2025
Muchtar E, Sanchorawala V, Hassan H, Hegenbart U, Schönland S, Lee HC, Qazilbash M, Kin A, Zonder J, Jacob E, Buadi F, Dispenzieri A, Dingli D, Arai S, Chin M, Chakraborty R, Lentzsch S, Magen H, Shkury E, Sarubbi C, Landau H, Gertz M. The Impact of Melphalan Conditioning and CD34+ Cell Dose and Schedule on Post-Transplant Outcomes in AL Amyloidosis. Am J Hematol. 2025 Apr 11. doi: 10.1002/ajh.27685. Epub ahead of print. PMID: 40214172. View Online
Prokaeva T, Jayaraman S, Klimtchuk E, Burke N, Spencer B, Nedelkov D, Chen H, Dasari S, McPhail ED, Pereira L, Payne MC, Wong S, Burks EJ, Sanchorawala V, Gursky O. An unusual phenotype of hereditary AApoAI amyloidosis caused by a novel Asp20Tyr substitution is linked to pH-dependent aggregation of apolipoprotein A-I. Biochim Biophys Acta Mol Basis Dis. 2025 Mar 29;1871(5):167820. doi: 10.1016/j.bbadis.2025.167820. Epub ahead of print. PMID: 40164396. View Online
Edwards CV, Ferri GM, Villegas-Galaviz J, Ghosh S, Singh Bawa P, Wang F, Klimtchuk E, Ajayi TB, Morgan GJ, Prokaeva T, Staron A, Ruberg FL, Sanchorawala V, Giadone RM, Murphy GJ. Abnormal global longitudinal strain and reduced serum inflammatory markers in cardiac AL amyloidosis patients without significant amyloid fibril deposition. Amyloid. 2025 Mar 25:1-14. doi: 10.1080/13506129.2025.2478397. Epub ahead of print. PMID: 40134188. View Online
Muchtar E, Dispenzieri A, Sanchorawala V, Hassan H, Mwangi R, Maurer M, Buadi F, Lee HC, Qazilbash M, Kin A, Zonder J, Arai S, Chin MM, Chakraborty R, Lentzsch S, Magen H, Shkury E, Sarubbi C, Landau H, Schönland S, Hegenbart U, Gertz M. A model for predicting day-100 stem cell transplant-related mortality in AL amyloidosis. Bone Marrow Transplant. 2025 Feb 24. PMID: 39994333; DOI: 10.1038/s41409-025-02535-z. View Online
Muchtar E, Grogan M, Aus dem Siepen F, Waddington-Cruz M, Misumi Y, Carroll AS, Clarke JO, Sanchorawala V, Milani P, Caccialanza R, Da Prat V, Pruthi R, Quintana LF, Bridoux F. Supportive care for systemic amyloidosis: International Society of Amyloidosis (ISA) expert panel guidelines. Amyloid. 2025 Feb 21:1-24. doi: 10.1080/13506129.2025.2463678. Epub ahead of print. PMID: 39985185.View Online
Dorbala S, Adigun R, Alexander KM, Brambatti M, Cuddy SAM, Dispenzieri A, Dunnmon P, Emdin M, Abou Ezzeddine OF, Falk RH, Fontana M, Grodin JL, Guthrie S, Jerosch-Herold M, Hofling AA, Hsu K, Lin G, Masri A, Maurer MS, Mittmann C, Prasad K, Quarta CC, Race JM, Rajendran JG, Ruberg FL, Sachdev V, Sanchorawala V, Signorovitch J, Sirac C, Soman P, Sorensen J, Sperry BW, Stephens AW, Stockbridge NL, Vest J, Wall JS, Wechalekar A, Welsh C, Lousada I. Development of Imaging Endpoints for Clinical Trials in AL and ATTR Amyloidosis: Proceedings of the Amyloidosis Forum. JACC Cardiovasc Imaging. 2025 Feb 20:S1936-878X(25)00023-3. doi: 10.1016/j.jcmg.2024.11.003. Epub ahead of print. PMID: 39985507. View Online
Chan N, Einstein AJ, Teruya S, Rodriguez C, Helmke S, Cuomo M, Santana D, Castillo M, Smiley D, Sabogal N, Lamour S, Winburn M, Fine D, de Freitas C, Kattan CG, Miller EJ, Maurer MS, Ruberg FL. The Impact of Active Ascertainment on Sex-Specific Differences in the Prevalence and Phenotype of Transthyretin Cardiac Amyloidosis: The Screening for Cardiac Amyloidosis With Nuclear Imaging in Minority Populations Study. Am J Cardiol. 2025 Feb 15;237:60-64. doi: 10.1016/j.amjcard.2024.11.019. Epub 2024 Nov 23. PMID: 39581521; PMCID: PMC11761373.View Online
Fontana M, Ioannou A, Cuddy S, Dorbala S, Masri A, Moon JC, Singh V, Clerc O, Hanna M, Ruberg F, Grogan M, Emdin M, Gillmore J. The Last Decade in Cardiac Amyloidosis: Advances in Understanding Pathophysiology, Diagnosis and Quantification, Prognosis, Treatment Strategies, and Monitoring Response. JACC Cardiovasc Imaging. 2025 Jan 3:S1936-878X(24)00458-3. doi: 10.1016/j.jcmg.2024.10.011. Epub ahead of print. PMID: 39797879. View Online
Cheng RK, Leedy D, Ruberg FL. Can Outpatient Worsening Heart Failure Serve as an Endpoint in Clinical Trials of Transthyretin Amyloidosis? J Am Coll Cardiol. 2025 Jan 16:S0735-1097(24)10558-X. doi: 10.1016/j.jacc.2024.10.126. Epub ahead of print. PMID: 39846939. View Online
Staron A, Sanchorawala V. Current landscape of clinical management for systemic light chain amyloidosis. Future Cardiol. 2025 Jan 31:1-3. doi: 10.1080/14796678.2025.2460392. Epub ahead of print. PMID: 39885778. View Online
2024
Morgan GJ, Nau AN, Wong S, Spencer BH, Shen Y, Hua A, Bullard MJ, Sanchorawala V, Prokaeva T. An updated AL-base reveals ranked enrichment of immunoglobulin light chain variable genes in AL amyloidosis. Amyloid. 2024 Dec 6:1-10. View Online
Morgan, G. J., Yung, Z., Spencer, B. H., Sanchorawala, V., & Prokaeva, T. (2024). Predicting Structural Consequences of Antibody Light Chain N-Glycosylation in AL Amyloidosis. Pharmaceuticals, 17(11), 1542. View Online
Urina-Jassir M, Teruya S, Blaner WS, Brun PJ, Prokaeva T, Tsai FJ, Kelly JW, Maurer MS, Ruberg FL. Differential Association of Transthyretin Stability with Variant and Wild-Type Transthyretin Amyloid Cardiomyopathy: The SCAN-MP Study. JACC Heart Fail. 2024 Jul 20:S2213-1779(24)00514-6. doi: 10.1016/j.jchf.2024.06.014. Epub ahead of print. PMID: 39115519. View Online
Klimtchuk ES, Prokaeva T, Spencer BH, Wong S, Ghosh S, Urdaneta A, Morgan G, Wales TE, Gursky O. Conformational differences in the light chain constant domain of immunoglobulin G and free light chain may influence proteolysis in AL amyloidosis. J Mol Biol. 2024 Oct 25:168837. doi: 10.1016/j.jmb.2024.168837. View Online
Morgan, G. J.; Yung, Z.; Spencer, B. H.; Sanchorawala, V.; Prokaeva, T. Structural Consequences of Antibody Light Chain N Glycosylation in AL Amyloidosis. Preprints 2024, 2024101407. https://doi.org/10.20944/preprints202410.1407.v1
Anand S, O’Neill-Dee M, Sanchorawala V, Verma A. Light Chain (AL) Amyloidosis for Nephrologists – Treatment Standard. Nephrology Dialysis Transplantation. 2024 Oct 7:gfae224. doi: 10.1093/ndt/gfae224. Epub ahead of print. PMID: 39375844. View Online
Sanchorawala V. Systemic Light Chain Amyloidosis. Reply. New England Journal of Medicine. 2024;391(12):1165-1166. View Online
Abdallah M, Arters F, Patel J, Edwards CV, Lerner A, Petrocca F, Dockerty M, Fuller B, Godfrey A, Staron A, Sanchorawala V, Sloan JM, Bertrand KA, Szalat RE. Febrile Neutropenia in Patients with Duffy-null Associated Neutrophil Counts and Multiple Myeloma or AL amyloidosis. Blood Advances. 2024; {prepublished online September 30, 2024} View Online
Sanchorawala V, Barroso B, Shelton AC, Goetz E, Mendelson L, Joshi T, Burke NJ, Sloan JM, Staron A. Defying the Odds: 30 Years in AL Amyloidosis Research, Recent Barriers to Clinical Trial Enrollment. Blood Advances. 2024 Sep 18 View Online
Staron A, Mendelson LM, Joshi T, Burke N, Sanchorawala V. Factors impeding organ recovery despite a deep haematological response in patients with systemic AL amyloidosis. British Journal of Haematology. 2024; {prepublished online September 14, 2024} View Online
Clerc OF, Vijayakumar S, Cuddy SAM, Bianchi G, Canseco Neri J, Taylor A, Benz DC, Datar Y, Kijewski MF, Yee AJ, Ruberg FL, Liao R, Falk RH, Sanchorawala V, Dorbala S. Functional Status and Quality of Life in Light-Chain Amyloidosis: Advanced Imaging, Longitudinal Changes, and Outcomes. JACC Heart Fail. 2024 Aug 20:S2213-1779(24)00562-6. doi: 10.1016/j.jchf.2024.07.007. Epub ahead of print. PMID: 39243245. View Online
Anand SK, Staron A, Mendelson LM, Joshi T, Burke N, Sanchorawala V, Verma A. Machine-learning based subgroups of AL amyloidosis and cumulative incidence of mortality and end stage kidney disease. Am J Hematol. 2024 Sep 11. doi: 10.1002/ajh.27472. Epub ahead of print. PMID: 39257247. View Online
Muchtar E, Gertz MA, Mwangi R, Hassan H, Dispenzieri A, Leung N, Buadi FK, Dingli D, Staron A, Sanchorawala V. Long-term outcomes of renal AL amyloidosis patients undergoing autologous stem cell transplantation: Validating the performance of the renal staging system. American Journal of Hematology. 2024; Aug 29. doi: 10.1002/ajh.27460. Epub ahead of print. PMID: 39207186. View Online
Marianna Fontana, M.D., Ph.D., John L. Berk, M.D., Julian D. Gillmore, M.D., Ph.D., Ronald M. Witteles, M.D., Martha Grogan, M.D., Brian Drachman, M.D., Thibaud Damy, M.D., Ph.D., Pablo Garcia-Pavia, M.D., Ph.D., Jorg Taubel, M.D., Scott D. Solomon, M.D., Farooq H. Sheikh, M.D., Nobuhiro Tahara, M.D., Ph.D., José González-Costello, M.D., Ph.D., Kenichi Tsujita, M.D., Ph.D., Caroline Morbach, M.D., Zoltán Pozsonyi, M.D., Mark C. Petrie, M.D., Diego Delgado, M.D., Peter Van der Meer, M.D., Ph.D., Andrew Jabbour, M.B., B.S., Ph.D., Antoine Bondue, M.D., Ph.D., Darae Kim, M.D., Ph.D., Olga Azevedo, M.D., Ph.D., Steen Hvitfeldt Poulsen, M.D., D.M.Sci., Ali Yilmaz, M.D., Ewa A. Jankowska, M.D., Ph.D., Vincent Algalarrondo, M.D., Ph.D., Andrew Slugg, B.Sc., Pushkal P. Garg, M.D., Katherine L. Boyle, M.D., Elena Yureneva, M.D., Nancy Silliman, Ph.D., Lilli Yang, Ph.D., Jihong Chen, Ph.D., Satish A. Eraly, M.D., Ph.D., John Vest, M.D., and Mathew S. Maurer, M.D. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. View Online
Chakraborty R, Zanwar S, Hegenbart U, Bhutani D, Gertz MA, Dispenzieri A, Kumar SK, D’Souza A, Patwari A, Cowan AJ, Chen G, Milani P, Palladini G, Sanchorawala V, Bodanapu G, Schönland S, Lentzsch S, Muchtar E. Prognostic impact of cytogenetic abnormalities by FISH in AL amyloidosis with daratumumab-based frontline therapy. Blood. 2024; {prepublished online August 28, 2024} View Online.
Verma K, Staron A, Zheng L, Cetin O, Doros G, Sanchorawala V.Systemic AL amyloidosis in patients younger than 40 years of age: Clinical presentation and outcomes. Clinical Lymphoma Myeloma and Leukemia. 2024; {prepublished online August 10, 2024} View Online
Muchtar E, Wisniowski B, Geyer S, Palladini G, Milani P, Merlini G, Schönland S, Veelken K, Hegenbart U, Leung N, Dispenzieri A, Kumar SK, Kastritis E, Dimopoulos MA, Liedtke M, Ulloa P, Sanchorawala V, Szalat R, Dooley K, Landau H, Petrlik E, Lentzsch S, Coltoff A, Bladé J, Cibeira MT, Cohen O, Foard D, Gillmore J, Lachmann H, Wechalekar A, Gertz MA. Graded Organ Response and Progression Criteria for Kidney Immunoglobulin Light Chain Amyloidosis. JAMA Oncol. 2024 Aug 1:e242629. doi: 10.1001/jamaoncol.2024.2629. Epub ahead of print. PMID: 39088206; PMCID: PMC11295065. View Online
Sanchorawala V. Systemic Light Chain Amyloidosis. New England Journal of Medicine. 2024; 390(24):2295-2307. View Online
Thel MC, Cochran JD, Teruya S, Hayashi O, Xie CR, Srinivasan AR, Chavkin NW, Arai Y, Sano S, Mirabal Santos A, De Los Santos J, Fine D, Sabogal N, Ullah I, Helmke S, Rodriguez C, Prokaeva T, Foster RH, Spencer BH, Izumiya Y, Maurer MS, Walsh K, Ruberg FL. Mosaic Loss of the Y Chromosome Is Enriched in Patients With Wild-Type Transthyretin Cardiac Amyloidosis and Associated With Increased Mortality. Circ Heart Fail. 2024 Jun 10:e011681. doi: 10.1161/CIRCHEARTFAILURE.124.011681. Epub ahead of print. PMID: 38853760. View Online
Adebayo AL, Rowan KE, Sanchorawala V, Boedicker MN, Boedicker DD. Evaluating the Amyloidosis Speakers Bureau: the influence of amyloidosis patients’ narratives on medical students’ knowledge, attitudes, and behavioral intent. MedEdPublish (2016). 2023 Jun 28;13:32. doi: 10.12688/mep.19631.1. PMID: 37753389; PMCID: PMC10518845. View Online
Ruberg FL, Maurer MS. Cardiac Amyloidosis Due to Transthyretin Protein: A Review. JAMA. 2024;331(9):778–791. doi:10.1001/jama.2024.0442. View Online
Anand SK, Sanchorawala V, Verma A. Systemic Amyloidosis and Kidney Transplantation: An Update. Semin Nephrol. 2024 Mar 14:151496. doi: 10.1016/j.semnephrol.2024.151496. Epub ahead of print. PMID: 38490903. View Online
2023
Stern LK, Grodin JL, Maurer MS, Ruberg FL, Patel AR, Khouri MG, Roth LR, Aras MA, Bhardwaj A, Bhattacharya P, Brailovsky Y, Drachman BM, Ebong IA, Fine NM, Gaggin H, Gopal D, Griffin J, Judge D, Kim P, Mitchell J, Mitter SS, Mohan RC, Ramos H, Reyentovich A, Sheikh FH, Sperry B, Carter S, Urey M, Vaishnav J, Vest AR, Kittleson MM, Patel JK. The Cardiac Amyloidosis Registry Study (CARS): Rationale, Design and Methodology. J Card Fail. 2023 Oct 30:S1071-9164(23)00379-2. doi: 10.1016/j.cardfail.2023.09.016. Epub ahead of print. PMID: 37907148. View Online
Wong S, West ME, Morgan GJ. Kinetic evidence for multiple aggregation pathways in antibody light chain variable domains. Protein Sci. 2023 Dec 15:e4871. doi: 10.1002/pro.4871. Epub ahead of print. PMID: 38100259. View Online
K. H. Vincent Lau, Tatiana Prokaeva, Luke Zheng, Gheorghe Doros, Michelle C. Kaku, Brian Spencer, John Berk & Vaishali Sanchorawala (2023) Neurofilament light chain kinetics as a biomarker for polyneuropathy in V122I hereditary transthyretin amyloidosis,Amyloid,DOI:10.1080/13506129.2023.2289340 View Online
Siddiqi OK, Gopal DM, Ruberg FL. Cardiac amyloidosis in 2023: A review of pathophysiology, diagnosis, and treatment. Heart Fail J India 2023;1:83-95. View Online
Adebayo AL, Rowan KE, Sanchorawala V, Boedicker MN, Boedicker DD. Evaluating the Amyloidosis Speakers Bureau: the influence of amyloidosis patients’ narratives on medical students’ knowledge, attitudes, and behavioral intent. MedEdPublish (2016). 2023 Jun 28;13:32. doi: 10.12688/mep.19631.1. PMID: 37753389; PMCID: PMC10518845.
Sarosiek S, Lee MH, Doros G, Edwards CV, Quillen K, Brauneis D, Shelton AC, Sanchorawala V, Sloan JM. Safety and Efficacy of Propylene Glycol-Free Melphalan in Patients with AL Amyloidosis Undergoing Autologous Stem Cell Transplantation: Results of a phase II study: Propylene Glycol-Free Melphalan Conditioning for Autologous Stem Cell Transplantation for AL Amyloidosis. Transplant Cell Ther. 2023 Aug 20:S2666-6367(23)01501-4. doi: 10.1016/j.jtct.2023.08.018. Epub ahead of print. PMID: 37607644. View Online
Gustine JN, Staron A, Mendelson L, Joshi T, Gopal DM, Ruberg FL, Siddiqi OK, Sanchorawala V. Predictors of treatment response and survival outcomes in patients with advanced cardiac AL amyloidosis. Blood Advances. 2023 Aug 15:bloodadvances.2023010324. doi: 10.1182/bloodadvances.2023010324. Epub ahead of print. PMID: 37581513. View Online
Sanchorawala V. A commentary on: Birtamimab plus standard of care in light chain amyloidosis: the phase 3 randomized placebo-controlled VITAL trial. PracticeUpdate website. Accessed August 08, 2023. View Online
Ghosh S, Villacorta-Martin C, Lindstrom-Vautrin J, Kenney D, Golden CS, Edwards CV, Sanchorawala V, Connors LH, Giadone RM, Murphy GJ. Mapping cellular response to destabilized transthyretin reveals cell- and amyloidogenic protein-specific signatures. Amyloid. 2023 Jul 13:1-15. doi: 10.1080/13506129.2023.2224494. Epub ahead of print. PMID: 37439769. View Online
Nader R, Angel-Korman A, Havasi A. Amyloidosis and the Kidney: An Update. Semin Nephrol. 2023 May 4;42(6):151343. View Online
Rizio AA, White MK, D’Souza A, Hsu K, Schmitt P, Quock TP, Signorovitch J, Lousada I, Sanchorawala V. Health-Related Quality of Life Instruments for Clinical Trials in AL Amyloidosis: Report from the Amyloidosis Forum HRQOL Working Group. Patient Relat Outcome Meas. 2023 May 18;14:153-169. View Online
Klimtchuk ES, Peterle D, Bullitt E, Connors LH, Engen JR, Gursky O. Role of complementarity-determining regions 1 and 3 in pathologic amyloid formation by human immunoglobulin κ1 light chains. Amyloid. 2023 May 22:1-15. View Online
Nau A, Shen Y, Sanchorawala V, Prokaeva T, Morgan GJ. Complete variable domain sequences of monoclonal antibody light chains identified from untargeted RNA sequencing data. Frontiers in Immunology 2023 Apr 18;14:1167235. View Online
Mendelson L, Prokaeva T, Lau KHV, Sanchorawala V, McCausland K, Spencer B, Dasari S, McPhail ED, Kaku MC. Hereditary gelsolin amyloidosis: a rare cause of cranial, peripheral and autonomic neuropathies linked to D187N and Y447H substitutions. Amyloid. 2023 May 4:1-7. View Online
Sanchorawala V. Eureka Moment-Innovation Comes of an Age in Light Chain Amyloidosis. JCO Oncol Pract. 2023 Feb 28:OP2300033. doi: 10.1200/OP.23.00033. Epub ahead of print. PMID: 36854072. View Online
Writing Committee; Kittleson MM, Ruberg FL, Ambardekar AV, Brannagan TH, Cheng RK, Clarke JO, Dember LM, Frantz JG, Hershberger RE, Maurer MS, Nativi-Nicolau J, Sanchorawala V, Sheikh FH. 2023 ACC Expert Consensus Decision Pathway on Comprehensive Multidisciplinary Care for the Patient With Cardiac Amyloidosis: A Report of the American College of Cardiology Solution Set Oversight Committee. Journal of American College of Cardiology. 2023; {prepublished online January 14, 2023}
Sanchorawala V, Wechalekar AD, Kim K, Schönland SO, Landau HJ, Kwok F, Suzuki K, Dispenzieri A, Merlini G, Comenzo RL, Cherepanov D, Hayden VC, Kumar A, Labotka R, Faller DV, Kastritis E. Quality of life and symptoms among patients with relapsed/refractory AL amyloidosis treated with ixazomib-dexamethasone versus physician’s choice. American Journal of Hematology. 2023; {prepublished online January 28, 2023}
Staron A, Mendelson LM, Joshi T, Ruberg FL, Sanchorawala V. Factors affecting the accuracy of amyloidosis identification and referral to a specialty centre. Amyloid. 2023; {prepublished online January 31, 2023}
2022
Minnema MC, Dispenzieri A, Merlini G, Comenzo RL, Kastritis E, Wechalekar AD, Grogan M, Witteles R, Ruberg FL, Maurer MS, Tran N, Qin X, Vasey SY, Weiss BM, Vermeulen J, Jaccard A. Outcomes by Cardiac Stage in Patients With Newly Diagnosed AL Amyloidosis: Phase 3 ANDROMEDA Trial. JACC CardioOncol. 2022 Nov 15;4(4):474-487. doi: 10.1016/j.jaccao.2022.08.011. PMID: 36444227; PMCID: PMC9700253. View Online
Gustine JN, Szalat RE, Staron A, Joshi T, Mendelson L, Sloan JM, Sanchorawala V. Light chain amyloidosis associated with Waldenström macroglobulinemia: treatment and survival outcomes. Haematologica. 2022 Dec 22. doi: 10.3324/haematol.2022.282264. Epub ahead of print. PMID: 36546447. View Online
Sykes A, Rebello S, Lousada I, Hsu K, Shore J, Sanchorawala V, Mendelson L, Sodowick A. Optimizing Patient-Provider Communication in Patients With Amyloidosis Using a Virtual Platform. Journal of Clinical Pathways. 2022; 8(7):36-41. View Online
Tsai FJ, Nelson LT, Kline GM, Jäger M, Berk JL, Sekijima Y, Powers ET, Kelly JW. Characterising diflunisal as a transthyretin kinetic stabilizer at relevant concentrations in human plasma using subunit exchange. Amyloid. 2022 Nov 29:1-5. doi: 10.1080/13506129.2022.2148094. Epub ahead of print. PMID: 36444793. View Online
Cuddy SA, Datar Y, Ovsak G, Saith S, Murphy SP, Bay CP, Haddad M, Lilleness B, Muralidhar V, Pipilas A, Vuong J, Guardino E, Maurer MS, Ruberg FL, Falk RH, Dorbala S. Optimal Echocardiographic Parameters to Improve the Diagnostic Yield of Tc-99m-Bone Avid Tracer Cardiac Scintigraphy for Transthyretin Cardiac Amyloidosis. Circ Cardiovasc Imaging. 2022 Nov;15(11):e014645. View Online
Prokaeva T, Klimtchuk ES, Feschenko P, Spencer B, Cui H, Burks EJ, Aslebagh R, Muneeruddin K, Shaffer SA, Varghese E, Berk JL, Connors LH. An additive destabilising effect of compound T60I and V122I substitutions in ATTRv amyloidosis. Amyloid. 2022 Oct 26:1-12. View Online
Muchtar E, Dispenzieri A, Wisniowski B, Palladini G, Milani P, Merlini G, Schönland S, Veelken K, Hegenbart U, Geyer SM, Kumar SK, Kastritis E, Dimopoulos MA, Liedtke M, Witteles R, Sanchorawala V, Szalat R, Landau H, Petrlik E, Lentzsch S, Coltoff A, Bladé J, Cibeira MT, Cohen O, Foard D, Wechalekar A, Gertz MA. Graded Cardiac Response Criteria for Patients With Systemic Light Chain Amyloidosis. J Clin Oncol. 2022 Oct 10:JCO2200643. View Online
Fraser CS, Spetz JKE, Qin X, Presser A, Choiniere J, Li C, Yu S, Blevins F, Hata AN, Miller JW, Bradshaw GA, Kalocsay M, Sanchorawala V, Sarosiek S, Sarosiek KA. Exploiting endogenous and therapy-induced apoptotic vulnerabilities in immunoglobulin light chain amyloidosis with BH3 mimetics. Nature Communications. 2022 13(1):5789. View Online
Alreshq R, Cozzolino M, Lilleness B, Pipilas A, Mendelson L, Joshi T, Muralidhar V, Guardino E, Berk JL, Siddiqi OK, Gopal DM, Sanchorawala V, Ruberg FL. Light Chain Testing Abnormalities Among Patients With Transthyretin Amyloid Cardiomyopathy Referred for Technetium-99m Pyrophosphate Imaging. Am J Cardiol. 2022 Aug 23:S0002-9149(22)00755-X. doi: 10.1016/j.amjcard.2022.06.064. Epub ahead of print. PMID: 36028388. View Online
Havasi A, Heybeli C, Leung N, Angel-Korman A, Sanchorawala V, Cohen O, Wechalekar A, Bridoux F, Jaffer I, Gutgarts V, Hassoun H, Levinson M, Rosenbaum C, Milani P, Palladini G, Merlini G, Hegenbart U, Schönland S, Veelken K, Pogrebinsky A, Doros G, Landau H. Outcomes of renal transplantation in patients with AL amyloidosis: an international collaboration through The International Kidney and Monoclonal Gammopathy Research Group. Blood Cancer J. 2022 Aug 18;12(8):119. doi: 10.1038/s41408-022-00714-5. PMID: 35982035. View Online
Hassan H, Verma K, Ferri G, Brauneis D, Quillen K, Sloan JM, Sanchorawala V, Edwards CV. Modified High-Dose versus High-Dose Melphalan Conditioning in Older Patients Undergoing Autologous Stem Cell Transplantation for Immunoglobulin Light Chain Amyloidosis. Transplantation and Cellular Therapy. 2022 Aug 12:. Epub ahead of print. PMID: 35970300.
Fogaren T, Shelton AC, Faller DV, Stull DM, Thuenemann S, Mendelson L. Understanding Amyloidosis: Unraveling the Complexities and Therapeutic Approaches for Oncology Nurses. Clin J Oncol Nurs. 2022 Jul 25;26(4):421-432. doi: 10.1188/22.CJON.421-432. PMID: 35939729. View Online
Adams D, Tournev IL, Taylor MS, Coelho T, Planté-Bordeneuve V, Berk JL, González-Duarte A, Gillmore JD, Low SC, Sekijima Y, Obici L, Chen C, Badri P, Arum SM, Vest J, Polydefkis M; HELIOS-A Collaborators. Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid. 2022 Jul 23:1-9. doi: 10.1080/13506129.2022.2091985. Epub ahead of print. PMID: 35875890. View Online
Wechalekar AD, Cibeira MT, Gibbs SD, Jaccard A, Kumar S, Merlini G, Palladini G, Sanchorawala V, Schönland S, Venner C, Boccadoro M, Kastritis E. Guidelines for non-transplant chemotherapy for treatment of systemic AL amyloidosis: EHA-ISA working group. Amyloid. 2022 Jul 15:1-15. View Online
Staron A, Zheng L, Doros G, Sanchorawala V. Differences in the cytogenetic underpinnings of AL amyloidosis among African Americans and Caucasian Americans. Blood Cancer Journal.2022; 12, 100. View Online
Ando Y, Adams D, Benson MD, Berk JL, Planté-Bordeneuve V, Coelho T, Conceição I, Ericzon BG, Obici L, Rapezz C, Sekijima Y, Ueda M, Palladini G, Merlini G. Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis. Amyloid. 2022 Jun 2:1-13. View Online
Prokaeva T, Joshi T, Klimtchuk ES, Gibson VM, Spencer B, Siddiqi O, Nedelkov D, Hu Y, Berk JL, Cuddy SAM, Dasari S, Chiu A, Choate LA, McPhail ED, Cui H, Chen H, Burks EJ, Sanchorawala V, Connors LH. A novel substitution of proline (P32L) destabilises β2-microglobulin inducing hereditary systemic amyloidosis. Amyloid. 2022 May 14:1-8. View Online
Abdallah M, Sanchorawala V. Update on the Contemporary Treatment of Light Chain Amyloidosis Including Stem Cell Transplantation. Am J Med. 2022 Apr;135 Suppl 1:S30-S37. doi: 10.1016/j.amjmed.2022.01.011. Epub 2022 Jan 23. PMID: 35081382. View Online
Staron A, Verma K, Sanchorawala V. Prevalence of plasma cell and lymphoproliferative disorders among blood relatives of patients with light chain amyloidosis. British Journal of Haematology. 2022 {Epub ahead of print May 2, 2022}. View Online
Mendelson L, Sanchorawala V, Connors L, Joshi T, Doros G, Pogrebinsky A, Havasi A. Correlation Between 24-Hour Urine Protein and Random Urine Protein-Creatinine Ratio in Amyloid Light-Chain Amyloidosis. Kidney Med. 2022 Feb 3;4(4):100427. View Online
Sanchorawala V, Palladini G, Minnema MC, Jaccard A, Lee HC, Gibbs S, Mollee P, Venner C, Lu J, Schönland S, Gatt M, Suzuki K, Kim K, Cibeira MT, Beksac M, Libby E, Valent J, Hungria V, Wong SW, Rosenzweig M, Bumma N, Chauveau D, Gries KS, Fastenau J, Tran NP, Qin X, Vasey SY, Weiss BM, Vermeulen J, Ho KF, Merlini G, Comenzo RL, Kastritis E, Wechalekar AD. Health-related quality of life in patients with AL amyloidosis treated with bortezomib, cyclophosphamide, and dexamethasone ± daratumumab: results from the ANDROMEDA study. Am J Hematol. 2022 View Online
Kaku M, Bhadola S, Berk JL, Connors LH, Sanchorawala V, Lau KHV. Neurological manifestations of hereditary transthyretin amyloidosis: a focus on diagnostic delays. 2022; Amyloid {prepublished online March 7, 2022} View Online
Nguyen VP, Mendelson L, Kaku M, Berk JL, Sanchorawala V. Systemic Amyloidosis Involving the Fallopian Tube. Archives of Clinical and Medical Case Reports 6 (2022): 110-115. View Online
Abdallah M, Sanchorawala V. Update on the Contemporary Treatment of Light Chain Amyloidosis Including Stem Cell Transplantation. Am J Med. 2022 Jan 23:S0002-9343(22)00031-6. View Online
Siddiqi OK, Mints YY, Berk JL, Connors L, Doros G, Gopal DM, Kataria S, Lohrmann G, Pipilas AR, Ruberg FL. Diflunisal treatment is associated with improved survival for patients with early stage wild-type transthyretin (ATTR) amyloid cardiomyopathy: the Boston University Amyloidosis Center experience. Amyloid. 2022 Jan 27:1-8. doi: 10.1080/13506129.2021.2000388. Epub ahead of print. PMID: 35083944. View Online
Hassan H, Sanchorawala V. Beyond Survival in AL amyloidosis: Identifying and Satisfying Patients’ Needs. Hemato 2022, 3, 38–46. View Online
2021
Rapezzi C, Elliott P, Damy T, Nativi-Nicolau J, Berk JL, Velazquez EJ, Boman K, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Efficacy of Tafamidis in Patients With Hereditary and Wild-Type Transthyretin Amyloid Cardiomyopathy: Further Analyses From ATTR-ACT. JACC Heart Fail. 2021 Feb;9(2):115-123. doi: 10.1016/j.jchf.2020.09.011. Epub 2020 Dec 9. PMID: 33309574. View Online
Adams D, Polydefkis M, González-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada López IA, Dispenzieri A, Quan D, Conceição IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Planté-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda M, Aldinc E, Wang JJ, White MT, Vest J, Berber E, Sweetser MT, Coelho T; patisiran Global OLE study group. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Lancet Neurol. 2021 Jan;20(1):49-59. doi: 10.1016/S1474-4422(20)30368-9. Epub 2020 Nov 16. Erratum in: Lancet Neurol. 2021 Feb;20(2):e2. PMID: 33212063. View Online
Brannagan TH 3rd, Auer-Grumbach M, Berk JL, Briani C, Bril V, Coelho T, Damy T, Dispenzieri A, Drachman BM, Fine N, Gaggin HK, Gertz M, Gillmore JD, Gonzalez E, Hanna M, Hurwitz DR, Khella SL, Maurer MS, Nativi-Nicolau J, Olugemo K, Quintana LF, Rosen AM, Schmidt HH, Shehata J, Waddington-Cruz M, Whelan C, Ruberg FL. ATTR amyloidosis during the COVID-19 pandemic: insights from a global medical roundtable. Orphanet J Rare Dis. 2021 May 6;16(1):204. doi: 10.1186/s13023-021-01834-0. PMID: 33957949; PMCID: PMC8100737. View Online
Benson MD, Berk JL, Dispenzieri A, Damy T, Gillmore JD, Hazenberg BP, Lavatelli F, Picken MM, Röcken C, Schönland S, Ueda M, Westermark P. Tissue biopsy for the diagnosis of amyloidosis: experience from some centres. Amyloid. 2021 Nov 12:1-6. doi: 10.1080/13506129.2021.1994386. Epub ahead of print. PMID: 34766859. View Online
Coelho T, Ando Y, Benson MD, Berk JL, Waddington-Cruz M, Dyck PJ, Gillmore JD, Khella SL, Litchy WJ, Obici L, Monteiro C, Tai LJ, Viney NJ, Buchele G, Brambatti M, Jung SW, St L O’Dea L, Tsimikas S, Schneider E, Geary RS, Monia BP, Gertz M. Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. Neurol Ther. 2021 Jun;10(1):375-389. doi: 10.1007/s40120-021-00235-6. Epub 2021 Feb 26. PMID: 33638113; PMCID: PMC8140170. View Online
Cuddy SAM, Jerosch-Herold M, Falk RH, Kijewski MF, Singh V, Ruberg FL, Sanchorawala V, Landau H, Maurer MS, Yee AJ, Bianchi G, Di Carli MF, Liao R, Kwong RY, Dorbala S. Myocardial Composition in Light-Chain Cardiac Amyloidosis More Than 1 Year After Successful Therapy. JACC Cardiovasc Imaging. 2021 Dec 10:S1936-878X(21)00832-9. doi: 10.1016/j.jcmg.2021.09.032. Epub ahead of print. PMID: 34922860. View Online
Furtado VF, Brauneis D, Weinberg J, Elhassan N, Sloan JM, Sanchorawala V. Early serum free light chain response after high-dose melphalan and stem cell transplantation predicts hematologic response in AL amyloidosis. Bone Marrow Transplantation. 2021 Nov 29. View Online
Morgan GJ. Transient disorder along pathways to amyloid. Biophysical Chemistry. 2021 Oct 30; 281:106711. View Online
Sanchorawala V, Boccadoro M, Gertz M, Hegenbart U, Kastritis E, Landau H, Mollee P, Wechalekar A, Palladini G. Guidelines for high dose chemotherapy and stem cell transplantation for systemic AL amyloidosis: EHA-ISA working group guidelines. Amyloid. 2021 Nov 16:1-7. View Online
Benson MD, Berk JL, Dispenzieri A, Damy T, Gillmore JD, Hazenberg BP, Lavatelli F, Picken MM, Röcken C, Schönland S, Ueda M, Westermark P. Tissue biopsy for the diagnosis of amyloidosis: experience from some centres. Amyloid. 2021 Nov 12:1-6. View Online
Peterle D, Klimtchuk ES, Wales TE, Georgescauld F, Connors LH, Engen JR, Gursky O. A Conservative Point Mutation in a Dynamic Antigen-binding Loop of Human Immunoglobulin λ6 Light Chain Promotes Pathologic Amyloid Formation. J Mol Biol. 2021 Oct 19;433(24):167310. View Online
Szalat RE, Gustine J, Sloan JM, Edwards CV, Sanchorawala V. Predictive factors of outcomes in patients with AL amyloidosis treated with Daratumumab. Am J Hematol. 2021 Nov 5. doi: 10.1002/ajh.26399. Epub ahead of print. View Online
Edwards CV, Rao N, Bhutani D, Mapara MY, Radhakrishnan J, Shames S, Maurer MS, Leng S, Solomon A, Lentzsch S, Eisenberger A. Phase 1a/b Study of Monoclonal Antibody CAEL-101 (11-1F4) in Patients with AL Amyloidosis. Blood. 2021 Sep 14:blood.2020009039. doi: 10.1182/blood.2020009039. Epub ahead of print. PMID: 34521113. View Online
Morgan GJ; Buxbaum JN; Kelly JW. Light Chain Stabilization: A Therapeutic Approach to Ameliorate AL Amyloidosis. Hemato 2021, 2, 645-659. View Online
Staron A, Zheng L, Doros G, Connors LH, Mendelson LM, Joshi T, Sanchorawala V. Marked progress in AL amyloidosis survival: a 40-year longitudinal natural history study. Blood Cancer J. 11, 139 (2021). View Online
Kastritis E, Palladini G, Minnema MC, Wechalekar AD, Jaccard A, Lee HC, Sanchorawala V, Gibbs S, Mollee P, Venner CP, Lu J, Schönland S, Gatt ME, Suzuki K, Kim K, Cibeira MT, Beksac M, Libby E, Valent J, Hungria V, Wong SW, Rosenzweig M, Bumma N, Huart A, Dimopoulos MA, Bhutani D, Waxman AJ, Goodman SA, Zonder JA, Lam S, Song K, Hansen T, Manier S, Roeloffzen W, Jamroziak K, Kwok F, Shimazaki C, Kim JS, Crusoe E, Ahmadi T, Tran N, Qin X, Vasey SY, Tromp B, Schecter JM, Weiss BM, Zhuang SH, Vermeulen J, Merlini G, Comenzo RL; ANDROMEDA Trial Investigators. Daratumumab-Based Treatment for Immunoglobulin Light-Chain Amyloidosis. New England Journal of Medicine. 2021 Jul 1;385(1):46-58. View Online
Dispenzieri A, Kastritis E, Wechalekar AD, Schönland SO, Kim K, Sanchorawala V, Landau HJ, Kwok F, Suzuki K, Comenzo RL, Berg D, Liu G, Kumar A, Faller DV, Merlini G. A randomized phase 3 study of ixazomib-dexamethasone versus physician’s choice in relapsed or refractory AL amyloidosis. Leukemia. 2021 Jun 24. View Online
Sarosiek S, Varga C, Jacob A, Fulciniti MT, Munshi N, Sanchorawala V. Detection of minimal residual disease by next generation sequencing in AL amyloidosis. Blood Cancer Journal. 2021;11, 117. View Online
Patel RK, Fontana M, Ruberg FL. Cardiac Amyloidosis: Multimodal Imaging of Disease Activity and Response to Treatment. Circulation Cardiovascular Imaging. 2021 Jun;14(6):e009025. View Online
Nader R, Zhen A, Angel-Korman A, Pavlovich SS, Pogrebinsky A, Doros G, Menn-Josephy H, Stern L, Sanchorawala V, Havasi A. Predictors and outcomes of acute kidney injury during autologous stem cell transplantation in AL amyloidosis. Nephrology Dialysis Transplantation. Nephrol Dial Transplant. 2021 May 27:gfab189. View Online
Yan NL, Santos-Martins D, Nair R, Chu A, Wilson IA, Johnson KA, Forli S, Morgan GJ, Petrassi HM, Kelly JW. Discovery of Potent Coumarin-Based Kinetic Stabilizers of Amyloidogenic Immunoglobulin Light Chains Using Structure-Based Design. J Med Chem. 2021 May 13;64(9):6273-6299. View Online
Brannagan TH 3rd, Auer-Grumbach M, Berk JL, Briani C, Bril V, Coelho T, Damy T, Dispenzieri A, Drachman BM, Fine N, Gaggin HK, Gertz M, Gillmore JD, Gonzalez E, Hanna M, Hurwitz DR, Khella SL, Maurer MS, Nativi-Nicolau J, Olugemo K, Quintana LF, Rosen AM, Schmidt HH, Shehata J, Waddington-Cruz M, Whelan C, Ruberg FL. ATTR amyloidosis during the COVID-19 pandemic: insights from a global medical roundtable. Orphanet Journal of Rare Diseases. 2021 May 6;16(1):204. View Online.
Ruberg FL, Alreshq R. Clinical approach to genetic testing in amyloid cardiomyopathy: from mechanism to effective therapies. Current Opinion Cardiology. 2021 May 1;36(3):309-317. View Online.
Hughes DM, DeMari S, Hassan H, Sanchorawala V, Sloan JM. Safety, Tolerability, and Efficacy of Selinexor in a Patient With Relapsed Light Chain (AL) Amyloidosis. Clinical Lymphoma, Myeloma, and Leukemia. 2021; S2152-2650 (21) 00001-X. {prepublished online on January 6, 2021} View Online
Benbrahim M, Norman K, Sanchorawala V, Siddiqi OK, Hughes D. A review of novel agents and clinical considerations in patients with ATTR cardiac amyloidosis. Journal of Cardiovascular Pharmacology. 2021; {prepublished online on February 19 2021}.View Online
Acevedo J, Doros G, Szalat R, Sanchorawala V. Clinical Characteristics, Treatment Regimens and Survival in Elderly Patients with AL amyloidosis. Clinical Lymphoma, Myeloma and Leukemia. 2021; S2152-2650 (21)00040-9. View Online
Palladini G, Schönland SO, Sanchorawala V, Kumar S, Wechalekar A, Hegenbart U, Milani P, Ando Y, Westermark P, Dispenzieri A, Merlini G. Clarification on the definition of complete haematologic response in light-chain (AL) amyloidosis. Amyloid. 2021 Jan 7:1-2. View Online
Premkumar VJ, Lentzsch S, Pan S, Bhutani D, Richter J, Jagannath S, Liedtke M, Jaccard A, Wechalekar AD, Comenzo R, Sanchorawala V, Royer B, Rosenzweig M, Valent J, Schönland S, Fonseca R, Wong S, Kapoor P. Venetoclax induces deep hematologic remissions in t(11;14) relapsed/refractory AL amyloidosis. Blood Cancer Journal. 2021; 11(1):10. View Online
Lovley A, Raymond K, Guthrie S, Pollock M, Sanchorawala V, White MK. Patient-reported burden of hereditary transthyretin amyloidosis on functioning and well-being. Journal of Patient Reported Outcomes. 2021; 5, 3. View Online
2020
Lohrmann G, Patel MA, Brauneis D, Sanchorawala V, Sarosiek S, Vellanki N, Siddiqi OK, Ruberg FL, Gopal DM. Left Atrial Mechanics Associates With Paroxysmal Atrial Fibrillation in Light-Chain amyloidosis Following Stem Cell Transplantation. JACC: Cardiooncology. 2020; 2(5): 721-731. View Online
Angel-Korman A, Havasi A. Kidney Transplantation in Systemic Amyloidosis. Transplantation. 2020; 104(10):2035-2047. View Online
Staron A., Connors LH, Zheng L, Doros G, Sanchorawala V. Race/ethnicity in systemic AL amyloidosis: perspectives on disease and outcome disparities. Blood Cancer Journal. 10, 118 (2020). View Online
Hoffman JE, Dempsey NG, Sanchorawala V. Systemic Amyloidosis Caused by Monoclonal Immunoglobulins: Soft Tissue and Vascular Involvement. Hematology Oncology Clinics of North America. 2020; 34(6):1099-1113. View Online
Hughes DM, Staron A, Sanchorawala V. A pharmacist’s review of the treatment of systemic light chain amyloidosis. Journal of Oncology Pharmacy Practices. 2020. View Online.
Inaugural Amyloidosis Forum Panelists, Lousada I. The Amyloidosis Forum: a public private partnership to advance drug development in AL amyloidosis. Orphanet J Rare Dis. 2020 Sep 29; 15(1):268. View Online
Sarosiek S, Zheng L, Sloan JM, Quillen K, Brauneis D, Sanchorawala V. Comparing measures of hematologic response after high-dose melphalan and stem cell transplantation in AL amyloidosis. Blood Cancer Journal. 2020; 10:88. View Online
Szalat R, Sarosiek S, Havasi A, Brauneis D, Sloan JM, Sanchorawala V. Organ responses after highdose melphalan and stem cell transplantation in AL amyloidosis. Leukemia. 2020 Jul 31. View Online
Yan NL, Santos-Martins D, Rennella E, Sanchez B, Chen J, Kay LE, Wilson IA, Morgan GJ, Forli S, Kelly JW. Structural basis for the stabilization of amyloidogenic immunoglobulin light chains by hydantoins. Bioorg Med Chem Lett. 2020 Aug 15;30(16):127356. View Online
Giadone R, Liberti D, Matte T, Rosarda J, Torres-Arancivia C, Ghosh S, Diedrich JK, Pankow S, Skvir N, Jean JC, Yates III J.R, Wilson AA, Connors L H., Kotton DN, Wiseman RL, Murphy GJ. Expression of amyloidgenic transthyretin drives hepatic proteostasis remodeling in an induced pluripotent stem cell model of systemic amyloid disease. Stem Cell Reports. 2020 July 30. View Online Also view video abstract for Giadone et al. 2020
Berk JL, Barroso F, Merlini G, Conceição I, Hughes SG, Kwoh J, Jung SW, Guthrie S, Pollock M, Benson MD, Gertz M, NEURO-TTR open-label extension investigators, Brannagan TH, et al. Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial. Eur J Neurol. 2020 Apr 28. doi: 10.1111/ene.14285. View Online
Takumi K, Staziaki PV, Hito R, Nadgir RN, Berk JL, Andreu-Arasa VC, Chavez W, Sakai O.Takumi K, et al. Among authors: Berk JL. Amyloidosis in the head and neck: CT findings with clinicopathological correlation. Eur J Radiol. 2020 Apr 30;128:109034. View Online
Kastritis E, Wechalekar A, Schönland S, Sanchorawala V, Merlini G, Palladini G, Minnema M, Roussel M, Jaccard A, Hegenbart U, Kumar S, Cibeira MT, Blade J, Dimopoulos MA. Challenges in the management of patients with systemic light chain (AL) amyloidosis during the COVID-19 pandemic. Br J Haematol. 2020 Jun 1. doi: 10.1111/bjh.16898. View Online
Kittleson MM, Maurer MS, Ambardekar AV, Bullock-Palmer RP, Chang PP, Eisen HJ, Nair AP, Nativi-Nicolau J, Ruberg FL. Cardiac amyloidosis: Evolving diagnosis and management: A scientific statement from the American Heart Association. Circulation. 2020 Jun 1. View Online
Cuddy SAM, Bravo PE, Falk RH, El-Sady S, Kijewski MF, Park MA, Ruberg FL, Sanchorawala V, Landau H, Yee AJ, Bianchi G, Di Carli MF, Cheng SC, Jerosch-Herold M, Kwong RY, Liao R, Dorbala S. Improved quantification of cardiac amyloid burden in systemic light chain amyloidosis: Redefining early disease? JACC Cardiovasc Imaging. 2020 May 8. S1936-878X(20)30251-5. doi: 10.1016/j.jcmg.2020.02.025. View Online
Sanchorawala V, Sarosiek S, Schulman A, Mistark M, Migre ME, Cruz R, Sloan JM, Brauneis D, Shelton AC. Safety, tolerability, and response rates of daratumumab in relapsed AL amyloidosis: results of a phase 2 study. Blood. 2020 Apr 30;135(18):1541-1547. View Online
Brannagan TH III, Wang AK, Coelho T, Waddington Cruz M, Polydefkis MJ, Dyck PJ, Plante-Bordeneuce V, Berk JL, Barroso F, Merlini G, Conceicao I, Hughes SG, Kwoh J, Jung SW, Guthrie S, Pollock M, Benson MD, Gertz M, NEURO-TTR open-label extension investigators. Early data on long-term efficacy and safety of Inotersen in patients with hereditary transthyretin amyloidosis: A 2-year update from the open label extension of NEURO-TTR Trial. Eur J Neurol. 2020 Apr 28. View Online
Coelho T, Yarlas A, Waddington-Cruz M, White MK, Sikora Kessler A, Lovley A, Pollock M, Guthrie S, Ackermann EJ, Hughes SG, Karam C, Khella S, Gertz M, Merlini G, Obici L, Schmidt HH, Polydefkis M, Dyck PJB, Brannagan III TH, Conceicao I, Benson MD, Berk JL. Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis. J Neurol. 2020 Apr;267(4): 1070-1079. View Online
Sanchorawala V. High-dose melphalan and autologous peripheral blood stem cell transplantation in AL amyloidosis. Acta Haematol. 2020 Apr 3:1-7. doi: 10.1159/000506498. View Online
Palladini G, Kastritis E, Maurer MS, Zonder JA, Minnema MC, Wechalekar AD, Jaccard A, Lee HC, Bumma N, Kaufman JL, Medvedova E, Kovacsovics TJ, Rosenzweig MA, Sanchorawala V, Qin X, Vasey SY, Weiss B, Vermeulen J, Merlini G, Comenzo RL. Daratumumab plus cyborD for patients with newly diagnosed AL amyloidosis: Safety run-in results of ANDROMEDA. Blood. 2020 Apr 3. pii: blood.2019004460. doi: 10.1182. View Online
Angel-Korman A, Stern L, Angel Y, Sarosiek S, Menn-Josephy H, Francis J, Ghai S, Sloan JM, Sanchorawala V, Havasi A. The role of kidney transplantation in monoclonal Ig deposition disease. Kidney Int Rep. 2020 Mar 9;5(4):485-493. View Online
Gonzalez-Duarte A, Berk JL, Quan D, Mauermann ML, Schmidt HH, Polydefkis M, Waddington-Cruz M, Ueda M, Conceicao IM, Kristen AV, Coelho T, Cauquil CA, Tard C, Merkel M, Aldinc E, Chen J, Sweetser MT, Wang JJ, Adams D. Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis. J Neurol. 2020 Mar;267(3):703-712. View Online
Ruberg, FL , Siddiqi OK. Hepatocyte growth factor and cardiac amyloidosis. JACC: CardioOncology. 2020 Mar 1. View Online
Staron A, Burks EJ, Lee JC, Sarosiek S, Sloan JM, Sanchorawala V. Assessment of minimal residual disease using multiparametric flow cytometry in patients with AL amyloidosis. Blood Adv. 2020 Mar 10;4(5):880-884. View Online
Obici L, Berk JL, González-Duarte A, Coelho T, Gillmore J, Schmidt H, Schilling M, Yamashita T, Labeyrie C, Brannagan III T, Ajroud-Driss S, Gorevic P, Kristen A, Franklin J, Chen J, Sweetser M, Wang J, Adams D. Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated Amyloidosis. J Neurol. 2020 Mar; 267(3):703-712. View Online
Lentzsch S, Lagos GG, Comenzo RL, Zonder JA, Osman K, Pan S, Bhutani D, Pregja S, Sanchorawala V, Landau H. Bendamustine with dexamethasone in relapsed/refractory systemic light-chain amyloidosis: Results of a phase II study. J Clin Oncol. 2020 May 1;38(13):1455-1462. View Online
Sanchorawala V, Sarosiek S, Schulman A, Mistark M, Migre ME, Cruz R, Sloan JM, Brauneis D, Shelton AC. Safety, tolerability, and response rates of daratumumab in relapsed AL amyloidosis results of a phase II study. Blood, 2020 published Apr 30;135(18):1541-15470 Online Jan 24. View Online
2019
Lohrmann G, Pipilas A, Mussineli R, Gopal DM, Berk JL, Connors LH, Vellanki N, Hellawell J, Siddiqi OK, Fox J, Maurer MS, Ruberg FL. Stabilization of cardiac function with diflunisal in transthyretin (ATTR) cardiac amyloidosis. J Card Failure. 2019.
Staron A, Kataria Y, Murray D, Sloan J.M, Sanchorawala V. Systemic AL amyloidosis with an undetectable plasma cell dyscrasia: a zebra without stripes. American Journal of Hematology. 17 Nov 2019.
Sarosiek S, Sanchorawala V. Treatment Options for Relapsed/Refractory Systemic Light-Chain (AL) Amyloidosis: Current Perspectives. J Blood Med. 2019 Oct 23; 10:373-380. View in PubMed
Elliot J, Zhou L, Chunyu C and Kaku M. A 63-Year-Old Man with Nausea, Vomiting, Orthostatic Dizziness, and Distal Limb Paresthesia. In: Zhou L, Burns D, Chunyu C. (A Case-Based Guide to Neuromuscular Pathology.) Springer, 2019. https://www.amazon.com/Case-Based-Guide-Neuromuscular-Pathology/dp/3030256812
Kaku M, Berk JL. Neuropathy Associated with Systemic Amyloidosis. Semin Neurol. 2019 Oct;39(5):578-588. doi: 10.1055/s-0039-1688994. Epub 2019 Oct 22. View in PubMed
A Angel-Korman, A Jaberi, V Sanchorawala, A Havasi. The utility of repeat kidney biopsy in systemic immunoglobulin light chain amyloidosis. Amyloid Journal 2019. 09 Oct 2019.
Gopal D, Ruberg FL, Siddiqi O Impact of Genetic Testing in Transthyretin (ATTR) Cardiac Amyloidosis. 2019 Sep 13. doi: 10.1007/s11897-019-00436-z View in PubMed
Lilleness B, Doros G, Ruberg FL, Sanchorawala V. Establishment of brain natriuretic peptide-based criteria for evaluating cardiac response to treatment in light chain (AL) amyloidosis. Br J Haematol. 2019 Sep 13. doi: 10.1111/bjh.16198. View in PubMed
Dorbala S, Ando Y, Bokhari S, Dispenzieri A, Falk RH, Ferrari VA, Fontana M, Gheysens O, Gillmore JD, Glaudemans AWJM, Hanna MA, Hazenberg BPC, Kristen AV, Kwong RY, Maurer MS, Merlini G, Miller EJ, Moon JC, Murthy VL, Quarta CC, Rapezzi C, Ruberg FL, Shah SJ, Slart RHJA, Verberne HJ, Bourque JM. SNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI Expert consensus recommendations for Multimodality Imaging in Cardiac Amyloidosis: Part 1 of 2 – Evidence base and Standardized methods of imaging. 2019 Aug 6. doi: 10.1016/J Card Fail 2019.08.001. View in PubMed
Dorbala S, Ando Y, Bokhari S, Dispenzieri A, Falk RH, Ferrari VA, Fontana M, Gheysens O, Gillmore JD, Glaudemans AWJM, Hanna MA, Hazenberg BPC, Kristen AV, Kwong RY, Maurer MS, Merlini G, Miller EJ, Moon JC, Murthy VL, Quarta CC, Rapezzi C, Ruberg FL, Shah SJ, Slart RHJA, Verberne HJ, Bourque JM.
ASNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI expert consensus recommendations for multimodality imaging in cardiac amyloidosis: Part 2 of 2-Diagnostic criteria and appropriate utilization. 2019 Aug 6. doi: 10.1016/J Card Fail 2019. 08.002. View in PubMed
Law ST, Reza Jafarzadeh S, Govender P, Sun X, Sanchorawala V, Kissin EY. Comparison of Ultrasound Features of major salivary glands in Sarcoidosis, Amyloidosis, and Sjögren’s Syndrome. Arthritis Care Res, (Hoboken). 2019 Jul 15. View in PubMed
Staron A, Connors LH, Ruberg FL, Mendelson LM, Sanchorawala, V. A new era of amyloidosis: the trends at a major US referral. Amyloid, The Journal of Protein Folding Disorders, July 2019. View online
Pudusseri A, Sanchorawala V, Sloan JM, Bever KM, Doros G, Kataria S, Sarosiek S. Prevalence and prognostic value of D-dimer elevation in patients with AL amyloidosis. Am J Hematol, 2019, Jul 11. doi: 10. 1002/ajh.25576 [Epub ahead of print]. View online
Ruberg FL, Grogan M, Hanna M, Kelly JW, Maurer MS. Transthyretin amyloid cardiomyopathy: JACC state-of-the-art review. J Am Coll Cardiol, 2019, June 11; 73 (22): 2872-2891. View in PubMed.
McCausland KL, Quock TP, Rizio AA, Bayliss MS, White MK, Guthrie SD, Sanchorawala V. Cardiac biomarkers and health-related quality of life in patients with light chain (AL) amyloidosis. British Journal of Haematology, 2019, 185, 940-1007. View online
Sanchorawala V, Doros G, Shelton, AC. Long term outcome of patients treated on clinical trials of immunomodulatory agents for the treatment of AL amyloidosis: A pooled analysis. Am J Hematol. 2019 April 24. doi: 10. 1022/ajh.25497. View in PubMed
Morgan GJ, Yan NL , Mortenson DE, Rennella E, Blundon JM, Gwin RM, Lin CY, Stanfield RL, Brown SJ, Rosen H, Spicer TP, Fernandez-Vega V, Merlini G, Kay LE, Wilson IA, Kelly JW. Stabilization of amyloidogenic immunoglobulin light chains by small molecules. Proceedings of National Academy of Sciences of the United States of America. 2019 Apr 10. doi: 10.1073/pnas.1817567116. View in PubMed
Ehman EC, El-Sady MS, Kijewski MF, Khor YM, Jacob S, Ruberg FL, Sanchorawala V, Landau H, Yee AJ, Bianchi G, Di Carli MF, Falk RH, Hyun H, Dorbala S. Early Detection of Multiorgan Light Chain (AL) Amyloidosis by Whole Body 18F-florbetapir PET/CT. Journal of Nuclear Medicine. 6 April 2019 dio:10.2967/jnumed.118.221770. View in PubMed
Gupta VK, Brauneis D, Shelton AC, Quillen K, Sarosiek S, Sloan JM, Sanchorawala V. Induction Therapy with Bortezomib and Dexamethasone and Conditioning with High-Dose Melphalan and Bortezomib Followed by Autologous Stem Cell Transplantation for AL Amyloidosis: Long Term Follow-Up Analysis. Biology of Blood and Marrow Transplantation. 11 Jan 2019. doi: 10.1016/j.bbmt.2019.01.007. View in PubMed
Lilleness B, Ruberg FL, Mussinelli R, Gheorghe D, and Sanchorawala V. Development and validation of a survival staging system incorporating BNP in patients with light chain amyloidosis. American Society of Hematology. 17 Jan 2019, 133 (2019). doi: https://doi.org/10.1182/blood-2018-06-858951. View in PubMed
Phull P, Sanchorawala V, Brauneis D, Sloan JM, Siddiqi OK, Quillen K, Sarosiek S. High-dose melphalan and autologous peripheral blood stem cell transplantation in patients with AL amyloidosis and cardiac defibrillators. Bone Marrow Transplantation. 21 Jan 2019. doi: 10.1038/s41409-019-0440-5. View in PubMed
Sanchorawala V. Delay treatment of AL amyloidosis at relapse until symptomatic: devil is in the details.. Blood Advances. 22 Jan 2019. doi: 10.1182/bloodadvances.2018021261. View in PubMed
2018
Angel-Korman A, Stern L, Sarosiek S, Sloan MJ, Doros G, Sanchorawala V, Havasi A. Long-term outcome of kidney transplantation in AL amyloidosis. Kidney International. 21 Dec 2018. doi: 10.1016/j.kint.2018.09.021. View in PubMed
McCausland KL, Quock TP, Rizio AA, Bayliss MS, White MK, Guthrie SD, Sanchorawala V. Cardiac biomarkers and health‐related quality of life in patients with light chain (AL) amyloidosis. British Journal of Haematology. 22 Nov 2018. doi:10.1111/bjh.15693. View in PubMed
Merlini G, Dispenzieri A, Sanchorawala V, Schönland SO, Palladini G, Hawkins PN, Gertz MA. Systemic immunoglobulin light chain amyloidosis. Nature Reviews Disease Primers. 4 Oct 2018, 38 (2018). doi: 10.1038/s41572-018-0034-3. View in Nature Reviews
Maurer MS, Ruberg FL. Early Diagnosis of Cardiac Amyloidosis by Carpal Tunnel Surgery: Is it All in the Wrist? Journal of the American College of Cardiology. Oct 2018, 72 (17) 2051-2053. doi:10.1016/j.jacc.2018.09.003. View in PDF
Waddington-Cruz M, Ackermann EJ, Polydefkis M, Heitner SB, Dyck PJ, Barroso FA, Wang AK, Berk JL, Dyck PJB, Monia BP, Hughes SG, Tai L, Kwoh TJ, Jung SW, Coelho T, Benson MD, Gertz MA. Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial. Amyloid. 31 Aug 2018: 1-9. doi: 10.1080/13506129.2018.1503593. View in PubMed
Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. New England Journal of Medicine 379(1), 22-31 2018 Jul 5. doi:10.1056/NEJMoa1716793. View in PubMed
Adams D, Gonzalez-Duarte A, O’Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. New England Journal of Medicine 379(1), 11-21 2018 Jul 5. doi:10.1056/NEJMoa1716153. View in PubMed
Mints YY, Doros Gheorghe, Berk JL, Connors LH & Ruberg FL. Features of atrial fibrillation in wild-type transthyretin cardiac amyloidosis: a systematic review and clinical experience. ESC Heart Failure (2018) 2018 Jun 19. doi:10.1002/ehf2.12308. View in Wiley Library
Copperman TS, Truong MT, Berk JL & Sobel RK. External beam radiation for localized periocular amyloidosis: a case series. Orbit, 37(3) 2018 Jun 11. doi:10.1080/01676830.2018.1483407. View in PubMed
White PS, Phull P, Brauneis D, Sloan JM, Quillen K, Sarosiek S & Sanchorawala V. High-dose melphalan and stem cell transplantation in AL amyloidosis with elevated cardiac biomarkers. Bone Marrow Transplantation, 2018 2018 June 8. doi:10.1038/s41409-018-0242-1. View in Springer Nature
Siddiqi OK, Sanchorawala V & Ruberg FL. Echocardiography and survival in light chain cardiac amyloidosis: back to basics. Circ Cardiovasc Imaging, 11(5) 2018 May 11. doi:10.1161/CIRCIMAGING.118.007826. View in PubMed
Jhaveri T, Sarosiek S, Ruberg FL, Siddiqi O, Berk JL & Sanchorawala V. Once AL amyloidosis: not always AL amyloidosis. Amyloid: The Journal of Protein Folding Disorders 2018 Mar 8. doi:10.1080/13506129.2018.1449104. View in Tandfonline | As Attachment
Hanson JLS, Arvanitis M, Koch CM, Berk JL, Ruberg FL, Prokaeva T & Connors LH. Use of serum transthyretin as a prognostic indicator and predictor of outcome in cardiac amyloid disease associated with wild-type transthyretin. Circulation: Heart Failure, 11 (2) 2018 Feb 15. doi: 10.1161/CIRCHEARTFAILURE.117.004000. View in Ahajournals
Havasi A, Doros G & Sanchorawala V. Predictive value of the new renal response criteria in AL amyloidosis treated with high dose melphalan and stem cell transplantation. American Journal of Hematology. 2018 Feb 12. doi: 10.1002/ajh.25066. View in PubMed
Phull P, Sanchorawala V, Connors LH, Doros G, Ruberg FL, Berk JL & Sarosiek S. Monoclonal gammopathy of undetermined significance in systemic transthyretin amyloidosis (ATTR). Amyloid: The Journal of Protein Folding Disorders. 2018 Feb 9. doi:10.1080/13506129.2018.1436048. View in Tandfonline
Parameswaran H, Huamao ML, Asche CV, Ren J, Yong C, Luptakova K, Faller DV & Sanchorawala V. Treatment patterns and health care resource utilization among patients with relapsed/refractory systemic light chain amyloidosis. Amyloid: The Journal of Protein Folding Disorders. 2018 Jan 5. doi:10.1080/13506129.2017.1411796. View in Tandfonline
Siddiqi OK, & Ruberg FL. Cardiac amyloidosis: an update on pathophysiology, diagnosis, and treatment. Trends in Cardiovascular Medicine, 28 (1), 22-23 2018 January. doi:10.1016/j.tcm.2017.07.004. View in ScienceDirect
2017
Lu Y, Jiang Y, Prokaeva T, Connors LH & Costello CE. Oxidative post-translational modifications of an amyloidogenic immunoglobulin light chain protein. Int J Mass Spectrom. 2017 May. doi: 10.1016/j.ijms.2016.11.006. View in PubMed
Nguyen VP, Brauneis D, Kaku M, Sloan JM, Sarosiek S, Quillen K, Shelton AC, & Sanchorawala V. Neuralgic amyotrophy following high-dose melphalan and autologous peripheral blood stem cell transplantation for AL amyloidosis. Bone Marrow Transplant. 2017 Dec 21. doi: 10.1038/s41409-017-0048-6. View in PubMed
White MK, McCausland KL, Sanchorawala V, Guthrie SD, & Bayliss MS. Psychometric validation of the SF-36 Health Survey in light chain amyloidosis: results from community-based and clinic-based samples. Patient Relate Outcome Mass. 2017 Dec 7; 8: 157-167. doi: 10.2147/PROM.S146849. eCollection 2017. View in PubMed
Sanchorawala V, Sarosiek S, Sloan JM, Brauneis Dina, Migre ME, Mistark M, Santos S, Fennessey S, & Shelton AC. 507 Safety and tolerability of daratumumab in patients with relapsed light chain (AL) amyloidosis: preliminary results of a phase II study. ASH 59th Annual Meeting & Exposition. 2017 Dec 10. View in ASH
Chan GC, Koch CM & Connors LH. Serum proteomic variability associated with clinical phenotype in familial transthyretin amyloidosis (ATTRm). J Proteome Res. 2017 Sept 18. doi: 10.1021/acs.jproteome,7b004479. View in PubMed
Batalini F, Econimo L, Quillen K, Sloan JM, Sarosiek S, Brauneis D, Havasi A, Stern L, Dember L & Sanchorawala V. High dose melphalan and stem cell transplantation in patients on dialysis due to AL amyloidosis and monoclanal immunoglobulin deposition disease. Biol Blood Marrow Transplant. 2017 Aug 30. doi: 10.1016/j.bbmt.2017.08.031. View in PubMed
Sanchorawala V, McCausland KL, White MK, Bayliss MS, Guthrie SD, Lo S & Skinner M. A longitudinal evaluation of health-related quality of life in patients with AL amyloidosis: associations with health outcomes over time. British Journal of Haematology. 2017 Aug 29. doi: 10.1111/bjh.14889. View in Wiley
Arun M, Brauneis D, Doros G, Shelton AC, Sloan JM, Quillen K, Ruberg FL, Sanchorawala V & Varga C. The incidence of atrial fibrillation among patients with AL amyloidosis undergoing high-dose melphalan and stem cell transplantation: experience at a single institution. Bone Marrow Transplantation. 2017 July 24. doi: 10.1038/bmt.2017.148. View in PubMed
Sanchorawala V, Palladini G, Kukreti V, Zonder JA, Cohen AD, Seldin DC, Dispenzieri A, Jaccard A, Schönland SO, Berg D, Yang H, Gupta N, Hui AM, Comenzo RL & Merlini G. A phase 1/2 study of the oral proteasome inhibitor ixazomib in relapsed or refractory light-chain (AL) amyloidosis. Blood 2017. 2017 May 26. doi: 10.1182/blood-2017-03-771220. View in PubMed
Browning S, Quillen K, Sloan JM, Doros G, Sarosiek S & Sanchorawala V. Hematologic relapse in AL amyloidosis after high-dose melphalan and stem cell transplantation. Blood First Edition Paper. 2017 July 11. doi: 10.1182/blood-2017-06-788729. View in Blood Journal
Klimtchuk ES, Prokaeva TB, Spencer BH, Gursky O & Connors LH. In vitro co-expression of human amyloidogenic immunoglobulin light and heavy chain proteins: a relevant cell-based model of AL amyloidosis. Amyloid: The Journal of Protein Folding Disorders. Vol 24. 2017 Jun 20. doi: 10.1080/13506129.2017.1336996. View in Tandfonline
Arvanitis M, Simon S, Chan G, Fine D, Beardsley P, LaValley M, Jacobson D, Koch C, Berk JL, Connors LH & Ruberg FL. Retinol binding protein 4 (RBP4) concentration identifies V122I transthyretin cardiac amyloidosis. Amyloid: The Journal of Protein Folding Disorders. Vol 24. 2017 Apr 22. doi: 10.1080/13506129.2017.1295371. View in PubMed
Siddiqi, OK & Ruberg FL. Challenging the myths of cardiac amyloidosis. European Heart Journal 2017: 0, 1-4. 2017 Apr 24. doi: 10.1093/eurheartj/ehx043. View in Oup
Govender P, Keyes CM, Hankinson EA, O’Hara CJ, Sanchorawala V & Berk JL. Transbrochial biopsies safely diagnose amyloid lung disease. Amyloid: The Journal of Protein Folding Disorders. 2017 Apr 10. doi: 10.1080/13506129.2017.1301917. View in Tandfonline
Pulido V, Doros G, Berk JL & Sanchorawala V. The six-minute walk test in patients with AL amyloidosis: a single centre case series. Br J Haematol. 2017 Mar 24. doi: 10.1111/bjh.14586. View in PubMed
Ruberg FL. Cardiac amyloidosis: a zebra hiding in plain sight?. Circ Cardiovasc Imaging (3): e006186. 2017 March 10. doi: 10.1161/CIRCIMAGING.117.006186. View in PubMed
Arvanitis M, Koch CM, Chan GC, Torres-Arancivia C, LaValley MP, Jacobson DR, Berk JL, Connors LH & Ruberg FL. Identification of transthyretin cardiac amyloidosis using serum retinol-binding protein 4 and a clincal predication model. JAMA Cardiol 2(3): 305-313. 2017 March 1. doi: 10.1001/jamacardio.2016.5864. View in: Pubmed.
Chan GG, Koch CM & Connors LH. Blood proteomic profiling in inherited (ATTRm) and acquired (ATTRwt) forms of transthyretin-associated cardiac amyloidosis. J Proteome Res. 2017 Feb 14. doi: 10.1021/acs.jproteome.6b00998. View in: PubMed
Pawar S, Haq M, Ruberg FL et al. Imaging options in cardiac amyloidosis: differentiating AL from ATTR. Curr Cardiovasc Imaging Rep, 2017, 2017 Feb 01. doi:10.1007/s12410-017-9399-z View in: SpringerLink
Buxbaum JN & Ruberg FL. Transthyretin V122I (pV142I) cardiac amyloidosis: an age dependent autosomal dominant cardiomyopathy too common to be overlooked as a cause of significant heart disease in elderly African Americans. Genetics in Medicine, 2017, 2017 Jan 19. View in: PubMed
2016
Salinaro F, Meier-Ewert HK, Miller E, Shivda P, Sanchorawala V, Berk JL, Seldin DC, Ruberg FL. Longitudinal systolic strain, cardiac function improvement, and survival following treatment of light-chain (AL) cardiac amyloidosis. European Heart Journal – Cardiovascular Imaging, 0, 1-8, 2016 Dec 12. View in: PubMed
Ruberg FL, Fontana M, Gillmore JD. Should histologic determination of Amyloid load determine management decisions in light-chain Amyloidosis? J Am Coll Cardiol. 68(22):2493-2494, 2016 Dec 6. View in: PubMed
Nolin AC, Mulhern RM, Panchenko MV, Pisarek-Horowitz A, Wang Z, Shirihai O, Borkan SC, Havasi A. Proteinuria causes dysfunctional autophagy in the proximal tubule.
Prokaeva T, Spencer B, Sun F, O’Hara RM, Seldin DC, Connors LH, Sanchorawala V. Immunoglobulin heavy light chain test quantifies clonal disease in patients with AL amyloidosis and normal serum free light chain ratio. 23(4):214-220, 2016 Dec. View in: PubMed
Castano A, Haq M, Narotsky DL, Goldsmith J, Weinberg RL, Morgenstern R, Pozniakoff T, Ruberg FL, Miller EJ, Berk JL, Dispenzieri A, Grogan M, Johnson G, Bokhari S, Maurer MS. Multicenter study of planar technetium 99m pyrophosphate cardiac imaging: predicting survival for patients with ATTR cardiac amyloidosis. 1(8):880-889, 2016 Nov 1. View in: PubMed
Havasi A, Stern L, Lo S, Sun F, Sanchorawala V. Validation of new renal staging system in AL amyloidosis treated with high dose melphalan and stem cell transplantation.
Havasi A, Lu W, Cohen HT, Beck LH Jr, Wang Z, Igwebuike C, Borkan SC. Blocking peptides & molecular mimicry as treatment for kidney disease.
Menn-Josephy H, Lee CS, Nolin A, Christov M, Rybin DV, Weinberg JM, Henderson J, Bonegio R, Havasi A. Renal interstitial fibrosis: an imperfect predictor of kidney disease progression in some patient cohorts. Am J Nephrol. 44(4):289-299, 2016 Sep 15. View in: PubMed
Sanchorawala V, Shelton AC, Lo S, Varga C, Sloan JM, Seldin DC. Pomalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 1 and 2 trial. Blood, 128(8):1059-1062, 2016. View in: PubMed
Haq M, Pawar S, Berk JL, Miller EJ, Ruberg FL. Can 99m-Tc-Pyrophosphate aid in early detection of cardiac involvement in asymptomatic variant TTR Amyloidosis? JACC Cardiovasc Imaging. pii: S1936-878X(16)30443-0, 2016 Aug 20. View in: PubMed
Gillmore JD, Maurer MS, Falk RH, Merlini G, Damy T, Dispenzieri A, Wechalekar AD, Berk JL, Quarta CC, Grogan M, Lachmann HJ, Bokhari S, Castano A, Dorbala S, Johnson GB, Glaudemans AW, Rezk T, Fontana M, Palladini G, Milani P, Guidalotti PL, Flatman K, Lane T, Vonberg FW, Whelan CJ, Moon JC, Ruberg FL, Miller EJ, Hutt DF, Hazenberg BP, Rapezzi C, Hawkins PN. Nonbiopsy diagnosis of cardiac transthyretin amyloidosis. Circulation. 133(24):2404-12. 2016 Jun 14. View in: PubMed
Lee SY, Brauneis D, Stern L, Sanchorawala V. Optimal dosing of high-dose melphalan prior to autologous hematopoietic stem cell transplantation in a patient with AL amyloidosis and a solitary kidney. Hematol Oncol Stem Cell Ther, 9(2):86-8, 2016. View in: PubMed
Havasi A, Dong Z. Autophagy and tubular cell death in the kidney.
Sanchorawala V, Gale RP. David C Seldin, MD, PhD: scientist, clinician, teacher, gentleman, 1957-2015. Bone Marrow Transplant, 51(3):323, 2016. View in: PubMed
Connors LH, Sam F, Skinner M, Salinaro F, Sun F, Ruberg FL, Berk JL, and Seldin DC. Heart failure due to age-related cardiac amyloid disease associated with wild-type transthyretin: a prospective, observational cohort study. Circulation, 133:282-290, 2016. View in: PubMed
Bever KM, Masha LI, Sun F, Stern L, Havasi A, Berk JL, Sanchorawala V, Seldin DC, Sloan JM. Risk factors for venous thromboembolism in immunoglobulin light chain amyloidosis. Haematologica, 101(1):86-90, 2016. View in: PubMed
2015
Greene MJ, Klimtchuk ES, Seldin DC, Berk JL, Connors LH. Cooperative stabilization of transthyretin by clusterin and diflunisal. Biochemistry, 54(2):268-78, 2015. View in: PubMed
Sanchorawala V, Sun F, Quillen K, Sloan JM, Berk JL, Seldin DC. Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation: 20-year experience. Blood, 126(20):2345-7, 2015. View in: PubMed
Sikora JL, Logue MW, Chan GG, Spencer BH, Prokaeva TB, Baldwin CT, Seldin DC, Connors LH. Genetic variation of the transthyretin gene in wild-type transthyretin amyloidosis (ATTRwt). Hum Genet, 134(1):111-21, 2015. View in: PubMed
Sarosiek S, Seldin DC, Connors LH, Spencer B, Murakami A, O’Hara C, and Sanchorawala V. Vertebral compression fractures as the initial presentation of AL amyloidosis: case series and review of literature. Amyloid, 22(3):156-62, 2015. View in: PubMed
Stern L, Havasi A. Renal transplantation in amyloidosis and MIDD. Front Biosci (Elite Ed), 7:149-57, 2015. View in: PubMed
Ruberg FL, Berk JL, Semigan MJ. V122I transthyretin variant in elderly black Americans. N Engl J Med, 372(18):1768, 2015. View in: PubMed
Renteria AS, Sanchorawala V, Niehaus ED, Sun F, Semigran MJ, Seldin DC. Serum free light chain trends between orthotopic heart transplantation and auto-SCT in patients with AL amyloidosis. Bone Marrow Transplant, 50(6):868-9, 2015. View in: PubMed
Sanchorawala V, Brauneis D, Shelton AC, Lo S, Sun F, Sloan JM, Quillen K, Seldin DC. Induction Therapy with Bortezomib Followed by Bortezomib-High Dose Melphalan and Stem Cell Transplantation for Light Chain Amyloidosis: Results of a Prospective Clinical Trial. Biol Blood Marrow Transplant, 21(8):1445-51, 2015. View in: PubMed
Suhr OB, Coelho T, Buades J, Pouget J, Conceicao I, Berk J, Schmidt H, Waddington-Cruz M, Campistol JM, Bettencourt BR, Vaishnaw A, Gollob J, Adams D. Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet J Rare Dis, 10:109, 2015. View in: PubMed
Sissoko M, Sanchorawala V, Seldin D, Sworder B, Angelino K, Broce M, Berk J, Sloan JM. Clinical presentation and treatment responses in IgM-related AL amyloidosis. Amyloid, 22(4):229-35, 2015. View in: PubMed
Gertz MA, Benson MD, Dyck PJ, Grogan M, Coelho T, Cruz M, Berk JL, Plante-Bordeneuve V, Schmidt HH, Merlini G. Diagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis. J Am Coll Cardiol, 66(21):2451-66, 2015. View in: PubMed
Bosch N, Renteria AS, Quillen K, Brauneis D, Santilli J, Kim D, Sanchorawala V. Nonoperative Management of Spontaneous Splenic Rupture in a Patient with Light-Chain Amyloidosis: A Case Report. J Vasc Interv Radiol, 26(10):1578-80, 2015. View in: PubMed
Skinner M, Sanchorawala V. In memoriam David C. Seldin, MD, PhD. Amyloid, 22(3):145-6, 2015. View in: PubMed
2014
Reddi HV, Jenkins S, Theis J, Thomas BC, Connors LH, Van Rhee F, Highsmith WE. Homozygosity for the V122I mutation in transthyretin is associated with earlier onset of cardiac amyloidosis in the African American population in the seventh decade of life. J Mol Diagn, 16(1):68-74, 2014. View in: PubMed
Mahmood S, Palladini G, Sanchorawala V, Wechalekar A. Update on treatment of light chain amyloidosis. Haematologica, (2):209-21, 2014. View in: PubMed
Merlini G, Comenzo RL, Seldin DC, Wechalekar A, Gertz MA. Immunoglobulin light chain amyloidosis. Expert Rev Hematol, 7(1):143-56, 2014. View in: PubMed
Gray Gilstrap L, Niehaus E, Malhotra R, Ton VK, Watts J, Seldin DC, Madsen JC, Semigran MJ. Predictors of survival to orthotopic heart transplant in patients with light chain amyloidosis. J Heart Lung Transplant, 33(2):149-56, 2014. View in: PubMed
Girnius S, Seldin DC, Meier-Ewert HK, Sloan JM, Quillen K, Ruberg FL, Berk JL, Doros G, Sanchorawala V. Safety and efficacy of high-dose melphalan and auto-SCT in patients with AL amyloidosis and cardiac involvement. Bone Marrow Transplant, 49(3):434-9, 2014. View in: PubMed
Phay M, Blinder V, Macy S, Greene MJ, Wooliver DC, Liu W, Planas A, Walsh DM, Connors LH, Primmer SR, Planque SA, Paul S, O’Nuallain B. Transthyretin aggregate-specific antibodies recognize cryptic epitopes on patient-derived amyloid fibrils. Rejuvenation Res, 17(2):97-104, 2014. View in: PubMed
von Keudell G, Sanchorawala V, O”Hara C, C Seldin D, Sloan JM. Simultaneous presentation of kappa-restricted chronic lymphocytic leukemia and lambda light chain AL amyloidosis. Amyloid: J Prot Folding Disorders, 21(2):124-7, 2014. View in: PubMed
Lichtman EI, Seldin DC, Shelton A, Sanchorawala V. Single agent lenalidomide three times a week induces hematologic responses in AL amyloidosis patients on dialysis. Am J Hematol, 89(7):706-8, 2014. View in: PubMed
Lee SY, Sanchorawala V, Seldin DC, Mark Sloan J, Andrea N, Quillen K. Plerixafor-augmented peripheral blood stem cell mobilization in AL amyloidosis with cardiac involvement: a case series. Amyloid: J Prot Folding Disorders, 21(3):149-53, 2014. View in: PubMed
Ruzhansky K, Scoon J, Weimer LH, Maurer MS, Berk JL, Brannagan TH. Discordant phenotype in monozygotic female twins with Lys35Thr TTR familial amyloidotic polyneuropathy. J Clin Neuromuscul Dis, 16(1):1-6, 2014. View in: PubMed
Suanprasert N, Berk JL, Benson MD, Dyck PJ, Klein CJ, Gollob JA, Bettencourt BR, Karsten V, Dyck PJ. Retrospective study of a TTR FAP cohort to modify NIS+7 for therapeutic trials. J Neurol Sci, 344(1-2):121-8, 2014. View in: PubMed
Guan J, Mishra S, Qiu Y, Shi J, Trudeau K, Las G, Liesa M, Shirihai OS, Connors LH, Seldin DC, Falk RH, MacRae CA, Liao R. Lysosomal dysfunction and impaired autophagy underlie the pathogenesis of amyloidogenic light chain-mediated cardiotoxicity. EMBO Mol Med, 6(11):1493-507, 2014. View in: PubMed
Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Bladé J, Fermand JP, Hassoun H, Heffner L, Kukreti V, Vescio RA, Pei L, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Long-term follow-up from a phase 1/2 study of single-agent bortezomib in relapsed systemic AL amyloidosis. Blood, 24(16):2498-506, 2014. View in: PubMed
Freeman B, Brauneis D, Seldin DC, Quillen K, Sloan JM, Renteria AS, Shelton AC, Teschner T, Finn KT, Sanchorawala V. Hospital admissions following outpatient administration of high-dose melphalan and autologous SCT for AL amyloidosis. Bone Marrow Transplant, 49(10):1345-6, 2014. View in: PubMed
Sanchorawala V. High dose melphalan and autologous peripheral blood stem cell transplantation in AL amyloidosis. Hematol Oncol Clin North Am, 28(6):1131-44, 2014. View in: PubMed
Fu J, Seldin DC, Berk JL, Sun F, O”Hara C, Cui H, Sanchorawala V. Lymphadenopathy as a manifestation of amyloidosis: a case series. Amyloid: J Prot Folding Disorders, 21(4):256-60, 2014. View in: PubMed
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