Treatment Options For Relapsed/refractory Systemic Light-Chain (AL) Amyloidosis: Current Perspectives

References

• Merlini G, Dispenzieri A, Sanchorawala V, et al. Systemic immunoglobulin light chain amyloidosis. Nat Rev Dis Prim. 2018;4(1):38. doi:10.1038/s41572-018-0034-330361521
   PubMed Web of Science ®Google Scholar
• Palladini G, Milani P, Foli A, et al. Oral melphalan and dexamethasone grants extended survival with minimal toxicity in AL amyloidosis : long-term results of a risk-adapted approach. Haematologica. 2014;99(4):743–750. doi:10.3324/haematol.2013.09546324213149
   PubMed Web of Science ®Google Scholar
• Palladini G, Sachchithanantham S, Milani P, et al. A European collaborative study of cyclophosphamide, bortezomib, and dexamethasone in upfront treatment of systemic AL amyloidosis. Blood. 2015;126(5):612–616. doi:10.1182/blood-2015-01-62030225987656
   PubMed Web of Science ®Google Scholar
• Sanchorawala V, Sun F, Quillen K, et al. Long term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation: 20-year experience. Blood. 2015;126(20):2345–2347. doi:10.1182/blood-2015-08-66272626443620
   PubMed Web of Science ®Google Scholar
• Gertz MA, Comenzo R, Falk RH, et al. Definition of organ involvement and treatment response in immunoglobulin light chain amyloidosis (AL): a consensus opinion from the 10th International Symposium on Amyloid and Amyloidosis. Am J Hematol. 2005;79(4):319–328. doi:10.1002/(ISSN)1096-865216044444
   PubMed Web of Science ®Google Scholar
• Warsame R, Bang SM, Kumar SK, et al. Outcomes and treatments of patients with immunoglobulin light chain amyloidosis who progress or relapse postautologous stem cell transplant. Eur J Haematol. 2014;92(6):485–490. doi:10.1111/ejh.2014.92.issue-624502602
   PubMed Web of Science ®Google Scholar
• Browning S, Quillen K, Sloan JM, et al. Hematologic relapse in AL amyloidosis after high-dose melphalan and stem cell transplantation. Blood. 2017;130(11):1383–1386. doi:10.1182/blood-2017-06-78872928698204
   PubMed Web of Science ®Google Scholar
• Sanchorawala V. Delay treatment of AL amyloidosis at relapse until symptomatic: devil is in the details. Blood Adv. 2019;3(2):216–218. doi:10.1182/bloodadvances.201802126130670538
   PubMed Web of Science ®Google Scholar
• Palladini G, Merlini G. When should treatment of AL amyloidosis start at relapse? Early, to prevent organ progression. Blood Adv. 2019;3(2):212–215. doi:10.1182/bloodadvances.201802125330670537
   PubMed Web of Science ®Google Scholar
• Reece DE, Hegenbart U, Sanchorawala V, et al. Long-term follow-up from a phase 1/2 study of single-agent bortezomib in relapsed systemic AL amyloidosis. Blood. 2014;124(16):2498–2506. doi:10.1182/blood-2014-04-56832925202139
   PubMed Web of Science ®Google Scholar
• Kastritis E, Anagnostopoulos A, Roussou M, et al. Treatment of light chain (AL) amyloidosis with the combination of bortezomib and dexamethasone. Haematologica. 2007;92(10):1351–1358. doi:10.3324/haematol.1132518024372
   PubMed Web of Science ®Google Scholar
• Kastritis E, Wechalekar AD, Dimopoulos MA, et al. Bortezomib with or without dexamethasone in primary systemic (light chain) amyloidosis. J Clin Oncol. 2010;28(6):1031–1037. doi:10.1200/JCO.2009.23.822020085941
   PubMed Web of Science ®Google Scholar
• Mikhael JR, Schuster SR, Jimenez-Zepeda VH, et al. Cyclophosphamide-bortezomib-dexamethasone (CyBorD) produces rapid and complete hematologic response in patients with AL amyloidosis. Blood. 2012;119(19):4391–4394. doi:10.1182/blood-2011-11-39093022331188
   PubMed Web of Science ®Google Scholar
• Kastritis E, Roussou M, Gavriatopoulou M, et al. Long-term outcomes of primary systemic light chain (AL) amyloidosis in patients treated upfront with bortezomib or lenalidomide and the importance of risk adapted strategies. Am J Hematol. 2015;90(4):E60–E65. doi:10.1002/ajh.2393625580702
   PubMed Web of Science ®Google Scholar
• Merz M, Salwender H, Haenel M, et al. Subcutaneous versus intravenous bortezomib in two different induction therapies for newly diagnosed multiple myeloma: an interim analysis from the prospective GMMG-MM5 trial. Haematologica. 2015;100(7):964–969. doi:10.3324/haematol.2015.12434725840597
   PubMed Web of Science ®Google Scholar
• Arnulf B, Pylypenko H, Grosicki S, et al. Updated survival analysis of a randomized phase III study of subcutaneous versus intravenous bortezomib in patients with relapsed multiple myeloma. Haematologica. 2012;97(12):1925–1928. doi:10.3324/haematol.2012.06779322689676
   PubMed Web of Science ®Google Scholar
• Cohen A, Landau H, Scott E, et al. Safety and efficacy of carfilzomib (CFZ) in previously-treated systemic light-chain (AL) amyloidosis. Blood. 2016;128(22):645. doi:10.1182/blood.V128.22.645.645
   Web of Science ®Google Scholar
• FDA grants breakthrough therapy status to ixazomib for AL amyloidosis and JCAR015 for relapsed/refractory ALL. Oncol Times. 2014;36(24):15. doi:10.1097/01.COT.0000459948.58065.8c
   Google Scholar
• Sanchorawala V, Palladini G, Kukreti V, et al. A phase 1/2 study of the oral proteasome inhibitor ixazomib in relapsed or refractory AL amyloidosis. Blood. 2017;130(5):597–605. doi:10.1182/blood-2017-03-77122028550039
   PubMed Web of Science ®Google Scholar
• Takeda Provides Update on TOURMALINE-AL1 Phase 3 Trial in AL Amyloidosis. 2019.
   Google Scholar
• Palladini G, Perfetti V, Perlini S, et al. The combination of thalidomide and intermediate-dose dexamethasone is an effective but toxic treatment for patients with primary amyloidosis (AL). Blood. 2005;105(7):2949–2951. doi:10.1182/blood-2004-08-323115572585
   PubMed Web of Science ®Google Scholar
• Wechalekar AD, Goodman HJB, Lachmann HJ, et al. Safety and efficacy of risk-adapted cyclophosphamide, thalidomide, and dexamethasone in systemic AL amyloidosis. Blood. 2007;109(2):457–464. doi:10.1182/blood-2006-07-03535216990593
   PubMed Web of Science ®Google Scholar
• Seldin D, Choufani E, Dember L, et al. Tolerability and efficacy of thalidomide for the treatment of patients with light chain-associated (AL) amyloidosis. Clin Lymphoma. 2003;3(4):241–246. doi:10.3816/CLM.2003.n.00512672274
   PubMedGoogle Scholar
• Dispenzieri A, Lacy M, Rajkumar S, et al. Poor tolerance to high doses of thalidomide in patients with primary systemic amyloidosis. Amyloid. 2003;10(4):257–261. doi:10.3109/1350612030904174314986485
   PubMed Web of Science ®Google Scholar
• Dispenzieri A, Lacy MQ, Zeldenrust SR, et al. The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis. Blood. 2007;109(2):465–470. doi:10.1182/blood-2006-07-03298717008538
   PubMed Web of Science ®Google Scholar
• Sanchorawala V, Wright D, Rosenzweig M, et al. Lenalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 2 trial. Blood. 2007;109(2):492–496. doi:10.1182/blood-2006-07-03054416960148
   PubMed Web of Science ®Google Scholar
• Mahmood S, Christopher P, Sachchithanantham S, et al. Lenalidomide and dexamethasone for systemic AL amyloidosis following prior treatment with thalidomide or bortezomib regimens. Br J Haematol. 2014;166:842–848. doi:10.1111/bjh.1297324930361
   PubMed Web of Science ®Google Scholar
• Specter R, Sanchorawala V, Seldin DC, et al. Kidney dysfunction during lenalidomide treatment for AL amyloidosis. Nephrol Dial Transplant. 2011;26(3):881–886. doi:10.1093/ndt/gfq48220693160
   PubMed Web of Science ®Google Scholar
• Tapan U, Seldin DC, Finn KT, et al. Increases in B-type natriuretic peptide (BNP) during treatment with lenalidomide in AL amyloidosis. Blood. 2010;116(23):5071–5072. doi:10.1182/blood-2010-09-30513621127185
   PubMed Web of Science ®Google Scholar
• Kastritis E, Terpos E, Roussou M, et al. A phase 1/2 study of lenalidomide with low-dose oral cyclophosphamide and low-dose dexamethasone (RdC) in AL amyloidosis. Blood. 2012;119(23):5384–5390. doi:10.1182/blood-2011-12-39690322517904
   PubMed Web of Science ®Google Scholar
• Kumar SK, Hayman SR, Buadi FK, et al. Lenalidomide, cyclophosphamide, and dexamethasone (CRd) for light-chain amyloidosis: long-term results from a phase 2 trial. Blood. 2012;119(21):4860–4867. doi:10.1182/blood-2012-01-40779122504925
   PubMed Web of Science ®Google Scholar
• Palladini G, Russo P, Milani P, et al. A phase II trial of cyclophosphamide, lenalidomide and dexamethasone in previously treated patients with AL amyloidosis. Haematologica. 2013;98(3):433–436. doi:10.3324/haematol.2012.07359322983583
   PubMed Web of Science ®Google Scholar
• Sanchorawala V, Shelton AC, Lo S, et al. Pomalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 1 and 2 trial. Blood. 2016;128(8):1059–1063. doi:10.1182/blood-2016-04-71082227381904
   PubMed Web of Science ®Google Scholar
• Dispenzieri A, Buadi F, Laumann K, et al. Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis. Blood. 2012;119(23):5397–5404. doi:10.1182/blood-2012-02-41316122493299
   PubMed Web of Science ®Google Scholar
• Palladini G, Milani P, Foli A, et al. A phase 2 trial of pomalidomide and dexamethasone rescue treatment in patients with AL amyloidosis. Blood. 2017;129(15):2120–2123. doi:10.1182/blood-2016-12-75652828130212
   PubMed Web of Science ®Google Scholar
• Jain T, Kosiorek H, Kung S, et al. Treatment with bortezomib-based therapy, followed by autologous stem cell transplantation, improves outcomes in light chain amyloidosis: a retrospective study. Clin Lymph Myeloma Leuk. 2018;18(7):486–492. doi:10.1016/j.clml.2018.04.006
   Web of Science ®Google Scholar
• Cornell RF, Zhong X, Atallah E, et al. Bortezomib-based induction for transplant ineligible AL amyloidosis and feasibility of later transplantation. Bone Marrow Transplant. 2015;50:914–917. doi:10.1038/bmt.2015.7325915809
   PubMed Web of Science ®Google Scholar
• Quillen K, Seldin DC, Finn KT, Sanchorawala V. A second course of high-dose melphalan and auto-SCT for the treatment of relapsed AL amyloidosis. Bone Marrow Transplant. 2011;46(7):976–980. doi:10.1038/bmt.2010.23920956955
   PubMed Web of Science ®Google Scholar
• Kastritis E, Leleu X, Arnulf B, et al. A randomized phase III trial of melphalan and dexamethasone (MDex) versus bortezomib, melphalan and dexamethasone (BMDex) for untreated patients with AL amyloidosis. Blood. 2016;128(22):646. doi:10.1182/blood.V128.22.646.646
   Web of Science ®Google Scholar
• Palladini G, Milani P, Foli A, et al. Presentation and outcome with second-line treatment in AL amyloidosis previously sensitive to nontransplant therapies. Blood. 2018;131(5):525–532. doi:10.1182/blood-2017-04-78054429101236
   PubMed Web of Science ®Google Scholar
• Milani P, Schönland S, Merlini G, et al. Treatment of AL amyloidosis with bendamustine: a study of 122 patients. Blood. 2018;132(18):1988–1991. doi:10.1182/blood-2018-04-84539630108065
   PubMed Web of Science ®Google Scholar
• Lentzsch S, Lagos GG, Comenzo RL, et al. Updated analysis of phase 2 study of bendamustine and dexamethasone in patients with relapsed/refractory systemic light chain (AL) amyloidosis. Amyloid. 2019;26(sup1):113–114. doi:10.1080/13506129.2019.158320031343313
   PubMed Web of Science ®Google Scholar
• Sher T, Fenton B, Akhtar A, Gertz MA. First report of safety and efficacy of daratumumab in 2 cases of advanced immunoglobulin light chain amyloidosis. Blood. 2016;128(15):1987–1989. doi:10.1182/blood-2016-06-72249627543437
   PubMed Web of Science ®Google Scholar
• Kaufman GP, Schrier SL, Lafayette RA, et al. Daratumumab yields rapid and deep hematologic responses in patients with heavily pretreated AL amyloidosis. Blood. 2017;130(7):900–902. doi:10.1182/blood-2017-01-76359928615223
   PubMed Web of Science ®Google Scholar
• Kimmich C, Schonland S, Ziehl R, et al. Daratumumab monotherapy in thirty-two heavily pre-treated patients with advanced systemic light-chain amyloidosis. Blood. 2017;130(Suppl 1):1837.
   Google Scholar
• Sanchorawala V, Sarosiek S, Sloan J, et al. Safety, tolerability and response rates of daratumumab in patients with relapsed light chain (AL) amyloidosis: results of a Phase II study. Blood. 2018;132(Suppl 1):2005. doi:10.1182/blood-2018-99-11299130409894
   PubMed Web of Science ®Google Scholar
• Jaccard A, Arnulf B, Stoppa A, et al. A prospective phase II study of daratumumab in previously-treated systemic light-chain (AL) amyloidosis. EHA Libr. 2018;214545:S851.
   Google Scholar
• Abeykoon JP, Zanwar S, Dispenzieri A, et al. Daratumumab-based therapy in patients with heavily-pretreated AL amyloidosis. Leukemia. 2019;33(2):531–536. doi:10.1038/s41375-018-0262-230267007
   PubMed Web of Science ®Google Scholar
• Sissoko M, Sanchorawala V, Seldin D, et al. Clinical presentation and treatment responses in Clinical presentation and treatment responses in IgM-related AL amyloidosis. Amyloid. 2015;22(4):229–235. doi:10.3109/13506129.2015.109243326488936
   PubMed Web of Science ®Google Scholar
• Palladini G, Foli A, Russo P, et al. Treatment of IgM-associated AL amyloidosis with the combination of rituximab, bortezomib, and dexamethasone. Clin Lymph Myeloma Leuk. 2011;11(1):143–145. doi:10.3816/CLML.2011.n.033
   Web of Science ®Google Scholar
• Manwani R, Sachchithanantham S, Mahmood S, et al. Treatment of IgM-associated immunoglobulin light-chain amyloidosis with rituximab-bendamustine. Blood. 2018;132(7):761LP–764LP. doi:10.1182/blood-2018-04-84649329925500
   PubMed Web of Science ®Google Scholar
• Pika T, Hegenbart U, Flodrova P, et al. First report of ibrutinib in IgM-related amyloidosis: few responses, poor tolerability, and short survival. Blood. 2018;131(3):368LP–371LP. doi:10.1182/blood-2017-09-80646329180400
   PubMed Web of Science ®Google Scholar
• Gertz MA, Landau HJ, Weiss BM. Organ response in patients with AL amyloidosis treated with NEOD001, an amyloid-directed monoclonal antibody. Am J Hematol. 2016;91(12):E506–E507. doi:10.1002/ajh.2456327648922
   PubMed Web of Science ®Google Scholar
• Richards DM, Cookson LM, Berges AC, et al. Therapeutic clearance of amyloid by antibodies to serum amyloid P component. N Engl J Med. 2015;373:1106–1114. doi:10.1056/NEJMoa150494226176329
   PubMed Web of Science ®Google Scholar
• Edwards CV, Bhutani D, Mapara M, et al. One year follow up analysis of the phase 1a/b study of chimeric fibril-reactive monoclonal antibody 11-1F4 in patients with AL amyloidosis. Amyloid. 2019;26(sup1):115–116. doi:10.1080/13506129.2019.158489231343323
   PubMed Web of Science ®Google Scholar
• Kumar S, Kaufman JL, Gasparetto C, et al. Efficacy of venetoclax as targeted therapy for relapsed/refractory t(11;14) multiple myeloma. Blood. 2017;130(22):2401–2409. doi:10.1182/blood-2017-06-78878629018077
   PubMed Web of Science ®Google Scholar
• Moreau P, Chanan-Khan A, Roberts AW, et al. Promising efficacy and acceptable safety of venetoclax plus bortezomib and dexamethasone in relapsed/refractory MM. Blood. 2017;130(22):2392–2400. doi:10.1182/blood-2017-06-78832328847998
   PubMed Web of Science ®Google Scholar
• Avet-Loiseau H, Attal M, Moreau P, et al. Genetic abnormalities and survival in multiple myeloma: the experience of the Intergroupe Francophone du Myélome. Blood. 2007;109(8):3489–3495. doi:10.1182/blood-2006-08-04041017209057
   PubMed Web of Science ®Google Scholar
• Fonseca R, Blood EA, Oken MM, et al. Myeloma and the t(11;14)(q13;q32); evidence for a biologically defined unique subset of patients. Blood. 2002;99(10):3735–3741. doi:10.1182/blood.V99.10.373511986230
   PubMed Web of Science ®Google Scholar
• Bochtler T, Hegenbart U, Cremer FW, et al. Evaluation of the cytogenetic aberration pattern in amyloid light chain amyloidosis as compared with monoclonal gammopathy of undetermined significance reveals common pathways of karyotypic instability. Blood. 2008;111(9):4700–4705. doi:10.1182/blood-2007-11-12210118305218
   PubMed Web of Science ®Google Scholar
• Bochtler T, Hegenbart U, Kunz C, et al. Translocation t(11;14) is associated with adverse outcome in patients with newly diagnosed AL amyloidosis when treated with bortezomib-based regimens. J Clin Oncol. 2015;33(12):1371–1378. doi:10.1200/JCO.2014.57.494725779559
   PubMed Web of Science ®Google Scholar
• Premkumar V, Comenzo R, Lentzsch S. Venetoclax in immunoglobulin light chain amyloidosis: is this the beginning or the end? Clin Lymph Myeloma Leuk. 2019. doi:10.1016/j.clml.2019.07.003
   Web of Science ®Google Scholar
• Leung N, Thomé SD, Dispenzieri A. Venetoclax induced a complete response in a patient with immunoglobulin light chain amyloidosis plateaued on cyclophosphamide, bortezomib and dexamethasone. Haematologica. 2018;103(3):e135–e137. doi:10.3324/haematol.2017.18374929351984
   PubMed Web of Science ®Google Scholar
• Le Bras F, Dupuis J, Lemonnier F, et al. Venetoclax induces sustained complete responses in refractory/relapsed patients with cardiac AL amyloidosis. J Clin Oncol. 2019;37(15_suppl):e19538–e19538.
   Web of Science ®Google Scholar