Induction Therapy with Bortezomib Followed by Bortezomib-High Dose Melphalan and Stem Cell Transplantation for Light Chain Amyloidosis: Results of a Prospective Clinical Trial

Biology of Blood and Marrow Transplantation

Volumn 21, Issue 8, 2015, Pages 1445-1451

  Sanchorawala, Vaishali ^a   Brauneis, Dina ^a   Shelton, Anthony C ^a   Lo, Stephen ^a   Sun, Fangui ^b   Sloan, J Mark ^a   Quillen, Karen ^a   Seldin, David C ^a  

^a BOSTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b BOSTON UNIVERSITY SCHOOL OF PUBLIC HEALTH   (United States)

Author keywords

AL amyloidosis; Bortezomib; Induction; Melphalan; Stem cell transplantation

Indexed keywords

ACICLOVIR; ANTIEMETIC AGENT; BIOLOGICAL MARKER; BORTEZOMIB; BRAIN NATRIURETIC PEPTIDE; BUDESONIDE; CORTICOSTEROID; CREATININE; DEXAMETHASONE; FLUCONAZOLE; GRANULOCYTE COLONY STIMULATING FACTOR; LEVOFLOXACIN; MELPHALAN; PROTON PUMP INHIBITOR; RITUXIMAB; TROPONIN I; ANTINEOPLASTIC AGENT;

ACUTE KIDNEY FAILURE; ADULT; AGED; AMYLOIDOSIS; ANTIVIRAL THERAPY; ARTICLE; ARTIFICIAL EMBOLISM; AUTOLOGOUS STEM CELL TRANSPLANTATION; BLOOD TOXICITY; BRONCHOPNEUMONIA; CAUSE OF DEATH; CLINICAL ARTICLE; CLINICAL TRIAL; COLON BIOPSY; CORTICOSTEROID THERAPY; CREATININE BLOOD LEVEL; DIARRHEA; DRUG DOSE REDUCTION; DRUG ERUPTION; DRUG MEGADOSE; DRUG PULSE THERAPY; DRUG RESPONSE; DRUG TOLERABILITY; DRUG WITHDRAWAL; END STAGE RENAL DISEASE; FAINTNESS; FEBRILE NEUTROPENIA; FEMALE; FOLLOW UP; GRAFT VERSUS HOST REACTION; HEART DISEASE; HEMODIALYSIS; HEPARIN INDUCED THROMBOCYTOPENIA; HUMAN; HUMAN TISSUE; IMMUNOSUPPRESSIVE TREATMENT; INDUCTION CHEMOTHERAPY; INFLUENZA A; INTENTION TO TREAT ANALYSIS; INVASIVE ASPERGILLOSIS; KIDNEY DISEASE; KIDNEY DYSFUNCTION; LIGHT CHAIN AMYLOIDOSIS; LIVER DISEASE; LOW DRUG DOSE; MALE; MORTALITY; MULTIPLE CYCLE TREATMENT; MULTIPLE ORGAN FAILURE; NAUSEA; NEUTROPHIL; ORTHOSTATIC HYPOTENSION; OVERALL SURVIVAL; PERIPHERAL EDEMA; PLASMA CELL DYSCRASIA; PROGRESSION FREE SURVIVAL; PROPHYLAXIS; PROSPECTIVE STUDY; RADICULOPATHY; RASH; RENAL REPLACEMENT THERAPY; RESPIRATORY FAILURE; SEPSIS; SPLEEN RUPTURE; STEM CELL MOBILIZATION; STOMATITIS; THROMBOCYTOPENIA; VOMITING; HEMATOPOIETIC STEM CELL TRANSPLANTATION; MIDDLE AGED; PROCEDURES; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING;

ADULT; AGED; AMYLOIDOSIS; ANTINEOPLASTIC AGENTS; BORTEZOMIB; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INDUCTION CHEMOTHERAPY; MALE; MELPHALAN; MIDDLE AGED; PROSPECTIVE STUDIES; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING;

EID: 84937731609     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2015.04.001     Document Type: Article

References (39)

1. Merlini G., Bellotti V. Molecular mechanisms of amyloidosis. NEngl J Med 2003, 349:583-596.
2. Skinner M., Anderson J., Simms R., et al. Treatment of 100 patients with primary amyloidosis: a randomized trial of melphalan, prednisone, and colchicine versus colchicine only. Am J Med 1996, 100:290-298.
3. Kyle R.A., Gertz M.A., Greipp P.R., et al. Atrial of three regimens for primary amyloidosis: colchicine alone, melphalan and prednisone, and melphalan, prednisone, and colchicine. NEngl J Med 1997, 336:1202-1207.
4. Cibeira M.T., Sanchorawala V., Seldin D.C., et al. Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients. Blood 2011, 118:4346-4352.
5. Gertz M.A., Lacy M.Q., Dispenzieri A., et al. Effect of hematologic response on outcome of patients undergoing transplantation for primary amyloidosis: importance of achieving a complete response. Haematologica 2007, 92:1415-1418.
6. Skinner M., Sanchorawala V., Seldin D.C., et al. High-dose melphalan and autologous stem-cell transplantation in patients with AL amyloidosis: an 8-year study. Ann Intern Med 2004, 140:85-93.
7. Parmar S., Kongtim P., Champlin R., et al. Auto-SCT improves survival in systemic light chain amyloidosis: a retrospective analysis with 14-year follow-up. Bone Marrow Transplant 2014, 49:1036-1041.
8. Dispenzieri A., Seenithamby K., Lacy M.Q., et al. Patients with immunoglobulin light chain amyloidosis undergoing autologous stem cell transplantation have superior outcomes compared with patients with multiple myeloma: a retrospective review from a tertiary referral center. Bone Marrow Transplant 2013, 48:1302-1307.
9. Devine S.M. Re-examining the role of high-dose chemotherapy in the treatment of light chain amyloidosis. Biol Blood Marrow Transplant 2014, 20:14-19.
10. Dember L.M., Sanchorawala V., Seldin D.C., et al. Effect of dose-intensive intravenous melphalan and autologous blood stem-cell transplantation on al amyloidosis-associated renal disease. Ann Intern Med 2001, 134:746-753.
11. Girnius S., Seldin D.C., Skinner M., et al. Hepatic response after high-dose melphalan and stem cell transplantation in patients with AL amyloidosis associated liver disease. Haematologica 2009, 94:1029-1032.
12. Meier-Ewert H.K., Sanchorawala V., Berk J., et al. Regression of cardiac wall thickness following chemotherapy and stem cell transplantation for light chain (AL) amyloidosis. Amyloid 2011, 18(Suppl 1):130-131.
13. Lachmann H.J., Gallimore R., Gillmore J.D., et al. Outcome in systemic AL amyloidosis in relation to changes in concentration of circulating free immunoglobulin light chains following chemotherapy. Br J Haematol 2003, 122:78-84.
14. Palladini G., Dispenzieri A., Gertz M.A., et al. New criteria for response to treatment in immunoglobulin light chain amyloidosis based on free light chain measurement and cardiac biomarkers: impact on survival outcomes. JClin Oncol 2012, 30:4541-4549.
15. Sanchorawala V., Wright D.G., Seldin D.C., et al. High-dose intravenous melphalan and autologous stem cell transplantation as initial therapy or following two cycles of oral chemotherapy for the treatment of AL amyloidosis: results of a prospective randomized trial. Bone Marrow Transplant 2004, 33:381-388.
16. Sanchorawala V., Wright D.G., Quillen K., et al. Tandem cycles of high-dose melphalan and autologous stem cell transplantation increases the response rate in AL amyloidosis. Bone Marrow Transplant 2007, 40:557-562.
17. Sanchorawala V., Hoering A., Seldin D.C., et al. Modified high-dose melphalan and autologous SCT for AL amyloidosis or high-risk myeloma: analysis of SWOG trial S0115. Bone Marrow Transplant 2013, 48:1537-1542.
18. Landau H., Hassoun H., Rosenzweig M.A., et al. Bortezomib and dexamethasone consolidation following risk-adapted melphalan and stem cell transplantation for patients with newly diagnosed light-chain amyloidosis. Leukemia 2013, 27:823-828.
19. Cohen A.D., Zhou P., Chou J., et al. Risk-adapted autologous stem cell transplantation with adjuvant dexamethasone+/- thalidomide for systemic light-chain amyloidosis: results of a phase II trial. Br J Haematol 2007, 139:224-233.
20. 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, 105:2949-2951.
21. Sanchorawala V., Wright D.G., Rosenzweig M., et al. Lenalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 2 trial. Blood 2007, 109:492-496.
22. Dispenzieri A., Lacy M.Q., Zeldenrust S.R., et al. The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis. Blood 2007, 109:465-470.
23. Reece D.E., 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, 124:2498-2506.
24. Kastritis E., Wechalekar A.D., Dimopoulos M.A., et al. Bortezomib with or without dexamethasone in primary systemic (light chain) amyloidosis. JClin Oncol 2010, 28:1031-1037.
25. Mikhael J.R., Schuster S.R., Jimenez-Zepeda V.H., et al. Cyclophosphamide-bortezomib-dexamethasone (CyBorD) produces rapid and complete hematologic response in patients with AL amyloidosis. Blood 2012, 119:4391-4394.
26. Sanchorawala V., Quillen K., Sloan J.M., et al. Bortezomib and high-dose melphalan conditioning for stem cell transplantation for AL amyloidosis: a pilot study. Haematologica 2011, 96:1890-1892.
27. Gertz M.A., Comenzo R., Falk R.H., 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, Tours, France, 18-22 April 2004. Am J Hematol 2005, 79:319-328.
28. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989, 10:1-10.
29. Dispenzieri A., Lacy M.Q., Kyle R.A., et al. Eligibility for hematopoietic stem-cell transplantation for primary systemic amyloidosis is a favorable prognostic factor for survival. JClin Oncol 2001, 19:3350-3356.
30. Perz J.B., Schonland S.O., Hundemer M., et al. High-dose melphalan with autologous stem cell transplantation after VAD induction chemotherapy for treatment of amyloid light chain amyloidosis: a single centre prospective phase II study. Br J Haematol 2004, 127:543-551.
31. Huang X., Wang Q., Chen W., et al. Induction therapy with bortezomib and dexamethasone followed by autologous stem cell transplantation versus autologous stem cell transplantation alone in the treatment ofrenal AL amyloidosis: a randomized controlled trial. BMC Med 2014, 12:2.
32. Cornell R.F., Hari P., Zhang M.J., et al. Divergent effects of novel immunomodulatory agents and cyclophosphamide on the risk of engraftment syndrome after autologous peripheral blood stem cell transplantation for multiple myeloma. Biol Blood Marrow Transplant 2013, 19:1368-1373.
33. Kline J., Subbiah S., Lazarus H.M., van Besien K. Autologous graft-versus-host disease: harnessing anti-tumor immunity through impaired self-tolerance. Bone Marrow Transplant 2008, 41:505-513.
34. Lazarus H.M., Sommers S.R., Arfons L.M., et al. Spontaneous autologous graft-versus-host disease in plasma cell myeloma autograft recipients: flow cytometric analysis of hematopoietic progenitor cell grafts. Biol Blood Marrow Transplant 2011, 17:970-978.
35. Reece D.E., Sanchorawala V., Hegenbart U., et al. Weekly and twice-weekly bortezomib in patients with systemic AL amyloidosis: results of a phase 1 dose-escalation study. Blood 2009, 114:1489-1497.
36. Shah G., Kaul E., Fallo S., et al. Bortezomib subcutaneous injection in combination regimens for myeloma or systemic light-chain amyloidosis: a retrospective chart review of response rates and toxicity in newly diagnosed patients. Clin Therap 2013, 35:1614-1620.
37. Dhakal B., Strouse C., D'Souza A., et al. Plerixafor and abbreviated-course granulocyte colony-stimulating factor for mobilizing hematopoietic progenitor cells in light chain amyloidosis. Biol Blood Marrow Transplant 2014, 20:1926-1931.
38. Girnius S., Seldin D.C., Meier-Ewert H.K., et al. Safety and efficacy of high-dose melphalan and auto-SCT in patients with AL amyloidosis and cardiac involvement. Bone Marrow Transplant 2014, 49:434-439.
39. Kongtim P., Qazilbash M.H., Shah J.J., et al. High-dose therapy with auto-SCT is feasible in high-risk cardiac amyloidosis. Bone Marrow Transplant 2015 Mar 2, doi: 10.1038/bmt.2015.21. [Epub ahead of print].