High dose melphalan and autologous peripheral blood stem cell transplantation in AL amyloidosis

Hematology/Oncology Clinics of North America

Volumn 28, Issue 6, 2014, Pages 1131-1144

  Sanchorawala, Vaishali ^a  

^a BOSTON MEDICAL CENTER   (United States)

Author keywords

AL amyloidosis; Melphalan; Patient selection; Stem cell transplantation; Supportive care; Treatment related mortality

Indexed keywords

AMIODARONE; AMYLOID PROTEIN; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; BLOOD CLOTTING FACTOR 9 COMPLEX; BORTEZOMIB; CALCIUM CHANNEL BLOCKING AGENT; CYCLOPHOSPHAMIDE; DEXAMETHASONE; DIGOXIN; DIURETIC AGENT; DOXORUBICIN; DULOXETINE; FLUDROCORTISONE; GABAPENTIN; GRANULOCYTE COLONY STIMULATING FACTOR; MELPHALAN; MIDODRINE; OCTREOTIDE; PLERIXAFOR; RECOMBINANT BLOOD CLOTTING FACTOR 7A; VINCRISTINE; ALKYLATING AGENT;

AL AMYLOIDOSIS; AMYLOIDOSIS; AUTOLOGOUS PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; BLOOD CLOTTING FACTOR 10 DEFICIENCY; CANCER PROGNOSIS; CANCER SURVIVAL; COMPRESSION STOCKING; DIARRHEA; DRUG MEGADOSE; END STAGE RENAL DISEASE; EVENT FREE SURVIVAL; GRAFT VERSUS HOST REACTION; HEART ARRHYTHMIA; HEART ATRIUM FIBRILLATION; HEMODIALYSIS; HUMAN; KIDNEY TRANSPLANTATION; MACROGLOSSIA; MUCOSAL BLEEDING; MULTIPLE MYELOMA; NEPHROTIC SYNDROME; NEUROPATHIC PAIN; ORTHOSTATIC HYPOTENSION; OVERALL SURVIVAL; PATIENT SELECTION; PERITONEAL DIALYSIS; PLASMA CELL DYSCRASIA; RECOMMENDED DRUG DOSE; REVIEW; SPLENECTOMY; STEM CELL MOBILIZATION; THERAPY EFFECT; TREATMENT DURATION; WHOLE BODY RADIATION; AUTOTRANSPLANTATION; MULTIMODALITY CANCER THERAPY; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; PROCEDURES; REMISSION; SURVIVAL; TREATMENT OUTCOME;

AMYLOIDOSIS; ANTINEOPLASTIC AGENTS, ALKYLATING; COMBINED MODALITY THERAPY; HUMANS; MELPHALAN; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; REMISSION INDUCTION; SURVIVAL ANALYSIS; TRANSPLANTATION, AUTOLOGOUS; TREATMENT OUTCOME;

EID: 84912074846     PISSN: 08898588     EISSN: 15581977     Source Type: Journal    
DOI: 10.1016/j.hoc.2014.08.013     Document Type: Review

References (56)

1. Merlini G., Bellotti V. Molecular mechanisms of amyloidosis. NEngl J Med 2003, 349:583-596.
2. Sipe J.D., Benson M.D., Buxbaum J.N., et al. Amyloid fibril protein nomenclature: 2012 recommendations from the Nomenclature Committee of the International Society of Amyloidosis. Amyloid 2012, 19:167-170.
3. Gertz M.A., Lacy M., Dispenzieri A. Amyloidosis. Hematol Oncol Clin North Am 1999, 13:1211-1220.
4. Kyle R.A., Linos A., Beard C.M., et al. Incidence and natural history of primary systemic amyloidosis in Olmsted County, Minnesota, 1950 through 1989. Blood 1992, 79:1817-1822.
5. Kyle R.A., Gertz M.A. Primary systemic amyloidosis: clinical and laboratory features in 474 cases. Semin Hematol 1995, 32:45-59.
6. Dember L.M. Emerging treatment approaches for the systemic amyloidoses. Kidney Int 2005, 68:1377-1390.
7. 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.
8. Girnius S., Seldin D.C., Cibeira M.T., et al. New hematologic response criteria predict survival in patients with immunoglobulin light chain amyloidosis treated with high-dose melphalan and autologous stem-cell transplantation. JClin Oncol 2013, 31:2749-2750.
9. Sanchorawala V., Seldin D.C., Magnani B., et al. Serum free light-chain responses after high-dose intravenous melphalan and autologous stem cell transplantation for AL (primary) amyloidosis. Bone Marrow Transplant 2005, 36:597-600.
10. 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.
11. Attal M., Harousseau J.L., Stoppa A.M., et al. Aprospective, randomized trial of autologous bone marrow transplantation and chemotherapy in multiple myeloma. Intergroupe Francais du Myelome. NEngl J Med 1996, 335:91-97.
12. Child J.A., Morgan G.J., Davies F.E., et al. High-dose chemotherapy with hematopoietic stem-cell rescue for multiple myeloma. NEngl J Med 2003, 348:1875-1883.
13. Comenzo R.L., Vosburgh E., Simms R.W., et al. Dose-intensive melphalan with blood stem cell support for the treatment of AL amyloidosis: one-year follow-up in five patients. Blood 1996, 88:2801-2806.
14. 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.
15. Sanchorawala V., Skinner M., Quillen K., et al. Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem-cell transplantation. Blood 2007, 110:3561-3563.
16. 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.
17. Comenzo R.L., Gertz M.A. Autologous stem cell transplantation for primary systemic amyloidosis. Blood 2002, 99:4276-4282.
18. Dispenzieri A., Gertz M.A., Kyle R.A., et al. Serum cardiac troponins and N-terminal pro-brain natriuretic peptide: a staging system for primary systemic amyloidosis. JClin Oncol 2004, 22:3751-3757.
19. Palladini G., Campana C., Klersy C., et al. Serum N-terminal pro-brain natriuretic peptide is a sensitive marker of myocardial dysfunction in AL amyloidosis. Circulation 2003, 107:2440-2445.
20. Gertz M., Lacy M., Dispenzieri A., et al. Troponin T level as an exclusion criterion for stem cell transplantation in light-chain amyloidosis. Leuk Lymphoma 2008, 49:36-41.
21. 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.
22. 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.
23. Oran B., Malek K., Sanchorawala V., et al. Predictive factors for hematopoietic engraftment after autologous peripheral blood stem cell transplantation for AL amyloidosis. Bone Marrow Transplant 2005, 35:567-575.
24. Comenzo R.L., Michelle D., LeBlanc M., et al. Mobilized CD34+ cells selected as autografts in patients with primary light-chain amyloidosis: rationale and application. Transfusion 1998, 38:60-69.
25. DiPersio J.F., Stadtmauer E.A., Nademanee A., et al. Plerixafor and G-CSF versus placebo and G-CSF to mobilize hematopoietic stem cells for autologous stem cell transplantation in patients with multiple myeloma. Blood 2009, 113:5720-5726.
26. Lee S.Y., Sanchorawala V., Seldin D.C., et al. Plerixafor-augmented peripheral blood stem cell mobilization in AL amyloidosis with cardiac involvement: a case series. Amyloid 2014, 21:149-153.
27. 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.
28. 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.
29. 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.
30. Seldin D.C., Anderson J.J., Sanchorawala V., et al. Improvement in quality of life of patients with AL amyloidosis treated with high-dose melphalan and autologous stem cell transplantation. Blood 2004, 104:1888-1893.
31. 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.
32. 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):125-126.
33. Meier-Ewert H.K., Sanchorawala V., Berk J.L., et al. Cardiac amyloidosis: evolving approach to diagnosis and management. Curr Treat Options Cardiovasc Med 2011, 13:528-542.
34. Choufani E.B., Sanchorawala V., Ernst T., et al. Acquired factor X deficiency in patients with amyloid light-chain amyloidosis: incidence, bleeding manifestations, and response to high-dose chemotherapy. Blood 2001, 97:1885-1887.
35. Oran B., Wright D.G., Seldin D.C., et al. Spontaneous rupture of the spleen in AL amyloidosis. Am J Hematol 2003, 74:131-135.
36. Tam M., Seldin D.C., Forbes B.M., et al. Spontaneous rupture of the liver in a patient with systemic AL amyloidosis undergoing treatment with high-dose melphalan and autologous stem cell transplantation: a case report with literature review. Amyloid 2009, 16:103-107.
37. Dispenzieri A., Kyle R.A., Lacy M.Q., et al. Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study. Blood 2004, 103:3960-3963.
38. Jaccard A., Moreau P., Leblond V., et al. High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. NEngl J Med 2007, 357:1083-1093.
39. Dey B.R., Chung S.S., Spitzer T.R., et al. Cardiac transplantation followed by dose-intensive melphalan and autologous stem-cell transplantation for light chain amyloidosis and heart failure. Transplantation 2010, 90:905-911.
40. Gillmore J.D., Goodman H.J., Lachmann H.J., et al. Sequential heart and autologous stem cell transplantation for systemic AL amyloidosis. Blood 2006, 107:1227-1229.
41. Lacy M.Q., Dispenzieri A., Hayman S.R., et al. Autologous stem cell transplant after heart transplant for light chain (Al) amyloid cardiomyopathy. JHeart Lung Transplant 2008, 27:823-829.
42. Seldin D.C., Berk J.L., Sam F., et al. Amyloidotic cardiomyopathy: multidisciplinary approach to diagnosis and treatment. Heart Fail Clin 2011, 7:385-393.
43. Sattianayagam P.T., Gibbs S.D., Pinney J.H., et al. Solid organ transplantation in AL amyloidosis. Am J Transplant 2010, 10:2124-2131.
44. Binotto G., Cillo U., Trentin L., et al. Double autologous bone marrow transplantation and orthotopic liver transplantation in a patient with primary light chain (AL) amyloidosis. Amyloid 2011, 18(Suppl 1):127-129.
45. Seldin D.C., Anderson J.J., Skinner M., et al. Successful treatment of AL amyloidosis with high-dose melphalan and autologous stem cell transplantation in patients over age 65. Blood 2006, 108:3945-3947.
46. Casserly L.F., Fadia A., Sanchorawala V., et al. High-dose intravenous melphalan with autologous stem cell transplantation in AL amyloidosis-associated end-stage renal disease. Kidney Int 2003, 63:1051-1057.
47. 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.
48. Madan S., Kumar S.K., Dispenzieri A., et al. High-dose melphalan and peripheral blood stem cell transplantation for light-chain amyloidosis with cardiac involvement. Blood 2012, 119:1117-1122.
49. Gertz M.A., Lacy M.Q., Dispenzieri A., et al. Transplantation for amyloidosis. Curr Opin Oncol 2007, 19:136-141.
50. Mollee P.N., Wechalekar A.D., Pereira D.L., et al. Autologous stem cell transplantation in primary systemic amyloidosis: the impact of selection criteria on outcome. Bone Marrow Transplant 2004, 33:271-277.
51. Schonland S.O., Perz J.B., Hundemer M., et al. Indications for high-dose chemotherapy with autologous stem cell support in patients with systemic amyloid light chain amyloidosis. Transplantation 2005, 80:S160-163.
52. Chow L.Q., Bahlis N., Russell J., et al. Autologous transplantation for primary systemic AL amyloidosis is feasible outside a major amyloidosis referral centre: the Calgary BMT Program experience. Bone Marrow Transplant 2005, 36:591-596.
53. Moreau P., Leblond V., Bourquelot P., et al. Prognostic factors for survival and response after high-dose therapy and autologous stem cell transplantation in systemic AL amyloidosis: a report on 21 patients. Br J Haematol 1998, 101:766-769.
54. Gertz M.A., Blood E., Vesole D.H., et al. Amulticenter phase 2 trial of stem cell transplantation for immunoglobulin light-chain amyloidosis (E4A97): an Eastern Cooperative Oncology Group Study. Bone Marrow Transplant 2004, 34:149-154.
55. Goodman H.J., Gillmore J.D., Lachmann H.J., et al. Outcome of autologous stem cell transplantation for AL amyloidosis in the UK. Br J Haematol 2006, 134:417-425.
56. Vesole D.H., Perez W.S., Akasheh M., et al. High-dose therapy and autologous hematopoietic stem cell transplantation for patients with primary systemic amyloidosis: a Center for International Blood and Marrow Transplant Research Study. Mayo Clin Proc 2006, 81:880-888.