Outcome of Transplantation for Myelofibrosis

Karen K. Ballen; Smriti Shrestha; Kathleen A. Sobocinski; Mei Jie Zhang; Asad Bashey; Brian J. Bolwell; Francisco Cervantes; Steven M. Devine; Robert Peter Gale; Vikas Gupta; Theresa E. Hahn; William J. Hogan; Nicolaus Kröger; Mark R. Litzow; David I. Marks; Richard T. Maziarz; Philip L. McCarthy; Gary Schiller; Harry C. Schouten; Vivek Roy; Peter H. Wiernik; Mary M. Horowitz; Sergio A. Giralt; Mukta Arora

doi:10.1016/j.bbmt.2009.10.025

Outcome of Transplantation for Myelofibrosis

Karen K. Ballen, Smriti Shrestha, Kathleen A. Sobocinski, Mei Jie Zhang, Asad Bashey, Brian J. Bolwell, Francisco Cervantes, Steven M. Devine, Robert Peter Gale, Vikas Gupta, Theresa E. Hahn, William J. Hogan, Nicolaus Kröger, Mark R. Litzow, David I. Marks, Richard T. Maziarz, Philip L. McCarthy, Gary Schiller, Harry C. Schouten, Vivek RoyPeter H. Wiernik, Mary M. Horowitz, Sergio A. Giralt, Mukta Arora

Medicine - Hematology, Oncology, Transplant

Research output: Contribution to journal › Article › peer-review

221 Scopus citations

Abstract

Myelofibrosis is a myeloproliferative disorder incurable with conventional strategies. Several small series have reported long-term disease-free survival (DSF) after allogeneic hematopoietic cell transplantation (HCT). In this study, we analyze the outcomes of 289 patients receiving allogeneic transplantation for primary myelofibrosis between 1989 and 2002, from the database of the Center for International Bone Marrow Transplant Research (CIBMTR). The median age was 47 years (range: 18-73 years). Donors were HLA identical siblings in 162 patients, unrelated individuals in 101 patients, and HLA nonidentical family members in 26 patients. Patients were treated with a variety of conditioning regimens and graft-versus-host disease (GVHD) prophylaxis regimens. Splenectomy was performed in 65 patients prior to transplantation. The 100-day treatment-related mortality was 18% for HLA identical sibling transplants, 35% for unrelated transplants, and 19% for transplants from alternative related donors. Corresponding 5-year overall survival (OS) rates were 37%, 30%, and 40%, respectively. DFS rates were 33%, 27%, and 22%, respectively. DFS for patients receiving reduced-intensity transplants was comparable: 39% for HLA identical sibling donors and 17% for unrelated donors at 3 years. In this large retrospective series, allogeneic transplantation for myelofibrosis resulted in long-term relapse-free survival (RFS) in about one-third of patients.

Original language	English (US)
Pages (from-to)	358-367
Number of pages	10
Journal	Biology of Blood and Marrow Transplantation
Volume	16
Issue number	3
DOIs	https://doi.org/10.1016/j.bbmt.2009.10.025
State	Published - Mar 2010

Bibliographical note

Funding Information:
Financial disclosure: The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement U24-CA76518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); a Grant/Cooperative Agreement 5U01HL069294 from NHLBI and NCI; a contract HHSH234200637015C with Health Resources and Services Administration (HRSA/DHHS); 2 Grants N00014-06-1-0704 and N00014-08-1-0058 from the Office of Naval Research; and grants from AABB; Aetna; American Society for Blood and Marrow Transplantation; Amgen, Inc.; anonymous donation to the Medical College of Wisconsin; Astellas Pharma US, Inc.; Baxter International, Inc.; Bayer HealthCare Pharmaceuticals; Be the Match Foundation; Biogen IDEC; BioMarin Pharmaceutical, Inc.; Biovitrum AB; BloodCenter of Wisconsin; Blue Cross and Blue Shield Association; Bone Marrow Foundation; Canadian Blood and Marrow Transplant Group; CaridianBCT; Celgene Corporation; CellGenix, GmbH; Centers for Disease Control and Prevention; Children's Leukemia Research Association; ClinImmune Labs; CTI Clinical Trial and Consulting Services; Cubist Pharmaceuticals; Cylex Inc.; CytoTherm; DOR BioPharma, Inc.; Dynal Biotech, an Invitrogen Company; Eisai, Inc.; Enzon Pharmaceuticals, Inc.; European Group for Blood and Marrow Transplantation; Gamida Cell, Ltd.; GE Healthcare; Genentech, Inc.; Genzyme Corporation; Histogenetics, Inc.; HKS Medical Information Systems; Hospira, Inc.; Infectious Diseases Society of America; Kiadis Pharma; Kirin Brewery Co., Ltd.; The Leukemia & Lymphoma Society; Merck & Company; The Medical College of Wisconsin; MGI Pharma, Inc.; Michigan Community Blood Centers; Millennium Pharmaceuticals, Inc.; Miller Pharmacal Group; Milliman USA, Inc.; Miltenyi Biotec, Inc.; National Marrow Donor Program; Nature Publishing Group; New York Blood Center; Novartis Oncology; Oncology Nursing Society; Osiris Therapeutics, Inc.; Otsuka America Pharmaceutical, Inc.; Pall Life Sciences; PDL BioPharma, Inc; Pfizer Inc; Pharmion Corporation; Saladax Biomedical, Inc.; Schering Corporation; Society for Healthcare Epidemiology of America; StemCyte, Inc.; StemSoft Software, Inc.; Sysmex America, Inc.; Teva Pharmaceutical Industries;; THERAKOS, Inc.; Thermogenesis Corporation; Vidacare Corporation; Vion Pharmaceuticals, Inc.; ViraCor Laboratories; ViroPharma, Inc.; and Wellpoint, Inc. The views expressed in this article do not reflect the official policy or position of the National Institutes of Health, the Department of the Navy, the Department of Defense, or any other agency of the U.S. Government.

Keywords

Allogeneic transplantation
Myelofibrosis

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Ballen, K. K., Shrestha, S., Sobocinski, K. A., Zhang, M. J., Bashey, A., Bolwell, B. J., Cervantes, F., Devine, S. M., Gale, R. P., Gupta, V., Hahn, T. E., Hogan, W. J., Kröger, N., Litzow, M. R., Marks, D. I., Maziarz, R. T., McCarthy, P. L., Schiller, G., Schouten, H. C., ... Arora, M. (2010). Outcome of Transplantation for Myelofibrosis. Biology of Blood and Marrow Transplantation, 16(3), 358-367. https://doi.org/10.1016/j.bbmt.2009.10.025

Ballen, KK, Shrestha, S, Sobocinski, KA, Zhang, MJ, Bashey, A, Bolwell, BJ, Cervantes, F, Devine, SM, Gale, RP, Gupta, V, Hahn, TE, Hogan, WJ, Kröger, N, Litzow, MR, Marks, DI, Maziarz, RT, McCarthy, PL, Schiller, G, Schouten, HC, Roy, V, Wiernik, PH, Horowitz, MM, Giralt, SA & Arora, M 2010, 'Outcome of Transplantation for Myelofibrosis', Biology of Blood and Marrow Transplantation, vol. 16, no. 3, pp. 358-367. https://doi.org/10.1016/j.bbmt.2009.10.025

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abstract = "Myelofibrosis is a myeloproliferative disorder incurable with conventional strategies. Several small series have reported long-term disease-free survival (DSF) after allogeneic hematopoietic cell transplantation (HCT). In this study, we analyze the outcomes of 289 patients receiving allogeneic transplantation for primary myelofibrosis between 1989 and 2002, from the database of the Center for International Bone Marrow Transplant Research (CIBMTR). The median age was 47 years (range: 18-73 years). Donors were HLA identical siblings in 162 patients, unrelated individuals in 101 patients, and HLA nonidentical family members in 26 patients. Patients were treated with a variety of conditioning regimens and graft-versus-host disease (GVHD) prophylaxis regimens. Splenectomy was performed in 65 patients prior to transplantation. The 100-day treatment-related mortality was 18% for HLA identical sibling transplants, 35% for unrelated transplants, and 19% for transplants from alternative related donors. Corresponding 5-year overall survival (OS) rates were 37%, 30%, and 40%, respectively. DFS rates were 33%, 27%, and 22%, respectively. DFS for patients receiving reduced-intensity transplants was comparable: 39% for HLA identical sibling donors and 17% for unrelated donors at 3 years. In this large retrospective series, allogeneic transplantation for myelofibrosis resulted in long-term relapse-free survival (RFS) in about one-third of patients.",

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author = "Ballen, {Karen K.} and Smriti Shrestha and Sobocinski, {Kathleen A.} and Zhang, {Mei Jie} and Asad Bashey and Bolwell, {Brian J.} and Francisco Cervantes and Devine, {Steven M.} and Gale, {Robert Peter} and Vikas Gupta and Hahn, {Theresa E.} and Hogan, {William J.} and Nicolaus Kr{\"o}ger and Litzow, {Mark R.} and Marks, {David I.} and Maziarz, {Richard T.} and McCarthy, {Philip L.} and Gary Schiller and Schouten, {Harry C.} and Vivek Roy and Wiernik, {Peter H.} and Horowitz, {Mary M.} and Giralt, {Sergio A.} and Mukta Arora",

note = "Funding Information: Financial disclosure: The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement U24-CA76518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); a Grant/Cooperative Agreement 5U01HL069294 from NHLBI and NCI; a contract HHSH234200637015C with Health Resources and Services Administration (HRSA/DHHS); 2 Grants N00014-06-1-0704 and N00014-08-1-0058 from the Office of Naval Research; and grants from AABB; Aetna; American Society for Blood and Marrow Transplantation; Amgen, Inc.; anonymous donation to the Medical College of Wisconsin; Astellas Pharma US, Inc.; Baxter International, Inc.; Bayer HealthCare Pharmaceuticals; Be the Match Foundation; Biogen IDEC; BioMarin Pharmaceutical, Inc.; Biovitrum AB; BloodCenter of Wisconsin; Blue Cross and Blue Shield Association; Bone Marrow Foundation; Canadian Blood and Marrow Transplant Group; CaridianBCT; Celgene Corporation; CellGenix, GmbH; Centers for Disease Control and Prevention; Children's Leukemia Research Association; ClinImmune Labs; CTI Clinical Trial and Consulting Services; Cubist Pharmaceuticals; Cylex Inc.; CytoTherm; DOR BioPharma, Inc.; Dynal Biotech, an Invitrogen Company; Eisai, Inc.; Enzon Pharmaceuticals, Inc.; European Group for Blood and Marrow Transplantation; Gamida Cell, Ltd.; GE Healthcare; Genentech, Inc.; Genzyme Corporation; Histogenetics, Inc.; HKS Medical Information Systems; Hospira, Inc.; Infectious Diseases Society of America; Kiadis Pharma; Kirin Brewery Co., Ltd.; The Leukemia & Lymphoma Society; Merck & Company; The Medical College of Wisconsin; MGI Pharma, Inc.; Michigan Community Blood Centers; Millennium Pharmaceuticals, Inc.; Miller Pharmacal Group; Milliman USA, Inc.; Miltenyi Biotec, Inc.; National Marrow Donor Program; Nature Publishing Group; New York Blood Center; Novartis Oncology; Oncology Nursing Society; Osiris Therapeutics, Inc.; Otsuka America Pharmaceutical, Inc.; Pall Life Sciences; PDL BioPharma, Inc; Pfizer Inc; Pharmion Corporation; Saladax Biomedical, Inc.; Schering Corporation; Society for Healthcare Epidemiology of America; StemCyte, Inc.; StemSoft Software, Inc.; Sysmex America, Inc.; Teva Pharmaceutical Industries;; THERAKOS, Inc.; Thermogenesis Corporation; Vidacare Corporation; Vion Pharmaceuticals, Inc.; ViraCor Laboratories; ViroPharma, Inc.; and Wellpoint, Inc. The views expressed in this article do not reflect the official policy or position of the National Institutes of Health, the Department of the Navy, the Department of Defense, or any other agency of the U.S. Government.",

year = "2010",

month = mar,

doi = "10.1016/j.bbmt.2009.10.025",

language = "English (US)",

volume = "16",

pages = "358--367",

journal = "Biology of Blood and Marrow Transplantation",

issn = "1083-8791",

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number = "3",

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T1 - Outcome of Transplantation for Myelofibrosis

AU - Ballen, Karen K.

AU - Shrestha, Smriti

AU - Sobocinski, Kathleen A.

AU - Zhang, Mei Jie

AU - Bashey, Asad

AU - Bolwell, Brian J.

AU - Cervantes, Francisco

AU - Devine, Steven M.

AU - Gale, Robert Peter

AU - Gupta, Vikas

AU - Hahn, Theresa E.

AU - Hogan, William J.

AU - Kröger, Nicolaus

AU - Litzow, Mark R.

AU - Marks, David I.

AU - Maziarz, Richard T.

AU - McCarthy, Philip L.

AU - Schiller, Gary

AU - Schouten, Harry C.

AU - Roy, Vivek

AU - Wiernik, Peter H.

AU - Horowitz, Mary M.

AU - Giralt, Sergio A.

AU - Arora, Mukta

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Outcome of Transplantation for Myelofibrosis

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