MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis

Damiano Rondelli, Judith D. Goldberg, Luis Isola, Leah S. Price, Tsiporah B. Shore, Michael Boyer, Andrea Bacigalupo, Alessandro Rambaldi, Marco Scarano, Rebecca B. Klisovic, Vikas Gupta, Bjorn Andreasson, John Mascarenhas, Meir Wetzler, Alessandro M. Vannucchi, Josef T. Prchal, Vesna Najfeld, Attilio Orazi, Rona S. Weinberg, Crystal Miller & 5 others Giovanni Barosi, Lewis R. Silverman, Giuseppe Prosperini, Roberto Marchioli, Ronald Hoffman

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Abstract

From 2007 to 2011, 66 patients with primary myelofibrosis or myelofibrosis (MF) preceded by essential thrombocythemia or polycythemia vera were enrolled into a prospective phase 2 clinical trial of reduced-intensity allogeneic hematopoietic stem cell transplantation (AHSCT), Myeloproliferative Disorder Research Consortium 101 trial. The study included patients with sibling donors (n = 32) receiving fludarabine/melphalan (FluMel) as a preparative regimen and patients with unrelated donors (n = 34) receiving conditioning with FluMel plus anti-thymocyte globulin (ATG). Patient characteristics in the 2 cohorts were similar. Engraftment occurred in 97% of siblings and 76% of unrelated transplants, whereas secondary graft failure occurred in 3% and 12%, respectively. With a median follow-up of 25 months for patients alive, the overall survival (OS) was 75% in the sibling group (median not reached) and 32% in the unrelated group (median OS: 6 months, 95% confidence interval [CI]: 3, 25) (hazard ratio 3.9, 95% CI: 1.8,8.9) (P <.001). Nonrelapse mortality was 22% in sibling and 59% in unrelated AHSCT. Survival correlated with type of donor, but not with the degree of histocompatibility match, age, or JAK2 V617F status. In patients with MF with sibling donors, AHSCT is an effective therapy, whereas AHSCT from unrelated donors with FluMel/ATG conditioning led to a high rate of graft failure and limited survival. This trial was registered at www.clinicaltrials.gov as #NCT00572897.

Original languageEnglish
Pages (from-to)1183-1191
Number of pages9
JournalBlood
Volume124
Issue number7
DOIs
Publication statusPublished - Aug 14 2014

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Primary Myelofibrosis
Hematopoietic Stem Cell Transplantation
Stem cells
Melphalan
Siblings
Prospective Studies
Antilymphocyte Serum
Grafts
Unrelated Donors
Survival
Tissue Donors
Transplants
Confidence Intervals
Essential Thrombocythemia
Myeloproliferative Disorders
Hazards
Polycythemia Vera
Histocompatibility
RC 101
Clinical Trials

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Rondelli, D., Goldberg, J. D., Isola, L., Price, L. S., Shore, T. B., Boyer, M., ... Hoffman, R. (2014). MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis. Blood, 124(7), 1183-1191. https://doi.org/10.1182/blood-2014-04-572545

MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis. / Rondelli, Damiano; Goldberg, Judith D.; Isola, Luis; Price, Leah S.; Shore, Tsiporah B.; Boyer, Michael; Bacigalupo, Andrea; Rambaldi, Alessandro; Scarano, Marco; Klisovic, Rebecca B.; Gupta, Vikas; Andreasson, Bjorn; Mascarenhas, John; Wetzler, Meir; Vannucchi, Alessandro M.; Prchal, Josef T.; Najfeld, Vesna; Orazi, Attilio; Weinberg, Rona S.; Miller, Crystal; Barosi, Giovanni; Silverman, Lewis R.; Prosperini, Giuseppe; Marchioli, Roberto; Hoffman, Ronald.

In: Blood, Vol. 124, No. 7, 14.08.2014, p. 1183-1191.

Research output: Contribution to journalArticle

Rondelli, D, Goldberg, JD, Isola, L, Price, LS, Shore, TB, Boyer, M, Bacigalupo, A, Rambaldi, A, Scarano, M, Klisovic, RB, Gupta, V, Andreasson, B, Mascarenhas, J, Wetzler, M, Vannucchi, AM, Prchal, JT, Najfeld, V, Orazi, A, Weinberg, RS, Miller, C, Barosi, G, Silverman, LR, Prosperini, G, Marchioli, R & Hoffman, R 2014, 'MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis', Blood, vol. 124, no. 7, pp. 1183-1191. https://doi.org/10.1182/blood-2014-04-572545
Rondelli, Damiano ; Goldberg, Judith D. ; Isola, Luis ; Price, Leah S. ; Shore, Tsiporah B. ; Boyer, Michael ; Bacigalupo, Andrea ; Rambaldi, Alessandro ; Scarano, Marco ; Klisovic, Rebecca B. ; Gupta, Vikas ; Andreasson, Bjorn ; Mascarenhas, John ; Wetzler, Meir ; Vannucchi, Alessandro M. ; Prchal, Josef T. ; Najfeld, Vesna ; Orazi, Attilio ; Weinberg, Rona S. ; Miller, Crystal ; Barosi, Giovanni ; Silverman, Lewis R. ; Prosperini, Giuseppe ; Marchioli, Roberto ; Hoffman, Ronald. / MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis. In: Blood. 2014 ; Vol. 124, No. 7. pp. 1183-1191.
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AU - Shore, Tsiporah B.

AU - Boyer, Michael

AU - Bacigalupo, Andrea

AU - Rambaldi, Alessandro

AU - Scarano, Marco

AU - Klisovic, Rebecca B.

AU - Gupta, Vikas

AU - Andreasson, Bjorn

AU - Mascarenhas, John

AU - Wetzler, Meir

AU - Vannucchi, Alessandro M.

AU - Prchal, Josef T.

AU - Najfeld, Vesna

AU - Orazi, Attilio

AU - Weinberg, Rona S.

AU - Miller, Crystal

AU - Barosi, Giovanni

AU - Silverman, Lewis R.

AU - Prosperini, Giuseppe

AU - Marchioli, Roberto

AU - Hoffman, Ronald

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N2 - From 2007 to 2011, 66 patients with primary myelofibrosis or myelofibrosis (MF) preceded by essential thrombocythemia or polycythemia vera were enrolled into a prospective phase 2 clinical trial of reduced-intensity allogeneic hematopoietic stem cell transplantation (AHSCT), Myeloproliferative Disorder Research Consortium 101 trial. The study included patients with sibling donors (n = 32) receiving fludarabine/melphalan (FluMel) as a preparative regimen and patients with unrelated donors (n = 34) receiving conditioning with FluMel plus anti-thymocyte globulin (ATG). Patient characteristics in the 2 cohorts were similar. Engraftment occurred in 97% of siblings and 76% of unrelated transplants, whereas secondary graft failure occurred in 3% and 12%, respectively. With a median follow-up of 25 months for patients alive, the overall survival (OS) was 75% in the sibling group (median not reached) and 32% in the unrelated group (median OS: 6 months, 95% confidence interval [CI]: 3, 25) (hazard ratio 3.9, 95% CI: 1.8,8.9) (P <.001). Nonrelapse mortality was 22% in sibling and 59% in unrelated AHSCT. Survival correlated with type of donor, but not with the degree of histocompatibility match, age, or JAK2 V617F status. In patients with MF with sibling donors, AHSCT is an effective therapy, whereas AHSCT from unrelated donors with FluMel/ATG conditioning led to a high rate of graft failure and limited survival. This trial was registered at www.clinicaltrials.gov as #NCT00572897.

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