Comparison of Outcomes of Allogeneic Transplantation for Primary Myelofibrosis among Hematopoietic Stem Cell Source Groups

Murata, Makoto; Takenaka, Katsuto; Uchida, Naoyuki; Ozawa, Yukiyasu; Ohashi, Kazuteru; Kim, Sung-Won; Ikegame, Kazuhiro; Kanda, Yoshinobu; Kobayashi, Hikaru; Ishikawa, Jun; Ago, Hiroatsu; Hirokawa, Makoto; Fukuda, Takahiro; Atsuta, Yoshiko; Kondo, Takeshi

doi:10.1016/j.bbmt.2019.02.019

Cited by 21 publications

(19 citation statements)

References 49 publications

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“…Takagi et al evaluated 14 UCB transplantation cases for myelofibrosis, including 11 secondary myelofibrosis from acute myeloid leukemia, and reported a 4-year OS of 29% ( 20 ). The recent nationwide retrospective study of HSCT for Japanese patients with primary myelofibrosis reported that the 1- and 4-year OS rates in UCB transplantation (n=29) were 48% and 27%, respectively, and that NRM was significantly higher in UCB transplantation than in HLA-matched related donor BM transplantation ( 21 ). Further analyses with more patients are required to evaluate the differences in the transplant outcomes for secondary myelofibrosis between UCB transplantation and related or unrelated donor transplantation.…”

Section: Discussionmentioning

confidence: 99%

Allogeneic Hematopoietic Stem Cell Transplantation for Post-essential Thrombocythemia and Post-polycythemia Vera Myelofibrosis

et al. 2020

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Objective Little information is available about the outcome of allogeneic hematopoietic stem cell transplantation (HSCT) for patients with secondary myelofibrosis from essential thrombocythemia (ET) and polycythemia vera (PV). A nationwide retrospective study of the outcome of HSCT for post-ET and post-PV myelofibrosis was conducted in Japan. Patients and Methods Clinical data for patients with post-ET (n=29) and post-PV (n=9) myelofibrosis who had received first allogeneic HSCT were extracted from the Transplant Registry Unified Management Program, which is a registry of the outcomes of HSCT in Japan. Results Five patients died without neutrophil recovery within 60 days after transplantation. The incidence of neutrophil recovery was significantly lower in umbilical cord blood (UCB) transplantation than in related donor transplantation (40% vs. 92%, p=0.010). The 1-year non-relapse mortality for post-ET and post-PV myelofibrosis was 35% and 27%, respectively (p=0.972). No patient or transplantation characteristics were associated with non-relapse mortality. The 4-year overall survival for post-ET and post-PV myelofibrosis was 46% and 65%, respectively (p=0.362). A univariate analysis identified UCB transplantation (vs. related donor, p=0.017) and ≥10 times red blood cell transfusions before transplantation (vs. <10 times, p=0.037) as predictive of a lower overall survival. Conclusion Allogeneic HSCT provides a long-term survival for at least some patients with post-ET and post-PV myelofibrosis. Further studies with more patients are required to determine the best alternative donor.

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Section: Discussionmentioning

confidence: 99%

Allogeneic Hematopoietic Stem Cell Transplantation for Post-essential Thrombocythemia and Post-polycythemia Vera Myelofibrosis

et al. 2020

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“…Compared with MSD BM transplantation, the incidence of neutrophil and platelet recovery after unrelated UCB transplantation were significantly lower, and NRM at 1 year was higher (41% versus 16%, p = 0.046). 72 Therefore, careful management is required for patients who proceed to UCB AHSCT, especially in the early period.…”

Section: Stem Cell Sourcementioning

confidence: 99%

Allogeneic hematopoietic stem-cell transplantation for myelofibrosis

Zhang

Yang

Feng

2020

Therapeutic Advances in Hematology

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Myelofibrosis is one of the Philadelphia chromosome (Ph)-negative myeloproliferative neoplasms with heterogeneous clinical course. Though many treatment options, including Janus kinase (JAK) inhibitors, have provided clinical benefits and improved survival, allogeneic hematopoietic stem-cell transplantation (AHSCT) remains the only potentially curative therapy. Considering the significant transplant-related morbidity and mortality, it is crucial to decide who to proceed to AHSCT, and when. In this review, we discuss recent updates in patient selection, prior splenectomy, conditioning regimen, donor type, molecular mutation, and other factors affecting AHSCT outcomes. Relapse is a major cause of treatment failure; we also describe recent data on minimal residual disease monitoring and management of relapse. In addition, emerging studies have reported pretransplant therapy with ruxolitinib for myelofibrosis showing favorable results, and further research is needed to explore its use in the post-transplant setting.

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“…Raj et al reported on 56 MF patients transplanted between 2009 and 2015 from a family mismatched donor (i.e., relatives with ≥2 Ag HLA different from recipient), with a 2-year overall survival rate of 56% but a rather high non-relapse mortality (NRM) of 38% at 2 years [55]. In a recent retrospective review, NRM and 4-year OS rates were similar in patients receiving their first transplant from HLA-matched related donor peripheral blood stem cells or from HLA-MUD bone marrow, while long-term survival was inferior in patients transplanted from umbilical cord blood, partly due to higher NRM [56]. Information about haploidentical related donor in MF is still scarce; in a comparison between HLA-identical sibling and "alternative" donors, including 20 haploidentical, transplanted in the years 2011-2014, Bregante et al found similar survival rates in the two cohorts (72% and 69%, respectively) [57].…”

Section: Pretransplant Evaluation: Prognostic Scores and Consideratiomentioning

confidence: 99%

The role of allogeneic stem-cell transplant in myelofibrosis in the era of JAK inhibitors: a case-based review

Tiribelli

Palandri

Sant’Antonio

et al. 2019

Bone Marrow Transplant

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Allogeneic hematopoietic stem-cell transplantation (HSCT) is, at present, the only potentially curative therapy for myelofibrosis (MF). Despite many improvements, outcomes of HSCT are still burdened by substantial morbidity and high transplant-related mortality. Allogeneic transplant is generally considered in intermediate-2 and high-risk patients aged <70 years, but the optimal selection of patients and timing of the procedure remains under debate, as does as the role of JAK inhibitors in candidates for HSCT. Starting from a real-life clinical case scenario, herein we examine some of the crucial issues of HSCT for MF in light of recent refinements on MF risk stratification, data on the use of ruxolitinib before and after transplant and findings on the impact of different conditioning regimens and donor selection.

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Comparison of Outcomes of Allogeneic Transplantation for Primary Myelofibrosis among Hematopoietic Stem Cell Source Groups

Cited by 21 publications

References 49 publications

Allogeneic Hematopoietic Stem Cell Transplantation for Post-essential Thrombocythemia and Post-polycythemia Vera Myelofibrosis

Allogeneic Hematopoietic Stem Cell Transplantation for Post-essential Thrombocythemia and Post-polycythemia Vera Myelofibrosis

Allogeneic hematopoietic stem-cell transplantation for myelofibrosis

The role of allogeneic stem-cell transplant in myelofibrosis in the era of JAK inhibitors: a case-based review

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