State-of-the-art review: allogeneic stem cell transplantation for myelofibrosis in 2019

Advances in understanding the pathogenesis and molecular landscape of myelofibrosis have occurred over the last decade. Treating physicians now have access to an ever-evolving armamentarium of novel agents to treat patients, although allogeneic hematopoietic stem cell transplantation remains the onl...

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Published inHaematologica (Roma) Vol. 104; no. 4; pp. 659 - 668
Main Authors McLornan, Donal P, Yakoub-Agha, Ibrahim, Robin, Marie, Chalandon, Yves, Harrison, Claire N, Kroger, Nicolaus
Format Journal Article
LanguageEnglish
Published Italy Ferrata Storti Foundation 01.04.2019
SeriesHaematologica
Subjects
Disease-Free Survival
Hematopoietic Stem Cell Transplantation
Humans
Life Sciences
Primary Myelofibrosis - blood
Primary Myelofibrosis - genetics
Primary Myelofibrosis - pathology
Primary Myelofibrosis - therapy
Recurrence
Review
Survival Rate
Transplantation Conditioning
Transplantation, Homologous
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Summary:Advances in understanding the pathogenesis and molecular landscape of myelofibrosis have occurred over the last decade. Treating physicians now have access to an ever-evolving armamentarium of novel agents to treat patients, although allogeneic hematopoietic stem cell transplantation remains the only curative approach. Improvements in donor selection, conditioning regimens, disease monitoring and supportive care have led to augmented survival after transplantation. Nowadays, there are comprehensive guidelines concerning allogeneic hematopoietic stem cell transplantation for patients with myelofibrosis. However, it commonly remains difficult for both physicians and patients alike to weigh up the risk-benefit ratio of transplantation given the inherent heterogeneity regarding both clinical course and therapeutic response. In this timely review, we provide an up-to-date synopsis of current transplantation recommendations, discuss usage of JAK inhibitors before and after transplantation, examine donor selection and compare conditioning platforms. Moreover, we discuss emerging data concerning the impact of the myelofibrosis mutational landscape on transplantation outcome, peritransplant management of splenomegaly, poor graft function and prevention/management of relapse.
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CNH and NK: joint senior authors
ISSN:0390-6078
1592-8721
DOI:10.3324/haematol.2018.206151