Safety and outcome of allogeneic stem cell transplantation in myelofibrosis

• Published in: European journal of haematology Vol. 96; no. 3; pp. 222 - 228
• Main Authors: Markiewicz, Miroslaw, Dzierzak Mietla, Monika, Wieczorkiewicz, Agata, Mizia, Sylwia, Helbig, Grzegorz, Kopera, Malgorzata, Bialas, Krzysztof, Rybicka, Malwina, Matyja, Mariusz, Koclega, Anna, Sedlak, Lech, Oleksy, Tomasz, Raman, Sundar, Kyrcz-Krzemien, Slawomira
• Format: Journal Article
• Language: English
• Published: England Blackwell Publishing Ltd 01.03.2016
• Subjects: Adult; Female; Graft vs Host Disease - etiology; Hematopoietic Stem Cell Transplantation - adverse effects; Humans; Janus Kinase 2 - genetics; Male; Middle Aged; Mutation; myelofibrosis; Primary Myelofibrosis - diagnosis; Primary Myelofibrosis - genetics; Primary Myelofibrosis - mortality; Primary Myelofibrosis - therapy; prognostic factors; Tissue Donors; transfusion; transplantation; Transplantation, Homologous; Treatment Outcome; Young Adult; myelofibrosis; prognostic factors; transfusion; transplantation
• ISSN: 0902-4441; 1600-0609
• DOI: 10.1111/ejh.12572

Objectives We evaluated the safety and outcome of allo‐HSCTs in myelofibrosis (MF). Methods A total of 27 patients with primary (n = 20) or secondary (n = 7) MF, aged 51 (21–63) yr, transplanted from HLA‐matched related (59%) or unrelated (41%) donors were analyzed. Conditioning was reduced in 26 and myeloablative in one patient; and ATG was used in 25. Sources of stem cells were as follows: peripheral blood (21), bone marrow (4) or both (2). Results Prognostic factors that adversely affected overall survival (OS) in the multivariate analysis were as follows: recipient age >45 yr (HR = 10.55, P = 0.025) and unrelated donor (HR=3.73, P = 0.026). Post‐transplant transfusion dependence adversely affected OS in the univariate analysis: dependence from either both RBCs and platelets (HR = 33.26, P = 0.001) or from either of them (HR = 10.53, P = 0.043). Of 16 JAK2V617F‐positive patients evaluated post‐transplant, it was eradicated in 69% and decreased in 25%. Acute GVHD III‐IV developed in 19% and extensive chronic GVHD in 26% of patients; the relapse in four patients was treated with second allo‐HSCT. Spleen decreased in all evaluated patients (n = 24). Fibrotic changes improved or disappeared in 80% of evaluated patients (n = 10). Conclusions Allo‐HSCT may prolong survival, provide disease regression and improve quality of life in MF, especially in patients ≤45 yr transplanted from matched related donors. Achieving transfusion independence post‐transplant indicates the favorable outcome.