Allogeneic stem cell transplantation in patients with myelofibrosis harboring the MPL mutation.

Abstract:

INTRODUCTION:Primary and post-ET/PV myelofibrosis are myeloproliferative neoplasms harboring in most cases driving mutations in JAK2, CALR or MPL, and a variable number of additional mutations in other genes. Molecular analysis represents a powerful tool to guide prognosis and clinical management. Only about 10% of patients with myelofibrosis harbor alterations in MPL gene. No data are available about the transplantation outcome in the specific MPL-mutated group. PATIENTS:We collected the data of 18 myelofibrosis patients(primary: 14; post-ET: 4) transplanted in 4 EBMT centers (Hamburg, Paris, Essen, and Hannover) between 2005 and 2016. RESULTS:Before the transplant, we explored the molecular profile by NGS and reported the frequency of mutations occurring in a panel of genes including JAK2, MPL, CALR, U2AF1, SRSF2, SF3B1, ASXL1, IDH1, IDH2, CBL, DNMT3A, TET2, EZH2, TP53, IKZF1, NRAS, KRAS, FLT3, SH2B3, and RUNX1. The 1-year transplant-related mortality was 16.5%, 5-years overall survival and 5-y relapse-free survival 83.5%. The only relapse occurred in a patient who harbored mutations in both ASXL1 and EZH2 genes. CONCLUSION:These retrospective data suggest that MPL-mutated myelofibrosis patients have a favorable outcome after allogeneic transplantation with very low rate of disease relapse (5.5%) in comparison with the available historical controls regarding myelofibrosis in all.

journal_name

Eur J Haematol

authors

Mannina D,Gagelmann N,Badbaran A,Ditschkowski M,Bogdanov R,Robin M,Cassinat B,Heuser M,Shahswar R,Thol F,Beelen D,Kröger N

doi

10.1111/ejh.13318

subject

Has Abstract

pub_date

2019-12-01 00:00:00

pages

552-557

issue

6

eissn

0902-4441

issn

1600-0609

journal_volume

103

pub_type

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