Allogeneic stem cell transplantation in patients with myelofibrosis harboring the MPL mutation
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Allogeneic stem cell transplantation in patients with myelofibrosis harboring the MPL mutation. / Mannina, Daniele; Gagelmann, Nico; Badbaran, Anita; Ditschkowski, Markus; Bogdanov, Rashit; Robin, Marie; Cassinat, Bruno; Heuser, Michael; Shahswar, Rabia; Thol, Felicitas; Beelen, Dietrich; Kröger, Nicolaus.
in: EUR J HAEMATOL, Jahrgang 103, Nr. 6, 12.2019, S. 552-557.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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T1 - Allogeneic stem cell transplantation in patients with myelofibrosis harboring the MPL mutation
AU - Mannina, Daniele
AU - Gagelmann, Nico
AU - Badbaran, Anita
AU - Ditschkowski, Markus
AU - Bogdanov, Rashit
AU - Robin, Marie
AU - Cassinat, Bruno
AU - Heuser, Michael
AU - Shahswar, Rabia
AU - Thol, Felicitas
AU - Beelen, Dietrich
AU - Kröger, Nicolaus
N1 - © 2019 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
PY - 2019/12
Y1 - 2019/12
N2 - 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.
AB - 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.
U2 - 10.1111/ejh.13318
DO - 10.1111/ejh.13318
M3 - SCORING: Journal article
C2 - 31446640
VL - 103
SP - 552
EP - 557
JO - EUR J HAEMATOL
JF - EUR J HAEMATOL
SN - 0902-4441
IS - 6
ER -
