Daniele Mannina1,2, Nico Gagelmann1, Anita Badbaran1, Markus Ditschkowski3, Rashit Bogdanov4, Marie Robin4, Bruno Cassinat4, Michael Heuser5, Rabia Shahswar5, Felicitas Thol5, Dietrich Beelen3, Nicolaus Kröger1. 1. Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany. 2. Department of Hematology and Bone Marrow Transplantation, San Raffaele Hospital, Milan, Italy. 3. Department of Bone Marrow Transplantation, West German Cancer Center, University Hospital of Essen, Essen, Germany. 4. Department of Hematology, Saint-Louis Hospital, APHP. Paris, Paris, France. 5. Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.
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.
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 myelofibrosispatients(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 myelofibrosispatients 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.
Authors: Jan Philipp Bewersdorf; Amar H Sheth; Shaurey Vetsa; Alyssa Grimshaw; Smith Giri; Nikolai A Podoltsev; Lohith Gowda; Roni Tamari; Martin S Tallman; Raajit K Rampal; Amer M Zeidan; Maximilian Stahl Journal: Transplant Cell Ther Date: 2021-05-28
Authors: Kate Downes; Pascal Borry; Katrin Ericson; Keith Gomez; Andreas Greinacher; Michele Lambert; Eva Leinoe; Patrizia Noris; Chris Van Geet; Kathleen Freson Journal: J Thromb Haemost Date: 2020-10 Impact factor: 5.824