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

MPL mutasyonu taşıyan miyelofibroz hastalarında allojeneik kök hücre nakli.

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ID: 63776
2019
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Abstract
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.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.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.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.
Primer ve post-ET/PV miyelofibrozis; çoğu olguda JAK2, CALR veya MPL genlerinde sürücü mutasyonlar ve diğer genlerde değişken sayıda ek mutasyon barındıran miyeloproliferatif neoplazilerdir. Moleküler analiz, prognoz ve klinik yönetime rehberlik etmede güçlü bir araçtır. Miyelofibrozisli hastaların yalnızca yaklaşık %10'u MPL geninde alterasyon taşımaktadır. Spesifik olarak MPL mutasyonlu gruptaki transplantasyon sonuçlarına ilişkin veri bulunmamaktadır. 2005 ile 2016 yılları arasında 4 EBMT merkezinde (Hamburg, Paris, Essen ve Hannover) nakil yapılan 18 miyelofibrozis hastasının (primer: 14; post-ET: 4) verilerini topladık. Transplantasyon öncesinde moleküler profili NGS ile inceledik ve JAK2, MPL, CALR, U2AF1, SRSF2, SF3B1, ASXL1, IDH1, IDH2, CBL, DNMT3A, TET2, EZH2, TP53, IKZF1, NRAS, KRAS, FLT3, SH2B3 ve RUNX1 genlerini içeren bir panelde meydana gelen mutasyon sıklıklarını bildirdik. 1 yıllık transplantasyon ilişkili mortalite %16,5, 5 yıllık genel sağkalım ve 5 yıllık nükssüz sağkalım ise %83,5 olarak saptandı. Gözlenen tek nüks, hem ASXL1 hem de EZH2 genlerinde mutasyon taşıyan bir hastada meydana geldi. Bu retrospektif veriler, genel miyelofibrozise ilişkin mevcut tarihsel kontrollerle karşılaştırıldığında, MPL mutasyonlu miyelofibrozis hastalarının allojenik transplantasyon sonrasında oldukça düşük hastalık nüksü oranı (%5,5) ile olumlu bir klinik sonlanıma sahip olduğunu düşündürmektedir.
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mannina2019allogeneiceuropean Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Mannina, Daniele;Gagelmann, Nico;Badbaran, Anita;Ditschkowski, Markus;Bogdanov, Rashit;Robin, Marie;Cassinat, Bruno;Heuser, Michael;Shahswar, Rabia;Thol, Felicitas;Beelen, Dietrich;Kröger, Nicolaus;
Journal European journal of haematology
Year 2019
DOI
10.1111/ejh.13318
URL
Keywords
transplantation bone marrow transplantation molecular biology of myelopoiesis myelofibrosis with myeloid metaplasia

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