Reduced intensity allogeneic stem cell transplantation for younger patients with myelofibrosis. (15th May 2019)

• Record Type: Journal Article
• Authors: Mannina, Daniele; Zabelina, Tatjana; Wolschke, Christine; Heinzelmann, Marion; Triviai, Ioanna; Christopeit, Maximilian; Badbaran, Anita; Bonmann, Stefan; von Pein, Ute‐Marie; Janson, Dietlinde; Ayuk, Francis; Kröger, Nicolaus
• Is Part Of: British journal of haematology. Volume 186:Number 3(2019)
• Page Start: 484
• Page End: 489
• Publication Date: 2019-05-15
• Subjects: myelofibrosis -- reduced‐intensity conditioning -- allogeneic stem cell transplantation -- younger age; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.15952
• Languages: English
• ISSNs: 0007-1048

Abstract

Summary: Allogeneic stem cell transplantation (alloSCT) is a curative procedure for myelofibrosis. Elderly people are mainly affected, limiting the feasibility of myeloablative regimens. The introduction of reduced‐intensity conditioning (RIC) made alloSCT feasible for older patients. Nevertheless, the incidence of myelofibrosis is not negligible in young patients, who are theoretically able to tolerate high‐intensity therapy. Very few data are available about the efficacy of RIC‐alloSCT in younger myelofibrosis patients. This study included 56 transplanted patients aged <55 years. Only 30% had a human leucocyte antigen (HLA)‐matched sibling donor, the others were transplanted from a fully‐matched (36%) or partially‐matched (34%) unrelated donor. All transplants were conditioned according the European Society for Blood and Marrow Transplantation protocol: busulfan‐fludarabine + anti‐thymocyte globulin, followed by ciclosporin and mycophenolate. One patient experienced primary graft failure. Incidence of graft‐versus‐host disease grade II‐IV was 44% (grade III/IV 23%). One‐year non‐relapse mortality was 7% and the 5‐year cumulative incidence of relapse was 19%. After a median follow‐up of 8·6 years, the estimated 5‐year progression‐free survival and overall survival (OS) was 68% and 82%, respectively. Patients with fully‐matched donor had a 5‐year OS of 92%, in contrast to 68% for those with a mismatched donor ( P = 0·03). The most important outcome‐determining factor is donor HLA‐matching. In conclusion, RIC‐alloSCT ensures optimal engraftment and low relapse rate in younger myelofibrosis patients, enabling the possibility of cure in this group.