Is allogeneic stem cell transplantation for myelofibrosis still indicated at the time of molecular markers and JAK inhibitors era?

• DOI: 10.1111/ejh.12891
• Publication Date: 2017-04-03T20:07:33Z
• Authors: Elsa Lestang; Pierre Peterlin; Yannick Le Bris; Viviane Dubruille; Jacques Delaunay; Catherine Godon; Olivier Theisen; Nicolas Blin; Beatrice Mahe; Thomas Gastinne; Alice Garnier; Cyrille Touzeau; Maud Voldoire; Marie C. Bene; Steven Le Gouill; Noel Milpied; Mohamad Mohty; Philippe Moreau; Thierry Guillaume; Patrice Chevallier
• Keywords: Adult; Male; Hematopoietic Stem Cell Transplantation; Janus Kinase 2; Middle Aged; Prognosis; 03 medical and health sciences; Treatment Outcome; 0302 clinical medicine; Primary Myelofibrosis; Mutation; Humans; Janus Kinase Inhibitors; Transplantation, Homologous; Female; Molecular Targeted Therapy; Biomarkers; Aged; Retrospective Studies

AbstractObjectiveThe role of allogenic stem cell transplantation (ASCT) is still debated in myelofibrosis (MF).MethodsA retrospective analyzed was performed to compare the outcome of 71 patients with intermediate‐2 or high‐risk Dynamic International Prognosis Scoring System+ (DIPSS+) primary (PMF) or secondary (SMF) myelofibrosis with an indication of ASCT as they ultimately underwent the procedure (n=34) or not (n=37).ResultsFive‐year overall survival (OS) was not statistically different between both groups (allograft: 52% vs no allograft: 34%, P=.12). However, progression to myelodysplastic syndrome or acute myeloid leukemia at 5 years was significantly lower in transplanted patients (14% vs 50%, P=.01). In univariate analysis, 5‐year OS was significantly higher for transplanted vs non‐transplanted patients with unfavorable karyotype (75% vs 0%, P=.001), SMF (71% vs 20%, P=.001) or high DIPSS+ score (46% vs 15%, P=.03). There was also a trend for better 5‐year OS in allografted patients with high JAK2V617F burden (>65%) (75% vs 8%, P=.07). Interestingly, the survival of patients who did not proceed to ASCT was dramatically increased by the use of ruxolitinib.ConclusionsNot all intermediate‐2/high‐risk DIPSS+ MF patients benefit from ASCT, especially since the introduction of JAK2 inhibitors.