Allogeneic haematopoietic stem cell transplantation for myelofibrosis: a report of the Société Française de Greffe de Moelle et de Thérapie Cellulaire (SFGM‐TC)

Abstract

SummaryAllogeneic haematopoietic stem-cell transplantation (HSCT) is the only curative treatment for myelofibrosis. We report an analysis of the Société Française de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC) registry including patients with myelofibrosis transplanted between 1997 and 2008. Potential risk factors affecting engraftment, non-relapse mortality (NRM), overall survival (OS) and progression-free survival (PFS) were analysed. One hundred and forty-seven patients, aged 20-68 (median 53) years, diagnosed with primary (53%) or secondary myelofibrosis underwent HSCT; 59% of patients were transplanted from a matched sibling donor. The conditioning regimen was myeloablative in 31% of patients. Ninety percent of the patients engrafted. Factors affecting favourably engraftment were splenectomy before HSCT, human leucocyte antigen (HLA) matched sibling donor, peripheral stem cell use as source of stem cells and absence of pre-transplant thrombocytopenia. Four-year OS, PFS and NRM survival were 39% (95%confidence interval [CI]: 31-50), 32% (95%CI: 24-43) and 39% (95%CI 30-48), respectively. Multivariate analysis indicated that HLAidentical sibling donor, chronic phase disease and splenectomy in men had favourable impact on OS.

Allo-SCT for myelofibrosis: reversing the chronic phase in the JAK inhibitor era?

“…36,83 Interestingly, a French study noted a favorable survival for male patients who underwent pretransplant splenectomy, while a German study demonstrated a higher relapse rate in the splenectomized patients. 17,84 In addition, there appears to be no agreement as to the actual size of the spleen which might, or might not, serve as a trigger for pretransplant therapy. There is now an emerging consensus to avoid splenic irradiation or splenectomy immediately prior to transplant due to the intrinsic complications of radiation and splenectomy and thus the notion of reducing splenomegaly medically is indeed attractive.…”

Allogeneic hematopoietic stem cell transplantation for primary and secondary myelofibrosis

“…No significant difference was observed in OS and NRM between patients with standard myeloablative and reduced-intensity regimens, which is also compatible with the results of previous reports. 23 tensity regimens (46%) is increased compared with that of those prepared by myeloablative conditioning (15%), approaching significance (P 0.06). 17 Therefore, allo-SCT with reduced-intensity conditioning could be the potential treatment of choice for MF patients who are not eligible for myeloablative conditioning and should be carefully chosen for younger MF patients, considering its association with a higher risk of relapse compared with myeloablative conditioning.…”

“…In univariate analysis, lower platelet count ( 10 10 4 / l) at transplantation and previous transformation into AML were associated with poor OS; these findings are consistent with those of a previous report. 23 Furthermore, lower platelet count at transplantation was significantly associated with TTP, which suggested that patients with a lower platelet count at transplantation were more likely to suffer a relapse after allo-SCT.…”

“…20,23 PV, ET, and PMF share a common background to some extent; therefore, it may be difficult to distinguish PMF from sMF if a patient is referred to hospital with advanced disease. In this context, whether we should distinguish between PMF and sMF when we consider the indications for allo-SCT is open to discussion.…”

Allo-SCT for Philadelphia-negative myeloproliferative neoplasms in blast phase: a study from the Societe Française de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC)

“…Although most MPN evolve to AML through a myelofibrosis step, engraftment was not a major concern and could be achieved in the majority of patients (>90%). 9,18 Moreover, TRM was relatively moderate,~22%, suggesting the feasibility of the procedure. The main cause for the poor outcome in our study was a high incidence of relapse.…”