Lestang Elsa, Peterlin Pierre, Le Bris Yannick, Dubruille Viviane, Delaunay Jacques, Godon Catherine, Theisen Olivier, Blin Nicolas, Mahe Beatrice, Gastinne Thomas, Garnier Alice, Touzeau Cyrille, Voldoire Maud, Bene Marie C, Le Gouill Steven, Milpied Noel, Mohty Mohamad, Moreau Philippe, Guillaume Thierry, Chevallier Patrice
Hematology Department, CHU Hotel-Dieu, Nantes, France.
Hematology/Biology Department, CHU Hotel-Dieu, Nantes, France.
Eur J Haematol. 2017 Jul;99(1):60-69. doi: 10.1111/ejh.12891. Epub 2017 May 5.
The role of allogenic stem cell transplantation (ASCT) is still debated in myelofibrosis (MF).
A 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).
Five-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 JAK2 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.
Not all intermediate-2/high-risk DIPSS+ MF patients benefit from ASCT, especially since the introduction of JAK2 inhibitors.
异基因干细胞移植（ASCT）在骨髓纤维化（MF）中的作用仍存在争议。
进行一项回顾性分析，比较71例国际动态预后评分系统升级版（DIPSS+）中2期或高危的原发性（PMF）或继发性（SMF）骨髓纤维化患者的结局，这些患者有ASCT指征，最终接受了该手术（n = 34）或未接受（n = 37）。
两组的5年总生存率（OS）无统计学差异（同种异体移植组：52% vs未进行同种异体移植组：34%，P = 0.12）。然而，移植患者5年时进展为骨髓增生异常综合征或急性髓系白血病的比例显著更低（14% vs 50%，P = 0.01）。在单因素分析中，具有不良核型（75% vs 0%，P = 0.001）、SMF（71% vs 20%，P = 0.001）或高DIPSS+评分（46% vs 15%，P = 0.03）的移植患者与未移植患者相比，5年OS显著更高。JAK2负荷高（＞65%）的同种异体移植患者的5年OS也有更好的趋势（75% vs 8%，P = 0.07）。有趣的是，使用鲁索替尼可显著提高未进行ASCT患者的生存率。
并非所有国际动态预后评分系统升级版（DIPSS+）中2期/高危MF患者都能从ASCT中获益，尤其是自JAK2抑制剂问世以来。
