Shanavas M, Messner H A, Atenafu E G, Kim D H, Kuruvilla J, Lipton J H, Uhm J, Seftel M, Alam N, Gupta V
Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Ontario, Canada.
Department of Biostatistics, Princess Margaret Cancer Center, Toronto, Ontario, Canada.
Bone Marrow Transplant. 2014 Sep;49(9):1162-9. doi: 10.1038/bmt.2014.131. Epub 2014 Jun 30.
Graft failure is one of the major barriers to the success of allogeneic hematopoietic cell transplantation (HCT) in myelofibrosis (MF). We report our institutional experience with 27 MF patients who underwent HCT using fludarabine-, intravenous BU- and low-dose total body irradiation (FBT)-based reduced-intensity (n=20) or full-intensity (n=7) conditioning regimens. Eight patients had prior exposure to JAK1/2 inhibitor therapy; six patients received JAK1/2 inhibitors leading on to HCT and two patients received transplant at the failure of JAK1/2 inhibitor therapy. No adverse impact of JAK1/2 inhibitor therapy was observed on early post-transplant outcomes. All evaluable patients had neutrophil recovery, and no primary graft failure was observed. Cumulative incidence of grades II-IV acute GVHD at day 100 was 48% (95% confidence interval (CI), 29-67%) and chronic GVHD at 2 years was 66% (95% CI, 49-84%). Cumulative incidences of nonrelapse mortality (NRM), relapse and probability of OS at 2 years were: 43% (95% CI, 12-74%), 10% (95% CI, 0-39%) and 56% (95% CI, 28-77%), respectively. FBT-based conditioning regimen has a favorable impact on engraftment; however, further efforts are required to reduce NRM.
移植物失败是骨髓纤维化（MF）患者接受异基因造血细胞移植（HCT）取得成功的主要障碍之一。我们报告了本机构对27例MF患者的治疗经验，这些患者接受了基于氟达拉滨、静脉注射白消安和低剂量全身照射（FBT）的减低强度（n = 20）或高强度（n = 7）预处理方案进行HCT。8例患者曾接受过JAK1/2抑制剂治疗；6例患者在接受JAK1/2抑制剂治疗后进行了HCT，2例患者在JAK1/2抑制剂治疗失败后接受了移植。未观察到JAK1/2抑制剂治疗对移植后早期结局有不良影响。所有可评估患者均实现中性粒细胞恢复，未观察到原发性移植物失败。移植后第100天II-IV级急性移植物抗宿主病（GVHD）的累积发生率为48%（95%置信区间（CI），29-67%），2年时慢性GVHD的累积发生率为66%（95%CI，49-84%）。2年时非复发死亡率（NRM）、复发率和总生存率（OS）的累积发生率分别为：43%（95%CI，12-74%）、10%（95%CI，0-39%）和56%（95%CI，28-77%）。基于FBT的预处理方案对植入有有利影响；然而，需要进一步努力降低NRM。
