Hematology Department, Hôpital Saint Antoine, Service d'Hématologie et Thérapie Cellulaire, Paris, France.
Hematology Department, Federico II University, Naples, Italy.
Bone Marrow Transplant. 2019 Sep;54(9):1499-1510. doi: 10.1038/s41409-019-0459-7. Epub 2019 Feb 4.
Refractory or relapsed acute myeloid leukemia (R/R-AML) has poor prognosis. Allogeneic hematopoietic stem-cell transplantation (HSCT) may provide cure in this scenario. We compared outcomes of HSCT from HLA-identical (HLA-id, n = 1654) sibling or haploidentical (Haplo, n = 389) donors in patients with R/R-AML, performed during the period 2007-2015. The Haplo group included patients receiving an unmanipulated graft (post-transplant cyclophosphamide, n = 278; in vivo T-cell depletion, n = 95; or both, n = 16). Median age at HSCT was 52 (range 18-74) years. Median follow-up was 16 and 22 months for HLA-id sibling and Haplo recipients, respectively (p = 0.11). Compared to MSD, Haplo HSCT were performed more recently (2013 vs 2011, p < 0.01), at longer interval from diagnosis (7 vs 5 months, p < 0.01), more frequently using bone marrow as stem cell source (47% vs 8%, p < 0.01) and with a reduced intensity conditioning regimen (50% vs 43%, p = 0.03). Engraftment was higher (93% vs 83%, p < 0.01) in HLA-id sibling. In multivariate analysis, Haplo HSCT was associated with lower GVHD/relapse-free survival, inferior LFS and OS and higher NRM, mainly due to a higher rate of infections (41% vs 25%, p < 0.01). For R/R-AML, HLA-id sibling donors remain the gold standard, when available, due to higher mortality in Haplo without significant gain in disease control.
难治或复发急性髓系白血病(R/R-AML)预后差。异基因造血干细胞移植(HSCT)可能为此提供治愈机会。我们比较了 2007 年至 2015 年间接受 HLA 匹配(HLA-id,n=1654)同胞或单倍体(Haplo,n=389)供者 HSCT 的 R/R-AML 患者的结果。Haplo 组包括接受未处理移植物(移植后环磷酰胺,n=278;体内 T 细胞耗竭,n=95;或两者均有,n=16)的患者。HSCT 时的中位年龄为 52 岁(18-74 岁)。HLA-id 同胞和 Haplo 受体的中位随访时间分别为 16 个月和 22 个月(p=0.11)。与 MSD 相比,Haplo HSCT 进行得更近(2013 年比 2011 年,p<0.01),从诊断到移植的时间间隔更长(7 个月比 5 个月,p<0.01),更常使用骨髓作为干细胞来源(47%比 8%,p<0.01),且强度更低的预处理方案(50%比 43%,p=0.03)。同胞 HLA-id 的植入率更高(93%比 83%,p<0.01)。多变量分析显示,Haplo HSCT 与较低的 GVHD/无复发存活率、较低的 LFS 和 OS 以及较高的 NRM 相关,主要是由于感染率较高(41%比 25%,p<0.01)。对于 R/R-AML,在有条件的情况下,HLA-id 同胞供者仍然是金标准,因为 Haplo 的死亡率更高,而疾病控制没有明显改善。