University College London NHS Foundation Trust, London, UK.
EBMT statistical Unit, Leiden, The Netherlands.
Blood Cancer J. 2022 Sep 28;12(9):140. doi: 10.1038/s41408-022-00729-y.
Myelodysplastic syndromes (MDS) are the second common indication for an Allo-HCT. We compared the outcomes of 1414 matched sibling (MSD) with 415 haplo-identical donors (HD) transplanted with post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis between 2014 and 2017. The median age at transplant with MSD was 58 and 61 years for HD. The median time to neutrophil engraftment was longer for HD being 20 vs 16 days for MSD (p < 0.001). Two-year overall survival (OS) and PFS (progression free survival) with MSD were significantly better at 58% compared with 50%, p ≤ 0.001, and 51% vs 47%, p = 0.029, with a HD. Relapse at 2 years was lower with a HD 23% than with MSD 29% (p = 0.016). Non relapse mortality (NRM) was higher with HD in the first 6 months post-transplant [HR 2.59 (1.5-4.48) p < 0.001] and was also higher at 2 years being 30% for HD and 20% for MSD, p ≤ 0.001. The incidence of acute GVHD grade II-IV and III-IV at 100 days was comparable for MSD and HD, however, chronic GVHD at 2 years was significantly higher with MSD being 44% vs 32% for HD (p < 0.001). After multivariable analysis, OS and primary graft failure were significantly worse for HD particularly before 6 months [HR 1.93(1.24-3.0)], and HR [3.5(1.5-8.1)]. The median age of HD 37 (IQR 30-47) years was significantly lower than sibling donors 56 (IQR 49-62 years) p < 0.001. However, there was no effect on NRM, relapse or PFS. This data set suggests that a MSD donor remains the preferred choice in MDS over a haplo donor. Transplants with haploidentical donors result in satisfactory long-term outcome, justifying it's use when no better donor is available.
骨髓增生异常综合征（MDS）是同种异体造血干细胞移植（Allo-HCT）的第二大适应证。我们比较了 2014 年至 2017 年间，1414 例匹配同胞（MSD）与 415 例单倍体相合供者（HD）接受移植后环磷酰胺（PTCy）作为移植物抗宿主病（GVHD）预防的结果。MSD 患者的中位年龄为 58 岁，HD 患者为 61 岁。HD 患者中性粒细胞植入的中位时间较长，为 20 天，而 MSD 患者为 16 天（p<0.001）。2 年总生存（OS）和无进展生存（PFS）MSD 分别为 58%和 51%，明显优于 HD 的 50%和 47%（p≤0.001），并且在 HD 中，2 年时复发率为 23%，而 MSD 为 29%（p=0.016）。HD 患者在移植后 6 个月内非复发死亡率（NRM）较高[HR 2.59（1.5-4.48），p<0.001]，2 年时 NRM 也较高，为 30%，而 MSD 为 20%，p≤0.001。100 天时 MSD 和 HD 的急性 GVHD Ⅱ-Ⅳ级和Ⅲ-Ⅳ级发生率相似，但 2 年时慢性 GVHD 明显较高，MSD 为 44%，而 HD 为 32%（p<0.001）。多变量分析后，OS 和原发性移植物失败在 HD 患者中明显更差，尤其是在 6 个月之前[HR 1.93（1.24-3.0）]，而 HR [3.5（1.5-8.1）]。HD 的中位年龄为 37 岁（IQR 30-47），明显低于同胞供者的 56 岁（IQR 49-62 岁）（p<0.001）。然而，这对 NRM、复发或 PFS 没有影响。本数据集表明，在 MDS 中，MSD 供者仍然是首选，而不是单倍体供者。使用单倍体相合供者进行移植可获得满意的长期结果，当没有更好的供者时，可考虑使用。
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