Anthony Nolan Research Institute, London, UK.
Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
Bone Marrow Transplant. 2020 Feb;55(2):441-451. doi: 10.1038/s41409-019-0680-4. Epub 2019 Sep 25.
This retrospective study explored the incidence of autoimmune cytopenia (AIC) in 530 paediatric and adult patients with acquired aplastic anaemia (aAA) who underwent first allogeneic HSCT between 2002 and 2012. AIC was a rare complication with a cumulative incidence of AIC at 1, 3, 5 and 10 years post HSCT of 2.5% (1.2-3.9 95% CI), 4.4% (2.6-6.2 95% CI), 4.6% (2.8-6.5 95% CI) and 5.1% (3.1-7.2 95% CI). Overall survival at 5 years after diagnosis of AIC was 85.9% (71-100 95% CI). Twenty-five patients were diagnosed with AIC at a median of 10.6 (2.6-91.5) months post HSCT. Eight (32%) patients were diagnosed with immune thrombocytopenia (ITP), seven (28%) with autoimmune haemolytic anaemia (AIHA), seven (24%) with Evans syndrome and four (16%) with autoimmune neutropenia (AIN). Treatment strategies were heterogeneous. Complete responses were seen in 12 of 25 patients, with death in three patients. In multivariable Cox analysis of a subgroup of 475 patients, peripheral blood stem cell (PBSC) transplant was associated with higher risk of AIC compared with bone marrow (BM) when conditioning regimens contained fludarabine and/or alemtuzumab (2.81 [1.06-7.49 95% CI]; p = 0.038), or anti-thymocyte globulin (ATG) (2.86 [1.11-7.37 95% CI]; p = 0.029). Myeloablative conditioning was associated with a lower risk of AIC compared with reduced intensity conditioning (RIC) in fludarabine and/or alemtuzumab (0.34 [0.12-0.98 95% CI]; p = 0.046) and ATG containing regimens (0.34 [0.12-0.95 95% CI]; p = 0.04). These findings provide clinically useful information regarding the incidence of a rare and potentially life-threatening complication of allogeneic HSCT for aAA, and further support for BM as the preferred stem cell source for transplant of patients with aAA.
这项回顾性研究探讨了 530 名在 2002 年至 2012 年间接受首次同种异体 HSCT 的获得性再生障碍性贫血（aAA）儿科和成年患者中自身免疫性细胞减少症（AIC）的发生率。AIC 是一种罕见的并发症，HSCT 后 1、3、5 和 10 年的 AIC 累积发生率分别为 2.5%（1.2-3.9 95%CI）、4.4%（2.6-6.2 95%CI）、4.6%（2.8-6.5 95%CI）和 5.1%（3.1-7.2 95%CI）。AIC 诊断后 5 年的总生存率为 85.9%（71-100 95%CI）。25 名患者在 HSCT 后中位 10.6（2.6-91.5）个月时被诊断为 AIC。8 名（32%）患者被诊断为免疫性血小板减少症（ITP），7 名（28%）为自身免疫性溶血性贫血（AIHA），7 名（24%）为 Evans 综合征，4 名（16%）为自身免疫性中性粒细胞减少症（AIN）。治疗策略存在异质性。25 名患者中有 12 名完全缓解，3 名患者死亡。在 475 名患者的亚组的多变量 Cox 分析中，与骨髓（BM）相比，当条件性治疗方案包含氟达拉滨和/或阿仑单抗时，外周血干细胞（PBSC）移植与 AIC 风险较高相关（2.81[1.06-7.49 95%CI]；p=0.038），或抗胸腺细胞球蛋白（ATG）（2.86[1.11-7.37 95%CI]；p=0.029）。与强度降低的条件性治疗（RIC）相比，骨髓消融性条件性治疗与 AIC 风险较低相关在氟达拉滨和/或阿仑单抗（0.34[0.12-0.98 95%CI]；p=0.046）和 ATG 中包含的方案（0.34[0.12-0.95 95%CI]；p=0.04）。这些发现为同种异体 HSCT 治疗 aAA 的罕见且潜在威胁生命的并发症提供了有用的临床信息，并进一步支持 BM 作为 aAA 患者移植的首选干细胞来源。
