Hacettepe University Faculty of Medicine, Department of Hematology, 06100, Ankara, Turkey.
Hacettepe University Faculty of Medicine, Department of Hematology, 06100, Ankara, Turkey.
Transfus Apher Sci. 2021 Apr;60(2):103050. doi: 10.1016/j.transci.2020.103050. Epub 2021 Jan 2.
Aplastic anemia (AA) is a life-threatening disorder and may be associated with significant morbidity and mortality Currently, the first treatment option is allogeneic hematopoietic stem cell transplant (allo-HSCT) for patients younger than 40 years. Bone marrow is recommended as the stem cell source due to less graft versus host disease (GVHD) risk and better outcomes than peripheral blood (PB)-derived stem cell. The aim of this study is to share the data of AA patients who have underwent PB-derived allo-HSCT in our bone marrow transplantation center.
Twenty-seven patients who underwent PB-derived allo-HSCT from human leukocyte antigen matched sibling donors were analyzed retrospectively.
The median follow-up time was 95.2 months (range, 4.8-235 months). The 10-year survival was 89 %. The median neutrophil and platelet engraftment time was 11 days (range, 9-16 days) and 13 days (range, 11-29 days), respectively. Primary platelet engraftment failure was observed in 1 patient (3.7 %). Acute and chronic GVHD observed in 2 (7.4 %) and 3 (11.1 %) patients, respectively. Neutropenic fever was observed in 13 (44.8 %) of patients until the engraftment after allo-HSCT. One patient died due to CMV infections, two died due to septic shock secondary to fungal infection.
Although there is no prospective data directly comparing BM with PB as stem cell source in AA, observational studies indicates better OS with BM. PB can be used in certain situations such as higher risk for graft failure and donor preference. This study demonstrated that PB-derived stem cell seems to be a reasonable alternative to BM.
再生障碍性贫血（AA）是一种危及生命的疾病，可能与显著的发病率和死亡率相关。目前，对于 40 岁以下的患者，首选治疗方案是异基因造血干细胞移植（allo-HSCT）。由于移植物抗宿主病（GVHD）风险较低和结局优于外周血（PB）来源的干细胞，骨髓被推荐为干细胞来源。本研究旨在分享在我们的骨髓移植中心接受 PB 来源 allo-HSCT 的 AA 患者的数据。
回顾性分析了 27 例接受 HLA 匹配的同胞供者 PB 来源 allo-HSCT 的患者。
中位随访时间为 95.2 个月（范围，4.8-235 个月）。10 年生存率为 89%。中性粒细胞和血小板植入的中位时间分别为 11 天（范围，9-16 天）和 13 天（范围，11-29 天）。1 例患者（3.7%）出现原发性血小板植入失败。2 例（7.4%）和 3 例（11.1%）患者分别发生急性和慢性 GVHD。13 例（44.8%）患者在 allo-HSCT 后中性粒细胞减少性发热。1 例患者因 CMV 感染死亡，2 例患者因真菌感染后继发感染性休克死亡。
虽然没有直接比较 BM 与 PB 作为 AA 干细胞来源的前瞻性数据，但观察性研究表明 BM 具有更好的 OS。在某些情况下，如移植物失败风险较高和供者偏好，可以使用 PB。本研究表明 PB 来源的干细胞似乎是 BM 的合理替代方案。
