Kobayashi S, Ito M, Sano H, Mochizuki K, Akaihata M, Waragai T, Ohara Y, Hosoya M, Ohto H, Kikuta A
Department of Pediatric Oncology.
Transfus Med. 2014 Oct;24(5):305-10. doi: 10.1111/tme.12150. Epub 2014 Sep 15.
Despite improvements in first-line therapies, the outcomes of relapsed or refractory childhood acute leukaemia that has not achieved complete remission after relapse, has relapsed after stem cell transplantation (SCT), has primary induction failure and has relapsed with a very unfavourable cytogenetic risk profile, are dismal.
We evaluated the feasibility and efficacy of T-cell-replete haploidentical peripheral blood stem cell transplantation (haplo-SCT) with low-dose anti-human thymocyte immunoglobulin (ATG), tacrolimus, methotrexate and prednisolone (PSL) in 14 paediatric patients with high-risk childhood acute leukaemia.
All patients achieved complete engraftment. The median time to reaching an absolute neutrophil count of more than 0.5 × 10(9) L(-1) was 14 days. Acute graft-vs-host disease (aGVHD) of grades II-IV and III-IV developed in 10 (71%) and 2 (14%) patients, respectively. Treatment-related mortality and relapse occurred in one (7%) patient and six (43%) patients, respectively. Eleven patients were alive and seven of them were disease-free with a median follow-up of 36 months (range: 30-159 months). The probability of event-free survival after 2 years was 50%.
These findings indicate that T-cell-replete haplo-SCT, with low-dose ATG and PSL, provides sustained remission with an acceptable risk of GVHD in paediatric patients with advanced haematologic malignancies.
尽管一线治疗有所改善，但复发或难治性儿童急性白血病的预后仍然很差，这些白血病包括复发后未达到完全缓解、干细胞移植（SCT）后复发、初次诱导失败以及复发时细胞遗传学风险特征非常不利的情况。
我们评估了在14例高危儿童急性白血病患儿中，采用低剂量抗人胸腺细胞免疫球蛋白（ATG）、他克莫司、甲氨蝶呤和泼尼松龙（PSL）进行富含T细胞的单倍体相合外周血干细胞移植（单倍体SCT）的可行性和疗效。
所有患者均实现完全植入。达到绝对中性粒细胞计数超过0.5×10⁹/L⁻¹的中位时间为14天。分别有10例（71%）和2例（14%）患者发生了II-IV级和III-IV级急性移植物抗宿主病（aGVHD）。治疗相关死亡率和复发分别发生在1例（7%）和6例（43%）患者中。11例患者存活，其中7例无病生存，中位随访时间为36个月（范围：30-159个月）。2年后无事件生存的概率为50%。
这些结果表明，对于晚期血液系统恶性肿瘤的儿科患者，采用低剂量ATG和PSL的富含T细胞的单倍体SCT可提供持续缓解，且移植物抗宿主病风险可接受。
