Department of Pediatrics, The University of Tokyo, Tokyo, Japan; Department of Cell Therapy and Transplantation Medicine, The University of Tokyo, Tokyo, Japan; Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.
Pediatr Blood Cancer. 2014 Oct;61(10):1860-6. doi: 10.1002/pbc.25121. Epub 2014 Jun 29.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curable approach for myelodysplastic syndrome (MDS) and myeloproliferative neoplasms (MPN); however, the event-free survival rate of patients with pediatric MDS and MPN is still only approximately 60%. Although salvage HSCT is the only curative approach for patients with the failure of previous HSCT, its safety and efficacy have yet to be determined.
We retrospectively analyzed 51 pediatric MDS or MPN who received salvage HSCT for relapse or graft failure following HSCT using registry data of the Japan Society for Hematopoietic Cell Transplantation. The indications used for salvage HSCT were relapse in 22 patients and graft failure in 29 patients.
The overall survival (OS) rate for salvage HSCT in relapsed patients was 49.0 ± 10.8% at 3 years. The cumulative incidence of relapse following salvage HSCT was 29.8 ± 10.7% at 3 years, whereas the incidence of non-relapse mortality (NRM) was 28.6 ± 10.2%. No significant differences were observed in the OS after salvage HSCT between disease types. Twenty-four of 29 patients who received salvage HSCT for graft failure achieved engraftment, resulting in an engraftment probability of 81.5 ± 8.0% on day 100. The OS rate after salvage HSCT for graft failure was 56.8 ± 9.6% at 3 years.
Second HSCT should be considered as a valuable option for the patients with relapse and graft failure in patients with pediatric MDS or MPN after HSCT, but high NRM is an important issue that needs to be addressed.
异基因造血干细胞移植（HSCT）是治疗骨髓增生异常综合征（MDS）和骨髓增殖性肿瘤（MPN）的唯一方法；然而，儿科 MDS 和 MPN 患者的无事件生存率仍约为 60%。尽管挽救性 HSCT 是既往 HSCT 失败患者的唯一治愈方法，但它的安全性和疗效尚待确定。
我们使用日本造血细胞移植学会的登记数据，对 51 例接受挽救性 HSCT 治疗 HSCT 后复发或移植物失败的儿科 MDS 或 MPN 患者进行了回顾性分析。挽救性 HSCT 的适应证为 22 例患者复发，29 例患者移植物失败。
复发患者挽救性 HSCT 的总生存率（OS）在 3 年内为 49.0±10.8%。挽救性 HSCT 后 3 年内复发的累积发生率为 29.8±10.7%，而非复发死亡率（NRM）为 28.6±10.2%。不同疾病类型间挽救性 HSCT 后的 OS 无显著差异。29 例因移植物失败而接受挽救性 HSCT 的患者中有 24 例获得了植入，第 100 天的植入概率为 81.5±8.0%。挽救性 HSCT 后移植物失败的 3 年 OS 率为 56.8±9.6%。
对于 HSCT 后儿科 MDS 或 MPN 患者的复发和移植物失败，应考虑进行二次 HSCT，但是高 NRM 是一个需要解决的重要问题。
