Paediatric Hematology, Oncology and Hematopoietic Stem Cell Transplantation Team, Department of Paediatrics and Adolescent Medicine, Hong Kong Children's Hospital, Hong Kong Special Administrative Region, China.
Pediatric Nephrology team, Department of Paediatrics and Adolescent Medicine, Hong Kong Children's Hospital, Hong Kong Special Administrative Region, China.
Pediatr Transplant. 2022 Nov;26(7):e14366. doi: 10.1111/petr.14366. Epub 2022 Jul 21.
Transplant-associated thrombotic microangiopathy (TA-TMA) is an under-recognized yet potentially devastating complication of hematopoietic stem cell transplantation (HSCT) which had increased awareness in recent years. This report summarizes the demographics and outcomes of pediatric TA-TMA in Hong Kong.
All patients aged below 18 years who underwent HSCT in the Hong Kong Children's Hospital and were diagnosed to have TA-TMA during the 2-year period from April 1, 2019 to March 31, 2021 were included.
A total of 73 transplants (51 allogeneic and 22 autologous) in 63 patients had been performed. Six patients (four males and two females) developed TA-TMA at a median duration of 2.5 months post-HSCT. The incidence rate was 9.52%. Of the six TA-TMA patients, five underwent allogenic one underwent autologous HSCT, respectively. Three of them were histologically proven. All four patients with cyclosporine had stopped the drug once TA-TMA was suspected. Median six doses of eculizumab were administered to five out of six patients. Three patients died (two due to fungal infection and one due to acute-on-chronic renal failure) within 3 months upon diagnosis of TA-TMA. Among three survivors, two stabilized with mild stage 2 chronic kidney disease (CKD) while the other suffered from stage 5 end-stage CKD requiring lifelong dialysis.
In conclusion, recognition and diagnosis of TA-TMA are challenging. Early recognition and prompt administration of complement blockage with eculizumab may be beneficial in selected cases. Further prospective research studies are recommended to improve the management and outcomes of TA-TMA.
移植相关性血栓性微血管病（TA-TMA）是造血干细胞移植（HSCT）中一种尚未被充分认识但潜在具有破坏性的并发症，近年来其认知度有所提高。本报告总结了香港儿科 TA-TMA 的人口统计学和结局。
所有在 2019 年 4 月 1 日至 2021 年 3 月 31 日期间在香港儿童医院接受 HSCT 且年龄在 18 岁以下的患者，若在移植后被诊断为 TA-TMA，则被纳入本研究。
共有 63 名患者的 73 次移植（51 次异基因和 22 次自体），6 名患者（4 名男性和 2 名女性）在 HSCT 后中位 2.5 个月出现 TA-TMA，发生率为 9.52%。6 例 TA-TMA 患者中，5 例接受异基因 HSCT，1 例接受自体 HSCT。其中 3 例经组织学证实。所有 4 例环孢素治疗的患者一旦怀疑 TA-TMA 均停止使用该药物。6 例患者中有 5 例接受了中位数为 6 剂的依库珠单抗治疗。3 例患者在确诊 TA-TMA 后 3 个月内死亡（2 例死于真菌感染，1 例死于急性肾损伤合并慢性肾功能衰竭）。在 3 例幸存者中，2 例稳定为轻度 2 期慢性肾脏病（CKD），而另 1 例患有 5 期终末期 CKD，需要终身透析。
总之，TA-TMA 的识别和诊断具有挑战性。在某些情况下，早期识别和及时使用依库珠单抗阻断补体可能有益。建议进一步开展前瞻性研究，以改善 TA-TMA 的管理和结局。
