Objective: To evaluate the efficacy and safety of eltrombopag for children with thrombocytopenia after hematopoietic stem cell transplantation (HSCT). Methods: Clinical data of 24 patients with thrombocytopenia after HSCT,who were treated with eltrombopag in the Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University from August 1, 2018 to April 1, 2019 were analyzed retrospectively. The response rate and adverse reactions of eltrombopag were evaluated. Patients were divided into groups by source of hematopoietic stem cells (umbilical cord blood group and peripheral stem cell group) and type of disease (malignant and non-malignant disease group) and the clinical outcomes between groups were compared. Rank Sum test was used for comparisons between groups. Results: Among 24 cases, 15 were males and 9 females, the age of starting eltrombopag was 7.7 (2.6-13.7) years, the time of eltrombopag treatment after HSCT was 27.5 (8.0-125.0) days, the time from treatment to complete response (CR) was 23.5 (6.0-83.0) days, with the treatment course 36.5 (8.0-90.0) days. The total dose of eltrombopag was 1 400(200-5 900) mg. Complete response rate was 92% (22/24),without eltrombopag related adverse reactions. Comparing with peripheral stem cell group (n=8), the course and total dose of eltrombopag in umbilical cord blood group (n=16) were significantly reduced(24.5 (8.0-81.0) vs. 65.5 (35.0-90.0) d, Z=-3.004, P=0.002; 900.0 (200.0-3 850.0) vs. 2 862.5 (1 175.0-5 900.0) mg, Z=-2.604, P=0.007), but no significant differences were found in the time from treatment to complete response, platelet count after 2 weeks of eltrombopag withdrawal or platelet count at the end point of follow-up (all P>0.05). Comparing malignant patients (n=12) and non-malignant patients (n=12), no significant differences were found in the time from treatment to complete response, course, total dose, platelet count after 2 weeks of eltrombopag withdrawal, and platelet count at the end point of follow-up in non-malignant patients (all P>0.05). Conclusion: Eltrombopag is safe and maybe effective for thrombocytopenia after HSCT, especially for umbilical cord blood transplantation.
目的： 评价艾曲波帕治疗儿童造血干细胞移植（HSCT）后血小板减少症的疗效与安全性。 方法： 回顾性分析2018年8月1日至2019年4月1日于中山大学孙逸仙纪念医院儿科接受HSCT后发生血小板减少症并采用艾曲波帕治疗的24例患儿的临床资料，评估治疗有效率及不良反应，根据造血干细胞来源分为脐带血组和外周干细胞组，根据疾病类型分为恶性病组和非恶性病组，分析各组间临床疗效。组间比较采用秩和检验。 结果： 24例患儿中男15例、女9例，艾曲波帕用药时的年龄7.7（2.6~13.7）岁，接受艾曲波帕治疗的时间为移植后第27.5（8.0~125.0） d，使用后完全有效的时间为23.5（6.0~83.0） d，用药疗程为36.5（8.0~90.0） d，艾曲波帕总剂量为1 400（200~5 900）mg，完全有效率92%（22/24），未发生艾曲波帕相关不良反应。脐带血干细胞移植（16例）的患儿使用艾曲波帕的疗程及总剂量均明显低于外周干细胞移植组（8例）［24.5（8.0~81.0）比65.5（35.0~90.0）d，900.0（200.0~3 850.0）比2 862.5 （1 175.0~5 900.0） mg，Z=-3.004、-2.604，P=0.002、0.007］，而达到完全有效的时间及停药2周后血小板计数、随访终点血小板计数的差异均无统计学意义（均P>0.05）。与恶性病患儿（12例）相比，非恶性病患儿（12例）用药后至获得完全有效的时间、用药疗程、总剂量及停药2周后血小板计数、随访终点血小板计数的差异均无统计学意义（均P>0.05）。 结论： 艾曲波帕用于儿童HSCT后血小板减少症有一定疗效，安全性高，尤其在脐带血移植中可能更有优势。.