儿科肿瘤溶解综合征治疗现状和疾病负担调查

Investigation on Current Status of Treatment and Burden of Disease in Pediatric Tumor Lysis Syndrome

目的评价儿科肿瘤溶解综合征(TLS)的治疗现状和疾病负担。方法利用问卷调查方法,对国内11个城市的共计20名临床医师进行面对面调查,内容包括收治患者状况、对TLS所采取的预防和治疗手段、治疗费用等。结果目前临床上TLS预防所使用的方式包括水化、碱化、别嘌呤醇、呋塞米、低分子右旋糖酐(DU)等在内的标准治疗方案(Standard of Care, SOC)。使用SOC预防TLS的失败率为5%~10%。对于TLS的治疗,需要在SOC基础上使用血液滤过(hemofiltration,HF)。在儿童急性髓细胞性白血病(acute myeloid leukemia, AML)、急性淋巴细胞白血病(acute lymphoblastic leukemia, ALL)和非霍奇金淋巴瘤(non-Hodgkin’s lymphoma, NHL)患者中,TLS治疗失败率分别为13.9%、14.9%和15.7%。在儿童AML、ALL和NHL患者中TLS预防或治疗的费用不同,总体来说检查费、治疗费以及HF费用较高。此外,SOC和HF会产生短期急性不良反应,HF还可能会产生肾损伤等长期不良反应。结论目前临床上均会对儿童血液肿瘤患者进行TLS常规预防,但TLS发生率仍处于较高水平。由于SOC在机制上无法处理已经存在于血液中的尿酸,临床上存在较高的HF使用率和治疗失败率,这给医疗体系和患者均产生较重的负担。

Objective To evaluate the current status and disease burden of pediatric tumor lysis syndrome(TLS).Methods A face-to-face questionnaire survey was conducted with 20 clinicians from 11 cities in China,to understand questions including the status of admitted patients,the prevention and treatment measures taken,and the cost of treatment for TLS.Results The clinical Standard of Care(SOC)for the prevention of TLS included hydration,alkalization,allopurinol,furosemide,low molecular weight dextran(DU),etc.The failure rate of SOC in the prevention of TLS was 5%to 10%.For the treatment of TLS,hemofiltration(HF)was required on top of SOC.Among children with acute myeloid leukemia(AML),acute lymphoblastic leukemia(ALL),and non-Hodgkin’s lymphoma(NHL),TLS-related treatment failure rate were 13.9%,14.9%,and 15.7%,respectively.The cost of TLS prevention or treatment varied among patients with AML,ALL,or NHL.In general,the costs of examination,treatment,and HF were higher.In addition,SOC and HF could cause short-term acute adverse reactions,and HF might also cause long-term adverse reactions such as kidney damage.Conclusion Currently in clinic,TLS prevention is routinely applied in children with hematological malignancies,but the incidence of TLS remains high.Since SOC cannot reduce preexisting uric acid,the rate of HF usage and treatment failure rate are significantly high in the clinical practice,which imposes a relatively heavy burden on the healthcare system and patients.