Division of Hematology/Oncology, Department of Pediatrics, Stanford University School of Medicine, Stanford, California, USA.
Department of Pediatrics, Division of Hematology/Oncology/Bone Marrow Transplantation, Emory University, Atlanta, GA, USA.
BMC Cancer. 2021 Apr 29;21(1):474. doi: 10.1186/s12885-021-08182-z.
Targeted anticancer therapies such as BCR-ABL tyrosine kinase inhibitors (TKIs) have improved outcomes for chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL). However, little is known about long-term risks of TKIs in children. Exposure-based survivorship guidelines do not include TKIs, thus surveillance practices may be variable.
We retrospectively examined surveillance for cardiac and endocrine late effects in children receiving TKIs for Ph + leukemias, diagnosed at < 21 years between 2000 and 2018. Frequency of echocardiogram (ECHO), electrocardiogram (EKG), thyroid stimulating hormone (TSH), dual-energy x-ray absorptiometry (DXA), and bone age testing were abstracted. Descriptive statistics were stratified by leukemia type.
66 patients (CML n = 44; Ph + ALL n = 22) met inclusion criteria. Among patients with CML, ≥1 evaluation was done: ECHO (50.0%), EKG (48.8%), TSH (43.9%), DXA (2.6%), bone age (7.4%). Among patients with Ph + ALL, ≥1 evaluation was done: ECHO (86.4%), EKG (68.2%), TSH (59.1%), DXA (63.6%), bone age (44.4%). Over a median 6.3 and 5.7 years of observation, respectively, 2% of patients with CML and 57% with Ph + ALL attended a survivorship clinic.
Despite common exposure to TKIs in survivors of Ph + leukemias, patterns of surveillance for late effects differed in CML and Ph + ALL, with the latter receiving more surveillance likely due to concomitant chemotherapy exposures. Targeted therapies such as TKIs are revolutionizing cancer treatment, but surveillance for late effects and referral to survivorship clinics are variable despite the chronicity of exposure. Evidence based guidelines and longer follow-up are needed.
针对癌症的靶向治疗方法，如 BCR-ABL 酪氨酸激酶抑制剂（TKI），已经改善了慢性髓性白血病（CML）和费城染色体阳性急性淋巴细胞白血病（Ph+ALL）的预后。然而，对于儿童接受 TKI 治疗后的长期风险知之甚少。基于暴露的生存指南不包括 TKI，因此监测实践可能存在差异。
我们回顾性地检查了 2000 年至 2018 年期间诊断为＜21 岁的接受 Ph+白血病 TKI 治疗的儿童的心脏和内分泌系统晚期效应的监测情况。摘录了超声心动图（ECHO）、心电图（EKG）、促甲状腺激素（TSH）、双能 X 线吸收法（DXA）和骨龄检查的频率。按白血病类型对描述性统计数据进行分层。
66 名患者（CML 患者 n=44；Ph+ALL 患者 n=22）符合纳入标准。在 CML 患者中，≥1 次评估为：ECHO（50.0%）、EKG（48.8%）、TSH（43.9%）、DXA（2.6%）、骨龄（7.4%）。在 Ph+ALL 患者中，≥1 次评估为：ECHO（86.4%）、EKG（68.2%）、TSH（59.1%）、DXA（63.6%）、骨龄（44.4%）。分别观察了中位数为 6.3 年和 5.7 年的时间后，2%的 CML 患者和 57%的 Ph+ALL 患者参加了生存随访门诊。
尽管 Ph+白血病幸存者普遍接受 TKI 治疗，但 CML 和 Ph+ALL 患者的晚期效应监测模式存在差异，后者接受了更多的监测，这可能是由于同时接受化疗暴露的原因。TKI 等靶向治疗正在彻底改变癌症治疗，但尽管暴露具有慢性，晚期效应监测和向生存随访门诊的转介仍存在差异。需要基于证据的指南和更长的随访。
