Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark
Department of Pediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark.
BMJ Open. 2021 Feb 9;11(2):e045543. doi: 10.1136/bmjopen-2020-045543.
More than 90% of patients diagnosed with childhood acute lymphoblastic leukaemia (ALL) today will survive. However, half of the survivors are expected to experience therapy-related chronic or late occurring adverse effects, reducing quality of life. Insight into underlying risk trajectories is warranted. The aim of this study is to establish a Nordic, national childhood ALL survivor cohort, to be investigated for the total somatic and psychosocial treatment-related burden as well as associated risk factors, allowing subsequent linkage to nation-wide public health registers.
This population-based observational cohort study includes clinical follow-up of a retrospective childhood ALL survivor cohort (n=475), treated according to a common Nordic ALL protocol during 2008-2018 in Denmark. The study includes matched controls. Primary endpoints are the cumulative incidence and cumulative burden of 197 health conditions, assessed through self-report and proxy-report questionnaires, medical chart validation, and clinical examinations. Secondary endpoints include organ-specific outcome, including cardiovascular and pulmonary function, physical performance, neuropathy, metabolic disturbances, hepatic and pancreatic function, bone health, oral and dental health, kidney function, puberty and fertility, fatigue, and psychosocial outcome. Therapy exposure, acute toxicities, and host genome variants are explored as risk factors.
The study is approved by the Regional Ethics Committee for the Capital Region in Denmark (H-18035090/H-20006359) and by the Danish Data Protection Agency (VD-2018-519). Results will be published in peer-reviewed journals and are expected to guide interventions that will ameliorate the burden of therapy without compromising the chance of cure.
如今，超过 90%被诊断患有儿童急性淋巴细胞白血病 (ALL) 的患者能够存活。然而，预计一半的幸存者会经历与治疗相关的慢性或迟发性不良影响，降低生活质量。因此，有必要深入了解潜在的风险轨迹。本研究旨在建立一个北欧的全国性儿童 ALL 幸存者队列，对其进行全面的躯体和心理社会治疗相关负担以及相关风险因素的调查，从而能够随后与全国性公共健康登记处进行关联。
这是一项基于人群的观察性队列研究，包括对一个回顾性儿童 ALL 幸存者队列（n=475）进行临床随访，这些患者在 2008-2018 年期间按照丹麦共同的北欧 ALL 方案进行治疗。该研究包括匹配对照。主要终点是通过自我报告和代理报告问卷、病历验证和临床检查评估的 197 种健康状况的累积发病率和累积负担。次要终点包括器官特异性结局，包括心血管和肺功能、身体表现、神经病变、代谢紊乱、肝和胰腺功能、骨骼健康、口腔和牙齿健康、肾功能、青春期和生育能力、疲劳和心理社会结局。治疗暴露、急性毒性和宿主基因组变异被探索为风险因素。
该研究已获得丹麦首都地区伦理委员会（H-18035090/H-20006359）和丹麦数据保护局（VD-2018-519）的批准。研究结果将发表在同行评议的期刊上，有望指导干预措施，在不影响治愈机会的情况下减轻治疗负担。
