Kleen Kristina, Gebauer Judith, Spix Claudia, Kronziel Lea L, König Inke, Baust Katja, Calaminus Gabriele, Simon Thorsten, Hero Barbara, Zolk Oliver, Graf Norbert, Abdul-Khaliq Hashim, Langer Thorsten
Paediatric Haematology and Oncology, Department of Paediatrics and Adolescent Medicine, University Hospital Schleswig-Holstein, Lübeck, Germany.
Medical Clinic 1, University Hospital Schleswig-Holstein, Lübeck, Germany.
Cancer Med. 2025 Aug;14(16):e71158. doi: 10.1002/cam4.71158.
Long-term childhood cancer survivors (CCS) may develop anthracycline-induced cardiomyopathy. Our cross-sectional study focused on the question of whether a central echocardiographic reference assessment is associated with a higher detection rate of cardiac dysfunction in a population-based cohort of affected children with neuroblastoma or nephroblastoma. We also examined the prevalence of anthracycline-induced cardiomyopathy and its risk factors.
The cohort of this subproject comprises 370 nephroblastoma or neuroblastoma survivors diagnosed with cancer between 1990 and 2012. At study entry, participants were younger than 18 years old, had been treated with anthracyclines, and had no documented previous cardiac disease. Data were collected via patient questionnaires, cardiologic examinations in the network of adults with congenital heart defects (Erwachsene mit angeborenem Herzfehler [EMAH]) and a reference assessment of the recorded echocardiography.
The prevalence of cardiomyopathy in the study cohort (mean age: 12 years) was 6.3% at a median of 9.1 years after initial cancer diagnosis. Risk factors were an age under 5 years at tumor diagnosis and concomitant treatment with cyclophosphamide or radiation. As a central and novel finding, the detection rates by the EMAH cardiologists and the reference center are similarly high but discrepant.
Limitations were mainly due to the low responder rate and incomplete data. This study established a nationwide competence network linking pediatric oncology and cardiology centers across six university hospitals in Germany, enabling data collection on pediatric CCS. Despite lower case numbers compared to adult CCS cohorts, meaningful data were gathered and analyzed.
Cardiac late effects after anthracycline-based therapy in childhood affect a relevant proportion of long-term CCS at pediatric age. In order to enable timely diagnosis and treatment, preventive examinations are essential and might benefit from additional central reference assessments. Discrepancy in detection of cardiomyopathy by reference and EMAH cardiologists requires further investigation.
儿童癌症长期幸存者（CCS）可能会发生蒽环类药物所致心肌病。我们的横断面研究聚焦于这样一个问题：在一个以人群为基础的神经母细胞瘤或肾母细胞瘤患儿队列中，心脏超声中央参考评估是否与心脏功能障碍的更高检出率相关。我们还研究了蒽环类药物所致心肌病的患病率及其危险因素。
该子项目的队列包括1990年至2012年间被诊断患有癌症的370名肾母细胞瘤或神经母细胞瘤幸存者。在研究开始时，参与者年龄小于18岁，接受过蒽环类药物治疗，且既往无心脏病记录。数据通过患者问卷、先天性心脏病成人网络（Erwachsene mit angeborenem Herzfehler [EMAH]）中的心脏检查以及记录的超声心动图参考评估来收集。
在初始癌症诊断后中位9.1年时，研究队列（平均年龄：12岁）中心肌病的患病率为6.3%。危险因素为肿瘤诊断时年龄小于5岁以及同时接受环磷酰胺或放疗。作为一个核心且新颖的发现，EMAH心脏病专家和参考中心的检出率同样高，但存在差异。
局限性主要归因于低应答率和不完整的数据。本研究建立了一个全国性的能力网络，将德国六所大学医院的儿科肿瘤学和心脏病学中心联系起来，从而能够收集儿科CCS的数据。尽管与成人CCS队列相比病例数较少，但仍收集并分析了有意义的数据。
儿童期基于蒽环类药物的治疗后的心脏远期效应影响了相当比例的儿科长期CCS。为了实现及时诊断和治疗，预防性检查至关重要，且可能受益于额外的中央参考评估。参考心脏病专家和EMAH心脏病专家在心肌病检测方面的差异需要进一步研究。
