本研究旨在评估父母在急性淋巴细胞白血病 (ALL) 治疗期间如何看待与治疗相关的副作用。在瑞典、芬兰和丹麦，诊断时 1-17.9 岁的儿童的父母还活着，并且在 ALL 治疗结束后 ≥6 个月内首次缓解，被要求回答有关长春新碱相关副作用如何影响他们的孩子的具体项目(VCR)、皮质类固醇、聚乙二醇天冬酰胺酶 (ASP) 和维持治疗，以及这些治疗的总体影响、一般并发症，以及与其他 ALL 儿童相比，他们对孩子影响的看法。 307名儿童的家长做出了回应。超过三分之一的人表示，他们的孩子受到 VCR (39.7%) 和皮质类固醇 (35.8%) 的严重影响，其中最常见和最严重的是行走困难、肌肉无力、疼痛、食欲改变和情绪波动症状。除周围麻痹（12.1%）外，NOPHO ALL2008 数据库中缺乏对这些毒性的报告。对于 NOPHO ALL2008 数据库中报告的不同毒性，例如血栓形成和胰腺炎，家长报告与数据库类似。尽管据报告治疗期间总体负面影响较高，但家长普遍认为其对孩子的影响与其他 ALL 儿童相比较小或相似。家长认为 VCR 和皮质类固醇治疗在 ALL 治疗期间尤其会对孩子产生负面影响，这一点在 NOPHO ALL2008 毒性报告中并未体现。我们的结果强调了将患者/家长报告的结果纳入毒性报告的重要性。© 2024 作者。约翰·威利 (John Wiley) 出版的 HemaSphere

This study aimed to assess how parents perceived treatment-related side effects during acute lymphoblastic leukemia (ALL) treatment. Parents of children 1-17.9 years at diagnosis in Sweden, Finland, and Denmark who were alive and in first remission ≥6 months after end of ALL treatment were asked to respond on specific items regarding how their child was affected by side effects related to vincristine (VCR), corticosteroids, peg-asparaginase (ASP), and maintenance therapy, as well as overall impact of these treatments, complications in general, and their perception of impact on their child in comparison with other children with ALL. Parents of 307 children responded. More than a third reported that their child had been affected to a high extent by VCR (39.7%) and corticosteroids (35.8%), with walking difficulties, muscular weakness, pain, changes in appetite, and mood swings as the most common and severe symptoms. Reporting of these toxicities was lacking from the NOPHO ALL2008 database, except for peripheral paralysis (12.1%). For distinct toxicities reported in the NOPHO ALL2008 database, for example, thrombosis and pancreatitis, parent reports were similar to the database. Although a high overall negative impact during treatment was reported, parents generally rated the impact on their child as less, or similar, to other children with ALL. Parents perceived VCR and corticosteroid therapy, in particular, to have a negative impact on their child during ALL treatment, which was not captured in the NOPHO ALL2008 toxicity reporting. Our results highlight the importance of including patient/parent-reported outcomes in toxicity reporting.© 2024 The Author(s). HemaSphere published by John Wiley & Sons Ltd on behalf of European Hematology Association.