儿童的真多细胞血和基本血小板血,仍然是儿科医生面临的挑战
Agathe Picard1, Sophie Bayart2, Marianna Deparis3
1Pediatric Oncohematology Department, Rennes University Hospital, Rennes, France. agathe.picard@chu-rennes.fr.
European journal of pediatrics
|February 4, 2025
概括
儿童骨髓增殖性瘤 (MPN) 如真多细胞血症 (PV) 和基本血栓血症 (ET) 缺乏管理指南. 医生培训和数据收集对于改善治疗这些罕见的儿童血液癌症至关重要.
科学领域:
- 儿科血液学 儿科血液学
- 在瘤学瘤学.
- 罕见疾病 罕见疾病
背景情况:
- 骨髓增殖性瘤 (MPNs) 是一种罕见的慢性疾病,主要影响成年人,数据有限,对于儿科患者没有确立的治疗指南.
- 多细胞血病 (PV) 和基本血小板血 (ET) 是在儿童,青少年和年轻成年人中诊断的特定的MPN,需要与成人协议不同的量身定制方法.
研究的目的:
- 评估目前在法国诊断为PV或ET的儿科患者的管理和后续实践.
- 了解儿科医生和成人血液学家在管理这些罕见的儿科血液学疾病时所面临的前景和挑战.
- 描述PV或ET儿童的现实世界临床过程和治疗.
主要方法:
- 进行了全国实践分析,以评估医生对儿科MPN管理的经验.
- 一项多中心,描述性,回顾性研究包括来自法国七个血液学部门的17名被诊断患有ET或PV的18岁以下患者.
- 医生采访和患者数据审查构成了分析的基础.
主要成果:
- 医生报告说,在诊断和管理儿科MPN方面,缺乏专业知识和培训.
- 与成人队列相比,研究队列 (15 ET,2 PV) 显示出高比例的无症状患者在诊断时 (41%) 和更高的"三阴性"状态率 (59%).
- 虽然76.5%的患者接受了治疗 (抗血栓剂,降细胞剂),但在45个月的中位随访期间没有观察到并发症,这表明治疗降级的可能性.
结论:
- 对于管理儿科MPN的医疗保健专业人员来说,有必要加强培训和教育.
- 积累有关儿科MPN的描述性数据对于制定基于证据的管理准则至关重要.
- 鉴于并发症率较低,对于患有PV和ET的儿科患者,应考虑与成人血液学家合作,采取快速治疗降级策略.
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