Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Germany.
Department of Pediatrics I, Division of Pediatric Neurology, Medical University of Innsbruck, Innsbruck, Austria.
J Neuromuscul Dis. 2020;7(1):41-46. doi: 10.3233/JND-190441.
The natural history of patients with spinal muscular atrophy (SMA) has changed due to advances in standard care and development of targeted treatments. Nusinersen was the first drug approved for the treatment of all SMA patients. The transfer of clinical trial data into a real-life environment is challenging, especially regarding the advice of patients and families to what extent they can expect a benefit from the novel treatment. We report the results of a modified Delphi consensus process among child neurologists from Germany, Austria and Switzerland about the indication or continuation of nusinersen treatment in children with SMA type 1 based on different clinical case scenarios.
由于标准治疗的进步和靶向治疗的发展，脊髓性肌萎缩症（SMA）患者的自然病程发生了变化。诺西那生钠是第一种被批准用于所有 SMA 患者治疗的药物。将临床试验数据转化到真实环境中具有挑战性，特别是关于患者和家属可以从新治疗中获得多大程度的获益的建议。我们报告了德国、奥地利和瑞士的儿童神经病学家就不同临床病例场景下基于诺西那生钠治疗 SMA 1 型患儿的适应证或继续治疗进行的改良 Delphi 共识过程的结果。
