GD2抗体达妥昔单抗β治疗神经母细胞瘤的临床应用专家共识（2021年版）

神经母细胞瘤(neuroblastoma,NB)是儿童最常见的颅外实体肿瘤,临床表现及预后具有高度异质性。低危患儿预后较好,高危患儿即使接受化疗、放疗、手术、造血干细胞移植等多种方法的综合治疗,长期存活率仍不足50%。探索潜在的治疗靶点对于提高高危患者的生存率具有重要意义。双唾液酸神经节苷脂抗原GD2在NB细胞高表达,达妥昔单抗β可与NB细胞膜表面过表达的GD2特定靶点结合,触发抗体依赖性细胞介导的细胞毒性作用和补体依赖的细胞毒性效应,通过双重免疫机制而发挥抗肿瘤作用。通过总结国外多项关键临床研究,并结合在海南和天津医疗先行区的上市前初步应用经验,我们对达妥昔单抗β的安全性、有效性、适用人群及使用方法进行总结和推荐,提出本共识,旨在为临床医师提供指导与帮助。     

The heart in myotonic dystrophy. Clinical and instrumental study in 17 patients (author's transl)

17 patients with myotonic dystrophy have been studied. The skeletal muscle disease has been assessed by history, physical examination, electro-myography and muscle biopsy. Cardiac evaluation has been obtained by history, physical examination, chest X-ray, ecg at rest and after exercise, vectorcardiography and echocardiography. Only two patients presented symptoms and clinical findings of cardiac disease, otherwise conduction disturbances have been founded in 15 cases, pseudoinfarct pattern in 5 cases and loss of anterior vectors in 2 cases. Has to be remembered that in myotonic dystrophy sudden death has a very high incidence, which could be explained by the development of complete a-v block, because of the impairment of the conduction system. Therefore, in those patients with bifascicular blocks (3 cases), the electrophysiological study may be useful in order to evaluate the opportunity to put on a permanent pace-maker, to prevent sudden death.