Faculty of medicine, Université Paris-Est Creteil (UPEC), F-94010, Créteil Cedex, France.
Assistance Publique-Hôpital Paris (AP-HP), Hôpital Henri-Mondor, Service de Dermatologie, F-94010, Créteil, France.
Orphanet J Rare Dis. 2020 Feb 3;15(1):37. doi: 10.1186/s13023-020-1310-3.
Neurofibromatosis type 1 is a relatively common genetic disease, with a prevalence ranging between 1/3000 and 1/6000 people worldwide. The disease affects multiple systems with cutaneous, neurologic, and orthopedic as major manifestations which lead to significant morbidity or mortality. Indeed, NF1 patients are at an increased risk of malignancy and have a life expectancy about 10-15 years shorter than the general population. The mainstay of management of NF1 is a patient-centered longitudinal care with age-specific monitoring of clinical manifestations, aiming at the early recognition and symptomatic treatment of complications as they occur. Protocole national de diagnostic et de soins (PNDS) are mandatory French clinical practice guidelines for rare diseases required by the French national plan for rare diseases. Their purpose is to provide health care professionals with guidance regarding the optimal diagnostic and therapeutic management of patients affected with a rare disease; and thus, harmonizing their management nationwide. PNDS are usually developed through a critical literature review and a multidisciplinary expert consensus. The purpose of this article is to present the French guidelines on NF1, making them even more available to the international medical community. We further dwelled on the emerging new evidence that might have therapeutic potential or a strong impact on NF1 management in the coming feature. Given the complexity of the disease, the management of children and adults with NF1 entails the full complement healthcare providers and communication among the various specialties.
神经纤维瘤病 1 型是一种较为常见的遗传性疾病,全球发病率在 1/3000 至 1/6000 之间。该疾病影响多个系统,主要表现为皮肤、神经和骨骼方面的异常,导致较高的发病率或死亡率。实际上,NF1 患者发生恶性肿瘤的风险增加,预期寿命比普通人群短 10-15 年左右。NF1 的主要治疗方法是以患者为中心的纵向管理,根据年龄特异性监测临床表现,旨在早期识别和对症治疗并发症。《法国罕见病诊断与治疗规范》(PNDS)是法国罕见病国家计划要求的强制性法国临床实践指南,旨在为医疗保健专业人员提供有关受罕见病影响的患者最佳诊断和治疗管理的指导;从而在全国范围内协调其管理。PNDS 通常通过批判性文献回顾和多学科专家共识制定。本文的目的是介绍 NF1 的法国指南,使其更能为国际医学界所了解。我们进一步探讨了新出现的可能具有治疗潜力或对 NF1 管理产生重大影响的新证据。鉴于该疾病的复杂性,NF1 患儿和成年患者的管理需要配备齐全的医疗保健提供者,并需要各专业之间进行沟通。