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阿拉伯海湾合作委员会地区1型神经纤维瘤病的诊断与管理:挑战与建议

Diagnosis and management of neurofibromatosis type 1 in Arabian Gulf Cooperation Council Region: challenges and recommendations.

作者信息

Bashiri Fahad A, Hundallah Khaled, Abukhaled Musaad, Alyahya Mossaed Mohammed, Al Futaisi Amna, Alshowaeir Daniah, Al Tawari Asmaa, Abdullah Shaker, Maaz Ata Ur Rehman, AlShamsi Eman Taryam, Alshuaibi Walaa, Alotaibi Faisal, Aldhalaan Hesham

机构信息

Division of Pediatric Neurology, Department of Pediatrics, College of Medicine, King Saud University Medical City, King Saud University, Riyadh, Saudi Arabia.

Prince Sultan Military Medical City, Riyadh, Saudi Arabia.

出版信息

Front Oncol. 2024 Aug 27;14:1323176. doi: 10.3389/fonc.2024.1323176. eCollection 2024.

Abstract

Neurofibromatosis type 1 (NF1) is a complex multisystem genetic disorder that requires long-term, age-specific monitoring and multidisciplinary care. NF1 symptom burden can significantly affect the quality of life and impose a substantial economic burden on patients and their families. The approval and widespread availability of mitogen-activated protein kinase (MEK) inhibitors such as selumetinib for NF1-related plexiform neurofibromas have revolutionized the standard of care for patients with NF1, however their effective utilization hinges on early recognition of NF1. We present a consensus manuscript describing the challenges observed in the Arabian Gulf Cooperation Council (GCC) for diagnosing and managing NF1. Experts from the GCC also present recommendations for the early recognition and management of NF1 and its complications. A referral pathway that can play a crucial role in helping primary healthcare providers refer their patients to experts is also proposed. Increasing the availability and accessibility of genetic testing at an affordable cost and optimizing personalized NF1 care are essential for NF1 management. Developing regional guidelines for NF1 management and establishing NF1 centers of excellence may facilitate better care and outcomes for patients with NF1 in the GCC region.

摘要

1型神经纤维瘤病(NF1)是一种复杂的多系统遗传性疾病,需要进行长期、针对不同年龄段的监测和多学科护理。NF1的症状负担会显著影响生活质量,并给患者及其家庭带来沉重的经济负担。丝裂原活化蛋白激酶(MEK)抑制剂(如司美替尼)被批准用于治疗与NF1相关的丛状神经纤维瘤,且广泛可得,这彻底改变了NF1患者的护理标准,然而,它们的有效利用取决于对NF1的早期识别。我们发表了一篇共识文稿,描述了在阿拉伯海湾合作委员会(GCC)诊断和管理NF1过程中所观察到的挑战。GCC的专家们还提出了早期识别和管理NF1及其并发症的建议。还提出了一条转诊途径,它在帮助初级医疗保健提供者将患者转诊给专家方面可发挥关键作用。以可承受的成本提高基因检测的可及性和可用性,以及优化个性化的NF1护理,对于NF1的管理至关重要。制定NF1管理的区域指南并建立卓越的NF1中心,可能有助于为GCC地区的NF1患者提供更好的护理和治疗结果。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d521/11385870/dbd3c1ed5433/fonc-14-1323176-g001.jpg

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