Potential endpoints for assessment of bone health in persons with neurofibromatosis type 1.
Neurofibromatosis type 1
bone
bone mineral density
clinical trials
osteoporosis
Journal
Clinical trials (London, England)
ISSN: 1740-7753
Titre abrégé: Clin Trials
Pays: England
ID NLM: 101197451
Informations de publication
Date de publication:
Feb 2024
Feb 2024
Historique:
medline:
13
2
2024
pubmed:
29
9
2023
entrez:
29
9
2023
Statut:
ppublish
Résumé
Neurofibromatosis type 1 is a genetic syndrome characterized by a wide variety of tumor and non-tumor manifestations. Bone-related issues, such as scoliosis, tibial dysplasia, and low bone mineral density, are a significant source of morbidity for this population with limited treatment options. Some of the challenges to developing such treatments include the lack of consensus regarding the optimal methods to assess bone health in neurofibromatosis type 1 and limited data regarding the natural history of these manifestations. In this review, the Functional Committee of the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration: (1) presents the available techniques for measuring overall bone health and metabolism in persons with neurofibromatosis type 1, (2) reviews data for use of each of these measures in the neurofibromatosis type 1 population, and (3) describes the strengths and limitations for each method as they might be used in clinical trials targeting neurofibromatosis type 1 bone manifestations. The Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration supports the development of a prospective, longitudinal natural history study focusing on the bone-related manifestations and relevant biomarkers of neurofibromatosis type 1. In addition, we suggest that the neurofibromatosis type 1 research community consider adding the less burdensome measurements of bone health as exploratory endpoints in ongoing or planned clinical trials for other neurofibromatosis type 1 manifestations to expand knowledge in the field.
Identifiants
pubmed: 37772407
doi: 10.1177/17407745231201338
pmc: PMC10920397
mid: NIHMS1928604
doi:
Types de publication
Review
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
29-39Subventions
Organisme : NICHD NIH HHS
ID : P50 HD105351
Pays : United States
Organisme : Intramural NIH HHS
ID : Z99 CA999999
Pays : United States
Déclaration de conflit d'intérêts
Declaration of conflicting interestsThe author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: AMG and LJK have served as unpaid advisors for AstraZeneca/Alexion; AMG has been an unpaid advisor for SpringWorks Therapeutics. DAS has been a paid consultant for Alexion. HS is CEO of Infixion Bioscience, Inc., a pharmaceutical R&D company targeting NF1.
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