Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas.
J Invest Dermatol
; 143(8): 1388-1396, 2023 08.
Article
em En
| MEDLINE
| ID: mdl-37294242
ABSTRACT
Cutaneous neurofibromas (cNFs) are benign tumors of the skin that affect >95% of adults with neurofibromatosis type 1. Despite their benign histology, cNFs can significantly impact QOL due to disfigurement, pain, and pruritus. There are no approved therapies for cNFs. Existing treatments are limited to surgery or laser-based treatments that have had mixed success and cannot be readily applied to a large number of tumors. We review cNF treatment options that are currently available and under investigation, discuss the regulatory considerations specific to cNFs, and propose strategies to improve cNF clinical trial design and standardize clinical trial endpoints.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Neoplasias Cutâneas
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Neurofibromatose 1
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Neurofibroma
Limite:
Adult
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Humans
Idioma:
En
Revista:
J Invest Dermatol
Ano de publicação:
2023
Tipo de documento:
Article