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Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas.
Ly, Ina; Romo, Carlos G; Gottesman, Sally; Kelly, Kristen M; Kornacki, Deanna; York, Zachary; Lee, Sang Y; Rhodes, Steven D; Staedtke, Verena; Steensma, Matthew R; Blakeley, Jaishri O; Wolkenstein, Pierre.
Afiliación
  • Ly I; Stephen E. and Catherine Pappas Center for Neuro-Oncology, Massachusetts General Hospital, Boston, Massachusetts, USA. Electronic address: ily@partners.org.
  • Romo CG; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Gottesman S; Neurofibromatosis Therapeutic Acceleration Program (NTAP), Baltimore, Maryland, USA; Children's Tumor Foundation, New York, New York, USA.
  • Kelly KM; Department of Dermatology, School of Medicine, University of California, Irvine, Irvine, California, USA.
  • Kornacki D; Incyte Corporation, Wilmington, Delaware, USA.
  • York Z; Independent, Neurofibromatosis type 1 patient.
  • Lee SY; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Rhodes SD; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, Indiana, USA.
  • Staedtke V; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Steensma MR; Helen Devos Children's Hospital, Spectrum Health System, Grand Rapids, Michigan, USA.
  • Blakeley JO; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Wolkenstein P; Department of Dermatology, Henri Mondor Hospital, University Paris East Créteil, Créteil, France.
J Invest Dermatol ; 143(8): 1388-1396, 2023 08.
Article en 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.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Neoplasias Cutáneas / Neurofibromatosis 1 / Neurofibroma Aspecto: Patient_preference Límite: Adult / Humans Idioma: En Revista: J Invest Dermatol Año: 2023 Tipo del documento: Article
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Neoplasias Cutáneas / Neurofibromatosis 1 / Neurofibroma Aspecto: Patient_preference Límite: Adult / Humans Idioma: En Revista: J Invest Dermatol Año: 2023 Tipo del documento: Article