Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas

Ina Ly; Carlos G. Romo; Sally Gottesman; Kristen M. Kelly; Deanna Kornacki; Zachary York; Sang Y. Lee; Steven D. Rhodes; Verena Staedtke; Matthew R. Steensma; Jaishri O. Blakeley; Pierre Wolkenstein

doi:10.1016/j.jid.2023.01.041

Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas

Ina Ly, Carlos G. Romo, Sally Gottesman, Kristen M. Kelly, Deanna Kornacki, Zachary York, Sang Y. Lee, Steven D. Rhodes, Verena Staedtke, Matthew R. Steensma, Jaishri O. Blakeley, Pierre Wolkenstein

School of Medicine

Research output: Contribution to journal › Review article › peer-review

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.

Original language	English (US)
Pages (from-to)	1388-1396
Number of pages	9
Journal	Journal of Investigative Dermatology
Volume	143
Issue number	8
DOIs	https://doi.org/10.1016/j.jid.2023.01.041
State	Published - Aug 2023

ASJC Scopus subject areas

Biochemistry
Molecular Biology
Dermatology
Cell Biology

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Ly, I., Romo, C. G., Gottesman, S., Kelly, K. M., Kornacki, D., York, Z., Lee, S. Y., Rhodes, S. D., Staedtke, V., Steensma, M. R., Blakeley, J. O., & Wolkenstein, P. (2023). Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas. Journal of Investigative Dermatology, 143(8), 1388-1396. https://doi.org/10.1016/j.jid.2023.01.041

Ly, I, Romo, CG, Gottesman, S, Kelly, KM, Kornacki, D, York, Z, Lee, SY, Rhodes, SD, Staedtke, V, Steensma, MR, Blakeley, JO & Wolkenstein, P 2023, 'Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas', Journal of Investigative Dermatology, vol. 143, no. 8, pp. 1388-1396. https://doi.org/10.1016/j.jid.2023.01.041

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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.",

author = "Ina Ly and Romo, {Carlos G.} and Sally Gottesman and Kelly, {Kristen M.} and Deanna Kornacki and Zachary York and Lee, {Sang Y.} and Rhodes, {Steven D.} and Verena Staedtke and Steensma, {Matthew R.} and Blakeley, {Jaishri O.} and Pierre Wolkenstein",

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year = "2023",

month = aug,

doi = "10.1016/j.jid.2023.01.041",

language = "English (US)",

volume = "143",

pages = "1388--1396",

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AU - Ly, Ina

AU - Romo, Carlos G.

AU - Gottesman, Sally

AU - Kelly, Kristen M.

AU - Kornacki, Deanna

AU - York, Zachary

AU - Lee, Sang Y.

AU - Rhodes, Steven D.

AU - Staedtke, Verena

AU - Steensma, Matthew R.

AU - Blakeley, Jaishri O.

AU - Wolkenstein, Pierre

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AB - 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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Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas

Abstract

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