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A Systematic Review of Recent and Ongoing Clinical Trials in Patients With the Neurofibromatoses.
Acar, Simge; Nieblas-Bedolla, Edwin; Armstrong, Amy E; Hirbe, Angela C.
Afiliação
  • Acar S; Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, Missouri; Koç University School of Medicine, Istanbul, Turkey.
  • Nieblas-Bedolla E; University of Washington School of Medicine, Seattle, Washington.
  • Armstrong AE; Division of Hematology and Oncology, Department of Pediatrics, Washington University School of Medicine, St. Louis, Missouri.
  • Hirbe AC; Division of Oncology, Department of Medicine, Washington University School of Medicine, St. Louis, Missouri. Electronic address: hirbea@wustl.edu.
Pediatr Neurol ; 134: 1-6, 2022 09.
Article em En | MEDLINE | ID: mdl-35759947
ABSTRACT

INTRODUCTION:

The neurofibromatoses comprise three different genetic conditions causing considerable morbidity and mortality neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2), and schwannomatosis (SWN). This review summarizes recent and ongoing clinical trials involving patients with neurofibromatoses to better understand the current state of clinical trial research centered around these conditions and inform areas of need.

METHODS:

A search was conducted using the Cochrane Central Register of Controlled Trials and clinicaltrials.gov databases. Inclusion and exclusion criteria were designed to identify clinical trials focused on patients with NF1, NF2, or SWN completed in or after 2010 and in process as of December 31, 2021. Information was collected using standardized guidelines.

RESULTS:

A total of 134 clinical trials were included, with 75 (56%) completed and 59 (44%) in process. For completed trials, 74% (n = 56) involved patients with NF1, and of those based on specific tumors (n = 26, 46%), the majority focused on plexiform neurofibromas (PNs) (n = 12, 46%). For ongoing trials, 79% (n = 47) involve patients with NF1, and of those based on specific tumors (n = 29, 61%), the majority are focused on PNs (n = 13, 45%).

CONCLUSION:

Both recent and ongoing clinical trials have primarily focused on patients with NF1 and the treatment of PNs. This research has led to the first FDA-approved drug for NF1-PN and has changed management of these tumors, allowing for systemic therapy rather than reliance on only a surgical modality. Trials evaluating comorbid psychiatric conditions and quality of life among patients with any of the neurofibromatoses appear less common. These areas may warrant focus in future studies to improve clinical management.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Neurofibromatose 2 / Neurofibromatose 1 / Neurofibromatoses / Neurofibroma Plexiforme / Neurilemoma Tipo de estudo: Clinical_trials / Guideline / Prognostic_studies / Systematic_reviews Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Neurofibromatose 2 / Neurofibromatose 1 / Neurofibromatoses / Neurofibroma Plexiforme / Neurilemoma Tipo de estudo: Clinical_trials / Guideline / Prognostic_studies / Systematic_reviews Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article