Phase II trial of pirfenidone in adults with neurofibromatosis type 1

Babovic‐Vuksanovic, Dusica; Ballman, Karla V.; Michels, Virginia V.; McGrann, Pamela; Lindor, N. M.; King, Brett; Camp, Jon J.; Micic, V.; Babović, Nada; Carrero, Xiomara W.; Spinner, Robert J.; O’Neill, Brian Patrick

doi:10.1212/01.wnl.0000243231.12248.67

Cited by 63 publications

(34 citation statements)

References 6 publications

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“…Therefore, more sensitive methods, such as volume segmentation, were developed for the analysis of PN and schwannomas. [10][11][12][13][14][15] These methods documented the feasibility of reproducibly measuring tumor volumes in natural history studies and treatment trials for PN and VS. 13,14,[16][17][18][19] Using the MEDx-based lesion detection method developed for PN, interobserver differences were less than 10% and intraobserver variation was #5%. 10 For VS measured using the Vitrea2 workstation, the coefficient of variation ranged from 0.6% to 6.8%.…”

Section: Standard Criteria For Response Evaluation In Clinical Trialsmentioning

confidence: 99%

Recommendations for imaging tumor response in neurofibromatosis clinical trials

et al. 2013

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Methods: Currently used imaging endpoints, designs of NF clinical trials, and knowledge of the natural history of NF-related tumors, in particular PN and VS, were reviewed. Consensus recommendations for response evaluation for future studies were developed based on this review and the expertise of group members.Results: MRI with volumetric analysis is recommended to sensitively and reproducibly evaluate changes in tumor size in clinical trials. Volumetric analysis requires adherence to specific imaging recommendations. A 20% volume change was chosen to indicate a decrease or increase in tumor size. Use of these criteria in future trials will enable meaningful comparison of results across studies. Conclusions:The proposed imaging response evaluation guidelines, along with validated clinical outcome measures, will maximize the ability to identify potentially active agents for patients with NF and benign tumors. 1,2 Linear measurements are performed for their ease of use and are suitable for most malignant lesions that rapidly change in size. Disease-specific recommendations have been developed for some diseases in which linear measurements are not practical or meaningful, such as the Response Assessment in Neuro-Oncology (RANO) criteria for brain tumors.3,4 In addition, an international effort is under way to develop measurement guidelines for pediatric brain tumors, which will be applicable to neurofibromatosis type 1 (NF1)-related gliomas.

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Section: Standard Criteria For Response Evaluation In Clinical Trialsmentioning

confidence: 99%

Recommendations for imaging tumor response in neurofibromatosis clinical trials

et al. 2013

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“…Pirfenidone was then studied in a phase II clinical trial in 24 patients with NF1. Of the 17 individuals who completed 2 years of treatment, the tumor volume remained stable in 11, increased in 3 (!15% volume increase), and decreased in 3 (15%-29% volume decrease) (Babovic-Vuksanovic et al, 2006).…”

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confidence: 86%

Neurofibromatosis Type I: From Genetic Mutation to Tumor Formation

Thomas¹,

Vries²

2009

Handbook of Neurochemistry and Molecular Neurobiology

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“…Otro fármaco estudiado es pirfenidone (PFD), el cual ha sido utilizado previamente para el manejo de fibrosis pulmonar idiopática y neurofibromatosis debido a sus características antifibróti-cas y antiinflamatorias [47][48] .…”

Section: Terapia Basada En Medicamentos Antifibróticos Y Antiinflamatunclassified

Nuevos tratamientos para la infección por virus de hepatitis C y el proceso de fibrosis hepática

2009

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Novel treatments for hepatitis C viral infection and the hepatic fibrosis Hepatitis C virus (HCV) infection represents a global health problem due to its evolution to hepatic cirrhosis and hepatocellular carcinoma. The viral pathogenesis and infectious processes are not yet fully understood. The development of natural viral resistance towards the host immune system represents a major challenge for the design of alternative therapeutic interventions and development of viral vaccines. The molecular mechanisms of hepatic fibrosis are well described. New alternatives for the treatment of patients with HCV infection and hepatic cirrhosis are under intensive research. New drugs such as viral protease inhibitors and assembly inhibitors, as well as immune modulators have been studied in clinical trials. Additional alternatives include antifibrotic drugs, which reverse the hepatic cellular damage caused by HCV infection. This review makes reference to viral infective mechanisms, molecular pathways of liver fibrosis and overviews conventional and new treatments for HCV infection and liver fibrosis (Rev Méd Chile 2009; 137: 280-8

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Phase II trial of pirfenidone in adults with neurofibromatosis type 1

Cited by 63 publications

References 6 publications

Recommendations for imaging tumor response in neurofibromatosis clinical trials

Recommendations for imaging tumor response in neurofibromatosis clinical trials

Neurofibromatosis Type I: From Genetic Mutation to Tumor Formation

Nuevos tratamientos para la infección por virus de hepatitis C y el proceso de fibrosis hepática

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