Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study
- Determine the effect of chronic vinblastine and methotrexate on time to disease
progression in children or young adults with progressive plexiform neurofibroma
associated with neurofibromatosis type 1.
- Determine the objective response rate in patients treated with this regimen.
- Determine the toxic effects of this regimen in these patients.
- Determine the quality of life of patients treated with this regimen.
OUTLINE: Patients are stratified according to tumor status (severely debilitating and/or
life-threatening vs cosmetically disfiguring).
Patients receive methotrexate and vinblastine IV weekly for 26 weeks and then every 2 weeks
for 26 weeks in the absence of disease progression or unacceptable toxicity.
Quality of life is assessed at baseline and then every 3 months during study participation.
Patients are followed every 3 months until disease progression.
PROJECTED ACCRUAL: A total of 35 patients will be accrued for this study within
approximately 3 years.
Masking: Open Label, Primary Purpose: Treatment
Time to disease progression after 6 months
Jean B. Belasco, MD
Children's Hospital of Philadelphia
|Children's Hospital of Philadelphia||Philadelphia, Pennsylvania 19104|