A Phase II Study of Imatinib Mesylate in Children and Adults With Sclerotic Skin Changes of Chronic Graft-Versus-Host Disease

Detailed Summary

Background: Chronic graft-versus-host disease (GVHD) is a common complication of stem cell transplant, resulting from the donor's immune cells attacking the cells of the body of the recipient. One effect of GVHD is fibrosis (scarring) of the skin that can lead to impaired function, decreased quality of life and increased risk of death. This is known as sclerotic skin changes of GVHD, or sclerodermatous graft versus host disease (ScGVHD). Imatinib mesylate (Gleevec) is a drug that has been approved by the Food and Drug Administration to treat cancer in humans and fibrosing conditions in animals. Objectives: To see if imatinib mesylate can improve ScGVHD and evaluate its effect on other GVHD symptoms To assess the side effects of imatinib mesylate in patients with GVHD To evaluate blood, body fluids and tissue samples in patients to try to better understand the biology of ScGVHD Eligibility: Patients 4 years of age and older with ScGVHD Design: Initial treatment: Participants take imatinib mesylate tablets once a day for up to 6 months, as long as their GVHD does not get worse and they do not develop unacceptable side effects of the drug. Evaluations: Participants are evaluated at 1, 3 and 6 months at the National Institutes of Health (NIH) Clinical Center with procedures that may include the following: Medical history and physical examination Blood and urine tests Lung function test Skin biopsy Magnetic resonance imaging (MRI) scan Specialty consultations (e.g., physical or rehabilitative therapy, dentist, eye doctor, dermatologist) Electrocardiogram (EKG) Echocardiogram (ultrasound test of the heart) Muga scan (nuclear medicine test of the heart) Quality-of-life questionnaires Apheresis (procedure for collecting quantities of white blood cells) Office visits with local physician once a week for 1 month, then once every 2 weeks for 5 months Followup visits at National Institutes of Health (NIH) every 6 months for 1 year Continuing treatment: Patients who improve continue to receive imatinib mesylate for up to 6 months after their best response and are followed for up to 2 years. Patients who continue to respond or who become worse after stopping treatment may receive additional treatment for up to 2 years.

Study Details

Timeline

Interventions