A Phase II Clinical Trial to Evaluate the Safety and Efficacy of Sirolimus for Secondary Treatment of Chronic Graft-versus-Host Disease

Date de début de l'étude : 1 avril 2002

Inclusion Criteria: * Biopsy-confirmed diagnosis of clinical extensive chronic GVHD with inadequate response to previous treatment and where secondary systemic therapy is indicated because of * Clinical progression of signs and symptoms of chronic GVHD in a previously involved organ, or * Development of signs and symptoms of chronic GVHD in a previously uninvolved organ, or * Absence of improvement after 3 months of primary treatment, or * Continued need for treatment with prednisone at doses \>= 1.0 mg/kg/day for more than 2 months, without qualification for type of donor, graft or conditioning regimen * Patient or guardian able and willing to provide informed consent * Stated willingness to use contraception in women of child-bearing potential (Food and Drug Administration \[FDA\] requirement) * Stated willingness of the patient to comply with study procedures and reporting requirements * Stated willingness of the physician most involved in management of chronic GVHD (the "managing physician,") to comply with study procedures and reporting requirements Exclusion Criteria: * Fungal or viral infection with no radiographic evidence of improvement during continued appropriate antimicrobial therapy * Cytomegalovirus (CMV) antigenemia unresponsive to antiviral therapy * Active disseminated varicella zoster virus (VZV) infection with persistent non-crusted lesions * Inability to tolerate oral medications * Absolute neutrophil count (ANC) \< 1500/uL * Platelet count \< 50,000/uL * Persistent or recurrent malignancy, including histopathologic evidence of myeloma or lymphoma; patients with breakpoint cluster region-abelson (bcr/abl) detected by polymerase chain reaction (PCR) assay as the only evidence of persistent chronic myeloid leukemia may be enrolled * Pregnancy * Known history of hypersensitivity to sirolimus