Combination therapy for refractory idiopathic thrombocytopenic purpura in adolescents

PURPOSE: To investigate combined immunosuppressive therapy with vincristine, methylprednisolone, and prolonged cyclosporine in adolescents with refractory idiopathic thrombocytopenic purpura (ITP). PATIENTS AND METHODS: Ten adolescent patients with ITP refractory to previous medical management, including gluco-corticosteroid, intravenous immunoglobulin or anti-Rh (D) IgG, or splenectomy, were treated with combination immunosuppressive therapy at the University of Michigan between 1997 and 2001. Therapy consisted of weekly doses of vincristine 1.5 mg/m intravenous push (IVP) (maximum dose 2 mg), weekly methylprednisolone 100 mg/m IVP, and cyclosporine (CSA) 5 mg/kg orally twice daily (goal: CSA trough of 100-200 mg/mL). Vincristine and methylprednisolone were given weekly until the platelet count was greater than 50,000/mm for a minimum of 2 doses and a maximum of 4 doses. CSA was continued until the platelet count was normal for 3 to 6 months. RESULTS: Seven patients had continuous complete responses (platelet count normal after cessation of CSA), a median of 13 months (9-37 months) since completion of therapy. One patient had a partial response (platelet count 80-120 x 10 /L off CSA for 3 months). Two patients were nonresponders (platelet count <40 x 10 /L), one of whom had all therapy discontinued after 2 weeks due to peripheral neuropathy. The median time to response was 7 days (range 7-67 days). CSA was administered for a median of 4 months (range 0.5-19 months). CONCLUSIONS: A combination immunosuppressive approach that includes prolonged cyclosporine therapy may be promising for refractory ITP and is associated with sustained disease remissions in some patients.