Intravesical rhGM-CSF for the treatment of late onset hemorrhagic cystitisafter bone marrow transplant

In the present study, we assessed the clinical effect of recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) in the treatmen t of refractory, grade III-IV hemorrhagic cystitis (HC) in six patients who underwent bone marrow transplantation (BMT), These were four males and two females, aged 24-40 years (median age 30.5 years). All received allogeneic BMT from HLA-identical siblings after preparation with busulfan-cyclophosp hamide. HC was evident 24.5 days (range 15-33 days) after BMT, Median durat ion of HC before treatment was 5 days (range 49 days). Treatment consisted of intravesical instillation of rhGM-CSF (400 mu g) for 3 consecutive days, A complete response was observed in three patients, the other three showed a partial response. Median time to achieve response was 36 h (range 0.2-72 h), Hematuria was controlled after the first (two patients), second (two p atients) or third (two patients) dose of intravesical rhGM-CSF, Patients we re discharged from the hospital 10.5 days (range 3-41 days) after treatment , All patients have been followed for up to 10 months and none have require d further treatment, No systemic or bladder side-effects have been observed , Although our results indicate that intravesical instillation of rhGM-CSF is effective in the treatment of HC, a phase II clinical trial, including a larger series of patients, is needed.