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

Abstract

In the present study, we assessed the clinical effect of recombinant human granulocyte–macrophage colony-stimulating factor (rhGM-CSF) in the treatment 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–cyclophosphamide. HC was evident 24.5 days (range 15–33 days) after BMT. Median duration of HC before treatment was 5 days (range 4–9 days). Treatment consisted of intravesical instillation of rhGM-CSF (400 μg) 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 patients) or third (two patients) dose of intravesical rhGM-CSF. Patients were 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 required 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.