Management of refractory hemorrhagic cystitis following hematopoietic stem cell transplantation in children.

HC is a complication associated with HSCT, but occurs rarely in solid organ recipients. The reported incidence varies from <10% to more than 70%. HC is characterized by hemorrhagic inflammation in urinary tract mucosa with symptoms varying from asymptomatic microscopic hematuria to frank hematuria with clot formation and urinary tract obstruction. Early onset HC may be explained by toxicity of chemo- and/or radiotherapy, while multiple factors including viral infections and their interplay seem to be involved in late onset HC. So far, only incidence of cyclophosphamide-associated HC has been reduced with preventive treatment. Likely, once HC is established, the treatment principles are similar regardless of the etiology and depend on the intensity of HC. Prevention of urinary tract obstruction, transfusion support, analgesic, and spasmolytic therapy are generally accepted in HC management. Treatment beyond this conservative approach entails higher risk for side effects, and thus treatment escalation proportional to HC intensity is warranted. No standard and evidence-based treatment escalation algorithm has been widely adopted yet. As severe HC following HSCT is a potentially life-threatening complication, a multidisciplinary and individual approach is required in children suffering from this devastating complication.