Cidofovir Treatment for Prevention of BK Virus-Associated Hemorrhagic Cystitis in Pediatric Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation.

OBJECTIVE

In this prospective observational study, we aim to investigate the role of cidofovir (CDV) as prophylactic treatment in preventing BK virus (BKV)-associated hemorrhagic cystitis (HC) in pediatric patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT).

METHODS

This study enrolled 28 patients who received prophylactic treatment with CDV following allogeneic hematopoietic stem cell transplantation (Allo-HSCT) and 25 Allo-HSCT patients who received conventional supportive care, which included hydration and analgesics, from January 2020 to January 2022. Patients in the CDV prophylactic treatment group received intravenous injections of CDV at a dose of 1 mg/kg once a week for 4 weeks. PCR was used to detect the copy number of BKV (BKV-DNA) in the urine of all study participants at weeks 1, 2, 3, 4, and 5 after HSCT.

RESULTS

From the third week onward, the BKV copy numbers in the CDV prophylactic treatment group were significantly lower than those in the conventional treatment group at subsequent time points. The proportion of patients with acute graft-versus-host disease (aGVHD), cytomegalovirus infection, and BKV copy numbers was significantly lower in the CDV group compared to the conventional treatment group. Additionally, the proportion of patients receiving CDV prophylactic treatment was significantly higher in the non-HC group. Receiver operating characteristic curve analysis indicated that BKV copy numbers could predict the occurrence and severity of HC. Furthermore, logistic regression analysis identified aGVHD, BKV copy numbers, and CDV prophylactic treatment as risk factors for the occurrence of HC in patients after Allo-HSCT.

CONCLUSION

We identified for the first time in the literature that prophylactic treatment with CDV could significantly reduce the incidence of BKV-associated hemorrhagic cystitis.

© 2025 Wiley Periodicals LLC.