Purpose:

Cyclosporine A (CsA) is the cornerstone prophylactic drug for graft versus host disease (GVHD) in allogeneic hematopoietic stem cell transplantation (allo-HSCT); however, its optimal trough level is yet to be determined. Therefore, in this study, we focused on the CsA trough levels and estimated their association with acute GvHD (aGVHD) risk in a consecutive cohort of 72 pediatric patients receiving allo-HSCT.

Method:

The trough CsA level was monitored 3–4 times in a week via mass spectrometry analysis during medication. The occurrence of GVHD, the trough of CsA level before and after allo-HSCT and other clinical information were recorded.

Results:

The cumulative incidence of aGVHD at 100 days was 19.44% for grade I and 23.61% for grades II–IV. Multivariable Cox regression analysis revealed that the optimal trough CsA level for aGVHD prophylaxis was >119 ng/mL, 146–214.5g/mL, >123.25 ng/mL, and 100.2–166 ng/mL on the −3 rd day, 3 rd day, 1 st week, and 2 nd month after HSCT, respectively. None of the cutoff values for CsA were significantly associated with the survival outcome.

Conclusion:

Our findings indicate that adequate management of CsA levels during the engraftment period might improve the clinical outcomes for pediatric patients undergoing hematopoietic stem cell transplantation. Clinical trial registration: China Clinical Trial Registration Center (ChiCTR2000034702). Registered 15 July 2020.