biostudies-literatureHong KTMinistry of Food and Drug Safety48https://www.ebi.ac.uk/biostudies/studies/S-EPMC11934747biostudies-literatureUnknown13(1)<h4>Background</h4>Post-transplantation cyclophosphamide (PTCy) has paved the way for the increased use of alternative donors, including haploidentical familial donors, with acceptable engraftment and graft-versus-host disease (GVHD) rates. However, pharmacokinetic studies of PTCy in the pediatric population following myeloablative conditioning regimens are scarce.<h4>Methods</h4>We conducted a prospective and comprehensive pharmacokinetic analysis of pre- and post-transplantation cyclophosphamide levels in pediatric patients undergoing haploidentical hematopoietic stem cell transplantation (HSCT) using a myeloablative busulfan-based conditioning regimen. A total of 14 samples were collected from each patient. Plasma concentrations of cyclophosphamide and carboxycyclophosphamide were analyBiomarker researchPharmacokinetics of post-transplant cyclophosphamide and its associations with clinical outcomes in pediatric haploidentical hematopoietic stem cell transplantation.PMC1193474721184MFDS286Hong KTSunwoo YCho JYChoi JYKang HJBae SOh JYu KSLee JPark HJKim BKfalsePharmacokinetics of post-transplant cyclophosphamide and its associations with clinical outcomes in pediatric haploidentical hematopoietic stem cell transplantation.<h4>Background</h4>Post-transplantation cyclophosphamide (PTCy) has paved the way for the increased use of alternative donors, including haploidentical familial donors, with acceptable engraftment and graft-versus-host disease (GVHD) rates. However, pharmacokinetic studies of PTCy in the pediatric population following myeloablative conditioning regimens are scarce.<h4>Methods</h4>We conducted a prospective and comprehensive pharmacokinetic analysis of pre- and post-transplantation cyclophosphamide levels in pediatric patients undergoing haploidentical hematopoietic stem cell transplantation (HSCT) using a myeloablative busulfan-based conditioning regimen. A total of 14 samples were collected from each patient. Plasma concentrations of cyclophosphamide and carboxycyclophosphamide were analy2025-01-01T00:00:00Z2025 Mar2025-06-25T03:04:12.977Z2025-06-25T03:04:12.977ZS-EPMC119347474012891910.1186/s40364-025-00749-3