Optimal treatment and timing of routine surveillance in children after allogeneic hematopoietic cell transplantaion
Wednesday, November 7, 2018 - 11:00am - 11:30am
In pediatric allogeneic hematopoietic cell transplantation (alloHCT), most of the guildelines for treatments and timing of routine surveillance originally developed for the adult patients. It has been challenging how to use such guidelines for individualized treatment for pediatric patients. I will use three examples to illustrate how to use simple statistical methods to tackle the challenging issues. The first example is on the treatment on acute graft-versus-host disease. Only a few studies in children have evaluated the efficacy of prophylactic regimens using tacrolimus on acute graft-versus-host disease. As a result, optimal tacrolimus levels in children after matched sibling donor allogeneic hematopoietic cell transplantation are not well defined. Using a dataset on 60 children during weeks 1 to 4 after alloHCT, we have identified optimal lower cutoff levels of tacrolimus using area under curve analysis. The second example is on the bone marrow harvest in pediatric sibling donors. The National Marrow Donor Program developed guidelines specifying that up to 20mL/kg of bone marrow can be harvested from adult donors. Using a data on 92 bone marrow harvests and clinical outcomes for 69 sibling recipient-donor duos, we have developed a guideline to optimize bone marrow harvest volume from pediatric matched sibling donors using receiver operating characteristic curve analysis with Youden's index criterion. The third example is on the utility and optimal timing of routine bone marrow and cerebrospinal fluid surveillance. Using a retrospective review of 108 childhood leukemia patients after alloHCT at Columbia, we have examined the existing routine practice and identified potential optimal surveillance time points using cumulative incidence analysis.