Making strides and meeting challenges in pediatric allogeneic hematopoietic cell transplantation clinical trials in the United States: Past, present and future

Over the past 20 years, the field of pediatric allogeneic hematopoietic cell transplantation has made ground-breaking strides in the successful treatment of patients with both malignant and non-malignant diseases. As the field advances, so does the need for high-quality studies including randomized controlled trials, aimed at answering clinically important questions about optimizing care and outcomes of children undergoing alloHCT. In an effort to actively address emerging clinical questions, three main cooperative groups in the U.S. have joined forces to develop and implement multiple clinical trials for pediatric alloHCT patients. These groups include the Blood and Marrow Transplant Clinical Trials Network, the Children's Oncology Group and the Pediatric Blood and Marrow Transplant Consortium. Though the field of stem cell transplantation continues to advance, conducting clinical trials in the pediatric population is a unique challenge and as a result, optimal outcomes have yet to be reached in this population. Because of the limited number of pediatric transplant patients at each institution in the U.S., trials aimed at answering important clinical questions still struggle to accrue acceptable numbers of patients in an appropriate amount of time and thus gathering statistically useful data has posed a challenge for the field. In an effort to mitigate some of the challenges associated with obtaining statistically and clinically meaningful information about pediatric alloHCT, the implementation of new cooperative group trials is active and ongoing.