| Summary: | For many pediatric indications, the use of hematopoietic stem-cell transplantation (HSCT) is not supported by high-quality trial data. In the absence of these data, the comparative effectiveness and harms of HSCT for these indications is uncertain. The Blue Cross and Blue Shield Association Technology Evaluation Center (BCBSA TEC) Evidence-based Practice Center (EPC) submitted a draft Comparative Effectiveness Review (CER) on HSCT in the pediatric population to the Agency for Healthcare Research and Quality. The six Key Questions addressed by the review evaluated the comparative effectiveness and/or harms of HSCT and alternative therapies for pediatric patients with (1) malignant solid tumors, (2) inherited metabolic diseases, and (3) autoimmune diseases. Overall there was a low to moderate strength of evidence for the various diseases and outcomes considered. Evidence consisted largely of case series and case reports. There was a preponderance of small uncontrolled studies, as is often the case in the study of rare diseases. Data synthesis was qualitative. Pooling was not attempted, as the data were not amenable to this approach. An effort was made to identify subgroups based on prognostic factors to see if these subgroups showed patterns of treatment success or failure. The objective of this Future Research Needs project was to systematically prioritize research gaps in the areas of HSCT for pediatric malignant solid tumors, inherited metabolic diseases, and autoimmune diseases, and to develop a list of research questions to address the prioritized gaps
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