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Future Research Needs for Hematopoietic Stem-Cell Transplantation in the Pediatric Population

Systematic Review ARCHIVED Feb 1, 2012

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Archived: This report is greater than 3 years old. Findings may be used for research purposes, but should not be considered current.

Excerpt

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.

Project Timeline

Future Research Needs for Hematopoietic Stem-Cell Transplantation in the Pediatric Population

Jan 27, 2012
Topic Initiated
Feb 1, 2012
Systematic Review Archived
Page last reviewed December 2019
Page originally created November 2017

Internet Citation: Systematic Review: Future Research Needs for Hematopoietic Stem-Cell Transplantation in the Pediatric Population. Content last reviewed December 2019. Effective Health Care Program, Agency for Healthcare Research and Quality, Rockville, MD.
https://effectivehealthcare.ahrq.gov/products/stem-cell-children-future/research

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