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.

Contents

• Preface
• Acknowledgments
• Key Informants
• Executive Summary
• Background
  • Clinical Context
  • Evidence Gaps
• Methods
  • Identification of Evidence Gaps
  • Criteria for Prioritization
  • Methods for Ranking Research Gaps
  • Engagement of Stakeholders, Researchers, Funders
  • Research Question Development and Study Design Considerations
• Results
  • Research Needs
• Discussion
• Conclusions
• References
• Acronyms
• Appendixes
  • Appendix A Key Questions From Comparative Effectiveness of HSCT for Pediatrics
  • Appendix B Conclusions From Draft Comparative Effectiveness Review of HSCT
  • Appendix C Conclusions for Which There Was Insufficient Evidence
  • Appendix D Search Strategy for Recently Published and Ongoing Studies
  • Appendix E Ongoing Studies
  • Appendix F Effective Health Care Program Selection Criteria
  • Appendix G Tool Used To Solicit KI Research Gap Ratings and Key Questions Via SurveyMonkey^® (June 16, 2011)
  • Appendix H Research Gap Rankings

Prepared for: Agency for Healthcare Research and Quality, U.S. Department of Health and Human Services¹, Contract No. HHSA 290-2007-10058I. Prepared by: Blue Cross and Blue Shield Association Technology Evaluation Center Evidence-based Practice Center, Chicago, IL

Suggested citation:

Belinson S, Mauger Rothenberg B, Chopra R, Aronson N. Future Research Needs for Hematopoietic Stem-Cell Transplantation in the Pediatric Population. Future Research Needs Paper No. 10. (Prepared by Blue Cross and Blue Shield Technology Evaluation Center Evidence-based Practice Center under Contract No. HHSA 290-2007-10058I.) AHRQ Publication No. 12-EHC019-EF. Rockville, MD: Agency for Healthcare Research and Quality; February 2012. www.effectivehealthcare.ahrq.gov/reports/final.cfm.

This report is based on research conducted by the Blue Cross and Blue Shield Association Technology Evaluation Center Evidence-based Practice Center (EPC) under contract to the Agency for Healthcare Research and Quality (AHRQ), Rockville, MD (Contract No. HHSA 290-2007-10058I). The findings and conclusions in this document are those of the authors, who are responsible for its contents; the findings and conclusions do not necessarily represent the views of AHRQ. Therefore, no statement in this report should be construed as an official position of AHRQ or of the U.S. Department of Health and Human Services.

The information in this report is intended to help health care researchers and funders of research make well-informed decisions in designing and funding research and thereby improve the quality of health care services. This report is not intended to be a substitute for the application of scientific judgment. Anyone who makes decisions concerning the provision of clinical care should consider this report in the same way as any medical research and in conjunction with all other pertinent information, i.e., in the context of available resources and circumstances.

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