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Cover of Hematopoietic Stem-Cell Transplantation in the Pediatric Population

Hematopoietic Stem-Cell Transplantation in the Pediatric Population

Comparative Effectiveness Reviews, No. 48

Investigators: Thomas A Ratko, PhD, Suzanne E Belinson, MPH, PhD, Heather M Brown, MD, Hussein Z Noorani, MS, Ryan D Chopra, MPH, Anne Marbella, MS, David J Samson, MS, Claudia J Bonnell, RN, MLS, Kathleen M Ziegler, PharmD, and Naomi Aronson, PhD.

Blue Cross and Blue Shield Association Technology Evaluation Center Evidence-based Practice Center
Rockville (MD): Agency for Healthcare Research and Quality (US); 2012 Feb.
Report No.: 12-EHC018-EF
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Structured Abstract

Objectives:

Assess comparative benefits and harms of hematopoietic stem-cell transplantation (HSCT) versus standard therapies or disease natural history in pediatric (age ≤21 years) patients with malignant solid tumors, inherited metabolic diseases, or autoimmune diseases.

Data Sources:

MEDLINE®, Embase, and the Cochrane Database of Systematic Reviews weresearched from January 1995 through August 2011. Additional studies were identified from reference lists and technical experts.

Review Methods:

Major data abstraction elements were patient and treatment characteristics, health outcomes (overall survival, remission, neurocognitive development, adverse events), and data analysis. The strength of the body of evidence for each indication was assessed according to the process developed by the Evidence-based Practice Center (EPC) Program of the Agency for Healthcare Research and Quality using four required domains specified in the EPC Methods Guide for Comparative Effectiveness Reviews: risk of bias, consistency, directness, and precision. In cases where there were no head-to-head comparative studies, directness was based on the outcome (e.g., overall survival) rather than on the comparison. For small series or a compilation of case reports in which the prognosis absent HSCT is uniformly fatal (e.g., Wolman's disease), the known natural history was considered an indirect comparator. An optional domain, strength of association (SOA, magnitude of effect) was applied to the body of evidence when there was an apparent benefit or harm, increasing the overall strength beyond what normally may be considered appropriate for such evidence. SOA was deemed not applicable for diseases where there was no clear evidence of benefit or harm with HSCT versus comparators, or if results of individual studies within a body of literature were inconsistent or conflicted. No quantitative scoring method was applied.

Results:

Among 6,416 records screened, 251 primary studies were included. The strength of evidence for specific indications was graded as high for 2 indications, moderate or low for 19, and insufficient for 39.

  • Evidence suggesting a benefit of HSCT for overall survival:
    • Wolman's disease compared to disease natural history (high strength)
    • Recurrent/progressive anaplastic astrocytoma compared to conventional therapy (low strength)
  • Evidence suggesting a benefit of HSCT for neuromuscular symptoms:
    • Farber's disease Type 2/3 compared to symptom management and disease natural history (high strength)
  • Evidence suggesting a benefit of HSCT for neurocognitive symptoms:
    • Infantile ceroid lipofuscinosis compared to symptom management or disease natural history (low strength)
    • Attenuated form of MPS (mucopolysaccharoidosis) II (Hunter's disease) compared to enzyme-replacement therapy (ERT) (low strength)
  • Evidence suggesting a benefit of HSCT for neurodevelopmental symptoms:
    • Attenuated and severe forms of MPS II (Hunter's disease) compared to ERT (both low strength)
  • Evidence suggesting no benefit of single HSCT for overall survival:
    • Metastatic rhabdomyosarcoma compared to conventional therapy (moderate strength)
    • Extraocular retinoblastoma with central nervous system involvement, high-risk Ewing's sarcoma family of tumors, high-risk relapsed Wilm's tumor compared to conventional therapy (all three low strength)
    • Niemann-Pick Type A compared to symptom management (low strength)
  • Evidence suggesting no benefit of HSCT for neurodevelopmental symptoms:
    • Gaucher Type III compared to ERT (low strength)
    • Juvenile form of GM1, juvenile Tay-Sachs compared to symptom management or disease natural history (both low strength)
    • MPS III (Sanfilippo) compared to symptom management, substrate reduction therapy, or disease natural history (low strength)
  • Evidence suggesting no benefit of HSCT for neurocognitive symptoms:
    • Severe form of MPS II (Hunter's disease) compared to symptom management or disease natural history (low strength)
    • MPS III (Sanfilippo) compared to symptom management, substrate reduction therapy, or disease natural history (low strength)
    • Gaucher Type III compared to ERT (moderate strength)
  • Evidence suggesting harm of HSCT for overall survival:
    • Nonanaplastic mixed or unspecified ependymoma compared to conventional therapy (both low strength)

Conclusions:

Evidence demonstrating benefit or harm of HSCT versus standard therapies or disease natural history was insufficient for most pediatric indications.

Contents

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

Suggested citation:

Ratko TA, Belinson SE, Brown HM, Noorani HZ, Chopra RD, Marbella A, Samson DJ, Bonnell CJ, Ziegler KM, Aronson N. Hematopoietic Stem-Cell Transplantation in the Pediatric Population. Comparative Effectiveness Review No. 48. (Prepared by the Blue Cross and Blue Shield Association Technology Evaluation Center Evidence-based Practice Center under Contract No. HHSA 290-2007-10058.) AHRQ Publication No. 12-EHC018-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 290-2007-10058). 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 decisionmakers—patients and clinicians, health system leaders, and policymakers, among others—make well-informed decisions and thereby improve the quality of health care services. This report is not intended to be a substitute for the application of clinical judgment. Anyone who makes decisions concerning the provision of clinical care should consider this report in the same way as any medical reference and in conjunction with all other pertinent information, i.e., in the context of available resources and circumstances presented by individual patients.

This report may be used, in whole or in part, as the basis for development of clinical practice guidelines and other quality enhancement tools, or as a basis for reimbursement and coverage policies. AHRQ or U.S. Department of Health and Human Services endorsement of such derivative products may not be stated or implied.

None of the investigators has any affiliations or financial involvement that conflicts with the material presented in this report.

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540 Gaither Road, Rockville, MD 20850; www​.ahrq.gov

AHRQ (US Agency for Healthcare Research and Quality)

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