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Hematopoietic Stem-Cell Transplantation for Rare Diseases in the Pediatric Population.


John M. Eisenberg Center for Clinical Decisions and Communications Science.


Comparative Effectiveness Review Summary Guides for Clinicians [Internet]. Rockville (MD): Agency for Healthcare Research and Quality (US); 2007-.
AHRQ Comparative Effectiveness Reviews.
2013 Sep 25.


A systematic review was undertaken to evaluate the evidence about the effectiveness, benefits, and adverse effects of using hematopoietic stem-cell transplantation (HSCT) to treat rare diseases in the pediatric population (age ≤ 21 years) for which no guidelines or recommendations based on extensive clinical study are available. HSCT was compared with standard therapies or the disease's natural history. The systematic review did not cover the pediatric indications for which substantive evidence supports the use of HSCT. The systematic review included 251 primary studies, mostly case series and case reports, published from January 1995 through August 2011. An online version of this summary provides links directly to the sections of the full report with references for individual findings, inclusion criteria for the studies, and an explanation of the methods for rating the studies and determining the strength of evidence for individual findings. The online version of this summary and the full report are available at

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