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Bone Marrow or Blood Stem Cell Transplants in Children With Certain Rare Inherited Metabolic Diseases: A Review of the Research for Parents and Caregivers

This summary will cover: Information about each rare inherited metabolic disease Possible benefits of an HSCT What researchers have found about treating children who have one of these diseases with an HSCT using stem cells from a donor What an HSCT is and how it is done Possible risks of an HSCT

Comparative Effectiveness Review Summary Guides for Consumers [Internet] - Agency for Healthcare Research and Quality (US).

Version: September 25, 2013

Hematopoietic Stem-Cell Transplantation in the Pediatric Population [Internet]

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.

Comparative Effectiveness Reviews - Agency for Healthcare Research and Quality (US).

Version: February 2012
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Hematopoietic Stem-Cell Transplantation for Rare Diseases in the Pediatric Population

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 www.effectivehealthcare.ahrq.gov/stem-cell-children.cfm.

Comparative Effectiveness Review Summary Guides for Clinicians [Internet] - Agency for Healthcare Research and Quality (US).

Version: September 25, 2013

Drug Class Review: HMG-CoA Reductase Inhibitors (Statins) and Fixed-dose Combination Products Containing a Statin: Final Report Update 5 [Internet]

In the United States, coronary heart disease and cardiovascular disease account for nearly 40% of all deaths each year. Coronary heart disease continues to be the leading cause of mortality and a significant cause of morbidity among North Americans. In 2006, coronary heart disease claimed 607 000 lives, translating into about 1 out of every 5 deaths in the United States. High levels of cholesterol, or hypercholesterolemia, are an important risk factor for coronary heart disease. The 3-hydroxy-3-methylglutaryl-coenzyme (HMG-CoA) reductase inhibitors, also known as statins, are the most effective class of drugs for lowering serum low-density lipoprotein cholesterol concentrations. They are first-line agents for patients who require drug therapy to reduce serum low-density lipoprotein cholesterol concentrations. The purpose of this review is to compare the benefits and harms of different statins in adults and children with hypercholesterolemia.

Drug Class Reviews - Oregon Health & Science University.

Version: November 2009
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Combination Therapy Versus Intensification of Statin Monotherapy: An Update [Internet]

To assess the benefits and harms of combination of statin and other lipid-modifying medication compared to intensification of statin monotherapy. This is an update to a 2009 review.

Comparative Effectiveness Reviews - Agency for Healthcare Research and Quality (US).

Version: February 2014
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Systematic Reviews in PubMed

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