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What does the 'Cochrane database of systematic reviews' tell us about hemophilia?

Emerito-Carlos Rodriguez-Merchan1

  • 1Department of Orthopaedic Surgery, La Paz University Hospital-IdiPaz, Madrid, Spain.

Expert Review of Hematology
|October 15, 2019
PubMed

Insights

Prophylaxis preserves joint function in children with hemophilia. Evidence suggests recombinant factor VII activated and activated prothrombin complex concentrates are similar in efficacy and safety for hemophilia treatment.

Area of Science:

  • Hematology
  • Evidence-based medicine
  • Systematic reviews

Background:

  • Hemophilia management relies on evidence from systematic reviews.
  • The Cochrane database of systematic reviews (CDBSR) is a key resource for hemophilia data.
  • Randomized controlled trials form the basis of evidence in the Cochrane Collaboration for hemophilia.

Purpose of the Study:

  • To review the hemophilia-related data available in the Cochrane database of systematic reviews.
  • To assess the quality and scope of evidence regarding hemophilia treatments.

Main Methods:

  • Systematic review of existing literature within the CDBSR.
  • Analysis of evidence from randomized controlled trials concerning hemophilia.

Main Results:

  • High-quality evidence supports prophylaxis over on-demand treatment for preserving joint function in children with hemophilia.
  • Recombinant factor VII activated (rFVIIa) and activated prothrombin complex concentrates (APPCs) demonstrate comparable efficacy and safety.
  • Antifibrinolytic therapy shows no proven benefit for preventing oral bleeding in hemophilia patients undergoing minor oral surgery.
  • The efficacy of gene therapy for hemophilia remains unproven.
  • Prophylaxis with bypassing agents may effectively reduce bleeding in hemophilic patients with inhibitors.

Conclusions:

  • Cochrane reviews provide high-level evidence for hemophilia management, particularly regarding prophylaxis.
  • Physicians should adhere to CDBSR recommendations for optimal hemophilia care.
  • Further research is needed to clarify the role of physical exercise and confirm gene therapy effectiveness in hemophilia.

Keywords:
Cochrane LibraryHemophiliadatabasesystematic reviewstreatment

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