EurekaMag.com logo
+ Site Statistics
References:
53,869,633
Abstracts:
29,686,251
+ Search Articles
+ Subscribe to Site Feeds
EurekaMag Most Shared ContentMost Shared
EurekaMag PDF Full Text ContentPDF Full Text
+ PDF Full Text
Request PDF Full TextRequest PDF Full Text
+ Follow Us
Follow on FacebookFollow on Facebook
Follow on TwitterFollow on Twitter
Follow on LinkedInFollow on LinkedIn

+ Translate

RCTs and observational studies to determine the effect of prophylaxis in severe haemophilia



RCTs and observational studies to determine the effect of prophylaxis in severe haemophilia



Haemophilia 13(4): 345-350



It has been suggested that more RCTs should be conducted in the evaluation of haemophilia treatment, including prophylaxis. To draw valid conclusions from experimental or observational studies, internal validity should be ensured. In particular, similarity of prognosis between treatment groups compared in a particular study is needed. Because the use of prophylaxis in observational studies is nonrandomized by definition, special efforts should be made to achieve comparability of prognosis, that is, to avoid 'confounding by indication'; in RCTs this is ensured by randomized allocation of treatment. However, the high costs of an RCT results in a limitation of both patient numbers and length of follow up. Observational studies tend to be more generalizable and cheaper, as they may include a wider spectrum of morbidities, use treatment protocols in agreement with routine care, and use available data. Therefore, observational studies can be much larger and of longer duration than RCTs; and are the preferred design for the evaluation of long term treatment effects. Several valid observational studies comparing prophylaxis and on demand treatment have reported both a short term reduction of 75-90% in bleeding frequency, and significant improvement in arthropathy, quality of life, and socioeconomic parameters after more than 20 years of prophylactic treatment. RCTs provide the most powerful tool for the evaluation of short term effects of any treatment, but are infeasible for the evaluation of long term effects. The definitive assessment of the long-term benefits of prophylaxis requires observational studies that should be performed according to rigorous standards.

(PDF emailed within 0-6 h: $19.90)

Accession: 055333889

Download citation: RISBibTeXText

PMID: 17610546

DOI: 10.1111/j.1365-2516.2007.01521.x



Related references

Systematic Review of Observational Studies and Rcts of Omalizumab in Severe Persistent Allergic Asthma and Meta-Analysis Feasibility Assessment. Value in Health 17(7): A589-A589, 2014

Survey of current prophylaxis practices and bleeding characteristics of children with severe haemophilia A in US haemophilia treatment centres. Haemophilia 18(1): 63-68, 2012

North American prophylaxis studies for persons with severe haemophilia: background, rationale and design. Haemophilia 9 Suppl 1: 44-8; Discussion 49, 2003

An International Prophylaxis Study Group (IPSG) survey of prophylaxis in adults with severe haemophilia. Haemophilia 23(5): E447-E450, 2017

Patient powered prophylaxis: A 12-month study of individualized prophylaxis in adults with severe haemophilia A. Haemophilia 23(6): 877-883, 2017

Cost-effectiveness analysis of pharmacokinetic-driven prophylaxis vs. standard prophylaxis in patients with severe haemophilia A. Blood Coagulation & Fibrinolysis 28(6): 425-430, 2016

An International Prophylaxis Study Group (IPSG) survey of prophylaxis in inhibitor positive children/adults with severe haemophilia. Haemophilia 23(5): E444-E447, 2017

Cost-utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on-demand therapy in young children with severe haemophilia A. Haemophilia 14(4): 743-752, 2008

Can observational studies approximate RCTs?. Value in Health 15(2): 215-216, 2012

Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort. Lancet. Haematology: -, 2018

How to achieve full prophylaxis in young boys with severe haemophilia A: different regimens and their effect on early bleeding and venous access. Haemophilia 21(4): 444-450, 2016

Patterns of tertiary prophylaxis in Canadian adults with severe and moderately severe haemophilia B. Haemophilia 20(3): E199-E204, 2015

When can RCTs and observational intervention studies mislead us and what can we do about it?. International Journal of Clinical Practice 63(11): 1562-1564, 2010

Lifelong prophylaxis in a large cohort of adult patients with severe haemophilia: a beneficial effect on orthopaedic outcome and quality of life. European Journal of Haematology 88(4): 329-335, 2012

The use of prophylaxis in 2663 children and adults with haemophilia: results of the 2006 Canadian national haemophilia prophylaxis survey. Haemophilia 14(5): 923-930, 2008