Long-term outcomes from prophylactic or episodic treatment of haemophilia A: A systematic review

Haemophilia : the Official Journal of the World Federation of Hemophilia
J O'HaraI Presch

Abstract

Evaluating treatment success in patients with haemophilia A (HA) remains a vigorous debate, especially concerning the interpretation of results from clinical and observational research. The benefits of short-term prophylaxis are well established, but long-term outcomes, particularly related to humanistic and economic burden, are not as well understood. We conducted a systematic literature review to evaluate the association of episodic or prophylactic bleed control with long-term clinical, humanistic and economic outcomes. Studies published in English between 1 January 2006 and 15 December 2016 were included. Participants had HA (with or without inhibitors), received prophylactic or episodic treatment and had at least 4 years of treatment or follow-up. Results were analysed qualitatively with descriptive findings. A total of 2091 records were screened, resulting in 19 studies from 20 publications for inclusion. Most studies included children (84%), were limited to patients with severe disease (74%) and were conducted in Europe or North America (89%). Ten studies (53%) included patients with inhibitors. Median study follow-up ranged from 5 to 19 years. Long-term bleeding and haemarthrosis outcomes were consistently better for pat...Continue Reading

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Citations

Oct 26, 2018·Blood Advances·Sandra Le Quellec, Claude Negrier
Apr 16, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Wolfgang MiesbachJörg Schüttrumpf
Mar 21, 2020·Journal of Thrombosis and Haemostasis : JTH·Karin M LövgrenParis Margaritis
Sep 10, 2019·Annals of Internal Medicine·Alfonso IorioUNKNOWN Data and Demographics Committee of the World Federation of Hemophilia
Oct 22, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Sonia O'HaraJamie O'Hara
Oct 24, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Charles R M HayCraig M Kessler
Feb 8, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Camila C AquinoAlfonso Iorio
Jun 3, 2021·Clinics and Practice·Maria Sol CruzLaura Forzani

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