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A survey of the outcome of prophylaxis, on‐demand treatment or combined treatment in 18–35‐year old men with severe haemophilia in six countries
Author(s) -
Noone D.,
O'Mahony B.,
Dijk J. P.,
Prihodova L.
Publication year - 2013
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/j.1365-2516.2012.02934.x
Subject(s) - medicine , haemophilia , quality of life (healthcare) , secondary prophylaxis , haemophilia a , pediatrics , on demand , demography , surgery , multimedia , computer science , nursing , sociology
Summary A number of studies have been published on the benefits of prophylactic treatment in adults with haemophilia. However, in many countries, it is considered as optional due to financial constraints. This survey was carried out to examine the long‐term effects of prophylaxis and the continuing benefit of the treatment into adulthood. Self‐assessed health‐related data and the EQ ‐5D questionnaire measuring health utility were collected from 124 men (26.9 ± 4.6 years) from C anada ( N  = 40), F rance ( N  = 14), I reland ( N  = 17), the N etherlands ( N  = 16), P oland ( N  = 24) and the UK ( N  = 13). The respondents were split into four groups: On‐Demand , <50% life on prophylaxis , ≥50% life on prophylaxis , Prophylaxis . Overall, long‐term prophylaxis results in lower presence of target joints ( P  ≤ 0.001), occurrence of serious bleeding episodes ( P  ≤ 0.05), recurring bleeding episodes ( P  ≤ 0.01) and requirement for surgical procedures ( P  ≤ 0.05). Furthermore, health utility ( P  ≤ 0.01) in the On‐demand group was significantly lower ( P  ≤ 0.01) compared to the ≥50% life on prophylaxis and the Prophylaxis group. No significant differences between countries were found except between the N etherlands and P oland, with P oland showing the lowest health utility ( P  ≤ 0.01) and the most problems with mobility ( P  ≤ 0.05) and pain/discomfort ( P  ≤ 0.001). The N etherlands showed the highest health utility (0.915) followed by C anada (0.791), I reland (0.786), UK (0.768), F rance (0.687) and P oland (0.629). The results demonstrate consistently higher quality of life of individuals who are on long‐term prophylactic treatment when compared to on‐demand treatment or intermittent prophylaxis and on ‐demand treatment.

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