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Assessment of the impact of treatment on quality of life of patients with haemophilia A at different ages: insights from two clinical trials on turoctocog alfa
Author(s) -
Santagostino E.,
Lentz S. R.,
Busk A. K.,
Regnault A.,
Iorio A.
Publication year - 2014
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/hae.12371
Subject(s) - medicine , haemophilia , quality of life (healthcare) , pediatrics , clinical trial , haemophilia a , regimen , health related quality of life , physical therapy , disease , nursing
Summary Haemophilia and its treatment interfere with patients' life, so health‐related quality of life ( HRQ oL) should be assessed when evaluating treatments. This study investigated the HRQ oL of patients with haemophilia A treated prophylactically with a new recombinant factor VIII . Two phase 3 trials investigated turoctocog alfa in patients with severe haemophilia A: one in children, one in adults and adolescents. HRQ oL was a secondary endpoint assessed by the HAEMO ‐ QOL age‐specific, self‐administered questionnaires. Parent‐completed versions were also included for parents of children and adolescents. All HAEMO ‐ QOL questionnaires allow the calculation of domain‐specific and total scores ranging from 0 to 100, lower scores indicating better HRQ oL. Mean change in all scores was described for 25 children aged 4–7 years, 21 children aged 8–12 years, 18 adolescents aged 13–18 years and 129 adults, overall, and according to the treatment regimen received prior to the study (on‐demand; prophylaxis; mixed). Mean changes in HAEMO ‐ QOL total score were 1.4 for children aged 4–7 years, −2.6 for children aged 8–12 years, −5.8 for adolescents and −1.6 for adults. In parent‐completed versions, mean changes in total score were −6.0 for children aged 4–7 years, −4.7 for children aged 8–12 years, and −10.0 for adolescents. Patients receiving on‐demand treatment before the trial showed greater improvement in HRQ oL scores than patients already on prophylaxis. HRQ oL of patients remained fairly stable over the course of the trials. However, improvements were observed for adolescents. Switching to prophylaxis was identified as a potential driver of improvement of HRQ oL in patients with haemophilia A.