Adherence to prophylaxis and bleeding outcome in haemophilia: a multicentre study

Abstract

Prevention of bleeding and joint damage in severe haemophilia is dependent on adherence to prophylactic replacement therapy. The aim of this study was to assess adherence to prophylaxis, including associations with age, bleeding and clotting factor consumption (CFC). In three Dutch haemophilia centres, semi-structured interviews about adherence to prophylaxis in the previous 2 weeks were conducted with patients or parents of a child with haemophilia. Patients were classified, according to pre-specified definitions, as adherent, sub-optimally adherent or non-adherent based on missing, timing, and dose of infusions. Association of annual bleeding rates, mean CFC, person performing the infusion (parents verus patients) with adherence categories were analysed. Overall, 241 patients with haemophilia using prophylaxis were studied. Parents were more adherent (66%; n = 48/73) than patients (43%; n = 72/168). Sub-optimal adherence occurred in 29% of parents and 37% of patients and was characterized by changes in timing of infusion (mostly from morning to evening), while missing <6% of infusions. Non-adherence occurred less often: in 5% of parents and 20% of patients. Reduced adherence was associated with lower CFC, but not with joint bleeding. In conclusion, non-adherence in haemophilia was relatively rare, yet 1/3 of patients struggled to administer prophylaxis at the appropriate time of day.

The behaviour and perception of illness: modulating variables of adherence in patients with haemophilia

“…However, we have not yet established other fundamental aspects that cause whether the patient follows treatment. As shown in our work and in other studies , the patients’ perception of their illness is essential in knowing how they will cope with the disease, including treatment.…”

“…Adherence understood as a complex and dynamic process, focuses on the different circumstances surrounding the individual to understand what determines it as a priority or not. The studies carried out so far, provide widely varying results ranging from 17% [8] to 59% [9] while the most recent studies render percentages of 70–80% . The causes behind lack of adherence include age, absence of symptoms, educational barriers (such as lack of knowledge), relationship with the health professionals, no self‐treatment skills and the belief that treatment is unnecessary .…”

“…The first studies carried out in this respect estimated 45‐60%, adherence, being higher in children than in patients of other ages . Some studies estimate rates between 80–87%, with a higher percentage in children and a lowest peak at between 20 and 45 years, then rising again after 60 years of age . Other studies support these results but do not find many differences between ages .…”

Manual and educational therapy in the treatment of hemophilic arthropathy of the elbow: a randomized pilot study

“…One of the possible causes could be the moderate adherence to the realization of domiciliary exercises. In patients with hemophilia, adherence to prophylactic treatment has been observed [24] greater than in other chronic diseases. However, the absence of well-defined protocols, clear scientific evidence and their implementation in the different treatment centers, indicates in the usual clinical practice a low-moderate adherence in the performance of domiciliary exercises, as we have observed in the patients of the educational group included in our study.…”

Unravelling adherence to prophylaxis in haemophilia: a patients’ perspective

“…In line with qualitative sampling strategies, maximum variation with respect to adherence level, bleeding frequency and age was sought for the initial sample [19]. Patients were selected from a previous study which quantified adherence in n = 241 patients (convenience sampling, comprising 71% of total population).…”