Health-Related Quality of Life and Adherence in Haemophilia

Karin Lindvall

Research output: ThesisDoctoral Thesis (compilation)

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Assessment of health-related quality of life (HRQoL) in haemophilia is important in order to provide information for clinical decision making and to verify the impact of haemophilia on patients and their families. Advances in haemophilia care and prophylactic treatment have improved HRQoL for the patients and where treatment is available, patients with haemophilia have almost reached the same life expectancy as the general population . However, development of an inhibitor is a severe complication of haemophilia related to replacement therapy and often occurs in children. An increased number of bleeds and a higher risk of disability are seen in these patients and may lead to increased concerns among the caregivers.
In (Paper I), patients with haemophilia responded to questions about their disease and adherence to treatment. Patients with mild haemophilia reported less knowledge of haemophilia compared to severe and moderate patients and were also less likely to treat a bleed. Patients with severe haemophilia estimated their health as worse than patients with mild haemophilia, but no statistical difference was found between patients on prophylaxis and those with no prophylaxis. Three studies evaluated HRQoL in patients (Paper II), partners (Paper III) and caregivers (Paper IV) where included subjects completed several HRQoL questionnaires. In Paper II, severely affected patients were more impaired in their ‘physical functioning’ than mild and moderate patients in the domains of SF-36. In contrast, moderate patients reported more impairment in ‘general health’ and ‘mental health’ than severe and mild patients. For patients undergoing orthopedic surgery, HRQoL increased in most of SF-36 domains. Partners (Paper III) of severe haemophilia patients on prophylaxis and partners of mild patients reported, in general, good HRQoL and low burden of the disease. The highest negative impact i.e. decreased HRQoL and higher burden, was seen in partners of moderate patients. In Paper IV, the comparison of SF-36 scores between caregivers of children with inhibitors and children on prophylaxis, revealed no statistically significant differences. In contrast, when these two groups were compared with caregivers of healthy children, significant differences were found, indicating a lower HRQoL in caregivers of haemophilia children especially in caregivers of inhibitor children. In Paper V, daily prophylaxis was compared with standard prophylaxis to assess if daily prophylaxis was feasible, reduced concentrate consumption and if it had an impact on patient´s HRQoL. Daily prophylaxis had a greater impact on daily life and the patients found the treatment more stressful. However, venous access and compliance was not a major problem. A cost reduction of approximately 30% was shown by switching from standard prophylaxis to daily prophylaxis.
If knowledge in the haemophilia population is to be increased, and HRQoL improved or maintained, these parameters must be evaluated regularly at the HTC more than is currently the case. As the patient gets older co-morbidities will add to haemophilic disease, and affect the well-being of haemophilia patients and their partners. The HTC should not only focus on the patient with haemophilia, and must take a more holistic view of the patient and his family.
Original languageEnglish
Awarding Institution
  • Clinical Coagulation, Malmö
  • Berntorp, Erik, Supervisor
  • Ljung, Rolf, Supervisor
Award date2013 May 30
ISBN (Print)978-91-87449-26-0
Publication statusPublished - 2013

Bibliographical note

Defence details

Date: 2013-05-30
Time: 09:00
Place: CRC, Malmö

External reviewer(s)

Name: Holme, Pål Andre
Title: MD, PhD,
Affiliation: Senter for sjeldne diagnoser/Rikshospitalet, Oslo Universitetssygehus, Norway



Paper IV: Increased Burden on Caregivers of Having a Child with Haemophilia Complicated by Inhibitors.
Page 11, Table 4. Total “Caregivers of children with inhibitor” should be change to 43.13 ± 10.3 and “Caregivers of children on prophylaxis” to 55.21 ± 11.4. Values should be changed accordingly in the Thesis text page 50, Table 10.

Paper V: Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy.
Figure 1. The pair of columns should be denoted in the following order: Mobility, Self-care, Usual activities, Pain/discomfort and Anxiety/depression.

Subject classification (UKÄ)

  • Cardiac and Cardiovascular Systems

Free keywords

  • Haemophilia
  • Adherence
  • Health-Related Quality of Life
  • Inhibitor
  • Patient
  • Partner
  • Caregive


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