OBJECTIVES:

Haemophilia is an inherited bleeding disorder which may result in recurrent bleeding and joint health deterioration. It poses a significant burden on the health-care system, but limited evidence is available on UK-specific-haemophilia-related direct medical cost (DMC) and patient characteristics that may be driving it. This analysis aimed to identify predictors of DMC of haemophilia in the UK.

METHODS:

UK data from the CHESS (Cost of Haemophilia in Europe: a Socioeconomic Survey) I/II cross-sectional burden-of-illness studies were analysed. Haemophilia-related twelve-month DMC were quantified using physician-reported outcomes obtained from medical records of adult males aged ≤65 years with haemophilia A or B, and no recent clinical trial participation. These included: haemophilia type and severity, inhibitor status, annual bleeding rate (ABR), number of problem joints (PJ; chronically damaged joints), treatment type and comorbidities. Generalised linear models were specified to estimate their effects on DMC.

RESULTS:

Of 378 patients included in the analysis, 299(79%) patients had haemophilia A and mean(standard deviation [SD]) age was 33.0(12.9) years. 198(52%), 98(26%), 53(14%) and 29(8%) patients had 0, 1, 2 or 3+ PJs, respectively. Mean(SD) per-patient-haemophilia-related DMC was £165,001(£192,398) and £4,091(£6,939), including and excluding factor-replacement therapy, respectively. Several covariates were statistically significant determinants of non-treatment DMC: PJ number, ABR and Treatment regimen (all p<0.001). Non-treatment DMC increased by £2,018, £3,101, and £5,785 for patients with an ABR of 1-5(n=255;67%), 6-10(n=53;14%) and ≥11(n=23;6%), respectively and £1,869 per PJ. Presence of joint damage (≥1PJ) was associated with a mean(SD) increase in non-treatment DMC of £3,229(£1,923).

CONCLUSIONS:

Haemophilia is associated with substantial DMC in the UK. Non-treatment DMC are related to key clinical outcomes, including frequency of bleeding events and chronic joint damage. Reducing ABR and preventing PJs could play a substantial role in reducing non-treatment DMC. These findings could be important in informing the economic evaluation of future haemophilia treatments in the UK.