The relationship between target joints and direct resource use in severe haemophilia

Abstract

ObjectivesTarget joints are a common complication of severe haemophilia. While factor replacement therapy constitutes the majority of costs in haemophilia, the relationship between target joints and non drug-related direct costs (NDDCs) has not been studied.MethodsData on haemophilia patients without inhibitors was drawn from the ‘Cost of Haemophilia across Europe – a Socioeconomic Survey’ (CHESS) study, a cost assessment in severe haemophilia A and B across five European countries (France, Germany, Italy, Spain, and the United Kingdom) in which 139 haemophilia specialists provided demographic and clinical information for 1285 adult patients. NDDCs were calculated using publicly available cost data, including 12-month ambulatory and secondary care activity: haematologist and other specialist consultant consultations, medical tests and examinations, bleed-related hospital admissions, and payments to professional care providers. A generalized linear model was developed to investigate the relationship between NDDCs and target joints (areas of chronic synovitis), adjusted for patient covariates.ResultsFive hundred and thirteen patients (42% of the sample) had no diagnosed target joints; a total of 1376 target joints (range 1–10) were recorded in the remaining 714 patients. Mean adjusted NDDCs for persons with no target joints were EUR 3134 (standard error (SE) EUR 158); for persons with one or more target joints, mean adjusted NDDCs were EUR 3913 (SE EUR 157; average mean effect EUR 779; p < 0.001).ConclusionsOur analysis suggests that the presence of one or more target joints has a significant impact on NDDCs for patients with severe haemophilia, ceteris paribus. Prevention and management of target joints should be an important consideration of managing haemophilia patients.

The societal burden of haemophilia A. I – A snapshot of haemophilia A in Australia and beyond

“…Reductions in HRQoL (measured by metrics such as the HRQoL index, EQ‐5D utility value and EQ‐VAS, the Haem‐A‐QoL scale and their various subscales/dimensions) are most noticeable for those with severe HA, or those with inhibitors to FVIII. HRQoL is also significantly influenced by target join disease as shown across five European countries in the CHESS study 29 . Reductions in quality of life are typically associated with poor physical health; inability to participate in sports, recreational or leisure activities; missed school or work; the perception of self; and the impact on the family 30‐34 .…”

“…As shown in the United States, while bleed‐related admissions are most common (17.0 admissions per 1000 patient‐years), PwHA may also be admitted due to major joint replacement (2.3 admissions per 1000 patient‐years), lower extremity and humerus procedures (2.1 admissions per 1000 patient‐years) and bone disease and arthropathy (2.0 admissions per 1000 patient‐years) 15 . In the CHESS study, 42.7% of people with a target joint had had surgery to the target joint in the preceding 12 months 29 . As with bleed‐related hospitalizations, hospitalizations for joint disease are more common in people with inhibitors (24.1% vs 6.2%) 46 .…”

How to discuss gene therapy for haemophilia? A patient and physician perspective

“…Joint damage Can result in chronic pain, disability and joint deformity at an early age 1,54,55 Poor health-related quality of life Closely linked to the extent of joint damage 54 Functional impairment…”

“…60,61 Pain/discomfort is an area where most individuals report experiencing 'extreme' issues. 54 Individuals may experience anger and frustration due to the pain, inconvenience and erratic nature of bleeds 62 Psychological Anxiety/depression are the areas where most individuals report experiencing 'extreme' issues 54 Personal productivity Adverse impact on educational achievement and work productivity due to absence and difficulties due to functional impairments and pain 57,63,64…”

Economic burden of hemophilia A and B: a case in Iran

“…Deficiencies in the blood clotting Factor VIII (FVIII) is called 'hemophilia A' and deficiencies in the blood clotting Factor IX (FIX) is known as 'hemophilia B' [2] which are categorized in terms of the severity into severe, moderate, and mild. Hemophilia imposes enormous health care costs on the patients, society and the health system [3]. In the United States and European countries, hemophilia is one of the most expensive diseases and a costly disorder that is highly considered not only because of its high direct medical costs but also because of its indirect costs [4][5][6][7].…”