Little data exist, especially for adolescent and young adult (AYA) persons with haemophilia (PWH), about the relationship between adherence to prescribed treatment regimen and chronic pain. We examined this relationship among PWH (moderate or severe) aged 13-25 via cross-sectional survey. Adherence was assessed using the Validated Hemophilia Regimen Treatment Adherence Scale (VERITAS)-Pro and VERITAS-PRN for prophylactic and on-demand participants respectively. VERITAS scores range from 24 (most adherent) to 120 (least adherent). Chronic pain was measured using the FPS-R and was dichotomized as high for FPS-R scores ≥4 and low for <4. Logistic regression models were constructed to assess factors associated with having high (vs. low) chronic pain. Of 80 AYA respondents (79 men), most had severe disease (91%), infused prophylactically (86%) and had haemophilia A (91%). Fifty-one per cent were aged 13-17 and most were white (76%), non-Hispanic (88%) and never married (93%). Chronic pain was reported as high for 35% of respondents. Mean VERITAS-Pro scores for those with high and low chronic pain were 53.6 ± 12.3 vs. 47.4 ± 12.9, P = 0.05. VERITAS-PRN scores were similar across chronic pain status. Logistic regression revealed that for each 10-point reduction (i.e. increase in adherence) in the combined VERITAS (Pro and PRN) and VERITAS-Pro scores there was a 35% (OR = 0.65; 95% CI = 0.44, 0.96; P = 0.03) and 39% (OR = 0.61; 95%CI = 0.39, 0.96; P = 0.03) reduction in odds of having high chronic pain respectively. Among AYA PWHs, better adherence was associated with significantly lower odds of having high chronic pain. Moreover, non-whites were >4 times as likely as whites to report high chronic pain.

Results suggest that adherence efforts should be especially targeted to young adults as they transition from adolescence (i.e. parental supervision) and assume primary responsibility for their bleeding disorder care. Healthcare providers should be mindful of AYAs whose mothers have less formal education and ensure that adequate time and resources are dedicated to family adherence education.

The primary goal of prophylaxis in patients with severe haemophilia is to convert the phenotype from severe to moderate and to prevent the development of chronic arthropathy. Prior studies have demonstrated that prophylaxis decreases episodes of joint bleeds and chronic arthropathy. Effectiveness depends on prescription of prophylaxis and adherence to the prescribed regimen. The aim of this study was to determine if prescription of prophylaxis for children with haemophilia and perceptions of adherence to prophylaxis have changed since publication of the Joint Outcome Study (JOS). A questionnaire was sent, in electronic and written formats, to health professionals who provide care to children with haemophilia at US haemophilia treatment centres (HTCs). The response rate was 56 of 128 (44%) of the targeted HTCs. There were a few missing data and denominators are provided. All responses agreed with the results of the JOS and 30/55 (55%) reported the JOS increased their prescription of prophylaxis. Nineteen of 56 (34%) physicians or HTC staff reported that they had not prescribed prophylaxis within the last year due to concerns about adherence, and 19/56 (34%) reported they had stopped prophylaxis due to concerns about adherence within the last year. Predicted adherence decreased with increasing age. Prescription of prophylaxis appears to be increasing since publication of the JOS. Strategies to improve adherence may increase the likelihood of physician prescription of prophylaxis and make prophylaxis easier to implement for individual patients, thereby improving the clinical outcome of children and adults with haemophilia.

PurposeDepression, anxiety, pain, and treatment adherence have reciprocal effects not characterized extensively in hemophilia. This study explored the relationships between depression, anxiety, chronic pain, and treatment adherence in adults with hemophilia.Patients and methodsAdults with self-reported hemophilia A or B completed the cross-sectional IMPACT QoL II survey. Depression (9-item Patient Health Questionnaire [PHQ-9]), anxiety (7-item Generalized Anxiety Disorder scale [GAD-7]), chronic pain (Faces Pain Scale–Revised [FPS-R]), social support (Duke UNC Functional Social Support questionnaire), level of pain control, clotting factor treatment adherence (VERITAS-Pro or -PRN), and previous depression/anxiety were analyzed.ResultsAmong 200 participants (male, 77.3%; female, 22.8%), 54% had PHQ-9 and 52% had GAD-7 scores indicating moderate to severe depression or anxiety without diagnosis of either disorder. Participants with PHQ-9 scores ≥10 (moderate to severe depression) were more likely to have lower treatment adherence than those with PHQ-9 scores <10 (P<0.05). Participants with PHQ-9 or GAD-7 scores ≥10 were more likely to report uncontrolled pain and less social support versus PHQ-9 or GAD-7 scores <10 (χ2 P<0.05). Significant correlations were found between PHQ-9 and GAD-7 (P<0.0001), PHQ-9 and FPS-R (P=0.0004), PHQ-9 and VERITAS (P=0.01), GAD-7 and FPS-R (P=0.02), and GAD-7 and VERITAS (P=0.001).ConclusionDepression and anxiety are underdiagnosed in hemophilia. Depression is associated with anxiety, pain, and lower treatment adherence. While treatment providers play an important role in diagnosis, social workers may play a pivotal role in depression and anxiety screening. This study highlights the importance of regular screening and treatment for these disorders.

BackgroundHealth-related quality of life (HRQoL) in adolescents and young adults with bleeding disorders is under-researched. We aimed to describe factors related to HRQoL in adolescents and young adults with hemophilia A or B or von Willebrand disease.MethodsA convenience sample of volunteers aged 13 to 25 years with hemophilia or von Willebrand disease completed a cross-sectional survey that assessed Physical (PCS) and Mental (MCS) Component Summary scores on the SF-36 questionnaire. Quantile regression models were used to assess factors associated with HRQoL.ResultsOf 108 respondents, 79, 7, and 14% had hemophilia A, hemophilia B, and von Willebrand disease, respectively. Most had severe disease (71%), had never developed an inhibitor (65%), and were treated prophylactically (68%). Half of patients were aged 13 to 17 years and most were white (80%) and non-Hispanic (89%). Chronic pain was reported as moderate to severe by 31% of respondents. Median PCS and MCS were 81.3 and 75.5, respectively. Quantile regression showed that the median PCS for women (61% with von Willebrand disease) was 13.1 (95% CI: 2.4, 23.8; p = 0.02) points lower than men. Ever developing an inhibitor (vs never) was associated with a 13.1-point (95% CI: 4.7, 21.5; p < 0.01) PCS reduction. MCS was 10.0 points (95% CI: 0.7, 19.3; p = 0.04) higher for prophylactic infusers versus those using on-demand treatment. Compared with patients with no to mild chronic pain, those with moderate to severe chronic pain had 25.5-point (95% CI: 17.2, 33.8; p < 0.001) and 10.0-point (95% CI: 0.8, 19.2; p = 0.03) reductions in median PCS and MCS, respectively.ConclusionsEfforts should be made to prevent and manage chronic pain, which was strongly related to physical and mental HRQoL, in adolescents and young adults with hemophilia and von Willebrand disease. Previous research suggests that better clotting factor adherence may be associated with less chronic pain.