Social determinants and health‐related dimensions of quality of life in adult patients with haemophilia

The availability of safe and effective factor replacement therapies, in persons with haemophilia (PWH), has in some countries answered the basic need for treatment of these patients. The findings suggest that adult patients who have always been on prophylaxis reported significantly better physical functioning, and thus better quality of life. This study is designed to evaluate the QoL in adult PWH, by focusing on social determinants of QoL and their relationship with health‐related dimensions, in Tabriz, Iran. The survey instrument was a self‐report 36 items questionnaire, ‘A36 Hemofilia – QoL’, which is a disease‐specific questionnaire for the assessment of the health‐related QoL in adults living with haemophilia. A total of 100 haemophilia A and B patients, aged over 17 years participated in this study within 1 year. QoL total score was 71.88 (±26.89 SD). Patients who treat in our Hemophilia Treatment Center, had better QoL score (P = 0.000), and education has a significant impact on the social aspects of QoL (P = 0.18). The QoL was very poor in urban area in contrast to patients who lived in the city (54.45 vs. 74.21 respectively). Single patients have a better QoL than married patients (76.56 vs. 68.50 respectively). Our results showed that low education and lack of awareness of the diseases among PWH lead to reduce of QoL and more disease complications. More and wider treatment and psychological care for improving quality of life of these patients are seriously recommended.     

Bone density and health‐related quality of life in adult patients with severe haemophilia

Summary. Severe haemophilia and reduced bone density can negatively influence perception of patient’s health‐related quality of life (HRQoL), especially considering future aspects, the risk of losing independence or pain suffering. The aim of this study was to assess levels of HRQoL in severe haemophilia patients and to compare HRQoL to those of the general population as well as to determine whether reduced bone density is correlated to the perceived HRQoL. Patients were divided into two groups based on timing of being treated with prophylaxis: Group A (started prophylaxis at age of ≤3 years; n = 22); Group B (at age of >3 years; n = 15). The bone mineral density (BMD g cm^?2) of different measured sites was measured by dual energy X‐ray absorptiometry (DXA). HRQoL was assessed using SF‐36 questionnaire. Group A have mean BMD T‐score >?1.0 (i.e. normal score) at all measured sites, and have almost similar scores in the SF‐36 domains compared with the reference population. Group B have mean BMD T‐score ?1.0 at lumbar spine and total body, and their scores in the SF‐36 domains were lower compared with the reference population. Moreover, significant correlations were found between BMD at femoral neck and total body with physical domains. With adequate long‐term prophylaxis since early childhood, adult patients with haemophilia report a comparable BMD and HRQoL to the Swedish reference population. Reduced BMD in group B correlated with impaired physical health, which underscores the importance of early onset of adequate prophylactic treatment.     

Cross‐cultural development and psychometric evaluation of a patient‐reported health‐related quality of life questionnaire for adults with haemophilia

Summary. Co‐morbidities of haemophilia, such as arthropathy and blood‐borne infections, can adversely affect the quality of life of adult patients with haemophilia. The purpose of this study was to develop and validate a haemophilia‐specific health‐related quality of life questionnaire for adults (HAEMO‐QoL‐A). Subjects with varying severities of haemophilia completed the HAEMO‐QoL‐A at baseline and 4 weeks. Other assessments included the SF‐36 and Health Assessment Questionnaire – Functional Disability Index (HAQ‐FDI). Two‐hundred and twenty‐one participants completed the 41‐item HAEMO‐QoL‐A covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns) and four independent items. Internal consistency was good‐to‐excellent (Cronbach’s α‐range: 0.75–0.95). Test–retest reproducibility was good, with intraclass correlation coefficients >0.80 except for the Emotional Impact domain (0.79). Concurrent validity between the HAEMO‐QoL‐A total and subscale scores and all SF‐36 subscale scores were generally good (correlations range: 0.13–0.87). Significant correlations between the HAEMO‐QoL‐A and the HAQ‐FDI ranged from ?0.14 to ?0.69. There were non‐significant correlations with the Treatment Concerns subscale and with the Worry subscale. The HAEMO‐QoL‐A discriminated significantly between adults with haemophilia by severity and HIV status. The Physical Functioning subscale discriminated between patients receiving prophylactic or on‐demand therapy. The HAEMO‐QoL‐A is a valid and reliable instrument for assessing quality of life in haemophilia patients.     

The relative burden of haemophilia A and the impact of target joint development on health‐related quality of life: results from the ADVATE Post‐Authorization Safety Surveillance (PASS) study

Summary. Studies with haemophilia A (HA) patients have shown burden in health‐related quality of life (HRQOL) when compared with general population norms. In the current study, HA patients’ SF‐36v2 health survey scores were compared with general population norms and to patients with other chronic conditions. The impact of target joints (TJs) on HRQOL was also examined. The sample was a subset of HA patients enrolled in the Post‐Authorization Safety Surveillance (PASS) programme: a prospective open‐label study in which ADVATE [Antihaemophilic Factor (Recombinant), Plasma/Albumin‐Free Method] was prescribed. A total of 205 patients who were ≥18 years old and had SF‐36v2 baseline scores were selected for this study. To measure the burden of HA on HRQOL, manova analyses compared these SF‐36v2 scores to age‐ and gender‐matched general population US and EU norms and to patients from other chronic condition groups. manova and correlational analyses examined the relations among TJ, age and SF‐36v2 scores. Comparisons with general population norms confirm that HA negatively impacts physical, but not mental, HRQOL. Comparison with other chronic conditions shows the physical burden of HA is greater than for chronic back pain but similar to diabetes and rheumatoid arthritis, while the mental burden of HA is less than for all three patient groups. The presence of TJs was negatively associated with physical HRQOL, although this association was much larger for older patients (45+ years) than for younger ones. Physical, but not mental, HRQOL is diminished in HA patients. Target joints are associated with lower physical HRQOL, although this effect is moderated by age.     

Improvement in health‐related quality of life with recombinant factor IX prophylaxis in severe or moderately severe haemophilia B patients: results from the BAX326 Pivotal Study

Little is known about the health‐related quality of life (HRQoL) burden of haemophilia B. The aim of this study was to assess HRQoL burden of haemophilia B, the benefit of recombinant factor IX (rFIX) prophylaxis and the HRQoL benefit of achieving a zero annual bleed rate. Subjects receiving rFIX (BAX326) prophylaxis or on‐demand completed the SF‐36 survey. Baseline SF‐36 scores were compared to the general US population scores to understand the HRQoL burden. Changes in SF‐36 scores between baseline and follow‐up were tested using t‐tests. Subgroup analysis was conducted to examine SF‐36 change among subjects who switched to BAX326 prophylaxis. SF‐36 scores were also compared between those with zero bleeds and those who bled during the study. Compared to the US norms, subjects reported lower average scores in all physical and several mental HRQoL domains. At follow‐up, prophylaxis subjects reported statistically significant and clinically meaningful improvements in overall physical HRQoL, as measured by the Physical Component Score (PCS) (mean change 2.60, P = 0.019), Bodily Pain (BP) (3.45, P = 0.015) and Role Physical (RP) domains (3.47, P = 0.016). Subjects who switched to prophylaxis from intermittent prophylaxis or on‐demand experienced more pronounced improvements not only in the PCS (3.21, P = 0.014), BP (3.71, P = 0.026), RP (4.43, P = 0.008) but also in Vitality (3.71, P = 0.04), Social Functioning (5.06, P = 0.002) and General Health domains (3.40, P = 0.009). Subjects achieving zero bleeds reported lower BP (P = 0.038). Prophylaxis with BAX326 significantly improved HRQoL in patients with moderately severe or severe haemophilia B by reducing bleeds.     

The impact of joint range of motion limitations on health‐related quality of life in patients with haemophilia A: a prospective study

In patients with haemophilia A, repeated occurrences of haemarthrosis and synovitis lead to limitations in range of motion (ROM) of major joints. However, the effect of limitations in joint ROM on health‐related quality of life (HRQOL) in these patients has not been studied previously. The aim of this study was to assess the impact of ROM limitations of 10 major joints (bilateral shoulders, elbows, hips, knees and ankles), combined with other possibly influential factors, on HRQOL in patients with haemophilia A. The ROM limitations in 13 movements and pain intensity of the 10 major joints were measured. The socio‐demographic and clinical data were recorded. Short‐Form 36 was used as the HRQOL measurement. Eighteen patients (mean age: 36.9 years) were included. Hip ROM limitations, knee ROM limitations and hip pain intensity predicted physical functioning scale (P < 0.001; adjusted R² = 0.553). Shoulder ROM limitations and age predicted role limitation were due to emotional problems scale (P < 0.001; adjusted R² = 0.373). Elbow ROM limitations and haemophilia severity predicted mental health scale (P = 0.001; adjusted R² = 0.320). Hip ROM limitations predicted social functioning scale (P = 0.041; adjusted R² = 0.091). Educational level and elbow ROM limitations predicted vitality scale (P < 0.001; adjusted R² = 0.416). The ROM limitations of hip, knee, shoulder and elbow could be predictors for HRQOL in patients with haemophilia A. Improving ROM of major joints could be an appropriate treatment strategy to enhance HRQOL in these patients.     

Prophylaxis with anti‐inhibitor coagulant complex improves health‐related quality of life in haemophilia patients with inhibitors: results from FEIBA NF Prophylaxis Study

The Pro‐FEIBA study reported health‐related quality of life (HRQoL) improved following 6‐month of Factor Eight Inhibitor Bypassing Activity (FEIBA) prophylaxis. This study investigates whether 12‐month of FEIBA prophylaxis improved HRQoL in haemophilia patients with inhibitors. Thirty‐six subjects in a 1‐year prospective, randomized, open‐label, parallel‐design study were randomized to prophylaxis (85 ± 15 U kg^?1 every other day) or on‐demand treatment. HRQoL was assessed at screening, 6 and 12‐month termination using the EQ‐5D, Haem‐A‐QoL, Haemo‐QoL and a general pain visual analog scale (VAS). To evaluate changes, paired t‐tests and criteria for minimally important differences were applied. Repeated measures regression tested the association between annualized bleeding rate (ABR) and physical HRQoL. At 6 and 12 months, prophylaxis subjects reported clinically meaningful improvement in EQ‐5D index (mean improvement, 0.10 and 0.08, respectively) and both clinically meaningful and statistically significant improvements in EQ‐VAS scores (16.9 and 15.7, respectively; P < 0.05) vs. baseline. General pain was significantly reduced during prophylaxis at each follow‐up (mean improvement, 20.3 and 23.2, respectively; both P <0.05). At 12 months, prophylaxis subjects achieved significant improvements in Haem‐A‐QoL Total Score and in four domains: Physical Health, Feeling, View, and Work and School (all P < 0.05). No statistically significant changes, except for Haem‐A‐QoL Physical Health at 6 months, were observed with on‐demand treatment. ABR was decreased by 72.5% with prophylaxis vs. on‐demand treatment (P = 0.0003) and reduced ABR was associated with better physical HRQoL (P < 0.05). FEIBA prophylaxis significantly reduced ABR and improved HRQoL in inhibitor patients. Subjects with lower ABR reported better physical HRQoL.     

Relationship between health‐related quality of life and blood pressure control in patients with uncontrolled hypertension

We sought to investigate the psychosocial characteristics of patients with uncontrolled hypertension and examine factors that influence blood pressure (BP) control. A total of 1011 patients with uncontrolled hypertension were enrolled in 13 tertiary hospitals. Uncontrolled hypertension was defined as systolic BP ≥140 mm Hg or diastolic BP ≥90 mm Hg despite on antihypertensive therapy. Socio‐demographics, anthropometrics, behavioral risk factors, medication pattern, adherence, and measures of health‐related quality of life (HRQoL; EuroQol 5D visual analog scale [EQ‐5D VAS]) were assessed at baseline and during follow‐up visits (3 and 6 months). Patients were divided into 2 groups based on BP control status at 6 months (controlled group [n = 532] vs uncontrolled group [n = 367]). There were no differences in clinical characteristics except the proportion of smokers and baseline BP between patients with controlled BP and uncontrolled BP. At 6 months, the adherence of antihypertensive medication did not differ between the groups but the proportion of combination therapy with ≥3 antihypertensives was significantly higher in patients with uncontrolled BP. EQ‐5D VAS at follow‐up was significantly lower in patients with uncontrolled BP despite similar baseline values. Multivariate logistic regression analysis revealed that EQ‐5D VAS at follow‐up significantly correlated with BP control. Patients with worse HRQoL had higher Charlson Comorbidity Index and higher proportion of taking ≥3 antihypertensives, but medication adherence was similar to those with better HRQoL. These findings suggest that along with pharmacologic intervention of hypertension, management of comorbid conditions or psychological support might be helpful for optimizing BP control in patients with uncontrolled hypertension.     

Health‐related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti‐inhibitor complex concentrate: results from the Pro‐FEIBA study

Patients with haemophilia A and inhibitors are at high risk for severe bleeding, progression of joint disease and deterioration of health‐related quality of life (HRQoL). To determine the impact of prophylaxis with an activated prothrombin complex concentrate (aPCC) on HRQoL, HRQoL was assessed using the Short‐Form (SF)‐36 Health Survey and the EQ‐5D questionnaire in subjects ≥14 years participating in a prospective, randomized, crossover study comparing 6 months of aPCC prophylaxis with 6 months of on‐demand therapy. Eighteen of 19 patients completed the survey or questionnaire before and after the on‐demand therapy and prophylaxis periods. A general trend towards improved HRQoL after prophylaxis was observed for the 18 evaluable patients in all SF‐36 dimensions except for vitality/energy and physical functioning. After prophylaxis, ‘good responders,’ defined as patients experiencing ≥50% reduction in bleeding, exhibited statistically and clinically significant differences in the physical component score (P = 0.021), role – physical (P = 0.042), bodily pain (P = 0.015), and social functioning (P = 0.036). Similarly, the EQ‐5D health profile showed a trend towards improvement after prophylaxis in all evaluable patients. Among the good responders, improvements did not differ from those observed after on‐demand treatment. EQ visual analogue scale values were slightly improved following prophylaxis for all evaluable patients and the EQ‐5D utility index improved in the good responders only. During prophylaxis, patients missed significantly fewer days from school or work because of bleeding than during on‐demand treatment (P = 0.01). In conclusion, by significantly reducing bleeding frequency in good responders, aPCC prophylaxis improved HRQoL compared with on‐demand treatment.