Risk assessment for coronary heart disease in patients with haemophilia: a single centre study in the United States

In haemophilia, coronary heart disease (CHD) occurs at a similar frequency as in the general population, but the contributing risk factors in haemophilia are incompletely understood. To investigate risk factors and 10‐year CHD risk in a single centre cohort of patients with haemophilia (PWH) ≥20 years old (n = 89). We retrospectively applied the modified Framingham National Cholesterol Education Program/Adult Treatment Panel (NCEP/ATP) III risk prediction equation. Three risk levels were defined: <10% (low), 10–20% (intermediate) and >20% (high). Results were compared to the National Health and Nutrition Examination Survey (NHANES). Mean age in both cohorts was similar. Compared to NHANES, systolic blood pressures were significantly higher in PWH, but current smoking and cholesterol were lower. CHD risk differed significantly between PWH and NHANES (P = 0.005) with a higher proportion of PWH classified at low risk (77.5% vs. 61.0%). The proportion of low risk patients was also significantly higher for severe haemophilia patients compared to non‐severe haemophilia patients (88.6% vs. 66.7%, P = 0.02). Among PWH, and compared to PWH who were hepatitis C (HepC) negative, HepC positive patients had significantly lower cholesterol, LDL and triglycerides. The CHD risk of HepC positive patients differed significantly from NHANES (P = 0.03) with a lower proportion of HepC positives being classified as high risk (5.7% vs. 17.3%). Favourable CHD risk classification in PWH may be influenced by low cholesterol associated with HepC infection. Estimates of CHD risk in PWH by composite scoring may not be accurate and will require studies correlating risk factors with incident CHD.     

Obesity in haemophilia patients: effect on bleeding frequency,clotting factor concentrate usage,and haemostatic and fibrinolytic parameters

The prevalence of obesity in patients with haemophilia (PWH) is increasing. We investigated the effect of obesity on bleeding frequency and clotting factor concentrate (CFC) usage in PWH and assessed whether prothrombotic changes observed in obesity differ between controls and PWH. Number of bleeds and CFC usage were compared between obese (N = 51) and non‐obese (N = 46) haemophilia A patients. Markers of haemostasis and fibrinolysis were compared between PWH, and gender‐, age‐ and body mass index (BMI)‐matched non‐haemophilic controls (N = 91). Median number of bleeds/patient‐month was comparable between obese and non‐obese patients with severe haemophilia (P = 0.791). Obese patients with severe haemophilia used 1.4 times more CFC/patient‐month than non‐obese patients (P = 0.036). When adjusting for weight this difference disappeared (P = 0.451). von Willebrand factor plasma concentration (VWF:Ag), factor VIII activity and endogenous thrombin potential were higher in obese than in non‐obese controls. Obesity did not influence these markers in PWH. Plasminogen activator inhibitor type 1 levels were higher in obese vs. non‐obese PWH (P < 0.001), whereas levels were comparable between PWH and controls (P = 0.912). Plasmin‐α2‐antiplasmin complex (PAP) levels appeared to be lower in obese vs. non‐obese subjects, both within controls (P = 0.011) and PWH (P = 0.008). However, in PWH, PAP levels were higher than in controls (P < 0.001). Obesity is associated with an increase in net CFC usage in PWH, but has no effect on bleeding frequency. In addition, obesity attenuates hyperfibrinolysis in PWH. Future research investigating whether obese PWH need CFC treatment dosed on weight or whether a lower dosage would suffice to prevent and treat bleedings is needed.     

Presentation and management of acute coronary syndromes among adult persons with haemophilia: results of an international,retrospective, 10‐year survey

Sparse data are available on presentation and management of acute coronary syndromes (ACS), including unstable angina and non‐ST‐ and ST‐elevation myocardial infarction, among persons with haemophilia (PWH). The aim of this study was to determine demographics, bleeding disorder characteristics, cardiovascular risk factors (CRFs), interventions, haemostatic protocol, revascularization outcomes and complications among PWH with ACS. Members of an international consortium comprising >2000 adult PWH retrospectively completed case report forms for episodes of ACS in a >10‐year follow‐up period (2003–2013). Twenty ACS episodes occurred among 19 patients [rate, 0.8% (95% CI 0.4, 1.2)]. Seven patients (37%) were aged <50 years; 10 (53%) had ≥3 CRFs. In 5/20 episodes (25%), the initial ACS management protocol was altered because of the bleeding disorder. None of the eight patients with severe haemophilia underwent coronary artery bypass grafting (CABG), compared with 54.5% of patients with non‐severe disease (P = 0.02). Revascularization with percutaneous coronary intervention (PCI) or CABG was rated successful in 13/13 cases, with no excessive bleeding during initial management. During chronic exposure to antiplatelet agents, secondary haemophilia prophylaxis was more prevalent in patients with severe haemophilia compared with non‐severe haemophilia (85.7% vs. 30%, P = 0.05). No ACS‐related deaths occurred during initial management, but one patient with severe haemophilia A died of undetermined cause 36 months after the ACS event while on aspirin therapy. ACS occurs even among relatively younger PWH, typically in association with multiple CRFs. Revascularization with PCI/CABG is feasible, and antiplatelet agents plus secondary prophylaxis appears to be well tolerated in selected PWH with ACS.     

Physical and mental quality of life in adult patients with haemophilia in Belgium: the impact of financial issues

In Belgium, where haemophilia affects approximately 1:7000 people (2011), data on patients' quality of life (QoL) is scarce. This project aims to assess physical and mental QoL (P‐QoL and M‐QoL) simultaneously, and to analyse the influence of different variables on these two aspects of QoL. After Ethics Committee approval, we contacted 84 adult haemophilia A (HA) and haemophilia B (HB) patients, without current inhibitors, on replacement therapy (on‐demand or secondary prophylaxis), regularly followed up at our comprehensive treatment centre. Seventy‐one (n = 59 HA, n = 12 HB) replied to our questionnaire, which included the SF36v2 QoL assessment forms. We analysed two groups of variables: one including variables previously associated with decreased QoL, and another including variables with unclear impact on QoL (e.g. patients' understanding of haemophilia‐related issues, economical concerns). In our population (mean ± SD age: 45.2 ± 14.7 years old), P‐QoL appeared more reduced than M‐QoL. P‐QoL was strongly influenced by the number of arthropathies while M‐QoL was primarily affected by patients' concern of personal costs due to haemophilia. Among this latter group, having knowledge of insurance coverage had a positive impact on M‐QoL. Scores did not depend on haemophilia type. QoL was impaired in our haemophilia patients. A simultaneous assessment of P‐QoL and M‐QoL confirmed the benefit of primary prophylaxis in P‐QoL, while originally pointing out the major burden of patients' concerns and poor understanding of haemophilia‐related economical issues on their M‐QoL. This might become a particularly challenging issue in times of financial crisis.     

A longitudinal study of family structure in Swedish persons with haemophilia

Haemophilia is an X‐linked inherited rare bleeding disorder affecting mainly men. The treatment consists of replacement therapy that has been associated with severe side effects, such as blood transmitted viral infections, but has markedly improved over the last decades. The aim of this study was to study family structure over time among Swedish persons with haemophilia (PWH), focusing on children, siblings and marital status. PWH A or B were identified from the haemophilia centres and the national Patient Registry. Each PWH was compared to five age‐ and gender‐matched controls. The national Multi‐Generation Registry was used to identify children and siblings. A total of 1365 children with a father suffering from haemophilia A or B and 1938 siblings of the PWH were identified. Having one or more children was significantly less common (P = 0.003) for PWH than for controls. Significantly lower rates of having a child were also found for the subgroups of persons suffering from severe haemophilia and those infected with HIV (P < 0.001). A higher proportion of PWH, with or without HIV and/or viral hepatitis had siblings compared to the controls (P < 0.001). However, the mean number of siblings was significantly lower for persons with severe haemophilia (P = 0.001). The number of marriages and divorces did not differ between PWH and controls. Our data indicate a negative impact of HIV and viral hepatitis on family structure for PWH despite the relatively good access to treatment in Sweden over the last few decades. This was particularly true for those with a severe form of haemophilia.     

Atrial fibrillation in patients with haemophilia: a cross‐sectional evaluation in Europe

Atrial fibrillation (AF) is a common health problem in the general population, but data on prevalence or management in patients with haemophilia (PWH) are lacking. The aims of this study were to analyse the prevalence of AF and risk factors for stroke using a cross‐sectional pan‐European design and to document current anticoagulation practice. The ADVANCE Working Group consists of members from 14 European haemophilia centres. Each centre retrieved data on their PWH with AF. From the total of 3952 adult PWH, 33 had AF with a mean age of 69 years (IQR 62–76). Haemophilia was severe in seven (21%), moderate in six (18%) and mild in 20 (61%) patients. The overall AF prevalence was 0.84% and increased with age; 0.42% in patients 40–60 years and 3.4% in patients >60 years. The mean CHA₂DS₂‐Vasc score was 1.3 (range 0–4), predominantly determined by age and hypertension. Hypertension was reported in 48% of PWH with AF. In 11 patients (33%), anticoagulation was started of whom nine aspirin and two vitamin K antagonists. Of these 11 patients, nine had mild haemophilia. Anticoagulation was given in 42% of patients with a CHA₂DS₂‐Vasc score ≥2. During follow‐up (mean 57 months), there were no thrombotic events reported, nor increases in bleeding severity. The prevalence of AF in haemophilia increases with age and is predominantly present in mild haemophilia. PWH have a low stroke risk based on their CHA₂DS₂‐Vasc scores, that might be even lower considering the hypocoagulable state. Only 33% of PWH with AF receives any form of anticoagulation therapy.     

Physical activity and functional abilities in adult males with haemophilia: a cross‐sectional survey from a single US haemophilia treatment centre

Physical activity and functional ability are important determinants of quality of life and these metrics are affected by both haemophilia and ageing. Outside haemophilic arthropathy, risk factors leading to reduced physical activity and function in people with haemophilia (PWH) are under‐explored. The purpose of this analysis was to determine risk factors for reduced physical activity and functional limitations in PWH. A secondary analysis was conducted on data indexing physical activity and functioning of 88 PWH using data originally collected as part of a cross‐sectional study at a single large haemophilia treatment centre. The Framingham Physical Activities Index (PAI), the Hemophilia Activities List (HAL) and the Timed Up‐and‐Go Test (TUG) were the outcome measures. The World Federation of Haemophilia (WFH) orthopaedic joint score was used as a measure of arthropathy. Multiple linear regression analysis was used to assess the relationship between the outcome measures and covariates. Worsening WFH joint score was independently associated with all three outcome measures (P < 0.05). Increasing age was associated with reduced PAI and increased TUG time (P < 0.05). The HAL summary score was decreased in patients with chronic liver disease (P = 0.006). The adjusted R² for each model was ≤0.35. This study provides evidence for the relationship between arthropathy and reduced physical functioning/activity, but also highlights that much of the variation in physical functioning/activity is not explained by haemophilia‐related characteristics.     

Better adherence to prescribed treatment regimen is related to less chronic pain among adolescents and young adults with moderate or severe haemophilia

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.     

Quadriceps strength,inter‐extremity difference (IED) and joint status in adult persons with severe haemophilia in different age stages

Quadriceps weakness seems to be a hallmark in adult persons with severe haemophilia (PWH). The purpose of this study was to compare PWH and non‐haemophilic controls in different age stages with reference to joint status and quadriceps strength. Further aims were to examine the extent of strength‐specific inter‐extremity‐difference (IED) and the prevalence of abnormal IED (AIED). A total of 106 adults with severe haemophilia (H) and 80 controls (C) had undergone an orthopaedic examination for classification of knee and ankle status using the WFH score. Quadriceps strength was evaluated unilaterally as well as bilaterally with a knee extensor device. Each group was divided into four age‐related subgroups (HA/CA: 18–29, HB/CB: 30–39, HC/CC: 40–49, HD/CD: 50–70; in years). H presented a worse knee and ankle status than C indicated by higher WFH scores (P < 0.01). Regarding the age‐matched subgroups only HB showed higher knee scores than CB (P < 0.05). The ankles were clinically more affected in HB‐HD compared with those in age‐matched controls (P < 0.05). H showed lower quadriceps strength than C (P < 0.05). In addition, all subgroups of H presented lower strength (HA: 10–17, HB: 19–23, HC: 35–36, HD: 53–61; in%, P < 0.05). IED was higher in H than in C [H: 12.0 (5.3/32.2) vs. C: 7.1 (2.9/10.9); Median (quartiles) in%, P < 0.001] and increased with age in H. We discovered an AIED in 35% of H. These findings highlight the importance for the early implementation of preventive and rehabilitative muscle training programmes in the comprehensive treatment of PWH.     

Haemophilia Experiences,Results and Opportunities (HERO) Study: survey methodology and population demographics

Psychosocial factors have a significant impact on the quality of life of persons with haemophilia (PWH). The Haemophilia Experiences, Results and Opportunities (HERO) initiative was developed to provide a greater understanding of the psychological components which influence the lives of PWH. This article describes the HERO methodology and the characteristics of respondents. Two online surveys (one for adult PWH ≥18 years and one for parents of children <18 years with haemophilia) were developed by an international advisory board and conducted in 10 countries. The surveys included demographic and treatment characteristics, relationships, sexual intimacy, quality of life, barriers to treatment and sources of information. A total of 675 PWH [age, median (range) 36 (18–86 years)] and 561 parents [39 (23–68 years)] completed the survey. PWH/parents reported haemophilia A (74%/76%), B (13%/16%) or with inhibitors (13%/8%). Spontaneous joint bleeding was reported in 76%/52% of PWH/children with haemophilia A, 67%/47% with haemophilia B and 93%/76% with inhibitors. Median number of bleeds (interquartile range) was 7 (2–20) for PWH and 4 (2–10) for children in the past year. Most PWH and children were treated with factor concentrate. PWH reported arthritis (49%) and HIV/HCV infections (18%/43%) related to haemophilia. Most PWH and parent respondents had received formal education (85%/89%) and were employed full‐ or part‐time (60%/72%). HERO is one of the largest multinational studies focused on psychosocial issues in haemophilia, including historical and treatment information that will allow for multivariate analyses of determinants of health in haemophilia.     

Haemophilia & Exercise Project (HEP): subjective and objective physical performance in adult haemophilia patients – results of a cross‐sectional study

Summary. Recurrent musculoskeletal haemorrhages in people with haemophilia (PWH) lead to restrictions in the locomotor system and consequently in physical performance. Patients’ perceptions of their health status have gained an important role in the last few years. The assessment of subjective physical performance in PWH is a new approach. This study aimed to compare the subjective physical performance of PWH with healthy controls and to correlate the results with objective data. Subjective physical performance was assessed via the new questionnaire HEP‐Test‐Q, which consists of 25 items pertaining to four subscales ‘mobility’, ‘strength & coordination’, ‘endurance’ and ‘body perception’. HEP‐Test‐Q subscales were compared with objective data in terms of range of motion, one‐leg‐stand and 12‐minute walk test. Forty‐eight patients (44 ± 11 years) with haemophilia A (43 severe, three moderate) or B (two severe) and 43 controls without haemophilia (42 ± 11 years) were enrolled. PWH showed an impaired subjective physical performance in all HEP‐Test‐Q subscales and in the total score (52 ± 20) compared with controls (77 ± 10; P ≤ 0.001). Correlation analyses for the total score of the HEP‐Test‐Q and objective data revealed values ranging from r = 0.403 (one‐leg‐stand) to r = 0.757 (12‐minute walk test) (P ≤ 0.001). PWH evaluated their physical performance poorer in comparison with healthy people. As self‐assessment did not always correlate highly with objective data, objective examinations of physical performance in PWH should be complemented with subjective perceptions.     

Prevalence of depression in adults with haemophilia

Multiple factors place adults with haemophilia at risk for depression. Health outcomes can be compromised in depressed patients secondary to increased risk taking behaviour and poor compliance with treatment recommendations. To assess the prevalence and risk factors associated with depression in adult patients with haemophilia treated at a haemophilia treatment centre. Adults with haemophilia were screened for depression during their annual clinic visit using the Patient Health Questionnaire 9 (PHQ‐9), a validated tool for depression screening in adults. Depression was defined as a PHQ‐9 score ≥ 5. Risk factors associated with depression were collected by chart review and correlated with depression scores. A total of 41 adult patients consented to the study and 37% met criteria for depression. Fifty‐three per cent of patients with depression reported moderate to severe symptoms of depression (PHQ‐9 score >10). Seventy‐six per cent of patients with depression reported suffering functional impairment due to their depressive symptoms. Lack of social support and unemployment were significantly associated with higher PHQ‐9 scores (P = 0.04 and P = 0.01 respectively). Adult patients with haemophilia have a high prevalence of depression. The addition of depression screening to the comprehensive care of adults with haemophilia may result in improved overall health outcomes and treatment adherence.     

Impact of joint status on contraction steadiness of m. quadriceps femoris in people with severe haemophilia

Impaired contraction steadiness of lower limb muscles affects functional performance and may increase injury risk. We hypothesize that haemophilic arthropathy of the knee and the strength status of quadriceps are relevant factors which compromise a steady contraction. This study addresses the questions if impaired steadiness of the quadriceps is verifiable in people with haemophilia (PWH) and whether a connection between the status of the knee joint and quadriceps strength exists. A total of 157 PWH and 85 controls (C) performed a strength test with a knee extensor device to evaluate their bilateral and unilateral maximal quadriceps strength and steadiness. Isometric steadiness was measured by the coefficient of variation of maximum peak torque (CV‐MVIC in %). For classification of the knee joint status the World Federation of Haemophilia (WFH) score was used. Lower steadiness (higher CV values) was found in PWH compared with C during bilateral [PWH vs. C; 0.63 (0.36/1.13) vs. 0.35 (0.15/0.72), median (Q25/Q75) P < 0.001] and unilateral trials [left leg: 0.70 (0.32/1.64) vs. 0.50 (0.23/1.04), P < 0.05; right leg: 0.68 (0.29/1.51) vs. 0.39 (0.18/0.68), P < 0.001]. PWH with a WFH score difference (≥1) between their extremities showed a less steady contraction in the more affected extremity (P < 0.05). More unsteady contractions have also been found in extremities with lower quadriceps strength compared with the contralateral stronger extremities (P < 0.001), whereby the weaker extremities were associated with a worse joint status (P < 0.001). The results of this study verify an impaired ability to realize a steady contraction of quadriceps in PWH and the influence of joint damage and strength on its manifestation.     

Haemophilia Experiences,Results and Opportunities (HERO) study: treatment‐related characteristics of the population

The HERO (Haemophilia Experiences, Results and Opportunities) quantitative surveys collected information on characteristics and perceptions of adult persons with haemophilia (PWH) and parents of children with haemophilia. The aim of this article is to describe the perceptions of PWH and parents on psychosocial aspects related to treatment. Two online surveys (one for PWH, one for parents) were conducted in 10 countries. Among 675 PWH respondents, 77% reported having responsibility for their own care; 72% of 561 parent respondents had the main responsibility for their son. PWH were most commonly treated on demand (45% of 648 adults using factor concentrate), with 32% on regular prophylaxis and 23% treated on demand with short‐term prophylaxis (e.g. for sports/physiotherapy). Children were most often treated with prophylaxis (65% of 549 children using factor concentrate), with 26% treated on demand and 8% treated on demand with short‐term prophylaxis. Factor was generally used as instructed at home. Some respondents (41% PWH; 30% parents) had difficulties/concerns with factor availability/affordability. PWH reported more bleeds in the last 12 months than parents reporting their son's bleeds (mean 17.8 vs. 8.7). Both PWH and parents generally perceived that overall, their (their son's) haemophilia was well controlled. Results differed by country. The HERO study captured new, patient‐based data regarding many facets of life relevant to PWH, including treatment. The information conveyed in this article largely represents new insights regarding perceptions of treatment and provides initial benchmark statistics for further research.     

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.     

Cancer detection and management in patients with haemophilia: a retrospective European multicentre study

Lymphomas or hepatocarcinomas related to blood‐borne transmitted diseases are well‐known malignancies in persons with haemophilia (PWH). However, rising life expectancy has increased the number of PWH suffering from other malignancies. This study aimed to collect cancer occurrence data in PWH followed in five European haemophilia treatment centres (Brussels, Geneva, Marseille, Montpellier and Paris‐Bicêtre) over the last 10 years and to analyse some particular features of cancer occurring in PWH. In total, 45 malignancies were diagnosed in 1067 PWH. The most common malignancies were hepatocellular carcinoma (12/45) and urogenital tract tumours (9/45). Bleeding at presentation or changes in bleeding pattern was indicative of cancer in four patients. Three patients with mild haemophilia developed anti‐factor VIII inhibitors after intensive substitution therapy prior to surgery or invasive procedures. There was no bleeding associated with chemotherapy or radiotherapy. A few bleeding complications occurred following invasive (3/39) or surgical procedures (2/27) as a result of insufficient hemostatic coverage or in spite of adequate substitution. No bleeding was noted after liver or prostate biopsies. Following cancer diagnosis, five patients were switched from on‐demand to prolonged prophylaxis substitution. In the majority of cases, the standard cancer treatment protocol was not modified on account of concomitant haemophilia. Thus, oncological treatments are not contraindicated and should not be withheld in PWH assuming that adequate haemostasis correction is undertaken. As shown by our study results, a change in bleeding pattern in adult PWH should raise suspicion of a malignancy. Intensive substitution must be considered a risk factor for inhibitor development.     

E-learning improves knowledge and practical skills in haemophilia patients on home treatment: a randomized controlled trial

Summary. Home treatment of haemophilia is currently the standard of care for patients with severe haemophilia. Home treatment increases the responsibility of the patients for their own treatment and care. Therefore, it is of utmost importance to attain a high level of knowledge and practical skills. The aim of our study was to investigate whether or not an educational e‐learning program improves knowledge and skills of adult patients with haemophilia on home treatment. Participants treated at the Haemophilia Treatment Center of the Erasmus University Medical Centre completed a questionnaire to test their knowledge of haemophilia, treatment of bleedings and of complications of treatment and were observed during the intravenous injection of clotting factor concentrate, using a standardized scoring list. Afterwards they were randomized to follow an e‐learning program or no intervention (control group). After 1 month they completed the same questionnaire again and practical skills were scored once more. At baseline, haemophilia patients (n = 30) scored 24 of 48 questions in the questionnaire correctly. Seventy‐five per cent of the items on the practical skills scoring list were performed correctly. One month later, the e‐learning group (n = 16; 36; 18–45) showed a higher level of theoretical knowledge compared to the control group (n = 14; 26; 19–32; P < 0.001). Also practical skills were significantly better in the group that followed the e‐learning program compared to the control group (respectively P = 0.002). Self‐efficacy of 90% vs. 80% the patients with haemophilia was high in all patients. Our study shows that in haemophilia patients with haemophilia, who are on home treatment, knowledge of haemophilia treatment and complications as well as practical skills can be improved by an educational e‐learning program.     

Burden of illness and costs among paediatric haemophilia patients with and without central venous access devices treated in US hospitals

Introduction

Central venous access devices (CVADs) facilitate repeated or urgent treatments for paediatric haemophilia patients, but are associated with complications. This study examined the burden of illness, healthcare utilization and costs for CVADs in a real‐world hospital setting.

Materials and Methods

This study included haemophilia patients ages ≤18 years with discharges during 2006‐2014 in the US Premier Healthcare Database. Haemophilia was identified using ICD‐9 diagnosis codes and CVAD exposure using billing information. After matching haemophilia patients with and without CVADs on demographic and clinical characteristics, we compared infection, thrombosis, length of stay (LOS), inflation‐adjusted hospital cost (2014 $USD) and readmission outcomes using generalized estimating equation models adjusted for hospital teaching status.

Results

Among 4793 paediatric haemophilia patients treated at one of 548 hospitals, a total of 197 patients were identified with CVAD exposure. The matched sample included 310 haemophilia patients (155 CVAD and 155 non‐CVAD). CVAD cases had greater frequencies of all‐cause infections (29% vs 17%, P = .01) and thrombosis (6% vs 1%, P = .06), longer adjusted mean LOS (9.5 vs 4.7 days, P = .002), higher adjusted mean inpatient total hospitalization costs ($47200 vs $25389, P = .02) as well as more inpatient and outpatient visits at 30‐, 60‐ and 90‐days (P < .05 for all differences) compared with non‐CVAD patients.

Conclusion

Paediatric haemophilia patients with CVADs experienced greater infection rates, healthcare utilization and higher hospitalization costs compared with non‐CVAD patients. The results of this study may inform further research efforts to understand the costs and benefits of novel treatment alternatives for young haemophilia patients requiring CVADs.