Quality of Life/Outcomes Research

Objective:

There are limited data on the impact of haemophilia on health status and health-related quality of life (HRQL) in people affected by non-severe haemophilia. Aim is to evaluate the health status of people living with mild or moderate haemophilia.

Methods:

A cross-sectional, multinational study was conducted as part of the Patient Reported Outcomes, Burdens and Experiences (PROBE) project. Respondents without bleeding disorder (NoBD) and those with mild or moderate haemophilia were included. Respondents were asked to complete the PROBE questionnaire, which contains haemophilia-related questions, general health questions and HRQL. Results were compared between unaffected individuals and people with mild or moderate haemophilia.

Summary:

A total of 862 respondents, of whom 144 with moderate haemophilia, 143 with mild haemophilia and 575 with NoBD were included. Median age (first-third quartile) was 33 (23-46),42 (25-55) and 43 (35-54), respectively. In relation to bleeding in the previous 12 months, respondents with mild reported less bleeding frequency than those with moderate haemophilia, with similar patterns of bleeding frequency seen in both male and female cohorts. Reporting of acute pain is less in those with NoBD compared to the mild to moderate cohorts for both genders (male - 33%, 67%, 77%; female - 38%, 52%, 67%, respectively). Thirteen percent of those with NoBD reported an impact on activities of daily living compared with mild and moderate haemophilia who reported of 35% and 61%, respectively. The impact on quality of life due to mild haemophilia compared to those with NoBD was a reduction of 5.2%, 5.0% and 9.3% in VAS, EQ-5D-5L and PROBE Score respectively (p≤0.001).

Conclusions:

People affected by mild or moderate hemophilia encountered a significant amount of haemophilia related sequalae. Future research is needed to identify the optimal management of moderate and mild hemophilia patients, with particular focus on early identification of patients with a severe clinical phenotype.

Objectives:

Von Willebrand disease (VWD) is the most common bleeding disorder in children and adolescents. Its varied clinical presentation contributes to challenges and delays in diagnosis and management. We characterized diagnosis, bleeding, and treatment patterns in children (2-11yrs) and adolescents (12-17yrs) with VWD.

Methods:

This retrospective database analysis utilized data from IQVIA PharMetrics Plus Database of medical insurance claims for VWD patients (ICD-9 286.4) from 01/01/2006 to 06/30/2015. Patients included had ≥2 medical claims for VWD and continuous enrollment for ≥2 years, to ensure higher likelihood of definitive VWD diagnosis, before/after their 1st VWD claim. Pre-diagnosis period included 18mos of data before diagnosis. Post-diagnosis period included 7-24mos post-diagnosis data. Data from the first 6-month post-diagnosis period were excluded due to data variability, suggestive of treatment optimization. Descriptive statistics were used to summarize patient demographic/clinical characteristics, including types of bleeding episode (BE), rates, outcomes; treating physician specialty; and type of VWD treatment, in both pre-/post-diagnosis periods.

Results:

475/1087 patients identified were children (43% female; mean age at diagnosis 6.9yrs; 612 were adolescents (74% female; mean age at diagnosis 14.9yrs). The top 3 treating physician specialties seen by children in the pre-/postdiagnosis periods, respectively, were hospitalists (21%/9%), primary care physicians (16%/7%), and hematologists (11%/3%). Adolescents were mostly seen by hospitalists (30%/15%), primary care physicians (25%/16%), and obstetrician gynecologists (19%/15%). 11% of children and adolescents saw a hematologist prior to diagnosis, compared with 3% and 5%, respectively, post-diagnosis. A 17%/15% decrease in bleed claims in the pre-/post-diagnosis period was observed among children (40%/23%) and adolescents (59%/44%), respectively. The most common type of BE among children in the pre-/post-diagnosis periods was epistaxis (19%/10%). Heavy menstrual bleeding was the most common BE among adolescents in both the pre-/post-diagnosis periods (40%/30%; in females 54%/40%). Epistaxis was the second most common BE among adolescents in both the pre-/post-diagnosis periods (11%/7%), and in females (9%/5%), but highest among males (17%/12%). Overall, VWD-related treatment claims increased between the pre-/post-diagnosis periods for both children (12%/23%) and adolescents (31%/50%). The most prescribed treatments for bleed management in children were aminocaproic acid (ACA), desmopressin (DDAVP) and nasal cauterization (pre-diagnosis: 5%, 4% and 4%, respectively; post-diagnosis: 11%, 13% and 3%, respectively). For adolescents, the most prescribed treatments, pre- and post-diagnosis respectively, were oral contraceptives (22% and 33%, DDAVP (9% and 19%) and ACA (4% and 11%).

Conclusions:

This analysis demonstrates a decrease in BE claims following VWD diagnosis and a rise in ACA and DDAVP treatment claims in both children and adolescents, and in oral contraceptive claims among female adolescents. Nevertheless, a considerable proportion of children and adolescents continue to experience BEs 6mos post-diagnosis. This emphasizes the need for treatment optimization and improvement in care and management of patients in these age groups.

Objectives:

To better understand what symptoms beyond bleeds are experienced, as well as the depth of impact of these symptoms and how they uniquely impact people living with hemophilia on a daily basis. Additionally, the study aims to better understand patients’ satisfaction with current treatments in addressing their hemophilia needs.

Design/Method:

An email invitation was sent to all U.S. members affiliated with hemophilia A of MyHemophiliaTeam, a social network of people diagnosed with or caring for someone with hemophilia. 54 members responded to a 24 question survey between April 19 and May 1, 2017.

Results:

Hemophilia had a significant negative impact on the day-to-day life of adults (72%) and children (52%). Pain was the most broadly and acutely experienced symptom: 60% of adults and 28% of caregivers felt that pain had a major impact on their lives and 33% of adults and 25% of caregivers considered mobility to be significantly impacted by hemophilia.

For adults, both pain and mobility limitations impacted sleep (71% and 45%, respectively), being able to perform chores (71% and 65%), and the ability to work (48% and 45%). For children, these conditions impacted school attendance (61% and 58%) and participation in high impact activities like running or playing soccer (56% and 75%).

Depression and anxiety were also common symptoms that impacted sleep across adults (71%, 61%) and children (60%, 55%). Adults most commonly reported feeling negative ones: stress (38%), fatigue (38%) and annoyance (35%).

81% of adults and 86% of caregivers were extremely or very satisfied with current treatment. However, needs beyond treating bleeds are currently not being met. Few felt their pain was adequately addressed by current therapies (74% of adults and 57% of children reported no relief). Mobility impairment issues were also not being adequately addressed. Time spent on treatment impacted people with hemophilia (39% of adults and 43% of children, respectively were not satisfied with the frequency of treatment).

Background:

While people with hemophilia are known to suffer from bleeding, numerous concomitant symptoms also burden these patients, including pain, mobility impairments, depression, and anxiety. These symptoms can have a significant impact on quality of life, limiting work and school attendance, causing social withdrawal, and encouraging inactivity. Additionally, available treatment options can sometimes fall short in treating the totality of hemophilia symptoms.

Conclusions:

People with hemophilia have many challenges beyond bleeds that are not currently being well addressed. This is particularly true for the pain experienced. As such, a more holistic approach to treating hemophilia beyond bleeds would be beneficial to patients living with hemophilia. Additionally, therapies that reduce the need and frequency for treatment could potentially lower the burden of disease.