Quality of Life/Outcomes Research

Introduction/Background:

Maintenance of oral health is frequently challenging for US adults with a bleeding disorder. Underlying causes are due mainly to (1) lack of dental insurance and (2) lack of knowledge among dentists about how to perform safely either primary dental care or oral surgery. Without regular care, patients are at risk for advanced dental disease, which is associated with chronic inflammatory conditions, including type 2 diabetes and cardiovascular disease. Research indicates that controlling periodontal disease reduces inflammatory markers throughout the body, including synovial joints.

Materials and Methods:

The Gulf States Hemophilia and Thrombophilia Center (GSHTC) in Houston, Texas serves a diverse urban population; historically, about 50% of adult patients receive no dental care. In October 2019, GSHTC added adult dental exams to comprehensive care services. Patients are evaluated by University of Texas School of Dentistry residents and scheduled for routine or specialized dental procedures as needed. To assess the scope of dental needs in the GSHTC patient population, we examined the range of procedures recommended and the proportion of patients reporting oral health care problems. We report here preliminary results from the initial six months of adult dental services, October 2019 – March 2020. 

Results:

In this period, 171 adults with a bleeding disorder received dental evaluations, and 146 completed the Oral Health Impact Profile (OHIP-14). The OHIP-14 is a validated survey instrument consisting of 14 questions measuring seven dimensions of oral health: functional limitation, physical pain, psychological discomfort, physical disability, psychological disability, social disability and handicap. Responses are rated on a 5-point Likert scale: 0=never, 1=hardly ever; 2=occasionally, 3=fairly often; 4=very often/every day. Total OHIP-14 scores can range from 0 to 56 and are calculated by summing the values for the 14 items. Higher scores indicate worse and lower scores indicate better health-related quality of life (HRQoL). Scores ranges from 0-52; 80 (55%) individuals had a score of 0-1 (no oral health problems); 48 (33%) scored between 2-9, and 18 (12%) had scores of 10 or greater. Since October 2019, nearly all adult patients seen in clinic have received a dental evaluation; approximately 40 patients in need of dental care have received treatment or a treatment plan. Procedures include deep cleaning, regular/surgical extractions, fillings, scaling, root planing, crowns, bridges and root canal, all of which require infusion of factor concentrate prior to treatment.

Conclusion:

A substantial proportion of patients who completed the OHIP-14 reported reduced HRQoL related to poor oral health; subsequent dental examination confirmed the urgent need for treatment in these individuals. Continued administration of the OHIP-14 will monitor the impact of dental treatment on patient HRQoL. Ultimately, we hope to provide evidence of the need for widespread inclusion of adult dental care among US Hemophilia Treatment Centers.

Objectives:

Incidence of hemophilia is commonly cited in the literature as 1/5,032 male births (Soucie et al., Am J Hematol 1998). Estimates of the true population prevalence of hemophilia A (HA; factor VIII deficiency) and B (HB; factor IX deficiency) are limited, and prevalence is influenced by the longer patient life expectancy now versus previous decades. We calculated updated epidemiologic estimates of HA and HB in the USA based on a literature review and other data sources.

Methods:

Data were collected from a systematic literature search (1970–2018) in Medline, EMBASE, conference proceedings, and other secondary data sources including registries (e.g., Registry for Bleeding Disorders Surveillance [part of Community Counts]). Keywords included: hemophilia, bleeding disorder, factor VIII or IX deficiency, incidence, prevalence, mortality, diagnosed, undiagnosed, severity. Eighteen references contributed to the USA analysis. Variables assessed comprised: incidence, prevalence (diagnosed, undiagnosed, total), and disease severity (mild, moderate, severe). Lastly, a simplified Markov model was developed to calculate the annual incoming patients, deaths and prevalence of HA or HB in a given year, including projections to 2020.

Summary:

As most literature references the 1/5,000 male birth incidence rate for HA and HB combined, we used this and assumed it remains constant. Based on assumptions from 2015, and an incidence rate of 15.49 for HA and 4.24 for HB per 100,000 male births there were 321 incident patients with HA and 88 with HB in the USA. Total USA prevalence was estimated to be 22,118 for HA and 6,058 for HB. Diagnosed and undiagnosed prevalence rates per 100,000 males were: 8.69 and 4.87 for HA, and 2.57 and 1.14 for HB, respectively. The corresponding percentage of patients with mild, moderate, and severe disease, respectively was 34, 16, and 51% for diagnosed HA and 39, 33, 28% for diagnosed HB. In addition, we assumed severity rates for undiagnosed HA were 70, 30, and 0% and 60, 40, 0% for undiagnosed HB. Given current treatment rates, we assumed prevalence rates plateaued around 2015. Therefore, 2015 prevalence rates were used to project prevalence in 2020. Based on the model, predicted total prevalence of hemophilia A and B in 2020 is estimated to be 22,913 and 6,276 patients. (Table)

Conclusions:

Overall, the model assumes constant incidence rates of HA and HB. Due to exclusion of undiagnosed patients in previous literature, this model’s total prevalence estimates are higher. In the USA, thousands of patients remaining undiagnosed suggest room for improvement in diagnosis and/or reporting.

Objectives:

Clinical profiles of hemophilia B range from mild to severe forms of the disease. Prior studies have investigated the economic burden of hemophilia B, but focused on outcomes within the overall study sample, without stratifying by disease severity or clinical profile. The present study sought to develop an algorithm to identify clinical profiles of hemophilia B for burden of illness assessment, based on indicators that are observable in US payer claims databases.

Methods:

Adult (≥18 year-old) male patients with ≥2 diagnoses of hemophilia B were identified from the Marketscan Commercial and Medicare Supplemental Databases (06/2011−02/2019). For each patient, an index date was randomly selected among all hemophilia B diagnosis dates meeting the requirement of continuous enrollment for 1 year pre-index (baseline) and 1 year post-index (study period). Clinical profile was categorized as severe, moderate-severe, moderate, or mild based on frequencies of FIX replacement claims and hemorrhage events at baseline. The selection of profile indicators was informed by literature and clinical expert opinion. To assess the discriminatory ability of the algorithm, healthcare costs were summarized by clinical profile during the study period.

Summary:

A total of 454 patients were included (mean age: 46 years). At baseline, the algorithm classified 66 (15%) as severe [≥6 FIX claims], 69 (15%) as moderate-severe [4-5 FIX claims, or ≤3 FIX claims and ≥1 hemorrhage], 118 (26%) as moderate [1-3 FIX claims and no hemorrhage], and 201 (44%) as mild [no FIX claims or hemorrhage]. During the study period, mean total healthcare costs were higher among patients identified as having more severe profiles (severe: $643,979; moderate-severe: $254,077; moderate: $141,101; mild: $83,291).

Conclusions:

The hemophilia B clinical profile algorithm developed in this study identified four subgroups with increasing healthcare costs according to severity. The development of the first claims-based algorithm to identify clinical profiles creates opportunities to expand potential uses of US payer claims databases for understanding disease burden and unmet medical needs in hemophilia B.

Background:

Von Willebrand disease (VWD) is an inherited bleeding disorder caused by a quantitative or qualitative deficiency of the protein, von Willebrand factor (VWF). There is a lack of clear guidance on best practices to inform the care of people with VWD.

Objectives:

Identify and prioritize the main topics of a collaborative guideline development effort.

Methods:

A scoping survey to prioritize topics to be addressed in a collaborative guideline for VWD was distributed to international stakeholders including patients, caregivers, clinicians, and allied healthcare professionals. The distribution strategy was coordinated by the guideline chairs and representatives of the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH). The survey was conducted in English, French, and Spanish. The survey focused on both diagnosis and management of VWD, using 7-point Likert-scale response options and open ended comments. Descriptive analysis of participants and comparative analysis of results by stakeholder subtype (patients/caregivers versus healthcare providers [HCP]), gender, and income setting was performed. Qualitative conventional content data was analyzed utilizing both deductive and inductive coding processes.

Results:

601 participants responded to the survey (49% patients/caregivers, and 51% HCPs). The highest priority topics identified were diagnostic criteria/classification, bleeding assessment tools, treatment options for women, and surgical patients. In contrast, screening for anemia and plasma-derived therapy versus recombinant therapies were rated the lowest priority topics (figures 1 – 2).

Conclusion:

The survey results highlighted areas of importance in the diagnosis and management of VWD across diverse groups of stakeholders and will direct future guideline efforts. The large number responses (601) and discrete comments (9,500) attest to the interest and involvement of the VWD community in this effort.
 

Objective:

While a new generation of therapies for patients with Hemophilia A are available, it is unclear what patient characteristics, perceptions, and barriers are associated with the decision to switch FVIII replacement therapies. This study assessed patient characteristics, health history, and reasons for switching from a FVIII product with more frequent dosing (³3x infusions/week) to a product with less frequent dosing (≤2x infusions/week) from patient/caregiver and physician perspectives.

Methods:

Data collection was a mix of qualitative and quantitative procedures. The qualitative portion consisted of two online discussion forums: patients (n=17) and caregivers of patients (n=11) receiving a FVIII product dosed ³3x/week, and patients (n=22) and caregivers of patients (n=5) who switched to a product dosed ≤2x/week. The quantitative portion was a retrospective medical chart review (n=207) which captured variables (e.g., bleed rate, treatment history) 6 months pre- and 6 months post-switching to a product with less frequent dosing.

Summary:

Prominent drivers among patients for starting a FVIII product with less frequent dosing included: 1) experiencing diminished effectiveness while on a product dosed ³3x/week resulting in increased breakthrough bleeding, 2) experiencing vein access issues, and 3) beginning prophylaxis as opposed to on-demand infusions after a bleed.

Key barriers to changing included: 1) fears regarding the process of switching being complicated, time consuming, and costly, 2) perceived risks associated with switching, and, 3) possible lack of healthcare provider support.

Physicians were most likely to report that patients switched products because they sought a newer product with twice weekly dosing or less per FDA-approved dosing recommendations (35.3%), followed by patient requested the switch (30.4%), and patient sought a reduction in infusion frequency to improve adherence (27.5%).

Switching to a product with less frequent dosing was associated with improvements in patient-reported bleeding-related outcomes. Patients were more likely to self-administer the treatment post-switch (63.8%) compared with pre-switch (48.8%; p<0.001) and had fewer infusions per week post-switch (2.8 vs. 3.3; p<0.001). Patients’ annualized bleed rate was lower (5.9) post-switch compared with pre-switch (7.7; p<0.001).

Both the number of spontaneous joint bleeds and joint bleeds after trauma or injury were lower (3.2 and 2.7) post-switch (3.6 and 4.3; p=0.044 and p<0.001). The bleeding event was less likely to be classified as moderate or severe (34.5% and 5.9%) post-switch compared with pre-switch (55.0% and 10.0%; p<0.001 and p=0.049). Fewer infusions were required to resolve the bleeding event post-switch (2.6 vs. 3.2; p<0.001).

Conclusion:

A prominent reason why patients switch treatment is to improve bleeding-related outcomes. Indeed, we found that switching to a FVIII product with less frequent dosing was associated with improved patient-reported bleeding-related outcomes. These findings are critical for improving patient outcomes and support the FDA mandate to incorporate patient perspectives in the regulatory process.

Background:

US Hemophilia Treatment Center (HTC) care reduces mortality and hospitalizations, and guidelines recommend this care model.  Yet national data that uniformly and longitudinally monitors patient experience with HTC care is limited.

Objective:

To assess patient satisfaction with HTC services and clinicians over time.

Methods:

The US HTC Network conducted the first ever nationally uniform patient satisfaction surveys on care received in 2014 and 2017. A Regional workgroup devised, piloted, and finalized an electronic, two-page survey for self-administration at clinic, or at home, in English or Spanish. Content was based on national instruments to enhance comparability and scientific robustness. Questions assessed demographics; satisfaction with HTC team members and services; insurance and language barriers. Respondents were anonymous but identified their HTC.  Participation was voluntary.  Patients with HTC contact in 2014 and 2017 were eligible. Data were collected for 4 months in 2015 and 6 in 2018; on average 130 HTCs (94%) from all US regions participated. Parents completed surveys for children under age 18. Data were entered, analyzed and aggregated at national, regional and HTC levels at a central site.

Results:

5006 and 4767 persons participated, respectively, in 2015 and 2018.  In both years, over 1400 (30%) respondents were female, nearly 80% were White, and 10% Hispanic. On average, 3038 had Factor 8 or 9, 1280 Von Willebrand, 186 other factor deficiencies and 369 other bleeding disorders.  Respondents reported being ‘always’ or ‘usually’ (A/U) satisfied with HTC staff and services from 90% - 97% of the time in both 2014 and 2017. In both years, >4400 gave these highest A/U ratings for HTC Hematologists and Nurses; 3300 for Social Workers; >2600 for Physical Therapists; 1400 for Genetic Counselors, and >1100 for Psychologists.  In both years, 96% were A/U satisfied overall with HTC services. Over 95% gave the A/U satisfaction ratings both years for these services: getting needed care and information, being treated respectfully, spending sufficient time with staff, and involved in shared decision making. 82% and 91% of respondents, respectively, gave the A/U satisfaction ratings for care coordination with primary care providers and other specialists.  Over 90% of >700 youth age 12-17 gave HTC teen transition services the A/U satisfaction ratings both years. 96% of >2760 respondents reported A/U satisfaction with their HTC Pharmacy (340B) Factor Program in 2017. Insurance and language barriers to HTC care posed problems A/U for 27% and 15%, respectively both years.

Conclusions:

Patients consistently report high levels of satisfaction with HTCs, documenting HTC value over time.  Patient satisfaction influences treatment adherence, can influence reimbursement, and is increasingly required by payers. A national uniform survey is feasible to conduct using a regional structure to implement, is well received by patients, and provides critical information to stakeholders.
 

Objective:

Hemophilia may negatively impact a patient’s health utility and quality of life (QoL). Health state utility values (HSUVs) and QoL are important inputs to the evaluation of novel treatment being developed in hemophilia, including gene therapies. This systematic literature review identified and evaluated HSUVs and QoL for people with hemophilia (PWH) type A and/or type B, as well as utility decrements relevant to the experience of PWH, by treatment and health state.

Methods:

Building on a review undertaken in 2014 (Grosse et al. 2015), we conducted a systematic literature review to March 2019 through a search of electronic medical databases, including MEDLINE®, Web of Science, Cochrane Library databases and the School of Health and Related Research Health Utilities Database (SCHARRHUD). Major clinical, patient, and pharmacoeconomic conferences in 2016-2019 were also queried. Studies were independently double screened by independent reviewers, after which data extraction was performed.  The information extracted included study design, description of treatment and health state, respondent details, instrument and tariff, HSUV and QoL estimates, quality of study, and appropriateness for use in economic evaluations of novel treatment.

Summary:

Of 1,511 titles and abstracts screened, 20 studies and 12 conference abstracts were included. The studies identified applied a mix of direct and indirect health utility elicitation techniques. Two studies applied direct time trade-off (TTO) methodology and the remaining 30 studies adopted indirect valuation methodologies. HSUVs were found to decrease with increasing disease severity. For example, in Hoxer et al. (2018), mean (standard deviation) HSUV were 0.80 (0.21), 0.73 (0.22) and 0.67 (0.25) in people with mild, moderate, and severe hemophilia, respectively.

Utility values were also found to vary by severity of musculoskeletal damage, frequency of bleed episodes, inhibitors, hemophilia subtype, treatment regimen, treatment adherence and other disease-related complications. Interestingly, HSUVs derived from valuations from the general public were found to be valued lower than those derived from PWH for similar health states. For example, in Carlsson et al. (2017), general population participants consistently rated significantly lower HSUVs for hemophilia disease states compared to PWH (range: 0.54-0.60 vs. 0.67-0.73).

Several hemophilia-specific QoL instruments were used alongside HSUV evaluations. These QoL findings further contribute to improving the understanding of the impact of hemophilia on PWH.

Conclusions:

This systematic review shows significant impact of hemophilia on health utilities and QoL among PWH. The substantial humanistic burden experienced by PWH highlights unmet needs remaining in hemophilia. Our review findings also suggest potential disease state adaptation among PWH, which warrants further research using robust patient preference studies.
 

Objective:

The impact of hemophilia on working life, the ability to pursue a desired career or sustain full employment have been previously reported. The aim of this analysis was to seek a better understanding of hemophilia-related health problems likely to impact employment.

Methods:

The analysis utilized data from the Patient Reported Outcomes Burdens and Experience (PROBE) study. Participants selected from among 7 possible responses (working full-time, working part-time, student full-time, student part-time, long-term disability, early retirement and other (e.g., unemployed, parental leave, retired)) to describe their current employment or school status. People with severe hemophilia A and B (PwSH) and controls with no bleeding disorder (NoBD) were compared for the proportion (percentage) reporting either working part-time due to their health or having retired early due to their health, the proportion reporting working full-time. Descriptive statistics were used to present the results as n (%), and odds ratio (95% CI) were calculated for the associations and assessed for their statistical significance.

Summary:

Data from 1009 participants (550 PwSH, 458 NoBD) ≥age 18 from 21 countries was analyzed. Mean age of participants was 39 (14.4 SD) for PwSH and 45.3 (13.7 SD) for NoBD. 250 PwSH (45.5%) and 263 NoBD (57.4%) reported working full time; 86 PwSH (15.6%) and 80 NoBD (17.5%) reported working part-time. 27 of the 86 PwSH (31.4%) and 3 of the 80 NoBD (3.8%) reported working part-time due to health. 52 PwSH (9.5%) and 28 NoBD (6.1%) reported taking early retirement. 25 of the 52 PwSH (48.1%) and 1 of the 28 NoBD (3.6%) reported taking early retirement due to health. Analysis of the association between reporting a health-related problem and reporting to be working part-time or taking early retirement due to health include [odds ratio (95% CI), positive numbers indicating #times higher chance of being part-time/retired early]: use of mobility aids 77.7 (3.8-1645), acute or chronic pain 41.2 (2-831.8), use of pain medication 23 (2.05-258.1), participants with any health problems 22.5 (2-252.6), chronic pain 16.5 (1.5-179.2), difficulties with activities of daily living 16.5 (1.5-179.2), and history of joint surgery 7.3 (0.4-148).

Conclusion:

PwSH are more likely to report working part-time or having taken early retirement due to health-related problems than people with NoBD. Among the study population, we find a significant negative impact of hemophilia on employment status. PwSH is associated with a higher rate of working part-time or retiring early due to health than age-matched controls. Use of mobility aids, acute and chronic pain, difficulties with activities of daily living and history of joint surgery are associated with working part-time or retiring early.

Objective:

To identify sociodemographic, clinical and treatment characteristics associated with the quality of life (QoL) of individuals with hemophilia B (HB) using longitudinal data in the HUGS Vb cohort.

Methods:

Between 2009-2012, 148 persons with HB were enrolled into HUGS Vb, a prospective cohort study examining individuals seen at ten federally supported U.S. hemophilia treatment centers (HTCs). Participants or parents of pediatric enrollees completed periodic surveys; data from 107 individuals with at least three follow-up surveys, clinician charts and dispensing records were included in the analyses. Data were analyzed at baseline and 6-month intervals across 2 years, yielding 5 time points. Periodic QoL assessments (SF-12 for adults and PedsQL for children), self-reported pain, employment and insurance status, time lost from work/school and treatment regimen were collected. Descriptive statistics and Spearman’s correlation coefficient test were used to examine the associations.

Summary:

Forty-six percent of the sample had severe HB; 50% were children (2-17 years). Among those with severe HB, 64% of children and 50% of adults treated prophylactically. 58% of adults were employed full-time. Individuals with mild hemophilia missed more work/school days due to disease-related issues (8 days) than those with moderate hemophilia (2 days) or severe hemophilia (3 days, P=0.03). QoL scores were similar over time among those using prophylactic and on-demand treatment for both adults and children. Median adult Mental Component Scores (MCS) and Physical Component Scores (PCS) measured at 5 time points ranged from 53.0 to 55.1 for MCS and 45.5 to 50.5 for PCS, with no significant changes observed over time. However, adults employed full-time had significantly higher median PCS at each time point than those working less than full-time (all Ps<0.05). Adults who reported pain had significantly lower median PCS than those who reported no pain/pain only when bleeding at each time point (all Ps<0.03). Median MCS remained similar between the two groups. Overall, we observed no longitudinal differences in children’s total PedsQL scores (range of median: 81.2-92.4) or in functioning subscales. However, among 18 children with QoL scores at both baseline and 24 months, missed school days were significantly correlated with decreased social functioning over time (rho=0.73, P<0.001). 8% of children who reported pain had consistently lower median total QoL scores than those reporting no pain/pain only when bleeding, despite having access to insurance and prophylactic treatment.

Conclusions:

Longitudinal data collected by HUGS Vb provide a valuable opportunity to examine the association of HB patient characteristics with measures of QoL in a multi-state sample. These data demonstrate that lower QoL was consistently associated over time with multiple factors, including absence from school, unemployment and pain. Continued analysis of this cohort will increase our understanding of the challenges faced by persons with HB.

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.

Objective:

Functional impairment from recurrent joint bleeding in people with hemophilia results in joint pain and reduces quality of life. The P-FiQ study formally evaluated patient- and site-reported functional assessment including responses to generic and hemophilia-specific patient-reported outcomes (PROs) tools. Psychometric analyses were used to evaluate reliability, validity, and consistency of responses.

Methods:

Adult males with hemophilia and a history of joint pain or bleeding completed a hemophilia history and 5 PROs assessing function: EQ-5D-5L, Brief Pain Inventory v2 Short Form (BPI), International Physical Activity Questionnaire (IPAQ), Short Form-36 v2 (SF-36v2), and Hemophilia Activities List (HAL). PROs were assessed for reliability, consistency, and correlation, with factors including patient-reported characteristics.

Summary:

A total of 381 adults (median age, 34 years; range, 18-86 years) were enrolled in P-FiQ. Most participants (66%) and sites (59%) reported functional disability in the past 6 months (CDC-UDC scale). Patients self-reported arthritis/bone/joint problems (65%) and history of joint procedures or surgeries (50%). On EQ-5D-5L, most reported problems “today” with mobility (61%) and usual activities (53%) but fewer with self-care (19%). On BPI, similar median pain interference scores (0- 10 scale, 10 is complete interference) were reported with general activity (3.0), walking ability (3.0), and normal work (3.0). On IPAQ, physical activity was reported by 49% of respondents over the prior week, with more reporting walking (35%) than moderate (16%) or vigorous (16%) activities. On SF-36v2, activities in the past 4 weeks that were most frequently limited were vigorous activities (80%), bending, kneeling, or stooping (67%), walking more than a mile (61%), and climbing several flights of stairs (59%). Physical problems caused participants to limit kinds of work/activities (69%), accomplish less than they would like (66%), have difficulty in performing work/activities (65%), and reduce time spent on work/activities (62%). On HAL, greater difficulties were seen for lower vs upper extremity functions/activities; within the lying/sitting/kneeling/standing domain, the most frequent problems in the previous month were squatting for a long time (74%), kneeling (73%) or standing (72%), and kneeling/squatting (70%). Similar items across different PROs were correlated with one another. Self- reported functional impairment was significantly differentiated by BPI pain interference, IPAQ total activity, SF-36v2 physical functioning, and all HAL domains and summary scores.

Conclusion:

PRO instruments assessing functional status range from simple/generic (EQ-5D-5L) to complex/disease-specific (HAL) and provide varying levels of detail. Greater use of formal PRO instruments in the clinical setting may improve dialogue between health care professionals and patients/caregivers and inform proactive approaches to specifically target patient identified functional limitations (eg, HAL) and identify areas for further targeted management strategies.

Objective:

Pain and functional impairment resulting from joint disease in patients with hemophilia (PWH) may impact emotional well-being, resulting in consistent reports of anxiety/depression. The P-FiQ study formally evaluated patient-reported anxiety/depression symptoms and treatment as well as responses to standardized patient-reported outcomes (PROs), and evaluated reliability, validity, and consistency of responses.

Methods:

At a comprehensive care visit, adult male PWH with a history of joint bleeding or pain completed a hemophilia history and 3 patient-reported outcomes (PROs) assessing anxiety/depression and quality of life (QoL): EQ-5D-5L, Brief Pain Inventory (BPI), and SF-36v2. PROs were assessed for reliability, consistency, and correlation with factors including patient-reported characteristics.

Summary:

A total of 381 PWH (median age, 34 years) were enrolled in P-FiQ; 77% had hemophilia A, 23% had hemophilia B, and 9% had inhibitors. Fewer than half (44%) were currently receiving routine infusions to prevent bleeding. More than half were employed full-time (53%), and 65% were married or had a long-term partner. Depression was reported by 19% and anxiety by 14%. On the EQ-5D-5L anxiety/depression item, 43% reported feeling anxious or depressed “today.” On BPI, most participants indicated that pain interfered in the previous week with mood, sleep, and enjoyment of life, and more than half (54%) indicated interference with relations with other people. On SF-36v2 (range 0- 100, higher scores indicate better QoL), median mental health summary score was 50.7; subdomains were similar (vitality, 49.0; social functioning, 45.6; role emotional, 55.9; mental health, 52.8). Emotional problems resulted in reduced time spent on work/activities (40%) and accomplishing less than they would like (47%). More than half (55%) had felt downhearted or depressed, and a large majority (93%) had felt tired in the past 4 weeks. Sixty percent of participants indicated that their physical or emotional problems had interfered with their normal social activities with family, friends, and other contacts. Similar items across PROs correlated with one another, and PRO scores (EQ-5D-5L anxiety/depression, SF-36 mental health) were significantly (P<0.05) correlated with self-reported anxiety/depression.

Conclusion:

Anxiety and depression in adults with hemophilia have been consistently reported in other studies and were identified in P-FiQ by self-report and across several PRO instruments. Emotional problems were reported to interfere with normal social activities and productivity. While the unmet need to address mental health in PWH has received increased recognition, it is not typically assessed formally. When compared with pain, management strategies and/or referral relationships may also not be as formally established. The findings presented here highlight the potential value of simple screening tools (eg, EQ-5D-5L) and opportunities to encourage patient dialogue about mental health within the comprehensive care setting and in referral networks.

Objective:

People with hemophilia frequently experience joint bleeds, resulting in pain and functional impairment. The P-FiQ study formally evaluated patient-reported pain descriptions, responses to standardized patient-reported outcomes (PROs) related to pain, and pain management strategies.

Methods:

Participants completed a pain/hemophilia history and 5 PRO instruments. Pain was assessed via 3 PRO instruments: EQ-5D-5L, Brief Pain Inventory v2 Short Form (BPI), and Short Form-36 v2 (SF- 36v2), and these instruments were assessed for reliability, consistency, and correlation with factors including patient-reported characteristics.

Summary:

P-FiQ enrolled 381 adult males with mild-severe hemophilia and a history of pain and/or joint bleeding. Most (65%) self-reported having arthritis/bone/joint problems. Thirty-two percent of participants reported experiencing both acute and chronic pain, 35% chronic pain only, 20% acute pain only, and 15% no pain. Of those reporting acute pain, most described the sensation as “sharp” (77%) or “aching” (65%); for those reporting chronic pain, most described the pain as “aching” (80%) or “nagging” (50%). Ankles (37%) and knees (24%) were commonly reported as the most painful joints. Many participants with acute/chronic pain reported using acetaminophen (62%/55%) or nonsteroidal anti-inflammatory drugs (34%/49%) to treat their pain in the past 6 months. Some participants indicated having moderate/severe/extreme (28%/12%/2%) pain/discomfort “today” as measured by the EQ-5D-5L pain/discomfort domain. For BPI (scale 0-10, 10 is most severe pain), median pain severity scores were 6.0 for worst pain, 3.0 for average pain, 2.0 for current pain, and 1.0 for least pain. Median BPI pain interference scores indicated interference with general activity (3.0), mood (3.0), walking ability (3.0), normal work (3.0), and enjoyment of life (2.0). On SF-36, most participants (90%) reported experiencing bodily pain, and 75% indicated that pain interfered with normal work in the last 4 weeks. Assessments of pain on PROs were highly correlated with one another. The following formal PRO assessments were associated with self-reported pain: pain/discomfort domain of EQ-5D-5L, BPI worst pain, least pain, average pain, and current pain, and SF-36 bodily pain. Greater extent of lifetime routine infusions was also associated with EQ-5D-5L pain/discomfort and SF-36 bodily pain.

Conclusion:

Pain severity and interference in people with hemophilia were identified consistently across several PROs, and correlated with patient-reported pain. In the comprehensive care setting, greater use of formalized assessment tools over time would improve dialogue and pain assessment between healthcare professionals and patients, document and validate the presence and extent of pain, establish and monitor individual goals for pain management interventions, and encourage the exploration of various pain management strategies and the evaluation of their overall quality and effectiveness.

Objectives:

The primary aims of this study are to 1) evaluate the prevalence of depression and anxiety among adult persons with hemophilia (PWH) and 2) explore relationships between depression, anxiety, chronic pain, and adherence to clotting-factor treatment.

Method:

This study used a subset of data from the IMPACT QoL II, a one-time, cross-sectional survey of 200 adults (age >18) with self-reported diagnosis of either Hemophilia A or B who were able to read, write, and speak English. The study was approved by the IRB in the primary investigator’s institution. Participants were a convenience sample recruited at bleeding disorders conferences in 2013-2014 and issued a randomly generated identification number to ensure anonymity. The 139-item survey was completed electronically through SurveyMonkeyTM on a study computer tablet. Participants were given $20 upon completion of the survey, which took 15-45 minutes to complete. The subset of variables evaluated for this analysis included the Faces of Pain Scale-Revised (FPS-R) (chronic pain), level of control over pain each participant feels they have, adherence to clotting factor measured by total scores (higher score=lower adherence) on the VERITAS-PRO or VERITAS-PRN, anxiety measured by the GAD- 7, depression measured by the PHQ-9, and self-report of ever receiving a diagnosis of depression and/or anxiety. The cut-off score for presence of moderate to severe depression or anxiety was 10 on both the PHQ-9 and the GAD-7, scores which have been validated by previous literature in other populations. Lower vs. higher adherence was defined by VERITAS scores in the highest and lowest quartile respectively.

Summary:

Participants with lower treatment adherence (VERITAS score >57) were more likely to have PHQ-9 scores >10 (P=0.02). GAD-7 scores >10 also demonstrated a trend to be associated with lower treatment adherence (P=0.15). 28% of participants reported a diagnosis of depression, but 53% with PHQ-9 scores >10 had not been diagnosed with depression. Similarly, 22% reported a diagnosis of anxiety but 52% with GAD-7 scores >10 had not been diagnosed. Participants who reported non-control of pain were more likely to have PHQ-9 scores or GAD-7 scores >10 (P=0.001 and P=0.013 respectively). There were significant correlations between PHQ-9 and GAD-7 (Rho=0.76, P<0.0001), PHQ-9 and FPS-R (chronic pain) (Rho=0.31, P=0.003), and GAD-7 and VERITAS (P=0.005). These data indicate that depression and anxiety are associated with greater severity of chronic pain, and depression is associated with poorer adherence to clotting factor treatment regimen (prophylaxis or other regimen). Both depression and anxiety appear to be under-diagnosed in PWH.

Objective:

To assess the effect of digital monitoring on bleeding rates in patients with hemophilia A using prophylaxis.

Methods:

A total of 294 eligible patients with hemophilia A were included in our observational study. Eligible patients used clotting factor concentrates and had no active inhibitors. Patients used a digital health tool powered by MicroHealth to log bleeds and infusions via smartphone, texting, or online. The study observational period was August 2014 to January 2015. Patients using the tool could choose to invite their care professionals for monitoring. For each patient, Hemophilia Treatment Center (HTC) monitoring was defined as having at least one HTC professional: 1. Linked to the patient and with online access to the infusion logs; 2. Receiving notifications when the patient had bleeds and/or had adherence below a threshold, and; 3. Having two or more patients under monitoring. There were a total of 35 and 259 patients in the monitoring and non-monitoring groups, respectively.

We conducted Bayesian analysis using linear mixed models and a negative binomial distribution to compare the relative risk of bleeding rates for patients with and without HTC monitoring.

Summary:

Patients using HTC monitoring had a relative bleeding rate of 0.60 vs. patients without monitoring, which is equivalent to a 40% reduction in bleeding rates for monitored patients (95% credible interval: 0.38—0.96).

Patients on the monitoring and no monitoring groups were comparable except that the monitoring group had 23% more pediatric patients (p<0.001). However, bleeding rates between pediatric and adults were comparable (p=0.500). Subgroup analysis showed no differences in the reductions of bleeding rates due to monitoring between pediatric-only and adult-only subgroups (p=0.353).

Conclusions:

The use of digital tools for chronic care monitoring is a growing global trend.

A reduction in the annualized bleeding rate of 40% (~2 bleeds a year) is both statistically and clinically significant and may have a cumulative protective impact on patients’ long-term outcomes. Observational studies are subject to sample bias, however, patients in both groups were technologically savvy and motivated enough to track their condition using a digital health tool. Given that this intervention is free, safe, and fits the accountable care model, we encourage clinicians to explore its adoption. Confirmatory studies on this topic are encouraged.

Objective:

Personalizing treatment to a patient’s lifestyle and promoting overall health and wellness in persons with hemophilia (PWH) is essential to optimizing outcomes. There is limited evidence that correlates how activity and infusions impact bleeding episodes and further data on this relationship is needed. The research objective of SPACE is to prospectively explore the association between activity level, timing of infusion, and occurrence of a bleeding episode in PWH using novel technology.

Methods:

This six-month prospective, observational study includes PWH A or B in the United States currently receiving ADVATE or RIXUBIS between the ages of 13 and 65 years. Enrolled PWH use a smartphone eDiary application to log information on activities, infusions, and bleeding episodes. As an additional measurement of activity, enrollees are given a FitBit, a consumer-based activity tracker that measures steps taken and calories burned. Activity types are assessed based on their level of perceived risk for collision, according to the NHF “Playing It Safe” brochure. We report here current study status and descriptive analysis of baseline data.

Results:

The interim analysis included 15 patients with a median age of 19 (Range: 13 to 47). At baseline, 87% of patients were on prophylaxis and 13% treated on-demand treatment. Fifty-three percent of patients had 0 target joints at baseline. Eighty-seven percent of patients indicated that they had discussed activity participation with their physician. Sixty-seven percent of patients considered themselves ‘very satisfied’ or ‘satisfied’ with their level of activity. Data collected from the FitBit indicated that patients in SPACE walked on average 7,367 (SD: 3250) steps per day and burned 979 (SD: 398) calories from their activity. For patients on prophylaxis, the mean number of days per week doing mild, moderate and strenuous activity were 3.57, 2.64, and 1.5 respectively. Of the data reported on bleeding episodes, 40% of patients reported no bleeds at the time of the interim analysis. Forty percent of patients did not report having a bleed at the time of the interim analysis. Of all bleeds reported, 34% were associated with physical activity.

Conclusions:

Current data from SPACE demonstrates that subjects are active and participating in various activities. Continued data will provide better understanding of the types of activities and infusion schedules that may be associated with risk as well as protective effects on bleeding episodes by infusing prior to activity. A personalized approach to treatment based on physical activity levels may minimize bleeding risk in PWH.

Background:

Until recently, the hemophilia B community has only had standard Factor IX (FIX-Fc) products as treatment choices. With the availability of additional standard rFIX products and extended half-life (EHL) rFIX-Fc, there has been much debate (1-4) regarding clinical and cost expectations, balanced with optimizing use in patients. To level the debate, an understanding of real-world prescription costs of patients who switch to EHL FIX-Fc products is warranted.

Objectives:

1) To characterize patients who switch from rFIX to EHL rFIX-Fc, and 2) to understand their change in factor costs.

Methods:

Retrospective analysis using national US specialty pharmacy dispensing databases from Jan 1, 2013-Apr 25, 2015 of Hemophilia B (ICD-9 code 286.1) individuals who switched from rFIX to rFIX-Fc. Descriptive statistics summarized patient characteristics. Patients who had at least 90 days of prescription coverage on both products were included. Utilization was averaged on a monthly basis pre and post switch. Costs were calculated by multiplying the units by the wholesale acquisition prices for each product (Red Book) and the percentage change in cost from rFIX to EHL-rFIX was compared. This analysis studied patients who switched but stayed on similar regimens. Cohorts were characterized as prophylaxis to prophylaxis (P to P), or on-demand to on-demand (OD to OD).

Results:

Sixteen switchers were included in the study. Hemophilia severity was reported as 62.5% severe, 12.5% moderate, 6.25% mild, and 18.75% of unknown severity. The P to P cohort comprised of the majority of patients at 87.5% (n=14) while OD to OD were smaller at 11% (n=2). The median ages for these cohorts were similar. The P to P median age was 15 (range: 5-51) and OD to OD was 14 (range: 13-15) years old. Median prescription costs increased in the P to P cohort by 40% (range: -56% to 181%), while OD to OD increased by 173% (range: 1% to 348%).

Conclusion:

While the availability of EHL rFIX-Fc allows hemophilia B patients an option with less frequent infusions, our findings demonstrate that for those who initially switched from prophylaxis to prophylaxis or from on-demand to on-demand regimens, costs increased for the majority of patients. This analysis provides initial real-world cost data for those who have switched to EHL and may be helpful in decision makers’ understanding of the value of EHL rFIX-Fc in hemophilia B.

Objective:

To assess pain and functional impairment through 5 patient-reported outcome (PRO) instruments in non-bleeding adults with hemophilia.

Methods:

Adult men with hemophilia (mild-severe) with history of joint pain/bleeding completed a hemophilia/pain history and 5 PROs (EQ-5D-5L with visual analog scale [VAS], Brief Pain Inventory v2 [BPI-SF], International Physical Activity Questionnaire [IPAQ-SF], SF- 36v2, and Hemophilia Activities List [HAL]) during routine visits. Initial patients were asked to complete the PROs again after their estimated 3-4 hour visit (retest population). PRO scores were calculated from published algorithms. Generally, higher scores indicate better health- related quality of life (HRQoL) and functional status, and greater pain severity/interference.

Summary:

381 Patients were enrolled between October 2013 and October 2014; 164 of the initial 187 completed the retest and are reported here. Median time for completion of the initial survey/PROs was 36.0 minutes and 21.0 minutes for the PRO retest. Most retest subjects had hemophilia A (74.4%) and were white-non-Hispanic (72.6%). Median (Q1, Q3) age was 33.9 (26.9, 46.0), 48.7% were married, 62.6% had some college or graduate education, 80.7% were employed, and 61.0% were overweight or obese. HCV (49.4%) was more common than HIV (16.5%); 61.0% self-reported arthritis/bone/joint problems. Median EQ-5D- 5L VAS was 80.0 (0-100 scale), and EQ-5D-5L health index 0.80 (-0.11-1 scale); 61.6% reported any problems with mobility (29.3% reported moderate/severe problems), 55.8% with usual activities (18.4% moderate/severe), and 22.0% with self-care (4.3% moderate/severe). 73.2% reported pain-discomfort (43.3% moderate/severe), and 41.1% anxiety-depression (14.7% moderate/severe). For BPI, median pain severity was 3.0 (0-10 scale) and pain interference 2.9 (0-10 scale); median worst pain was 6.0, least pain 2.0, average pain 3.0, and current pain 2.0. Pain most impacted general activity, mood, walking ability, and normal work, and least impacted relations with other people. Ankles were the most frequently reported site of pain. Median IPAQ total activity was 693.0 MET/min/week; 49.3% reported no activity in the prior week. Median SF-36v2 scores (0-100 scale) were lower for physical health (39.6) than for mental health (51.6). Median overall HAL score was 76.1 (0-100 scale); complex lower extremity activities were the most impacted activity domain.

Conclusions:

These 5 PRO instruments provide different levels of detail describing the impact of hemophilia on pain and function, and consequently, have varied burdens of administration. PRO data from the retest population demonstrate that most adults with hemophilia experience pain and functional impairment that impacts HRQoL, highlighting the importance of assessments and patient dialogue.

Background and Objective:

Standard outcome measures in hemophilia, such as the annual bleeding rate, have inherent limitations in both clinical practice and in research, including lack of both sensitivity and personalization. Goal attainment scaling (GAS) is a method for evaluating outcome that is patient-centered and sensitive to change in individuals and populations; collaborative goal setting may also be viewed through the biopsychosocial lens as a potential resource to strengthen disease-related coping. GAS has been used successfully in several disease areas. However, feasibility can be an issue. To address this, standardized goal attainment menus can be developed that preserve individualization, while allowing greater ease of use by non-experts. We initiated a process to develop a novel outcome measure in hemophilia, the GAS-Hem, by creating a goal attainment menu for patients living with hemophilia.

Methods:

We conducted 2 multidisciplinary workshops with participants from medicine, nursing, social work, and physical therapy (n=12). During the first workshop, we developed a list of goal areas, each with an associated set of descriptors of attainment levels. At the second workshop, a second group of experts (n=8) critically reviewed each goal to evaluate its importance and relevance for inclusion in the GAS-Hem.

Summary:

28 goal areas with associated descriptors of attainment levels were developed, covering 3 broad categories: ability to manage hemophilia, ability to recognize and treat complications, and ability to cope with the impact of hemophilia on daily life. Descriptors incorporated several key parameters for each goal (eg. skill level, desire for change, utilization of available resources) (Table).

Conclusions:

We developed a novel, patient- centered outcome measure for patients with hemophilia. A comprehensive, preliminary list of goal areas and attainment levels was successfully created. The next step will be to incorporate feedback from patients and families, after which a feasibility study will be conducted to evaluate content and construct validity and overall ease of use.

Table. Examples of Goal Areas and Descriptors

Objective:

Hemophilia is marked by frequent joint bleeding, resulting in acute and chronic pain and functional impairment. Surveys in US adults with hemophilia demonstrate suboptimal pain management and quality of life (QoL). The objective of P-FiQ was to methodologically assess QoL parameters, including functional impairment and pain, and pain management strategies.

Methods:

Adult men with mild-severe hemophilia with a history of joint pain and/or bleeding completed a hemophilia/pain history and various patient-reported outcome assessments (completed twice in “retest” population of initially enrolled patients).

Summary:

Of 164 adults with hemophilia (median age 34) in the “retest” population, more had hemophilia A (74%) than B (26%); 6% had inhibitors. Most had some college-or-above education (63%), 81% were employed, 61% were overweight/obese, and 61% self-reported arthritis/bone/joint problems. Current patient-reported treatment regimens were prophylaxis (42%), on-demand (39%), or mostly on-demand (19%; 25/31 using infusions ahead of activity). One-third (32.9%) reported unrestricted school/work/recreational activities in the prior 6 months; 6.2% reported needing assistance for school/work/self-care, or did not participate in recreational activities because of pain, loss of motion, and weakness. Some patients (31.3%) reported using a cane/crutches/walker (3.8% always) and 7.6% a wheelchair (1.3% always), and 47.0% reported a history of joint surgery (41 knee, 37 ankle, 29 elbow). Patients reported losing an average of 3.7 and 1.8 school/work days in the previous 6 months due to lower and upper extremity problems, respectively. Patients reported that during the prior 6 months they had experienced acute pain only (24%), chronic pain only (33%), or both (29%); 15% reported no pain. Acute pain was most frequently described as sharp (77%), aching (66%), shooting (57%), and throbbing (55%), and chronic pain as aching (74%), nagging (49%), throbbing (44%), and sharp (40%). Most common analgesics in the past 6 months for acute/chronic pain were acetaminophen (69%/58%), NSAIDs (40%/52%), hydrocodone-acetaminophen (29%/33%), oxycodone (12%/11%), and oxycodone- acetaminophen (9%/8%). Most common nonanalgesic treatment strategies reported for acute/chronic pain in the past 6 months were ice (73%/37%), rest (48%/34%), factor or bypassing agent (48%/24%), elevation (34%/28%), relaxation (31%/23%), compression (27%/21%), and heat (25%/15%); other reported strategies include medical marijuana (17%/9%), physical therapy (12%/9%), prayer (11%/8%), faith (9%/8%), alcohol (8%/7%), aquatherapy (5%/6%), illicit drugs (4%/2%), acupuncture (2%/1%), hypnosis (2%/1%), and biofeedback (1%/1%).

Conclusions:

Initial data corroborate the high prevalence of pain and functional disability in adults with hemophilia and highlight opportunities to address clinical assessment, patient dialogue, and management strategies to improve outcomes.

Objective:

Clinical knowledge gaps of hemophilia can affect patient outcomes through delayed diagnosis/referral as well as improper monitoring and interventions. A study was undertaken to identify and characterize clinical practice gaps and confidence levels in the management of hemophilia specific to pediatricians.

Methods:

Building upon a previous assessment developed in 2014, an updated global, hemophilia-specific continuing medical education-accredited clinical practice assessment survey was developed utilizing current evidence-based consensus guidelines and best practices, including guidelines from the National Hemophilia Foundation and the World Federation of Hemophilia. The assessment included both knowledge- and case -based, multiple-choice questions that healthcare providers completed confidentially on-line between March 23, 2015 and April 9, 2015. Areas such as appropriate triggers for initiating prophylaxis and use of physical therapy were assessed. Responses from pediatric providers were de- identified and aggregated prior to analysis.

Summary:

660 pediatricians (30% of total respondents) completed the survey, from the following locales: North America (36%), Asia (23%), Europe (15%), Middle East (10%), Africa (7%), Central/South America (6%), and Australia (4%). Academic (31%), private practice (27%), community hospital (19%), community clinic (12%), and hemophilia treatment center (3%) practice settings were identified. Analysis of pediatricians who indicated professional interaction with hemophilia patients (87% of pediatrician respondents) demonstrated knowledge gaps including (% incorrect responses): classification of severity of hemophilia (37%); optimal use of prophylactic therapy, e.g., when to initiate (31%), at what dose (53%), prophylaxis in active patients (26%); likelihood of inhibitors (75%); using bypassing therapy (58%); comprehensive care model (61%); supporting overall joint health and quality of life (70%); and adherence (60%). A low level of confidence in the ability to identify when to use prophylaxis was reported among 31% of pediatricians. The top barriers to the administration of prophylaxis identified by the pediatric providers included lack of availability of FVIII or FIX concentrates, lack of venous access, and insurance coverage (29%, 22%, and 21% for respondents, respectively).

Conclusions:

This study demonstrated gaps in knowledge and confidence about the assessment and optimal care of hemophilia for pediatricians, suggesting that further education specific to the needs of these providers is warranted.

Objective:

To evaluate the effect of bleeding episodes on hemophilia patient burden of illness using observational data.

Methods:

Between 2005-2007 and 2009-2012, the Hemophilia Utilization Group Studies Va and Vb, respectively, recruited patients from ten Hemophilia Treatment Centers (HTCs) in eleven states. Adult patients or parents of children with hemophilia A or B completed an initial survey assessing socio-demographics, clinical characteristics, and treatment regimen. Work absenteeism, underemployment due to hemophilia, and unpaid hemophilia-related caregiver time were recorded at regular intervals over two years to estimate indirect costs using the human capital approach. Direct costs were estimated using healthcare services utilization and drug dispensing records. All costs were annualized and converted to 2014 US dollars. Annual mean bleeding episodes were calculated from patient-reported number of bleeds recorded in follow-up interviews, and used to stratify patients into bleeding categories of 0, 1-3, 4-6, 7-9, and 10+ bleeds. Associations between bleeding episodes and healthcare utilization, work productivity losses, and total costs were analyzed in patient subgroups based on both severity and treatment regimen.

Results:

Of 477 recruited patients, 352 with complete healthcare utilization and dispensing records and at least three months of follow-ups were included. A larger proportion of hemophilia A patients had severe disease and used prophylaxis compared to those with hemophilia B, but no socio-demographic variables differed significantly between the groups. Among severe patients, adults compared to children and episodic treatment compared to prophylaxis users had significantly more average annual bleeds [respective mean(standard deviation): 16.24(13.9) vs 5.54(9.47), p<0.0001 and 15.69(12.65) vs 8.39(11.1), p<0.0001]. Nearly two-thirds of the 82 severe patients using episodic treatment (63.4%) had 10 or more annual bleeds. Higher bleeding categories (more annual bleeds) were significantly associated with higher annual mean indirect costs for mild/moderate patients using episodic treatment [mean range across categories: $423-$21,434 (p=0.0003)] and severe patients on prophylaxis [$6,467-$14,890 (p=0.005]. Increased bleeding was also significantly associated with higher mean total costs in episodic treatment users with mild/moderate disease [$17,373- $136,552 (p<0.0001)] and severe disease [$83,957-$226,614 (p=0.008)]. Across all subgroups, increased bleeding was associated with more emergency room visits, outpatient procedures, and missed days of work, oftentimes reaching statistical significance.

Conclusions:

A larger proportion of severe hemophilia patients treating episodically have poor bleed management compared to those on prophylaxis. Overall, higher bleeding frequency is associated with both higher direct and indirect costs for individuals with hemophilia A and B across disease severity and treatment regimen.

Objective:

Patient satisfaction with healthcare services enhances patient experience, improves outcomes, and is increasingly mandated by public and private payers. While many US Hemophilia Treatment Centers (HTC) periodically assess patient satisfaction, the lack of a uniform survey hampered national measurement. To remedy this knowledge gap, the US HTC Network implemented a national patient satisfaction survey in 2015.

Methods:

A Regional HTC Coordinator workgroup devised, piloted, and finalized a two-page survey for self-administration online, at clinic, or at home, in English or Spanish and mailed to households. Survey content and format were based on national health surveys to enhance comparability and scientific robustness, informed by legacy regional HTC surveys. Questions assessed patient demographics; satisfaction with services, team members, and care processes; and Healthy People 2020 adolescent transition objectives. Surveys included open ended questions to obtain qualitative data. Respondents were anonymous but identified with their respective HTCs. Participation was voluntary. Persons with genetic bleeding disorders who had HTC contact in 2014 were eligible. During February 2015, 124/130 HTCs sent surveys to 27,563 households. Parents completed surveys for children under age 15. No reminders were sent. Data were entered and analyzed at a central site and aggregated at national, regional and HTC levels.

Results:

Over 4800 households (17.4%) returned surveys by April 30, 2015. National analyses on 4332 surveys reveal that 96.6% were ‘always’ or ‘usually’ satisfied with HTC care. Over 80% were ‘always’ satisfied with the core HTC team members. Three quarters of 12-17 year olds were ‘always’ satisfied with HTC encouragement regarding becoming more independent, and how the HTC discussed caring for a bleeding disorder upon reaching adulthood. Eighty– 90% were ‘always’ or ‘usually’ satisfied with care processes, e.g. shared decision making, care coordination, ease of obtaining timely information and services, and being treated respectfully. Insurance and language were ‘always’ a problem for 20%. 29.0% of respondents were female and 10.3% Hispanic. 83.4% were Caucasian, 5.8 African- American, 3.1% Asian/Pacific Islander or Native Hawaiian, 4.3% Multiple races, and 4% Other. Over half had severe or moderate FVIII or FIX deficiency or VWD Type 3. Ages ranged from newborns to 96 years: 38% under 18, 20% age 18 – 34, and 42% over age 35.

Conclusions:

Implementing a National Patient Satisfaction Survey for the US HTCN is feasible, and provides valuable information. Satisfaction with HTC services is high, but insurance and language ‘always’ pose problems for one fifth. Further analyses will examine regional differences.