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.
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.
Objective:
Emicizumab is indicated for prophylaxis in persons with hemophilia A with or without factor VIII (FVIII) inhibitors to reduce or prevent bleeds. This study provides an early view of individuals treated with emicizumab, stratified by FVIII inhibitor status.
Methods:
This study used commercial claims data from MarketScan Commercial Research (11/2016–12/2018) and PharMetrics Plus (11/2016–3/2019) Databases. To be included in the study, individuals were required to have evidence of emicizumab use, and to be continuously enrolled in the 12-month period preceding first emicizumab use. Demographics, clinical characteristics (major bleeds, arthropathy, and pain), utilization of FVIII and bypassing agents (BPA), and healthcare resource use were examined in the 12-month period preceding first emicizumab use. An algorithm developed by Shrestha et al. (2017) was used to identify major bleeds, defined as a cluster of bleeding claims for the same body part in a 7-day period.
Summary:
A total of 107 patients with at least one emicizumab claim were identified. All patients were male (100%); average age was 24 years (standard deviation [SD]±17.6; range=1–62) with 17% (n=18) aged ≤5 years. In the 12-month period preceding first emicizumab use, 28% (n=30) had evidence of major bleeds, with an average of 2.2 bleeds (SD±1.9; range=1–8) among those with at least one major bleed; 17% (n=18) had evidence of arthropathy and related disorders, and 12% (n=13) had a diagnosis of pain. A total of 79% (n=85) had evidence of FVIII use and 17% (n=18) had a BPA claim (evidence of FVIII inhibitors) with an average of 10.3 (SD±12.3) and 8.6 (SD±7.9) prescriptions, respectively. Individuals with FVIII inhibitors were younger (mean age±SD: 18.0±13.3 vs. 25.4±18.2 years), and a larger proportion experienced major bleeds (44% vs. 25%), had evidence of arthropathy (44% vs. 11%) and pain (44% vs. 6%) compared to those without FVIII inhibitors. Of the 107 patients identified, 35% (n=37) had at least one emergency room visit (mean±SD=0.7±1.4); 14% (n=15) had at least one inpatient stay with mean length of stay of 1.1 (SD±3.5) days; 82% (n=88) had at least one outpatient hospital visit (mean±SD=4.1±9.7); and 88% (n=94) had at least one office visit (mean±SD=8.0±9.7). Generally, individuals with FVIII inhibitors had higher healthcare resource use compared to those without FVIII inhibitors.
Conclusions:
To our knowledge, this is the first real-world claims study, which describes the characteristics of individuals initiating emicizumab, and underscores their disease and treatment burden. Results show utilization of emicizumab in patients with a broad range of clinical characteristics and across various age groups. Longer follow-up data will help to further examine these real-world outcomes.
Objective:
Understanding characteristics of the aging hemophilia A (HA) population is critical to informing appropriate management strategies. There is paucity of published literature describing the aging HA population. This study aims to examine real-world demographic and clinical characteristics, treatment patterns, and outcomes among treated persons with hemophilia A (PwHA), stratified by age.
Methods:
This retrospective study used data from the Humana Research Database from 01/2007–07/2018. PwHA enrolled in commercial or Medicare Advantage Prescription Drug plans were identified based on diagnosis codes and treatments for congenital HA. Index was the first evidence for treatment during the identification period. Persons included were required to be enrolled for 12 months after inclusion in the study. Unadjusted comparisons on characteristics, clinical outcomes, and healthcare resource utilization (HCRU) were conducted across age groups (<18, 18–55, >55 years).
Summary:
A total of 294 PwHA were identified; the majority were adults (18–55 years=60%, >55 years=17%), males (96%), 59% were seen in a hemophilia treatment center, and 91% had ≥1 FVIII claim. Overall, 30% of patients had evidence of prophylactic treatment. The most common comorbidities were arthritis (40%) and hepatitis C (19%). Severity could be identified in 19%; the majority of which were mild (50.9%). Prophylactic treatment was higher among PwHA aged <18 years (45%) compared with those aged 18–55 years (31%) and >55 years (<20%), while on demand use was more common among those aged 18–55 years (63%) and >55 years (69%) compared with those aged <18 years (49%). Overall, a total of 21% of patients experienced ≥1 bleeding event in the 12-month post-index period, and 41% and 53% had evidence of arthropathy or related disorders, and pain, respectively; all were highest among PwHA aged >55 years. Generally, HCRU increased with age and was highest in PwHA aged >55 years.
Conclusions:
PwHA represent a medically complex group and incur substantial HCRU. Insights on differences in treatment, outcomes, and HCRU as PwHA age may identify opportunities for enhanced disease management particularly for older patients.
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:
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.
Helping teens with bleeding disorders prepare to manage their care as they transition to adulthood is a national priority for US Hemophilia Treatment Centers (HTC). The National HTC Patient Satisfaction Surveys (PSS) reveal high satisfaction with HTC teen transition services. Yet how satisfaction differs comparing HTCs that primarily care for children to HTCs that care for patients throughout the lifespan is unknown.
Objective:
To assess variation in patient satisfaction with US HTC teen transition services by HTC type.
Methods:
The US HTC Network conducted nationally uniform patient satisfaction surveys in 2015 and 2018 on care received, respectively, 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. Participation was voluntary. Respondents were anonymous but identified their HTC. Parents completed surveys for children under age 18. The PSS included two teen transition questions for respondents age 12-17 to complete. HTC type was categorized as ‘pediatric’ if >80% of responses were from patients/caregivers of individuals under age 18, and ‘adult’ if >80% were from patients over age 24. All other HTCs were categorized as ‘lifespan’. For both years, approximately 26% of HTCs were classified as pediatric, 52% as life-span, and 22% as adult.
Results:
Over 700 teens age 12-17 (or their parents/guardians) from an average of 130 HTCs (94.0%) from all US regions participated in 2015 and 2018. Approximately 96.5% of teens at pediatric HTCs (96.4% - 96.5%) and 96.2% at lifespan HTCs (95.9% - 96.5%) reported being ‘always’ or ‘usually’ (A/U) satisfied with HTC services overall. On average, 90.4% of teens at pediatric HTCs (90.1% - 90.7%) and 91.0% at lifespan HTCs (90.3%–91.6%) reported being A/U satisfied with how HTC clinic staff talked about how to care for the bleeding disorder as they became an adult. Similarly, 92.5% (92.0%– 92.9%) of teens at pediatric HTCs and 92.5% (92.3%-92.7%) reported being A/U satisfied with how the HTC clinic staff encouraged them to become more independent in managing their bleeding disorder.
Conclusions:
HTC patients age 12-17 years consistently report very high levels of satisfaction with HTC teen transition services, regardless if the HTC primarily cares for patients up to age 17, or throughout the life-span. This suggests teens receive support and tools to successfully transition to adult care across the US HTC Network. A national uniform HTC Patient Satisfaction Survey provides vital information, is feasible to conduct using a regional structure, and well received nationwide.
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.
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:
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.
