Objective:
To determine the medical and educational needs reported by persons with Type 3 and other severe types of Von Willebrand Disease (VWD) who attended the second USA National Type 3/Severe VWD Conference held in Florida in June 2018.
Little research has been done concerning medical issues and education in Type 3/Severe VWD. As patient identification increases, it is vital that education, support and resources are available for these patients.
Methods:
A survey of 48 questions was developed and administered to 74 vetted patient attendees. Responses for any individual question varied between 62-66. The survey was administered through an Audience Response System (ARS) utilizing handheld clickers. The responses were compiled and immediately visually available to the respondents via a projector screen.
The multiple-choice questions were used to identify basic demographics, medical and psychosocial concerns, and educational needs.
Summary:
In this self-reported ARS survey, basic demographic data was obtained. This sample of VWD patients reported a need for more education on several issues related to their medical and psychosocial issues including depression/mental health issues, lab results and product choices. In addition, subjects reported significant needs for care, treatment and education in the fields of orthopedic services and genetic counseling.
Respondents' answers expressed a lack of orthopedic care despite a need for it. Only 8 (13%) patients reported having an orthopedic surgeon attend his/her bleeding disorder clinic. Forty-two (67%) did not know of any orthopedic resources. However, 18 (28%) reported that he/she had already had at least one joint surgery/procedure due to VWD and 5 (*%) plan to have surgery in the future. Eight (12%) had had joint replacements.
Only 25 (40%) of respondents knew that they had undergone genetic testing related to their bleeding disorder, 30 (48%) have not had genetic testing, 8 (13%) were unsure. When asked, “Were your parents diagnosed with a bleeding disorder before your birth?” of the 63 who answered, 51 (81%) stated “no, neither parent”. When asked if a parent was diagnosed with a bleeding disorder after the respondent’s birth, 24 (38%) responded “yes” to one or both parents. Twelve (19%) respondents have had their diagnosis change since first being identified with a bleeding disorder.
Conclusion:
Orthopedic care, genetic testing and education are vital services wanted by Type 3/Severe VWD patients. The community should further evaluate these needs and take action to respond. These results may also empower persons with Type 3/Severe VWD to seek support from professional and social members of their community.
Objective:
To investigate how bleeding disorder characteristics influence patient perceived challenges and management strategies.
Methods:
This is a mixed-method, retrospective, cross-sectional continuation of a pilot study identifying themes of self-perceived challenges and management strategies for persons with bleeding disorders. Sixty-one participants with a bleeding disorder (BD), either hemophilia (PWH) or Von Willebrand disease (PWVWD), were asked what their most significant challenge was in managing their BD and how they managed that challenge. Data were collected from March, 2017 through December, 2018, coded for themes and uploaded to NVivo. Similar themes were grouped for analysis. Subject-level data was extracted from the electronic medical record including demographics, disease type, severity and presence of joint disease (JD). Pain interference was determined from participant response to the Brief Pain Inventory (BPI).
Results:
Conclusions:
Gaining insight to patient-perceived challenges and management strategies is important to be able to tailor an effective treatment approach that is individualized and meets the changing needs of PWBD across circumstance and life-course.
Introduction:
Photovoice is a qualitative research method that has been used for communities to share pictures as a tool for discussion that is often used at a grassroots advocacy level. Photovoice can show both strengths about a topic or concerns. Photovoice can create empowerment by sharing perspectives and can also create a foundation to advocate for awareness and change.
Long-term Goal:
To create more awareness surrounding bleeding disorders during the month of March, which is bleeding disorders awareness month.
Objectives:
Methods:
People that follow the Tri-State Bleeding Disorder Foundation on social media as well as members of a closed social media group that consist of parents of children who are patients at Cincinnati Children’s Hospital were asked to participate in the pilot Photovoice project. Participants were asked to share pictures on their own social media pages and to use hashtags to link the photos to the Tri-State Bleeding Disorder Foundation’s page. The project was promoted by sharing an infographic that explained Photovoice and the details of the project. Several community stakeholders were identified as people active on social media and they were personally asked to participate so that examples of the project could be shared with others. There were weekly themes and a weekly contest for pictures that best exemplified that week’s theme with the winners winning a small gift card.
Summary:
This innovative pilot project applied the methodology of Photovoice to social media to generate awareness and advocacy during bleeding disorders awareness month. The theme of this Photovoice project was “Living with Hemophilia”. Weekly themes consisted of: living with a new diagnosis, living with treatment, living and learning about a bleeding disorder, and living with health and being physically active.
Conclusion:
Utilizing Photovoice and applying this methodology to social media as a pilot project with the bleeding disorder population is an innovative idea. This grass roots level movement is a modern way for people to share their story of living with a bleeding disorder. To date, the use of this methodology with the bleeding disorder population has not been documented in the literature. Participants in this project reported satisfaction with being a part of the project. The project’s authors reported that it was a positive and creative way to create more awareness on a personal level about bleeding disorders and they plan to repeat the project in the future.
Introduction and Objectives:
Up to an estimated 1% of women in the United States have a bleeding disorder, but many with symptoms go undiagnosed. The National Hemophilia Foundation (NHF) conducted a needs assessment of currently diagnosed women to understand their path to diagnosis to help inform creation of an awareness campaign called Better You Know.
Materials and Methods:
In 2015, NHF fielded a survey of diagnosed women, yielding 184 responses. Informed by the needs assessment and input from a working group of medical providers and consumers, NHF launched the Better You Know campaign in 2016, which includes a website with a validated screening tool, resources on where to find providers on the path to diagnosis and treatment, outreach videos, social media posts, promotional postcards, paid media articles, accredited medical provider webinars, and mini-grants to select chapters for local outreach.
Results:
NHF found that women finally sought care for their symptoms for the following reasons: significant bleeding incident (surgery, childbirth, etc.), symptoms got too bad, or a family member was diagnosed. Women reported seeing the following providers first on their path to diagnosis: 38% hematologist; 23% primary care physician, 16% OB/GYN, and 15% pediatrician. About 80% of women also reported going to other people they know with a bleeding disorder for information and support. This lead to the creation of betteryouknow.org and other related campaign elements. From launch in July 2016 through October 2017, the website drew 4107 sessions, with 413 completing the screening tool and 86% of those being at risk. There were 108 clinicians who received accreditation for the provider webinars. NHF has developed partnerships with feminine hygiene product companies to spread the word, and pushes out campaign messaging via social media, chapters and some paid media. Total audience (website visits, social media impressions, etc.) for the campaign to date is over 252,500,000.
Conclusions:
Undiagnosed women with bleeding disorders face true challenges due to their bleeding symptoms. NHF will continue to raise awareness with providers and women, utilizing findings for effective methods of communication and education, through the Better You Know campaign.
Objective:
Physicians prescribing Factor VIII for haemophilia A patients are presented with an array of dose and dose frequency in the package insert making it difficult to precisely prescribe. To clarify the treatment outcome across products, a model of the published half-life was used to provide the expected % IU/dl +/- SD at 24, 48, 72, and 96 hours post dose at a normalized dose and at the high end of the recommended dosage (dose and freq.).
Methods:
Eleven rFVIII products recently marketed in the US were assessed using a model of the halflife +/- SD to calculate the expected %IU/dl +/- SD at 24, 48, 72, and 96 hrs post-dose. Terminal half-life data for adults was obtained from each published package insert (PI). The one-stage clotting assay data was used for all except Afstyla© (chromogenic assay only). Variance reported as coefficient of variation or confidence intervals was converted to standard deviation.
The first comparison was made using a standard dose of 50 IU/kg across all products. The second comparison used the maximum recommended dose and frequency for routine prophylaxis defined in the PI. Full data sets and curves of %IU/dl+/-SD under standard doses of 50 IU/kg and under maximum dose and frequency for routine prophylaxis have been generated.
Summary:
Graphing the single dose comparison (50 iu/kg) revealed 3 general clusters and one outlier at 24 hrs post dose. The first cluster had 4 products with a mean range of 21-25 %IU/dl at 24 hrs post dose. The second cluster had 5 products with a range of 30-32 %IU/dl at 24 hrs. The third cluster had 1 product (Eloctate©) with 43% IU/dl at 24 hrs. The outlier (Nuwiq©) showed exceptional variance compared to others and removed from discussion.
Expected %IU/dl was recalculated using the most frequent and highest dose recommended for individual products. Assessing the –SD value: 10 of 10 products achieved > 1% trough at 24 hrs; 8 of 10 at 48 hrs; 2 of 10 at 72 hrs; and 1 of 10 at 96 hrs. Note at 72hrs, only 2 of 10 products achieve minimal trough at –SD level when 4 of 10 claim q3day dosing.
Conclusions:
Initial dosing for routine prophylaxis relies on the mean half-life. The PK parameters are based on only 18 to 30 patients under well-controlled conditions. There is waste on both sides of the PK half-life distribution curve. Would it be reasonable to assure minimal breakthrough bleeds and less over-dosage by initiating treatment with a personal PK profile for each patient in order to identify the correct dose and frequency? This model could be used to find optimal dose and frequency with input from a personalized product half-life.
Objective:
Knowledge gaps among clinicians regarding diagnosis, classification, and/or management in hemophilia can potentially delay diagnosis/referral and lead to adverse clinical outcomes. A study was undertaken to identify hemophilia clinical practice gaps among pediatricians.
Methods:
A global, hemophilia-specific continuing medical education-accredited clinical practice assessment survey was developed based on 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 21, 2014 and April 16, 2014. 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 analyses.
Summary:
817 pediatricians (42% of total respondents) completed the survey, from the following locales: North America (29%), Asia (23%), Europe (16%), Middle East (13%), Africa (9%), Central/South America (6%), and Australia (3%). Academic (36%), private practice (26%), community hospital (24%), community clinic (9%), and hemophilia treatment center (1%) practice settings were identified. For most responses, the proportion of incorrect responses appeared to be consistent regardless of whether pediatricians indicated professional interaction with hemophilia patients (Group A: 60%) or not (Group B: 40%). Pediatrician knowledge gaps included (% incorrect responses): classification of severity of hemophilia (28% A v. 39% B; P=.0030); optimal use of prophylactic therapy, e.g., when to initiate (29% A v. 30% B; P=.83), at what dose (16% A v. 17% B; P=.93); likelihood of inhibitors (51% A v. 55% B; P=.14); and adolescent care, e.g., adherence (25% A v. 22% B; P=.33), transitioning (14% A v. 14% B; P=.36), and long-term prophylaxis (76% A v. 76% B; P=.68). Differences in correct responses were observed when comparing Australia, Europe, and North America versus Africa, Asia, Central/South America, and the Middle East on topics such as classification of severity of hemophilia (P<.0001) and when to initiate prophylaxis (P=.04), although knowledge gaps existed in both groups. A low level of confidence in ability to identify when to use prophylaxis was reported among 42% [95% CI: 40%-46%] of pediatricians. The top barriers to the administration of prophylaxis included cost and lack of availability of FVIII or FIX (41% and 26% for all respondents, respectively).
Conclusions:
Substantial knowledge gaps permeate pediatric clinical practice in the diagnosis and optimal care of hemophilia. Educational efforts tailored to the practice setting and geographic locales are warranted.
Objective:
To characterize the one-year bleeding pattern and assess the burden of bleeding among persons with hemophilia B, a subgroup of individuals with hemophilia that is frequently under-represented in population-based and health services surveillance.
Methods:
Hemophilia Utilization Group Study Part Vb (HUGS-Vb) collected prospective information about bleeding episodes, healthcare utilization, and burden of illness among persons with hemophilia B who obtained comprehensive care at one of ten hemophilia treatment centers in eleven geographically diverse states. Participants completed an initial interview and quarterly follow-up surveys. This analysis reports on baseline and one-year follow-up data from 93 participants. Wilcoxon-Mann-Whitney tests were used to determine the differences among subgroups. Spearman correlation coefficients (rho) were used to assess the relationship between bleeding episodes and utilization of health-related care.
Results:
Of the 93 participants, 50 (54%) were children. Forty-four (47%) participants (24 children) had severe hemophilia B, of whom 26 (59%) (16 children) reported using prophylactic therapy at initial interview. Fifty-eight (62%) participants reported having at least one bleeding episode within the one-year follow-up period. Mean number of bleeding episodes among participants with mild, moderate or severe hemophilia were 2.11±2.75, 3.47±6.25 and 6.53±7.66, respectively. Participants with severe hemophilia on prophylactic therapy had significantly (p=0.0203) fewer bleeding episodes (4.67±6.31), compared to those on episodic therapy (9.22±8.76). Significantly fewer outpatient visits (rho=0.2831, p=0.0060) and emergency room visits (rho=0.2810, p=0.0064) were reported among participants with fewer bleeding episodes. Mean absent days from work/school due to hemophilia among all participants was 2.24±4.58, and it was 0.98±2.16 days among parents of children under 18 years. Increased bleeding episodes were positively associated with increased days absent from work/school among both participants (rho=0.4598, p<0.0001) and parents of children (rho=0.3433, p=0.0147). Moreover, Positive relationship were found between increased bleeding episodes and increased time spent on the telephone with hemophilia centers (rho=0.5399, p<0.0001), pharmacists (rho=0.3373, p=0.0009) and employer/school personnel (rho=0.2230, p=0.0317).
Conclusions:
The HUGS Vb study documents the considerable burden of illness imposed by frequent bleeding episodes on persons with hemophilia B. These episodes increase patient and caregiver absenteeism from work/school, due to increased healthcare utilization and work/school coordination. As participants on prophylactic therapy have significantly fewer bleeding episodes than those on episodic therapy, prophylaxis should be encouraged among persons with hemophilia.
