scholarly journals Characterization and Disease Management of Persons with Hemophilia A Treated with Emicizumab: A Survey Study of Hematologists in the US

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4941-4941
Author(s):  
Wendy E. Owens ◽  
Anisha M. Patel ◽  
Kathleen Poulos ◽  
Janet Bernard ◽  
Janet Powers ◽  
...  

Introduction Emicizumab (EMI) is a subcutaneously administered, bispecific, humanized monoclonal antibody that bridges activated factor IX (FIXa) and FX to restore the function of missing FVIIIa in persons with hemophilia A (PwHA). It was approved in the US for prophylaxis in PwHA with FVIII inhibitors in November 2017 and for PwHA without FVIII inhibitors in October 2018. In May 2019, an online survey of 50 board-certified hematologists (HEMs) was conducted with the objective of understanding the characteristics of PwHA who are being treated with EMI (PwHA-EMI) and assessing any changes in disease management of these individuals by HEMs. Methods Descriptive analysis was conducted on data collected from May 8 to May 20, 2019, through a 30-minute, online survey of 50 HEMs treating adult and pediatric PwHA with or without FVIII inhibitors in the US. These HEMs were required to have ≥2 years of experience post-residency and experience treating ≥3 PwHA per month with EMI. Results Of the 50 HEMs from 22 states who were surveyed in this study, 28% (n=14) primarily treated pediatric PwHA and 76% (n=38) practiced at federally funded hemophilia treatment centers. HEMs reported that the majority of their PwHA-EMI had severe HA, were previously treated, and did not have FVIII inhibitors. HEMs reported presence of inhibitors (40%, n=20) and high treatment burden with previous treatments (16%, n=8) as the top factors that were considered when initiating treatment with EMI. HEMs reported that they are testing less frequently for FVIII activity (52%, n=26) and inhibitors (28%, n=14) in PwHA-EMI. Most HEMs reported using multiple methods for breakthrough bleed recognition in their PwHA-EMI, such as advising them to call a healthcare provider (HCP) for diagnosis support (70%, n=35) and providing educational materials (68%, n=34). Further, 34% (n=17) reported a change in their guidance on bleed treatment in PwHA-EMI. Guidance varied by inhibitor status and type of bleed; these HEMs were more likely to recommend that their PwHA-EMI with FVIII inhibitors (65%, n=11/17) and without FVIII inhibitors (53%, n=9/17) call a HCP to discuss symptoms before treating the bleed. Also, 42% (n=21) of HEMs typically advised keeping 3-4 doses of bypassing agent or FVIII at hand in case of breakthrough bleed treatment in PwHA-EMI. A majority of HEMs (84%, n=42) reported that the recreational physical activity levels of their PwHA have remained the same or increased after starting EMI; 48% (n=24) of HEMs recommended that their patients have a gradual increase in these activities after starting EMI. Among HEMs who provided specific guidance on recreational physical activities (54%; n=27), most develop customized plans for their patients based on fitness goals (82%; n=22/27), disease severity (74%; n=20/27), and age (56%; n=15/27). For disease management support, 58% (n=29) of HEMs reported no change in the support they provide, even though EMI is a relatively new treatment. HEMs mainly attributed this to fewer bleeds in PwHA-EMI. For routine and follow-up care, 72% (n=36) and 56% (n=28) of HEMs, respectively, reported that their PwHA-EMI seek about the same levels of such care as they did prior to starting EMI, but 32% (n=16) of HEMs reported that their PwHA-EMI need less non-routine care after starting treatment with EMI. Breakthrough bleeds (22%, n=11) and trauma (20%, n=10) were reported as the top reasons for any non-routine care in PwHA-EMI. As for treatment adherence, 90% (n=45) of HEMs reported that their PwHA-EMI have similar or better adherence compared with those on other HA treatments. Lastly, 46% (n=23) of HEMs reported that their PwHA-EMI regularly experience insurance issues, including ones related to coverage for EMI and coverage for care and breakthrough bleed treatments while on EMI. Conclusions Based on the findings of this survey study, most HEMs have retained similar disease management support and practices for their PwHA-EMI. Although the frequency of routine care has not changed for most PwHA-EMI, HEMs indicated a reduction in the amount of non-routine care needed by these individuals. As EMI is prescribed and used over a longer period post approval, patient characteristics, treatment methods, and disease management practices should continue to be monitored to support HEMs' treatment and care of PwHA. Disclosures Owens: PRO Unlimited: Employment; Genentech, Inc.: Consultancy. Patel:Roche/Genentech: Equity Ownership; Genentech: Employment. Poulos:MedPanel, Inc.: Employment. Powers:MedPanel: Employment. Ko:Genentech, Inc.: Employment.

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5020-5020
Author(s):  
Anisha Patel ◽  
Karina Raimundo ◽  
Corey Stepule ◽  
Jennifer Whiteley ◽  
Richard H Ko

Abstract Introduction: Emicizumab, a bispecific antibody to factors IXa/X, was approved by the Food and Drug Administration in 2017 for prophylactic treatment of persons with hemophilia A (PwHA) with inhibitors. Compared with other treatments for PwHA, emicizumab has a novel mechanism of action. It is the first hemophilia A (HA) treatment administered subcutaneously and has a much longer half-life than previous treatments; hence, disease management with emicizumab can be different. This study aimed to evaluate how physicians manage HA in patients treated with emicizumab. Methods: This qualitative analysis is the first phase of a planned mixed-methods approach study and comprised 1-hour, blinded telephone interviews with 5 hematologists in all 4 geographic regions of the US. Physicians were included if they met the following criteria: 1) MD or DO degree; 2) board-certified hematologist; 3) practice in the US; 4) ≥2 years of experience post-residency; and 5) currently treat PwHA. To evaluate experience with emicizumab, physicians were also required to have treated a minimum number of patients receiving emicizumab (5 for physicians in hemophilia treatment centers [HTCs] and 3 for physicians in non-HTCs). A total of 5 qualitative interviews were conducted in May and June 2018 to understand the physicians' perspectives on treating PwHA with emicizumab. Interview topics included general management of PwHA, inhibitor screening and testing, management of PwHA with inhibitors, and physicians' experiences treating patients with emicizumab. We report common themes that descriptively emerged from the qualitative interviews, which will inform survey development for the quantitative phase of the study. Results: Of the 5 physicians, 3 were adult hematologists and 4 physicians reported seeing patients in an HTC. All physicians reported having at least 10 years of experience in practice post-residency. Regarding management of PwHA, physicians indicated disease severity, inhibitor status and annual bleed rate as critical factors in guiding treatment decision making. Patients who develop inhibitors require more medical engagement and frequent monitoring. Physicians reported that, from their experience, emicizumab results in a reduction in bleeds, leading to fewer office visits and less monitoring in PwHA with inhibitors. In addition, physicians reported that the need for disease management, even among patients with a phenotype with more bleeding, is lower as a result of emicizumab treatment due to decreased bleed rate and a reduction in need for additional treatments. Physicians reported that emicizumab's subcutaneous, once-weekly dosing leads to improved patients' quality of life and confidence in performing activities without bleeds. Acute bleed management with emicizumab was reported to be slightly different than before, given the boxed warning around concomitant treatment with activated prothrombin complex concentrate; however, the physicians indicated that they still recommend to treat bleeds as soon as one is suspected. Physicians articulated that some questions remain, including the role of immune therapy induction (ITI), use in low-titer inhibitors, and surgical management in patients treated with emicizumab. Physicians reported that there have been no significant access challenges and none of their patients have discontinued therapy; however, long-term monitoring and safety data are needed. Conclusions: This qualitative study suggests that management of PwHA with inhibitors receiving emicizumab is evolving. The interviewed physicians reported that their patients receiving emicizumab experience fewer bleeds and have an improved quality of life. However, questions regarding the role of ITI, surgical management, and long-term safety of emicizumab remain. Future research will provide additional insights into the current management of PwHA with inhibitors treated with emicizumab among US physicians. Disclosures Patel: Genentech Inc: Employment. Raimundo:Genentech Inc: Employment, Other: Ownership interests PLC. Stepule:MedPanel, Inc.: Employment. Whiteley:Genentech, Inc.: Employment, Other: Ownership interests none PLC. Ko:Genentech Inc: Employment; F. Hoffmann-La Roche Ltd: Other: Ownership interests non-PLC.


2018 ◽  
Vol 36 (4_suppl) ◽  
pp. 400-400
Author(s):  
Daniel M. Halperin ◽  
Lynn Huynh ◽  
Jennifer L Beaumont ◽  
Beilei Cai ◽  
Rachel Bhak ◽  
...  

400 Background: Limited information is available on the longitudinal impact of CS symptoms and QoL in CS patients (pts) who received SSAs in real-world setting. This study aimed to examine change in CS symptoms and QoL in CS pts treated with SSAs using the validated FACT-G instrument. Methods: Pts with CS symptoms in the US were recruited through an advocacy group to complete a two-part, anonymous online survey. Time point 1 (T1) survey was fielded from July-October 2016 and time point 2 (T2) was administered 6 months later. Eligible pts were ≥ 18 years old with CS symptoms and treated with SSA or non-SSA. Analyses were performed to assess change in FACT-G QoL scores between T1 and T2. Severity ratings of CS symptoms in the past month were classified as mild, moderate, severe, or not applicable. Duration of SSA treatment was categorized as ≤ 2, > 2-5, and > 5 years. Results: Among 89 pts who completed T1 and T2 surveys, 98% were treated with SSA (T1 or T2; median duration of 5 years). Higher proportion of pts in T2 did not report diarrhea (16% vs. 7%, p < 0.05) or flushing (28% vs. 18%, p < 0.05) vs. T1. Among pts treated with SSAs for ≤ 2, > 2-5 and > 5 years, mean change in FACT-G total score was +3.7, 0.0, and -1.2, respectively. Pts treated with SSAs for ≤ 2 years showed a clinically relevant improvement in QoL on FACT-G by exceeding the minimal important difference (MID) of > 3.0. Additionally, in a subgroup of pts who experienced improvement in flushing (N=17) and diarrhea (N=31), a mean change of +3.0 in FACT-G total score was observed for flushing, indicating better QoL, whereas a nominal mean change was reported for diarrhea. Pts whose flushing and diarrhea symptoms worsened had a mean change of -2.2 and -1.2 in FACT-G total score, respectively, showing worsened QoL. Conclusions: This longitudinal survey study showed that improvement in flushing resulted in positive benefit in QoL, while worsening of flushing/diarrhea resulted in decline in QOL. Improvement in CS symptoms and QoL was most pronounced in the early years after SSA treatment initiation. Less impact on change in QoL may be observed in later years possibly due to disease progression and late-effects of cancer treatment.


2007 ◽  
Vol 31 (4) ◽  
pp. 499 ◽  
Author(s):  
Caitlin F Francis ◽  
Anne-Marie Feyer ◽  
Ben J Smith

The evaluation of the Sharing Health Care Initiative addressed the translation of different models of chronic disease self-management into health and community service contexts in Australia. Across seven projects, four intervention models were adopted: (1) the Stanford Chronic Disease Self Management course; (2) generic disease management planning, training and support; (3) tailored disease management planning, training and support, and; (4) telephone coaching. Targeted recruitment through support groups and patient lists was most successful for reaching high-needs clients. Projects with well developed organisational structures and health system networks demonstrated more effective implementation. Engagement of GPs in recruitment and client support was limited. Future self-management programs will require flexible delivery methods in the primary health care setting, involving practice nurses or the equivalent. After 12 months there was little evidence of potential sustainability, although structures such as consumer resource centres and client support clubs were established in some locations. Only one project was able to use Medicare chronic disease-related items to integrate self-management support into routine general practice. Participants in all projects showed improvements in self-management practices, but those receiving Model 3, flexible and tailored support, and Model 4, telephone coaching, reported the greatest benefits


2021 ◽  
Vol 9 ◽  
Author(s):  
Ankita Sinharoy ◽  
Shekhar Pal ◽  
Jishu Das ◽  
Pritish Mondal

Rationale: India and the USA, the worst affected countries by COVID-19, experienced very different pandemic courses. By 2020, COVID-19 cases had steadily declined in India, whereas the fight continued in the US. The people of India and the USA perhaps perceived threats very differently, influenced by their knowledge, available healthcare facilities, and social security. We conducted an online survey study to compare COVID-related perceptions between Indian participants (IND-P) and US-based participants (US-P).Methods: COVID-related perceptions such as stress, knowledge, and preventive behaviors were measured with specific questionnaires, and normalized scores were computed. T-tests were used to compare the perception scores, while the Kruskal-Wallis-H (KWH) tests were used to compare socioeconomic distributions between participants from two countries. Generalized linear model (GLM) adjusted for sociodemographic confounders estimated the association between the country of residence and COVID-perception.Results: The IND-P (N = 242) were younger and male-dominated compared with the US-P (N = 531) (age: KWH = 97.37, p &lt; 0.0001, gender: KWH = 140.38, p &lt; 0.0001). Positive attitudes toward preventive guidelines were associated with higher perceived risk and stress (r = 0.35, p &lt; 0.001, and r = 0.21, p &lt; 0.001, respectively) but not with the knowledge (r = −0.05, p = 0.14). Compared with the US-P, the IND-P had lower knowledge (5.19 ± 1.95 vs. 7.82 ± 1.35; t-test: p &lt; 0.0001), higher stress (7.01 ± 1.51 vs. 6.07 ± 1.61; t-test: p &lt; 0.0001), and better adherence to preventive guidelines (8.84 ± 1.30 vs. 8.34 ± 2.09; t-test: p = 0.0006). GLM demonstrated a significant association between the country and COVID-perception scores.Conclusion: The IND-P experienced higher stress and perceived threat during COVID-19 than the US-P, perhaps due to a lack of faith in the healthcare system and insecurity. Despite lower knowledge, the IND-P had better acceptance of preventive guidelines than the US-P.


PLoS ONE ◽  
2021 ◽  
Vol 16 (4) ◽  
pp. e0251060
Author(s):  
Jie Li ◽  
Leslie J. Verteramo Chiu ◽  
Miguel I. Gómez ◽  
Nelson L. Bills

During the COVID-19 lockdown in the US, many businesses were shut down temporarily. Essential businesses, most prominently grocery stores, remained open to ensure access to food and household essentials. Grocery shopping presents increased potential for COVID-19 infection because customers and store employees are in proximity to each other. This study investigated shoppers’ perceptions of COVID-19 infection risks and put them in context by comparing grocery shopping to other activities outside home, and examined whether a proactive preventive action by grocery stores influence shoppers’ perceived risk of COVID-19 infection. Our data were obtained via an anonymous online survey distributed between April 2 and 10, 2020 to grocery shoppers in New York State (the most affected by the pandemic at the time of the study) and Washington State (the first affected by the pandemic). We found significant factors associated with high levels of risk perception on grocery shoppers. We identified some effective preventive actions that grocery stores implement to alleviate anxiety and risk perception. We found that people are generally more concerned about in-store grocery shopping relative to other out-of-home activities. Findings suggest that a strict policy requiring grocery store employees to use facemasks and gloves greatly reduced shoppers’ perceived risk rating of infection of themselves by 37.5% and store employees by 51.2%. Preventive actions by customers and businesses are critical to reducing the unwitting transmission of COVID-19 as state governments prepare to reopen the economy and relax restrictions on activities outside home.


1995 ◽  
Vol 74 (05) ◽  
pp. 1255-1258 ◽  
Author(s):  
Arnaldo A Arbini ◽  
Pier Mannuccio Mannucci ◽  
Kenneth A Bauer

SummaryPatients with hemophilia A and B and factor levels less than 1 percent of normal bleed frequently with an average number of spontaneous bleeding episodes of 20–30 or more. However there are patients with equally low levels of factor VIII or factor IX who bleed once or twice per year or not at all. To examine whether the presence of a hereditary defect predisposing to hypercoagulability might play a role in amelio rating the hemorrhagic tendency in these so-called “mild severe” hemophiliacs, we determined the prevalence of prothrombotic defects in 17 patients with hemophilia A and four patients with hemophilia B selected from 295 and 76 individuals with these disorders, respectively, followed at a large Italian hemophilia center. We tested for the presence of the Factor V Leiden mutation by PCR-amplifying a fragment of the factor V gene which contains the mutation site and then digesting the product with the restriction enzyme Mnll. None of the patients with hemophilia A and only one patient with hemophilia B was heterozygous for Factor V Leiden. None of the 21 patients had hereditary deficiencies of antithrombin III, protein C, or protein S. Our results indicate that the milder bleeding diathesis that is occasionally seen among Italian hemophiliacs with factor levels that are less than 1 percent cannot be explained by the concomitant expression of a known prothrombotic defect.


1980 ◽  
Vol 44 (01) ◽  
pp. 039-042 ◽  
Author(s):  
Philip M Blatt ◽  
Doris Ménaché ◽  
Harold R Roberts

SummaryThe treatment of patients with hemophilia A and anti-Factor VIII antibodies is difficult. Between July 1977 and June 1978, a survey was carried out by an ad hoc working party of the subcommittee on Factor IX concentrates of the International Committee on Thrombosis and Hemostasis to assess the effectiveness of Prothrombin Complex Concentrates in controlling hemorrhage in these patients. The results are presented in this paper and, although subjective, support the view that these concentrates are not as effective in patients with inhibitors as Factor VIII concentrates are in patients without inhibitors.


2020 ◽  
Author(s):  
Michael Bollwerk ◽  
Bernd Schlipphak ◽  
Joscha Stecker ◽  
Jens Hellmann ◽  
Gerald Echterhoff ◽  
...  

Threat perceptions towards immigrants continue to gain importance in the context of growing international migration. To reduce associated intergroup conflicts, it is crucial to understand the personal and contextual determinants of perceived threat. In a large online survey study (N = 1,184), we investigated the effects of ideology (i.e., Right-Wing Authoritarianism and Social Dominance Orientation), subjective societal status (SSS) and their interaction effects in predicting symbolic and realistic threat perceptions towards Middle Eastern immigrants. Results showed that ideology (higher RWA and SDO) and lower SSS significantly predicted both symbolic and realistic threat, even after controlling for income, education, age, and gender. Furthermore, ideology and SSS interacted significantly in predicting realistic threat, with higher levels of SDO and RWA enhancing the effect of SSS. In the discussion, we focus on the implications of our findings with respect to understanding societal conflicts, discuss methodological limitations, and provide directions for future research.


2020 ◽  
Author(s):  
Khanh Ngoc Cong Duong ◽  
Tien Nguyen Le Bao ◽  
Phuong Thi Lan Nguyen ◽  
Thanh Vo Van ◽  
Toi Phung Lam ◽  
...  

BACKGROUND The first nationwide lockdown due to the COVID-19 pandemic was implemented in Vietnam from April 1 to 15, 2020. Nevertheless, there has been limited information on the impact of COVID-19 on the psychological health of the public. OBJECTIVE This study aimed to estimate the prevalence of psychological issues and identify the factors associated with the psychological impact of COVID-19 during the first nationwide lockdown among the general population in Vietnam. METHODS We employed a cross-sectional study design with convenience sampling. A self-administered, online survey was used to collect data and assess psychological distress, depression, anxiety, and stress of participants from April 10 to 15, 2020. The Impact of Event Scale-Revised (IES-R) and the Depression, Anxiety, and Stress Scale-21 (DASS-21) were utilized to assess psychological distress, depression, anxiety, and stress of participants during social distancing due to COVID-19. Associations across factors were explored using regression analysis. RESULTS A total of 1385 respondents completed the survey. Of this, 35.9% (n=497) experienced psychological distress, as well as depression (n=325, 23.5%), anxiety (n=195, 14.1%), and stress (n=309, 22.3%). Respondents who evaluated their physical health as average had a higher IES-R score (beta coefficient [B]=9.16, 95% CI 6.43 to 11.89), as well as higher depression (B=5.85, 95% CI 4.49 to 7.21), anxiety (B=3.64, 95% CI 2.64 to 4.63), and stress (B=5.19, 95% CI 3.83 to 6.56) scores for DASS-21 than those who rated their health as good or very good. Those who self-reported their health as bad or very bad experienced more severe depression (B=9.57, 95% CI 4.54 to 14.59), anxiety (B=7.24, 95% CI 3.55 to 10.9), and stress (B=10.60, 95% CI 5.56 to 15.65). Unemployment was more likely to be associated with depression (B=3.34, 95% CI 1.68 to 5.01) and stress (B=2.34, 95% CI 0.84 to 3.85). Regarding worries about COVID-19, more than half (n=755, 54.5%) expressed concern for their children aged &lt;18 years, which increased their IES-R score (B=7.81, 95% CI 4.98 to 10.64) and DASS-21 stress score (B=1.75, 95% CI 0.27 to 3.24). The majority of respondents (n=1335, 96.4%) were confident about their doctor’s expertise in terms of COVID-19 diagnosis and treatment, which was positively associated with less distress caused by the outbreak (B=–7.84, 95% CI –14.58 to –1.11). CONCLUSIONS The findings highlight the effect of COVID-19 on mental health during the nationwide lockdown among the general population in Vietnam. The study provides useful evidence for policy decision makers to develop and implement interventions to mitigate these impacts. CLINICALTRIAL


2020 ◽  
Author(s):  
Muhammad Jee ◽  
Alsa Khan

BACKGROUND Understanding teens' relationship with technology is a pressing topic in this digital era. There seem to be both benefit and harmful implications that originate from use of technology by teens. Approximately 95% of teenagers have access to a smartphone, and several studies show a positive correlation between screen addiction and trends of Anxiety and Depression. While, at the same time, research shows that two-thirds of teenagers believe that technology is a necessity for connecting and making new friends. OBJECTIVE The aim of this research is to understand teens’ perceptions of their own and others’ relationship with personal technology and what future relationships do teens aspire to have with their technology. This will be fundamental in helping designers, healthcare practitioners and youth counselors inform design in technology accordingly. METHODS This research paper explores teens and technology relationship via three lenses. Lens-1 is teens' perception of their current relationship with their personal technology as users. Lens-2 is teens' perception of others' (parents, siblings or friends) relationship with personal technology as bystanders. Lens-3 is teens’ aspiration of how they wish to relate to their personal technology. We conducted an online survey with 619 teens in the 13-19 years age range. RESULTS We found that teens selected 'Essential,' 'Distractive,' and 'Addictive' most commonly to describe their own and others' relationship with personal technology. Whereas teens selected 'Provides an escape' more to describe their own relationship with technology. In contrast, they selected 'It's just a tool' and 'Creates Barrier' more to describe others' relationship with technology. These trends are consistent across ages and genders. Additionally, we found that 13-15 year-olds, particularly the majority of females in this age range, wish to see their future technology as their 'best friend.' However, 16-17 year-olds’ top choice was 'I don't believe in personal connection with mobile technology,’ and 18-19 year-olds’ top choice was 'My personal assistant.’ CONCLUSIONS Our three lenses approach is holistic as it allowed comparison of how teens perceive their relationship with personal technology as users vs. bystanders and from current vs. aspirational perspectives. This research not just confirms what is already known, but uncovers critical new associations more exclusive to teens' own relationship with technology. This paper presents related design implications to inform personal tech design thinking, including our guideline to shift 'user experience design' process to 'user relationship design.'


Sign in / Sign up

Export Citation Format

Share Document