- Research Article
- 10.1182/blood-2024-211444
Unwavering Priorities: Global Physician Alignment on Gene Therapy for Hemophilia
- Nov 05, 2024
- Blood
- Tze Yen Loo + 4 more +4
Publications from 2021 to 2026
Showing 10 of 32 papers
Unwavering Priorities: Global Physician Alignment on Gene Therapy for Hemophilia
Comparison of drug treatment intent in stage IV solid tumors in EU4+UK.
e23323 Background: The goal of cancer treatment may vary based on multiple factors. This study uses real-world data to compare treatment intent, and its evolution over time among patients with solid tumors; whilst we focus on stage IV cohorts, a comparison vs. stage I-III patients is also made, for context. Methods: Ipsos’ Global Oncology Monitor is a multi-stakeholder, physician-reported syndicated patient record database. 3,308 solid tumor-treating physicians (France = 834, Germany = 709, Italy = 578, Spain = 626, UK = 561) were screened for seniority and caseload and submitted data on 329,154 patients (France = 64,985, Germany = 77,709, Italy = 64,507, Spain = 65,427, UK = 56,526). Data were collected online from January 2020 to September 2023. Sample data were projected to the wider clinical population using market sizing extrapolations. Results: Of all solid tumor patients reported, regardless of stage, control cancer spread and/or growth (extend life) is the most common cited goal by sampled physicians when choosing drug therapy for the patient in question (average ~50% reported patients). This is followed by intent of eliminating cancer cells to undetectable levels (curative), while the intent to ease cancer symptoms (palliative) is typically less likely to be selected. Treatment intent at stage I-III showed similar trends, with curative being the most common goal (average ~70% of reported patients). When focusing on Stage IV solid tumors we observe life extension generally emerging as the main intention; a more nuanced analysis reveals similarity between Germany and Spain, where palliative intent is cited more often by sampled physicians compare to other regional counterparts. These differences are shown in Table 1. Little change seen in curative intent from 2020 to 2023 across all regions, except UK, where a downward trend is reflected from Q3 2022 onwards with a concurrent increase in extend life. Conclusions: This study indicates that treating to cure is a common aim for EU4+UK sampled physicians when giving drug therapy to stage I-III solid tumor patients, whilst extending life is the notable goal when considering stage IV patients. Additional analysis of tumor- and region-specific changes over time may help establish any potential influences on drug treatment choice due to factors such as reimbursement and treatment type availability. Further investigation using comparator cohort is warranted. [Table: see text]
Read moreDeveloping a National Longitudinal Tobacco Cohort of Youth and Young Adults: The Tobacco Epidemic Evaluation Network (TEEN+) Study
Introduction:Timely and relevant data are critical to monitoring the rapidly changing youth vaping epidemic and for understanding the prevalence, patterns of use, knowledge, and perceptions of tobacco products. While e-cigarettes have been the most used tobacco product among youth for nearly a decade, new nicotine delivery products continue to be introduced to the US market. Flavored tobacco products, including flavored e-cigarettes, menthol cigarettes, and flavored cigars, drive disparities in use by young people.Aims and Methods:To examine tobacco use among youth and young adults, the Monitoring E-Cigarette Use among Youth project established a longitudinal cohort of youth and young adults (13–24)—the Tobacco Epidemic Evaluation Network (TEEN+) study. TEEN+ focuses on e-cigarette and other tobacco products use and also includes questions about other substance use (eg, marijuana, alcohol), physical health, mental health, and social determinants of health (eg, discrimination, poverty, sexual and gender identity). Geocoding of responses allows for the evaluation of local tobacco control policies. The cohort includes an oversample of California residents to generate reliable and representative state-level estimates. This manuscript provides an overview of methods and baseline demographics from Wave 1.Results:The initial Wave 1 TEEN+ cohort included 10 255 in the national sample and 2761 in the California sample.Conclusions:TEEN+ study data complement nationally-representative cross-sectional studies and allow for rapid evaluation of local and state policies. This manuscript describes the study’s probability-based sample recruitment. Furthermore, we identify this initiative as a resource for evaluating the impact of flavored tobacco restriction policies and informing policy implementation efforts.Implications:This manuscript provides an overview of the methodology and baseline characteristics for a new longitudinal cohort of youth and young adults, the Tobacco Epidemic Evaluation Network (TEEN+) study. The TEEN+ study data can be used to evaluate the impact of flavored tobacco product restriction policies and informing policy implementation efforts.
Read moreThe impact of classic Hodgkin lymphoma on informal caregivers: results from the CONNECT cross-sectional survey
PurposeAs part of the CONNECT study, we evaluated the caregiver role in treatment decision-making when caring for patients with classic Hodgkin lymphoma (cHL) in the USA.MethodsThe CONNECT caregiver survey was administered online December 2020–March 2021 to self-identified adult caregivers of cHL patients recruited from patient referrals and online panels. The caregiver’s role in treatment decision-making, health-related quality of life (HRQoL, PROMIS-Global), and work impacts (WPAI:CG) were assessed.ResultsWe surveyed 209 caregivers (58% women; median age 47 years; 54% employed; 53% spouse/partner); 69% of patients cared for were diagnosed with cHL in the past 1–2 years, with 48% having stage III/IV cHL and 29% in remission. More spouse/partner than other caregivers were involved in caregiving at symptom onset (61% vs 27%), whereas more other than spouse/partner caregivers began after first treatment (34% vs 5%). Cure, caregivers’ top treatment goal (49%), was rated higher by spouse/partner than other caregivers (56% vs 42%). More spouse/partner than other caregivers were involved in treatment option discussions with physicians (52% vs 28%), were involved in patients’ treatment decisions (54% vs 23%), and were aligned with patients’ treatment goals (93% vs 79%). While caregivers reported HRQoL similar to that of the general population, nearly 30% of employed caregivers reported work impairment.ConclusionCure was caregivers’ top treatment goal. Spouse/partner vs other caregivers were more involved, were involved earlier, and reported greater alignment with patient treatment goals and decision-making. Caregivers reported good HRQoL; however, caregiving impacted work productivity regardless of patient relationship.
Read moreAB1060 EXPLORING DISEASE ACTIVITY AND PATIENT CHARACTERISTICS IN ANKYLOSING SPONDYLITIS (AS) IN EUROPE
BackgroundAnkylosing Spondylitis (AS) is a form of arthritis that primarily affects the spine. It causes inflammation of the spinal joints that can lead to severe, chronic pain and discomfort[1]. The Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) is the most frequently used patient-reported outcome to measure disease activity in axial spondyloarthritis. There is no validated definition for disease remission using BASDAI, although a threshold of ≤3 has been used in clinical trials for low disease activity[2].ObjectivesThe objective of this study was to examine AS patient characteristics of those with low disease activity (LDA) (BASDAI score ≤3), highlighting any nuances in treating these patients.MethodsA multi-centre online medical chart review study of patients with AS was conducted between July 2022 – September 2022 among UK, FR, DE, IT & ES rheumatologists practicing across hospital and private practices. Physicians were screened for practice duration and patient volume. Charts of patients prescribed with biologics were included in the analysis.Results262 sampled physicians collectively reported 524 AS patients. From the reported AS patients, 277 were recorded as having achieved LDA (‘LDA’) and 165 were recorded as not having achieved LDA (‘not LDA’). Among not LDA reported patients, a higher proportion were female vs the LDA cohort (32% vs 19%). Reported not LDA patients were more likely to suffer any additional co-morbidities vs LDA cohort (63% vs 44%).When analysing disease severity (physician-defined) status between these two patient groups, reported not LDA patients were more likely to be recorded as ‘mild’ at diagnosis vs LDA (14% vs 4%). In contrast, current (i.e. at time of consult) disease severity was more likely to be recorded as moderate/severe for the not LDA cohort vs the LDA cohort (76% vs 20%). In addition, reported not LDA patients recorded with higher mean disease and haematology scores.Table 1.Reported patient disease and haematology scores (mean)Reported AS patientsTender joint count (TJC)Swollen joint count (SJC)C-reactive protein (CRP)Erythrocyte Sedimentation Rate (ESR)Achieved LDA0.80.410.73.6Not achieved LDA3.42.228.915.5The most prominent biologic therapy class used to treat both patient groups are still TNFis; notable JAKi usage is more prominent in the not LDA cohort vs the LDA cohort (9% vs 3%).ConclusionFrom the sample surveyed, it appears the not achieved LDA group are milder at diagnosis but show a greater disease severity/activity on their most recent consultation compared to the LDA cohort – it could be inferred that initial mild severity resulted in a lesser need to treat earlier and, as such, disease progression ensued. There may therefore be an argument to treat early with advanced therapies, regardless of initial disease severity. Further investigation using comparator cohort is warranted.
Read moreDefining and validating the system is broken index
Abstract Objective: The aim of this article is to define and validate the “system is broken” index. The index measures anti‐establishment attitudes cross‐nationally.Methods: Using survey data from 25 countries, we test the reliability of the system is broken index and then demonstrate its convergent and divergent validity.Results: The results reveal that the system is broken index is a reliable and valid measure capturing perceptions that the political system, in any given country, is broken and is not working for the average person. The index predicts related attitudes (nativism and populism) and provides stable estimates of anti‐establishment attitudes over time.Conclusions: The system is broken index helps to predict the conditions that give rise to anti‐establishment candidates and populist movements. Tracking the measure across three decades provides unique insights into the challenges confronting contemporary politics, including the rise of populism movements.
Read more33925 Global survey shows strong impact of pandemic on skincare routines and skin health
COVID-19 Vaccine Acceptance Among US Parents: A Nationally Representative Survey
BackgroundLittle was known about US parental attitudes, beliefs, and intentions surrounding coronavirus disease 2019 (COVID-19) vaccines for children before their introduction.MethodsAn online cross-sectional nationally representative survey of US parents/guardians of children < 18 years old via Ipsos KnowledgePanel, fielded from October 26, 2021 to November 30, 2021.ResultsResponse rate was 64.2% (3230/5034). For children ages 0–4 years, 51.5% of parents were likely to have their children vaccinated, and for ages 5–11 and 12–17, 54.0% and 69.7% of parents, respectively, reported they were likely to vaccinate or had already vaccinated their children. Among respondents with unvaccinated children, 25.2% (ages 0–4) and 22.0% (ages 5–11) reported they would seek COVID-19 vaccination for their children as soon as authorization occurred. Factors associated with willingness to have children receive a COVID-19 vaccine were: belief in benefits of COVID-19 vaccination (odds ratio [OR] = 6.44, 5.68, 4.57 in ages 0–4, 5–11, and 12–17 respectively), acceptance of routine childhood vaccines (OR = 6.42, 5.48, 1.76), parental COVID-19 vaccination (OR = 1.85, 3.70, 6.16), perceptions that pediatric COVID-19 is severe (OR = 1.89, 1.72, 1.35), Hispanic ethnicity (OR = 2.07, 2.29, 2.60), influenza vaccine acceptance (OR = 1.07, 0.88, 1.62), presence of children of another age group in the household (OR = 0.71, 0.71, 0.65), and attitudinal barriers to COVID-19 vaccination (OR = 0.30, 0.26, 0.49).ConclusionsBelief in the benefits of COVID-19 vaccination and acceptance of routine childhood vaccines are the strongest predictors of intention to vaccinate children. Further research is needed to track how parental attitudes change as more data about pediatric COVID-19 vaccines become available and how intentions translate into pediatric vaccine uptake.
Read morePsychosocial determinants of adherence to public health and social measures (PHSMs) in 18 African Union Member States during the early phase of the COVID-19 pandemic: results of a cross-sectional survey
ObjectiveThe objective of this study was to gain a better understanding of the psychosocial and sociodemographic factors that affected adherence to COVID-19 public health and social measures (PHSMs), and to identify the factors that most strongly related to whether citizens followed public health guidance.DesignCross-sectional study.Setting and participantsNationally representative telephone surveys were conducted from 4–17 August 2020 in 18 African Union Member States. A total of 21 600 adults (mean age=32.7 years, SD=11.4) were interviewed (1200 in each country).Outcome measuresInformation including sociodemographics, adherence to PHSMs and psychosocial variables was collected. Logistic regression models examined the association between PHSM adherence (eg, physical distancing, gathering restrictions) and sociodemographic and psychosocial characteristics (eg, risk perception, trust). Factors affecting adherence were ranked using the Shapley regression decomposition method.ResultsAdherence to PHSMs was high, with better adherence to personal than community PHSMs (65.5% vs 30.2%, p<0.05). Psychosocial measures were significantly associated with personal and community PHSMs (p<0.05). Women and older adults demonstrated better adherence to personal PHSMs (adjusted OR (aOR): women=1.43, age=1.01, p<0.05) and community PHSMs (aOR: women=1.57, age=1.01, p<0.05). Secondary education was associated with better adherence only to personal PHSMs (aOR=1.22, p<0.05). Rural residence and access to running water were associated with better adherence to community PHSMs (aOR=1.12 and 1.18, respectively, p<0.05). The factors that most affected adherence to personal PHSMs were: self-efficacy; trust in hospitals/health centres; knowledge about face masks; trust in the president; and gender. For community PHSMs they were: gender; trust in the president; access to running water; trust in hospitals/health centres; and risk perception.ConclusionsPsychosocial factors, particularly trust in authorities and institutions, played a critical role in PHSM adherence. Adherence to community PHSMs was lower than personal PHSMs since they can impose significant burdens, particularly on the socially vulnerable.
Read moreAn Analysis of Patient Experience and Adherence to Diabetes Medication among Military Health System Beneficiaries.
Few studies have investigated the relationship between patient experience and diabetes medication adherence among Military Health System (MHS) beneficiaries. We explored the link between patient experience survey ratings and adherence to diabetes medication. The hypothesis was that adherent patients would report better provider-patient experience than non-adherent patients. Data included 2,599 patient surveys and pharmacy refill records. Adherence was determined using proportion of days covered (PDC) methodology where a patient must have had medications available 80% or more of the time during the observation period. Analysis involved multivariable logistic regression. Medication adherence was 60.2%. Regarding patient experience, those who were with their provider for 5 years or more had greater odds of adherence (OR 1.86[95%CI 1.19, 2.90]) Most of the patients in this study had high morbidity and high care utilization. Patient characteristics that significantly (p is less than 0.05) differentiated adherent versus non-adherent patients were race, mental health status, multiple medication use, glycated hemoglobin (HbA1c) levels, and health utilization. Two key factors of adherence that emerged from this study are that moderate (OR 2.54[95%CI 1.35, 4.75]) and elevated (OR 2.35[95%CI 1.29, 4.30]) HbA1c and patients with 7+ health care providers (OR 1.56[95%CI 1.06,2.29]) had greater odds of adherence. Findings suggest that ability to see provider when needed and provider continuity support adherence to treatment. The practice implications of this study are health practitioners can leverage patient experience and pharmacy data to identify patterns of adherence among patients in the MHS.
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