Self-reported prophylaxis treatment experience among persons living with hemophilia A in the US: A cross-sectional analysis of survey results combined with medical records
Bibliographic record
Abstract
Abstract Background: In hemophilia A (HA), prophylaxis is used to prevent bleeding and joint damage. With the expansion of non-factor therapies (NFT), there is limited evidence on patient experience and satisfaction, which are crucial to identify contemporary care gaps and facilitate shared decision making (SDM) when selecting prophylaxis. This study aims to assess patient-reported satisfaction, adherence, and SDM with treatment classes among persons with hemophilia A (PwHA) in the United States using PicnicHealth research platform. Methods: This non-interventional, retrospective, cross-sectional study used PicnicHealth research platform, including longitudinal medical records from routine clinical care and data from a one-time custom survey, Hemophilia Patient Experience Survey (HPES), deployed between February – May 2025. Key eligibility criteria included moderate/severe HA and at least one prophylaxis since 2019 in the medical records. HPES included 9 questions about effectiveness, side effects, cost, ease of preparation, frequency, pain upon administration, interference with daily activities, anxiety related to administration, and overall satisfaction with the current/most recent prophylaxis, adherence and shared decision making regarding prophylactic switches. HPES response date was defined as index date. Data was summarized descriptively using means and proportions. Results: Of 334 PwHA, 305 (91%) met eligibility criteria, among whom 193 (63%) responded to the HPES (analytic cohort; 99% male). Currently or most recently prior to index, 35 (18%) of the cohort received standard half-life (SHL), 52 (27%) extended half-life (EHL), and 106 (55%) NFT. The mean (standard deviation [SD]) age of PwHA in the cohort was 33 (15), with 17% <18 years. By group: SHL 34 (14), 14% <18; EHL 36 (13), 8% <18; NFT 32 (16), 8% <18. 86% of the overall cohort had severe HA (86% SHL; 81% EHL; 89% NFT). The overall cohort had a mean (SD) annualized bleed rate (ABR) of 0.82 (1.66) during at least 180-days of continuous index prophylaxis use 1-year before the index date, with comparable values observed across treatment classes. In the overall cohort, 83% of PwHA were satisfied/very satisfied with the effectiveness of their prophylactic treatment in preventing bleeding, while 16% were dissatisfied/very dissatisfied with the anxiety related to administering prophylaxis; 15% with cost; and 10% with pain upon prophylaxis administration. Additionally, 13%, 24%, 33%, and 31% responded neutrally to these attributes, respectively. When asked about overall satisfaction with index prophylaxis, 77% of SHL, 79% of EHL, and 86% of NFT patients reported being satisfied/very satisfied. In the SHL cohort, 80% were satisfied/very satisfied with prophylactic effectiveness, while 29% were dissatisfied/very dissatisfied with anxiety related to prophylaxis administration, and 23% with both administration frequency and cost. Neutral responses were 17% for effectiveness, 23% for anxiety related to prophylaxis administration and frequency, and 43% for cost. In the EHL cohort, 87% were satisfied/very satisfied with side effects; 15% were dissatisfied/very dissatisfied with both cost and treatment interfering with work, school, travel and social activities, and 12% with anxiety related to prophylaxis administration. Neutral responses were 12% (side effects), 33% (cost), 25% (interference), and 27% (anxiety). In the NFT cohort, 85% were satisfied/very satisfied with effectiveness, 13% were dissatisfied/very dissatisfied with administration anxiety, 12% with pain, and 11% with cost. Neutral responses were 12%, 23%, 25%, and 29% for these attributes, respectively. Of all respondents, 71% had changed prophylaxis. Decision-making in choosing/changing treatment was reported as doctor-led in 16% of cases, patient-led in 29%, and shared equally in 55%. Among respondents, 54% in the SHL cohort, 73% in EHL and 69% in the NFT, reported rarely or never missing or delaying prophylaxis. Often/very often missing or delaying were reported by 17% in SHL, 2% in EHL, and 7% in NFT cohorts. Conclusions: In this study, PwHA were overall satisfied with their current prophylaxis. However, PwHA were less satisfied with the anxiety experienced during prophylaxis, cost and pain of administration. Evaluating the gaps in treatment satisfaction for PwHA may facilitate meaningful SDM when selecting prophylaxis. Acknowledgements: The authors would like to thank the PwHA on the PicnicHealth research platform.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.005 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.001 | 0.002 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.001 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".