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
Notice bibliographique
Résumé
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.
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Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,002 | 0,005 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,001 | 0,002 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,001 | 0,001 |
| Science ouverte | 0,000 | 0,001 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,001 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».