Physical and Psychological Burden on People with Hemophilia: Global Real-World Data
Notice bibliographique
Résumé
Background: Although current and emerging prophylactic treatments can reduce bleeding risk for people with hemophilia (PWH), a residual physical and psychological burden associated with treatment may persist (Barry V et al. Haemophilia. 2021;27(3):375-382). Aims: This study investigated the remaining clinical needs of people with hemophilia A (HA) and B (HB) receiving prophylaxis, from a global perspective. The current analysis assessed the physical and psychological burden reported by PWH. Methods: This cross-sectional real-world survey captured patient-reported outcomes, experiences, and clinical data from PWH and parents/guardians of PWH (PPWH) in eight countries (Canada, France, Germany, India, Italy, Saudi Arabia, Spain and the US). Participants were recruited via interaction with treating healthcare professionals, social media, online panels and patient groups. Eligible participants were ≥1 year of age with HA and ≥6 months of prophylaxis or HB who were on prophylactic and/or on-demand treatment. Exclusions included mental incapacity, language barriers, clinical trial participation, or receipt of investigative/compassionate treatments. Between December 2023 and March 2024, participants completed a 30-minute online survey. Disease impact on physical and psychological health was measured using the Patient-Reported Outcomes Measurement Information System 29+2 version 2.1 (PROMIS®-29+2 Profile v2.1 [PROMIS-29]), a validated questionnaire covering eight domains (physical functioning, ability to participate in social roles and activities, cognitive function, fatigue, sleep disturbance, pain interference, anxiety, and depressive symptoms). PROMIS-29 scores were transformed into T-scores using a reference sample from the general population with a mean of 50. Lower scores for physical, social, and cognitive domains reflect higher burden, while higher scores for the other domains indicate greater burden. This analysis focuses on results for people with severe hemophilia without inhibitors (woI) by treatment class: standard half-life (SHL) and extended half-life (EHL) for factor VIII in HA or factor IX in HB, and non-factor therapy (NFT) in HA. Results: Of 495 PWH/PPWH who completed the survey, most were self-completing PWH (n=323, 65%). Mean age (standard deviation, SD) of PWH was 27.1 (17.1) years. Of the 229 people with severe HAwoI 26% (n=59) received SHL, 28% (n=64) EHL, 40% (n=91) NFT and 6% (n=15) unknown. Of the 46 people with severe HBwoI 20% (n=9) received SHL, 78% EHL (n=36), and 2% (n=1) unknown. Adult PROMIS-29 scores indicated that burden of disease for people with severe HAwoI in all domains was similar regardless of treatment class. Median scores (interquartile range) by treatment class, SHL (n=46)/EHL (n=36)/NFT (n=62), were 44 (38-57)/46 (41-57)/44 (39-57) for physical functioning, 48 (44-56)/52 (49-64)/52 (46-58) for social roles and activities, 56 (54-58)/56 (54-58)/58 (54-60) for sleep disturbance, and 59 (52-64)/56 (50-60)/56 (42-61) for pain interference. Similar results were observed for people with severe HBwoI, SHL (n=5)/EHL (n=21), at 46 (42-51)/48 (39-57) for physical functioning, 54 (50-60)/56 (46-64) for social roles and activities, 58 (57-60)/56 (54-58) for sleep disturbance, and 56 (53-57)/52 (42-61) for pain interference. In response to two independent survey questions, people with severe HAwol (SHL [n=59]/EHL [n=64]/NFT [n=91]) reported feeling anxious (39%/38%/30%) or worried (34%/34%/26%), at least sometimes, that their treatment might not adequately protect them from bleeds. Corresponding responses for 45 people with severe HBwoI (SHL [n= 9]/EHL [n=36]) were 22%/22% (anxious) and 33%/31% (worried). The mean [SD] number of self-reported bleeds from people with severe HAwoI in the 12 months before the survey or since therapy switch was 6.8 [6.1] (SHL, n=59), 6.6 [9.7] (EHL, n=64), and 2.3 [4.2] (NFT, n=91). Corresponding mean [SD] self-reported bleeds from people with severe HBwoI were 3.2 [2.8] (SHL, n=9) and 4.8 [5.8] (EHL, n=36). Conclusion: Assessing treatment burden using standard tools is an important part of comprehensive care to individually optimize care and outcomes. Irrespective of treatment class, individuals with severe hemophilia woI continue to experience physical burden from the disease, manifested as pain, sleep disturbances and psychological burden of anxiety, and worry about breakthrough bleeds.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
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 | 0,001 | 0,004 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,001 |
| 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 | 0,001 |
| 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 ».