Inhibitors Significantly Impair Quality of Life for Hemophilia a Patients
Notice bibliographique
Résumé
Background: Hemophilia A is a rare, inherited bleeding disorder that can severely affect a patient's quality of life (QoL) across all age groups. The development of inhibitors, antibodies that neutralize clotting factor therapies, may further exacerbate the disease burden in this patient population. Aims: This study aims to assess the impact of inhibitors on key disease burden factors and their association with decreased QoL in hemophilia A patients. Methods: The Ipsos Hemophilia Therapy Monitor, a multi-country quantitative online survey, was conducted from November 2023 to February 2024.The study collected 805 hemophilia A patient records (US n=149; UK n=113; France n=110; Germany n=87; Italy n=144; Spain n=154; Canada n= 48) from participating physicians, including 113 patients with inhibitors and 692 patients without inhibitors. Physicians were screened for duration of practice in their specialty and caseload, and provided data on the presence of target joints (defined as joints in which spontaneous bleeding has occurred on 3 or more occasions within the past 12 months), chronic pain levels (0-10 scale), and treated bleeds (a bleeding event where coagulation factors are administered to treat signs or symptoms of bleeding (e.g., pain, swelling, etc.) in the past 12 months). Patients were considered to have decreased QoL if they reported the presence of target joints, chronic pain levels of 5 or above, and at least one treated bleed in the past 12 months. Results: In the study, reported patients with inhibitors were significantly more likely to have target joints (30% vs. 12%, p<0.01), chronic pain levels of 5 or above (45% vs. 21%, p<0.01), and had at least one treated bleed in the past 12 months (61% vs. 43%, p<0.01) compared to those without inhibitors. Inhibitor patients were also more likely to experience all 3 impairments simultaneously (19% vs. 6%, p<0.01), indicating a higher prevalence of decreased QoL. Overall, only 26% of the inhibitor patients reported none of the 3 disease burdens, compared to 48% of non-inhibitor patients (p<0.01). Interestingly, 40% of patients who had previously developed inhibitors but are now inhibitor-free reported an absence of the three impairments. This suggests that patients who have overcome inhibitors may experience an improvement in their QoL, potentially regaining functionality and reduced disease burden compared to those who continue to struggle with persistent inhibitors. Summary/Conclusion: In this study, hemophilia patients with inhibitors, across all age groups, experienced a severe, multifaceted disease burden, that significantly undermined their quality of life. However, eradicating inhibitors may lead to improved QoL outcomes. These findings highlight the critical need for enhanced treatment strategies and comprehensive care approaches to alleviate the unique challenges faced by inhibitor hemophilia A patients and optimize their overall well-being. Further research using comparator data is warranted.
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 | 0,000 |
| Bibliométrie | 0,000 | 0,001 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,001 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,003 | 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 ».