Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A
Notice bibliographique
Résumé
Background: The real-world data on outcome of hemophilia A patients with inhibitors (HAI) is sparse, especially from developing countries. In a setting of inequitable healthcare opportunities for hemophilia patients, especially those with inhibitors, low-dose practices of emicizumab are emerging. In the present article, we describe our experience of managing HAI patients on low-dose emicizumab over a period of 56 months (from December 2019 to August 2024). Methods: The present study reports the response of patients with inhibitor-positive severe hemophilia A (HAI) and a high annual bleed rate to two-dose schedules of emicizumab prophylaxis. All patients with HAI were previously managed with on-demand bypassing agents (BPAs) before being shifted to emicizumab. Seven patients were treated on standard dose of 3 mg/kg weekly for 4 weeks followed by once in 2 weeks, whereas 25 patients were started on low dose of 3 mg/kg once in 4 weeks with or without loading as per clinical decision. Bleed frequency, joint involvement, trough drug level and hemophilia joint health score (HJHS) were documented serially till in September 2023 (median of 16.4 months of follow-up). After September 2023, all patients were shifted to low dose of 3 mg/kg once in 4 weeks, following which 18 more patients were added, and this regimen has continued to date. Results: Thirty-two patients were initiated on emicizumab prophylaxis between December 2019 and December 2022. The median duration of follow-up of this cohort was 16.4 months (7.7 - 27.3 months). There was a significant reduction in bleed rate and improvement in HJHS in both arms after initiation of emicizumab. During a cumulative follow-up period of 562.8 months involving the 32 patients, only one patient experienced a bleed that required treatment. At 12 months post-initiation, the median baseline HJHS improved from 9 to 0 in children who received full dose and from 12 to 4 in those who received low dose. The mean emicizumab trough level observed in September 2023 in both groups were 29.92 ± 2.53 µg/mL and 12.6 ± 3.79 µg/mL, respectively. No significant difference was noted either in treated bleeds or HJHS score between patients who received standard or low-dose emicizumab. In view of clinical equivalence, the standard-dose patients were also shifted to low dose, and 18 more patients were subsequently added to this arm since September 2023. The last date of follow-up for this analysis was 31 Aug 2024. The cost of treatment on low-dose emicizumab in India compared to on-demand BPAs modeled on a child weighing 10 kg is analyzed. Conclusions: Emicizumab prophylaxis even in lower doses is effective in preventing bleeds and improving joint outcome in HAI with pre-existing high bleed rate and arthropathy. This opens up an avenue for providing equity in healthcare delivery for HAI in low- and middle-income countries (LMICs) such as India.
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 distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,000 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,000 |
| Bibliométrie | 0,001 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,000 | 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) | 0,000 | 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 tête enseignante, 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 ».