PF734 PATIENT PREFERENCES FOR THE TREATMENT OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: RESULTS OF A PATIENT SURVEY OF RAVULIZUMAB (ALXN1210) AND ECULIZUMAB
Notice bibliographique
Résumé
Background: Since 2007, eculizumab has transformed the management of paroxysmal nocturnal hemoglobinuria (PNH). However, it has a treatment burden associated with q2w dosing. Recently, the FDA approved ravulizumab for PNH treatment. Ravulizumab administered q8w demonstrated noninferiority to eculizumab in two phase 3 trials on all primary and key secondary efficacy measures. In view of these two treatment options, it is important to consider patient preference in determining a treatment plan for these patients (pts). Aims: To assess patient preference for ravulizumab or eculizumab treatment, in clinical trial substudy ALXN1210‐PNH‐302 s. Methods: The substudy enrolled PNH pts who participated in the extension period of the phase 3 study (ALXN1210‐PNH‐302) and provided informed consent. In the phase 3 study, adult PNH pts stable on eculizumab for at least 6 months received ravulizumab or eculizumab in accordance with protocol for 26 wks (randomized period). All pts in the extension period received ravulizumab. In the substudy, patient treatment preference was evaluated at one time point using an 11‐item PNH‐specific Patient Preference Questionnaire (PNH‐PPQ©). Prior to the administration of the PNH‐PPQ, pts must have received at least two doses of ravulizumab during the extension period. Differences in preference between ravulizumab and eculizumab were examined with an exact binomial test (Q1), frequency distributions (Q2–3), and paired t‐tests (Q4–11), with P < 0.05 considered statistically significant. Standardized effect sizes (d) were calculated for differences between evaluations of ravulizumab and eculizumab on Q4‐11. Results: Of 98 pts enrolled, 95 pts from 8 countries (European Union, North America, and Australia) completed PNH‐PPQs per protocol for analysis. The mean age of responders was 50 years (range: 22–78); 56% were male and 44% female. Mean time since diagnosis was 14 years (range: 2–48) and the mean number of days between the last randomized study treatment and completion of PNH‐PPQ was 306 (range: 196–457). Overall, 93% of pts (n = 88) preferred ravulizumab vs 7% (n = 7; P < 0.001) who had no preference (n = 6) or preferred eculizumab (n = 1) (Table). With regard to specific aspects of treatment, ravulizumab was widely preferred on frequency of infusions (98%), ability to plan activities (98%), overall quality of life (88%), convenience of receiving treatment (85%), and effectiveness of medication until the next infusion (78%). Among pts who preferred eculizumab or had no preference, side effects of treatment (n = 52; 55%) and anxiety related to infusions (n = 49; 51%) were the leading factors in determining their choice. Frequency of infusions was selected by the most respondents (n = 41; 43%) as the most important factor determining treatment preference, followed by overall quality of life (n = 22; 23%). Moderate to large effect sizes were observed for factors favoring ravulizumab over eculizumab, including the frequency of infusions disrupting everyday life (d = –1.46, P < 0.001), feeling fatigued after infusions (d = –0.56, P < 0.001), and being able to enjoy life while receiving treatment (d = 0.88, P < 0.001). Summary/Conclusion: In this clinical substudy of eculizumab‐experienced PNH pts, a vast majority preferred ravulizumab due to reduced infusion frequency (q8w vs q2w), ability to plan activities, overall quality of life, convenience of treatment, and effectiveness of medication compared to eculizumab. The study had a global outreach and imparts important patient perspective for treatment of PNH when there is more than one treatment option. image
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 | 0,002 | 0,005 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,001 |
| Bibliométrie | 0,000 | 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 | 0,001 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,002 | 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 ».