RESULTS FROM VERIFY, A PHASE 3, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF RUSFERTIDE FOR THE TREATMENT OF POLYCYTHEMIA VERA
Notice bibliographique
Résumé
Polycythemia vera (PV) is characterized by red blood cell overproduction. Rusfertide is a subcutaneous, self-injected, first-in-class peptide hepcidin mimetic that decreases erythrocytosis. VERIFY (NCT05210790) is a global, ongoing phase 3 study designed to assess rusfertide vs placebo (PBO) in phlebotomy (PHL)-dependent patients with PV receiving standard of care (SOC) therapy. In VERIFY Part 1a (Weeks 0–32), pts requiring frequent PHL with or without stable cytoreductive therapy (CRT) to control hematocrit (Hct) were randomized (1:1) to receive once-weekly rusfertide or PBO. Pts were stratified by concurrent PV therapy. All patients completing Part 1a were eligible for open-label rusfertide in Part 1b (Weeks 32–52). Patients who completed Part 1b were eligible to continue receiving rusfertide. The primary efficacy endpoint was the proportion of patients achieving a clinical response (i.e., absence of PHL eligibility with no PHLs from Weeks 20–32, and Part 1a completion). Key secondary endpoints (Weeks 0–32) included mean number of PHLs, proportion of patients with Hct <45%, and the mean change from baseline at end of Part 1a (Week 32) in the (1) PROMIS Fatigue Short Form 8a (SF-8a) total T-score and (2) the MFSAF v4.0 Total Symptom Score (TSS). A total of 293 patients (male, 73.0%; median age, 57 [range, 27–86] years) were randomized to receive rusfertide (n = 147) or PBO (n = 146). In the rusfertide and PBO groups, 56.5% (n = 83) and 55.5% (n = 81) of patients, respectively, received concurrent CRT. During Weeks 20–32, significantly more patients in the rusfertide group (76.9%) achieved a clinical response vs PBO (32.9%) (p < 0.0001). The mean (standard error) number of PHLs (Weeks 0–32) was 0.5 (0.2) with rusfertide vs 1.8 (0.2) with PBO (p < 0.0001). More patients treated with rusfertide maintained Hct <45% from Weeks 0–32 vs PBO (rusfertide, 62.6%; PBO, 14.4%; p < 0.0001). For patient-reported outcomes (PROs), patients treated with rusfertide demonstrated a statistically significant improvement in the PROMIS Fatigue SF-8a total T-score and MFSAF TSS (p < 0.03). During Part 1a, the most common treatment-emergent adverse events (AEs) in the rusfertide and PBO groups, respectively, were injection site reactions (55.9% and 32.9%), anemia (15.9% and 4.1%), and fatigue (15.2% and 15.8%). Serious AEs occurred in 3.4% (rusfertide) and 4.8% (PBO) of patients; none were considered related to rusfertide. During Part 1a, new malignancies were reported in 1 (rusfertide) and 7 (PBO) patients. In patients with PV receiving SOC therapy, rusfertide resulted in a statistically significant reduction in the mean number of PHLs and improved Hct control. Rusfertide is the first investigational agent to target the hepcidin pathway to control Hct and the first agent to prospectively demonstrate a statistically significant improvement in the PROMIS Fatigue SF-8a and MFSAF PROs in patients with PV. Rusfertide had a safety and tolerability profile consistent with rusfertide in prior studies. ©2025 American Society of Clinical Oncology, Inc. Reused with permission. This abstract was accepted and previously presented at the 2025 ASCO Annual Meeting. All rights reserved.
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 | 0,000 | 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 | 0,000 | 0,000 |
| 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 ».