Treatment characteristics and outcomes in lower-risk, non-del(5q) myelodysplastic syndromes: findings from a medical record review in the USA, Canada and Europe
Notice bibliographique
Résumé
Aim: To assess treatment patterns and outcomes in patients with non-del(5q) lower-risk myelodysplastic syndromes. Methods: Patient medical records were reviewed in the USA, Canada (CAN), UK and the EU. Results: Analysis included 119 patients in the USA/CAN (median age, 61.5 years) and 245 patients in the UK/EU (median age, 67.3 years). Most patients received erythropoiesis-stimulating agents (ESAs) as first-line (1L) therapy (USA/CAN: 89.0%; UK/EU: 90.2%). A substantial proportion of 1L erythropoiesis-stimulating agent-treated patients were transfusion dependent before 1L (USA/CAN: 37.1%; UK/EU: 51.2%); a small percentage of these patients achieved transfusion independence during 1L therapy (USA/CAN: 2.8%; UK/EU: 14.4%). Conclusion: These findings highlight an unmet need for more effective treatments among patients with non-del(5q) lower-risk myelodysplastic syndromes. Patients with lower-risk myelodysplastic syndromes (LR-MDS) often present with anemia and frequently require red blood cell transfusions (RBCTs). Transfusion dependence is associated with reduced survival and poor quality of life. Therefore, the goal of LR-MDS treatment is to manage anemia and reduce transfusion burden. In patients with non-del(5q) LR-MDS, erythropoiesis-stimulating agents (ESAs), with or without granulocyte colony-stimulating factor, are an established treatment for anemia in patients who are ESA-naive. Treatment options are limited for patients who are unresponsive to ESAs. Currently, little is known about the real-world treatment patterns and clinical outcomes of patients with non-del(5q) LR-MDS. This retrospective medical record review aimed to describe patient characteristics, treatment patterns and hematologic outcomes among patients with non-del(5q) LR-MDS in the USA, Canada (CAN), UK and EU (France, Germany, Spain). Data were abstracted for 119 patients with non-del(5q) LR-MDS in the USA/CAN and 245 patients in the UK/EU. Most patients initiated first-line (1L) therapy (USA/CAN: 109 [91.6%]; UK/EU: 225 [91.8%]) with a mean time from MDS diagnosis to initiation of 1L treatment of 10.1 months in the USA/CAN and 9.6 months in the UK/EU. The majority of patients received ESAs as 1L therapy (USA/CAN: 97 [89.0%]; UK/EU: 203 [90.2%]) and the median duration of 1L ESA treatment was 25.8 months in the USA/CAN and 31.8 months in the UK/EU. Prior to 1L treatment, 36 patients (37.1%) treated with 1L ESA-containing regimens were transfusion dependent (TD) in the USA/CAN, while 104 patients (51.2%) were TD in the UK/EU. Among these patients who were TD before 1L, only a small proportion (2.8% USA/CAN; 14.4% UK/EU) achieved transfusion independence during 1L. An RBCT reduction was achieved by 41.7% and 52.9% of 1L ESA-treated patients who were TD at treatment initiation in the USA/CAN and UK/EU, respectively. A hemoglobin improvement was achieved by 32.1% of 1L ESA-treated patients in the USA/CAN and 44.8% in the UK/EU. These findings highlight an unmet need in anemia management among patients with non-del(5q) LR-MDS and a requirement for new and more effective treatment options.
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 | 0,006 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,004 | 0,010 |
| Études des sciences et des technologies | 0,001 | 0,000 |
| Communication savante | 0,001 | 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) | 0,001 | 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 ».