P1523: DEMOGRAPHICS, CLINICAL CHARACTERISTICS, AND REAL-WORLD TREATMENT PATTERNS AMONG PATIENTS WITH BETA-THALASSEMIA: A RETROSPECTIVE MEDICAL RECORD ABSTRACTION STUDY
Notice bibliographique
Résumé
Background: Beta-thalassemias (BTs) are genetic disorders of hemoglobin (Hb) synthesis defined by deficient or absent synthesis of the beta-globin subunit. As a result, patients with BT experience hypochromic anemia and excess alpha-globin chain, causing ineffective erythropoiesis and low-grade hemolysis; however, patient characteristics and treatment patterns are not well understood. Aims: This retrospective medical record abstraction study evaluated patients prior to introduction of luspatercept with transfusion-dependent (TD) and non-transfusion-dependent (NTD) BT for demographics, clinical characteristics, and treatment patterns. Methods: Medical records of adults with TD or NTD BT in the United Kingdom (UK), France, Germany, Spain, and Canada with ≥5 years of visit history within the practice were evaluated. The target sample size was 50 patients in all countries (except Canada: N=20 patients), with an even distribution of patients with TD and NTD BT. Information on demographics, clinical characteristics, and treatment patterns was abstracted from the medical records. Results: In total, 118 patients with TD and 96 patients with NTD BT were identified. Among patients with TD BT, mean (standard deviation [SD]) patient age was 36.1 (11.9) years, and 28.8% were female; among patients with NTD BT, mean (SD) patient age was 36.6 (9.8) years, and 38.5% were female (Table). Table shows characteristics and results per country. Among patients with TD BT, 90/118 (76.3%) were receiving transfusions at least once every 4 weeks, with 5.1% receiving transfusions weekly, 16.1% every 2 weeks, 28.8% every 3 weeks, 26.3% every 4 weeks, 3.4% every 5 weeks, 17.8% every 6 weeks, and 2.5% other or unknown. Patients with TD BT had an average (SD) of 2.4 (0.6) units of blood transfused per session (range, 1.9 in Spain to 2.7 in UK), with an average (SD) Hb level before the transfusion of 6.9 (1.3) g/dL (range, 5.9 in Canada to 7.3 in Germany). Patients with TD BT had been on the transfusion regimen for an average of 72.6 months (range, 45.2 in Canada to 104.9 in UK) during the observation period. All patients with NTD BT had previously had ≥1 transfusion at any point in time that the physician was aware of, with an average (SD) of 2.9 (0.9) transfusions per patient (range, 2.6 in Canada to 3.1 in Germany). Among patients with NTD BT with detailed transfusion data available post-index date (N=81; 84.4%), the mean (SD) number of units of blood per transfusion was 2.2 (0.6) (range, 1.9 in Spain to 2.7 in France), and the mean (SD) Hb level before transfusion was 7.4 (1.2) g/dL (range, 6.2 in Canada to 8.2 in Spain). Iron chelation therapy was received in 70.3% of TD patients (range, 44.4% in the UK and 92.6% in Germany) and in 45.8% of NTD patients (range, 37.0% in France to 80.0% in Canada). The percentages of patients with hepatitis B, C, and D were low (ie, all <2%) for both the TD and NTD cohorts, as were the percentages of patients with cerebrovascular disease (2.5% for TD and 1.0% for NTD) and congestive heart failure (5.1% for TD and 2.1% for NTD). Image:Summary/Conclusion: Real-world data on clinical practice and outcomes in patients with BT are lacking. This study found that NTD and TD patients with BT have similarly low pre-transfusion Hb levels. A high number of patients, especially TD patients, are not treated according to the current recommendations, highlighting the importance of national reference centers to improve outcomes and quality of life in these patients.
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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 | 0,001 |
| 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 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 ».