Evaluating efficacy and safety of hydroxyurea and thalidomide for beta-thalassemia: A systematic review andmeta-analysis.
Notice bibliographique
Résumé
Abstract INTRODUCTION Therapeutic interventions such as hydroxyurea and thalidomide have individually demonstrated a reduction in transfusion requirements in beta-thalassemia; however, the combined effects of these agents remain inadequately studied. This meta-analysis aims to determine the efficacy and safety of the combined use of hydroxyurea and thalidomide in transfusion-dependent beta-thalassemia patients. METHODS Using PRISMA guidelines a comprehensive search was conducted across PubMed, Scopus, Cochrane Library, and ClinicalTrials.gov from inception till May 30, 2025. Four studies evaluating the safety and efficacy of thalidomide plus hydroxyurea in beta-thalassemia patients were included. Data was analyzed using OpenMetaAnalyst software. Pooled estimates were reported as mean ± standard deviation (SD) and 95 % confidence intervals (CI), using a random-effects model. Heterogeneity was assessed using I2 statistics. Subgroup analyses were conducted based on follow-up duration (≤ 6 months vs. > 6 months). Quality assessment was performed using the Newcastle-Ottawa Scale. Some characteristics were described systematically. RESULTS A total of 372 patients (60% male; age range: 2-50 years) from 4 observational studies were included in this analysis. The median baseline hemoglobin was 7.3 g/dL (range, 6.4-9), ferritin was 2436 ng/ml (range, 1482-2974), and the median serum bilirubin was 1.865 mg/dl (range, 1.8-1.9). The treatment regimen consisted of hydroxyurea, thalidomide and aspirin. The pooled rates for overall response (OR) and complete response (CR) were 72% (95% CI 0.679-0.776, I2= 16.39%, p = 0.310) and 61.6% (95% CI 0.521-0.707, I2 = 71.57%, p = 0.014), respectively. The pooled rates for increase in hemoglobin and reduction in serum ferritin were -87% (95% CI -1.526 to -0.219, I2 = 94.12%, p < 0.0.001) and 58% (95% CI 0.229-0.931, I2 = 80.65%, p = 0.001). Subgroup analysis reported an ORR of 72.1% (95% CI: 58.4 to 85.9%, I2 = 66.36 %, p = 0.085) for ≤ 6 months and 71.0% (95% CI: 65.9 to77.0%, I2 = 0%, p = 0.074) for > 6months, respectively. CR was 57.3% (95%CI: 38.2 - 76.3%, I2 = 79.85%, p= 0.026) and 64.6% (95% CI: 49.7 - 79.6%, I2 = 82.26%, p= 0.018) in the respective subgroups. Ferritin levels showed non-significant change at < 6 months (0.560 ng/dl; 95% CI: -0.197 to 1.316 ng/dl, I2 = 88.4%, p= 0.003), but a significant reduction at > 6 months (0.639 ng/dl; 95% CI: 0.294 to 0.984, I2 = 61.29%, p= 0.108). Hemoglobin increased by 1.385 g/dl (95% CI: 0.947 to 1.823, I2 = 63.24%, p= 0.099) and 0.37 g/dl (95% CI: 0.170 to 0.570, I2 = 0%, p= 0.346), respectively. Billirubin levels demonstrated no significant change (-0.03 mg/dl 95% CI: -0.200 to 0.140, I2 = 90.62%, p= 0.001). Adverse effects were reported in 29.9% (95% CI: 5.7 to 54%, I2 = 0%, p= 0.001) of patients. CONCLUSION The combination therapy of hydroxyurea and thalidomide has shown safety and efficacy in the treatment of beta-thalassemia. The results have shown improved haemoglobin levels and reduced ferritin levels, while bilirubin levels showed no significant change. However, given lack of high quality data, further randomised controlled trials are required to validate these results and assess long-term outcomes.
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 | 0,014 | 0,029 |
| Méta-épidémiologie (sens strict) | 0,002 | 0,001 |
| Méta-épidémiologie (sens large) | 0,016 | 0,024 |
| Bibliométrie | 0,008 | 0,009 |
| Études des sciences et des technologies | 0,001 | 0,001 |
| Communication savante | 0,003 | 0,002 |
| Science ouverte | 0,002 | 0,001 |
| Intégrité de la recherche | 0,002 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,004 | 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 ».