Hydroxyurea for β-Thalassemia Major: A Meta-Analysis
Notice bibliographique
Résumé
Abstract Background β-thalassemia major (β-TM) is one of the most common inherited diseases worldwide, characterized by a reduced ability to produce hemoglobin resulting in life-long transfusion-dependent anemia. Chronic transfusions carry significant risks such as infection, and result in iron overload that can cause significant multisystem organ damage. Hydroxyurea, an oral chemotherapeutic drug, is anticipated to decrease the need for transfusions, either completely or partially by raising hemoglobin levels and thus decreasing the short and long term complications of chronic transfusions. Objectives To evaluate the clinical efficacy and safety of hydroxyurea in β-thalassemia major (β-TM) patients of any age. Search strategy We searched MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials (CENTRAL), ongoing trials registers, and major preceding conferences. Hand searches were also conducted using reference lists from primary studies. All searches were updated to June 5, 2014. Selection criteria Randomized controlled trials (RCTs) and observational studies (sample size ≥ 5) assessing the clinical efficacy of hydroxyurea alone for three months or longer, for the treatment of patients with β-TM were included. Data collection and analysis Two authors acted as reviewers and independently assessed study quality and extracted data from the included studies. Authors of included studies were contacted if further information was required. β-TM includes the classical β-TM as well as severe hemoglobin E/β thalassemia, both of which are characterized by lifelong transfusion needs. The effect size was estimated as a proportion (those showing response to treatment over the total number treated) and reported as overall response rate (ORR) or complete response rate (CRR). ORR was defined as ≥ 50% reduction in transfusion need and CRR was defined as complete cessation of regular transfusion. All data was analyzed using Stata, Version 13.0. Results A total of 10 observational studies involving 620 patients were included. Hydroxyurea was associated with a statistically significant decrease in transfusion need with CRR of 36% (95% CI, 23-50%) and ORR of 66% (95% CI, 52-79%). All of the studies had several limitations, such as small sample size, lack of comparison group, under-reporting of data and methods, and being observational studies. Adverse events (AEs) were transient and improved with temporary cessation of the drug and/or adjustment of the dose. No long-term AEs, including cancer or end organ damage were reported. Authors’ conclusion Hydroxyurea appears to be effective in the management of β-TM by decreasing the need for chronic blood transfusions completely or partially in a significant number of patients. It appears to be well tolerated and associated with mild and transient AEs. Patients with β-TM may benefit from a trial of hydroxyurea, though large RCTs assessing efficacy should be done to confirm the findings of this meta-analysis. Disclosures Off Label Use: Hydroxyurea for β-Thalassemia.
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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 | 0,001 |
| 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 | 0,000 | 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 ».