Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis
ObjectiveOur aim is to review the safety and efficacy of hydroxyurea (HU) on β-thalassemia patients.MethodsStudies that evaluated the safety and efficacy of HU on β-thalassemia patients were searched in Pub-Med, Cochrane Databases, Web of Science, China-Biology-Medicine, CNKI, Embase, VIP, and WanFa...
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Frontiers Media S.A.
2025-01-01
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| Series: | Frontiers in Medicine |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fmed.2024.1480831/full |
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| author | Tianmin Huang Huixian Jiang Ganling Tang Jingyi Li Xiaoman Huang Zhenguang Huang Hongliang Zhang |
| author_facet | Tianmin Huang Huixian Jiang Ganling Tang Jingyi Li Xiaoman Huang Zhenguang Huang Hongliang Zhang |
| author_sort | Tianmin Huang |
| collection | DOAJ |
| description | ObjectiveOur aim is to review the safety and efficacy of hydroxyurea (HU) on β-thalassemia patients.MethodsStudies that evaluated the safety and efficacy of HU on β-thalassemia patients were searched in Pub-Med, Cochrane Databases, Web of Science, China-Biology-Medicine, CNKI, Embase, VIP, and WanFang data. The proportions of response rate (RR) (50% fall in transfusion need in transfusion-dependent β-thalassemia patients, or 1 g/dL elevate in hemoglobin (Hb) levels in transfusion-independent β-thalassemia patients) and good RR (transfusion-free in transfusion-dependent β-thalassemia patients or 2 g/dL elevate in Hb levels in transfusion-independent β-thalassemia patients) were utilized to evaluate the effect size (ES). The secondary outcomes were the adverse events incidence rates of HU in β-thalassemia patients.ResultsTwo randomized controlled trials (RCTs) and 25 single-armed observational studies with typically 1,748 individuals were involved in our analysis. All 27 clinical trials were reported with fair quality. HU, in transfusion-dependent β-thalassemia patients, was related to a significant decrease in transfusion requirements [a pooled RR of 0.37 and a pooled good RR of 0.65 (95% CI, 0.53–0.76)]; in transfusion-independent β-thalassemia patients, it was correlated to an excellent raise in Hb levels [a pooled RR of 0.20 (95% CI, 0.08–0.35) and a pooled good RR of 0.53 (95% CI, 0.41–0.65)]. Neutropenia and leucopenia were the most prevalent adverse events in β-thalassemia patients treated with HU, while the incidence rates of other side effects were relatively lower.ConclusionOur findings demonstrated that β-thalassemia patients tolerated and responded well to HU. Due to the control arms absence in the involved studies, more double-masked RCTs are essential for proving the safety and efficacy of HU in β-thalassemia patients. |
| format | Article |
| id | doaj-art-1cd2261c925f400ba0b31a6e3a464cb4 |
| institution | Kabale University |
| issn | 2296-858X |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Medicine |
| spelling | doaj-art-1cd2261c925f400ba0b31a6e3a464cb42025-01-15T06:10:32ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2025-01-011110.3389/fmed.2024.14808311480831Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysisTianmin HuangHuixian JiangGanling TangJingyi LiXiaoman HuangZhenguang HuangHongliang ZhangObjectiveOur aim is to review the safety and efficacy of hydroxyurea (HU) on β-thalassemia patients.MethodsStudies that evaluated the safety and efficacy of HU on β-thalassemia patients were searched in Pub-Med, Cochrane Databases, Web of Science, China-Biology-Medicine, CNKI, Embase, VIP, and WanFang data. The proportions of response rate (RR) (50% fall in transfusion need in transfusion-dependent β-thalassemia patients, or 1 g/dL elevate in hemoglobin (Hb) levels in transfusion-independent β-thalassemia patients) and good RR (transfusion-free in transfusion-dependent β-thalassemia patients or 2 g/dL elevate in Hb levels in transfusion-independent β-thalassemia patients) were utilized to evaluate the effect size (ES). The secondary outcomes were the adverse events incidence rates of HU in β-thalassemia patients.ResultsTwo randomized controlled trials (RCTs) and 25 single-armed observational studies with typically 1,748 individuals were involved in our analysis. All 27 clinical trials were reported with fair quality. HU, in transfusion-dependent β-thalassemia patients, was related to a significant decrease in transfusion requirements [a pooled RR of 0.37 and a pooled good RR of 0.65 (95% CI, 0.53–0.76)]; in transfusion-independent β-thalassemia patients, it was correlated to an excellent raise in Hb levels [a pooled RR of 0.20 (95% CI, 0.08–0.35) and a pooled good RR of 0.53 (95% CI, 0.41–0.65)]. Neutropenia and leucopenia were the most prevalent adverse events in β-thalassemia patients treated with HU, while the incidence rates of other side effects were relatively lower.ConclusionOur findings demonstrated that β-thalassemia patients tolerated and responded well to HU. Due to the control arms absence in the involved studies, more double-masked RCTs are essential for proving the safety and efficacy of HU in β-thalassemia patients.https://www.frontiersin.org/articles/10.3389/fmed.2024.1480831/fullβ-thalassemiahydroxyureaefficacysafetymeta-analysis |
| spellingShingle | Tianmin Huang Huixian Jiang Ganling Tang Jingyi Li Xiaoman Huang Zhenguang Huang Hongliang Zhang Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis Frontiers in Medicine β-thalassemia hydroxyurea efficacy safety meta-analysis |
| title | Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis |
| title_full | Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis |
| title_fullStr | Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis |
| title_full_unstemmed | Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis |
| title_short | Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis |
| title_sort | efficacy and safety of hydroxyurea therapy on patients with β thalassemia a systematic review and meta analysis |
| topic | β-thalassemia hydroxyurea efficacy safety meta-analysis |
| url | https://www.frontiersin.org/articles/10.3389/fmed.2024.1480831/full |
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