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Review
. 2023 Apr 26;15(4):e38135.
doi: 10.7759/cureus.38135. eCollection 2023 Apr.

Efficacy of Hydroxyurea in Transfusion-Dependent Major β-Thalassemia Patients: A Meta-Analysis

Modather I Hatamleh  1 , Venkata Sai Harshabhargav Chenna  2 , Hazel Contractor  3 , Gautham Varun Krishna Mohan  4 , Gayathri Tirumandyam  5 , Nada Dammas  6 , Muhammad Waqas Khan  7 , Shamsha Hirani  8
Affiliations
Review

Efficacy of Hydroxyurea in Transfusion-Dependent Major β-Thalassemia Patients: A Meta-Analysis

Modather I Hatamleh et al. Cureus. .

Abstract

The present meta-analysis was conducted to determine the efficacy of hydroxyurea in patients with transfusion dependent major β-thalassemia. The present meta-analysis was conducted following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) and Meta-analyses of Observational Studies in Epidemiology (MOOSE) guidelines. A systematic search was carried out to evaluate the efficacy of hydroxyurea in patients with transfusion-dependent B-thalassaemia using electronic databases, including MEDLINE, Cochrane Central Register of Controlled Trials, and EMBASE. The keywords used to search for relevant studies included "hydroxyurea", "thalassemia", "transfusion-dependent", and "efficacy". Outcomes assessed in the present meta-analysis included transfusion in one year and intervals between transfusions (in days). Other outcomes assessed in the present meta-analysis were fetal hemoglobin (%), hemoglobin (%), and ferritin levels (ng/dl). Total of five studies were included in the analysis enrolling 294 patients with major B-thalassemia. The pooled analysis reported that the mean interval between transfusions was significantly higher in patients receiving hydroxyurea compared to those not receiving hydroxyurea (mean deviation {MD}: 10.07, 95% CI: 2.16, 17.99). Hemoglobin was significantly higher in patients receiving hydroxyurea compared to its counterparts (MD: 1.71, 95% CI: 0.84, 2.57). Patients receiving hydroxyurea had significantly lower ferritin levels compared to those not receiving hydroxyurea (MD: -299.65, 95% CI: -518.35, -80.96). These findings suggest that hydroxyurea may be a promising and cost-effective alternative to blood transfusions and iron chelation therapies for beta-thalassemia patients. However, the authors noted that further randomized controlled trials are needed to validate these findings and to determine the optimal dosages and treatment regimens for hydroxyurea in this patient population.

Keywords: efficacy; hydroxyurea; meta-analysis; transfusion; transfusion-dependent β-thalassemia patient.

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Conflict of interest statement

The authors have declared that no competing interests exist.

Figures

Figure 1
Figure 1. Flowchart of selection of studies
Figure 2
Figure 2. Forest plot showing effect of hydroxyurea on number of blood transfusion
Sources: References [14,17]
Figure 3
Figure 3. Forest plot showing effect of hydroxyurea on interval between blood transfusion
Sources: References [14,17]
Figure 4
Figure 4. Forest plot showing effect of hydroxyurea on HbF
Sources: References [15-16,18]
Figure 5
Figure 5. Forest plot showing effect of hydroxyurea on Hemoglobin
Sources: References [14,16]
Figure 6
Figure 6. Forest plot showing effect of hydroxyurea on ferritin
Sources: References [14-15]
See this image and copyright information in PMC

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