Meta-analysis on effectiveness of hydroxyurea to treat transfusion-dependent beta-thalassemia.

Objective To determine the efficacy of hydroxyurea therapy on transfusion-dependent beta-thalassemia patients. Methods This study collected articles from databases, applied inclusion and exclusion criteria, and analyzed them for bias. The effects of hydroxyurea on transfusion requirements were categorized according to the following definitions. 'Good responders' were participants who became transfusion independent after treatment. 'Moderate responders' were participants who were still transfusion dependent, however, experienced a significant decline in their transfusion requirements. 'Poor responders' were defined as participants who did not respond to hydroxyurea. Results A total of 13 studies met all the inclusion and exclusion criteria providing a total of 1196 study participants. The weighted average of the odds ratio using the random effect model (P < 0.001) was determined to be 0.493 for good responders, 0.270 for moderate responders, and 0.229 for poor responders. Discussion Until now, there has not been any double-blinded placebo-controlled studies performed looking at the effectiveness of hydroxyurea with this regard, and this substantially limits this meta-analysis. More studies should be conducted to determine whether hydroxyurea preferentially treats particular type of mutations over others. Conclusion This study suggests that hydroxyurea provides some benefit to patients, and therefore, in certain clinical situations, it may be understandable to start a trial therapy of hydroxyurea to qualifying patients. However, a double-blinded placebo-controlled studies should be performed before its efficacy can be considered established.