Hydroxyurea-Induced Changes in Hematology, Iron and Renal Profiles in β-Thalassemia Patients: Impact on Hematological Parameters, Iron Status, and Renal Function

Najaf Abbas  Ghafoor; Naseem Aslam  Channa; Rubina  Ghani; Khalida  Bano; Syeda Zainab  Abbas; Zoya

Authors

Najaf Abbas Ghafoor University of Sindh, Jamshoro, Pakistan
Naseem Aslam Channa University of Sindh, Jamshoro, Pakistan https://orcid.org/0000-0003-0629-4209
Rubina Ghani Sohail University, Karachi, Pakistan
Khalida Bano Jalawan Medical College, Khuzdar, Pakistan
Syeda Zainab Abbas Jinnah University for Women, Karachi, Pakistan
Zoya Institute of Mother and Child Healthcare Hospital, Shaheed Benazirababd, Pakistan

Keywords:

Blood cell count, Erythrocyte transfusion, Gender differences, Hydroxyurea, β-thalassemia

Abstract

Background: β-thalassemia is a hereditary blood disorder characterized by reduced hemoglobin production, leading to chronic anemia and various complications. Recent evidence suggests that gender-related differences may influence disease progression and treatment outcomes in β-thalassemia patients. Hydroxyurea has emerged as a promising disease-modifying therapy, but limited research exists on gender-specific treatment responses. This study aimed to evaluate gender-specific hematological effects and safety profiles of Hydroxyurea treatment in pediatric β-thalassemia patients.

Methods: A prospective interventional study was conducted involving 150 β-thalassemia patients aged 0-15 years at Saylani Blood Bank and Transfusion Centre, Karachi. Patients were divided into two groups: males (n=78) and females (n=72). All participants were given Hydroxyurea at 5 mg/kg/day orally till six days per week. Complete blood count parameters, iron profile (ferritin), and kidney function markers (serum urea and creatinine) had been checked pre and post treatment. Statistical analysis was also applied using Student's t-test with significance set at p<0.05.

Results: Hydroxyurea showcased marked therapeutic effectiveness along with notable gender-specific differences in responses. Improvement in hemoglobin levels among women (p=0.001) as well as other hematological parameters was more pronounced compared to men who only had significant improvement in hematocrit (p=0.001) and red blood cell count (p=0.001). Treatment effectively mitigated iron overload as evidenced by persistent ferritin reduction (p=0.001) in both gender groups with differing response patterns. Renal function was either stable or improved on treatment which suggests favorable safety in both groups.

Conclusion: Hydroxyurea proved to be an effective disease-modifying therapy for β-thalassemia providing significant outcomes influenced by patient gender. These findings support the need for gender-specific treatment approaches and monitoring protocols to optimize therapeutic benefits in β-thalassemia management.

DOI: https://doi.org/10.59564/amrj/03.03/009

Author Biographies

Najaf Abbas Ghafoor, University of Sindh, Jamshoro, Pakistan

PhD Scholar

Naseem Aslam Channa, University of Sindh, Jamshoro, Pakistan

Professor, Institute of Biochemistry/Director, High Tech Central Resource Laboratory

Rubina Ghani, Sohail University, Karachi, Pakistan

Professor, Head of Department

Khalida Bano, Jalawan Medical College, Khuzdar, Pakistan

Assistant Professor, Department of Biochemistry

Syeda Zainab Abbas, Jinnah University for Women, Karachi, Pakistan

Student, Department of Pharmacy

Zoya, Institute of Mother and Child Healthcare Hospital, Shaheed Benazirababd, Pakistan

Consultant Peadiatrician

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