| ORIGINAL ARTICLE |
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| Year : 2020 | Volume
: 11
| Issue : 3 | Page : 137-142 |
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Comparison of efficacy, safety, and humanistic outcomes of iron-chelation therapy in pediatrics suffering from transfusion-dependent β-thalassemia major
Narayana Goruntla1, P Stephen Azarya1, Mahitha Chalagundla1, Dasaratha Ramaiah Jinka2, Pradeepkumar Bhupalam3, Vishnuvandana Bandaru4
1 Department of Pharmacy Practice, Raghavendra Institute of Pharmaceutical Education and Research (RIPER) - Autonomous, Anantapur, Andhra Pradesh, India
2 Department of Paediatrics, RDT Hospital, Anantapur, Andhra Pradesh, India
3 Department of Pharmacology, Raghavendra Institute of Pharmaceutical Education and Research (RIPER) - Autonomous, Anantapur, Andhra Pradesh, India
4 Department of Pharmaceutical Analysis, Balaji College of Pharmacy, Anantapur, Andhra Pradesh, India
Correspondence Address:
Mr. Narayana Goruntla
Department of Pharmacy Practice, Raghavendra Institute of Pharmaceutical Education and Research (RIPER) - Autonomous, K.R. Palli Cross, Anantapur - 515 721, Andhra Pradesh
India
 Source of Support: None, Conflict of Interest: None
DOI: 10.4103/INJMS.INJMS_23_20
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Introduction: Beta-thalassemia major (β-TM) is a severe form of genetic disorder that affects the patient's quality of life. Iron-chelation therapy (ICT) is recommended in these patients to reduce the complications (iron overload) associated with repeated blood transfusion. Objective: The present study aims to compare the efficacy, safety, and Pediatric Quality of Life (PedsQL) of the single and combinational ICT regimens in β-TM. Materials and Methods: This prospective, comparative study was conducted in the outpatient department of a daycare pediatric hospital located in South India. Patients diagnosed with β-TM, ages between 2 and 12 years, under ICT regimens were recruited. A suitable data collection form and PedsQL questionnaire was used to obtain demographics, blood transfusion, serum ferritin, hematological, biochemical, and PedsQL data of the study participants. One-way ANOVA and Chi-square test were used to compare the safety, efficacy, and PedsQL. Results: A total of 97 participants who met the study criteria were enrolled in the study and divided into three groups: Group I (n = 45) Deferasirox alone, Group II (n = 28) Deferasirox + Deferiprone, and Group III (n = 24) deferasirox + deferoxamine. The mean difference of ferritin concentration in Group III (695.3 ± 1252.4) was high compared to Group I (91.31 ± 702.6) and Group II (392.5 ± 1083.5) regimens with a P = 0.0053. In Group III, a significant reduction in physical and school functioning was observed. Conclusion: Oral Deferasirox combined with subcutaneous deferoxamine shows a great significant reduction in ferritin levels, but this combination is associated with a significant rise in hepatic enzymes, and poor physical and school functioning. An oral Deferasirox combined with oral Deferiprone is a safe and effective regimen in the treatment of β-TM, without affecting any domain of the Health-Related Quality of Life.
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