Evaluation the Toxicity, Safety, and Efficacy of Deferasirox Locally Manufactured Product in Iron Chelation of Hemoglubinopathic Patients in Iraq
DOI:
https://doi.org/10.59992/IJSR.2025.v4n5p10Keywords:
Blood transfusion, Derferasirox, Iron overload, ThalassemiamAbstract
Background: Thalassemia is a genetic condition that requires at least one parent to be a carrier, this condition is inherited. Certain types of blood diseases are characterized by frequent transfusions of blood, including sickle cell disease, anemia, and thalassemia. Blood transfusions offer many advantages, but they might cause the body to store too much iron. Extra iron in the body can lead to serious complications. Eliminating excess iron may reduce the risk of certain disorders. IpJade (deferasirox) is a medication used to treat chronically high iron levels in the body caused by frequent blood transfusions.
Objective: The aim of the present study is to identify the safety and efficacy of deferasirox locally manufactured product in iron chelation of hemoglubinopathic patients in Iraq.
Patients and Methods: This study was done at the Children’s Educational Hospital / Hemoglobinopathy center in Karbala. 100 patients’ medical records were acquired randomly who were diagnosed with B-thalassemia Major between the ages of 5 to 27. The information recorded included whether the patient was male or female, the daily dose of Deferasirox prescribed for each patient, and common side effects the patients were experiencing, as well as hepatic and renal function biomarkers.
Results: The results obtained from this study showed slight changes in some biomarkers (WBC, AST, serum ferritin) while clear and strong changes in other (ALT. Alkaline phosphate, serum creatinine) before and after the treatment. The findings of this study revealed a statistically association between the serum creatinine and Deferasirox doses in addition to hepatic enzymes.
Conclusion: This study revealed that the iron chelating agent (Deferasirox) is proven to be the best for patient compliance due to its favored administration compared to other types, it is shown to be well tolerated and efficacious in patients despite some negative effect which need periodic monitoring.
References
1. Kishimoto S, Maruhashi T, Kajikawa M, Matsui S, Hashimoto H, Takaeko Y, et al. Hematocrit, hemoglobin and red blood cells are associated with vascular function and vascular structure in men. Scientific reports. 2020;10(1):11467.
2. Vallelian F, Buehler PW, Schaer DJ. Hemolysis, free hemoglobin toxicity, and scavenger protein therapeutics. Blood, The Journal of the American Society of Hematology. 2022;140(17):1837-44.
3. Ahmed MH, Ghatge MS, Safo MK. Hemoglobin: structure, function and allostery. Vertebrate and invertebrate respiratory proteins, lipoproteins and other body fluid proteins. 2020:345-82.
4. Hussain Z, Mosa AU, Atiyeh MN, Sahib AS, Mahmood HS. The Effect of Deferasirox on Liver Function Tests of Thalassemia Patients in Karbala.
5. Cazzola M. Introduction to a review series on normal and pathologic erythropoiesis. Blood, The Journal of the American Society of Hematology. 2022;139(16):2413-4.
6. Shiri R, Mahdieh N. An overview on thalassemia: genetics of beta thalassemia in Iran. Koomesh. 2024;17(1):1-17.
7. Garland JM, Rudin CE. Introduction to the hematopoietic system. Colony-Stimulating Factors: CRC Press; 2020. p. 1-34.
8. Pena RSL. Molecular characterization of alpha-thalassemia and the long-range regulation of alpha-globin gene expression 2022.
9. Ali S, Mumtaz S, Shakir HA, Khan M, Tahir HM, Mumtaz S, et al. Current status of beta‐thalassemia and its treatment strategies. Molecular genetics & genomic medicine. 2021;9(12):e1788.
10. Al-khafaji KM, AL-Mosawy WF, Naser IH, Almusawi IM. Correlation Between Serum Ferritin Level and Endocrine Disorder in Female Patients with Beta-Thalassemia Major In Kerbala, Iraq. 2022.
11. Saliba A, Taher A. Iron overload in transfusion-dependent thalassemia. Hematology. 2015;20(5):311-2.
12. Stumpf JL. Deferasirox. American Journal of Health-System Pharmacy. 2007;64(6):606-16.
13. Díaz-García JD, Gallegos-Villalobos A, Gonzalez-Espinoza L, Sanchez-Nino MD, Villarrubia J, Ortiz A. Deferasirox nephrotoxicity—the knowns and unknowns. Nature reviews nephrology. 2014;10(10):574-86.
14. Chang HH, Lu MY, Liao YM, Lin PC, Yang YL, Lin DT, et al. Improved efficacy and tolerability of oral deferasirox by twice‐daily dosing for patients with transfusion‐dependent β‐thalassemia. Pediatric blood & cancer. 2011;56(3):420-4.
15. Allegra S, De Francia S, Cusato J, Arduino A, Massano D, Longo F, et al. Deferasirox pharmacogenetic influence on pharmacokinetic, efficacy and toxicity in a cohort of pediatric patients. Pharmacogenomics. 2017;18(6):539-54.
16. Tanaka C. Clinical pharmacology of deferasirox. Clinical pharmacokinetics. 2014;53(8):679-94.
17. Yang LP, Keam SJ, Keating GM. Deferasirox: a review of its use in the management of transfusional chronic iron overload. Drugs. 2007;67:2211-30.
18. Chirnomas D, Smith AL, Braunstein J, Finkelstein Y, Pereira L, Bergmann AK, et al. Deferasirox pharmacokinetics in patients with adequate versus inadequate response. Blood, The Journal of the American Society of Hematology. 2009;114(19):4009-13.
19. Huang WF, Chou HC, Tsai YW, Hsiao FY. Safety of deferasirox: a retrospective cohort study on the risks of gastrointestinal, liver and renal events. Pharmacoepidemiology and drug safety. 2014;23(11):1176-82.
20. Althanoon ZA, Alkazzaz NA. Comparison of The Effects Of Deferasirox And Deferoxamine On Uric Acid And Renal Function In Patients with Beta Thalassemia. Systematic Reviews in Pharmacy. 2020;11(11).
21. Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, et al. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up. Blood, The Journal of the American Society of Hematology. 2011;118(4):884-93.
22. Soliman A, Yassin M, Al Yafei F, Al-Naimi L, Almarri N, Sabt A, et al. Longitudinal study on liver functions in patients with thalassemia major before and after deferasirox (DFX) therapy. mediterranean journal of hematology and Infectious diseases. 2014;6(1).
23. Zargari A, Wu S, Greenway A, Cheng K, Kaplan Z. Effects of dual chelation therapy with deferasirox and deferoxamine in patients with beta thalassaemia major. Vox Sanguinis. 2022;117(5):733-7.
24. Nolte F, Angelucci E, Breccia M, Gattermann N, Santini V, Vey N, et al. Updated recommendations on the management of gastrointestinal disturbances during iron chelation therapy with Deferasirox in transfusion dependent patients with myelodysplastic syndrome–Emphasis on optimized dosing schedules and new formulations. Leukemia research. 2015;39(10):1028-33.