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Hemoglobin
international journal for hemoglobin research
Volume 48, 2024 - Issue 1
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Research Articles

Efficacy and Safety of a Dispersible Tablet of GPO-Deferasirox Monotherapy among Children with Transfusion-Dependent Thalassemia and Iron Overload

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Pages 47-55 | Received 17 Oct 2023, Accepted 23 Jan 2024, Published online: 18 Feb 2024

References

  • Schnall SF, Benz JEJ. Abnormalities of hemoglobin. In: Mazza JJ, editor. Manual of Clinical Hematology. Boston (MA): Marshfield; 1998. p. 118–122.
  • Premawardhena A, De Silver S, Arambepola M, et al. Hemoglobin E-β-thalassemia: progress report from the International Study Group. Ann N Y Acad Sci. 2005;1054(1):33–39. doi: 10.1196/annals.1345.005.
  • Winichagoon P, Thonglairoam V, Fucharoen S, et al. Severity differences in β-thalassemia/haemoglobin E syndromes: implication of genetic factors. Br J Haematol. 1993;83(4):633–639. doi: 10.1111/j.1365-2141.1993.tb04702.x.
  • Viprakast V. Comprehensive management for thalassemia. J Hematol Transf Med. 2013;23:303–320.
  • Viprakasit V, Nuchprayoon I, Chuansumrit A, et al. Deferiprone (GPO-L-One®) monotherapy reduces iron overload in transfusion-dependent thalassemia: 1-year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO-L-ONE; A001) from Thailand. Am J Hematol. 2013;88(4):251–260. doi: 10.1002/ajh.23386.
  • Cappellini MD, Bejaoui M, Agaoglu L, et al. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5-years’ follow-up. Blood. 2011;118(4):884–893. doi: 10.1182/blood-2010-11-316646.
  • Cappellini MD, Porter J, El-Beshlawy A, et al. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemia. Haematologica. 2010;95(4):557–566. doi: 10.3324/haematol.2009.014696.
  • Capellini MD, Cohen A, Eleftheriou A, et al. Guidelines for the clinical management of thalassemia. Nicosia, Cyprus: Thalassemia International Federation; 2008. p. 33–61.
  • Tanner MA, He T, Westwood MA, et al. Multi-center validation of the transferability of the magnetic resonance T2* technique for the quantification of tissue iron. Hematologica. 2006;91(10):1388–1391.
  • Anderson LJ, Holden S, Davis B, et al. Cardiovascular T2-star (T2*) magnetic resonance for early diagnosis of myocardial iron overload. Eur Heart J. 2001;22(23):2171–2179. doi: 10.1053/euhj.2001.2822.
  • Hoffbrand AV, Al-Refaie F, Davis B, et al. Long-term trial of deferiprone in 51 transfusion-dependent iron overload patients. Blood. 1998;91(1):295–300. doi: 10.1182/blood.V91.1.295.
  • Chuansumrit A, Songdej D, Sirachainan N, et al. Safety and efficacy of a liquid formulation of deferiprone (GPO-L-ONE) monotherapy among iron-overloaded young children with transfusion-dependent thalassemia. J Hematol Transfus Med. 2021;31:47–56.
  • Taher A, Cappellini MD, Vichinsky E, et al. Efficacy and safety of deferasirox doses of >30 mg/kg per day in patients with transfusion-dependent anemia and iron overload. Br J Haematol. 2009;147(5):752–759. doi: 10.1111/j.1365-2141.2009.07908.x.
  • Pennell DJ, Porter JB, Cappellini MD, et al. Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β thalassemia major. Haematologica. 2012;97(6):842–848. doi: 10.3324/haematol.2011.049957.
  • Chirnomas D, Smith AL, Braunstein J, et al. Deferasirox pharmacokinetics in patients with adequate versus inadequate response. Blood. 2009;114(19):4009–4013. doi: 10.1182/blood-2009-05-222729.
  • Chang HH, Lu MY, Liao YM, et al. Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for patients with transfusion-dependent β thalassemia. Pediatr Blood Cancer. 2011;56(3):420–424. doi: 10.1002/pbc.22826.
  • Pongtanakul B, Viprakasit V. Twice daily deferasirox significantly improves clinical efficacy in transfusion-dependent thalassemia who were inadequate responders to once daily dose. Blood Cells Mol Dis. 2013;51(2):96–97. doi: 10.1016/j.bcmd.2013.03.004.
  • Buaboonnam J, Takpradit C, Viprakasit V, et al. Long-term effective, safety and tolerability of twice-daily dosing with deferasirox in children with transfusion-dependent thalassemia unresponsive to standard once-daily dosing. Mediterr J Hematol Infect Dis. 2021;13(1):e2021065. doi: 10.4084/MJHID.2021.065.
  • Otto-Duessel M, Aguilar M, Nick H, et al. Comparison of twice-daily vs once daily deferasirox dosing in gerbil model of iron cardiomyopathy. Exp Hematol. 2007;35(7):1069–1073. doi: 10.1016/j.exphem.2007.04.001.
  • Sevimli C, Yilmaz Y, Bayramoglu Z, et al. Pancreatic MR imaging and endocrine complications in patients with beta-thalassemia: a single-center experience. Clin Exp Med. 2022;22(1):95–101. doi: 10.1007/s10238-021-00735-7.
  • Restaino G, Meloni A, Positano V, et al. Regional and global pancreatic T2* MRI for iron overload assessment in a large cohort of healthy subjects: normal values and correlation with age and gender. Magn Reson Med. 2011;65(3):764–769. doi: 10.1002/mrm.22640.
  • Meloni A, Restaino G, Missere M, et al. Pancreatic iron overload by T2* MRI in a large cohort of well-treated thalassemia major patients: can it tell us heart iron distribution and function? Am J Hematol. 2015;9(9):E189–E190.
  • Noetzli LJ, Papudesi J, Coates TD, et al. Pancreatic iron loading predicts cardiac iron loading in thalassemia. Blood. 2009;114(19):4021–4026. doi: 10.1182/blood-2009-06-225615.
  • Chaudhary P, Pullarkat V. Deferasirox: appraisal of safety and efficacy in long-term therapy. J Blood Med. 2013;4:101–110. doi: 10.2147/JBM.S35478.
  • Yacobovich J, Stark P, Barzilai-Birenbaum S, et al. Acquired proximal renal tubular dysfunction in β-thalassemia patients treated with deferasirox. J Pediatr Hematol Oncol. 2010;32(7):564–567. doi: 10.1097/MPH.0b013e3181ec0c38.
  • Rheault MN, Bechtel H, Neglia JP, et al. Reversible Fanconi syndrome in a pediatric patient on deferasirox. Pediatr Blood Cancer. 2011;56(4):674–676. doi: 10.1002/pbc.22711.
  • Scoglio M, Cappellini MD, D’Angelo E, et al. Kidney tubular damage secondary to deferasirox: systematic literature review. Children. 2021;8(12):1104. doi: 10.3390/children8121104.
  • Rafat C, Fakhouri F, Ribeil JA, et al. Fanconi syndrome due to deferasirox. Am J Kidney Dis. 2009;54(5):931–934. doi: 10.1053/j.ajkd.2009.03.013.
  • Gottwald EM, Schuh CD, Drücker P, et al. The iron chelator deferasirox causes severe mitochondrial swelling without depolarization due to a specific effect on inner membrane permeability. Sci Rep. 2020;10(1):1577. doi: 10.1038/s41598-020-58386-9.
  • Wong P, Polkinghorne K, Kerr PG, et al. Deferasirox at therapeutic doses is associated with dose-dependent hypercalciuria. Bone. 2016;85:55–58. doi: 10.1016/j.bone.2016.01.011.

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