Checklists References

Checklist: Guidelines for Managing Transfusion Therapy for Thalassemia

  1. Cazzola, M., Borgna-Pignatti, C., Locatelli, F., Ponchio, L., Beguin, Y. & De Stefano, P. (1997) A moderate transfusion regimen may reduce iron loading in beta-thalassemia major without producing excessive expansion of erythropoiesis. Transfusion, 37, 135-140.
  2. Cazzola, M., De Stefano, P., Ponchio, L., Locatelli, F., Beguin, Y., Dessi, C., Barella, S., Cao, A. & Galanello, R. (1995) Relationship between transfusion regimen and suppression of erythropoiesis in beta-thalassaemia major. Br J Haematol, 89, 473-478.
  3. Lawson, J.P., Ablow, R.C. & Pearson, H.A. (1981) The ribs in thalassemia. I. The relationship to therapy. Radiology, 140, 663-672.
    Pasricha, S.R., Frazer, D.M., Bowden, D.K. & Anderson, G.J. (2013) Transfusion suppresses erythropoiesis and increases hepcidin in adult patients with beta-thalassemia major: a longitudinal study. Blood, 122, 124-133.
  4. Piomelli, S., Karpatkin, M.H., Arzanian, M., Zamani, M., Becker, M.H., Geneiser, N., Danoff, S.J. & Kuhns, W.J. (1974) Hypertransfusion regimen in patients with Cooley’s anemia. Ann N Y Acad Sci, 232, 186-192.
  5. Vichinsky, E., Neumayr, L., Trimble, S., Giardina, P.J., Cohen, A.R., Coates, T., Boudreaux, J., Neufeld, E.J., Kenney, K., Grant, A., Thompson, A.A. & Investigators, C.D.C.T. (2014) Transfusion complications in thalassemia patients: a report from the Centers for Disease Control and Prevention (CME). Transfusion, 54, 972-981..
  6. Viprakasit, V., Tanphaichitr, V.S., Mahasandana, C., Assteerawatt, A., Suwantol, L., Veerakul, G., Kankirawatana, S., Pung-Amritt, P. & Suvatte, V. (2001) Linear growth in homozygous beta-thalassemia and beta-thalassemia/hemoglobin E patients under different treatment regimens. J Med Assoc Thai, 84, 929-941.

Checklist: Supplementary Material: Findings from Research Studies to Assess Optimal Pre-transfusion Hemoglobin Levels

  1. Pasricha, S.R., Frazer, D.M., Bowden, D.K. & Anderson, G.J. (2013) Transfusion suppresses erythropoiesis and increases hepcidin in adult patients with beta-thalassemia major: a longitudinal study. Blood, 122, 124-133.
  2. Cazzola, M., De Stefano, P., Ponchio, L., Locatelli, F., Beguin, Y., Dessi, C., Barella, S., Cao, A. & Galanello, R. (1995) Relationship between transfusion regimen and suppression of erythropoiesis in beta-thalassaemia major. Br J Haematol, 89, 473-478.
  3. Cazzola, M., Borgna-Pignatti, C., Locatelli, F., Ponchio, L., Beguin, Y. & De Stefano, P. (1997) A moderate transfusion regimen may reduce iron loading in beta-thalassemia major without producing excessive expansion of erythropoiesis. Transfusion, 37, 135-140.
  4. Lawson, J.P., Ablow, R.C. & Pearson, H.A. (1981) The ribs in thalassemia. I. The relationship to therapy. Radiology, 140, 663-672.
  5. Piomelli, S., Karpatkin, M.H., Arzanian, M., Zamani, M., Becker, M.H., Geneiser, N., Danoff, S.J. & Kuhns, W.J. (1974) Hypertransfusion regimen in patients with Cooley’s anemia. Ann N Y Acad Sci, 232, 186-192.
  6. Viprakasit, V., Tanphaichitr, V.S., Mahasandana, C., Assteerawatt, A., Suwantol, L., Veerakul, G., Kankirawatana, S., Pung-Amritt, P. & Suvatte, V. (2001) Linear growth in homozygous beta-thalassemia and beta-thalassemia/hemoglobin E patients under different treatment regimens. J Med Assoc Thai, 84, 929-941.
  7. Propper, R.D., Button, L.N. & Nathan, D.G. (1980) New approaches to the transfusion management of thalassemia. Blood, 55, 55-60.
  8. Masera, G., Terzoli, S., Avanzini, A., Fontanelli, G., Mauri, R.A., Piacentini, G. & Ferrari, M. (1982) Evaluation of the supertransfusion regimen in homozygous beta-thalassaemia children. Br J Haematol, 52, 111-113.

Checklist: Guidelines for Monitoring of Iron Overload in Transfusion-Dependent Thalassemia

  1. Angelucci E, Brittenham GM, McLaren CE, et al. Hepatic iron concentration and total body iron stores in thalassemia major. N Engl J Med. 2000; 343:327-31.
  2. Wood JC, Enriquez C, Ghugre N, et al. MRI R2 and R2* mapping accurately estimates hepatic iron concentration in transfusion-dependent thalassemia and sickle cell disease patients. Blood. 2005;106(4):1460-5.
  3. Pierre TG, Clark PR, Chua-anusorn W, et al. Noninvasive measurement and imaging of liver iron concentration using proton magnetic resonance. Blood. 2005; 105: 855-61.
  4. Anderson LJ, Holden S, Davis B, et al. Cardiovascular T2-star (T2*) magnetic resonance for the early diagnosis of myocardial iron overload. Eur Heart J. 2001; 22(23):2171-9.
  5. Wood JC, Noetzli L. Cardiovascular MRI in thalassemia major. Ann N Y Acad Sci. 2010:173-9.
  6. Noetzli LJ, Carson SM, Nord AS, Coates TD, Wood JC. Longitudinal analysis of heart and liver iron in thalassemia major. 2008;112(7):2973-8.

Checklist: Monitoring Deferasirox Therapy

  1. Novartis Pharmaceuticals Corporation. Highlights of prescribing information: JADENU®. New Jersey, US: Novartis; 2015. [Accessed Deptember 29, 2017].
  2. Galanello R, Piga A, Cappellini MD, et al. Effect of Food, Type of Food, and Time of Food Intake on Deferasirox Bioavailability: Recommendations for an Optimal Deferasirox Administration Regimen. J. Clin. Pharmacol. 2008;48(4):428–435.
  3. Goldberg SL, Giardina PJ, Chirnomas D, et al. The Palatability and Tolerability of Deferasirox Taken With Different Beverages or Foods. Pediatr. Blood Cancer. 2013;n/a–n/a.
  4. Taher AT, Origa R, Perrotta S, et al. New film-coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower-risk MDS: Results of the randomized, phase II ECLIPSE study. Am. J. Hematol. 2017;92(5):420–428.
  5. Aydinok Y, Kattamis A, Cappellini MD, et al. Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload. Blood. 2015;blood-2014-07-586677.
  6. Taher A, Cappellini MD, Vichinsky E, et al. Efficacy and safety of deferasirox doses of >30 mg/kg per d in patients with transfusion-dependent anaemia and iron overload. Br. J. Haematol. 2009;147(5):752–759.
  7. Taher A, Elalfy MS, Al Zir K, et al. Importance of optimal dosing ≥30 mg/kg/d during deferasirox treatment: 2.7‐yr follow‐up from the ESCALATOR study in patients with β‐thalassaemia. Eur. J. Haematol. 2011;87(4):355–365.
  8. Bauters T, Mondelaers V, Robays H, Hunninck K, de Moerloose B. Gastric ulcer in a child treated with deferasirox. Pharm World Sci. 2010;32(2):112–3.
  9. Chandra J, Chaudhary H, Pemde H, Singh V, Dutta AK. Safety and efficacy of deferasirox in multitransfused Indian children with beta-thalassaemia major. Ann Trop Paediatr. 2011;31(1):47–51.
  10. Pennell DJ, Porter JB, Piga A, et al. A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in beta-thalassemia major (CORDELIA). Blood. 2014;123(10):1447–54.
  11. Pennell D, Porter JB, Cappellini MD, et al. Deferasirox for up to 3 Years Leads to Continued Improvement of Myocardial T2* in Patients with Beta-Thalassemia Major. Haematologica. 2012;
  12. FDA. Jadenu: Full Prescribing Information. 2016;
  13. Porter JB, Elalfy MS, Taher AT, et al. Efficacy and safety of deferasirox at low and high iron burdens: results from the EPIC magnetic resonance imaging substudy. Ann. Hematol. 2013;92(2):211–219.
  14. 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.
  15. Pennell DJ, Porter JB, Piga A, et al. Sustained improvements in myocardial T2* over 2 years in severely iron-overloaded patients with beta thalassemia major treated with deferasirox or deferoxamine. Am J Hematol. 2015;90(2):91–6.
  16. Merchant R, Ahmed J, Krishnan P, Jankharia B. Efficacy and safety of deferasirox for reducing total body and cardiac iron in thalassemia. Indian Pediatr. 2012;49(4):281–5.
  17. Murphy N, Elramah M, Vats H, Zhong W, Chan MR. A case report of deferasirox-induced kidney injury and Fanconi syndrome. WMJ. 2013;112(4):177–80.
  18. Papadopoulos N, Vasiliki A, Aloizos G, Tapinis P, Kikilas A. Hyperchloremic metabolic acidosis due to deferasirox in a patient with beta thalassemia major. Ann Pharmacother. 2010;44(1):219–21.
  19. Rafat C, Fakhouri F, Ribeil J-A, Delarue R, Le Quintrec M. Fanconi Syndrome Due to Deferasirox. Am. J. Kidney Dis. 2009;54(5):931–934.
  20. Wei HY, Yang CP, Cheng CH, Lo FS. Fanconi syndrome in a patient with beta-thalassemia major after using deferasirox for 27 months. Transfusion (Paris). 2011;51(5):949–54.
  21. Dell’Orto VG, Bianchetti MG, Brazzola P. Hyperchloraemic metabolic acidosis induced by the iron chelator deferasirox: a case report and review of the literature. J Clin Pharm Ther. 2013;38(6):526–7.
  22. Milat F, Wong P, Fuller PJ, et al. A case of hypophosphatemic osteomalacia secondary to deferasirox therapy. J Bone Min. Res. 2012;27(1):219–22.
  23. Rheault MN, Bechtel H, Neglia JP, Kashtan CE. Reversible Fanconi syndrome in a pediatric patient on deferasirox. Pediatr Blood Cancer. 2011;56(4):674–6.
  24. Yacobovich J, Stark P, Barzilai-Birenbaum S, et al. Acquired Proximal Renal Tubular Dysfunction in [beta]-Thalassemia Patients Treated With Deferasirox. J. Pediatr. Hematol. 2010;32(7):564–567.
  25. Viprakasit V, Ibrahim H, Ha SY, et al. Clinical efficacy and safety evaluation of tailoring iron chelation practice in thalassaemia patients from Asia-Pacific: a subanalysis of the EPIC study of deferasirox. Int J Hematol. 2011;93(3):319–28.
  26. Alavi S, Ebadi M, Ghazizadeh F, et al. Efficacy and safety of deferasirox in beta-thalassemia major patients in Iran: a prospective study from a single referral center in Iran. Pediatr Hematol Oncol. 2014;31(1):76–86.
  27. Taher AT, Cappellini MD, Aydinok Y, et al. Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study. Blood Cells Mol Dis. 2016;57:23–9.
  28. Vichinsky E, Bernaudin F, Forni GL, et al. Long-term safety and efficacy of deferasirox (Exjade®) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease. Br. J. Haematol. 2011;154(3):387–397.

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