Deferasirox in a Refractory Anemia After Other Treatment Options: Case Report and Literature Review

Overview

Journal Clin Case Rep

Date 2015 Jul 18

PMID 26185629

Authors

Palma Manduzio

Affiliations

Soon will be listed here.

Abstract

Deferasirox, represents an effective iron chelator drug in lower risk myelodysplastic syndromes. Reduction in oxidative stress is responsible of the hematologic improvement but further explanation may impact on its benefit. Biological and clinical studies are necessary to better define mechanisms of action, assess toxicities, and predicting factors of response.

References

Cheong J, Kim H, Lee K, Yoon S, Lee J, Park H . Deferasirox improves hematologic and hepatic function with effective reduction of serum ferritin and liver iron concentration in transfusional iron overload patients with myelodysplastic syndrome or aplastic anemia. Transfusion. 2013; 54(6):1542-51. DOI: 10.1111/trf.12507. View

Gattermann N, Finelli C, Porta M, Fenaux P, Ganser A, Guerci-Bresler A . Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study. Leuk Res. 2010; 34(9):1143-50. DOI: 10.1016/j.leukres.2010.03.009. View

Cheson B, Greenberg P, Bennett J, Lowenberg B, Wijermans P, Nimer S . Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood. 2006; 108(2):419-25. DOI: 10.1182/blood-2005-10-4149. View

Mittelman M, Lugassy G, Merkel D, Tamary H, Sarid N, Rachmilewitz E . Iron chelation therapy in patients with myelodysplastic syndromes: consensus conference guidelines. Isr Med Assoc J. 2008; 10(5):374-6. View

Santini V, Girelli D, Sanna A, Martinelli N, Duca L, Campostrini N . Hepcidin levels and their determinants in different types of myelodysplastic syndromes. PLoS One. 2011; 6(8):e23109. PMC: 3158762. DOI: 10.1371/journal.pone.0023109. View

Lyons R, Cosgriff T, Modi S, Gersh R, Hainsworth J, Cohn A . Hematologic response to three alternative dosing schedules of azacitidine in patients with myelodysplastic syndromes. J Clin Oncol. 2009; 27(11):1850-6. DOI: 10.1200/JCO.2008.17.1058. View

Santini V . Clinical use of erythropoietic stimulating agents in myelodysplastic syndromes. Oncologist. 2011; 16 Suppl 3:35-42. DOI: 10.1634/theoncologist.2011-S3-35. View

Rose C, Brechignac S, Vassilief D, Pascal L, Stamatoullas A, Guerci A . Does iron chelation therapy improve survival in regularly transfused lower risk MDS patients? A multicenter study by the GFM (Groupe Francophone des Myélodysplasies). Leuk Res. 2010; 34(7):864-70. DOI: 10.1016/j.leukres.2009.12.004. View

Hoffbrand A, Taher A, Cappellini M . How I treat transfusional iron overload. Blood. 2012; 120(18):3657-69. DOI: 10.1182/blood-2012-05-370098. View

10.

Messa E, Carturan S, Maffe C, Pautasso M, Bracco E, Roetto A . Deferasirox is a powerful NF-kappaB inhibitor in myelodysplastic cells and in leukemia cell lines acting independently from cell iron deprivation by chelation and reactive oxygen species scavenging. Haematologica. 2010; 95(8):1308-16. PMC: 2913079. DOI: 10.3324/haematol.2009.016824. View

11.

Bowen D, Culligan D, Jowitt S, Kelsey S, Mufti G, Oscier D . Guidelines for the diagnosis and therapy of adult myelodysplastic syndromes. Br J Haematol. 2003; 120(2):187-200. DOI: 10.1046/j.1365-2141.2003.03907.x. View

12.

Roy N, Myerson S, Schuh A, Bignell P, Patel R, Wainscoat J . Cardiac iron overload in transfusion-dependent patients with myelodysplastic syndromes. Br J Haematol. 2011; 154(4):521-4. DOI: 10.1111/j.1365-2141.2011.08749.x. View

13.

Nolte F, Hochsmann B, Giagounidis A, Lubbert M, Platzbecker U, Haase D . Results from a 1-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral Deferasirox in patients diagnosed with low and int-1 risk myelodysplastic syndrome (MDS) and transfusion-dependent iron overload. Ann Hematol. 2012; 92(2):191-8. DOI: 10.1007/s00277-012-1594-z. View

14.

Porter J, Galanello R, Saglio G, Neufeld E, Vichinsky E, Cappellini M . Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study. Eur J Haematol. 2007; 80(2):168-76. PMC: 2268958. DOI: 10.1111/j.1600-0609.2007.00985.x. View

15.

Lyons R, Marek B, Paley C, Esposito J, Garbo L, DiBella N . Comparison of 24-month outcomes in chelated and non-chelated lower-risk patients with myelodysplastic syndromes in a prospective registry. Leuk Res. 2013; 38(2):149-54. DOI: 10.1016/j.leukres.2013.11.004. View

16.

Valent P, Krieger O, Stauder R, Wimazal F, Nosslinger T, Sperr W . Iron overload in myelodysplastic syndromes (MDS) - diagnosis, management, and response criteria: a proposal of the Austrian MDS platform. Eur J Clin Invest. 2008; 38(3):143-9. PMC: 2438601. DOI: 10.1111/j.1365-2362.2007.01915.x. View

17.

Mainous 3rd A, Tanner R, Hulihan M, Amaya M, Coates T . The impact of chelation therapy on survival in transfusional iron overload: a meta-analysis of myelodysplastic syndrome. Br J Haematol. 2014; 167(5):720-3. PMC: 4991026. DOI: 10.1111/bjh.13053. View

18.

Gattermann N, Finelli C, Porta M, Fenaux P, Stadler M, Guerci-Bresler A . Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes. Haematologica. 2012; 97(9):1364-71. PMC: 3436237. DOI: 10.3324/haematol.2011.048546. View

19.

Ohyashiki J, Kobayashi C, Hamamura R, Okabe S, Tauchi T, Ohyashiki K . The oral iron chelator deferasirox represses signaling through the mTOR in myeloid leukemia cells by enhancing expression of REDD1. Cancer Sci. 2009; 100(5):970-7. PMC: 11158870. DOI: 10.1111/j.1349-7006.2009.01131.x. View

20.

Marsella M, Borgna-Pignatti C . Transfusional iron overload and iron chelation therapy in thalassemia major and sickle cell disease. Hematol Oncol Clin North Am. 2014; 28(4):703-27, vi. DOI: 10.1016/j.hoc.2014.04.004. View