Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes

Haematologica

Volumn 97, Issue 9, 2012, Pages 1364-1371

  Gattermann, Norbert ^a   Finelli, Carlo ^b   Porta, Matteo Della ^c   Fenaux, Pierre ^d   Stadler, Michael ^e   Guerci Bresler, Agnes ^f   Schmid, Mathias ^g   Taylor, Kerry ^h   Vassilieff, Dominique ⁱ   Habr, Dany ^j   Marcellari, Andrea ^j   Roubert, Bernard ^j   Rose, Christian ^k  

^a HEINRICH HEINE UNIVERSITY   (Germany)

^b UNIVERSITY OF BOLOGNA   (Italy)

^c FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^d AVICENNE HOSPITAL   (France)

^e HANNOVER MEDICAL SCHOOL   (Germany)

^f CHU NANCY BRABOIS   (France)

^g TRIEMLI HOSPITAL   (Switzerland)

^h Raymond Terrace   (Australia)

ⁱ Institut Cochin   (France)

^j NOVARTIS PHARMA AG   (Switzerland)

^k HÔPITAL SAINT VINCENT DE PAUL   (France)

more..

Author keywords

Deferasirox; Hematologic response; Iron chelation therapy; Iron overload; Myelodysplastic syndromes

Indexed keywords

DEFERASIROX; HEMOGLOBIN; BENZOIC ACID DERIVATIVE; IRON CHELATING AGENT; TRIAZOLE DERIVATIVE;

ADOLESCENT; ADULT; AGED; ARTICLE; CHILD; DIARRHEA; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ERYTHROCYTE CONCENTRATE; ERYTHROCYTE TRANSFUSION; ERYTHROID CELL; FEMALE; FERRITIN BLOOD LEVEL; GASTROINTESTINAL SYMPTOM; HEMATOCRIT; HEMATOLOGICAL PARAMETERS; HUMAN; IRON BLOOD LEVEL; IRON OVERLOAD; MAJOR CLINICAL STUDY; MALE; MYELODYSPLASTIC SYNDROME; NEUTROPHIL COUNT; PHASE 3 CLINICAL TRIAL (TOPIC); RECOMMENDED DRUG DOSE; SCHOOL CHILD; THROMBOCYTE COUNT; TREATMENT RESPONSE; ADVERSE EFFECTS; BLOOD TRANSFUSION; CLINICAL TRIAL; COMPLICATION; DRUG EFFECTS; FOLLOW UP; MIDDLE AGED; MORTALITY; MULTICENTER STUDY; MYELODYSPLASTIC SYNDROMES; NEOPLASM RECURRENCE, LOCAL; NEUTROPHIL; PATHOLOGY; PHASE 3 CLINICAL TRIAL; PROGNOSIS; PROSPECTIVE STUDY; SURVIVAL RATE; THROMBOCYTE; VERY ELDERLY; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; BENZOATES; BLOOD PLATELETS; BLOOD TRANSFUSION; CHILD; FEMALE; FOLLOW-UP STUDIES; HEMOGLOBINS; HUMANS; IRON CHELATING AGENTS; IRON OVERLOAD; MALE; MIDDLE AGED; MYELODYSPLASTIC SYNDROMES; NEOPLASM RECURRENCE, LOCAL; NEUTROPHILS; PROGNOSIS; PROSPECTIVE STUDIES; SURVIVAL RATE; TRIAZOLES; YOUNG ADULT;

EID: 84865848632     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2011.048546     Document Type: Article

References (48)

1. Cazzola M, Malcovati L. Myelodysplastic syndromes-coping with ineffective hematopoiesis. N Engl J Med. 2005;352(6): 536-8.
2. Cheson BD, Bennett JM, Kantarjian H, Pinto A, Schiffer CA, Nimer SD, et al. Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood. 2000;96(12):3671-4.
3. Malcovati L, Della Porta MG, Pascutto C, Invernizzi R, Boni M, Travaglino E, et al. Prognostic factors and life expectancy in myelodysplastic syndromes classified according to WHO criteria: a basis for clinical decision making. J Clin Oncol. 2005;23(30): 7594-603.
4. Sanz G, Nomdedeu B, Such E, Bernal T, Belkaid M, Ardanaz T, et al. Independent impact of iron overload and transfusion dependency on survival and leukemic evolution in patients with myelodysplastic syndrome. Blood. 2008;112(11):abst 640.
5. Gattermann N. Iron overload in myelodysplastic syndromes. Hematol Oncol Clin North Am. 2005;19(Suppl 1):1-26.
6. National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology v.2: Myelodysplastic Syndromes. 2010. Available at: http://www.nccn.org/professionals/physician_gls/pdf/mds.pdf.
7. Bowen D, Culligan D, Jowitt S, Kelsey S, Mufti G, Oscier D, et al. Guidelines for the diagnosis and therapy of adult myelodysplastic syndromes. Br J Haematol. 2003; 120(2):187-200.
8. Gattermann N, Porter J, Lopes LF, Seymour J. Consensus statement on iron overload in myelodysplastic syndromes. Hematol Oncol Clin North Am. 2005;19(Suppl 1):18-25.
9. Santini V, Alessandrino PE, Angelucci E, Barosi G, Billio A, Di MM, et al. Clinical management of myelodysplastic syndromes: update of SIE, SIES, GITMO practice guidelines. Leuk Res. 2010;34(12):1576-88.
10. Bennett JM. Consensus statement on iron overload in myelodysplastic syndromes. Am J Hematol. 2008;83(11):858-61.
11. Arrizabalaga B, del Cañizo C, Remacha A, Sanz G, Villegas A. Guía clínica de quelación del paciente con síndrome mielodisplásico [Clinical guide to chelation therapy for patients with myelodysplastic syndrome (Spanish Guidelines)]. Haematologica. 2008; 93(Suppl 1):3-10.
12. Mittelman M, Lugassy G, Merkel D, Tamary H, Sarid N, Rachmilewitz E, et al. Iron chelation therapy in patients with myelodysplastic syndromes: consensus conference guidelines. Isr Med Assoc J. 2008;10(5):374-6.
13. Suzuki T, Tomonaga M, Miyazaki Y, Nakao S, Ohyashiki K, Matsumura I, et al. Japanese epidemiological survey with consensus statement on Japanese guidelines for treatment of iron overload in bone marrow failure syndromes. Int J Hematol. 2008;88(1):30-5.
14. Valent P, Krieger O, Stauder R, Wimazal F, Nösslinger T, Sperr WR, et al. 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.
15. Wells RA, Leber B, Buckstein R, Lipton JH, Hasegawa W, Grewal K, et al. Iron overload in myelodysplastic syndromes: a Canadian consensus guideline. Leuk Res. 2008;32(9): 1338-53.
16. Gattermann N, Finelli C, Della Porta M, Fenaux P, Ganser A, Guerci-Bresler A, et al. 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.
17. List AF, Baer MR, Steensma DP, Raza A, Esposito J. Martinez-Lopez N, et al. Deferasirox reduces serum ferritin and labile plasma iron in RBC transfusiondependent patients with myelodysplastic syndrome. J Clin Oncol. 2012;30(17):2134-9.
18. Messa E, Cilloni D, Messa F, Arruga F, Roetto A, Saglio G. Deferasirox treatment improved the hemoglobin level and decreased transfusion requirements in four patients with the myelodysplastic syndrome and primary myelofibrosis. Acta Haematol. 2008;120(2): 70-4.
19. Capalbo S, Spinosa G, Franzese MG, Palumbo G. Early deferasirox treatment in a patient with myelodysplastic syndrome results in a long-term reduction in transfusion requirements. Acta Haematol. 2009; 121(1):19-20.
20. Okabe H, Suzuki T, Omori T, Mori M, Uehara E, Hatano K, et al. Hematopoietic recovery after administration of deferasirox for transfusional iron overload in a case of myelodysplastic syndrome. Rinsho Ketsueki. 2009;50(11):1626-9.
21. Badawi MA, Vickars LM, Chase JM, Leitch HA. Red blood cell transfusion independence following the initiation of iron chelation therapy in myelodysplastic syndrome. Adv Hematol. 2010;2010:164045.
22. Oliva EN, Ronco F, Marino A, Alati C, Pratico G, Nobile F. Iron chelation therapy associated with improvement of hematopoiesis in transfusion-dependent patients. Transfusion. 2010;50(7):1568-70.
23. Molteni A, Riva M, Speziale V, Morra E, Cappellini MD. Iron-chelation therapy in MDS/IMF patients: does it really impact on transfusion requirements? Haematologica. 2010;95(suppl 2):abst 1410.
24. Nishiuchi T, Okutani Y, Fujita T, Yoshida K, Ohnishi H, Haba R. Effect of iron chelator deferasirox on chronic anemia and thrombocytopenia in a transfusion-dependent patient with myelodysplastic syndrome. Int J Hematol. 2010;91(2):333-5.
25. Breccia M, Loglisci G, Salaroli A, Cannella L, Santopietro M, Alimena G. Deferasirox treatment interruption in a transfusionrequiring myelodysplastic patient led to loss of erythroid response. Acta Haematol. 2010;124(1):46-8.
26. Guariglia R, Martorelli MC, Villani O, Pietrantuono G, Mansueto G, D'Auria F, et al. Positive effects on hematopoiesis in patients with myelodysplastic syndrome receiving deferasirox as oral iron chelation therapy: a brief review. Leuk Res. 2011;35 (5):566-7.
27. Jensen PD, Jensen IM, Ellegaard J. Desferrioxamine treatment reduces blood transfusion requirements in patients with myelodysplastic syndrome. Br J Haematol. 1992;80(1):121-4.
28. Jensen PD, Heickendorff L, Pedersen B, Bendix-Hansen K, Jensen FT, Christensen T, et al. The effect of iron chelation on haemopoiesis in MDS patients with transfusional iron overload. Br J Haematol. 1996;94(2):288-99.
29. Cappellini MD, Porter JB, El-Beshlawy A, Li C-K, Seymour JF, Elalfy M, et al. Tailoring iron chelation by iron intake and serum ferritin trends: the prospective multicenter EPIC study of deferasirox in 1744 patients with various transfusion-dependent anemias. Haematologica. 2010;95(4):557-66.
30. Cheson BD, Greenberg PL, Bennett JM, Lowenberg B, Wijermans PW, Nimer SD, et al. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood. 2006;108(2):419-25.
31. Esposito BP, Breuer W, Sirankapracha P, Pootrakul P, Hershko C, Cabantchik ZI. Labile plasma iron in iron overload: redox activity and susceptibility to chelation. Blood. 2003;102(7):2670-7.
32. Smeets ME, Vreugdenhil G, Holdrinet RS. Improvement of erythropoiesis during treatment with deferiprone in a patient with myelofibrosis and transfusional hemosiderosis. Am J Hematol. 1996; 51(3):243-4.
33. Di Tucci AA, Murru R, Alberti D, Rabault B, Deplano S, Angelucci E. Correction of anemia in a transfusion-dependent patient with primary myelofibrosis receiving iron chelation therapy with deferasirox (Exjade®, ICL670). Eur J Haematol. 2007; 78(6):540-2.
34. Park SJ, Han CW. Complete hematopoietic recovery after continuous iron chelation therapy in a patient with severe aplastic anemia with secondary hemochromatosis. J Korean Med Sci. 2008;23(2):320-3.
35. Ghaffari S. Oxidative stress in the regulation of normal and neoplastic hematopoiesis. Antioxid Redox Signal. 2008;10(11):1923-40.
36. List AF, Baer MR, Steensma D, Raza A, Esposito J, Virkus J, et al. Deferasirox (ICL670; Exjade®) reduces serum ferritin (SF) and labile plasma iron (LPI) in patients with myelodysplastic syndromes (MDS). Blood. 2007;110(11):abst 1470.
37. Ghoti H, Fibach E, Merkel D, Perez-Avraham G, Grisariu S, Rachmilewitz EA. Changes in parameters of oxidative stress and free iron biomarkers during treatment with deferasirox in iron-overloaded patients with myelodysplastic syndromes. Haematologica. 2010;95(8):1433-4.
38. Messa E, Carturan S, Maffé C, Pautasso M, Bracco E, Roetto A, et al. Deferasirox is a powerful NF-kB inhibitor in myelodysplastic cells and in leukemia cell lines acting independently from intracellular iron chelation and reactive oxygen species scavenging. Haematologica. 2010;95(8):1308-16.
39. Braun T, Carvalho G, Coquelle A, Vozenin MC, Lepelley P, Hirsch F, et al. NF-kappaB constitutes a potential therapeutic target in high-risk myelodysplastic syndrome. Blood. 2006;107(3):1156-65.
40. Aggarwal BB. Nuclear factor-kappaB: the enemy within. Cancer Cell. 2004;6(3):203-8.
41. Vreugdenhil G, Smeets M, Feelders RA, van Eijk HG. Iron chelators may enhance erythropoiesis by increasing iron delivery to haematopoietic tissue and erythropoietin response in iron-loading anaemia. Acta Haematol. 1993;89(2):57-60.
42. Lyons RM, Cosgriff TM, Modi SS, Gersh RH, Hainsworth JD, Cohn AL, et al. Hematologic response to three alternative dosing schedules of azacitidine in patients with myelodysplastic syndromes. J Clin Oncol. 2009;27(11):1850-6.
43. Sekeres MA, List AF, Cuthbertson D, Paquette R, Ganetzky R, Latham D, et al. Phase I combination trial of lenalidomide and azacitidine in patients with higher-risk myelodysplastic syndromes. J Clin Oncol. 2010;28(13):2253-8.
44. Raza A, Mehdi M, Mumtaz M, Ali F, Lascher S, Galili N. Combination of 5-azacytidine and thalidomide for the treatment of myelodysplastic syndromes and acute myeloid leukemia. Cancer. 2008;113(7): 1596-604.
45. Vidaza (azacitidine) Basic Prescribing Information. 2011. Available at: http://www.vidaza.com/pdf/PI_FINAL.pdf.
46. REVLIMID® (lenalidomide) Basic Prescribing Information. 2011. Available at: http://www.revlimid.com/pdf/REVLIMID_P I.pdf.
47. Nimer SD. Clinical management of myelodysplastic syndromes with interstitial deletion of chromosome 5q. J Clin Oncol. 2006;24(16):2576-82.
48. Malcovati L, Germing U, Kuendgen A, Della Porta MG, Pascutto C, Invernizzi R, et al. Time-dependent prognostic scoring system for predicting survival and leukemic evolution in myelodysplastic syndromes. J Clin Oncol. 2007;25(23):3503-10.