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Deferasirox for managing transfusional iron overload in people with sickle cell disease

  1. Joerg J Meerpohl1,*,
  2. Lisa K Schell1,
  3. Gerta Rücker2,
  4. Edith Motschall3,
  5. Nigel Fleeman4,
  6. Charlotte M Niemeyer5,
  7. Dirk Bassler6

Editorial Group: Cochrane Cystic Fibrosis and Genetic Disorders Group

Published Online: 27 MAY 2014

Assessed as up-to-date: 6 MAY 2014

DOI: 10.1002/14651858.CD007477.pub3


How to Cite

Meerpohl JJ, Schell LK, Rücker G, Motschall E, Fleeman N, Niemeyer CM, Bassler D. Deferasirox for managing transfusional iron overload in people with sickle cell disease. Cochrane Database of Systematic Reviews 2014, Issue 5. Art. No.: CD007477. DOI: 10.1002/14651858.CD007477.pub3.

Author Information

  1. 1

    Medical Center - University of Freiburg, German Cochrane Centre, Freiburg, Germany

  2. 2

    Medical Center - University of Freiburg, Department of Medical Biometry and Medical Informatics, Freiburg, Germany

  3. 3

    Medical Center - University of Freiburg, Center for Medical Biometry and Medical Informatics, Freiburg, Germany

  4. 4

    University of Liverpool, Liverpool Reviews & Implementation Group, Liverpool, UK

  5. 5

    University Medical Center Freiburg, Pediatric Hematology & Oncology, Center for Pediatrics & Adolescent Medicine, Freiburg, Germany

  6. 6

    University Children's Hospital, Department of Neonatology, Tuebingen, Germany

*Joerg J Meerpohl, German Cochrane Centre, Medical Center - University of Freiburg, Berliner Allee 29, Freiburg, 79110, Germany. meerpohl@cochrane.de. joerg.meerpohl@uniklinik-freiburg.de.

Publication History

  1. Publication Status: New search for studies and content updated (no change to conclusions)
  2. Published Online: 27 MAY 2014

SEARCH

References

References to studies included in this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
  4. References to other published versions of this review
Vichinsky 2007 {published data only}
  • Cappellini MD, Vichinsky E, Ford JM, Rabault B, Porter J. Evaluation of deferasirox (Exjade (R), ICL670) therapy in patients with transfusional iron overload who achieve serum ferritin (SF) <= 1000 ng/mL in long-term studies. Blood 2007; Vol. 110, issue 11 Pt 2:16B.
  • Cappellini MD, Vichinsky E, Galanello R, Piga A, Williamson P, Porter JB. Long-term treatment with deferasirox (Exjade (R), ICL670), a once-daily oral iron chelator, is effective in patients with transfusion-dependent anemias. Blood 2007; Vol. 110, issue 11 Pt 1:816A.
  • Piga A, Vichinsky E, Forni GL, Kilinc Y, Maseruka H, Kattamis A. Long-term efficacy and safety with deferasirox (Exjade (R), ICL670), a once-daily oral iron chelator, in pediatric patients. Blood 2007; Vol. 110, issue 11 Pt 1:815A.
  • Vichinsky E. Patient reported outcomes with chelation therapy in patients with sickle cell disease (SCD) on either deferasirox (Exjade®, ICL670) or deferoxamine (DFO) [abstract]. 29th Annual Meeting of The National Sickle Cell Disease Program; 2006 Apr 8-12; Memphis, USA 2006:Abstract no: 174.
  • Vichinsky E. Results of a randomized, controlled phase two trials of deferasirox (Exjade®, ICL670) in sickle cell disease patients with chronic overload [abstract]. 29th Annual Meeting of The National Sickle Cell Disease Program; 2006 Apr 8-12; Memphis, USA. 2006:Abstract no: 175.
  • Vichinsky E, Bernaudin F, Forni GL, Gardner R, Hassell K, Heeney MM, et al. Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease. British Journal of Haematology 2011;154(3):387-97.
  • Vichinsky E, Coates T, Thompson AA, Bernaudin F, Rodriguez M, Rojkjaer L, et al. Deferasirox (Exjade (R)), the Once-Daily Oral Iron Chelator, Demonstrates Safety and Efficacy in Patients with Sickle Cell Disease (SCD): 3.5-Year Follow-up. Blood 2008;112(11):506.
  • Vichinsky E, Coates TD, Thompson AA, Mueller BU, Lagrone D, Heeney MM. Long-term efficacy and safety of deferasirox (Exjade (R), ICL670), a once-daily oral iron chelator, in patients with sickle cell disease (SCD). Blood 2007; Vol. 110, issue 11 Pt 1:995A.
  • Vichinsky E, Fischer R, Fung E, Onyekwere O, Porter J, Swerdlow P, et al. A randomized, controlled phase II trial in sickle cell disease patients with chronic iron overload demonstrates that the once-daily oral iron chelator deferasirox (Exjade((R)) ICL670) is well tolerated and reduces iron burden. Blood 2005; Vol. 106, issue 11 Pt 1:95A.
  • Vichinsky E, Fischer R, Pakbaz Z, Onyekwere O, Porter J, Swerdlow P, et al. Satisfaction and convenience of chelation therapy in patients with sickle cell disease (SCD): Comparison between deferasirox (Exjade((R)), ICL670) and deferoxamine (DFO). Blood 2005; Vol. 106, issue 11 Pt 1:656A-7A.
  • Vichinsky E, Onyekwere O, Porter J, Swerdlow P, Eckman J, Lane P, et al. A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. British Journal of Haematology 2007;136(3):501-8. [PUBMED: 17233848]
  • Vichinsky E, Pakbaz Z, Onyekwere O, Porter J, Swerdlow P, Coates T, et al. Patient-reported outcomes of deferasirox (Exjade, ICL670) versus deferoxamine in sickle cell disease patients with transfusional hemosiderosis. Substudy of a randomized open-label phase II trial. Acta Haematologica 2008;119(3):133-41. [PUBMED: 18408362]
Vichinsky 2011 {published and unpublished data}
  • Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients. http://clinicaltrials.gov/ct2/show/results/NCT00110617 (accessed 01 February 2014).
  • Vichinsky E, Torres M, Glass J, Minniti CP, Barrette S, Habr D, et al. A randomized phase II study evaluating the efficacy and safety of deferasirox versus deferoxamine in patients with sickle cell disease (SCD): 2-year results including pharmacokinetics (PK) and safety of deferasirox with concomitant hydroxyurea therapy [abstract]. Blood 2011;118(21):497.
  • Vichinsky E, Torres M, Minniti CP, Barrette S, Habr D, Zhang Y, et al. Efficacy and safety of deferasirox compared with deferoxamine in sickle cell disease: two-year results including pharmacokinetics and concomitant hydroxyurea. American Journal of Hematology 2013;88(12):1068-73.

References to studies excluded from this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
  4. References to other published versions of this review
Ware 2011 {published data only}
  • Aygun B, Mortier NA, Kesler K, Schultz WH, Alvarez OA, Rogers ZR, et al. Therapeutic Phlebotomy in Children with Sickle Cell Anemia, Stroke, and Iron Overload: The SWiTCH Experience. Blood 2011; Vol. 118, issue 21:481-2.
  • Kwiatkowski JL, Cohen AR, Garro J, Alvarez O, Nagasubramanian R, Sarnaik S, et al. Transfusional iron overload in children with sickle cell anemia on chronic transfusion therapy for secondary stroke prevention. American Journal of Hematology 2012; Vol. 87, issue 2:221-3.
  • Ware RE, Helms RW. Stroke with transfusions changing to hydroxyurea (SWITCH). Blood 2012;119(17):3925-32.

Additional references

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
  4. References to other published versions of this review
Adams 1998
  • Adams RJ, McKie VC, Hsu L, Files B, Vichinsky E, Pegelow C, et al. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. New England Journal of Medicine 1998;339(1):5-11.
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Cappellini 2007a
  • Cappellini MD, Bejaoui M, Agaoglu L, Porter J, Coates T, Jeng M, et al. Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia. Clinical Therapeutics 2007;29(5):909-17. [PUBMED: 17697909]
Cappellini 2007b
  • Cappellini MD, Vichinsky E, Ford JM, Rabault B, Porter J. Evaluation of deferasirox (Exjade (R), ICL670) therapy in patients with transfusional iron overload who achieve serum ferritin (SF) <= 1000 ng/mL in long-term studies. Blood 2007; Vol. 110, issue 11, Part 2:16B.
Cappellini 2007c
  • Cappellini MD, Vichinsky E, Galanello R, Piga A, Williamson P, Porter JB. Long-term treatment with deferasirox (Exjade (R), ICL670), a once-daily oral iron chelator, is effective in patients with transfusion-dependent anemias. Blood 2007; Vol. 110, issue 11, Part 1:816A.
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  • Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, et al. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica 2010;95(4):557–66.
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McLeod 2009
  • McLeod C, Fleeman N, Kirkham J, Bagust A, Boland A, Chu P, et al. Deferasirox for the treatment of iron overload associated with regular blood transfusions (transfusional haemosiderosis) in patients suffering with chronic anaemia: a systematic review and economic evaluation. Health Technology Assessment (Winchester, England) 2009;13(1):iii-iv, ix-xi, 1-121. [PUBMED: 19068191]
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  • Vichinsky E, Fischer R, Fung E, Onyekwere O, Porter J, Swerdlow P, et al. A randomized, controlled phase II trial in sickle cell disease patients with chronic iron overload demonstrates that the once-daily oral iron chelator deferasirox (Exjade((R)) ICL670) is well tolerated and reduces iron burden. Blood 2005; Vol. 106, issue 11, Part 1:95A.
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  • Vichinsky E, Fischer R, Pakbaz Z, Onyekwere O, Porter J, Swerdlow P, et al. Satisfaction and convenience of chelation therapy in patients with sickle cell disease (SCD): Comparison between deferasirox (Exjade((R)), ICL670) and deferoxamine (DFO). Blood 2005; Vol. 106, issue 11, Part 1:656A-7A.
Vichinsky 2006a
  • Vichinsky E. Results of a randomized, controlled phase two trials of deferasirox (Exjade®, ICL670) in sickle cell disease patients with chronic overload [abstract]. 29th Annual Meeting of The National Sickle Cell Disease Program; 2006 April 8-12; Memphis, USA. 2006:Abstract no: 175.
Vichinsky 2006b
  • Vichinsky E. Patient reported outcomes with chelation therapy in patients with sickle cell disease (SCD) on either deferasirox (Exjade®, ICL670) or deferoxamine (DFO) [abstract]. 29th Annual Meeting of The National Sickle Cell Disease Program; 2006 April 8-12; Memphis, USA 2006:Abstract no: 174.
Vichinsky 2007a
Vichinsky 2007b
  • Vichinsky E, Coates TD, Thompson AA, Mueller BU, Lagrone D, Heeney MM. Long-term efficacy and safety of deferasirox (Exjade (R), ICL670), a once-daily oral iron chelator, in patients with sickle cell disease (SCD). Blood 2007; Vol. 110, issue 11, Part 1:995A.
Vichinsky 2008a
  • Vichinsky E, Pakbaz Z, Onyekwere O, Porter J, Swerdlow P, Coates T, et al. Patient-reported outcomes of deferasirox (Exjade, ICL670) versus deferoxamine in sickle cell disease patients with transfusional hemosiderosis. Substudy of a randomized open-label phase II trial. Acta Haematologica 2008;119(3):133-41. [PUBMED: 18408362]
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  • Vichinsky E, Coates T, Thompson AA, Bernaudin F, Rodriguez M, Rojkjaer L, et al. Deferasirox (Exjade (R)), the Once-Daily Oral Iron Chelator, Demonstrates Safety and Efficacy in Patients with Sickle Cell Disease (SCD): 3.5-Year Follow-up. Blood 2008;112(11):506.
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References to other published versions of this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
  4. References to other published versions of this review
Meerpohl 2010