Aplastic anemia

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9 regimens on this page
12 variants on this page


Initial therapy

ATG (Horse) & Cyclosporine

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Regimen

Study Evidence Comparator
Marsh et al. 1999 Phase III Cyclosporine
Teramura et al. 2007 Phase III ATG (Horse), Cyclosporine, G-CSF
Scheinberg et al. 2009 Phase III ATG (Horse), Cyclosporine, Sirolimus
Tichelli et al. 2011 Phase III ATG (Horse), Cyclosporine, G-CSF
Scheinberg et al. 2011 Phase III ATG (Rabbit) & Cyclosporine
  • Antithymocyte globulin, horse ATG (Atgam) 40 mg/kg IV once per day on days 1 to 4
  • Cyclosporine A (type not specified) as follows:
    • Starting dose: 10 mg/kg in divided doses q12h (15 mg/kg for children younger than 12)
    • Dose adjusted to keep trough blood level of 200 to 400 ng/ml

Supportive medications:

References

  1. Marsh J, Schrezenmeier H, Marin P, Ilhan O, Ljungman P, McCann S, Socie G, Tichelli A, Passweg J, Hows J, Raghavachar A, Locasciulli A, Bacigalupo A. Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood. 1999 Apr 1;93(7):2191-5. link to original article PubMed
  2. Teramura M, Kimura A, Iwase S, Yonemura Y, Nakao S, Urabe A, Omine M, Mizoguchi H. Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan. Blood. 2007 Sep 15;110(6):1756-61. Epub 2007 May 25. link to original article PubMed
  3. Scheinberg P, Wu CO, Nunez O, Scheinberg P, Boss C, Sloand EM, Young NS. Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study. Haematologica. 2009 Mar;94(3):348-54. Epub 2009 Jan 30. link to original article PubMed
  4. Tichelli A, Schrezenmeier H, Socié G, Marsh J, Bacigalupo A, Dührsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Höchsmann B, Barrois A, Champion K, Passweg JR. A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2011 Apr 28;117(17):4434-41. Epub 2011 Jan 13. link to original article PubMed
  5. Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, Young NS. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011 Aug 4;365(5):430-8. link to original article PubMed

ATG (Horse), Cyclosporine, G-CSF

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Regimen

Study Evidence Comparator
Teramura et al. 2007 Phase III ATG (Horse) & Cyclosporine
Tichelli et al. 2011 Phase III ATG (Horse) & Cyclosporine

To be completed

References

  1. Teramura M, Kimura A, Iwase S, Yonemura Y, Nakao S, Urabe A, Omine M, Mizoguchi H. Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan. Blood. 2007 Sep 15;110(6):1756-61. Epub 2007 May 25. link to original article PubMed
  2. Tichelli A, Schrezenmeier H, Socié G, Marsh J, Bacigalupo A, Dührsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Höchsmann B, Barrois A, Champion K, Passweg JR. A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2011 Apr 28;117(17):4434-41. Epub 2011 Jan 13. link to original article PubMed

ATG (Horse), Cyclosporine, Sirolimus

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Regimen

Study Evidence Comparator
Scheinberg et al. 2009 Phase III ATG (Horse) & Cyclosporine

To be completed

References

  1. Scheinberg P, Wu CO, Nunez O, Scheinberg P, Boss C, Sloand EM, Young NS. Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study. Haematologica. 2009 Mar;94(3):348-54. Epub 2009 Jan 30. link to original article PubMed

ATG (Rabbit) & Cyclosporine

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Regimen

Study Evidence Comparator
Scheinberg et al. 2011 Phase III ATG (Horse) & Cyclosporine
  • Antithymocyte globulin, rabbit ATG (Thymoglobulin) 3.5 mg/kg IV once per day on days 1 to 5
  • Cyclosporine A (type not specified) as follows:
    • Starting dose: 10 mg/kg in divided doses q12h (15 mg/kg for children younger than 12)
    • Dose adjusted to keep trough blood level of 200 to 400 ng/ml

Supportive medications:

References

  1. Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, Young NS. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011 Aug 4;365(5):430-8. link to original article PubMed

Cyclosporine

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Regimen

Study Evidence Comparator
Marsh et al. 1999 Phase III ATG (Horse) & Cyclosporine

To be completed

References

  1. Marsh J, Schrezenmeier H, Marin P, Ilhan O, Ljungman P, McCann S, Socie G, Tichelli A, Passweg J, Hows J, Raghavachar A, Locasciulli A, Bacigalupo A. Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood. 1999 Apr 1;93(7):2191-5. link to original article PubMed

Relapsed/Refractory

ATG (Rabbit) & Cyclosporine

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Regimen

Study Evidence
Scheinberg et al. 2006 Phase II

To be completed

References

  1. Scheinberg P, Nunez O, Young NS. Retreatment with rabbit anti-thymocyte globulin and ciclosporin for patients with relapsed or refractory severe aplastic anaemia. Br J Haematol. 2006 Jun;133(6):622-7. link to original article PubMed

Eltrombopag (Promacta)

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Regimen

Study Evidence
Olnes et al. 2012 Phase II
  • Eltrombopag (Promacta) 50 mg PO once per day, increased by 25 mg every 2 weeks until platelet count > 20,000 per µL or maximum of 150 mg daily dose

References

  1. Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, Parikh AR, Soto S, Biancotto A, Feng X, Lozier J, Wu CO, Young NS, Dunbar CE. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9. Erratum in: N Engl J Med. 2012 Jul 19;367(3):284. link to original article contains verified protocol PubMed
    1. Update: Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M, Parikh AR, Broder K, Calvo KR, Wu CO, Young NS, Dunbar CE. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014 Mar 20;123(12):1818-25. Epub 2013 Dec 17. link to original article contains verified protocol PubMed
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