Difference between revisions of "Aplastic anemia"

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Revision as of 05:58, 4 December 2016

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Is there a regimen missing from this list? Would you like to share a different dosage/schedule or an additional reference for a regimen? Have you noticed an error? Do you have an idea that will help the site grow to better meet your needs and the needs of many others? You are invited to contribute to the site.

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, Eltrombopag

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Regimen

Study Evidence
Townsley et al. 2015 Phase II

In the abstract, the authors state that all patients received "standard" ATG (horse) and cyclosporine, without further specification. The doses below are from the published papers for ATG (horse) & cyclosporine. There were also 3 cohorts of eltrombopag, none were mentioned as preferred.

  • 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
  • Eltrombopag (Promacta) on one of the following 3 schedules:
    • Cohort 1: 150 mg PO once per day starting on day 14 and continuing for 6 months
    • Cohort 2: 150 mg PO once per day starting on day 14 and continuing for 3 months
    • Cohort 3: 150 mg PO once per day starting on day 1 and continuing for 6 months

Supportive medications:

References

  1. Abstract: Townsley et al. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia Accelerates Count Recovery and Increases Response Rates. ASH 2015 Annual Meeting LBA-2 link to abstract

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
Di Bona et al. 1999 Phase II
Scheinberg et al. 2006 Phase II

To be completed

References

  1. Di Bona E, Rodeghiero F, Bruno B, Gabbas A, Foa P, Locasciulli A, Rosanelli C, Camba L, Saracco P, Lippi A, Iori AP, Porta F, De Rossi G, Comotti B, Iacopino P, Dufour C, Bacigalupo A. Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Br J Haematol. 1999 Nov;107(2):330-4. Erratum in: Br J Haematol 2000 Feb;108(2):461. De Rossi V [corrected to De Rossi G]. link to original article PubMed
  2. 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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