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Diagnosis and Treatment of Aplastic Anemia

  • Published:2017-11-16 Issue:12 Volume:18 Page:
  • ISSN:1527-2729
  • Container-title:Current Treatment Options in Oncology
  • language:en
  • Short-container-title:Curr. Treat. Options in Oncol.

Author:

Peslak Scott A.,Olson Timothy,Babushok Daria V.

Funder

National Heart, Lung, and Blood Institute

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Oncology

Reference99 articles.

1. Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012;120(6):1185–96. https://doi.org/10.1182/blood-2011-12-274019 .

2. Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012;367(1):11–9. https://doi.org/10.1056/NEJMoa1200931 .

3. •• Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M, et al. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014;123(12):1818–25. https://doi.org/10.1182/blood-2013-10-534743 . A follow-up of a phase 2 study of eltrombopag monotherapy in patients with refractory SAA showing an overall response of 40%, with a subset of patients achieving trilineage hematopoietic responses.

4. •• Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med. 2017;376(16):1540–50. https://doi.org/10.1056/NEJMoa1613878 . A prospective phase 1-2 study of immunosuppressive therapy plus eltrombopag in newly-diagnosed SAA showing that addition of eltrombopag improves hematologic response as compared to historical IST controls.

5. • Dufour C, Veys P, Carraro E, Bhatnagar N, Pillon M, Wynn R, et al. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT working party, Paediatric diseases working party and severe aplastic Anaemia working party of EBMT. Br J Haematol. 2015;171(4):585–94. https://doi.org/10.1111/bjh.13614 . A retrospective analysis of 29 consecutive children who underwent unrelated donor transplantation with Fludarabine/Cyclophosphamide/Alemtuzumab (FCC) conditioning in an upfront setting, which showed outcomes similar to historical controls treated with matched sibling donor transplantation and superior to IST and to unrelated donor transplant after IST failure, suggesting that front-line therapy with unrelated donor BMT may be considered in selected pediatric patients without a matched sibling donor.
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