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Front-line immunosuppressive treatment of acquired aplastic anemia

Bone Marrow Transplantation volume 48, pages 174–177 (2013)Cite this article

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Abstract

In this article, front-line immunosuppressive therapy (IST) for acquired plastic anemia (AA) is illustrated and discussed. Also second-line and salvage options are briefly illustrated. First-line IST should consist of horse anti-thymocyte globulin (ATG) and CsA that has been shown to result in response rates between 60 and 80%. CsA should be given for 12 months until transfusion independence is achieved and then tapered very slowly in the presence of a CR. Patients with a partial response are usually continued on CsA. Tight monitoring of the blood count during CsA tapering is necessary to identify early loss of response. G-CSF 5 μg/kg/day s.c. in the first 30 days has been shown to reduce infections and hospitalization and to identify early responders, as those who achieve neutrophils count of⩾0.5 × 109/L by day +30. This schedule is recommended in the first month of therapy. Afterward, G-CSF can be considered in neutropenic febrile episodes. Patients not achieving transfusion independence after a first course of IST may be considered for second-line IST, or for an allogeneic hematopoietic SCT depending on patient age, ongoing infection, neutrophil count and transfusion requirements. Third-line IST is rarely given, but some options are discussed.

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Acknowledgements

ERG spa, SAAR Depositi Oleari Portuali, Rimorchiatori Riuniti and Cambiaso and Risso are acknowledged for their support to the activity of the Clinical and Experimental Hematology Unit of G Gaslini Children’s Hospital, Genova, Italy.

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Authors and Affiliations

  1. Clinical and Experimental Hematology Unit, G Gaslini Children’s Hospital, Genova, Italy

    C Dufour & J Svahn

  2. Second Hematology Department, San Martino Hospital, Genova, Italy

    A Bacigalupo

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on behalf of the Severe Aplastic Anemia–Working Party of the EBMT

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Correspondence to C Dufour.

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Competing interests

Small research funding to the Hematology Unit of G Gaslini Children’s Hospital from Pfizer in 2010.

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Dufour, C., Svahn, J., Bacigalupo, A. et al. Front-line immunosuppressive treatment of acquired aplastic anemia. Bone Marrow Transplant 48, 174–177 (2013). https://doi.org/10.1038/bmt.2012.222

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