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01-12-2017 | Leukemia (PH Wiernik, Section Editor)

Diagnosis and Treatment of Aplastic Anemia

Authors: Scott A. Peslak, MD, PhD, Timothy Olson, MD, PhD, Daria V. Babushok, MD, PhD

Published in: Current Treatment Options in Oncology | Issue 12/2017

Opinion statement

Acquired aplastic anemia (AA) is a rare, life-threatening bone marrow failure (BMF) disorder that affects patients of all ages and is caused by lymphocyte destruction of early hematopoietic cells. Diagnosis of AA requires a comprehensive approach with prompt evaluation for inherited and secondary causes of bone marrow aplasia, while providing aggressive supportive care. The choice of frontline therapy is determined by a number of factors including AA severity, age of the patient, donor availability, and access to optimal therapies. For newly diagnosed severe aplastic anemia, bone marrow transplant should be pursued in all pediatric patients and in younger adult patients when a matched sibling donor is available. Frontline therapy in older adult patients and in all patients lacking a matched sibling donor involves immunosuppressive therapy (IST) with horse antithymocyte globulin and cyclosporine A. Recent improvements in upfront therapy include encouraging results with closely matched unrelated donor transplants in younger patients and the emerging benefits of eltrombopag combined with initial IST, with randomized studies underway. In the refractory setting, several therapeutic options exist, with improving outcomes of matched unrelated donor and haploidentical bone marrow transplantation as well as the addition of eltrombopag to the non-transplant AA armamentarium. With the recent appreciation of frequent clonal hematopoiesis in AA patients and with the growing use of next-generation sequencing in the clinic, utmost caution should be exercised in interpreting the significance of somatic mutations in AA. Future longitudinal studies of large numbers of patients are needed to determine the prognostic significance of somatic mutations and to guide optimal surveillance and treatment approaches to prevent long-term clonal complications.

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Title: Diagnosis and Treatment of Aplastic Anemia
Authors: Scott A. Peslak, MD, PhD
Timothy Olson, MD, PhD
Daria V. Babushok, MD, PhD
Publication date: 01-12-2017
Publisher: Springer US
Published in: Current Treatment Options in Oncology / Issue 12/2017
Print ISSN: 1527-2729
Electronic ISSN: 1534-6277
DOI: https://doi.org/10.1007/s11864-017-0511-z

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