Unresponsive Severe Aplastic Anemia in a Young Patient: Case Report and Short Review of the Literature

Aplastic anemia or medullary aplasia is a medical condition characterized by pancytopenia and is associated with a high prevalence of morbidity and mortality. In patients in whom bone marrow transplantation cannot be attempted, an immunosuppressive regimen is considered to be the first-line therapy. Also, the addition of eltrombopag from the first day of immunosuppressive treatment seems to significantly increase response rate. Unfortunately, there are a small number of patients who remain unresponsive to all these therapies. Here we present the case of a young woman who was referred by the family doctor complaining of marked physical asthenia, new onset dyspnea, and dizziness. Apart from a severe pancytopenia, no further changes have been brought to light by paraclinical investigations. After multiple secondary causes were excluded, the patient was diagnosed with idiopathic aplastic anemia. Even if bone marrow transplant was the first-line therapy in this case, because of the severe leukopenia, it was not possible to determine the HLA type. Therefore, the patient was prescribed immunosuppressive treatment. Despite the three drug-associated therapy (horse anti-thymocyte globulin, cyclosporin A, and eltrombopag), the response was unsatisfactory, with the persistence of severe pancytopenia.