Pb2069: eltrombopag with immuno suppressive therapy in severe acquired aplastic anemia in children and adolescents: a monocentric experience

Topic: 12. Bone marrow failure syndromes incl. PNH - Clinical Background: Severe acquired aplastic anemia (SAA) is a rare, life-threatening disorder. Immunosuppressive therapy with transfusion support represented the standard treatment. Recently eltrombopag was added to improve the outcome. Aims: This study aims to report the clinical and laboratory findings at the time of diagnosis, the treatment approach, and the outcome of children and adolescents with AA treated in our institution with eltrombopag and immunosuppressive therapy. Methods: We retrospectively evaluated 8 children and adolescents with (AA), treated with immunosuppressive therapy +eltrombopag and followed at the hematology department at Farhat Hached University hospital in Sousse-Tunisa between 2016 and 2022. Results: Eight patients, 5 males and 3 females with AA were analyzed. The mean age at diagnosis was 12 years (5 - 17 years). One patient had a type I diabetes. Another patient developed AA during treatment with isotretinoin. Infection was the most frequent reason for consultation in 5 patients followed by anemia (3 patients) and hemorrhagic signs in 2 patients. At the time of diagnosis, the average neutrophil count was 150/mm3, the Hb was 5.45 g/dl and the platelets count was 13400/mm3. According to the initial laboratory findings and the Camitta’s criteria, all patients were classified as very severe acquired aplastic anemia. All patients received equine anti-thymocyte globulin (ATGAM) + Cyclosporine associated with eltrombopag (150mg/day) and supportive care. Four patients achieved a complete response, a partial response in 2 cases. Two patients died of infectious complications. One of the2 patients who had a partial response underwent matched family donor allogenic stem cell transplantation (the donor weight initially was under 20 kg) and achieved a complete response, all 5 patients are transfusion independent and the 6th is currently in partial remission requiring often transfusion. No grade 3 or 4 toxicity was noted. The overall survival was 75% Summary/Conclusion: The addition of eltrombopag to standard immunosuppressive treatment was well tolerated and resulted in satisfactory hematological outcome in this single-institution experience. Keywords: Aplastic anemia, Immunosuppressive therapy, Children, Age