الفهرس 
Descriptive Study of Pediatric Patients Suffering from Aplastic Anemia; A Single Center Experience
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Abstract
Introduction:
Aplastic anemia (AA) is heterogeneous group of disorders characterized by a hypoplastic, fatty bone marrow (BM) with profound reductions in hematopoietic stem cells (HSCs) that lead to peripheral pancytopenia and defective mature blood cell production, and can be inherited or acquired (Luzzatto & Risitano, 2018).
Aim of the work:
To study the epidemiological characteristics of aplastic anemia patients, identify causes of aplastic anemia in our cohort, differentiation between the inherited form and the acquired form of aplastic anemia, recognition the most frequent symptoms and signs, the response to treatment and the effectiveness of different combination of immunosuppressive drugs.
Methods:
The medical records for patients were reviewed and data collected including: Age, gender, cause of aplastic anemia, family history, detailed infection history, detailed bleeding history, frequency of transfusions before and after treatment, anthropometric measures of patients, detailed general and local examination, investigations, with special reference to different treatment received and response to treatment.
Results:
Our patients were 43 (44.3%) females and 54 (55.7%) males,
median age of presentation was 5 (3 – 9) years, 68 (70.1%) of patients had acquired aplastic anemia, 17 (17.5%) had Fanconi anemia, and 12 (12.4%) had pure red cell aplasia. Most of our patients received immunosuppressive therapy (IST) alone 31 (31.9%), (61.3%) of them achieved response and (29.0%) died. Nineteen patients (19.6%) were treated with bone marrow transplantation (BMT), 68.4% of them achieved response and 31.5% died. Seventeen patients (17.5%) received triple therapy (IST, Eltrombopag (EPAG) and anti thymocyte globulin (ATG)), 64.7% of them achieved response and 52.9% died. Thirteen patients (13.4%) received EPAG and IST, 76.9% of them achieved response and 15.4% died. Seven patients (7.2%) received danazoland
steroids, 42.9% achieved response and all of them (100%) are alive. Ten patients (10.3%) did not receive any treatment, 20.0% of them achieved spontaneous resolution and 80.0% died. In acquired aplastic anemia resolution (complete or partial) was achieved in 12/17 (70.6%) of patients treated with BMT, and 5/14 (35.7%) of patients treated with cyclosporine alone, and 10/13 (76.9%) of patients treated with cyclosporine and EPAG, and 12/16 (75.0%) patients treated with ATG, cyclosporine and EPAG. In patients who did not receive any treatment, only one patient achieved full resolution (1/8) as he was moderate form of acquired aplastic anemia. In patients with Fanconi anemia resolution (complete or partial) was obtained in 6/8 (75.0%) of cases on steroid alone, and 1/2 (50.0%) of patients who treated with BMT achieved full resolution, and in patients who received both steroid and androgen 1/4 (25%) achieved partial resolution, and 1/2 (50.0%) of patients who did not receive any treatment achieved full resolution as he was moderate form of Fanconi anemia. In patients with Pure red cell aplasia (PRCA) complete resolution was obtained in 6/9 (66.6%) of cases on steroids alone, and in 1/3 (33.3%) of patients who received steroids andandrogen.