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Aplastic anemia (AA) is an uncommon disorder in children, with hematopoietic stem cell transplant being the 1st line therapy; immunosuppressive therapy (IST) is the alternative therapy and is the most commonly used modality of treatment. There is paucity of data from the developing countries regarding treatment outcome with IST. We aimed to assess the outcome of IST in children with AA. Data for 43 children treated with IST from January 2012 to January 2017 (5 years) were retrieved from clinic records. IST included equine antithymocyte globulin (ATG) along with cyclosporine A. Complete response, partial response and nonresponse was seen in 9 (21%), 14 (32.5%) and 20(46.5%) patients, respectively. The median time to best response in the whole cohort was 19.1 months. However, complete response occurred nearly 2-year post-IST. There was no difference in outcome related to severity of AA, the presence of PNH clone, higher ALC or different available brands of ATG. There was a significantly better rate of response ( value: .03) at 6 months in patients who went on to achieve a CR vs patients who achieved a final PR only. An overall response rate, including partial and complete response, of 53.5% was seen in our cohort with a 3-year OS of 63%. This is not at par to the outcome reported from developed nations. The available brands of ATG were similar in terms of response kinetics as well as survival. A delayed time to complete response with prolonged requirement of cyclosporine therapy was seen in the cohort.
This article was published in the following journal.
Name: Pediatric hematology and oncology
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Comparison of outcomes of frontline immunosuppressive therapy and frontline haploidentical hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA-matched sibling donor.
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To the investigator's Knowledge this is the first study that will assess Treatment with thrombopoietin Mimetic plus immunosuppressiveTherapy in Egyptian Patients with Aplastic Anaemia. ...
PRECIS - Most acquired aplastic anemia ensues from immune-mediated destruction of hematopoietic stem and progenitor cells (Young, 2013). - Immunosuppression is the defin...
A form of anemia in which the bone marrow fails to produce adequate numbers of peripheral blood elements.
A severe sometimes chronic anemia, usually macrocytic in type, that does not respond to ordinary antianemic therapy.
A megaloblastic anemia occurring in children but more commonly in later life, characterized by histamine-fast achlorhydria, in which the laboratory and clinical manifestations are based on malabsorption of vitamin B 12 due to a failure of the gastric mucosa to secrete adequate and potent intrinsic factor. (Dorland, 27th ed)
Manipulation of the host's immune system in treatment of disease. It includes both active and passive immunization as well as immunosuppressive therapy to prevent graft rejection.
The type species of GYROVIRUS, a small, non-enveloped DNA virus originally isolated from contaminated vaccines in Japan. It causes chicken infectious anemia and may possibly play a key role in hemorrhagic anemia syndrome, anemia dermatitis, and blue wing disease.
Blood is a specialized bodily fluid that delivers necessary substances to the body's cells (in animals) – such as nutrients and oxygen – and transports waste products away from those same cells. In vertebrates, it is composed of blo...
Pediatrics is the general medicine of childhood. Because of the developmental processes (psychological and physical) of childhood, the involvement of parents, and the social management of conditions at home and at school, pediatrics is a specialty. With ...