An Analysis of Efficacy of Androgenic Steroids in patients with Acquired Aplastic Anemia.

Nisham, P N (2014) An Analysis of Efficacy of Androgenic Steroids in patients with Acquired Aplastic Anemia. Masters thesis, Christian Medical College, Vellore.


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INTRODUCTION : Acquired aplastic anemia (AA) is a rare disease of unknown pathogenesis and disputed etiology. The diagnosis is based on the criteria proposed by international aplastic anemia and agranulocytosis study group. There are several etiological factors proposed for the same based on epidemiological studies which include drugs, toxic chemicals, radiation,etc. Most cases of aplastic anemia have an immune mediated pathogenesis. This is revealed by its response to immunosuppressive drugs. However non immune factors also play an important role. The role of HLA DR2 allele, cytokine gene polymorphisms and telomeres in aplastic anemia have been widely discussed. The treatment of choice of acquired aplastic anemia in young patients without comorbidities is haematopoietic stem cell transplant. Other important treatment modality is immunosuppressive drugs. Androgenic steroids gives good response in a subset of patients with acquired aplastic anemia. The exact mechanism of action is however not known. Recent studies have shown that it tends to protect from telomere shortening in haematopoietic tissues by increasing Telomerase activity, the specialized reverse transcriptase which helps in adding telomeres to chromosome ends. AIMS AND OBJECTIVES : 1. To analyze the clinical profile of patients (adults and children) with acquired aplastic anemia treated with androgenic steroids. 2. To assess the response to androgenic steroids in the above patients with acquired aplastic anemia. 3. To identify the demographic, clinical, and laboratory parameters that can predict response to androgenic steroids in the above mentioned patients. MATERIALS AND METHODS : This study protocol was approved by our Institutional Review Board (IRB). This is a retrospective analysis of patients diagnosed to have acquired aplastic anemia and treated with androgenic steroids from January 2008 to December 2012. Diagnostic criteria: Aplastic Anemia was diagnosed in patients presenting with cytopenia(s) (defined by International Agranulocytosis and Aplastic anemia study group in 1987 as presence of at least two of the following ie. Haemoglobin < 100g/L, neutrophil count < 1.5 x109/L and platelet count < 50 x 109/L) associated with a hypoplastic bone marrow (ie.bone marrow cellularity <25%, <50% if less <30% of the bone marrow is haematopoietic cells). Inclusion Criteria: All adults (age≥15 years) and children (age 1-14 years) diagnosed to have acquired Aplastic anemia and treated with androgenic steroids from January 2008 to December 2012, and with a follow up of at least 12 weeks. Exclusion Criteria: 1. Patients with inherited bone marrow failure syndromes 2. Patients who had been on any other medications prior to starting androgenic steroids. 3. Patients on androgenic steroids with less than 12 weeks follow up. 4. Patients with acquired Aplastic anemia and on androgenic steroids whose data are not retrievable. Methods: After approval by the IRB, the patient data base at our institution was reviewed to identify all patients (adults and children) diagnosed to have acquired Aplastic anemia at our institute from January 2008 to December 2012. Medical information regarding the clinical/laboratory details at diagnosis, post treatment response and adverse events were obtained from the hospital records (laboratory reports/ physician documentation in hospital charts/hospital discharge summaries). Only patients who had at least 12 weeks follow up after initiating therapy were categorized as ‘evaluable‘ for assessment of response and survival. RESULTS : Between January 2008 and December 2012, a total of 8980 out patients were seen in the Haematology department, of which 1065 (3.67%) were diagnosed to have Aplastic anemia – both constitutional and acquired. Of this only 97 (9.1%) patients received androgenic steroid as first line treatment, while 208 (19.5%) received ATG, 334 (31.4%) were given CSA, 84 (7.9%) underwent Allogeneic PBSCT and the remaining 342 (32.1%) patients had received androgenic steroids after receiving other therapies. There were a total of 97 patients diagnosed to have acquired aplastic anemia and who received androgenic steroids as initial therapy who fulfilled the inclusion criteria for the study. Out of this 77 (79.3%) were adults (age ≥15 years) and 22 (20.7%) were children (age 1-14 years). CONCLUSION : 1. Androgens still remains a viable option in the treatment of aquired AA in resource constraint situations, with no major drug related adverse effects. 2. There is no significant association between severity and response to androgens. 3. Response to Danazol is superior to Stanozolol in adult patients. 4. Further prospective studies are needed to delineate the mechanisms involved in androgen action and the factors predicting response to therapy.

Item Type: Thesis (Masters)
Uncontrolled Keywords: Efficacy ; Androgenic Steroids ; Patients ; Acquired Aplastic Anemia ; Analysis.
Subjects: MEDICAL > Clinical Haematology
Depositing User: Subramani R
Date Deposited: 16 Aug 2017 00:39
Last Modified: 16 Aug 2017 03:44

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