A pilot study investigating the aetiology of aplastic anaemia and the response to immunosuppressive therapy at the Charlotte Maxeke Johannesburg Academic Hospital.

Abstract

Introduction There is a paucity of South African data documenting the various causes of Aplastic Anaemia (AA), as well as the response to combination immunosuppressive therapy. The vast differences in treatment protocols internationally may be as a result of the poor representation in literature owing to the rarity of the disorder. Here, an analysis of possible secondary causes of AA as well as individual responses to treatment was reviewed at the Charlotte Maxeke Johannesburg Academic Hospital. Objective To determine the absolute number of cases, possible causes and response to immunosuppressive therapy in aplastic anaemia at the Charlotte Maxeke Johannesburg Academic Hospital. Method A retrospective cross-sectional analysis of all laboratory confirmed cases of aplastic anaemia at the Charlotte Maxeke Johannesburg Academic Hospital for the period of January 2014 to June 2019. Results There were 35 confirmed cases of AA for the given study period; 21 of which were males and 14 females. Sixty-five percent of the study sample had no identifiable cause for AA and were defined as idiopathic. Twelve patients had identifiable secondary causes for AA. Of these, 5 patients were exposed to a toxin (hydrocarbons), 2 patients were exposed to drugs (azathioprine and zidovudine), two patients had active Hepatitis B infection, two patients had a documented auto-immune condition (Systemic Lupus Erythematosus and Rheumatoid Arthritis) and one patient was pregnant at initial presentation. All patients were treated with a combination of antithymocyte globulin (ATG), rabbit or horse derived, cyclosporine and corticosteroids. None of the patients had received a stem-cell transplant. Page | vi Based on the World Health Organization (WHO) Haematological Toxicity Scale, 46% of patients had a poor response to treatment, 26% had a partial response to treatment and 28% had a complete response to treatment. Twenty-seven percent of patients died, many of whom had no clearly documented or suspected immediate cause of death. However, mortality cannot be commented on, due to the absence of complete follow-up data. Conclusion Aplastic Anaemia is a rare disease which carries a high mortality rate. All possible secondary causes should be sought by taking a detailed patient history and conducting relevant viral serology and immunological assays. All patients must be treated with a standard treatment protocol and any exposure to toxins or offending drugs should be terminated. A South African Bone Marrow Registry (SABMR) donor search should be conducted on all patients eligible for a stem-cell transplant to improve the number of patients receiving transplant therapy. The rationale for the absence of bone marrow transplants conducted must be investigated and acted upon. Every effort to improve patient follow-up and continuation of care should be made to establish concrete data on mortality and long-term complications.