Outcomes of paediatric patients with acquired aplastic anaemia treated with immunosuppressive therapy at two Johannesburg Hospitals
No Thumbnail Available
Date
2020
Authors
Van der Nest, Alison
Journal Title
Journal ISSN
Volume Title
Publisher
Abstract
Background
Aplastic anaemia (AA) is a life-threatening disorder of bone marrow failure, due to an
autoimmune destruction of stem cells in the bone marrow. Treatment options include
haematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST).
Most frequently, IST is used for treatment of AA in South Africa as HSCT is often
unavailable. This study describes the population of paediatric patients with acquired AA in
South Africa and determines their response to IST. Methods and materials
A retrospective record review was done of patients diagnosed with AA between January 2002
and December 2011 at two Johannesburg centres. Forty-one records were included. The
patient demographics are illustrated. The main outcomes reviewed include overall survival,
survival with treatment, response to therapy, rate of relapse and progression to clonal
disorders and malignancy, and causes of death.Results
61% of patients were male. The median age at diagnosis was 10.1 years. The majority (67%)
had severe AA. Overall survival rates were 68% for all patients and 75% for patients treated
with IST. 83% of patients received IST (38% one course, 62% two courses). At the six month
evaluations, 6% had a complete response, 59% had a partial response and 15% had no
response. The relapse rate was 15% and progression to clonal disorder was 5%. Thirteen
patients (32%) demised.Conclusion
This study demonstrates patient demographics, survival rates and response to IST in keeping
with international findings. The use of IST can be justified in this setting when HSCT cannot
be provided.
Description
A research report submitted in partial fulfilment of the requirements for the degree of
Master of Medicine in Paediatrics to the Faculty of Health Sciences, University of the
Witwatersrand, Johannesburg, 2020