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Single institution outcomes of treatment of severe aplastic anaemia

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Background: In severe aplastic anaemia, the treatment of choice for young patients with a human leucocyte antigen-matched sibling is now established as allogeneic bone marrow transplantation (BMT). In older patients and in those without a matched sibling donor, immunosuppressive therapy is the usual first option. ‘Alternative’ marrow donors are emerging as an option for those without a matched sibling donor.

Aims: To review 10 years of local experience in treating severe aplastic anaemia with BMT and immunosuppressive therapy with emphasis on long-term outcomes.

Methods: A retrospective analysis was performed of all patients with severe aplastic anaemia presenting to the Royal Brisbane and Royal Children’s Hos- pitals between 1989 and 1999. Data were abstracted regarding patient demographics, pretreatment characteristics and outcome measures, including response rates, overall survival and long-term complications.

Results: Twenty-seven consecutive patients were identified, 12 treated with immunosuppression alone and 15 with BMT. In these two groups, transfusion independence was attained in 25% and 100%, respectively, with overall survival being 36% and 100%, respectively. Those treated with immunosuppression were significantly older (median 41.5 versus 22 years, P = 0.008). Long-term survivors of either treatment had extremely low morbidity. Three patients carried pregnancies to term post-transplant. Three patients received alternative donor BMT with correspondingly excellent survival.

Conclusions: Patients treated with allogeneic BMT for severe aplastic anaemia enjoyed extremely good long-term survival and minimal morbidity. Patients treated with immunosuppressive therapy had a poorer outcome reflecting their older age and different usage of therapies over the past decade. Optimal treatment strategies for severe aplastic anaemia remain to be determined. (Intern Med J 2001; 31: 337–342)
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Keywords: aplastic anaemia; bone marrow transplantation; immunosuppression; unrelated donor transplantation

Document Type: Research Article

Affiliations: 1: Bone Marrow Transplant Unit 2: Haematology/Oncology Unit, Royal Children’s Hospital 3: Haematology Unit, Royal Brisbane Hospital 4: Queensland Health Pathology Service, Brisbane, Queensland, Australia

Publication date: 2001-08-01

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