BOF: Red Cell (Haemoglobinopathy)

Severe beta thalassaemia major may be cured with bone marrow transplantation in carefully selected patients, with the best outcomes seen in children. However, their relapse risk may be high. Which of the following interventions has NOT been shown to improve the high relapse risk?

a) Addition of fludarabine

b) Addition of hydroxycarbamide

c) Busulphan dose increase

d) Cyclophosphamide dose increase

e) Hypertransfusion to target Hb 140-150g/l

Answer

c) Busulphan dose increase

Explanation

The Pesaro group in Italy have some of the most extensive experience of transplantation under these circumstances. The poor outcomes encountered in children were improved by suppression of the erythron through hypertransfusion and ongoing iron chelation, and hydroxyurea supported with GCSF, by increasing the immunosuppression by the addition of fludarabine and azathioprine, and by the use of a higher dose of cyclophosphamide. The busulfan dose was unchanged.

Reference:

Angelucci E, Matthes-Martin S, Baronciani D, et al. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel. Haematologica 2014; 99:811.