Bone marrow transplant for sickle cell disease--the dilemma

Sickle cell disease is the family of clinically significant haemoglobin disorders which have in common the inheritance of the sickle beta haemoglobin chain gene. The homozygous SS condition, also known as sickle cell anaemia, can present a varied clinical picture from asymptomatic through to frequent painful vaso-occlusive crises and even death as a result of complications of the disease. S beta zero thalassaemia and the rate haemoglobin SD disease present a similar picture to SS, while S beta plus thalassaemia and haemoglobin SC disease generally have milder clinical features than SS and present somewhat later in life. The present debate about bone marrow transplant (BMT) for sickle cell disease, among both physicians and community, relates to SS patients, definition and markers of disease severity, the effect of BMT on sickle related organ damage, the efficacy of BMT for SS, and its complications, both early and long-term.