Bone marrow transplantation in thalassaemia

This is not, at present conventional treatment of beta thalassaemia. However results of studies of allogenic bone marrow transplantation by Lucarelli and his colleagues from Pesaro, Italy have been encouraging.

Reported probabilities of survival, rejection-free survival, death unrelated to rejection and rejection were reported at 0.92, 0.85, 0.06, and 0.08 respectively.

• first successful allogeneic stem cell transplant from an HLA-identical sibling donor was reported in 1982
• Italian group led by Lucarelli has the most experience with this procedure
  • documented a 90% long-term survival rate in patients with favorable characteristics (young age, HLA match, no organ dysfunction)

Transplantation-related issues such as graft versus host disease, graft failure, chronic immunosuppressive therapy, and transplantation-related mortality are factors that should be carefully considered prior to proceeding with this approach.

Reference:

• Thomas ED, Buckner CD, Sanders JE, Papayannopoulou T, Borgna-Pignatti C, De Stefano P. Marrow transplantation for thalassaemia. Lancet. Jul 31 1982;2(8292):227-9
• Lucarelli G, Galimberti M, Polchi P. Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N Engl J Med. Sep 16 1993;329(12):840-4