A new bone marrow transplant procedure has successfully treated adult patients with sickle cell disease, it has been announced.
The procedure was developed by the US National Institutes of Health – and now doctors in Illinois, USA, say they have successfully adapted it to achieve a near total success rate in their first 13 patients.
The procedure has been developed to enable adult patients to receive tissue from healthy, matched siblings.
It avoids the need for chemotherapy to destroy the patients’ haematopoietic cells as this is considered too risky for adult patients.
The treatment involves giving the patients immunosuppressive drugs just before the transplant – together with a low dose of total body irradiation.
The patients then continue taking immunosuppressants for at least a year.
Doctors in Illinois treated 13 patients, aged between 17 and 40. Two patients were HLA-matched but had a different blood type from their siblings.
Reporting in Biology of Blood & Marrow Transplantation, the doctors say that one patient did not continue with the drugs after treatment – leading to a failure of the transplant.
After 12 months, the remaining 12 patients had normal hemoglobin concentrations and improved cardiopulmonary function. Four were able to stop the immunotherapy treatment.
Researcher Professor Damiano Rondelli said: "With this chemotherapy-free transplant, we are curing adults with sickle cell disease, and we see that their quality of life improves vastly within just one month of the transplant."
He added: "Adults with sickle cell disease can be cured without chemotherapy – the main barrier that has stood in the way for them for so long.
"Our data provide more support that this therapy is safe and effective and prevents patients from living shortened lives, condemned to pain and progressive complications."
Biology of Blood & Marrow Transplantation September 2015
Leave a Reply