RIC BMT

http://www.hematologytimes.com/ht/p_article.do?id=676&section=Transplantation

RIC HSCT could cure sickle cell disease

HT Staff   Print | Email | Discuss
Published: 11/12/08   

Sickle cell
Allogeneic hematopoietic stem cell transplant (HSCT) following a reduced intensity conditioning (RIC) regimen has the potential to cure sickle cell disease, according to a small study.

Lakshmanan Krishnamurti, MD, of Children’s Hospital of Pittsburgh, and colleagues found that 6 of 7 sickle cell patients who received RIC HSCT in the last decade engrafted successfully and are free from symptoms of sickle cell disease.

“Through the reduced-intensity approach we developed, the potential for complications is dramatically lessened,” Dr Krishnamurti said. “This study offers hope for a cure to thousands of patients with severe sickle cell disease.”

The 7 high-risk sickle cell patients enrolled in the study underwent HSCT from an HLA-matched sibling donor following an RIC regimen. The conditioning regimen was comprised of busulfan, fludarabine, equine antithymocyte globulin, and total lymphoid irradiation with shielding of the liver, lungs, heart, and gonads on day 1. Patients also received GVHD prophylaxis consisting of cyclosporine and mycophenolate mofetil.

All patients experienced hematopoietic recovery and were able to tolerate the regimen. Six of the patients are stably engrafted off immunosuppression and have no sickle cell-related symptoms at 2 years to 8.5 years after HSCT.

Dr Krishnamurti was the first physician in the world to perform an RIC bone marrow transplant in a patient with sickle cell disease while at the University of Minnesota in 1999. He joined Children's Hospital in 2003, and that year, he performed the region's first successful bone marrow transplant in a patient with sickle cell disease.

Austin Jones, then 5, of Indiana, Pennsylvania, underwent an RIC bone marrow transplant August 8, 2003, with donor marrow from his brother, Anthony Jr. Today, Austin, now 10, is free of sickle cell disease.

Dr Krishnamurti’s study was published in the November issue of Biology of Blood and Marrow Transplantation.

Dr. Krishnamurti is a wonderful doctor - I have known him for the past 10 years and he has been very dedicated to this work. 

Sharmin

Sharmin,

I didn't know you knew Dr. Krishnamurti. I am such a fan of his work! Thanks for the update. It's wonderful to see his approach continue to meet with success. I posted about him awhile back at

http://www.thalassemiapatientsandfriends.com/index.php?topic=471.msg3475#msg3475

Please keep us posted about any new developments and progress.

While we are waiting for gene therapy, better and safer (involving less toxicity, less gvhd, lower chances of mortality) methods of bone marrow transplant are also vital.  I think that research such as this, RIC HSCT can change the outcome of transplants for people.  I will post anything else that I learn from him.

Sharmin