With a current waiting list of 30 to 40 children, and a referral base that is worldwide, the Duke Pediatric Blood and Marrow Transplant (BMT) Program is the largest children’s transplant program in the world, now transplanting some 100 children annually.

It is a program of last resort: These children have a life expectancy of less than one year without a transplant.

Innovation and Research

Duke was the first to use umbilical cord blood from unrelated donors to cure life-threatening cancers, rare immune deficiencies, and metabolic diseases. Because patients do not require an exact blood “HLA” match with cord blood as they do with bone marrow, it is easier to find a cord blood match providing access to this life-saving therapy for patients unable to find a fully matched donor.

Duke was also the first to prove that bone marrow transplants could cure severe combined immune deficiency if administered in the first months of life.

Recently, our researchers showed that transplantation can also be an effective treatment for Hurler’s syndrome, a rare metabolic disease (MPS1) in which patients suffer from progressive brain, liver, heart, bone, cartilage, and cornea damage due to a missing enzyme.

In addition, transplantation has proved effective in treating some cases of Krabbe disease, Sanfilippo syndrome (MPS III), metachromatic leukodystrophy (MLD), and adenoleukodystrophy (ALD). Duke researchers are continually studying other conditions that might be successfully treated with transplantation.