Stem cell therapy has cured Diamond-Blackfan anemia in a patient with Duchenne muscular dystrophy - a research team from the Department of Haematology & Bone Marrow Transplantation in Army Hospital, India, reports.
The doсtors conducted a single-patient study to see if allogeneic hematopoietic stem cell transplantation (using cells from a donor) could treat anemia in a patient suffering from Duchenne muscular dystrophy at the same time. The boy was diagnosed Diamond-Blackfan anemia at eight months old as he had low red blood cell count despite normally developed bone marrow. When the boy was five years old, he was diagnosed with Duchenne muscular dystrophy.
As the boy's older brother had identical human leukocyte antigen (HLA) matching, the patient received his brother’s hematopoietic stem cells, although some medications to prevent graft-versus-host disease were used before stem cell transplantation. Later, the doctors closely monitored how many cells were successfully transferred and grafted.
As a result of the treatment, the anemia was cured, and the boy has been transfusion-free at least two months post-transplant.
There were no pronounced effects of the therapy on Duchenne muscular dystrophy course in the patient so far, but future clinical follow-ups and muscle biopsies from this patient could shed light whether hematopoietic stem cell transplantation worked for treating this disease not.