Brescia Performs First Bone Marrow Transplant for Leukodystrophy in Child
Successfully performed in recent months at the Clinica pediatrica del Civile, a French girl has undergone a bone marrow transplant (the first in Brescia) to treat metachromatic leukodystrophy, a severe inherited metabolic disease of the nervous system that affects one in every 40,000 children.
The transplant saved Clara, a nearly five-year-old girl. Her older sister was also affected by the same disease. The salvation for Clara came precisely from her sister’s tragedy: the disease was diagnosed early enough so that intervention was possible.
Only thanks to early diagnosis made at a hospital in Paris
Now Clara is improving and is still under care at the Civile, housed at the Casa dei bambini. The family’s ordeal began about two years ago, when Clara’s younger sister started first grade.
The diagnostic and therapeutic pathway
With the start of school, the girl began experiencing problems with incontinence. The situation continued to worsen, and after a medical check-up, the terrible metabolic disease was diagnosed, causing degenerations at motor and intellectual levels.
After the diagnosis of her older sister, doctors also performed examinations on Clara, confirming she was affected by the same disease. The substances involved in a person’s metabolism accumulate abnormally in the cells, causing neurological damage.
Only a bone marrow transplant can halt the progression of the disease. However, the transplant can only be performed if the disease is diagnosed early. For little Clara, the metabolic disease was diagnosed in February at a Parisian hospital.
The French doctors requested the availability of the Clinica pediatrica del Civile for the transplant: the Brescia center is an international reference point. The operation was made possible by identifying a compatible donor registered in the bone marrow bank.
