CHILDHOOD CEREBRAL FORM OF ADRENOLEUKODYSTROPHY - SHORT-TERM EFFECT OF BONE-MARROW TRANSPLANTATION ON BRAIN MR OBSERVATIONS

PURPOSE: To report the serial brain MR observations in patients with c hildhood-onset cerebral adrenoleukodystrophy 1 to 2 years after bone m arrow transplantation. METHODS: Eight boys with childhood-onset cerebr al adrenoleukodystrophy have undergone successful transplantation at o ur institution. Seven patients (mean age, 8 years 10 months; range, 5 years 3 months to 11 years 9 months) had serial MR studies before and after transplantation. An MR severity score (0 to 34) based on disease location and the presence or absence of focal atrophy was calculated for each patient scan. RESULTS: Posttransplantation serial MR showed i mprovement in two patients, stabilization in three patients, and worse ning of MR signal changes in two patients. The patient with the most s triking progression had systemic graft-versus-host disease. Although t he adrenoleukodystrophy MR severity score did not change in three pati ents after transplantation, two of these patients did show improved ma rgination of disease. CONCLUSION: Bone marrow transplantation can affe ct brain MR observations in childhood-onset cerebral adrenoleukodystro phy. Although brain MR findings do not typically resolve, they do seem to stabilize, which is an improvement over the natural MR history of the disease.