Bone marrow transplantation for the treatment of X-linked adrenoleukodystrophy

The effects of bone marrow transplantation (BMT) in X-linked childhood adre noleukodystrophy (ALD) are described in four Japanese patients. Two older p atients, 10-year-old boys with IQ 60 and difficulty in school, had favourab le results. IQ levels and MRI findings were generally maintained after BMT. One patient showed improvement of gait disturbance. They have both attende d ordinary schools after BMT, although a learning disorder persists. On the other hand, two other younger patients with a rapid course and indetermina te IQ at BMT showed deterioration of neurological functions. Indication for BMT seems to be a maintained IQ level, preferably higher than 80, since it seems to be difficult to normalize IQ level after BMT. Younger patients ha ve higher risk of developing a rapidly progressive form of the disease. Ide ntification of presymptomatic boys, and serial and careful follow-up by neu ropsychological and neuroradiological studies, are essential prerequisites to successful BMT in X-ALD.