Three children with adrenoleukodystrophy (ALD) were treated with allogeneic bone marrow transplantation (BMT) at Huddinge University Hospital, Sweden. Patient 1, a boy aged 9, presented with attention deficit disorder and learning disability; MRI showed demyelination in the frontal lobes which progressed over a 4 year follow-up, in association with mental deterioration. The 2nd and 3rd patients had no symptoms but white matter lesions were present on MRI at diagnosis. One had Addison’s disease at 6 years of age, and ALD was confirmed biochemically and by MRI six months later. He showed worsening of attention deficits before, and he died 18 months after BMT. Patient 3 was the younger brother of patient 2, and ALD and Addison’s disease were diagnosed at 4 years. This boy is healthy 3.5 years after BMT. [1]
COMMENT. The authors recommend bone marrow transplantation implemented early in the treatment of ALD, even without symptoms, provided that the MRI shows signs of demyelination and a suitable donor is available.
Attention deficit disorder was the presenting clinical manifestation of ALD in one of these patients, emphasizing the neurologist’s role in the management of children with ADHD.