Key result
Hematopoietic cell transplantation for cerebral ALD in childhood did not prevent the onset of adrenomyeloneuropathy in adulthood, with 3 of 5 patients (60%) developing signs of myelopathy.
Why the study?
Does hematopoietic cell transplantation in childhood prevent the onset of adrenomyeloneuropathy in adulthood in patients with X-linked adrenoleukodystrophy?
Observational (n=5)
Does hematopoietic cell transplantation in childhood prevent the onset of adrenomyeloneuropathy in adulthood in patients with X-linked adrenoleukodystrophy?
Hematopoietic cell transplantation for cerebral ALD in childhood does not appear to prevent the onset of adrenomyeloneuropathy in adulthood.
Suggests childhood HCT may not prevent adult myelopathy in cerebral ALD; hypothesis-generating, needs larger prospective validation.
BACKGROUND: X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 years). Cerebral ALD is treated with haematopoetic cell transplantation (HCT). It is unknown if AMN still develops in patients with X-ALD that underwent HCT for cerebral ALD in childhood. PATIENTS AND METHODS: A retrospective observational study was performed by selecting all adult patients with X-ALD in our cohort that underwent HCT in childhood. RESULTS: This retrospective study found that three out of five patients in our cohort who underwent HCT in childhood developed signs of myelopathy in adulthood. CONCLUSION: These data suggest that HCT for cerebral ALD in childhood does not prevent the onset of AMN in X-ALD in adulthood.
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Geel et al. (2014) conducted an observational in X-linked adrenoleukodystrophy (X-ALD) (n=5). Hematopoietic cell transplantation (HCT) was evaluated on Signs of myelopathy in adulthood. Hematopoietic cell transplantation for cerebral ALD in childhood did not prevent the onset of adrenomyeloneuropathy in adulthood, with 3 of 5 patients (60%) developing signs of myelopathy.
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