Adrenoleukodystrophy (ALD): treatment

Lorenzo’s oil

• The evidence for this is not established
• It may help if started early: before the age of 6 years
• It has no effect on brain lesions or disease course

Immunotherapy

Statins

Allogenic haematopoietic stem cell transplantation

Haematopoietic stem cell gene therapy

Investigational treatments: Leriglitazone

References

1. Engelen M, van Ballegoij WJC, Mallack EJ, et al. International recommendations for the diagnosis and management of patients with adrenoleukodystrophy: a consensus-based approach. Neurology 2022; 99:940-951.
2. Mukherjee S, Newby E, Harvey JN. Adrenomyeloneuropathy in patients with `Addison's disease': genetic case analysis. J R Soc Med 2006; 99:245-249.
3. van Geel BM, Assies J, Wanders RJ, Barth PG. X linked adrenoleukodystrophy: clinical presentation, diagnosis, and therapy. JNNP 1997; 63:4-14.
4. Restuccia D, Di Lazzaro V, Valeriani M, et al. Neurophysiologic follow-up of long-term dietary treatment in adult-onset adrenoleukodystrophy. Neurology 1999; 52:810-816.
5. Kühl JS, Suarez F, Gillett GT, et al. Long-term outcomes of allogeneic haematopoietic stem cell transplantation for adult cerebral X-linked adrenoleukodystrophy. Brain 2017; 140:953-966. 
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Related checklists

• Adrenoleukodystrophy (ALD): genetics and pathology
• Adrenoleukodystrophy (ALD): neurological features
• Adrenoleukodystrophy (ALD): systemic features
• Adrenoleukodystrophy (ALD): differential diagnosis
• Adrenoleukodystrophy (ALD): investigations
• Adrenoleukodystrophy (ALD): surveillance