Adrenoleukodystrophy medical therapy: Difference between revisions
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief:
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Overview
The mainstay of treatment for adrenoleukodystrophy is Hematopoietic stem cell transplantation. Supportive therapy includes Lorenzo's oil, adrenal hormone replacement therapy and Lovastatin therapy.
Medical Therapy
Hematopoietic stem cell transplantation
The only standard therapy currently available is hematopoietic stem cell transplantation in cerebral ALD boys. It is the gold standard for cALD with a Loes score below 8. HSCT prevents demyelination in ALD patients but improvement of microglial peroxisome activity or reduction of VLCFA levels has not been identified as a result of HSCT.[1]
Lorenzo’s oil
While there is currently no cure for the disease, some dietary treatments, for example, Lorenzo's oil in combination with a diet low in VLCFA, have been used with limited success, especially before disease symptoms appear. A recent study by Moser et al (2005) shows positive long-term results with this approach;[2] see also the Myelin Project.
Lorenzo oil is a 4:1 mixture of oleic and erucic acids that, when combined with a fat-restricted diet, normalizes plasma VLCFA levels.
Adrenal Replacement Therapy
Adrenal hormone replacement therapy should be prescribed to more than 70% of male X-ALD patients with adrenal insufficiency. This can stop a life-threatening adrenal crisis, but it has not been proven to slow down the neurological course of the condition. [3]
Metabolic modulators
Lovastatin is an anti cholesterol drug that normalized plasma VLCFA in one study but showed no normalization to placebo in randomized double‐blind crossover trial. [4]
Several other studies are being conducted on sobetirome, a thyroid hormone receptor agonist[5], and pioglitazone, a PPARγ agonist.[6]
References
- ↑ Turk, Bela R.; Moser, Ann B.; Fatemi, Ali (2017). "Therapeutic strategies in adrenoleukodystrophy". Wiener Medizinische Wochenschrift. 167 (9–10): 219–226. doi:10.1007/s10354-016-0534-2. ISSN 0043-5341.
- ↑ Moser HW, Raymond GV, Lu S-E, Muenz LR, Moser AB, Xu J, Jones RO, Loes DJ, Melhem ER, Dubey P, Bezman L, Brereton NH, Odone A. Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's Oil. Arch Neurol 2005;62;1073-80. PMID 16009761.
- ↑ Moser HW, Raymond GV, Dubey P (2005). "Adrenoleukodystrophy: new approaches to a neurodegenerative disease". JAMA. 294 (24): 3131–4. doi:10.1001/jama.294.24.3131. PMID 16380594.
- ↑ Engelen, Marc; Ofman, Rob; Dijkgraaf, Marcel G.W.; Hijzen, Michiel; van der Wardt, Lucinda A.; van Geel, Bjorn M.; de Visser, Marianne; Wanders, Ronald J.A.; Poll-The, Bwee Tien; Kemp, Stephan (2010). "Lovastatin in X-Linked Adrenoleukodystrophy". New England Journal of Medicine. 362 (3): 276–277. doi:10.1056/NEJMc0907735. ISSN 0028-4793.
- ↑ Hartley, Meredith D.; Kirkemo, Lisa L.; Banerji, Tapasree; Scanlan, Thomas S. (2017). "A Thyroid Hormone–Based Strategy for Correcting the Biochemical Abnormality in X-Linked Adrenoleukodystrophy". Endocrinology. 158 (5): 1328–1338. doi:10.1210/en.2016-1842. ISSN 0013-7227.
- ↑ Morató, Laia; Galino, Jorge; Ruiz, Montserrat; Calingasan, Noel Ylagan; Starkov, Anatoly A.; Dumont, Magali; Naudí, Alba; Martínez, Juan José; Aubourg, Patrick; Portero-Otín, Manuel; Pamplona, Reinald; Galea, Elena; Beal, M. Flint; Ferrer, Isidre; Fourcade, Stéphane; Pujol, Aurora (2013). "Pioglitazone halts axonal degeneration in a mouse model of X-linked adrenoleukodystrophy". Brain. 136 (8): 2432–2443. doi:10.1093/brain/awt143. ISSN 1460-2156.