Progression of abnormalities in adrenomyeloneuropathy and neurologically asymptomatic X-linked adrenoleukodystrophy despite treatment with "Lorenzo's oil"

Journal of Neurology Neurosurgery and Psychiatry

Volumn 67, Issue 3, 1999, Pages 290-299

  Van Geel, B M ^a,d   Assies, J ^b   Haverkort, E B ^a   Koelman, J H T M ^c   Verbeeten Jr , B ^a   Wanders, R J A ^a   Barth, P G ^d  

^a Department of Neurology ^*

^b Departments of Internal Medicine and Psychiatry

^c Division of Clinical Neurophysiology

^d UNIVERSITY OF AMSTERDAM   (Netherlands)

Author keywords

Adrenomy eloneuropathy; Lorenzo's oil; Peroxisome; X linked adrenoleukodystrophy

Indexed keywords

HEMOGLOBIN; LIVER ENZYME; LORENZO OIL; VERY LONG CHAIN FATTY ACID;

ADOLESCENT; ADRENOLEUKODYSTROPHY; ADULT; ARTICLE; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; DISABILITY; DISEASE SEVERITY; ENDOCRINE FUNCTION; EVOKED RESPONSE; FATTY ACID BLOOD LEVEL; FEMALE; GASTROINTESTINAL SYMPTOM; GINGIVITIS; HEMOGLOBIN DETERMINATION; HUMAN; LEUKOCYTE COUNT; MALE; NERVE CONDUCTION; NUCLEAR MAGNETIC RESONANCE IMAGING; PRIORITY JOURNAL; THROMBOCYTOPENIA; X CHROMOSOME LINKAGE;

ADOLESCENT; ADRENOLEUKODYSTROPHY; ADULT; BRAIN; CHILD; CHILD, PRESCHOOL; DIETARY FATS, UNSATURATED; DRUG COMBINATIONS; ENDOCRINE SYSTEM; ERUCIC ACIDS; EVOKED POTENTIALS; FEMALE; HUMANS; LINKAGE (GENETICS); MAGNETIC RESONANCE IMAGING; MALE; TRIOLEIN; X CHROMOSOME;

EID: 0032790614     PISSN: 00223050     EISSN: None     Source Type: Journal    
DOI: 10.1136/jnnp.67.3.290     Document Type: Article

References (53)

1. Singh I, Moser AE, Goldfischer S, et al. Lignoceric acid is oxidized in the peroxisome: implications for the Zellweger cerebro-hepato-renal syndrome and adrenoleukodystrophy. Proc Nad Acad Sci USA 1984;81:4203-7.
2. Wanders RJA, van Roermund CWT, van Wijland MJA, et al. X linked adrenoleukodystrophy: defective peroxisomal oxidation of very long chain fatty acids but not of very long chain fatty acyl-CoA esters. Clin Chim Acta 1987;165:321-9.
3. Lazo O, Contreras M, Hashmi M, et al. Peroxisomal lignoceroyl-CoA ligase deficiency in childhood adrenoleukodystrophy and adrenomyeloneuropathy. Proc Natl Acad Sci USA 1988;85:7647-51.
4. Powers JM. Adreno-leukodystrophy (adreno-testiculoleuko-myelo-neuropathic-complex). Clin Neuropathol 1985;4:181-99.
5. Moser HW, Smith KD, Moser AB. X linked adrenoleukodystrophy. In: Scriver CR, Beaud A, Sly WS, et al, eds. The metabolic and molecular bases of inherited disease. 7th ed. New York: McGraw-Hill, 1995:2325-49.
6. Aubourg P, Chaussain JL Adrenoleukodystrophy presenting as Addison's disease in children and adults. Trends Endocrinol Metab 1991;2:49-52.
7. van Geel BM, Assies J, Weverling GJ, Barth PG. Predominance of the adrenomyeloneuropathy phenotype of X linked adrenoleukodystrophy in the Netherlands: a survey of 30 kindreds. Neurology 1994;44:2343-6.
8. van Geel BM, Assies J, Wanders RJA, et al. X linked adrenoleukodystrophy: clinical presentation, diagnosis and therapy. J Neurol Neurosurg Psychiatry 1997;63:4-14.
9. Moser HW, Moser AB, Smith KD, et al. Adrenoleukodystrophy: phenotypic variability and implications for therapy. J Inher Metab Dis 1992;15:645-64.
10. Brown III FR, van Duyn MA, Moser AB, et al. Adrenoleukodystrophy: effects of of dietary restriction and of administration of carnitine and clofibrate on clinical status and plasma fatty acids. Johns Hopkins Medical Journal 1982;151:164-72.
11. Tsuji S, Sano T, Ariga T, et al. Increased synthesis of hexacosanoic acid (C26:0) by cultured skin fibroblasts from patients with adrenoleukodystrophy (ALD) and adrenomyeloneuropathy (AMN). J Biochem 1981;90:1233-6.
12. Rizzo WB, Watkins PA, Phillips MW, et al. Adrenoleukodystrophy: oleic acid lowers fibroblast saturated C22-26 fatty acids. Neurology 1986;36:357-61.
13. Rizzo WB, Phillips MW, Dammann AL, et al. Adrenoleukodystrophy: dietary oleic acid lowers hexacosanoate levels. Ann Neurol 1987;21:232-9.
14. Moser AB, Borel J, Odone A, et al. A new dietary therapy for adrenoleukodystrophy: biochemical and preliminary clinical results in 36 patients. Ann Neurol 1987;21:240-9.
15. Rizzo WB, Leshner RT, Odone A, et al. Dietary erucic acid therapy for X linked adrenoleukodystrophy. Neurology 1989;39:1415-22.
16. Odone A, Odone M. Lorenzo's oil: a new treatment for adrenoleukodystrophy. J Pediatr Neurosci 1989;5:55-61.
17. Uziel G, Bertini E, Bardelli P, et al. Experience on therapy of adrenoleukodystrophy and adrenomyeloneuropathy. Dev Neurosci 1991;13:274-9.
18. Cappa M, Cambiaso P, del Balzo P, et al. Adrenoleukodystrophy: clinical and therapeutic updating. In: Bhatt HR, James VHT, Besser GM, et al, eds. Advances in Thomas Addison's diseases. Vol 1. Bristol, UK: Journal of Endocrinology, 1994:171-80.
19. Asano J, Suzuki Y, Yajima S, et al. Effects of erucic acid therapy on Japanese patients with X linked adrenoleukodystrophy. Brain Dev 1994;16:454-8.
20. Korenke GC, Hunneman DH, Kohler W, et al. Glyceroltrioleate / glyceroltrierucate therapy in 16 patients with X chromosomal adrenoleukodystrophy/adrenomyeloneuropathy: effect on clinical, biochemical and neurophysiological parameters. Eur J Pediatr 1995;154:64-70.
21. Aubourg P, Adamsbaum C, Lavallard-Rousseau MC, et al. A 2 year trial of oleic and erucic acids (Lorenzo's oil) as treatment for adrenomyeloneuropathy. N Engl J Med 1993; 329:745-52.
22. Moser HW, Moser AB, Frayer KK, et al. Adrenoleukodystrophy: increased plasma content of saturated very long chain fatty acids. Neurology 1981;31:1241-6.
23. Moser HW, Moser AB, Kawamura N, et al. Adrenoleukodystrophy: elevated C26 fatty acid in cultured skin fibroblasts. Ann Neurol 1980;7:542-9.
24. Kurtzke JF. Rating neurologic impairment in multiple sclerosis: an expanded disability status scale (EDSS). Neurology 1983;33:1444-52.
25. Wade DT, Collin C. The Barthel ADL index: a standard measure of physical disability? International Disability Studies 1988;10:64-7.
26. van Swieten JC, Koudstaal PJ, Visser MC, et al. Interobserver agreement for the assessment of handicap in stroke patients. Stroke 1988;19:604-7.
27. Assies J, Gooren LJG, van Geel B, et al. Signs of testicular insufficiency in adrenoleukodystrophy and neurologically asymptomatic X linked adrenoleukodystrophy: a retrospective study. Int J Andrology 1997;20:315-21.
28. van Geel BM, Koelman JHTM, Barth PG, et al. Peripheral nerve abnormalities in adrenomyeloneuropathy: a clinical and electrodiagnostic study. Neurology 1996;46:112-18.
29. Aramideh M, Hoogendijk JE, Aalfs CM, et al. Somatosensory evoked potentials, sensory nerve potentials and sensory nerve conduction in hereditary motor and sensory neuropathy type I. J Neurol 1992;239:277-83.
30. Aalfs CM, Koelman JHTM, Meyjes FE, et al. Posterior tibial and sural nerve somatosensory evoked potentials: a study in spastic paraparesis and spinal cord lesions. Electroencephabgr Clin Neurophysiol 1993;89:437-11.
31. Aubourg P, Blanche S, Jambaqué I, et al. Reversal of early neurologic and neuroradiologic manifestations of X linked adrenoleukodystrophy by bone marrow transplantation. N Engl J Med 1990;322:1860-6.
32. Krivit W, Lockman LA, Watkins PA, et al. The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome. J Inherit Metab Dis 1995;18:398-412.
33. Moser HW, Borel J. Dietary management of X linked adrenoleukodystrophy. Annu Rev Nutr 1995;15:379-97.
34. Powers JM, Liu Y, Moser AB, et al. The inflammatory myelinopathy of adreno-leukodystrophy: cells, effector molecules, and pathogenetic implications. J Neuropathol Exp Neurol 1992;51:630-43.
35. Rasmussen M, Moser AB, Borel J, et al. Brain, liver, and adipose tissue erucic and very long chain fatty acid levels in adrenoleukodystrophy patients treated with glyceryl trierucate and trioleate oils (Lorenzo's oil). Neurochem Res 1994; 8:1073-82.
36. Poulos A, Gibson R, Sharp P, et al. Very long chain fatty acids in X linked adrenoleukodystrophy brain after treatment with Lorenzo's oil. Ann Neurol 1994;36:741-6.
37. Moser HW. Clinical and therapeutic aspects of adrenoleukodystrophy and adrenomyeloneuropathy. J Neuropathol Exp Neurol 1995;54:740-5.
38. Assies J, van Geel BM, Weverling GJ, et al. Endocrine evaluation during dietary therapy for adrenomyeloneuropathy. In: Bhatt HR, James VHT, Besser GM, et al, eds. Advances in Thomas Addison's diseases. Vol 1. Bristol, UK: Journal of Endocrinology 1994:191-8.
39. Korenke GC, Roth C, Krasemann E, et al. Variability of endocrinological dysfunction in 55 patients with X linked adrenoleukodystrophy: clinical, laboratory and genetic findings. Eur J Endocrinol 1997;137:40-7.
40. Padgett DA, Loria RM. In vitro potentiation of lymphocyte activation by dehydroepiandrosterone, androstenediol and androstenetriol. J Immunol 1994;153:1544-52.
41. Konijnenberg A, van Geel BM, Sturk A, et al. Lorenzo's oil and platelet activation in adrenomyeloneuropathy and asymptomatic X linked adrenoleukodystrophy. Platelets 1998;9:41-8.
42. Unkrig CJ, Schröder R, Scharf RE. Lorenzo's oil and lymphocytopenia. N Engl J Med 1994;330:577.
43. Zierz S, Schröder R, Unkrig CJ. Thrombocytopenia induced by erucic acid therapy in patients with X linked adrenoleukodystrophy. Clin Invest 1993;71:802-5.
44. Zinkham WH, Kickler T, Borel J, et al. Lorenzo's oil and thrombocytopenia in patients with adrenoleukodystrophy. N Engl J Med 1993;328:1126-7.
45. Toplak H, Wascher TC, Pleschko B, et al. Reduced stimulability of platelet surface adhesion molecules under treatment with Lorenzo's oil. J Inherit Metab Dis 1994;17: 628-9.
46. Sedlmayr P, Pleschko B, Paschke E, et al. Severely depressed natural killer cell activity of patients with adrenoleukodystrophy under treatment with Lorenzo's oil. J Inherit Metab Dis 1995;18:101-2.
47. Stumpf DA, Hayward A, Haas R, et al. Adrenoleukodystrophy: failure of immunosuppression to prevent neurological progression. Arch Neurol 1981;38:48-9.
48. Naidu S, Bresnan MJ, Griffin D, et al. Childhood adrenoleukodystrophy: failure of intensive immunosuppression to arrest neurological progression. Arch Neurol 1988;45:846-8.
49. Korenke GC, Christen H-J, Kruse B, et al. Progression of X linked adrenoleukodystrophy under interferon-β therapy. J Inherit Metab Dis 1997;20:59-66.
50. Cappa M, Bertini E, del Balzo P, et al. High dose immunoglobulin IV treatment in adrenolsukodystrophy. J Neurol Neurosurg Psychiatry 1994;57(suppl):69-70.
51. Lu J-F, Lawler AM, Watkins PA, et al. A mouse model for X linked adrenoleukodystrophy. Proc Natl Acad Sci USA 1997;94:9366-71.
52. Singh I, Khan M, Key L, et al. Lovastatin for X linked adrenoleukodystrophy. N Engl J Med 1998;339:702-3.
53. Kemp S, Wei H-M, Lu J-F, et al. Gene redundancy and pharmacological gene therapy: implications for X linked adrenoleukodystrophy. Nat Med 1998;4:1261-8.