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Titolo:
GLYCEROLTRIOLEATE GLYCEROLTRIERUCATE THERAPY IN 16 PATIENTS WITH X-CHROMOSOMAL ADRENOLEUKODYSTROPHY ADRENOMYELONEUROPATHY - EFFECT ON CLINICAL, BIOCHEMICAL AND NEUROPHYSIOLOGICAL PARAMETERS
Autore:
KORENKE GC; HUNNEMAN DH; KOHLER J; STOCKLER S; LANDMARK K; HANEFELD F;
Indirizzi:
UNIV GOTTINGEN,DEPT PAEDIAT & NEUROPAEDIAT,ROBERT KOCH ALLEE 40 D-37075 GOTTINGEN GERMANY
Titolo Testata:
European journal of pediatrics
fascicolo: 1, volume: 154, anno: 1995,
pagine: 64 - 70
SICI:
0340-6199(1995)154:1<64:GGTI1P>2.0.ZU;2-6
Fonte:
ISI
Lingua:
ENG
Soggetto:
ERUCIC-ACID THERAPY; CHAIN FATTY-ACIDS; LINKED ADRENOLEUKODYSTROPHY; LORENZO OIL; ADRENOMYELONEUROPATHY; TRIAL;
Keywords:
X-CHROMOSOMAL ADRENOLEUKODYSTROPHY; ADRENOMYELONEUROPATHY; VERY LONG-CHAIN FATTY ACIDS; THERAPY; PEROXISOMAL DISORDER;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Science Citation Index Expanded
Citazioni:
22
Recensione:
Indirizzi per estratti:
Citazione:
G.C. Korenke et al., "GLYCEROLTRIOLEATE GLYCEROLTRIERUCATE THERAPY IN 16 PATIENTS WITH X-CHROMOSOMAL ADRENOLEUKODYSTROPHY ADRENOMYELONEUROPATHY - EFFECT ON CLINICAL, BIOCHEMICAL AND NEUROPHYSIOLOGICAL PARAMETERS", European journal of pediatrics, 154(1), 1995, pp. 64-70

Abstract

We have investigated the effect of glyceroltrioleate/glyceroltrierucate (GTO/GTE) therapy on X-chromosomal adrenoleukodystrophy in 16 patients with adrenoleukodystrophy (n = 6), adrenomyeloneuropathy (n = 3), Addison disease without neurological involvement (n = 2), and neurologically and endocrinologically asymptomatic patients (n = 5). Therapy was carried out for 19.4 +/- 10 months. All patients showed a normalization of C 26:0 plasma fatty acid concentrations. None of the seven neurologically asymptomatic patients developed neurological symptoms. Somatosensory evoked potentials of the tibialis nerve was the most sensitive electrophysiological parameter, showing a slight improvement in neurologically asymptomatic patients during therapy. In none of the patients with normal cranial MRI at start of therapy (n = 6) has MRI deterioration been observed whilst on therapy. Follow up of the neurologically asymptomatic children supports the hypothesis that GTO/GTE therapymight prevent the development of neurological symptoms. Six of the nine neurologically symptomatic patients deteriorated to varying degreeswhilst on therapy. MRT alterations have worsened in all patients withclinical deterioration. Conclusion GTO/GTE treatment should be initiated in all neurological asymptomatic boys before first neurological symptoms develop. To discover these patients very long-chain fatty acid determination should be performed in all family members at risk when adrenoleukodystrophy or adrenomyeloneuropathy is diagnosed.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 01/10/20 alle ore 04:16:58