NR AOTS
AU Haik,S.; Brandel,J.P.; Salomon,D.; Sazdovitch,V.; Delasnerie-Laupretre,N.; Fierville,F.; Laplanche,J.L.; Faucheux,B.A.; Hauw,J.J.; Alperovitch,A.
TI Clinical and neuropathological features of CJD patients treated with Quinacrine in France
QU International Conference - Prion diseases: from basic research to intervention concepts - TSE-Forum, 08.10.-10.10.2003, Gasteig, München - Poster session - IV-26
PT Konferenz-Poster
AB After Quinacrine was reported as an antiprion agent in cell lines chronically infected with scrapie and the announcement, in August 2001, of a potential effect of this drug in a case of variant Creutzfeldt-Jakob disease (vCJD) a therapeutic compassionate procedure was initiated in France. All forms of probable CJD were included. A loading dose of 1000 mg per os was delivered in the first 24 hours, followed by a daily dose of 100 mg third a day. Of the 30 sporadic CJD treated cases, mean duration between disease onset and treatment was 6.4 months (range: 1 to 17). Mean treatment duration was 36 days (range: 1 to 265). No obvious clinical improvement was observed. The mean survival time (9 months) of the group of treated patients was not statistically different from the one (8,4 months) of a group of non-treated patients matched on age, codon 129 polymorphism of PRNP and period of notification. Neuropathological investigation could be performed for 11 patients. Profile and severity of lesions of 7 patients treated longer than 30 days were compared with those of a non-treated control group. The pattern of prion protein deposit and the profile of the accumulated abnormal prion protein were also studied by immunohistochemistry and Western blot respectively. The data of two autopsied vCJD patients included in this study will be also presented. Keeping in mind the methodological limits of such an open compassionate procedure, our study does not provide any evidence for an effect of Quinacrine on clinical course or neuropathological pattern of CJD. However, the interval between first symptoms and treatment was long compared to the survival time. This strengthens the importance of developing early diagnosis markers in order to better examine the efficiency of future therapeutic strategies.
AD S. Haïk, V. Sazdovitch, F. Fierville, B.A. Faucheux, J.J. Hauw, Laboratoire de Neuropathologie R Escourolle, G.H. Pitié-Salpêtrière, Paris, France; S. Haïk, J.P. Brandel, D. Salomon, V. Sazdovitch, N. Delasnerie-Lauprêtre, F. Fierville, B.A. Faucheux, J.J. Hauw, A. Alpérovitch, INSERM U360, G.H. Pitié-Salpêtrière, Paris, France; J.P. Brandel, Cellule Nationale de Référence de la MCJ, G.H. Pitié-Salpêtrière, Paris, France; J.L. Laplanche, Laboratoire Central de Biochimie, Hôpital Lariboisière, Paris, France
SP englisch
PO Deutschland