RT Journal Article
SR Electronic
T1 Doxycycline in early CJD: a double-blinded randomised phase II and observational study
JF Journal of Neurology, Neurosurgery & Psychiatry
JO J Neurol Neurosurg Psychiatry
FD BMJ Publishing Group Ltd
SP 119
OP 125
DO 10.1136/jnnp-2016-313541
VO 88
IS 2
A1 Daniela Varges
A1 Henrike Manthey
A1 Uta Heinemann
A1 Claudia Ponto
A1 Matthias Schmitz
A1 Walter J Schulz-Schaeffer
A1 Anna Krasnianski
A1 Maren Breithaupt
A1 Fabian Fincke
A1 Katharina Kramer
A1 Tim Friede
A1 Inga Zerr
YR 2017
UL http://jnnp.bmj.com/content/88/2/119.abstract
AB Objectives The main objective of the present study is to study the therapeutic efficiency of doxycycline in a double-blinded randomised phase II study in a cohort of patients with sporadic Creutzfeldt-Jakob disease (sCJD).Methods From the National Reference Center of TSE Surveillance in Germany, patients with probable or definite sCJD were recruited for a double-blinded randomised study with oral doxycycline (EudraCT 2006-003934-14). In addition, we analysed the data from patients with CJD who received compassionate treatment with doxycycline in a separate group. Potential factors which influence survival such as age at onset, gender, codon 129 polymorphism and cognitive functions were evaluated. The primary outcome measure was survival.Results Group 1: in the double-blinded randomised phase II study, 7 patients in the treatment group were compared with 5 controls. Group 2: 55 patients with sCJD treated with oral doxycycline were analysed and compared with 33 controls by a stratified propensity score applied to a Cox proportional hazard analysis. The results of both studies were combined by means of a random-effects meta-analysis. A slight increase in survival time in the doxycycline treatment group was observed (p=0.049, HR=0.63 (95% CI 0.402 to 0.999)).Conclusions On the basis of our studies, a larger trial of doxycycline should be performed in persons in the earliest stages of CJD.Trial registration number EudraCT 2006-003934-14; Results.