An orally administered, fixed-dose coformulation of sodium phenylbutyratetaurursodiol (PB-TURSO) significantly slowed functional decline in a randomized, placebo-controlled, phase 2 trial in ALS (CENTAUR). Herein we report results of a long-term survival analysis of participants in CENTAUR. In CENTAUR, adults with ALS were randomized 2:1 to PB-TURSO or placebo. Participants completing the 6-month (24-week) randomized phase were eligible to receive PB-TURSO in the open-label extension. An all-cause mortality analysis (35-month maximum follow-up post-randomization) incorporated all randomized participants. Participants and site investigators were blinded to treatment assignments through the duration of followup of this analysis. Vital status was obtained for 135 of 137 participants originally randomized in CENTAUR. Median overall survival was 25.0 months among participants originally randomized to PB-TURSO and 18.5 months among those originally randomized to placebo (hazard ratio, 0.56; 95% confidence interval, 0.34-0.92; P = .023). Initiation of PB-TURSO treatment at baseline resulted in a 6.5-month longer median survival as compared with placebo. Combined with results from CEN-TAUR, these results suggest that PB-TURSO has both functional and survival benefits in ALS.

Introduction: Peritoneal dialysis (PD) is reported to be underused in the autosomal dominant polycystic kidney disease (ADPKD) population because doctors fear technical failure caused by reduced abdominal space and high intraperitoneal pressure (IPP). Methods:We designed a multicenter retrospective study to be carried out in 15 French centers recruiting 60 patients with ADPKD treated with PD to identify factors associated with IPP. Inclusion criteria were start of PD between 2010 and 2017, available tomodensitometry, and IPP measurement in the first year of dialysis. The clinical and radiological data for each patient were reviewed by the same operator. Total kidney volume (TKV), liver volume, and the volume of the abdominal cavity were measured using contouring.Results: TKV and the volume of the abdominal cavity in women and men were, respectively, 2397 ml versus 3758 ml and 9402 ml versus 12,920 ml. In the univariate analysis, IPP was significantly and positively associated with body surface area (P ¼ 0.0024), body mass index (BMI) (P < 0.0001), the volume of the abdominal cavity (P ¼ 0.0005), and the volume of the dialysate infused in the peritoneal cavity (IPV) (P ¼ 0.0108). In the multivariate analysis, only BMI was still significantly associated with IPP (P ¼ 0.0004) Conclusions: Our results identified BMI as the main factor linked to IPP in patients with ADPKD. Despite a reliable assessment of the volume of their organs we did not find any correlation between liver and kidney volumes and IPP. To our knowledge, this is the first study designed to identify factors associated with IPP in patients with ADPKD on PD.

Concern has been expressed recently regarding the proliferation of angioplasty and/or stenting of cerebral vessels. However, little is known about the volume of angioplasties being performed or the number of experienced interventionalists. A questionnaire was mailed to directors of accredited radiology residency programs in the United States, to define the level of expertise available at teaching hospitals in terms of angioplasty and/or stenting. Of 200 programs surveyed, 111 responded (56 %). Of 111 program directors 47 (42%) indicated that cerebral angioplasty was being performed at their center. The greatest experience is currently for angioplasty of post-subarachnoid hemorrhage vasospasm (mean 16 procedures performed) and the least experience for dilation of basilar artery atherosclerosis (mean five procedures performed). The reported stroke and/or death rate in centers performing angioplasty of the extracranial carotid system is 1.5 %. Comparisons with other medical specialties (e.g., cardiologists, neurologists, neurosurgeons) are necessary to determine the full scope of extracranial neurovascular procedures being performed and the corresponding complication rates.

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