title: Randomised clinical trial: the efficacy and safety of propionyl-L-carnitine therapy in patients with ulcerative colitis receiving stable oral treatment.
abstract: BACKGROUND
  Ulcerative colitis (UC) is characterised by impaired fatty-acid oxidation; l-carnitine has a key role in fatty-acid metabolism and short-chain fatty acids such as butyrate and propionate are important energy source for intestinal epithelial cells.
  
  
  OBJECTIVE
  To evaluate efficacy and safety of colon-release propionyl-L-carnitine (PLC) in patients with mild-to-moderate UC receiving stable oral aminosalicylate or thiopurine therapy.
  
  
  METHODS
  In a multicentre, phase II, double-blind, parallel-group trial, patients were randomised to receive PLC 1 g/day, PLC 2 g/day or placebo. Main inclusion criteria were as follows: age 18-75; disease activity index (DAI) score 3-10 inclusive, be under oral stable treatment with aminosalicylate or thiopurine. The primary endpoint was clinical/endoscopic response, defined as a decrease in DAI score ≥ 3 points or remission, defined as a DAI score ≤ 2 with no individual sub-score > 1.
  
  
  RESULTS
  Of 121 patients who were randomised, 57 of 79 (72%) patients receiving PLC (combined 1 g and 2 g cohort) had a clinical/endoscopic response vs. 20 of 40 (50%) receiving placebo (P = 0.02). Specifically, in PLC 1 g/day group, 30 of 40 (75%) patients had clinical/endoscopic response (P = 0.02 vs. placebo) and 27 of 39 (69%) in the PLC 2 g/day group (P = 0.08 vs. placebo). Rates of remission were 22/40 (55%), 19/39 (49%), 14/40 (35%) in the PLC 1 g, PLC 2 g, and placebo groups, respectively. PLC had a similar safety profile to placebo; the most common adverse events were gastrointestinal.
  
  
  CONCLUSIONS
  Propionyl-L-carnitine 1 g/day should be investigated further as a co-treatment for mild-to-moderate ulcerative colitis (NCT-01026857).
authors.affiliation: State Scientific Centre of Coloproctology, Rosmedtechnology, Moscow, Russia.
authors.forename: T L
authors.initials: TL
authors.lastname: Mikhailova
authors.forename: E
authors.initials: E
authors.lastname: Sishkova
authors.forename: E
authors.initials: E
authors.lastname: Poniewierka
authors.forename: K P
authors.initials: KP
authors.lastname: Zhidkov
authors.forename: I G
authors.initials: IG
authors.lastname: Bakulin
authors.forename: L
authors.initials: L
authors.lastname: Kupcinskas
authors.forename: K
authors.initials: K
authors.lastname: Lesniakowski
authors.forename: V B
authors.initials: VB
authors.lastname: Grinevich
authors.forename: E
authors.initials: E
authors.lastname: Malecka-Panas
authors.forename: S
authors.initials: S
authors.lastname: Ardizzone
authors.forename: A
authors.initials: A
authors.lastname: D'Arienzo
authors.forename: D
authors.initials: D
authors.lastname: Valpiani
authors.forename: M
authors.initials: M
authors.lastname: Koch
authors.forename: G
authors.initials: G
authors.lastname: Denapiene
authors.forename: G
authors.initials: G
authors.lastname: Vago
authors.forename: P
authors.initials: P
authors.lastname: Fociani
authors.forename: P
authors.initials: P
authors.lastname: Zerbi
authors.forename: M
authors.initials: M
authors.lastname: Ceracchi
authors.forename: R
authors.initials: R
authors.lastname: Camerini
authors.forename: G
authors.initials: G
authors.lastname: Gasbarrini
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000008:administration & dosage* / Q000009:adverse effects; D002331:Carnitine / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D015331:Cohort Studies; D003093:Colitis, Ulcerative / Q000188:drug therapy*; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D012720:Severity of Illness Index; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000894:Anti-Inflammatory Agents, Non-Steroidal; C003223:propionylcarnitine; D002331:Carnitine