PMID- 35951014
OWN - NLM
STAT- MEDLINE
DCOM- 20221011
LR  - 20221029
IS  - 1530-0366 (Electronic)
IS  - 1098-3600 (Linking)
VI  - 24
IP  - 10
DP  - 2022 Oct
TI  - Safety and preliminary efficacy on cognitive performance and adaptive 
      functionality of epigallocatechin gallate (EGCG) in children with Down syndrome. 
      A randomized phase Ib clinical trial (PERSEUS study).
PG  - 2004-2013
LID - S1098-3600(22)00820-6 [pii]
LID - 10.1016/j.gim.2022.06.011 [doi]
AB  - PURPOSE: Although some caregivers are using epigallocatechin gallate (EGCG) off 
      label in hopes of improving cognition in young adults with Down syndrome (DS), 
      nothing is known about its safety, tolerability, and efficacy in the DS pediatric 
      population. We aimed to evaluate safety and tolerability of a dietary supplement 
      containing EGCG and if EGCG improves cognitive and functional performance. 
      METHODS: A total of 73 children with DS (aged 6-12 years) were randomized. 
      Participants received 0.5% EGCG (10 mg/kg daily dose) or placebo for 6 months 
      with 3 months follow up after treatment discontinuation. RESULTS: In total, 72 
      children were treated and 66 completed the study. A total of 38 participants were 
      included in the EGCG group and 35 in the placebo group. Of 72 treated 
      participants, 62 (86%) had 229 treatment-emergent adverse events (AEs). Of 37 
      participants in the EGCG group, 13 (35%) had 18 drug-related treatment-emergent 
      AEs and 12 of 35 (34%) from the placebo group had 22 events. In the EGCG group, 
      neither severe AEs nor increase in the incidence of AEs related to safety 
      biomarkers were observed. Cognition and functionality were not improved compared 
      with placebo. Secondary efficacy outcomes in girls point to a need for future 
      work. CONCLUSION: The use of EGCG is safe and well-tolerated in children with DS, 
      but efficacy results do not support its use in this population.
CI  - Copyright (c) 2022 The Authors. Published by Elsevier Inc. All rights reserved.
FAU - Cieuta-Walti, Cecile
AU  - Cieuta-Walti C
AD  - Institut Jerome Lejeune, Paris, France; Sherbrooke University, Quebec, Canada.
FAU - Cuenca-Royo, Aida
AU  - Cuenca-Royo A
AD  - Integrative Pharmacology and Systems Neuroscience research group, IMIM-Hospital 
      del Mar Medical Research Institute, Barcelona, Spain.
FAU - Langohr, Klaus
AU  - Langohr K
AD  - Integrative Pharmacology and Systems Neuroscience research group, IMIM-Hospital 
      del Mar Medical Research Institute, Barcelona, Spain; Department of Statistics 
      and Operations Research, Universitat Politecnica de Catalunya/BarcelonaTECH, 
      Barcelona, Spain.
FAU - Rakic, Claire
AU  - Rakic C
AD  - Institut Jerome Lejeune, Paris, France.
FAU - Lopez-Vilchez, Ma Angeles
AU  - Lopez-Vilchez MA
AD  - Pediatric Sevice, IMIM-Hospital del Mar Medical Research Institute, Barcelona, 
      Spain.
FAU - Lirio, Julian
AU  - Lirio J
AD  - Social Pediatrics Division, Hospital Infantil Universitario Nino Jesus, Madrid, 
      Spain.
FAU - Gonzalez-Lamuno Leguina, Domingo
AU  - Gonzalez-Lamuno Leguina D
AD  - Division of Pediatrics, Hospital Universitario Marques de Valdecilla - 
      Universidad de Cantabria - Instituto de Investigacion Valdecilla (IDIVAL), 
      Santander, Cantabria, Spain.
FAU - Gonzalez, Teresa Bermejo
AU  - Gonzalez TB
AD  - Instituto Hispalense de Pediatria, Sevilla, Spain.
FAU - Garcia, Jordi Garcia
AU  - Garcia JG
AD  - Pediatric Sevice, IMIM-Hospital del Mar Medical Research Institute, Barcelona, 
      Spain.
FAU - Roure, Maria Rimblas
AU  - Roure MR
AD  - Division of Pediatrics, Hospital Universitari Sant Joan de Reus, Tarragona, 
      Spain.
FAU - Aldea-Perona, Ana
AU  - Aldea-Perona A
AD  - Clinical Research Unit, IMIM- Hospital del Mar Medical Research Institute, 
      Barcelona, Spain.
FAU - Forcano, Laura
AU  - Forcano L
AD  - Integrative Pharmacology and Systems Neuroscience research group, IMIM-Hospital 
      del Mar Medical Research Institute, Barcelona, Spain.
FAU - Gomis-Gonzalez, Maria
AU  - Gomis-Gonzalez M
AD  - Integrative Pharmacology and Systems Neuroscience research group, IMIM-Hospital 
      del Mar Medical Research Institute, Barcelona, Spain.
FAU - Ces, Sebastia Videla
AU  - Ces SV
AD  - Clinical Research Support Unit [HUB.IDIBELL], Clinical Pharmacology Department, 
      Bellvitge University Hospital, Barcelona, Spain; Pharmacology Unit, Department of 
      Pathology and Experimental Therapeutics, School of Medicine and Health Sciences, 
      IDIBELL, University of Barcelona, Barcelona, Spain.
FAU - Lacaille, Florence
AU  - Lacaille F
AD  - Service de Gastro-enterologie-Hepatologie-Nutrition Pediatriques, Hopital 
      Necker-Enfants Malades Paris, Paris, France.
FAU - Ravel, Aime
AU  - Ravel A
AD  - Institut Jerome Lejeune, Paris, France.
FAU - Mircher, Clotilde
AU  - Mircher C
AD  - Institut Jerome Lejeune, Paris, France.
FAU - Walti, Herve
AU  - Walti H
AD  - Institut Jerome Lejeune, Paris, France.
FAU - Janel, Nathalie
AU  - Janel N
AD  - Unite de Biologie Fonctionnelle et Adaptative (BFA), Laboratoire Processus 
      degeneratifs, Stress et Vieillissement, UMR 8251, CNRS, Universite de Paris, 
      Paris, France.
FAU - Dairou, Julien
AU  - Dairou J
AD  - UMR 8601 CNRS, Laboratoire de Chimie et Biochimie Pharmacologiques et 
      Toxicologiques, Universite de Paris, Paris, France.
FAU - Levy, Marilyne
AU  - Levy M
AD  - Centre de Reference Malformations Cardiaques Congenitales Complexes, M3C-Necker, 
      Hopital Universitaire Necker-Enfants Malades, APHP, Universite de Paris, Paris, 
      France.
FAU - Durand, Sophie
AU  - Durand S
AD  - Institut Jerome Lejeune, Paris, France.
FAU - Dierssen, Mara
AU  - Dierssen M
AD  - Cellular & Systems Neurobiology, Systems Biology Program. Center for Genomic 
      Regulation, Barcelona, Spain.
FAU - Sacco, Silvia
AU  - Sacco S
AD  - Institut Jerome Lejeune, Paris, France.
FAU - Fornell, Rafael de la Torre
AU  - Fornell RT
AD  - Sherbrooke University, Quebec, Canada; Department of Experimental and Health 
      Sciences, Universitat Pompeu Fabra, Barcelona, Spain. Electronic address: 
      rtorre@imim.es.
CN  - PERSEUS Study Group
LA  - eng
PT  - Clinical Trial, Phase I
PT  - Journal Article
PT  - Randomized Controlled Trial
DEP - 20220811
PL  - United States
TA  - Genet Med
JT  - Genetics in medicine : official journal of the American College of Medical 
      Genetics
JID - 9815831
RN  - 8R1V1STN48 (Catechin)
RN  - BQM438CTEL (epigallocatechin gallate)
SB  - IM
MH  - *Catechin/adverse effects/analogs & derivatives
MH  - Child
MH  - Cognition
MH  - Dietary Supplements
MH  - Double-Blind Method
MH  - *Down Syndrome/drug therapy
MH  - Female
MH  - Humans
MH  - Male
OTO - NOTNLM
OT  - Children
OT  - Cognition
OT  - Down syndrome
OT  - EGCG
OT  - Safety
COIS- Conflict of Interest The authors declare no conflicts of interest.
FIR - Guerrico, Ion Alvarez
IR  - Guerrico IA
FIR - Goday, Albert
IR  - Goday A
FIR - Llop, Cristina
IR  - Llop C
FIR - Mateus, Julian
IR  - Mateus J
FIR - Matilla, Iris
IR  - Matilla I
FIR - Esther
IR  - Esther
FIR - Menoyo
IR  - Menoyo
FIR - Hernandez-Ligero, Lorena
IR  - Hernandez-Ligero L
FIR - Perez, Marta
IR  - Perez M
FIR - Pizarro, Neus
IR  - Pizarro N
FIR - Principe, Alessandro
IR  - Principe A
FIR - Cortesi, Anna
IR  - Cortesi A
FIR - Gomez, Alba
IR  - Gomez A
FIR - Paredes, Noelia
IR  - Paredes N
FIR - Gomez, Cristina
IR  - Gomez C
FIR - Graell, Montserrat
IR  - Graell M
FIR - Lopez, Pilar
IR  - Lopez P
FIR - Lozano, Rosario
IR  - Lozano R
FIR - Medrano, Constancio
IR  - Medrano C
FIR - Andrade-Navarro, Maria Teresa
IR  - Andrade-Navarro MT
FIR - Daza, Gema
IR  - Daza G
FIR - Garcia, Sandra
IR  - Garcia S
FIR - Gayan, Ma Luisa
IR  - Gayan ML
FIR - Guerra-Colorado, Cristina
IR  - Guerra-Colorado C
FIR - Luna, Beatriz
IR  - Luna B
FIR - Maya, Enriqueta
IR  - Maya E
FIR - Millan, Delia
IR  - Millan D
FIR - Reina, Macarena
IR  - Reina M
FIR - Amigo, Maria Teresa
IR  - Amigo MT
FIR - Fernandez, Jose Luis
IR  - Fernandez JL
FIR - Garcia-Martinez, Maria
IR  - Garcia-Martinez M
FIR - Perez-Poyato, Maria Del Socorro
IR  - Perez-Poyato MDS
FIR - Pozueta, Ana
IR  - Pozueta A
FIR - Tato, Emma
IR  - Tato E
FIR - Viadero, Maria Teresa
IR  - Viadero MT
FIR - Akkaya, Magnolia
IR  - Akkaya M
FIR - Falquero, Segolene
IR  - Falquero S
FIR - Gambarini, Alicia
IR  - Gambarini A
FIR - Akkaya, Magnolia
IR  - Akkaya M
FIR - Falquero, Segolene
IR  - Falquero S
FIR - Gambarini, Alicia
IR  - Gambarini A
EDAT- 2022/08/12 06:00
MHDA- 2022/10/12 06:00
CRDT- 2022/08/11 11:03
PHST- 2022/03/14 00:00 [received]
PHST- 2022/06/27 00:00 [revised]
PHST- 2022/06/29 00:00 [accepted]
PHST- 2022/08/12 06:00 [pubmed]
PHST- 2022/10/12 06:00 [medline]
PHST- 2022/08/11 11:03 [entrez]
AID - S1098-3600(22)00820-6 [pii]
AID - 10.1016/j.gim.2022.06.011 [doi]
PST - ppublish
SO  - Genet Med. 2022 Oct;24(10):2004-2013. doi: 10.1016/j.gim.2022.06.011. Epub 2022 
      Aug 11.