PMID- 37288719
OWN - NLM
STAT- MEDLINE
DCOM- 20230814
LR  - 20230817
IS  - 1478-3231 (Electronic)
IS  - 1478-3223 (Linking)
VI  - 43
IP  - 9
DP  - 2023 Sep
TI  - Efficacy and safety of treatment with sofosbuvir/velpatasvir in patients aged 
      6-18 years with chronic hepatitis C-Results of the PANDAA-PED study.
PG  - 1871-1878
LID - 10.1111/liv.15637 [doi]
AB  - BACKGROUND AND AIMS: The aim of this non-commercial, open-label, real-life, 
      non-randomized clinical trial was to analyse the efficacy and safety of a 
      pangenotypic regimen sofosbuvir/velpatasvir (SOF/VEL) in patients aged 6-18 years 
      with chronic hepatitis C virus (HCV) infection. METHODS: Fifty patients qualified 
      for the 12-week treatment were divided into two weight groups: 15 children 
      weighting between 17 and <30 kg received a fixed dose of 200/50 mg of SOF/VEL 
      (tablet) once daily, and 35 patients weighting >/=30 kg were treated with 
      400/100 mg SOF/VEL. The primary endpoint of the study was efficacy defined as 
      sustained viral response (undetectable HCV RNA using an real-time polymerase 
      chain reaction method) at 12 weeks posttreatment (SVR12). RESULTS: Median age of 
      the participants was 10 (IQR 8-12) years, 47 were infected vertically, and 3 
      patients were previously ineffectively treated with pegylated interferon and 
      ribavirin. Thirty-seven participants were infected with HCV genotype 1, 10 with 
      HCV genotype 3 and the remaining 3 with genotype 4. There was no case of 
      cirrhosis. SVR12 was 100%. Thirty-three reported adverse events (AEs) were 
      considered related to the administration of SOF/VEL, all of them were mild or 
      moderate. Children presenting with AEs were older compared to these without AEs: 
      12 (9.5-13) versus 9 (IQR 8-11) years (p = 0.008). CONCLUSIONS: Results of the 
      PANDAA-PED study indicated a 100% effectiveness of a 12-week therapy with SOF/VEL 
      in children aged 6-18 years with chronic HCV infection and its good safety 
      profile, in particular in younger patients.
CI  - (c) 2023 The Authors. Liver International published by John Wiley & Sons Ltd.
FAU - Pokorska-Spiewak, Maria
AU  - Pokorska-Spiewak M
AUID- ORCID: 0000-0001-7783-6904
AD  - Department of Children's Infectious Diseases, Medical University of Warsaw, 
      Warsaw, Poland.
AD  - Department of Pediatric Infectious Diseases, Regional Hospital of Infectious 
      Diseases in Warsaw, Warsaw, Poland.
FAU - Talarek, Ewa
AU  - Talarek E
AD  - Department of Children's Infectious Diseases, Medical University of Warsaw, 
      Warsaw, Poland.
AD  - Department of Pediatric Infectious Diseases, Regional Hospital of Infectious 
      Diseases in Warsaw, Warsaw, Poland.
FAU - Aniszewska, Malgorzata
AU  - Aniszewska M
AD  - Department of Children's Infectious Diseases, Medical University of Warsaw, 
      Warsaw, Poland.
AD  - Department of Pediatric Infectious Diseases, Regional Hospital of Infectious 
      Diseases in Warsaw, Warsaw, Poland.
FAU - Pluta, Magdalena
AU  - Pluta M
AD  - Department of Children's Infectious Diseases, Medical University of Warsaw, 
      Warsaw, Poland.
AD  - Department of Pediatric Infectious Diseases, Regional Hospital of Infectious 
      Diseases in Warsaw, Warsaw, Poland.
FAU - Dobrzeniecka, Anna
AU  - Dobrzeniecka A
AD  - Department of Pediatric Infectious Diseases, Regional Hospital of Infectious 
      Diseases in Warsaw, Warsaw, Poland.
FAU - Marczynska, Magdalena
AU  - Marczynska M
AD  - Department of Children's Infectious Diseases, Medical University of Warsaw, 
      Warsaw, Poland.
AD  - Department of Pediatric Infectious Diseases, Regional Hospital of Infectious 
      Diseases in Warsaw, Warsaw, Poland.
FAU - Indolfi, Giuseppe
AU  - Indolfi G
AUID- ORCID: 0000-0003-3830-9823
AD  - Department of Neurofarba, Meyer Children's University of Florence, Florence, 
      Italy.
LA  - eng
PT  - Clinical Trial
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20230608
PL  - United States
TA  - Liver Int
JT  - Liver international : official journal of the International Association for the 
      Study of the Liver
JID - 101160857
RN  - WJ6CA3ZU8B (Sofosbuvir)
RN  - KCU0C7RS7Z (velpatasvir)
RN  - 0 (Antiviral Agents)
RN  - 0 (Heterocyclic Compounds, 4 or More Rings)
SB  - IM
MH  - Child
MH  - Humans
MH  - *Sofosbuvir/adverse effects
MH  - *Hepatitis C, Chronic/drug therapy
MH  - Antiviral Agents/adverse effects
MH  - Treatment Outcome
MH  - Heterocyclic Compounds, 4 or More Rings/adverse effects
MH  - Hepacivirus/genetics
MH  - Genotype
MH  - Sustained Virologic Response
OTO - NOTNLM
OT  - children
OT  - direct acting antivirals
OT  - hepatitis C virus
OT  - sofosbuvir/velpatasvir
EDAT- 2023/06/08 13:08
MHDA- 2023/08/14 06:42
CRDT- 2023/06/08 06:33
PHST- 2023/05/15 00:00 [revised]
PHST- 2023/03/15 00:00 [received]
PHST- 2023/05/26 00:00 [accepted]
PHST- 2023/08/14 06:42 [medline]
PHST- 2023/06/08 13:08 [pubmed]
PHST- 2023/06/08 06:33 [entrez]
AID - 10.1111/liv.15637 [doi]
PST - ppublish
SO  - Liver Int. 2023 Sep;43(9):1871-1878. doi: 10.1111/liv.15637. Epub 2023 Jun 8.