PMID- 32516282
OWN - NLM
STAT- MEDLINE
DCOM- 20210715
LR  - 20221005
IS  - 1532-0987 (Electronic)
IS  - 0891-3668 (Print)
IS  - 0891-3668 (Linking)
VI  - 39
IP  - 8
DP  - 2020 Aug
TI  - Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: A 
      Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial 
      (miniSTONE-2).
PG  - 700-705
LID - 10.1097/INF.0000000000002747 [doi]
AB  - BACKGROUND: Baloxavir marboxil (baloxavir) is a novel, cap-dependent endonuclease 
      inhibitor that has previously demonstrated efficacy in the treatment of influenza 
      in adults and adolescents. We assessed the safety and efficacy of baloxavir in 
      otherwise healthy children with acute influenza. METHODS: MiniSTONE-2 
      (Clinicaltrials.gov: NCT03629184) was a double-blind, randomized, active 
      controlled trial enrolling children 1-<12 years old with a clinical diagnosis of 
      influenza. Children were randomized 2:1 to receive either a single dose of oral 
      baloxavir or oral oseltamivir twice daily for 5 days. The primary endpoint was 
      incidence, severity and timing of adverse events (AEs); efficacy was a secondary 
      endpoint. RESULTS: In total, 173 children were randomized and dosed, 115 to the 
      baloxavir group and 58 to the oseltamivir group. Characteristics of participants 
      were similar between treatment groups. Overall, 122 AEs were reported in 84 
      (48.6%) children. Incidence of AEs was similar between baloxavir and oseltamivir 
      groups (46.1% vs. 53.4%, respectively). The most common AEs were gastrointestinal 
      (vomiting/diarrhea) in both groups [baloxavir: 12 children (10.4%); oseltamivir: 
      10 children (17.2%)]. No deaths, serious AEs or hospitalizations were reported. 
      Median time (95% confidence interval) to alleviation of signs and symptoms of 
      influenza was similar between groups: 138.1 (116.6-163.2) hours with baloxavir 
      versus 150.0 (115.0-165.7) hours with oseltamivir. CONCLUSIONS: Oral baloxavir is 
      well tolerated and effective at alleviating symptoms in otherwise healthy 
      children with acute influenza. Baloxavir provides a new therapeutic option with a 
      simple oral dosing regimen.
FAU - Baker, Jeffrey
AU  - Baker J
AD  - From the Clinical Research Prime, Idaho Falls, Idaho.
FAU - Block, Stanley L
AU  - Block SL
AD  - Kentucky Pediatric and Adult Research Inc., Bardstown, Kentucky.
FAU - Matharu, Balpreet
AU  - Matharu B
AD  - F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.
FAU - Burleigh Macutkiewicz, Laura
AU  - Burleigh Macutkiewicz L
AD  - F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.
FAU - Wildum, Steffen
AU  - Wildum S
AD  - F. Hoffmann-La Roche Ltd, Basel, Switzerland.
FAU - Dimonaco, Sophie
AU  - Dimonaco S
AD  - F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.
FAU - Collinson, Neil
AU  - Collinson N
AD  - F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.
FAU - Clinch, Barry
AU  - Clinch B
AD  - F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.
FAU - Piedra, Pedro A
AU  - Piedra PA
AD  - Baylor College of Medicine, Houston, Texas.
LA  - eng
SI  - ClinicalTrials.gov/NCT03629184
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - Pediatr Infect Dis J
JT  - The Pediatric infectious disease journal
JID - 8701858
RN  - 0 (Antiviral Agents)
RN  - 0 (Dibenzothiepins)
RN  - 0 (Morpholines)
RN  - 0 (Pyridones)
RN  - 0 (Triazines)
RN  - 4G86Y4JT3F (baloxavir)
RN  - EC 3.1.- (Endonucleases)
SB  - IM
MH  - Acute Disease/therapy
MH  - Administration, Oral
MH  - Antiviral Agents/*administration & dosage/pharmacokinetics/*therapeutic use
MH  - Child
MH  - Child, Preschool
MH  - Dibenzothiepins/*administration & dosage/pharmacokinetics/*therapeutic use
MH  - Double-Blind Method
MH  - Drug Administration Schedule
MH  - Endonucleases/antagonists & inhibitors
MH  - Female
MH  - Global Health
MH  - Humans
MH  - Infant
MH  - Influenza, Human/*drug therapy
MH  - Male
MH  - Morpholines/*administration & dosage/pharmacokinetics/*therapeutic use
MH  - Pyridones/*administration & dosage/pharmacokinetics/*therapeutic use
MH  - Triazines/*administration & dosage/pharmacokinetics/*therapeutic use
PMC - PMC7360097
EDAT- 2020/06/10 06:00
MHDA- 2021/07/16 06:00
PMCR- 2020/07/14
CRDT- 2020/06/10 06:00
PHST- 2020/06/10 06:00 [pubmed]
PHST- 2021/07/16 06:00 [medline]
PHST- 2020/06/10 06:00 [entrez]
PHST- 2020/07/14 00:00 [pmc-release]
AID - 00006454-202008000-00012 [pii]
AID - 10.1097/INF.0000000000002747 [doi]
PST - ppublish
SO  - Pediatr Infect Dis J. 2020 Aug;39(8):700-705. doi: 10.1097/INF.0000000000002747.