title: Individualized therapy for persistent asthma in young children.
abstract: Phenotypic presentations in young children with asthma are varied and might contribute to differential responses to asthma controller medications.
  
  
  
  The Individualized Therapy for Asthma in Toddlers study was a multicenter, randomized, double-blind, double-dummy clinical trial in children aged 12 to 59 months (n = 300) with asthma necessitating treatment with daily controller (Step 2) therapy. Participants completed a 2- to 8-week run-in period followed by 3 crossover periods with daily inhaled corticosteroids (ICSs), daily leukotriene receptor antagonists, and as-needed ICS treatment coadministered with albuterol. The primary outcome was differential response to asthma medication based on a composite measure of asthma control. The primary analysis involved 2 stages: determination of differential response and assessment of whether 3 prespecified features (aeroallergen sensitization, previous exacerbations, and sex) predicted a differential response.
  
  
  
  Seventy-four percent (170/230) of children with analyzable data had a differential response to the 3 treatment strategies. Within differential responders, the probability of best response was highest for a daily ICS and was predicted by aeroallergen sensitization but not exacerbation history or sex. The probability of best response to daily ICS was further increased in children with both aeroallergen sensitization and blood eosinophil counts of 300/μL or greater. In these children daily ICS use was associated with more asthma control days and fewer exacerbations compared with the other treatments.
  
  
  
  In young children with asthma necessitating Step 2 treatment, phenotyping with aeroallergen sensitization and blood eosinophil counts is useful for guiding treatment selection and identifies children with a high exacerbation probability for whom treatment with a daily ICS is beneficial despite possible risks of growth suppression.
authors: Emory University, Department of Pediatrics, Atlanta, GA.
authors: Anne M
authors: AM
authors: Fitzpatrick
authors: University of Wisconsin School of Medicine and Public Health, Pediatrics Section of Allergy, Immunology and Rheumatology, Madison, WI.
authors: Daniel J
authors: DJ
authors: Jackson
authors: Penn State University, College of Medicine, Department of Public Health Sciences, Hershey, PA.
authors: David T
authors: DT
authors: Mauger
authors: Penn State University, College of Medicine, Department of Public Health Sciences, Hershey, PA.
authors: Susan J
authors: SJ
authors: Boehmer
authors: Boston Children's Hospital, Division of Allergy/Immunology, Harvard Medical School, Boston, MA.
authors: Wanda
authors: W
authors: Phipatanakul
authors: Boston Children's Hospital, Division of Allergy/Immunology, Harvard Medical School, Boston, MA.
authors: William J
authors: WJ
authors: Sheehan
authors: Stroger Hospital of Cook County, Department of Pediatrics, Rush University Medical Center, Chicago, IL.
authors: James N
authors: JN
authors: Moy
authors: Penn State University, College of Medicine, Department of Pediatrics, Hershey, PA.
authors: Ian M
authors: IM
authors: Paul
authors: Washington University in St. Louis School of Medicine and St. Louis Children's Hospital, Department of Pediatrics. St Louis, MO.
authors: Leonard B
authors: LB
authors: Bacharier
authors: University of California - San Francisco, Medicine, San Francisco, CA.
authors: Michael D
authors: MD
authors: Cabana
authors: The University of Pittsburgh Asthma Institute at UPMC/UPSOM, Pittsburgh, PA.
authors: Ronina
authors: R
authors: Covar
authors: University of Wisconsin School of Medicine and Public Health, Pediatrics, Madison, WI.
authors: Fernando
authors: F
authors: Holguin
authors: University of Arizona, Arizona Respiratory Center, Tuscon, AZ.
authors: Robert F
authors: RF
authors: Lemanske
authors: Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL.
authors: Fernando D
authors: FD
authors: Martinez
authors: Washington University School of Medicine and St. Louis Children's Hospital, Department of Pediatrics. St Louis, MO.
authors: Jacqueline A
authors: JA
authors: Pongracic
authors: Boston Children's Hospital, Division of Allergy/Immunology, Harvard Medical School, Boston, MA.
authors: Avraham
authors: A
authors: Beigelman
authors: University of California San Francisco Benioff Children's Hospital Oakland, Oakland, CA.
authors: Sachin N
authors: SN
authors: Baxi
authors: Nemours Children's Health System, Jacksonville, FL.
authors: Mindy
authors: M
authors: Benson
authors: Department of Pediatrics, Case Western Reserve University School of Medicine, Rainbow Babies and Children's Hospital, Cleveland, OH.
authors: Kathryn
authors: K
authors: Blake
authors: National Jewish Health, Pediatrics, Denver, CO.
authors: James F
authors: JF
authors: Chmiel
authors: University of Arizona, Arizona Respiratory Center, Tuscon, AZ.
authors: Cori L
authors: CL
authors: Daines
authors: University of Arizona, Arizona Respiratory Center, Tucson, AZ.
authors: Michael O
authors: MO
authors: Daines
authors: Boston Children's Hospital, Division of Respiratory Diseases, Harvard Medical School, Boston, MA.
authors: Jonathan M
authors: JM
authors: Gaffin
authors: Allegheny General Hospital, Department of Pediatrics, Pittsburgh, PA.
authors: Deborah Ann
authors: DA
authors: Gentile
authors: Wake Forest School of Medicine, Winston-Salem, NC.
authors: W Adam
authors: WA
authors: Gower
authors: Brigham & Women's Hospital, Harvard Medical School, Boston, MA.
authors: Elliot
authors: E
authors: Israel
authors: University of Illinois at Chicago, Chicago, IL.
authors: Harsha Vardhan
authors: HV
authors: Kumar
authors: Nemours Children's Hospital, University of Central Florida College of Medicine, Orlando, FL.
authors: Jason E
authors: JE
authors: Lang
authors: University of California, San Francisco, Medicine, San Francisco, CA.
authors: Stephen C
authors: SC
authors: Lazarus
authors: Nemours Children's Health System, Jacksonville, FL.
authors: John J
authors: JJ
authors: Lima
authors: University of California - San Francisco, Airway Clinical Research Center, San Francisco, CA.
authors: Ngoc
authors: N
authors: Ly
authors: University of California San Francisco Benioff Children's Hospital Oakland, Oakland CA.
authors: Jyothi
authors: J
authors: Marbin
authors: University of Arizona, Arizona Respiratory Center, Tucson, AZ.
authors: Wayne
authors: W
authors: Morgan
authors: Rainbow Babies and Children's Hospital, Cleveland, OH.
authors: Ross E
authors: RE
authors: Myers
authors: National Jewish Health, Department of Pediatrics, Denver, CO.
authors: J Tod
authors: JT
authors: Olin
authors: Wake Forest University School of Medicine, Winston-Salem, NC.
authors: Stephen P
authors: SP
authors: Peters
authors: University of New Mexico, Pediatrics / Pulmonary, Albuquerque, NM.
authors: Hengameh H
authors: HH
authors: Raissy
authors: Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL.
authors: Rachel G
authors: RG
authors: Robison
authors: Department of Pediatrics, Case Western Reserve University School of Medicine, Rainbow Babies and Children's Hospital, Cleveland, OH.
authors: Kristie
authors: K
authors: Ross
authors: University of Wisconsin-Madison, Madison, WI.
authors: Christine A
authors: CA
authors: Sorkness
authors: Olive View-UCLA Medical Center, Department of Pediatrics, David Geffen School of Medicine at UCLA, Los Angeles, CA.
authors: Shannon M
authors: SM
authors: Thyne
authors: Children's Hospital Colorado, The Breathing Institute, and University of Colorado School of Medicine, Aurora, CO.
authors: Stanley J
authors: SJ
authors: Szefler
mesh_terms: D000280:Administration, Inhalation; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D000420:Albuterol / Q000627:therapeutic use; D001249:Asthma / Q000188:drug therapy*; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D020024:Leukotriene Antagonists / Q000627:therapeutic use*; D008297:Male; D057285:Precision Medicine; D012008:Recurrence; D016896:Treatment Outcome; D014481:United States
chemical_list: D000305:Adrenal Cortex Hormones; D020024:Leukotriene Antagonists; D000420:Albuterol
keywords: Asthma; asthma biomarkers; asthma phenotype; asthma treatment; inhaled corticosteroid; leukotriene receptor antagonist; personalized medicine; treatment response