Individualized therapy for persistent asthma in young children

National Institutes of Health/National Heart, Lung, and Blood Institute AsthmaNet

Research output: Contribution to journalArticle

78 Citations (Scopus)

Abstract

Background Phenotypic presentations in young children with asthma are varied and might contribute to differential responses to asthma controller medications. Methods 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. Results 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. Conclusions 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.

Original languageEnglish (US)
Pages (from-to)1608-1618.e12
JournalJournal of Allergy and Clinical Immunology
Volume138
Issue number6
DOIs
StatePublished - Dec 1 2016

Fingerprint

Asthma
Adrenal Cortex Hormones
Therapeutics
Eosinophils
Leukotriene Antagonists
Albuterol
History
Clinical Trials
Growth

Keywords

  • Asthma
  • asthma biomarkers
  • asthma phenotype
  • asthma treatment
  • inhaled corticosteroid
  • leukotriene receptor antagonist
  • personalized medicine
  • treatment response

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology

Cite this

National Institutes of Health/National Heart, Lung, and Blood Institute AsthmaNet (2016). Individualized therapy for persistent asthma in young children. Journal of Allergy and Clinical Immunology, 138(6), 1608-1618.e12. https://doi.org/10.1016/j.jaci.2016.09.028

Individualized therapy for persistent asthma in young children. / National Institutes of Health/National Heart, Lung, and Blood Institute AsthmaNet.

In: Journal of Allergy and Clinical Immunology, Vol. 138, No. 6, 01.12.2016, p. 1608-1618.e12.

Research output: Contribution to journalArticle

National Institutes of Health/National Heart, Lung, and Blood Institute AsthmaNet 2016, 'Individualized therapy for persistent asthma in young children', Journal of Allergy and Clinical Immunology, vol. 138, no. 6, pp. 1608-1618.e12. https://doi.org/10.1016/j.jaci.2016.09.028
National Institutes of Health/National Heart, Lung, and Blood Institute AsthmaNet. Individualized therapy for persistent asthma in young children. Journal of Allergy and Clinical Immunology. 2016 Dec 1;138(6):1608-1618.e12. https://doi.org/10.1016/j.jaci.2016.09.028
National Institutes of Health/National Heart, Lung, and Blood Institute AsthmaNet. / Individualized therapy for persistent asthma in young children. In: Journal of Allergy and Clinical Immunology. 2016 ; Vol. 138, No. 6. pp. 1608-1618.e12.
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abstract = "Background Phenotypic presentations in young children with asthma are varied and might contribute to differential responses to asthma controller medications. Methods 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. Results 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. Conclusions 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.",
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T1 - Individualized therapy for persistent asthma in young children

AU - National Institutes of Health/National Heart, Lung, and Blood Institute AsthmaNet

AU - Fitzpatrick, Anne M.

AU - Jackson, Daniel J.

AU - Mauger, David T.

AU - Boehmer, Susan J.

AU - Phipatanakul, Wanda

AU - Sheehan, William J.

AU - Moy, James N.

AU - Paul, Ian M.

AU - Bacharier, Leonard B.

AU - Cabana, Michael D.

AU - Covar, Ronina

AU - Holguin, Fernando

AU - Lemanske, Robert F.

AU - Martinez, Fernando

AU - Pongracic, Jacqueline A.

AU - Beigelman, Avraham

AU - Baxi, Sachin N.

AU - Benson, Mindy

AU - Blake, Kathryn

AU - Chmiel, James F.

AU - Daines, Cori L

AU - Daines, Michael O

AU - Gaffin, Jonathan M.

AU - Gentile, Deborah Ann

AU - Gower, W. Adam

AU - Israel, Elliot

AU - Kumar, Harsha Vardhan

AU - Lang, Jason E.

AU - Lazarus, Stephen C.

AU - Lima, John J.

AU - Ly, Ngoc

AU - Marbin, Jyothi

AU - Morgan, Wayne J

AU - Myers, Ross E.

AU - Olin, J. Tod

AU - Peters, Stephen P.

AU - Raissy, Hengameh H.

AU - Robison, Rachel G.

AU - Ross, Kristie

AU - Sorkness, Christine A.

AU - Thyne, Shannon M.

AU - Szefler, Stanley J.

PY - 2016/12/1

Y1 - 2016/12/1

N2 - Background Phenotypic presentations in young children with asthma are varied and might contribute to differential responses to asthma controller medications. Methods 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. Results 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. Conclusions 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.

AB - Background Phenotypic presentations in young children with asthma are varied and might contribute to differential responses to asthma controller medications. Methods 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. Results 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. Conclusions 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.

KW - Asthma

KW - asthma biomarkers

KW - asthma phenotype

KW - asthma treatment

KW - inhaled corticosteroid

KW - leukotriene receptor antagonist

KW - personalized medicine

KW - treatment response

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