Related Experiment Video
Updated: Oct 4, 2025
Bronchial Thermoplasty: A Novel Therapeutic Approach to Severe Asthma
Published on: November 4, 2010
Treatment by biomarker-informed endotype vs guideline care in children with difficult-to-treat asthma
Theresa W Guilbert1, Jocelyn M Biagini2, Rachelle R Ramsey3
1Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio.
Insights
A new biomarker-informed treatment model improved outcomes for difficult-to-treat asthma in children. Personalized plans reduced emergency visits and enhanced asthma control, suggesting superiority over standard guideline care.
Area of Science:
- Pulmonology
- Genetics
- Immunology
Background:
- Asthma management is challenging due to its heterogeneous nature.
- Difficult-to-treat (DTT) asthma requires novel therapeutic strategies.
Purpose of the Study:
- To develop and pilot a biomarker-informed treatment model for DTT asthma.
- To assess the feasibility and impact of a personalized, biomarker-guided approach.
Main Methods:
- A pilot study enrolled 21 school-aged children with DTT asthma.
- Guideline care and adherence interventions were initiated, followed by biomarker assessment.
- A personalized treatment algorithm based on biomarkers (treatment by endotype) was implemented.
Main Results:
- Personalized treatment plans significantly reduced emergency department visits (median 1 vs 0, P=.04).
- Asthma control improved, with increased Asthma Control Test scores (median 22.5 vs 23.0, P=.01).
- Specific biomarkers identified distinct asthma phenotypes (allergic, nonallergic, mixed).
Conclusions:
- A biomarker-based algorithm effectively guided additional interventions beyond standard care for DTT asthma.
- This treatment-by-endotype approach is feasible, potentially superior to guideline care alone.
- Findings support a definitive trial for biomarker-informed asthma management.
Background:
Asthma is heterogeneous, contributing to difficulty in disease management.
Objective:
To develop a biomarker-informed treatment model for difficult-to-treat (DTT) asthma and conduct a pilot feasibility study.
Methods:
School-aged children (n = 21) with DTT asthma were enrolled and completed 3 medical visits (V1-V3). V2 and V3 were completed approximately 3.5 months and 12 months after V1, respectively. At V1, guideline care and adherence interventions were initiated, and blood samples were collected for asthma biomarker assessment. A personalized treatment algorithm was developed based on biomarkers (treatment by endotype) and was implemented at V2. Asthma outcomes were compared from V1 to V2 (guideline-based care) to V2 to V3 (guideline + biomarker-informed care).
Results:
Overall retention was 86%. There was an even distribution of participants with allergy, without allergy, and with mixed allergies. The participants received an average of 5.9 interventions (range, 3-9). The allergic phenotype was characterized by increased CDHR3 risk genotype and high transepidermal water loss. High serum interleukin-6 level was most notable in the mixed allergic subgroup. The nonallergic phenotype was characterized by vitamin D deficiency and poor steroid treatment responsiveness. The personalized treatment plans were associated with decreased emergency department visits (median, 1 vs 0; P = .04) and increased asthma control test scores (median, 22.5 vs 23.0; P = .01).
Conclusion:
The biomarker-based treatment algorithm triggered interventions on top of guideline care in all children with DTT asthma studied, supporting the need for this type of multipronged approach. Our findings identify the minimal biomarker set that is informative, reveal that this treatment-by-endotype intervention is feasible and may be superior to guideline care alone, and provide a strong foundation for a definitive trial.
Trial Registration:
ClinicalTrials.gov identifier: NCT04179461.
Related Concept Videos
01:30Asthma-IV: Diagnostic and Management
Clinical Assessment for Asthma:
This is the first step in diagnosing and managing asthma. It includes:
01:25Antiasthma Drugs: Mast Cell Stabilizers and Anti-IgE Drugs
Mast cell stabilizers, such as cromolyn (also known as sodium cromoglycate) and nedocromil (Tilade), are effective drugs in asthma management. These stabilizers hinder histamine release by skillfully obstructing the activation of mast cells and other cellular entities. Notably, they navigate this task without...
01:20Asthma: Pathogenesis and Management
Asthma is classified as allergic and non-allergic. Allergens such as dust mites, pollen, and pet dander trigger allergic asthma, while factors like cold air, intense emotions, or exercise can induce non-allergic asthma.
01:26Asthma-II: Pathophysiology and Classification
Additionally, environmental and genetic factors play crucial roles in determining an individual's susceptibility to asthma and the severity of their condition.
Critical processes in asthma pathophysiology include:
01:30Asthma-IV: Nursing Management
First, in...
01:26COPD: Management Using Bronchodilators and Corticosteroids