Clinical trial NCT05309590: Efficacy of Low-Dose Topical Steroids in Maintaining Remission of Eosinophilic Esophagitis in Children
Official title: Prospective Pilot Study Evaluating the Efficacy of Low-Dose Topical Steroids in Maintaining Histologic Remission of Eosinophilic Esophagitis in Children
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Eosinophilic Esophagitis
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
First posted: 2022-04-04
Enrollment: 50
Sex: ALL
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: By design, this study involves the participation of children. It is not anticipated that any other special groups be enrolled in a proportion other than that representative of the US population. Enrollment will not be limited by race or gender.

Brief summary: This study evaluates the decrease in steroid dosing for patients who have achieved remission on a full dose of steroids. Once a patient is in remission they will be enrolled in this study if they choose to decrease the steroid dosing.

Detailed description: As the disease usually recurs once steroids are discontinued, and there is concern for complications of untreated disease, continuation of steroids is indefinite after induction of remission. The short-term safety of topical steroids has been well established; however, long-term safety has not been studied in EoE. Concerns for long-term complications of chronic steroid therapy include effects on bone density and stature as has been suggested by literature in patients with asthma treated with inhaled corticosteroids chronically.4, 5 It remains unclear whether symptom and histologic remission, once achieved, can be maintained with a lower, to be defined, dosage of topical steroids.6 This is critical to prevent the major complications associated with uncontrolled eosinophilic inflammation: fibrotic remodeling (scarring) of the esophagus and eventual stricture (narrowing of the esophagus).7-11

Several studies have demonstrated that remission with induction (approximately 3 months of therapy) dose topical steroids resulted in resolution of inflammation and concurrent reversal of sub-epithelial early-stage fibrosis.12-18 It has not, however, been established whether fibrosis recurs when patients are maintained on induction doses long-term or whether lower "maintenance" dosing is sufficient. Studies are therefore needed to demonstrate whether fibrosis can be prevented from recurring long-term, ideally at a lower maintenance dose to minimize potential complications of therapy. Therapeutic strategies that achieve continuous and sustained remission with resolution of eosinophilic inflammation will prevent chronic symptoms along with remodeling, fibrosis and eventual esophageal stricture.

The rationale for utilizing lower dose topical steroids for maintaining clinical and histologic remission in children is the potential to lower the steroid-related side effects including contracting oral and/or esophageal fungal infections (a yeast infection which causes white patches in the mouth and throat), loss of bone density, suppression of adrenal-cortical axis (turns off the body's usual production of cortisol, which helps the body maintain a balance and respond to stress), and failure to achieve linear growth (height) potential. There are no studies that have properly quantified the risk of swallowed steroids in EoE, thus the investigators are presuming and postulating a lower cumulative risk of adverse effects from long-term use of the medication with this lower dose than the standard dose.

Eligibility criteria: Inclusion Criteria:

* Patients diagnosed with EoE
* Patients less than 21 years of age at enrollment
* Patients who within the past 12 months have demonstrated histologic remission (\<15 eos/hpf as assessed by upper esophagogastroduodenoscopy (EGD) with biopsy ) to the standard induction dose of topical steroids and have remained on this treatment:

  * Topical steroid treatment includes only those with previously published results: Swallowed topical fluticasone (i.e. Flovent®), and Swallowed viscous budesonide (i.e. Pulmicort Respules®) mixed with Honey or Splenda®
* Patients who are interested in lowering their dose of topical steroids.

Exclusion Criteria:

* Patients with EoE who have not demonstrated histologic remission (\<15 eos/hpf) to topical steroids.
* Patients who are unable or unwilling to take topical steroids.
* Patients who have changed the vehicle or carrier (e.g. Splenda®or honey ) used to mix with the actual steroid drug, after demonstrating histologic remission.

Primary outcomes:
measure: Efficacy of Low Dose Topical Steroid
timeFrame: At standard of care endoscopies from the date of enrollment; estimated every 6-12 months
Secondary outcomes:
measure: Visual and histologic assessment of the esophagus
timeFrame: At standard of care endoscopies from the date of enrollment; estimated every 6-12 months
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H Lurie Childjren's Hospital of Chicago
state: Illinois
Study officials:
affiliation: Ann & Robert H Lurie Children's Hospital of Chicago
name: Joshua B Wechsler, MD
role: PRINCIPAL_INVESTIGATOR
References:
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