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Comparative efficacy of therapies for eosinophilic esophagitis
Investigator (PI): Rothenberg, Marc
Performing Organization (PO): (Current): Cincinnati Children's Hospital Medical Center, Division of Allergy and Immunology / (513) 636-2601
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2015
Final Year: 2019
Record Source/Award ID: PCORI/CER-1403-11593
Funding: Total Award Amount: $2,484,263
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Imagine a life without food while people all around you are eating. Patients with eosinophilic esophagitis (EoE), a chronic disease associated with esophageal problems, face this reality when eosinophils, a type of white blood cell, invade the esophagus. Unlike typical food allergies, EoE is a chronic disorder, as patients are allergic to main food groups, such as milk, egg, and wheat. EoE patients must survive on a restricted diet, representing a medical, psychological, and social nightmare. Our research has shown that children with eosinophilic gastrointestinal disorders have the lowest quality of life (QOL) compared to ten other chronic childhood diseases. Further, the financial burden for families is high due to the cost of regular endoscopies and anesthesia and expensive special formulas and medicines. Dietary therapy does not usually require medication, but food restrictions require deep commitment. Current standard therapies use either an allergy test directed diet elimination (eliminate all foods triggering positive allergy testing) or empiric 6 food elimination diet (eliminate the six most commonly allergenic foods--milk, soy, wheat, egg, fish/shellfish, and peanuts/treenuts); these approaches tend to improve symptoms and inflammation in ~70% of patients. Alternatively, a diet requiring a patient to only consume a completely broken down liquid formula (no other food allowed) causes resolution of symptoms and inflammation in >90% of patients. Finally, the use of swallowed steroids has been shown to improve inflammation and symptoms in 50-90% of patients. There are many limitations to these treatments, including the difficulty staying on extremely restricted diets, the need for repeated endoscopies, and the potential side effects of long-term use of steroids. Given this, our primary goal is to determine whether simpler, markedly less restricted diets might also effectively treat EoE. We plan to compare response to a 3-month trial of either a 1-food (milk alone) or 3-food (milk, wheat, and egg) elimination diet, as measured by examining biopsies from the esophagus under the microscope to see if the abnormal eosinophils have resolved on the diet. We will also compare the results of this microscopic exam to reports from patients on symptoms and quality of life, as well as some other new outcome measurements. We will also assess whether patients who do not improve on these diets might still respond to swallowed steroids by treating them with a 3-month trial of steroids and then examining biopsies as well as symptoms. This study will be important to patients by potentially identifying simpler diet management protocols, which will make following these diets easier, require fewer endoscopies, and improve QOL. It will also be useful in terms of testing newer methods to accurately measure how active the disease is beyond microscopic examination of biopsies.
MeSH Terms:
  • Anesthesia
  • Biopsy
  • Child
  • Chronic Disease
  • Comparative Effectiveness Research
  • * Diet
  • Endoscopy
  • Eosinophilic Esophagitis /psychology
  • /*therapy
  • Esophagus /pathology
  • Food Hypersensitivity /psychology
  • /*therapy
  • Humans
  • Inflammation
  • Quality of Life
  • Steroids /therapeutic use
  • Treatment Outcome
Country: United States
State: Ohio
Zip Code: 45229
UI: 20152199
Project Status: Completed
Record History: ('2017: Project extended to 2019.',)