What are the treatments for patients with mast cell disorders, eosinophilic gastroenteritis, or eosinophilic esophagitis?

Treatment of Mast Cell Disorders, Eosinophilic Gastroenteritis, and Eosinophilic Esophagitis

Mast Cell Disorders

For mast cell disorders (mastocytosis), cromolyn sodium oral solution is the FDA-approved treatment and should be used to manage symptoms including diarrhea, flushing, headaches, vomiting, urticaria, abdominal pain, nausea, and itching. 1

• Cromolyn sodium is a mast cell stabilizer that prevents the release of inflammatory mediators 1
• This is the only FDA-approved oral medication specifically indicated for mastocytosis 1

Eosinophilic Esophagitis (EoE)

Topical corticosteroids are the first-line treatment for eosinophilic esophagitis and should be strongly preferred over no treatment. 2

Primary Treatment Options:

• Topical steroids (swallowed, not inhaled) are highly effective and represent the strongest recommendation from multiple guidelines 2
• Proton pump inhibitors (PPIs) are an alternative first-line option, though if they cause side effects (diarrhea, GI infections, magnesium deficiency), switch to topical steroids or dietary therapy 2
• Dietary elimination therapy is effective but requires supervision by an experienced dietitian 2, 3

Maintenance Therapy:

• The rate of clinical relapse is high after withdrawal of topical steroids, so maintenance treatment should be recommended after clinical review 2
• Medical treatment with topical steroids likely reduces the development of strictures 2

Treatments to AVOID in EoE:

• Montelukast is NOT recommended - showed no benefit in reducing eosinophilia (OR 0.48,95% CI 0.10-2.16, p=0.33) 2, 4, 3
• Cromolyn sodium is NOT recommended - only 1 of 16 patients achieved remission in trials 2, 4, 3
• Antihistamines are NOT recommended for EoE management 2, 4
• Anti-IgE therapy (omalizumab) is NOT recommended - showed no effect on esophageal eosinophilia or symptoms 2, 4
• Immunomodulators (azathioprine, 6-mercaptopurine) are NOT recommended 2
• Anti-TNF and anti-integrin therapies are NOT recommended 2

Novel Biologics (Emerging):

• Dupilumab, cendakimab, and benralizumab show promise but are currently recommended only in the context of clinical trials or for patients with significant concomitant atopic disease 2, 3

Management of Complications:

• For fibrostenotic disease with strictures: Endoscopic dilation is safe and effective using either balloon or bougie dilators 2
• Combine therapeutic dilation with topical steroids for better clinical outcomes 2
• EoE is the most common cause of spontaneous esophageal perforation at any age 2

Eosinophilic Gastroenteritis (EGE)

Systemic corticosteroids are the most effective treatment for eosinophilic gastroenteritis, inducing both clinical and histological remission in the majority of patients. 3, 5, 6

Treatment Algorithm:

1. First-line: Systemic corticosteroids - induce remission in approximately 95% of patients 3, 5, 6
2. Consider PPIs as complementary therapy at omeprazole 20 mg twice daily for 8-12 weeks, particularly if acid-related symptoms are present 3
3. Elimination diets should be conducted only under dietitian supervision, starting with two-food elimination (milk +/- wheat or egg) for 8-12 weeks if food allergy is suspected 3
4. Elemental diets have the highest efficacy but lowest compliance; reserve for refractory cases 3

Treatments That Do NOT Work for EGE:

• Sodium cromoglycate failed to demonstrate clinical improvement despite laboratory immunological changes 3
• Montelukast is NOT effective - no statistically significant difference versus placebo 3
• Antihistamines have no demonstrated efficacy for eosinophilic gastrointestinal disorders 3

Novel Biologics:

• Anti-IL-5 agents (mepolizumab) reduced eosinophil counts but showed variable clinical response and are not recommended for routine use 3, 6
• Dupilumab, cendakimab, and benralizumab are recommended only for patients with coexisting allergic diseases, not as primary therapy 3, 6

Critical Clinical Pitfalls

• Do not rely on symptoms alone - endoscopy with biopsy is essential to evaluate histological response, as symptoms may not correlate with histological activity 2, 3, 7
• Repeat endoscopy should be performed no sooner than 4 weeks after the last therapeutic intervention 2
• Approximately 30-40% of EGE cases may have spontaneous remission, but most require ongoing treatment 5, 6

When to Refer

• Patients with EoE or EGE refractory to treatment and/or with significant concomitant atopic disease should be jointly managed by a gastroenterologist and specialist allergist 2, 3
• If eosinophilia persists ≥1.5 × 10⁹/L for more than 3 months without an identified cause, refer to hematology 4