Table 1.
Topical steroids in pediatric eosinophilic esophagitis, prospective and randomized controlled trials
| Study | Type of Study |
Control Group (n) |
Histologic Criteria |
Drug (n) |
Dose (mg) | Length of Treatment |
Primary Outcomes |
Drug Efficacya (%) |
Control Group Response (%) |
Other Outcomes | Adverse Events |
Comments |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Teitelbaum et al, 2002 | Prospective | NA | >15 eos/hpf, superficial layering, and/or eosinophil microabscesses | FP (13) | 2–4 yo: 88 BID 5–10 yo: 220 BID 2:11 yo: 440 BID |
8 wk | Clinical improvement/resolution of symptoms | 100 | NA | 70% Still had abnormal endoscopy (loss of vascular pattern, thickened longitudinal folds), but improved histology | 18% With esophageal candidiasis, 9% (n 5 1) symptomatic | 8-wk PPI trial before diagnosis. Normal 24-h continuous pH monitoring; 9 of the patients who responded clinically to FP had failed allergy testing–based diet restriction. |
| Konikoff et al, 2006 | Randomized, double-blind, placebo-controlled | Placebo (15) | >24 eos/hpf in any x400 HPF and epithelial hyperplasia | FP (21) | 440 BID | 3 mo | Complete response: <1 eos/hpf | 50 | 9 | All FP responders: resolved distal furrowing, epithelial hyperplasia, and vomiting | Incidental esophageal candidiasis in 9% of FP pts (1/11) | Prior acid suppression therapy was not necessary for diagnosis. FP response higher in nonallergic individuals. FP response negatively correlates with patient age, height, and weight. |
| Partial response: 1–24 eos/hpf | 15 | 9 | ||||||||||
| Schaefer et al, 2008 | Randomized, comparator controlled | Prednisone 1 mg/kg/d (40) | 2:15 eos/hpf with negative pH probe studies | FP (40) | 1–10 yo: 220 QID 11–18 yo: 440 QID |
4-wk Induction | Complete histologic resolution | 50 | 81 | 97% FP group had resolution of symptoms. 100% of prednisone group had resolution of symptoms. | Incidental esophageal candidiasis in 15% of FP patients; hyperphagia, weight gain in 40% of prednisone patients. | Symptom relapse in 44% of FP patients, 45% of prednisone 12 wk after treatment stopped. |
| Improvement in biopsy grade (score based on basal cell zone % and # eos/hpf) | 94 | 94 | ||||||||||
| Dohil et al, 2010 | Randomized, double-blind, placebo-controlled | Placebo (9) | Peak eos/hpf 2:20 | OVB (15) | <5 ft Tall:1000/d 2:5 ft Tall:2000/d |
3 mo | Responders: <6 eos/hpf | 87 | 0 | Endoscopy score improved more in OVB vs placebo. Symptom score improved in OVB but not placebo group. | Oral candidiasis that responded to nystatin. Serum cortisol unchanged | All patients received PPI during drug period. <10 yo: Lansoprazole 15 mg BID; 2:10 yo: lansoprazole 30 mg BID. Placebo and PPI did not improve eosinophilia at any level. |
| Partial responders: 7–19 eos/hpf | 6.7 | 11 | ||||||||||
| Nonresponders 2:20 eos/hpf | 6.7 | 89 | ||||||||||
| Boldorini et al, 2013 | Prospective | NA | >15 eos/hpf | FP (34) | 750 TID | 6 wk | Responders: :s6 eos/hpf | 74 | NA | All children had symptomatic improvement irrespective of histologic results. Responders had more severe inflammation (higher median peak eos/hpf, higher likelihood of eosinophilic microabscesses, and peak mast cells/HPF). | No adverse events seen | All children were nonresponders to PPI or 24-h pH monitoring was negative for gastroesophageal reflux. 4 Children had celiac disease, 3 were responders 1 was not. Age, weight, and height, did not affect response. |
| Borderline: 7–20 eos/hpf | 0 | |||||||||||
| Nonresponders: >20 eos/hpf | 26 |