Safety and Efficacy of Budesonide Oral Suspension Maintenance Therapy in Patients With Eosinophilic Esophagitis

Evan S. Dellon, David A. Katzka, Margaret H. Collins, Sandeep Gupta, Lan Lan, James Williams, Ikuo Hirano

Research output: Contribution to journalArticle

10 Citations (Scopus)

Abstract

Background & Aims: We aimed to determine the safety and efficacy of budesonide oral suspension (BOS) maintenance therapy in patients with eosinophilic esophagitis (EoE). Methods: We performed an open-label extension study of a 12-week, multicenter, randomized, double-blind, placebo-controlled trial. Patients with EoE (11–40 years old) who completed double-blind BOS (n = 45) or placebo therapy (n = 37) received 24 weeks’ open-label BOS (2.0 mg once daily for 12 weeks, with optional dose increase [1.5–2.0 mg twice daily] for 12 weeks thereafter). Predefined efficacy outcomes included: proportion of patients with a histologic response (≤6 eosinophils/high-power field [eos/hpf]) and change in mean peak eosinophil counts after 24 weeks. Analyses were stratified by patients who received placebo (placebo/BOS) or BOS (BOS/BOS) during the double-blind trial. Results: BOS was well tolerated and drug-related adverse events were uncommon (placebo/BOS, 19% [7/37]; BOS/BOS, 4% [2/45]). Incidence of oral candidiasis (1 per group) and esophageal candidiasis (placebo/BOS group, n = 4) remained low. Changes in morning serum cortisol levels were not clinically relevant. A histologic response was observed in 49% (16/33) of patients receiving placebo/BOS and 23% (9/39) receiving BOS/BOS. Mean peak eosinophil counts (baseline vs week 24 or early termination) were: placebo/BOS, 118.8 vs 29.1; P <.001 and BOS/BOS, 38.1 vs 72.4; P =.01. Of the patients who responded to double-blind therapy, 42% maintained a histologic response during the open-label extension; 4% of nonresponders gained response. Conclusions: In an open-label extension study of patients with EoE, BOS was well tolerated and drug-related adverse events were uncommon. BOS maintained a histologic response in some initial responders, but few initial nonresponders had a response. ClinicalTrials.gov no: NCT01642212.

Original languageEnglish (US)
JournalClinical Gastroenterology and Hepatology
DOIs
StateAccepted/In press - Jan 1 2019
Externally publishedYes

Fingerprint

Eosinophilic Esophagitis
Budesonide
Suspensions
Safety
Placebos
Therapeutics
Eosinophils
Drug-Related Side Effects and Adverse Reactions

Keywords

  • Clinical Trial
  • Corticosteroid
  • Esophagus
  • Treatment

ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology

Cite this

Safety and Efficacy of Budesonide Oral Suspension Maintenance Therapy in Patients With Eosinophilic Esophagitis. / Dellon, Evan S.; Katzka, David A.; Collins, Margaret H.; Gupta, Sandeep; Lan, Lan; Williams, James; Hirano, Ikuo.

In: Clinical Gastroenterology and Hepatology, 01.01.2019.

Research output: Contribution to journalArticle

Dellon, Evan S. ; Katzka, David A. ; Collins, Margaret H. ; Gupta, Sandeep ; Lan, Lan ; Williams, James ; Hirano, Ikuo. / Safety and Efficacy of Budesonide Oral Suspension Maintenance Therapy in Patients With Eosinophilic Esophagitis. In: Clinical Gastroenterology and Hepatology. 2019.
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abstract = "Background & Aims: We aimed to determine the safety and efficacy of budesonide oral suspension (BOS) maintenance therapy in patients with eosinophilic esophagitis (EoE). Methods: We performed an open-label extension study of a 12-week, multicenter, randomized, double-blind, placebo-controlled trial. Patients with EoE (11–40 years old) who completed double-blind BOS (n = 45) or placebo therapy (n = 37) received 24 weeks’ open-label BOS (2.0 mg once daily for 12 weeks, with optional dose increase [1.5–2.0 mg twice daily] for 12 weeks thereafter). Predefined efficacy outcomes included: proportion of patients with a histologic response (≤6 eosinophils/high-power field [eos/hpf]) and change in mean peak eosinophil counts after 24 weeks. Analyses were stratified by patients who received placebo (placebo/BOS) or BOS (BOS/BOS) during the double-blind trial. Results: BOS was well tolerated and drug-related adverse events were uncommon (placebo/BOS, 19{\%} [7/37]; BOS/BOS, 4{\%} [2/45]). Incidence of oral candidiasis (1 per group) and esophageal candidiasis (placebo/BOS group, n = 4) remained low. Changes in morning serum cortisol levels were not clinically relevant. A histologic response was observed in 49{\%} (16/33) of patients receiving placebo/BOS and 23{\%} (9/39) receiving BOS/BOS. Mean peak eosinophil counts (baseline vs week 24 or early termination) were: placebo/BOS, 118.8 vs 29.1; P <.001 and BOS/BOS, 38.1 vs 72.4; P =.01. Of the patients who responded to double-blind therapy, 42{\%} maintained a histologic response during the open-label extension; 4{\%} of nonresponders gained response. Conclusions: In an open-label extension study of patients with EoE, BOS was well tolerated and drug-related adverse events were uncommon. BOS maintained a histologic response in some initial responders, but few initial nonresponders had a response. ClinicalTrials.gov no: NCT01642212.",
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T1 - Safety and Efficacy of Budesonide Oral Suspension Maintenance Therapy in Patients With Eosinophilic Esophagitis

AU - Dellon, Evan S.

AU - Katzka, David A.

AU - Collins, Margaret H.

AU - Gupta, Sandeep

AU - Lan, Lan

AU - Williams, James

AU - Hirano, Ikuo

PY - 2019/1/1

Y1 - 2019/1/1

N2 - Background & Aims: We aimed to determine the safety and efficacy of budesonide oral suspension (BOS) maintenance therapy in patients with eosinophilic esophagitis (EoE). Methods: We performed an open-label extension study of a 12-week, multicenter, randomized, double-blind, placebo-controlled trial. Patients with EoE (11–40 years old) who completed double-blind BOS (n = 45) or placebo therapy (n = 37) received 24 weeks’ open-label BOS (2.0 mg once daily for 12 weeks, with optional dose increase [1.5–2.0 mg twice daily] for 12 weeks thereafter). Predefined efficacy outcomes included: proportion of patients with a histologic response (≤6 eosinophils/high-power field [eos/hpf]) and change in mean peak eosinophil counts after 24 weeks. Analyses were stratified by patients who received placebo (placebo/BOS) or BOS (BOS/BOS) during the double-blind trial. Results: BOS was well tolerated and drug-related adverse events were uncommon (placebo/BOS, 19% [7/37]; BOS/BOS, 4% [2/45]). Incidence of oral candidiasis (1 per group) and esophageal candidiasis (placebo/BOS group, n = 4) remained low. Changes in morning serum cortisol levels were not clinically relevant. A histologic response was observed in 49% (16/33) of patients receiving placebo/BOS and 23% (9/39) receiving BOS/BOS. Mean peak eosinophil counts (baseline vs week 24 or early termination) were: placebo/BOS, 118.8 vs 29.1; P <.001 and BOS/BOS, 38.1 vs 72.4; P =.01. Of the patients who responded to double-blind therapy, 42% maintained a histologic response during the open-label extension; 4% of nonresponders gained response. Conclusions: In an open-label extension study of patients with EoE, BOS was well tolerated and drug-related adverse events were uncommon. BOS maintained a histologic response in some initial responders, but few initial nonresponders had a response. ClinicalTrials.gov no: NCT01642212.

AB - Background & Aims: We aimed to determine the safety and efficacy of budesonide oral suspension (BOS) maintenance therapy in patients with eosinophilic esophagitis (EoE). Methods: We performed an open-label extension study of a 12-week, multicenter, randomized, double-blind, placebo-controlled trial. Patients with EoE (11–40 years old) who completed double-blind BOS (n = 45) or placebo therapy (n = 37) received 24 weeks’ open-label BOS (2.0 mg once daily for 12 weeks, with optional dose increase [1.5–2.0 mg twice daily] for 12 weeks thereafter). Predefined efficacy outcomes included: proportion of patients with a histologic response (≤6 eosinophils/high-power field [eos/hpf]) and change in mean peak eosinophil counts after 24 weeks. Analyses were stratified by patients who received placebo (placebo/BOS) or BOS (BOS/BOS) during the double-blind trial. Results: BOS was well tolerated and drug-related adverse events were uncommon (placebo/BOS, 19% [7/37]; BOS/BOS, 4% [2/45]). Incidence of oral candidiasis (1 per group) and esophageal candidiasis (placebo/BOS group, n = 4) remained low. Changes in morning serum cortisol levels were not clinically relevant. A histologic response was observed in 49% (16/33) of patients receiving placebo/BOS and 23% (9/39) receiving BOS/BOS. Mean peak eosinophil counts (baseline vs week 24 or early termination) were: placebo/BOS, 118.8 vs 29.1; P <.001 and BOS/BOS, 38.1 vs 72.4; P =.01. Of the patients who responded to double-blind therapy, 42% maintained a histologic response during the open-label extension; 4% of nonresponders gained response. Conclusions: In an open-label extension study of patients with EoE, BOS was well tolerated and drug-related adverse events were uncommon. BOS maintained a histologic response in some initial responders, but few initial nonresponders had a response. ClinicalTrials.gov no: NCT01642212.

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KW - Corticosteroid

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KW - Treatment

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