TY - JOUR
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 K.
AU - Lan, Lan
AU - Williams, James
AU - Hirano, Ikuo
N1 - Funding Information:
The authors thank all MPI-101-06 investigators (listed in the Supplementary Material ) and participants for their contribution to this study. The authors thank Mohamed Hamdani of Shire, who previously provided statistical support for this study. They also thank Luci Witcomb, PhD, of PharmaGenesis London, for providing medical writing support funded by Shire International GmbH .
PY - 2019/3
Y1 - 2019/3
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.
KW - Clinical Trial
KW - Corticosteroid
KW - Esophagus
KW - Treatment
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U2 - 10.1016/j.cgh.2018.05.051
DO - 10.1016/j.cgh.2018.05.051
M3 - Article
C2 - 29902649
AN - SCOPUS:85060640315
VL - 17
SP - 666-673.e8
JO - Clinical Gastroenterology and Hepatology
JF - Clinical Gastroenterology and Hepatology
SN - 1542-3565
IS - 4
ER -