Budesonide Oral Suspension Improves Symptomatic, Endoscopic, and Histologic Parameters Compared With Placebo in Patients With Eosinophilic Esophagitis

Evan S. Dellon, David A. Katzka, Margaret H. Collins, Mohamed Hamdani, Sandeep K. Gupta, Ikuo Hirano, Amir Kagalwalla, Jeffrey Lewis, Jonathan Markowitz, Samuel Nurko, John Wo, Evan Dellon, Thirumazhisai S. Gunasekaran, Ikuo Hirano, Sandeep Gupta, Brad Pasternak, Mark Ellis, Kathryn Peterson, Gary Falk, John LeungLaurel Prestridge, Michael Hart, Neal Leleiko, Michael Vaezi, Rebecca Cherry, David Katzka, Keith Friedenberg, Yehudith Assouline-Dayan, Vincent Mukkada

Research output: Contribution to journalArticle

54 Citations (Scopus)

Abstract

Background & Aims Pharmacologic treatment of eosinophilic esophagitis (EoE) is limited to off-label use of corticosteroids not optimized for esophageal delivery. We performed a randomized, controlled phase 2 trial to assess the ability of budesonide oral suspension (BOS), a novel muco-adherent topical steroid formulation, to reduce symptoms and esophageal eosinophilia in adolescents and adults with EoE. Methods In this multicenter, randomized, double-blind, placebo-controlled, parallel-group trial, 93 EoE patients between the ages of 11 and 40 years with dysphagia and active esophageal eosinophilia were randomized to receive either BOS 2 mg or placebo twice daily for 12 weeks. Co-primary outcomes were change in Dysphagia Symptom Questionnaire (DSQ) score from baseline, and proportion of patients with a histologic response (≤6 eosinophils/high-power field) after treatment. Endoscopic severity scores and safety parameters were assessed. Results At baseline, mean DSQ scores were 29.3 and 29.0, and mean peak eosinophil counts were 156 and 130 per hpf in the BOS and placebo groups, respectively. After treatment, DSQ scores were 15.0 and 21.5, and mean peak eosinophil counts were 39 and 113 per high-power field, respectively (P < .05 for all). For BOS vs placebo, change in DSQ score was −14.3 vs −7.5 (P = .0096), histologic response rates were 39% vs 3% (P < .0001), and change in endoscopic severity score was −3.8 vs 0.4 (P < .0001). Adverse events were similar between groups. Conclusions Treatment with BOS was well tolerated in adolescent and young adult patients with EoE and resulted in improvement in symptomatic, endoscopic, and histologic parameters using validated outcome instruments. ClinicalTrials.gov ID NCT01642212.

Original languageEnglish (US)
Pages (from-to)776-786.e5
JournalGastroenterology
Volume152
Issue number4
DOIs
StatePublished - Mar 1 2017

Fingerprint

Eosinophilic Esophagitis
Budesonide
Deglutition Disorders
Suspensions
Placebos
Eosinophils
Eosinophilia
Off-Label Use
Therapeutics
Young Adult
Adrenal Cortex Hormones
Steroids
Safety
Surveys and Questionnaires

Keywords

  • Corticosteroids
  • Dysphagia
  • Eosinophilic Esophagitis
  • Patient-Reported Outcomes

ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology

Cite this

Dellon, Evan S. ; Katzka, David A. ; Collins, Margaret H. ; Hamdani, Mohamed ; Gupta, Sandeep K. ; Hirano, Ikuo ; Kagalwalla, Amir ; Lewis, Jeffrey ; Markowitz, Jonathan ; Nurko, Samuel ; Wo, John ; Dellon, Evan ; Gunasekaran, Thirumazhisai S. ; Hirano, Ikuo ; Gupta, Sandeep ; Pasternak, Brad ; Ellis, Mark ; Peterson, Kathryn ; Falk, Gary ; Leung, John ; Prestridge, Laurel ; Hart, Michael ; Leleiko, Neal ; Vaezi, Michael ; Cherry, Rebecca ; Katzka, David ; Friedenberg, Keith ; Assouline-Dayan, Yehudith ; Mukkada, Vincent. / Budesonide Oral Suspension Improves Symptomatic, Endoscopic, and Histologic Parameters Compared With Placebo in Patients With Eosinophilic Esophagitis. In: Gastroenterology. 2017 ; Vol. 152, No. 4. pp. 776-786.e5.
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abstract = "Background & Aims Pharmacologic treatment of eosinophilic esophagitis (EoE) is limited to off-label use of corticosteroids not optimized for esophageal delivery. We performed a randomized, controlled phase 2 trial to assess the ability of budesonide oral suspension (BOS), a novel muco-adherent topical steroid formulation, to reduce symptoms and esophageal eosinophilia in adolescents and adults with EoE. Methods In this multicenter, randomized, double-blind, placebo-controlled, parallel-group trial, 93 EoE patients between the ages of 11 and 40 years with dysphagia and active esophageal eosinophilia were randomized to receive either BOS 2 mg or placebo twice daily for 12 weeks. Co-primary outcomes were change in Dysphagia Symptom Questionnaire (DSQ) score from baseline, and proportion of patients with a histologic response (≤6 eosinophils/high-power field) after treatment. Endoscopic severity scores and safety parameters were assessed. Results At baseline, mean DSQ scores were 29.3 and 29.0, and mean peak eosinophil counts were 156 and 130 per hpf in the BOS and placebo groups, respectively. After treatment, DSQ scores were 15.0 and 21.5, and mean peak eosinophil counts were 39 and 113 per high-power field, respectively (P < .05 for all). For BOS vs placebo, change in DSQ score was −14.3 vs −7.5 (P = .0096), histologic response rates were 39{\%} vs 3{\%} (P < .0001), and change in endoscopic severity score was −3.8 vs 0.4 (P < .0001). Adverse events were similar between groups. Conclusions Treatment with BOS was well tolerated in adolescent and young adult patients with EoE and resulted in improvement in symptomatic, endoscopic, and histologic parameters using validated outcome instruments. ClinicalTrials.gov ID NCT01642212.",
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author = "Dellon, {Evan S.} and Katzka, {David A.} and Collins, {Margaret H.} and Mohamed Hamdani and Gupta, {Sandeep K.} and Ikuo Hirano and Amir Kagalwalla and Jeffrey Lewis and Jonathan Markowitz and Samuel Nurko and John Wo and Evan Dellon and Gunasekaran, {Thirumazhisai S.} and Ikuo Hirano and Sandeep Gupta and Brad Pasternak and Mark Ellis and Kathryn Peterson and Gary Falk and John Leung and Laurel Prestridge and Michael Hart and Neal Leleiko and Michael Vaezi and Rebecca Cherry and David Katzka and Keith Friedenberg and Yehudith Assouline-Dayan and Vincent Mukkada",
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Dellon, ES, Katzka, DA, Collins, MH, Hamdani, M, Gupta, SK, Hirano, I, Kagalwalla, A, Lewis, J, Markowitz, J, Nurko, S, Wo, J, Dellon, E, Gunasekaran, TS, Hirano, I, Gupta, S, Pasternak, B, Ellis, M, Peterson, K, Falk, G, Leung, J, Prestridge, L, Hart, M, Leleiko, N, Vaezi, M, Cherry, R, Katzka, D, Friedenberg, K, Assouline-Dayan, Y & Mukkada, V 2017, 'Budesonide Oral Suspension Improves Symptomatic, Endoscopic, and Histologic Parameters Compared With Placebo in Patients With Eosinophilic Esophagitis', Gastroenterology, vol. 152, no. 4, pp. 776-786.e5. https://doi.org/10.1053/j.gastro.2016.11.021

Budesonide Oral Suspension Improves Symptomatic, Endoscopic, and Histologic Parameters Compared With Placebo in Patients With Eosinophilic Esophagitis. / Dellon, Evan S.; Katzka, David A.; Collins, Margaret H.; Hamdani, Mohamed; Gupta, Sandeep K.; Hirano, Ikuo; Kagalwalla, Amir; Lewis, Jeffrey; Markowitz, Jonathan; Nurko, Samuel; Wo, John; Dellon, Evan; Gunasekaran, Thirumazhisai S.; Hirano, Ikuo; Gupta, Sandeep; Pasternak, Brad; Ellis, Mark; Peterson, Kathryn; Falk, Gary; Leung, John; Prestridge, Laurel; Hart, Michael; Leleiko, Neal; Vaezi, Michael; Cherry, Rebecca; Katzka, David; Friedenberg, Keith; Assouline-Dayan, Yehudith; Mukkada, Vincent.

In: Gastroenterology, Vol. 152, No. 4, 01.03.2017, p. 776-786.e5.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Budesonide Oral Suspension Improves Symptomatic, Endoscopic, and Histologic Parameters Compared With Placebo in Patients With Eosinophilic Esophagitis

AU - Dellon, Evan S.

AU - Katzka, David A.

AU - Collins, Margaret H.

AU - Hamdani, Mohamed

AU - Gupta, Sandeep K.

AU - Hirano, Ikuo

AU - Kagalwalla, Amir

AU - Lewis, Jeffrey

AU - Markowitz, Jonathan

AU - Nurko, Samuel

AU - Wo, John

AU - Dellon, Evan

AU - Gunasekaran, Thirumazhisai S.

AU - Hirano, Ikuo

AU - Gupta, Sandeep

AU - Pasternak, Brad

AU - Ellis, Mark

AU - Peterson, Kathryn

AU - Falk, Gary

AU - Leung, John

AU - Prestridge, Laurel

AU - Hart, Michael

AU - Leleiko, Neal

AU - Vaezi, Michael

AU - Cherry, Rebecca

AU - Katzka, David

AU - Friedenberg, Keith

AU - Assouline-Dayan, Yehudith

AU - Mukkada, Vincent

PY - 2017/3/1

Y1 - 2017/3/1

N2 - Background & Aims Pharmacologic treatment of eosinophilic esophagitis (EoE) is limited to off-label use of corticosteroids not optimized for esophageal delivery. We performed a randomized, controlled phase 2 trial to assess the ability of budesonide oral suspension (BOS), a novel muco-adherent topical steroid formulation, to reduce symptoms and esophageal eosinophilia in adolescents and adults with EoE. Methods In this multicenter, randomized, double-blind, placebo-controlled, parallel-group trial, 93 EoE patients between the ages of 11 and 40 years with dysphagia and active esophageal eosinophilia were randomized to receive either BOS 2 mg or placebo twice daily for 12 weeks. Co-primary outcomes were change in Dysphagia Symptom Questionnaire (DSQ) score from baseline, and proportion of patients with a histologic response (≤6 eosinophils/high-power field) after treatment. Endoscopic severity scores and safety parameters were assessed. Results At baseline, mean DSQ scores were 29.3 and 29.0, and mean peak eosinophil counts were 156 and 130 per hpf in the BOS and placebo groups, respectively. After treatment, DSQ scores were 15.0 and 21.5, and mean peak eosinophil counts were 39 and 113 per high-power field, respectively (P < .05 for all). For BOS vs placebo, change in DSQ score was −14.3 vs −7.5 (P = .0096), histologic response rates were 39% vs 3% (P < .0001), and change in endoscopic severity score was −3.8 vs 0.4 (P < .0001). Adverse events were similar between groups. Conclusions Treatment with BOS was well tolerated in adolescent and young adult patients with EoE and resulted in improvement in symptomatic, endoscopic, and histologic parameters using validated outcome instruments. ClinicalTrials.gov ID NCT01642212.

AB - Background & Aims Pharmacologic treatment of eosinophilic esophagitis (EoE) is limited to off-label use of corticosteroids not optimized for esophageal delivery. We performed a randomized, controlled phase 2 trial to assess the ability of budesonide oral suspension (BOS), a novel muco-adherent topical steroid formulation, to reduce symptoms and esophageal eosinophilia in adolescents and adults with EoE. Methods In this multicenter, randomized, double-blind, placebo-controlled, parallel-group trial, 93 EoE patients between the ages of 11 and 40 years with dysphagia and active esophageal eosinophilia were randomized to receive either BOS 2 mg or placebo twice daily for 12 weeks. Co-primary outcomes were change in Dysphagia Symptom Questionnaire (DSQ) score from baseline, and proportion of patients with a histologic response (≤6 eosinophils/high-power field) after treatment. Endoscopic severity scores and safety parameters were assessed. Results At baseline, mean DSQ scores were 29.3 and 29.0, and mean peak eosinophil counts were 156 and 130 per hpf in the BOS and placebo groups, respectively. After treatment, DSQ scores were 15.0 and 21.5, and mean peak eosinophil counts were 39 and 113 per high-power field, respectively (P < .05 for all). For BOS vs placebo, change in DSQ score was −14.3 vs −7.5 (P = .0096), histologic response rates were 39% vs 3% (P < .0001), and change in endoscopic severity score was −3.8 vs 0.4 (P < .0001). Adverse events were similar between groups. Conclusions Treatment with BOS was well tolerated in adolescent and young adult patients with EoE and resulted in improvement in symptomatic, endoscopic, and histologic parameters using validated outcome instruments. ClinicalTrials.gov ID NCT01642212.

KW - Corticosteroids

KW - Dysphagia

KW - Eosinophilic Esophagitis

KW - Patient-Reported Outcomes

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