Ustekinumab for Intestinal Behçet's Syndrome With Myelodysplastic Syndrome

Efficacy and Safety of Ustekinumab for Intestinal Behçet's Syndrome Complicated by Myelodysplastic Syndrome

Behçet's syndrome is a systemic vasculitis. Gastrointestinal involvement in Behçet's syndrome complicated by myelodysplastic syndrome represents a rare and severe subtype for which no standardized treatment guidelines currently exist, posing significant challenges in clinical practice. Ustekinumab, a biologic agent targeting IL-12/IL-23, has demonstrated favorable efficacy in both gastrointestinal Behçet's syndrome and inflammatory bowel disease. This study aims to evaluate the efficacy and safety of ustekinumab in patients with intestinal Behçet's syndrome and coexisting myelodysplastic syndrome.

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndrome; Behcet's Syndrome, Intestinal Type
• Intervention / Treatment: Drug: Ustekinumab 90 mg

Detailed Description

This is a multicenter, single-arm clinical trial designed to evaluate the efficacy and safety of ustekinumab in patients with intestinal Behçet's syndrome complicated by myelodysplastic syndrome (MDS). A total of 8 patients with intestinal Behçet's syndrome and concomitant MDS will be enrolled. Prior to enrollment, all patients will discontinue any previous biologic agents. Ustekinumab will be administered subcutaneously at a dose of 90 mg at weeks 0, 4, and 8, followed by a maintenance dose of 90 mg every 12 weeks (every 3 months). All patients will be followed for a total of 24 months.

Clinical manifestations, inflammatory biomarkers, and endoscopic findings will be documented throughout the study period. Concomitant medications will be recorded, and adverse events will be systematically monitored to evaluate the efficacy and safety of the treatment.

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100044
    • Department of Rheumatology and Immunology, Peking University People's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Meet the 2014 International Criteria for Behçet's Disease (ICBD) for the diagnosis of Behçet's syndrome; Have a confirmed diagnosis of myelodysplastic syndrome (MDS) by bone marrow aspiration and/or biopsy; Have confirmed intestinal ulcers on colonoscopy; Aged between 18 and 70 years; Have active disease at enrollment, defined as a Behçet's Disease Current Activity Form (BDCAF) score ≥ 1; Provide signed informed consent.

Exclusion Criteria:

Presence of one or more other autoimmune diseases; Involvement of other vital organs (e.g., cardiovascular, neurological) requiring treatment with other biologic agents or high-dose corticosteroids; Receipt of surgical treatment; Severe trilineage cytopenia attributed to myelodysplastic syndrome; Presence of acute or chronic active infection (e.g., bacterial, or viral infections such as EBV, CMV, HIV, or active hepatitis virus) within 4 weeks prior to enrollment; Current or prior history of any malignancy; Pregnancy or within 6 months postpartum; Presence of severe liver failure (Child-Pugh Class C) or end-stage renal disease requiring dialysis.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treated with ustekinumab; Eight patients with intestinal Behçet's syndrome complicated by myelodysplastic syndrome were treated with ustekinumab.	Drug: Ustekinumab 90 mg; Ustekinumab will be administered subcutaneously at a dose of 90 mg at weeks 0, 4, and 8, followed by a maintenance dose of 90 mg every 12 weeks (every 3 months).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patients achieving complete remission, marked improvement, and improvement	The primary endpoint was defined as the proportion of patients achieving each response category at week 24. The response categories included complete remission, marked improvement, and improvement, as defined by the Global Gastrointestinal Symptoms criteria, with the proportion of patients achieving each category expressed as a percentage (%).	Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes of Behcet's Disease Current Activity Form (BDCAF) score of patients	Clinical manifestations are recorded at enrollment and at week 24, and changes in the BDCAF score from baseline to week 24 are evaluated. The score of BDCAF ranges from 0 to 12, with higher scores indicating greater severity.	Week 24
Changes of Disease Activity Index for Intestinal Behcet's Disease (DAIBD) of patients	Intestinal-related clinical manifestations are documented at enrollment and at week 24. Changes in the DAIBD score from baseline to week 24 are evaluated. The score of DAIBD ranges from 0 to 295, with higher scores indicating greater severity.	Week 24
Changes of C-reactive protein	Blood samples were collected from all patients and the concentration of C-reactive protein (mg/L) were recorded.	Week 24
Changes of erythrocyte sedimentation rate	Blood samples were collected from all patients and the erythrocyte sedimentation rates (mm/h) were recorded.	Week 24
Changes of dosage of glucocorticoids from baseline	The dosage of glucocorticoids (mg/day) of all patients were recorded during the follow-up.	Week 24

Collaborators and Investigators

• Sponsor: Liu Tian

Study record dates

Study Major Dates

• Study Start (Actual): March 24, 2024
• Primary Completion (Actual): February 23, 2026
• Study Completion (Actual): February 23, 2026

Study Registration Dates

• First Submitted: April 23, 2026
• First Submitted That Met QC Criteria: May 7, 2026
• First Posted (Actual): May 13, 2026

Study Record Updates

• Last Update Posted (Actual): May 13, 2026
• Last Update Submitted That Met QC Criteria: May 7, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Myelodysplastic Syndrome; Ustekinumab; Behcet's Syndrome, Intestinal Type
• Additional Relevant MeSH Terms: Mouth Diseases; Stomatognathic Diseases; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Eye Diseases; Hematologic Diseases; Skin Diseases; Bone Marrow Diseases; Skin Diseases, Vascular; Skin Diseases, Genetic; Uveal Diseases; Vasculitis; Panuveitis; Uveitis, Anterior; Uveitis; Hereditary Autoinflammatory Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Behcet Syndrome; Amino Acids, Peptides, and Proteins; Proteins; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Serum Globulins; Globulins; Ustekinumab
• Other Study ID Numbers: Peking University PH; PAYJ-023 (Other Grant/Funding Number: Bethune-Puai Medical Research Fund)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No