Behcet's Syndrome, Intestinal Type, Myelodysplastic Syndrome
Conditions
Keywords
Behcet's Syndrome, Intestinal Type, Myelodysplastic Syndrome, Ustekinumab
Brief summary
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.
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.
Interventions
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).
Sponsors
Liu Tian
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Eight patients with intestinal Behçet's syndrome complicated by myelodysplastic syndrome
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Patients achieving complete remission, marked improvement, and improvement | Week 24 | 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 (%). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes of Behcet's Disease Current Activity Form (BDCAF) score of patients | Week 24 | 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. |
| Changes of Disease Activity Index for Intestinal Behcet's Disease (DAIBD) of patients | Week 24 | 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. |
| Changes of C-reactive protein | Week 24 | Blood samples were collected from all patients and the concentration of C-reactive protein (mg/L) were recorded. |
| Changes of erythrocyte sedimentation rate | Week 24 | Blood samples were collected from all patients and the erythrocyte sedimentation rates (mm/h) were recorded. |
| Changes of dosage of glucocorticoids from baseline | Week 24 | The dosage of glucocorticoids (mg/day) of all patients were recorded during the follow-up. |
Countries
China
Outcome results
None listed