Optimal Treatment Approaches to Intestinal Behçet's Disease Complicated by Myelodysplastic Syndrome: The KASID and KSBD Multicenter Study

Abstract

Purpose: Studies on intestinal Behçet's disease (BD) complicated by myelodysplastic syndrome (MDS) are rare, and no established therapeutic guidelines exist. This study aimed to evaluate the clinical presentation and outcomes of patients with intestinal BD complicated by MDS (intestinal BD-MDS) and suggest a treatment strategy.

Materials and methods: Data from patients with intestinal BD-MDS from four referral centers in Korea who were diagnosed between December 2000 and December 2022 were retrospectively analyzed. Clinical features and prognosis of intestinal BD-MDS compared with age-, sex-matched intestinal BD without MDS were investigated.

Results: Thirty-five patients with intestinal BD-MDS were included, and 24 (70.6%) had trisomy 8. Among the 35 patients, 23 (65.7%) were female, and the median age at diagnosis for intestinal BD was 46.0 years (range, 37.0-56.0 years). Medical treatments only benefited eight of the 32 patients, and half of the patients underwent surgery due to complications. Compared to 70 matched patients with intestinal BD alone, patients with intestinal BD-MDS underwent surgery more frequently (51.4% vs. 24.3%; p=0.010), showed a poorer response to medical and/or surgical treatment (75.0% vs. 11.4%; p<0.001), and had a higher mortality (28.6% vs. 0%; p<0.001). Seven out of 35 patients with intestinal BD-MDS underwent hematopoietic stem cell transplantation (HSCT), and four out of the seven patients had a poor response to medical treatment prior to HSCT, resulting in complete remission of both diseases.

Conclusion: Patients with intestinal BD-MDS frequently have refractory diseases with high mortalities. HSCT can be an effective treatment modality for medically refractory patients with intestinal BD-MDS.

Keywords: Behçet's syndrome; case–control study; myelodysplastic syndrome; trisomy 8.

Fig. 1. Flowchart showing medical treatment and outcomes of patients with intestinal BD complicated by MDS (A) and controls (B). The number of patients who underwent surgery is represented in parentheses “( ).” BD, Behçet’s disease; MDS, myelodysplastic syndrome; 5-ASA, 5-aminosalicylic acid; CS, corticosteroid; TNF, tumor necrosis factor; HSCT, hematopoietic stem cell transplantation.

Fig. 2. Cumulative survival from index date in the intestinal BD complicated by MDS and intestinal BD alone groups using the Kaplan–Meier method. Index date: the earlier date of diagnosis between intestinal BD and MDS in the intestinal BD complicated by MDS group and the date of intestinal BD diagnosis in the intestinal BD alone group. BD, Behçet’s disease; MDS, myelodysplastic syndrome.