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Abstract：
OBJECTIVE: 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.
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.
CONCLUSIONS: 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.
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.
CONCLUSIONS: 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.
摘要：
目的：关于肠Behçet病（BD）并发骨髓增生异常综合征（MDS）的研究很少，并且没有既定的治疗指南。本研究旨在评估肠道BD并发MDS（肠道BD-MDS）患者的临床表现和预后，并提出治疗策略。
方法：回顾性分析了2000年12月至2022年12月间来自韩国四个转诊中心的肠道BD-MDS患者的数据。肠道BD-MDS的临床特征及预后与年龄、研究了无MDS的性别匹配的肠道BD。
结果：纳入35例肠道BD-MDS患者，24例(70.6%)有三体8。在35名患者中，23人（65.7%）为女性，诊断为肠BD的中位年龄为46.0岁(范围，37.0-56.0年）。药物治疗仅使32名患者中的8名受益，一半的患者因并发症接受了手术。与70例仅肠道BD的匹配患者相比，肠道BD-MDS患者接受手术的频率更高(51.4%vs.24.3%；p=0.010），对药物和/或手术治疗的反应较差(75.0%vs.11.4%；p<0.001)，死亡率较高(28.6%vs.0%；p<0.001)。35例肠道BD-MDS患者中有7例接受了造血干细胞移植（HSCT），七名患者中有四名在HSCT之前对药物治疗的反应较差，导致两种疾病的完全缓解。
结论：肠道BD-MDS患者常有难治性疾病，死亡率高。HSCT可作为难治性肠道BD-MDS患者的有效治疗方法。
方法：回顾性分析了2000年12月至2022年12月间来自韩国四个转诊中心的肠道BD-MDS患者的数据。肠道BD-MDS的临床特征及预后与年龄、研究了无MDS的性别匹配的肠道BD。
结果：纳入35例肠道BD-MDS患者，24例(70.6%)有三体8。在35名患者中，23人（65.7%）为女性，诊断为肠BD的中位年龄为46.0岁(范围，37.0-56.0年）。药物治疗仅使32名患者中的8名受益，一半的患者因并发症接受了手术。与70例仅肠道BD的匹配患者相比，肠道BD-MDS患者接受手术的频率更高(51.4%vs.24.3%；p=0.010），对药物和/或手术治疗的反应较差(75.0%vs.11.4%；p<0.001)，死亡率较高(28.6%vs.0%；p<0.001)。35例肠道BD-MDS患者中有7例接受了造血干细胞移植（HSCT），七名患者中有四名在HSCT之前对药物治疗的反应较差，导致两种疾病的完全缓解。
结论：肠道BD-MDS患者常有难治性疾病，死亡率高。HSCT可作为难治性肠道BD-MDS患者的有效治疗方法。
