Schiepatti Annalisa, Maimaris Stiliano, Scalvini Davide, Raju Suneil A, Ingham Katerina E, Johnson Calvin M, Rubio-Tapia Alberto, Maruggi Chiara, Malamut Georgia, Lenti Marco Vincenzo, Di Sabatino Antonio, Caio Giacomo, Volta Umberto, Zingone Fabiana, Marasco Giovanni, Barbara Giovanni, Makharia Govind, Mehra Lalita, Das Prasenjit, Lundin Knut E A, Cross Simon S, Sanders David S, Biagi Federico
Department of Internal Medicine and Medical Therapeutics, University of Pavia, Italy.
Istituti Clinici Scientifici Maugeri IRCCS, Gastroenterology Unit of Pavia Institute, Pavia, Italy.
Am J Gastroenterol. 2025 Jan 29. doi: 10.14309/ajg.0000000000003331.
Long-term prognosis of nonceliac enteropathies (NCEs) is poorly understood. We aimed to evaluate long-term outcomes and develop a prognostic score for NCEs.
NCEs patients from an international multicenter cohort (4 Italian centers, 1 United Kingdom, 1 French, 1 Norwegian, 1 United States, 1 Indian) followed-up over 30 years were enrolled. Complications and mortality were analyzed with Kaplan-Meier curves, standardized mortality ratios (SMR), and multivariate Cox regression. A clinical score to identify patients at risk of poor outcomes was developed.
Two hundred sixty-one patients were enrolled (144 female, mean age at diagnosis 49 ± 18 years, median follow-up 70 months, interquartile range 24-109). The most common etiologies were idiopathic villous atrophy (39%), drug related (17%), common variable immune deficiency (15%), infectious (10%), and autoimmune enteropathy (9%). Five-year and 10-year complication-free survival were 89% and 77%, respectively, whereas 5-year and 10-year overall survival were 88% and 74%, respectively. Causes of death included sepsis/major infections (22%), lymphoproliferative disorders (22%), solid-organ malignancies (12%), cardiovascular/metabolic disease (10%), and was unknown in 33%. Mortality was increased in NCEs compared with the general population (SMR 3.17, 95% confidence interval [CI] 2.24-4.34). Older age at diagnosis ( P < 0.001), anemia (hazard ratio [HR] 2.53, 95% CI 1.33-4.80, P < 0.01), and lack of clinical (HR 3.21, 95% CI 1.68-6.18, P < 0.01) and histological response (HR 2.14, 95% CI 1.08-4.23, P = 0.04) were independent predictors of mortality at Cox regression. A 5-point score was developed to identify high-risk patients: very low risk (0 pts), low risk (1-2 pts), intermediate risk (3 pts), and high risk (4-5 pts), with 10-year survival rates of 100%, 87%, 62%, and 16%, respectively.
Mortality in NCEs is increased because of complications and lack of response to current therapies. We developed a clinical score to personalize follow-up. Targeted treatments are needed to improve outcomes.
非乳糜泻性肠病（NCEs）的长期预后尚不清楚。我们旨在评估NCEs的长期结局并制定一个预后评分。
纳入来自一个国际多中心队列（4个意大利中心、1个英国中心、1个法国中心、1个挪威中心、1个美国中心、1个印度中心）且随访超过30年的NCEs患者。采用Kaplan-Meier曲线、标准化死亡比（SMR）和多变量Cox回归分析并发症和死亡率。制定一个临床评分以识别预后不良风险的患者。
共纳入261例患者（144例女性，诊断时平均年龄49±18岁，中位随访70个月，四分位间距24 - 109个月）。最常见的病因是特发性绒毛萎缩（39%）、药物相关（17%）、常见可变免疫缺陷（CVID，15%）、感染性（10%）和自身免疫性肠病（9%）。5年和10年无并发症生存率分别为89%和77%，而5年和10年总生存率分别为88%和74%。死亡原因包括败血症/严重感染（22%）、淋巴增殖性疾病（22%）、实体器官恶性肿瘤（12%）、心血管/代谢疾病（10%），33%原因不明。与一般人群相比，NCEs患者死亡率增加（SMR 3.17，95%置信区间[CI] 2.24 - 4.34）。诊断时年龄较大（P < 0.001）、贫血（风险比[HR] 2.53，95% CI 1.33 - 4.80，P < 0.01）以及缺乏临床（HR 3.21，95% CI ）和组织学反应（HR 2.14，95% CI 1.08 - 4.23，P = 0.04）是Cox回归中死亡率的独立预测因素。制定了一个5分评分来识别高危患者：极低风险（0分）、低风险（1 - 2分）、中度风险（3分）和高风险（4 - 5分），10年生存率分别为100%、87%、62%和16%。
由于并发症以及对当前治疗缺乏反应，NCEs患者死亡率增加。我们制定了一个临床评分以实现随访个体化。需要有针对性的治疗来改善结局。
