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Development and validation of pFIB scores for exclusion of significant liver fibrosis in pediatric MASLD
Hepatology ( IF 12.9 ) Pub Date : 2024-07-19 , DOI: 10.1097/hep.0000000000001016
Sander Lefere 1, 2 , Antonella Mosca 3 , Christian Hudert 4 , Ellen Dupont 5 , Emer Fitzpatrick 6, 7 , Eirini Kyrana 8 , Anil Dhawan 6 , Laura Kalveram 4 , Andrea Pietrobattista 3 , Anja Geerts 1, 2 , Ruth De Bruyne 9
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Background & Aims: Metabolic dysfunction-associated steatotic liver disease (MASLD) is the most prevalent pediatric liver disease, yet accurate risk scores for referral of children/adolescents with suspected clinically significant liver fibrosis are currently lacking. Approach & Results: Clinical and biochemical variables were collected in a prospective cohort of 327 children and adolescents with severe obesity, in whom liver fibrosis was evaluated by transient elastography. Logistic regression was performed to establish continuous (pFIB-c) and simplified (pFIB-6) diagnostic scores that accurately exclude significant (≥F2) fibrosis. Performance for each was compared to established non-invasive fibrosis scores. These scores were validated in elastography (n=504) and multiple biopsy-proven MASLD (n=261) cohorts. Patient sex, ethnicity, weight z-score, HOMA-IR index, ALT, and presence of hypertension were included in the scores. The pFIB-c and pFIB-6 exhibited good discriminatory capacity (c-statistic of 0.839 and 0.826), outperforming existing indices. Negative predictive values (NPV) were >90% for both scores in the derivation and elastography validation cohorts. Performance in the histological cohorts varied (AUROCs for the pFIB-c between 0.710 and 0.770), as the scores were less accurate when applied to populations in tertiary referral centers characterized by a high prevalence of significant fibrosis and high ALT levels. Conclusions: Analyzing several cohorts totaling approximately 1100 children and adolescents, we developed novel risk scores incorporating readily available clinical variables. In accordance with the aim of excluding pediatric MASLD-associated fibrosis, the scores performed better in non-selected cohorts of children and adolescents living with obesity than in patients referred to tertiary liver units.

中文翻译:


用于排除儿童 MASLD 中显着肝纤维化的 pFIB 评分的制定和验证



背景和目的:代谢功能障碍相关的脂肪性肝病(MASLD)是最常见的儿科肝病,但目前缺乏针对疑似临床显着肝纤维化的儿童/青少年转诊的准确风险评分。方法和结果:在 327 名严重肥胖儿童和青少年的前瞻性队列中收集临床和生化变量,通过瞬时弹性成像评估肝纤维化情况。进行逻辑回归以建立连续(pFIB-c)和简化(pFIB-6)诊断评分,准确排除显着(≥F2)纤维化。将每个人的表现与已建立的非侵入性纤维化评分进行比较。这些评分在弹性成像 (n=504) 和经多次活检证实的 MASLD (n=261) 队列中得到验证。评分中包括患者性别、种族、体重 z 评分、HOMA-IR 指数、ALT 和是否存在高血压。 pFIB-c 和 pFIB-6 表现出良好的判别能力(c 统计量分别为 0.839 和 0.826),优于现有指数。推导和弹性成像验证队列中两个评分的阴性预测值 (NPV) 均 >90%。组织学队列中的表现各不相同(pFIB-c 的 AUROC 在 0.710 和 0.770 之间),因为当应用于三级转诊中心以显着纤维化发生率高和 ALT 水平高为特征的人群时,评分不太准确。结论:通过分析总计约 1100 名儿童和青少年的多个队列,我们​​结合现成的临床变量制定了新的风险评分。 根据排除儿科 MASLD 相关纤维化的目标,未选择的肥胖儿童和青少年队列的评分优于转诊至三级肝病单位的患者。
更新日期:2024-07-19
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