National Clinical Research Centre for Digestive Diseases, Beijing Friendship Hospital, Capital Medical University, 95# Yong-an Road, Xi Cheng District, Beijing, 100050, China.
China Standard Medical Information Research Centre, Shenzhen, Guangdong, China.
Orphanet J Rare Dis. 2020 Jun 19;15(1):154. doi: 10.1186/s13023-020-01446-0.
Patients with isolated methylmalonic acidemia (MMA) usually experience recurrent episodes of acute metabolic decompensation or metabolic stroke, require frequent hospitalization, and have a relatively high mortality rate. The aim of our study was to assess factors predicting the in-hospital death of pediatric patients with isolated MMA. We performed a retrospective study using data from the Hospital Quality Monitoring System, a national inpatient database in China collected from 2013 to 2017. All patients under 18 years old with a diagnosis of isolated MMA were included. Demographic, hospital-related, and clinical features were collected. Poisson regression was performed to identify potential influencing variables associated with in-hospital death.
From 2013 to 2017, among 2317 admissions for pediatric patients diagnosed with isolated MMA, 1.77% had the outcome of death. In the univariate analysis, patients aged under 1 year had a higher risk of death than did those aged 1 year or older (odds ratio [OR] = 2.63, 95% confidence interval [CI]: 1.36-5.07). There was a higher risk of in-hospital death for patients admitted through emergency departments or via referrals than for those admitted through other routes (OR = 3.76, 95% CI: 1.84-7.67). Deaths were higher in hospitals with volumes of less than 50 patients with isolated MMA during the five study years (OR = 2.92, 95% CI: 1.46-5.83). Moreover, the risk of in-hospital death gradually decreased over time (OR = 0.72, 95% CI: 0.57-0.90). In the multivariate analysis, the abovementioned associations with the risk of in-hospital death remained statistically significant. However, no significant associations were observed between specific clinical signs and in-hospital death in either the univariate or the multivariate analysis.
Younger age, admission to hospitals with low patient volumes, and admission through emergency departments or referrals are associated with higher risk of in-hospital death. The co-existence of specific clinical signs appears to have no effect on in-hospital death.
孤立性甲基丙二酸血症（MMA）患者通常会反复发作急性代谢失代偿或代谢性卒中，需要频繁住院，死亡率相对较高。我们的研究旨在评估预测孤立性 MMA 儿科患者住院期间死亡的因素。我们使用中国国家住院患者数据库（2013 年至 2017 年）中的医院质量监测系统数据进行了回顾性研究。纳入所有年龄在 18 岁以下诊断为孤立性 MMA 的患者。收集人口统计学、医院相关和临床特征。采用泊松回归分析确定与住院死亡相关的潜在影响因素。
2013 年至 2017 年，2317 例儿科患者因孤立性 MMA 住院，其中 1.77%的患者死亡。在单因素分析中，年龄在 1 岁以下的患者死亡风险高于年龄在 1 岁或以上的患者（比值比 [OR] = 2.63，95%置信区间 [CI]：1.36-5.07）。通过急诊或转科入院的患者住院死亡风险高于其他途径入院的患者（OR = 3.76，95%CI：1.84-7.67）。在五年研究期间，收治 MMA 患者少于 50 人的医院死亡人数较高（OR = 2.92，95%CI：1.46-5.83）。此外，住院期间死亡风险随时间逐渐降低（OR = 0.72，95%CI：0.57-0.90）。多因素分析显示，上述与住院期间死亡风险的关联仍具有统计学意义。然而，在单因素或多因素分析中，特定临床体征与住院期间死亡之间均无显著关联。
年龄较小、收入量少的医院和通过急诊或转科入院与住院期间死亡风险增加相关。特定临床体征的共存似乎对住院期间死亡没有影响。
