Clinical manifestations, healthcare resource utilization, and costs among patients with long-chain fatty acid oxidation disorders: a retrospective claims database analysis.

IF 4.6 Q2 MATERIALS SCIENCE, BIOMATERIALS ACS Applied Bio Materials Pub Date : 2024-09-18 DOI:10.1080/03007995.2024.2405124
Erru Christy Yang,Zhenzhen Fang,Ruixin Tan,Yun Guo,Siyi He,Eliza Kruger,Justin Nedzesky,Deborah Marsden
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Abstract

OBJECTIVE Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of rare genetic inborn errors of metabolism. Clinical manifestations may result in frequent healthcare visits, hospitalizations, and early death. This retrospective cohort study assessed manifestations, healthcare resource use (HRU), direct medical costs, and the impact of COVID-19 on HRU among patients with LC-FAOD. METHODS The IQVIA PharMetrics Plus database was searched for pediatric (0-17 years) and adult (≥18 years) patients with confirmed LC-FAOD (ICD-10-CM Diagnosis Code E71.310) and ≥12 months continuous enrollment (CE) between January 2016-February 2020. A non-LC-FAOD general population cohort was randomly selected and matched using 1:20 exact matching on age, gender, payer type, and CE start year. Manifestations were identified via ICD-10 diagnosis codes (any billing position). Overall HRU and attributable costs were stratified by care setting. Pre-COVID-19 (March 2019-February 2020) and during COVID-19 (March 2020-February 2021) HRU was assessed among a subgroup of patients and the general population. Outcomes were evaluated among children and adults, respectively. RESULTS 423 patients with LC-FAOD (47% female; 79.7% children) were included. Mean enrollment duration was 2.6 ± 1.2 years. 22.6% of children with LC-FAOD had at least one major clinical event (MCE), consisting of rhabdomyolysis (10.1%), hypoglycemia (9.8%), or cardiomyopathy (8.6%) versus 1.5% overall occurrence in the general population. Adults with LC-FAOD had higher incidence of MCEs (37.2%) than children with LC-FAOD. Annualized all-cause HRU in all care settings and mean total annualized medical costs (children: $17,082 vs $4,144; adults: $43,602 vs $3,949) were higher in patients with LC-FAOD versus the general population. Patients with LC-FAOD had substantially fewer healthcare visits during COVID-19 across care settings than during the pre-COVID-19 period. CONCLUSIONS LC-FAOD impart a high burden on patients. Extended hospital stays and increased outpatient management were especially pronounced for adults and for patients with ≥1 MCE, resulting in substantially higher medical costs than the general population.
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长链脂肪酸氧化紊乱患者的临床表现、医疗资源利用率和成本:一项回顾性索赔数据库分析。
目的长链脂肪酸氧化紊乱(LC-FAOD)是一组罕见的遗传性先天性代谢错误。临床表现可导致频繁就医、住院和过早死亡。这项回顾性队列研究评估了LC-FAOD患者的表现、医疗资源使用(HRU)、直接医疗费用以及COVID-19对HRU的影响。方法在IQVIA PharMetrics Plus数据库中搜索了2016年1月至2020年2月期间确诊为LC-FAOD(ICD-10-CM诊断代码E71.310)且连续入组(CE)时间≥12个月的儿童(0-17岁)和成人(≥18岁)患者。随机选取了非 LC-FAOD 普通人群队列,并根据年龄、性别、付款人类型和 CE 开始年份采用 1:20 精确配对法进行匹配。通过 ICD-10 诊断代码(任何计费位置)确定病症表现。总体 HRU 和可归因成本按护理环境进行分层。在 COVID-19 之前(2019 年 3 月至 2020 年 2 月)和 COVID-19 期间(2020 年 3 月至 2021 年 2 月),对患者亚群和一般人群的 HRU 进行了评估。结果纳入了 423 名 LC-FAOD 患者(47% 为女性;79.7% 为儿童)。平均入组时间为 2.6 ± 1.2 年。22.6%的LC-FAOD儿童至少发生过一次重大临床事件(MCE),包括横纹肌溶解(10.1%)、低血糖(9.8%)或心肌病(8.6%),而普通人群的总体发生率为1.5%。成人 LC-FAOD 患者的 MCE 发生率(37.2%)高于儿童 LC-FAOD患者。与普通人群相比,LC-FAOD 患者在所有医疗环境中的年化全因 HRU 和平均年化医疗总费用(儿童:17,082 美元 vs 4,144 美元;成人:43,602 美元 vs 3,949 美元)都更高。在 COVID-19 期间,LC-FAOD 患者在各种医疗机构就诊的次数大大少于 COVID-19 前。延长住院时间和增加门诊管理对成人和≥1个MCE的患者尤为明显,导致医疗费用大大高于普通人群。
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来源期刊
ACS Applied Bio Materials
ACS Applied Bio Materials Chemistry-Chemistry (all)
CiteScore
9.40
自引率
2.10%
发文量
464
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