一项随机交叉试验，以评估大学医院生产的熊去氧胆酸治疗原发性胆汁性胆管炎的疗效及成本降低情况。
A randomized crossover trial to assess therapeutic efficacy and cost reduction of acid ursodeoxycholic manufactured by the university hospital for the treatment of primary biliary cholangitis.
作者信息
Nakano Larissa Akeme, Cançado Eduardo Luiz Rachid, Chaves Cleuber Esteves, Madeira Maria Cristina Vaz, Katayose Jéssica Toshie, Nabeshima Mariana Akemi, Fossaluza Victor, Uhrigshardt Gabriela Guimarães, Liting Zheng, Pinto Vanusa Barbosa, Carrilho Flair José, Ono Suzane Kioko
机构信息
Department of Gastroenterology, Division of Clinical Gastroenterology and Hepatology, Hospital das Clinicas, University of São Paulo School of Medicine, Av. Dr. Enéas Carvalho de Aguiar, 255, ICHC, 9th Floor, office 9159, São Paulo, SP 05403-000, Brazil.
Laboratory of Medical Investigation LIM 06, Institute of Tropical Medicine, University of São Paulo, São Paulo, Brazil.
出版信息
BMC Gastroenterol. 2020 Aug 5;20(1):253. doi: 10.1186/s12876-020-01399-5.
BACKGROUND
Health care costs are growing faster than the rest of the global economy, according to the World Health Organization (WHO). Countries' health expenditures include paying for general medicine, diagnostic procedures, hospitalizations and surgeries, as well as medications and prescribed treatment. Primary biliary cholangitis (PBC) is a rare autoimmune liver disease and the first line available treatment is ursodeoxycholic acid (UDCA), however, direct and indirect treatment costs are expensive. Main aim of this trial was to assess if the therapeutic efficacy of UDCA manufactured by the university hospital is equivalent to that of standard UDCA and treatment cost reduction in patients with PBC.
METHODS
It is a prospective, interventional, randomized, and crossover study in patients diagnosed with PBC. UDCA 300 mg tablets and capsules were developed and manufactured by the university hospital. Thirty patients under treatment with standard UDCA, in stable doses were randomized in sequence A and B, 15 patients in each arm. The groups were treated for 12 weeks and after, the UDCA formulation was changed, following for another 12 weeks of continuous therapy (tablets and capsules / capsules and tablets). Laboratory tests were performed at time T0 (beginning of treatment), T1 (at the 12 week-therapy, before the crossing-over) and T2 (end of treatment). The evaluation was done by comparing the hepatic parameters ALP, GGT, ALT, AST and total bilirubin, also considering the adverse events. The comparison of costs was based on price of the manufactured UDCA and standard UDCA price of the hospital.
RESULTS
Hospital reduced 66.1% the PBC treatment costs using manufactured UDCA. There were no differences in the biochemical parameters between sequence (A and B) and tablets or capsules of UDCA formulations applied in the treatment of PBC.
CONCLUSIONS
The study showed that there was no significant difference between manufactured UDCA (capsule and tablet) and standard UDCA. Hospital reduced the PBC treatment costs using the manufactured UDCA by the university hospital.
TRIAL REGISTRATION
ClinicalTrials.gov: NCT03489889 retrospectively registered on January 12th, 2018; Ethics Committee approved the study (ID: 1.790.088) on October 25th, 2016.
背景
根据世界卫生组织（WHO）的数据，医疗保健成本的增长速度超过了全球经济的其他领域。各国的医疗支出包括支付普通药物、诊断程序、住院和手术费用，以及药物和规定治疗的费用。原发性胆汁性胆管炎（PBC）是一种罕见的自身免疫性肝病，一线可用治疗药物是熊去氧胆酸（UDCA），然而，直接和间接治疗成本都很高。本试验的主要目的是评估大学医院生产的UDCA的治疗效果是否等同于标准UDCA，以及能否降低PBC患者的治疗成本。
方法
这是一项针对确诊为PBC患者的前瞻性、干预性、随机交叉研究。大学医院研发并生产了300毫克的UDCA片剂和胶囊。30名接受标准UDCA稳定剂量治疗的患者按顺序A和B随机分组，每组15名。两组接受12周治疗，之后更换UDCA制剂，继续治疗12周（片剂和胶囊/胶囊和片剂）。在T0（治疗开始时）、T1（12周治疗时，换药前）和T2（治疗结束时）进行实验室检查。通过比较肝参数碱性磷酸酶（ALP）、γ-谷氨酰转肽酶（GGT）、谷丙转氨酶（ALT）、谷草转氨酶（AST）和总胆红素，并考虑不良事件来进行评估。成本比较基于生产的UDCA价格和医院标准UDCA价格。
结果
使用生产的UDCA，医院将PBC治疗成本降低了66.1%。在治疗PBC时，UDCA制剂的序列（A和B）以及片剂或胶囊之间的生化参数没有差异。
结论
研究表明，生产的UDCA（胶囊和片剂）与标准UDCA之间没有显著差异。大学医院生产的UDCA降低了医院的PBC治疗成本。
试验注册
ClinicalTrials.gov：NCT03489889于2018年1月12日进行回顾性注册；伦理委员会于2016年10月25日批准该研究（编号：1.790.088）。
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