Abstract：
Data on cost-effectiveness of first-line infliximab in paediatric patients with Crohn\'s disease are limited. Since biologics are increasingly prescribed and accompanied by high costs, this knowledge gap needs to be addressed.
To investigate the cost-effectiveness of first-line infliximab compared to conventional treatment in children with moderate-to-severe Crohn\'s disease.
We included patients from the Top-down Infliximab Study in Kids with Crohn\'s disease randomised controlled trial. Children with newly diagnosed moderate-to-severe Crohn\'s disease were treated with azathioprine maintenance and either five induction infliximab (biosimilar) infusions or conventional induction treatment (exclusive enteral nutrition or corticosteroids). Direct healthcare consumption and costs were obtained per patient until week 104. This included data on outpatient hospital visits, hospital admissions, drug costs, endoscopies and surgeries. The primary health outcome was the odds ratio of being in clinical remission (weighted paediatric Crohn\'s disease activity index<12.5) during 104 weeks.
We included 89 patients (44 in the first-line infliximab group and 45 in the conventional treatment group). Mean direct healthcare costs per patient were €36,784 for first-line infliximab treatment and €36,874 for conventional treatment over 2 years (p = 0.981). The odds ratio of first-line infliximab versus conventional treatment to be in clinical remission over 104 weeks was 1.56 (95%CI 1.03-2.35, p = 0.036).
First-line infliximab treatment resulted in higher odds of being in clinical remission without being more expensive, making it the dominant strategy over conventional treatment in the first 2 years after diagnosis in children with moderate-to-severe Crohn\'s disease.
NCT02517684.
To investigate the cost-effectiveness of first-line infliximab compared to conventional treatment in children with moderate-to-severe Crohn\'s disease.
We included patients from the Top-down Infliximab Study in Kids with Crohn\'s disease randomised controlled trial. Children with newly diagnosed moderate-to-severe Crohn\'s disease were treated with azathioprine maintenance and either five induction infliximab (biosimilar) infusions or conventional induction treatment (exclusive enteral nutrition or corticosteroids). Direct healthcare consumption and costs were obtained per patient until week 104. This included data on outpatient hospital visits, hospital admissions, drug costs, endoscopies and surgeries. The primary health outcome was the odds ratio of being in clinical remission (weighted paediatric Crohn\'s disease activity index<12.5) during 104 weeks.
We included 89 patients (44 in the first-line infliximab group and 45 in the conventional treatment group). Mean direct healthcare costs per patient were €36,784 for first-line infliximab treatment and €36,874 for conventional treatment over 2 years (p = 0.981). The odds ratio of first-line infliximab versus conventional treatment to be in clinical remission over 104 weeks was 1.56 (95%CI 1.03-2.35, p = 0.036).
First-line infliximab treatment resulted in higher odds of being in clinical remission without being more expensive, making it the dominant strategy over conventional treatment in the first 2 years after diagnosis in children with moderate-to-severe Crohn\'s disease.
NCT02517684.
摘要：
背景：在克罗恩病儿科患者中一线英夫利昔单抗的成本效益数据有限。由于生物制剂的处方越来越多，并且伴随着高昂的成本，这种知识差距需要解决。
目的：探讨中重度克罗恩病患儿一线英夫利昔单抗与常规治疗相比的成本-效果。
方法：我们纳入了Top-downInfliximab研究儿童克罗恩病随机对照试验的患者。新诊断为中度至重度克罗恩病的儿童接受硫唑嘌呤维持治疗和5次诱导英夫利昔单抗（生物仿制药）输注或常规诱导治疗（独家肠内营养或皮质类固醇）。直到第104周，每位患者都获得了直接的医疗保健消费和费用。这包括门诊就诊的数据，入院,药费,内窥镜检查和手术。主要健康结果是104周内临床缓解（加权小儿克罗恩病活动指数<12.5）的比值比。
结果：我们纳入了89例患者（一线英夫利昔单抗组44例，常规治疗组45例）。在2年内，一线英夫利昔单抗治疗每位患者的平均直接医疗费用为36,784欧元，常规治疗为36,874欧元（p=0.981）。一线英夫利昔单抗与常规治疗在104周内临床缓解的比值比为1.56（95CI1.03-2.35，p=0.036）。
结论：一线英夫利昔单抗治疗在不更昂贵的情况下获得临床缓解的几率更高，在中重度克罗恩病患儿诊断后的前2年内，使其成为常规治疗的主导策略。
背景：NCT02517684。
目的：探讨中重度克罗恩病患儿一线英夫利昔单抗与常规治疗相比的成本-效果。
方法：我们纳入了Top-downInfliximab研究儿童克罗恩病随机对照试验的患者。新诊断为中度至重度克罗恩病的儿童接受硫唑嘌呤维持治疗和5次诱导英夫利昔单抗（生物仿制药）输注或常规诱导治疗（独家肠内营养或皮质类固醇）。直到第104周，每位患者都获得了直接的医疗保健消费和费用。这包括门诊就诊的数据，入院,药费,内窥镜检查和手术。主要健康结果是104周内临床缓解（加权小儿克罗恩病活动指数<12.5）的比值比。
结果：我们纳入了89例患者（一线英夫利昔单抗组44例，常规治疗组45例）。在2年内，一线英夫利昔单抗治疗每位患者的平均直接医疗费用为36,784欧元，常规治疗为36,874欧元（p=0.981）。一线英夫利昔单抗与常规治疗在104周内临床缓解的比值比为1.56（95CI1.03-2.35，p=0.036）。
结论：一线英夫利昔单抗治疗在不更昂贵的情况下获得临床缓解的几率更高，在中重度克罗恩病患儿诊断后的前2年内，使其成为常规治疗的主导策略。
背景：NCT02517684。
