RESUMO
BACKGROUND AND AIMS: We aimed to assess cost-effectiveness of increasing adalimumab dose intervals compared to the conventional dosing interval in patients with Crohn's disease [CD] in stable clinical and biochemical remission. DESIGN: We conducted a pragmatic, open-label, randomized controlled non-inferiority trial, comparing increased adalimumab intervals with the 2-weekly interval in adult CD patients in clinical remission. Quality of life was measured with the EQ-5D-5L. Costs were measured from a societal perspective. Results are shown as differences and incremental net monetary benefit [iNMB] at relevant willingness to accept [WTA] levels. RESULTS: We randomized 174 patients to the intervention [nâ =â 113] and control [nâ =â 61] groups. No difference was found in utility (difference: -0.017, 95% confidence interval [-0.044; 0.004]) and total costs (-943, [-2226; 1367]) over the 48-week study period between the two groups. Medication costs per patient were lower (-2545, [-2780; -2192]) in the intervention group, but non-medication healthcare (+474, [+149; +952]) and patient costs (+365 [+92; 1058]) were higher. Cost-utility analysis showed that the iNMB was 594 [-2099; 2050], 69 [-2908; 1965] and -455 [-4,096; 1984] at WTA levels of 20 000, 50 000 and 80 000, respectively. Increasing adalimumab dose intervals was more likely to be cost-effective at WTA levels below 53 960 per quality-adjusted life year. Above 53 960 continuing the conventional dose interval was more likely to be cost-effective. CONCLUSION: When the loss of a quality-adjusted life year is valued at less than 53 960, increasing the adalimumab dose interval is a cost-effective strategy in CD patients in stable clinical and biochemical remission. CLINICAL TRIAL REGISTRATION NUMBER: ClinicalTrials.gov, number NCT03172377.
Assuntos
Doença de Crohn , Adulto , Humanos , Adalimumab/uso terapêutico , Doença de Crohn/tratamento farmacológico , Análise de Custo-Efetividade , Qualidade de Vida , Anticorpos Monoclonais Humanizados/uso terapêutico , Resultado do Tratamento , Análise Custo-BenefícioRESUMO
BACKGROUND AND AIMS: Histological remission [HR] is a potential treatment target in ulcerative colitis [UC]. Limited 'real world' data are available on the reliability of histological scoring when assessing minimal histological inflammation. The aim of this study was to investigate the reliability of UC histological scores in colonic biopsies showing mucosal healing [MH] and limited histological inflammation, and to compare the 'daily practice' histological assessment with expert reviews by gastrointestinal [GI] pathologists. METHODS: We performed a retrospective single-centre study. Colonic biopsies from UC patients with MH [Mayo score ≤ 1] were included. All biopsies assessed in daily practice were reassessed by three blinded GI pathologists using three histological scores (Geboes score [GS], Riley score [RS], Harpaz [Gupta] Index [HGI]) and a global visual scale [GVS]. We evaluated inter- and intra-observer variation between GI pathologists and correlations between scores including the initial histological assessment using Cronbach's alpha and Spearman rho analysis. RESULTS: In total, 270 biopsies from 39 UC patients were included. The inter-observer concordance for all histological indexes was substantial to almost perfect [GS 0.84; HGI 0.61; GVS 0.74, RS 0.91]. Correlation between the RS and GS was almost perfect [R = 0.86], but we found no correlation between the primary histological assessment and reassessment by GI pathologists. CONCLUSIONS: Current UC histological scores reliably assess limited histological inflammation in UC patients. The discrepancy between the initial histological assessment and the reassessment by dedicated GI pathologists suggests a gap between daily practice and academic expertise. This issue may limit the implementation of HR as a treatment target for UC in daily practice.