Patrizia Alvisi, Serena Arrigo, Salvatore Cucchiara, Paolo Lionetti, Erasmo Miele, Claudio Romano, Alberto Ravelli, Daniela Knafelz, Stefano Martelossi, Graziella Guariso, Salvatore Accomando, Giovanna Zuin, Costantino De Giacomo, Lucio Balzani, Monia Gennari, Marina Aloi
{"title":"阿达木单抗作为二线治疗在英夫利昔单抗治疗失败的克罗恩病儿童队列中的疗效:意大利儿科胃肠病学、肝病学和营养学学会的经验","authors":"Patrizia Alvisi, Serena Arrigo, Salvatore Cucchiara, Paolo Lionetti, Erasmo Miele, Claudio Romano, Alberto Ravelli, Daniela Knafelz, Stefano Martelossi, Graziella Guariso, Salvatore Accomando, Giovanna Zuin, Costantino De Giacomo, Lucio Balzani, Monia Gennari, Marina Aloi","doi":"10.2147/BTT.S183088","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Adalimumab (Ada) treatment is an available option for pediatric Crohn's disease (CD) and the published experience as rescue therapy is limited.</p><p><strong>Objectives: </strong>We investigated Ada efficacy in a retrospective, pediatric CD cohort who had failed previous infliximab treatment, with a minimum follow-up of 6 months.</p><p><strong>Methods: </strong>In this multicenter study, data on demographics, clinical activity, growth, laboratory values (CRP) and adverse events were collected from CD patients during follow-up. Clinical remission (CR) and response were defined with Pediatric CD Activity Index (PCDAI) score ≤10 and a decrease in PCDAI score of ≥12.5 from baseline, respectively.</p><p><strong>Results: </strong>A total of 44 patients were consecutively recruited (mean age 14.8 years): 34 of 44 (77%) had active disease (mean PCDAI score 24.5) at the time of Ada administration, with a mean disease duration of 3.4 (range 0.3-11.2) years. At 6, 12, and 18 months, out of the total of the enrolled population, CR rates were 55%, 78%, and 52%, respectively, with a significant decrease in PCDAI scores (<i>P</i><0.01) and mean CRP values (mean CRP 5.7 and 2.4 mL/dL, respectively; <i>P</i><0.01) at the end of follow-up. Steroid-free remission rates, considered as the total number of patients in CR who were not using steroids at the end of this study, were 93%, 95%, and 96% in 44 patients at 6, 12, and 18 months, respectively. No significant differences in growth parameters were detected. In univariate analysis of variables related to Ada efficacy, we found that only a disease duration >2 years was negatively correlated with final PCDAI score (<i>P</i><0.01). Two serious adverse events were recorded: 1 meningitis and 1 medulloblastoma.</p><p><strong>Conclusion: </strong>Our data confirm Ada efficacy in pediatric patients as second-line biological therapy after infliximab failure. Longer-term prospective data are warranted to define general effectiveness and safety in pediatric CD patients.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":"13 ","pages":"13-21"},"PeriodicalIF":5.3000,"publicationDate":"2019-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/BTT.S183088","citationCount":"7","resultStr":"{\"title\":\"Efficacy of adalimumab as second-line therapy in a pediatric cohort of Crohn's disease patients who failed infliximab therapy: the Italian Society of Pediatric Gastroenterology, Hepatology, and Nutrition experience.\",\"authors\":\"Patrizia Alvisi, Serena Arrigo, Salvatore Cucchiara, Paolo Lionetti, Erasmo Miele, Claudio Romano, Alberto Ravelli, Daniela Knafelz, Stefano Martelossi, Graziella Guariso, Salvatore Accomando, Giovanna Zuin, Costantino De Giacomo, Lucio Balzani, Monia Gennari, Marina Aloi\",\"doi\":\"10.2147/BTT.S183088\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Adalimumab (Ada) treatment is an available option for pediatric Crohn's disease (CD) and the published experience as rescue therapy is limited.</p><p><strong>Objectives: </strong>We investigated Ada efficacy in a retrospective, pediatric CD cohort who had failed previous infliximab treatment, with a minimum follow-up of 6 months.</p><p><strong>Methods: </strong>In this multicenter study, data on demographics, clinical activity, growth, laboratory values (CRP) and adverse events were collected from CD patients during follow-up. Clinical remission (CR) and response were defined with Pediatric CD Activity Index (PCDAI) score ≤10 and a decrease in PCDAI score of ≥12.5 from baseline, respectively.</p><p><strong>Results: </strong>A total of 44 patients were consecutively recruited (mean age 14.8 years): 34 of 44 (77%) had active disease (mean PCDAI score 24.5) at the time of Ada administration, with a mean disease duration of 3.4 (range 0.3-11.2) years. At 6, 12, and 18 months, out of the total of the enrolled population, CR rates were 55%, 78%, and 52%, respectively, with a significant decrease in PCDAI scores (<i>P</i><0.01) and mean CRP values (mean CRP 5.7 and 2.4 mL/dL, respectively; <i>P</i><0.01) at the end of follow-up. Steroid-free remission rates, considered as the total number of patients in CR who were not using steroids at the end of this study, were 93%, 95%, and 96% in 44 patients at 6, 12, and 18 months, respectively. No significant differences in growth parameters were detected. In univariate analysis of variables related to Ada efficacy, we found that only a disease duration >2 years was negatively correlated with final PCDAI score (<i>P</i><0.01). Two serious adverse events were recorded: 1 meningitis and 1 medulloblastoma.</p><p><strong>Conclusion: </strong>Our data confirm Ada efficacy in pediatric patients as second-line biological therapy after infliximab failure. Longer-term prospective data are warranted to define general effectiveness and safety in pediatric CD patients.</p>\",\"PeriodicalId\":9025,\"journal\":{\"name\":\"Biologics : Targets & Therapy\",\"volume\":\"13 \",\"pages\":\"13-21\"},\"PeriodicalIF\":5.3000,\"publicationDate\":\"2019-01-03\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.2147/BTT.S183088\",\"citationCount\":\"7\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Biologics : Targets & Therapy\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.2147/BTT.S183088\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2019/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q1\",\"JCRName\":\"MEDICINE, RESEARCH & EXPERIMENTAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Biologics : Targets & Therapy","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.2147/BTT.S183088","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2019/1/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
Efficacy of adalimumab as second-line therapy in a pediatric cohort of Crohn's disease patients who failed infliximab therapy: the Italian Society of Pediatric Gastroenterology, Hepatology, and Nutrition experience.
Background: Adalimumab (Ada) treatment is an available option for pediatric Crohn's disease (CD) and the published experience as rescue therapy is limited.
Objectives: We investigated Ada efficacy in a retrospective, pediatric CD cohort who had failed previous infliximab treatment, with a minimum follow-up of 6 months.
Methods: In this multicenter study, data on demographics, clinical activity, growth, laboratory values (CRP) and adverse events were collected from CD patients during follow-up. Clinical remission (CR) and response were defined with Pediatric CD Activity Index (PCDAI) score ≤10 and a decrease in PCDAI score of ≥12.5 from baseline, respectively.
Results: A total of 44 patients were consecutively recruited (mean age 14.8 years): 34 of 44 (77%) had active disease (mean PCDAI score 24.5) at the time of Ada administration, with a mean disease duration of 3.4 (range 0.3-11.2) years. At 6, 12, and 18 months, out of the total of the enrolled population, CR rates were 55%, 78%, and 52%, respectively, with a significant decrease in PCDAI scores (P<0.01) and mean CRP values (mean CRP 5.7 and 2.4 mL/dL, respectively; P<0.01) at the end of follow-up. Steroid-free remission rates, considered as the total number of patients in CR who were not using steroids at the end of this study, were 93%, 95%, and 96% in 44 patients at 6, 12, and 18 months, respectively. No significant differences in growth parameters were detected. In univariate analysis of variables related to Ada efficacy, we found that only a disease duration >2 years was negatively correlated with final PCDAI score (P<0.01). Two serious adverse events were recorded: 1 meningitis and 1 medulloblastoma.
Conclusion: Our data confirm Ada efficacy in pediatric patients as second-line biological therapy after infliximab failure. Longer-term prospective data are warranted to define general effectiveness and safety in pediatric CD patients.
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