6533b832fe1ef96bd129a3d4

RESEARCH PRODUCT

Disease status, reasons for discontinuation and adverse events in 1038 Italian children with juvenile idiopathic arthritis treated with etanercept

Marta TorcolettiAchille MarinoFrancesco La TorreGiovanni ContiPatrizia BaroneDonato RiganteFrancesco ZulianFrancesca RovelliAngelo RavelliAngelo RavelliFabrizio De BenedettiGianfranco D'angeloAdele CivinoAlessandro De FantiRebecca NicolaiValeria GerloniIrene PontikakiSilvia Magni-manzoniFrancesca BovisMaria Cristina MaggioNicolino RupertoSerena PastoreC FedeDenise Pires MarafonAlberto MartiniAlberto MartiniChiara SandrinFabrizia CoronaRomina GallizziSergio DavìMg AlpigianiRita ConsoliniSara VerazzaAlessandro ConsolaroAlessandro ConsolaroGiorgia MartiniAntonella InsalacoMauro JoriniAlma Nunzia OlivieriRolando CimazRosanna PoddaValentina MuratoreLuciana BredaMarco CattaliniElisabetta Cortis

subject

MaleBiologic therapieBiologic therapies; Etanercept; Juvenile idiopathic arthritis; Pediatric rheumatology; TNF inhibitors; Adolescent; Antirheumatic Agents; Arthritis Juvenile; Child; Child Preschool; Cross-Sectional Studies; Drug Substitution; Etanercept; Female; Humans; Male; Methotrexate; Patient Outcome Assessment; Retrospective Studies; Treatment Outcome; Pediatrics Perinatology and Child Health; Rheumatology; Immunology and AllergyArthritisJuvenilePediatricsInflammatory bowel diseaseEtanerceptEtanerceptTNF inhibitorsSettore MED/38 - Pediatria Generale E Specialistica0302 clinical medicineQuality of lifeRetrospective StudieImmunology and AllergyPediatric rheumatology030212 general & internal medicineChildBiologic therapies; Etanercept; Juvenile idiopathic arthritis; Pediatric rheumatology; TNF inhibitors; Pediatrics Perinatology and Child Health; Immunology and Allergy; RheumatologyDrug SubstitutionBiologic therapies; Etanercept; Juvenile idiopathic arthritis; Pediatric rheumatology; TNF inhibitors; Adolescent; Antirheumatic Agents; Arthritis Juvenile; Child; Child Preschool; Cross-Sectional Studies; Drug Substitution; Etanercept; Female; Humans; Male; Methotrexate; Patient Outcome Assessment; Retrospective Studies; Treatment Outcome; Pediatrics Perinatology and Child Health; Immunology and Allergy; RheumatologyAntirheumatic AgentPerinatology and Child Health3. Good healthTreatment OutcomeSettore MED/38 - PEDIATRIA GENERALE E SPECIALISTICAChild PreschoolAntirheumatic AgentsFemaleResearch ArticleHumanmedicine.drugBiologic therapies; Etanercept; Juvenile idiopathic arthritis; Pediatric rheumatology; TNF inhibitors; Adolescent; Antirheumatic Agents; Arthritis Juvenile; Child; Child Preschool; Cross-Sectional Studies; Drug Substitution; Etanercept; Female; Humans; Male; Methotrexate; Patient Outcome Assessment; Retrospective Studies; Treatment Outcomemedicine.medical_specialtyAdolescent03 medical and health sciencesJuvenile idiopathic arthritiRheumatologyInternal medicineBiologic therapies; Etanercept; Juvenile idiopathic arthritis; Pediatric rheumatology; TNF inhibitorsmedicineHumansPediatrics Perinatology and Child HealthAdverse effectPreschoolRetrospective StudiesCross-Sectional Studie030203 arthritis & rheumatologybusiness.industryArthritisRetrospective cohort studyJuvenile idiopathic arthritismedicine.diseaseArthritis JuvenileRheumatologyDiscontinuationPatient Outcome AssessmentBiologic therapiesCross-Sectional StudiesMethotrexatePediatrics Perinatology and Child HealthPhysical therapybusinessTNF inhibitor

description

Background: Data from routine clinical practice are needed to further define the efficacy and safety of biologic medications in children with juvenile idiopathic arthritis (JIA). The aim of this analysis was to investigate the disease status, reasons for discontinuation and adverse events in Italian JIA patients treated with etanercept (ETN). Methods: In 2013, all centers of the Italian Pediatric Rheumatology Study Group were asked to make a census of patients given ETN after January 2000. Patients were classified in three groups: group 1 = patients still taking ETN; group 2 = patients discontinued from ETN for any reasons; group 3 = patients lost to follow-up while receiving ETN. All three groups received a retrospective assessment; patients in group 1 also underwent a cross-sectional assessment. Results: 1038 patients were enrolled by 23 centers: 422 (40.7%) were in group 1, 462 (44.5%) in group 2, and 154 (14.8%) in group 3. Median duration of ETN therapy was 2.5 years. At cross-sectional assessment, 41.8% to 48.6% of patients in group 1 met formal criteria for inactive disease, whereas 52.4% of patients in group 2 and 55.8% of patients in group 3 were judged in clinical remission by their caring physician at last visit. A relatively greater proportion of patients with systemic arthritis were discontinued or lost to follow-up. Parent evaluations at cross-sectional visit in group 1 showed that 52.4% of patients had normal physical function, very few had impairment in quality of life, 51.2% had no pain, 76% had no morning stiffness, and 82.7% of parents were satisfied with their child's illness outcome. Clinically significant adverse events were reported for 27.8% of patients and ETN was discontinued for side effects in 9.5%. The most common adverse events were new onset or recurrent uveitis (10.2%), infections (6.6%), injection site reactions (4.4%), and neuropsychiatric (3.1%), gastrointestinal (2.4%), and hematological disorders (2.1%). Ten patients developed an inflammatory bowel disease and 2 had a malignancy. One patient died of a fulminant streptococcal sepsis. Conclusions: Around half of the patients achieved complete disease quiescence under treatment with ETN. The medication was overall well tolerated, as only one quarter of patients experienced clinically significant adverse events and less than 10% had treatment discontinued for toxicity.

10.1186/s12969-016-0126-0http://hdl.handle.net/11577/3331179