Subcutaneous dosing regimens of tocilizumab in children with systemic or polyarticular juvenile idiopathic arthritis.


Por: Ruperto N, Brunner HI, Ramanan AV, Horneff G, Cuttica R, Henrickson M, Anton-Lopez J, Boteanu AL, Penades IC, Minden K, Schmeling H, Hufnagel M, Weiss JE, Pardeo M, Nanda K, Roth J, Rubio-Pérez N, Hsu JC, Wimalasundera S, Wells C, Bharucha K, Douglass W, Bao M, Mallalieu NL, Martini A, Lovell D, De Benedetti F and Paediatric Rheumatology INternational Trials Organisation (PRINTO) and the Pedia

Publicada: 2 oct 2021 Ahead of Print: 28 ene 2021
Resumen:
OBJECTIVES: To determine s.c. tocilizumab (s.c.-TCZ) dosing regimens for systemic JIA (sJIA) and polyarticular JIA (pJIA). METHODS: In two 52-week phase 1 b trials, s.c.-TCZ (162 mg/dose) was administered to sJIA patients every week or every 2 weeks (every 10 days before interim analysis) and to pJIA patients every 2 weeks or every 3 weeks with body weight =30 kg or <30 kg, respectively. Primary end points were pharmacokinetics, pharmacodynamics and safety; efficacy was exploratory. Comparisons were made to data from phase 3 trials with i.v. tocilizumab (i.v.-TCZ) in sJIA and pJIA. RESULTS: Study participants were 51 sJIA patients and 52 pJIA patients aged 1-17 years who received s.c.-TCZ. Steady-state minimum TCZ concentration (Ctrough) >5th percentile of that achieved with i.v.-TCZ was achieved by 49 (96%) sJIA and 52 (100%) pJIA patients. In both populations, pharmacodynamic markers of disease were similar between body weight groups. Improvements in Juvenile Arthritis DAS-71 were comparable between s.c.-TCZ and i.v.-TCZ. By week 52, 53% of sJIA patients and 31% of pJIA patients achieved clinical remission on treatment. Safety was consistent with that of i.v.-TCZ except for injection site reactions, reported by 41.2% and 28.8% of sJIA and pJIA patients, respectively. Infections were reported in 78.4% and 69.2% of patients, respectively. Two sJIA patients died; both deaths were considered to be related to TCZ. CONCLUSION: s.c.-TCZ provides exposure and risk/benefit profiles similar to those of i.v.-TCZ. S.c. administration provides an alternative administration route that is more convenient for patients and caregivers and that has potential for in-home use. TRIAL REGISTRATION: ClinicalTrials.gov, http://clinicaltrials.gov, NCT01904292 and NCT01904279.

Filiaciones:
Ruperto N:
 IRCCS Istituto Giannina Gaslini, Clinica Pediatrica e Reumatologia-PRINTO, Genoa, Italy

Brunner HI:
 Pediatric Rheumatology Collaborative Study Group (PRCSG), Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA

Ramanan AV:
 University Hospitals Bristol NHS Foundation Trust & Bristol Medical School, University of Bristol, Bristol, UK

Horneff G:
 Department of General Paediatrics, Asklepios Clinic Sankt Augustin, Sankt Augustin, Germany

 Department of Paediatric and Adolescents Medicine, University Hospital of Cologne, Cologne, Germany

Cuttica R:
 Rheumatology Section, Hospital Pedro de Elizalde, Buenos Aires, Argentina

Henrickson M:
 Pediatric Rheumatology Collaborative Study Group (PRCSG), Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA

Anton-Lopez J:
 Hospital Sant Joan de Déu, Universitat de Barcelona, Unidad de Reumatología Pediátrica, Esplugues de Llobregat (Barcelona), Spain

Boteanu AL:
 Pediatric Rheumatology Unit, University Hospital Ramón y Cajal, Madrid, Spain

Penades IC:
 Pediatric Rheumatology Unit, Hospital Universitario y Politécnico La Fe, Valencia, Spain

Minden K:
 German Rheumatism Research Centre Berlin, and Department of Rheumatology and Clinical Immunology, Charité University Medicine, Berlin, Germany

Schmeling H:
 Department of Pediatrics, Alberta Children's Hospital and Cumming School of Medicine/University of Calgary, Alberta, Canada

Hufnagel M:
 University Medical Center Freiburg, Department of Pediatrics and Adolescent Medicine, Division of Pediatric Infectious Diseases and Rheumatology, Medical Faculty, University of Freiburg, Freiburg, Germany

Weiss JE:
 Hackensack University Medical Center, Pediatric Rheumatology, Hackensack, NJ, USA

Pardeo M:
 Division of Rheumatology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy

Nanda K:
 Seattle Children's Hospital, Seattle, WA, USA

Roth J:
 University of Ottawa and Division of Pediatric Dermatology & Rheumatology, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada

Rubio-Pérez N:
 Universidad Autónoma de Nuevo León, Facultad de Medicina, Departamento de Pediatria, Hospital Universitario "Dr. J. E. González", Monterrey, NL, Mexico

Hsu JC:
 Roche Innovation Center, New York, NY, USA

Wimalasundera S:
 Roche Products Ltd, Welwyn Garden City, UK

Wells C:
 Roche Products Ltd, Welwyn Garden City, UK

Bharucha K:
 Genentech, South San Francisco, CA, USA

Douglass W:
 Roche Products Ltd, Welwyn Garden City, UK

Bao M:
 Genentech, South San Francisco, CA, USA

Mallalieu NL:
 Roche Innovation Center, New York, NY, USA

Martini A:
 Università degli Studi di Genova, Dipartimento di Neuroscienze, Riabilitazione, Oftalmologia, Genetica e Scienze Materno-Infantili, Genoa, Italy

Lovell D:
 Pediatric Rheumatology Collaborative Study Group (PRCSG), Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA

De Benedetti F:
 Division of Rheumatology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
ISSN: 14620324





RHEUMATOLOGY
Editorial
OXFORD UNIV PRESS, GREAT CLARENDON ST, OXFORD OX2 6DP, ENGLAND, Reino Unido
Tipo de documento: Article
Volumen: 60 Número: 10
Páginas: 4568-4580
WOS Id: 000709572600024
ID de PubMed: 33506875
imagen Green Published, hybrid

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