Safety and effectiveness of abatacept in juvenile idiopathic arthritis: results from the PRINTO/PRCSG registry
Authors: Lovell Daniel J , Tzaribachev Nikolay , Henrickson Michael , Simonini Gabriele , Griffin Thomas A , Alexeeva Ekaterina , Bohnsack John F , Zeft Andrew , Horneff Gerd , Vehe Richard K , Staņēviča Valda , Tarvin Stacey , Trachana Maria , del Río Ana Quintero , Huber Adam M , Kietz Daniel , Orbán Ilonka , Dare Jason , Foeldvari Ivan , Quartier Pierre , Dominique Alyssa , Simon Teresa A , Martini Alberto , Brunner Hermine I , Ruperto Nicolino ,   for PRINTO and the Pediatric Rheumatology Collaborative Study Group (PRCSG)§, Brunner Jurgen , Fernandes Taciana , Appenzeller Simone , Oliveira Sheila , Terreri Maria Teresa , Tzaribachev Nikolay , Minden Kirsten , Hufnagel Mark , Foeldvari Ivan , Horneff Gerd , Helling-Bakki Astrid , Herlin Troels , Moreno Estefania , Anton Jordi , del-Castillo Pablo Mesa- , Udaondo Clara , Penades Inmaculada Calvo , Quartier Pierre , Brochard Karine , Ramanan Athimalaipet , Trachana Maria , Orban Ilonka , Hashkes Philip (Pinchas) , Ruperto Nicolino , Simonini Gabriele , Olivieri Alma Nunzia , Zulian Francesco , Montin Davide , Peroni Diego , Stanevicha Valda , Cornejo Gabriel Vega , Wulffraat Nico , Kamphuis Sylvia , Gastanaga Maria Eliana Paz , Miraval Tatiana , Oliveira-Ramos Filipa , Lazar Calin , Nikishina Irina , Alexeeva Ekaterina , Sarychev Aleksej , Chasnyk Vyacheslav , Grebenkina Lyudmila , Suwairi Wafaa Mohammed Saad , Koskova Elena , Ally Mahmood , Louw Ingrid , Breedt Johannes , Brunner Hermine , Ting Tracy , Taylor Janalee , Huggins Jennifer , Henrickson Michael , DeWitt Esi Morgan , Grom Alexei , Lovell Daniel , Schulert Grant , Rodriguez-Smith Jackeline , Dare Jason , Morris Paula , Sukumarain Sukesh , Gitelman Marissa Klein , Miller Michael , Curran Megan , Alperin Risa , Ardalan Kaveh , De Ranieri Deirdre , Hiskey Megan , Nolan Brian , Chalom Beth , Zelf Andy , Spalding Steven , Costanzo Denise , Rennebohm Robert , Waugaman Brenda , Brodus Elizabeth , Robinson Angela , Panupattanapong Sirada , Kietz Dan , Rosenkranz Margalit , Cassidy Elaine , Torok Kathryn , Kingsbury Dan , Cartwright Victoria , Lasky Andrew , Brown Diane , Reiff Andreas , Shaham Bracha , Marzan Katherine , Wagner-Weiner Linda , Onel Karen , Tesher Melissa , Edens Cuoghi , Moore Terry , Syed Reema , Pepmueller Peri , Tuttle Paul , Dalrymple Austin , Barhula Srikanth , Feller Lance , Horwitz Mara , Justice Matt , Nocton James , Olson Judyann , Williams Calvin , Versbsy James , Co Dominic , Roth-Wojcicki Elizabeth , Correll Colleen , Vehe Richard , Binstadt Bryce , Hobday Patricia , Brueck Danielle , Griffin Tom , Gillispie-Taylor Miriah , Vora Sheetal , Tarvin Stacey , O'Neil Kathleen , Ballinger Susan , Blakley Michael , Klausmeier Thomas , Oliver Melissa , Stevens Brandi , Rodriguez Martha , Go Ellen , Bohnsack John , Inman Christi , Hersh Aimee , Stern Sara , Woodward Amy , Durkee Debbie , Boulva Sylvie Fadrhonc , James Karen , Treemarcki Erin , Goldsmith Donald , Lvovich Svetlana , Toib Dana , Patel Julisa , Jerath Rita , Sharma Nirupma , Newhall Lauren , Carrasco Ruy , Moorthy Nandini , Boneparth Alexis , Quintero Ana , Graham Thomas , Spence Stephanie , Davis Alaina , Gotte Alisa , Mehta Jay , Walters Heather , Mian Zanab , Parkinson Elizabeth , Hui-Yen Joyce , Steigerwald Katherine , Guzman Marla , Gottlieb Beth , Quintero Ana , Whitaker Connie , Kelly Leslie , Carrasco Ruy , Succimarri Rosie , Hazel Elizabeth , Chedeville Gaelle , Compillo Sarah , LeBlance Claire , Tucker Lori , Cabral David , Houghton Kristin , Guzman Jamie , Morishita Kim , Huber Adam , Stringer Elizabeth , Ramsey Suzanne , Lang Bianca , Levy Deborah , Silverman Earl , Schmeling Heinrike , Johnson Nicole , Luca Nadia , Dhalla Muhammed 
Published: January 2024

Abstract

Objective
The aim of this study was to report the interim 5-year safety and effectiveness of abatacept in patients with JIA in the PRINTO/PRCSG registry.

Methods
The Abatacept JIA Registry (NCT01357668) is an ongoing observational study of children with JIA receiving abatacept; enrolment started in January 2013. Clinical sites enrolled patients with JIA starting or currently receiving abatacept. Eligible patients were assessed for safety (primary end point) and effectiveness over 10 years. Effectiveness was measured by clinical 10-joint Juvenile Arthritis Disease Activity Score (cJADAS10) in patients with JIA over 5 years. As-observed analysis is presented according to the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) guidelines.

Results
As of 31 March 2020, 587 patients were enrolled; 569 are included in this analysis (including 134 new users) with 1214.6 patient-years of safety data available. Over 5 years, the incidence rate (IR) per 100 patient-years of follow-up of serious adverse events was 5.52 (95% CI: 4.27, 7.01) and of events of special interest was 3.62 (95% CI: 2.63, 4.86), with 18 serious infections [IR 1.48 (95% CI: 0.88, 2.34)]. As early as month 3, 55.9% of patients achieved cJADAS10 low disease activity and inactive disease (20.3%, 72/354 and 35.6%, 126/354, respectively), sustained over 5 years. Disease activity measures improvement over 5 years across JIA categories.

Conclusion
Abatacept was well tolerated in patients with JIA, with no new safety signals identified and with well-controlled disease activity, including some patients achieving inactive disease or remission.

Trial registration
Clinicaltrials.gov, NCT01357668.