Related Experiment Video
Updated: Feb 8, 2026
In Silico Clinical Trials for Cardiovascular Disease
Published on: May 27, 2022
Growth During Tocilizumab Therapy for Polyarticular-course Juvenile Idiopathic Arthritis: 2-year Data from a Phase
Kamal N Bharucha1,2, Hermine I Brunner1,2, Inmaculada Calvo Penadés1,2
1From Genentech, San Francisco; Consultant, Santa Monica, California, USA; Division of Rheumatology, Department of Pediatrics, University of Cincinnati, Cincinnati Children's Hospital Medical Center (CCHMC), Pediatric Rheumatology Collaborative Study Group (PRCSG) Coordinating Center, Cincinnati, Ohio, USA; Paediatric Rheumatology Department, Hospital Universitario La Fe, Valencia, Spain; Pediatric Department, V.A. Nasonova Research Institute of Rheumatology; Department of Pediatric Rheumatology, Hospital of Childhood Diseases, JCA-UNIT, Sechenov Moscow Medical Academy, Moscow, Russia; Department of Pediatrics, Hospital Universitario Dr. J.E. González, Universidad Autónoma de Nuevo, Monterrey, Mexico; Universidade Federal do Rio de Janeiro, Instituto de Puericultura e Pediatria Martagao Gesteira (IPPMG); Hospital Universitario Pedro Ernesto, Nucleo de Estudos da Saúde do Adolescente, Universidade do Estado do Rio de Janeiro, Rio de Janeiro, Brazil; Wojewodzki Szpital Dzieciecy, Oddzial Pediatrii, Hematologii Onkologii i Reumatologii, Bydgoszcz, Poland; Department of Pediatrics, Alberta Children's Hospital and University of Calgary, Calgary, Alberta, Canada; Klinikum Bremen-Mitte, Professor Hess-Kinderklinik, Pädiatrische Intensivmedizin, Bremen, Germany; Rheumatology Unit, Department of Woman and Child Health, University of Padua, Padua; Istituto Giannina Gaslini, Pediatria II, Rheumatologia, Paediatric Rheumatology International Trials Organisation (PRINTO) Coordinating Centre, Genoa; Institute for Research and Health Care (IRCCS) Ospedale Pediatrico Bambino Gesù, Rome, Italy; Royal Children's Hospital, Pediatric Rheumatology, Melbourne, Victoria; Department of Rheumatology, Sydney Children's Hospital Network, Randwick and Westmead, Sydney, Australia; Roche Products Ltd., Welwyn Garden City, UK.
Insights
Tocilizumab (TCZ) treatment improved growth in children with polyarticular-course juvenile idiopathic arthritis (pcJIA). Over two years, height SDS increased significantly in patients with early-stage disease, with 72% showing improvement.
Area of Science:
- Pediatric Rheumatology
- Endocrinology
- Clinical Trials
Background:
- Juvenile idiopathic arthritis (JIA) can impact growth in children.
- Polyarticular-course JIA (pcJIA) affects multiple joints and may necessitate advanced therapies.
- Growth impairment is a significant concern in children with chronic inflammatory conditions like pcJIA.
Purpose of the Study:
- To evaluate the effect of tocilizumab (TCZ) on growth in patients with pcJIA over a 2-year period.
- To assess changes in height velocity and World Health Organization (WHO) height standard deviation score (SDS) in pcJIA patients treated with TCZ.
- To determine the proportion of patients achieving improved growth parameters during TCZ therapy.
Main Methods:
- A phase III clinical trial involving patients with pcJIA and inadequate response to methotrexate.
- Patients received open-label intravenous tocilizumab (TCZ) for 16 weeks, followed by randomization to TCZ or placebo for 24 weeks, with an open-label extension to 104 weeks.
- Growth parameters, including height velocity and WHO height SDS, were measured in patients with Tanner stage ≤ 3 at baseline, excluding those on growth hormone therapy.
Main Results:
- The study included 187 patients, with a mean baseline WHO height SDS of -0.5 ± 1.2.
- Among 103 patients at Tanner stage ≤ 3 who completed 2 years of data, mean height SDS increased by +0.40 (p < 0.0001).
- 72% (74/103) of these patients demonstrated an increase in height SDS from baseline, with a mean height velocity of 6.7 ± 2.0 cm/year.
Conclusions:
- Tocilizumab (TCZ) treatment led to significant growth improvement in patients with polyarticular-course juvenile idiopathic arthritis (pcJIA).
- A majority of patients (72%) with early-stage disease (Tanner stage ≤ 3) experienced enhanced height SDS over two years.
- TCZ is a viable therapeutic option for improving growth outcomes in children with pcJIA.
Objective:
Evaluate growth in patients with polyarticular-course juvenile idiopathic arthritis (pcJIA) treated with tocilizumab (TCZ) for up to 2 years in a phase III trial.
Methods:
Patients with pcJIA lasting at least 6 months and inadequate response to methotrexate received open-label TCZ intravenously every 4 weeks (randomly assigned to 8 or 10 mg/kg if they weighed < 30 kg; received 8 mg/kg if they weighed ≥ 30 kg) for 16 weeks. Patients with JIA American College of Rheumatology 30 response at Week 16 were randomly assigned to TCZ or placebo for 24 weeks, with an open-label extension through Week 104. Mean ± SD height velocity (cm/yr) and World Health Organization (WHO) height SD score (SDS) were measured in patients receiving ≥ 1 dose of TCZ who did not receive growth hormone and in patients whose baseline Tanner stage was ≤ 3.
Results:
The study included 187 of 188 patients (99.5%) with mean WHO height SDS -0.5 ± 1.2, which was unrelated to age or disease duration (Spearman rank correlations r = 0.08 and r = -0.12, respectively). There were 123 patients at Tanner stage ≤ 3 at baseline, among whom 103 completed the study with 2 years of height SDS data. Mean height SDS increased from baseline to year 2 (+0.40, p < 0.0001). In 74 of 103 patients (72%), height SDS was greater than at baseline, and mean height velocity was 6.7 ± 2.0 cm/year.
Conclusion:
Among patients with pcJIA at Tanner stage ≤ 3 at baseline, 72% (74/103) had increased height SDS at the end of the study.
Related Concept Videos
01:12Statistical Software for Data Analysis and Clinical Trials
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...
01:11Clinical Trials: Overview
01:05Data Collection III
The principles to begin the physical assessment include conducting a comprehensive or problem-related history in a quiet, well-lit room, emphasizing privacy and comfort for the...
01:23Gastritis III: Clinical Manifestations and Management
Clinical manifestations of acute gastritis
The patient with acute gastritis may have a rapid onset of symptoms, such as epigastric pain or discomfort, dyspepsia, anorexia, hiccups, or nausea and vomiting, which can last from a few hours to a few days. Erosive or hemorrhagic gastritis may cause bleeding, which may manifest as blood in vomit or as...
01:26Heart Failure III: Clinical Manifestations