Functional Ability and Health‐Related Quality of Life in Randomized Controlled Trials of Tocilizumab in Patients With Juvenile Idiopathic Arthritis

Brunner, Hermine I.; Chen, Chen; Bovis, Francesca; Benedetti, Fabrizio; Espada, Graciela; Joos, Rik; Akikusa, Jonathan; Chaitow, Jeffrey; Boteanu, Alina; Kimura, Yukiko; Rietschel, Christoph; Siri, Daniel; Smolewska, Elżbieta; Schmeling, Heinrike; Brown, Diane E.; Martini, Alberto; Lovell, Daniel J.; Huang, Bin; Ruperto, Nicolino

doi:10.1002/acr.24384

Cited by 7 publications

(5 citation statements)

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“…Two studies in patients with JIA (ages 2 to 17 years) who were treated with either tocilizumab or golimumab have demonstrated improvements in PROs (CHAQ‐DI, Child Health Questionnaire [CHQ]) over 16 weeks of treatment (37,38). Other studies in patients ages between 8 and 24 years have also reported improvements in PROs (CHAQ‐DI, CHQ, Health Utilities Index mark 3) with both short‐ and long‐term bDMARD treatment (39–41). Two registries have reported improvements in health‐related quality of life (HRQoL) in patients with JIA treated with either adalimumab plus MTX versus MTX over 1 year or with IV or SC abatacept over 5 years (42,43).…”

Section: Discussionmentioning

confidence: 93%

“…‡ For the 2-to 5-year-old cohort, the median (IQR) values at baseline were absolute numbers of missed school days, 1.0 (0.0-5.0); number of paid care days, 0.0 (0.0-0.0); and number of missed parent/caregiver activity days, 3.0 (0.0-7.0). (39)(40)(41). Two registries have reported improvements in healthrelated quality of life (HRQoL) in patients with JIA treated with either adalimumab plus MTX versus MTX over 1 year or with IV or SC abatacept over 5 years (42,43).…”

Section: Discussionmentioning

confidence: 99%

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Patient‐Reported Outcomes Among Patients Ages Two to Seventeen Years With Polyarticular‐Course Juvenile Idiopathic Arthritis Treated With Subcutaneous Abatacept: Two‐Year Results From an International Phase III Study

Ruperto

Lovell

Berman

et al. 2023

Arthritis Care & Research

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Objective To describe longitudinal changes in patient‐reported outcomes (PROs) in children with polyarticular‐course juvenile idiopathic arthritis (pJIA) treated with subcutaneous abatacept. Methods Secondary analysis of a single‐arm, open‐label 24‐month study of patients ages 6–17 years and 2–5 years. PROs included Childhood Health Assessment Questionnaire‐Disability Index (CHAQ‐DI), parent global assessment of child well‐being (PaGA), pain assessment, and Activity Limitation Questionnaire (ALQ). Clinical outcomes included 50% or greater improvement in JIA American College of Rheumatology (ACR) criteria, clinically inactive disease, and Juvenile Arthritis Disease Activity Score. Results For the 6‐ to 17‐year‐old (n = 173) and 2‐ to 5‐year‐old (n = 46) cohorts, respectively, median (Q1, Q3) changes from baseline in CHAQ‐DI at months 4 and 24 were −0.3 (−0.8, 0.0) and −0.5 (−1.0, −0.1), and −0.4 (−0.8, 0.0) and −0.5 (−1.0–−0.1). Median pain scores were below cutoff threshold for clinically relevant pain (<35 mm) by month 1 (6 to 17 years, 32.3 mm; 2 to 5 years, 25.7 mm), reaching a nadir at month 24 (6 to 17 years, 6.0 mm; 2 to 5 years, 2.0 mm). For the 6‐ to 17‐year‐old and 2‐ to 5‐year‐old cohorts, respectively, median PaGA scores were 47.8 (n = 172) and 42.1 (n = 46) at baseline and 6.3 (n = 107) and 2.0 (n = 37) at month 24. In both cohorts, ALQ components improved from baseline to month 4 and were largely maintained to month 24. Clinical outcomes improved through to month 24. Conclusion Early and sustained PRO improvements were reported in this phase III, open‐label trial of subcutaneous abatacept in patients with pJIA.

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Section: Discussionmentioning

confidence: 93%

Section: Discussionmentioning

confidence: 99%

Patient‐Reported Outcomes Among Patients Ages Two to Seventeen Years With Polyarticular‐Course Juvenile Idiopathic Arthritis Treated With Subcutaneous Abatacept: Two‐Year Results From an International Phase III Study

Ruperto

Lovell

Berman

et al. 2023

Arthritis Care & Research

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“…During the biologic era, disability in patients with JIA has decreased. A study found that patients with systemic JIA and patients with polyarticular JIA who were treated with tocilizumab had significantly reduced disability [ 29 ]. Tanya, et al reported disability and joint damage to be rare in their Singaporean JIA cohort; and 36% of their patients were treated with biologic agents [ 30 ].…”

Section: Discussionmentioning

confidence: 99%

Disability and disease-related damage in Thai children and adolescents with juvenile idiopathic arthritis

2023

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Background Children and adolescents with juvenile idiopathic arthritis (JIA) may suffer from disability and disease-related damage. This study aimed to investigate the prevalence of disability and damage, and identify the factors associated with articular and extra-articular damage in children and adolescents with JIA in a resource-restricted setting in Thailand. Methods This cross-sectional study enrolled JIA patients during June 2019-June 2021. Disability was assessed using the Child Health Assessment Questionnaire (CHAQ) and Steinbrocker classification criteria. Damage was evaluated using the Juvenile Arthritis Damage Index (JADI) and the modified-JADI (mJADI) tools. Results There were 101 patients (50.5% female) with median age of 11.8 years. Median disease duration was 32.7 months. Enthesitis-related arthritis (ERA) was the most common subtype (33.7%), followed by systemic JIA (25.7%). Thirty-three (32.7%) patients had delayed diagnosis ≥ 6 months. Moderate to severe disability was found in 20 (19.8%) patients. Patients with Steinbrocker functional classification > class I were seen in 17.9%. Thirty-seven (36.6%) patients had articular damage. Extra-articular complications were observed in 24.8%. Growth failure and striae were the most common complications in 7.8%. Leg-length discrepancy was documented in 5.0%. Ocular damage was found in 1 patient with ERA. Multivariable logistic regression analysis revealed Steinbrocker functional classification > class I (aOR: 18.1, 95% CI: 3.9–84.6; p < 0.001), delayed diagnosis ≥ 6 months (aOR: 8.5, 95%CI: 2.7–27.0; p < 0.001), and ERA (aOR: 5.7, 95%CI: 1.8–18.3; p = 0.004) as independent predictors of articular damage. Systemic corticosteroids use was the independent predictor of extra-articular damage (aOR: 3.8, 95%CI: 1.3–11.1; p = 0.013). Conclusions Disability and disease-related damage was identified in one-fifth and one-third of JIA patients. Early detection and treatment are essential for preventing permanent damage.

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“…A CHQ pain score of 100 indicates no pain. The CHQ pain score had been applied in pain research earlier (46,67). We categorized CHQ pain score 0-30 as severe, 40-60 as moderate, and 70-90 as mild pain.…”

Section: Discussionmentioning

confidence: 99%

“…We believed that the grading of pain severity should be independent from daily activities because the reference frames for daily activities would differ greatly (Papers I-III). Questions on pain from CHQ were used in previous research in children with CP (46,50,81), and recently in treatment evaluation in the children with juvenile idiopathic arthritis (67).…”

Section: Choice Of Variablesmentioning

confidence: 99%

Daytime contacts and general practitioner consultations, and pain as a reason for encounter in children with cerebral palsy; a Norwegian national registry linkage study

Larsen

Eide

Brunborg

et al. 2022

Scandinavian Journal of Primary Health Care

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Background: Cerebral palsy (CP) is the most common disorder of movement and posture in childhood, often accompanied by secondary challenges in cognition, epilepsy, and pain. Reports show that up to 75 % of all children with CP have pain, and that pain is often undertreated. Thus, pain has been considered a major challenge in care for children with CP since it influences health-related quality of life (HRQoL) negatively. The knowledge base on pain in children with CP is constantly increasing. Still, longitudinal studies on pain and HRQoL are scarce. Many different health care professions are involved in care for children with CP. Still, it is unknown if children with CP encounter a general practitioner (GP), or if they seek GP advice when in pain.Aims: The overall aim of this PhD thesis was to investigate pain and pain management in children with CP outside hospital. We investigated possible changes in pain, hip pain and HRQoL and relevant factors that could be associated with these variables, in a cohort of children, now youth with CP who were dependent on waking aid and who followed national CP surveillance program (Paper I-III). We also investigated if a diagnosis of CP in children ( 0-17 years) influenced the frequency of daytime contacts and consultations with a GP, and the frequency of pain as a reason for consultation (Paper IV). Methods: Papers I-III are based on a follow-up study with both cross-sectional and longitudinal design. All non-ambulatory children registered in Norwegian Quality and Surveillance Registry for Cerebral Palsy (NorCP) with bilateral CP, GMFCS levels III to V, born 2002-2006, living in South-Eastern Norway were invited to participate. Two identical data collections were performed five years apart, in childhood (7-12 years) and adolescence (12-17 years). One primary caregiver responded on behalf of each participant at both time points. Pain last four weeks was assessed using questions on pain frequency and intensity from Child Health Questionnaire (CHQ), a body map to localize pain sites and two questions on pain interference with activities of daily living and sleep from the Brief Pain Inventory (BPI), and an interview on pain, hip pain and the use of first-line analgesics. Hip displacement was measured on radiographs of the pelvis taken through NorCP. HRQoL was assessed using five of six CPCHILD domains. Paper IV is based on a registry study of GPs' daytime contacts available from the Norwegian Directorate of Health's database for the control and reimbursement of health expenses. All daytime contacts with regard 9 to type of contact and reason for encounter (RFE), in period 2006-2018 were analyzed for persons born in 1996 -2012. Linkage to NorCP identified children with CP. Comparison was performed between children registered in NorCP and children not registered in NorCP.Results: Papers I-III: Pain and hip pain were assessed twice in 67 children. The prevalence of recurrent pain, number of pain sites, pain intensity and frequency, and pain interference with activities of daily livin...

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Functional Ability and Health‐Related Quality of Life in Randomized Controlled Trials of Tocilizumab in Patients With Juvenile Idiopathic Arthritis

Cited by 7 publications

References 30 publications

Patient‐Reported Outcomes Among Patients Ages Two to Seventeen Years With Polyarticular‐Course Juvenile Idiopathic Arthritis Treated With Subcutaneous Abatacept: Two‐Year Results From an International Phase III Study

Patient‐Reported Outcomes Among Patients Ages Two to Seventeen Years With Polyarticular‐Course Juvenile Idiopathic Arthritis Treated With Subcutaneous Abatacept: Two‐Year Results From an International Phase III Study

Disability and disease-related damage in Thai children and adolescents with juvenile idiopathic arthritis

Daytime contacts and general practitioner consultations, and pain as a reason for encounter in children with cerebral palsy; a Norwegian national registry linkage study

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