Clinical Characteristics and Factors Associated With Disability and Impaired Quality of Life in Children With Juvenile Systemic Sclerosis: Results From the Childhood Arthritis and Rheumatology Research Alliance Legacy Registry

and the Childhood Arthritis and Rheumatology Research Alliance Registry Investigators

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Objective: To investigate clinical manifestations of juvenile systemic sclerosis (SSc; scleroderma), including disease characteristics and patient quality of life, using the multinational Childhood Arthritis and Rheumatology Research Alliance (CARRA) Legacy Registry. Methods: Patients with juvenile SSc were prospectively enrolled between 2010 and 2013. The diagnosis of juvenile SSc was determined by the enrolling pediatric rheumatologist, with the requirement for disease onset prior to age 18 years. Collected data included demographics, disease characteristics, medication exposure, and quality of life metrics. Results: In total, 64 patients with juvenile SSc were enrolled a median of 3.6 years after disease onset, which occurred at a median age of 10.3 years. The most common organ manifestations were dermatologic and vascular, followed by musculoskeletal, gastrointestinal, and pulmonary; in 38% of patients, ≥4 organ systems were affected. Patients with juvenile SSc had significantly more disability at enrollment compared with CARRA Legacy Registry patients with juvenile idiopathic arthritis, dermatomyositis, or systemic lupus erythematosus. Although physician-reported measures correlated most closely with arthritis, dermatologic manifestations, and pulmonary manifestations, poor patient-reported measures were associated with gastrointestinal involvement. During >50 person-years of follow-up, most organ manifestations remained stable, and no mortality or development of new solid organ involvement after enrollment was reported. Conclusion: In the first multicenter prospective cohort of patients with juvenile SSc in North America, the disease burden was high: multiorgan manifestations were common, and functional disability was greater than that observed in patients with other childhood-onset rheumatic diseases. Gastrointestinal involvement had the greatest impact on quality of life.

Original languageEnglish (US)
Pages (from-to)1806-1813
Number of pages8
JournalArthritis Care and Research
Volume70
Issue number12
DOIs
StatePublished - Dec 1 2018

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Systemic Scleroderma
Rheumatology
Arthritis
Registries
Quality of Life
Research
Lung
Dermatomyositis
Juvenile Arthritis
North America
Rheumatic Diseases
Systemic Lupus Erythematosus
Blood Vessels
Demography
Pediatrics
Physicians
Mortality

ASJC Scopus subject areas

  • Rheumatology

Cite this

Clinical Characteristics and Factors Associated With Disability and Impaired Quality of Life in Children With Juvenile Systemic Sclerosis : Results From the Childhood Arthritis and Rheumatology Research Alliance Legacy Registry. / and the Childhood Arthritis and Rheumatology Research Alliance Registry Investigators.

In: Arthritis Care and Research, Vol. 70, No. 12, 01.12.2018, p. 1806-1813.

Research output: Contribution to journalArticle

@article{9c2de36e51da4bd1a53571388f545164,
title = "Clinical Characteristics and Factors Associated With Disability and Impaired Quality of Life in Children With Juvenile Systemic Sclerosis: Results From the Childhood Arthritis and Rheumatology Research Alliance Legacy Registry",
abstract = "Objective: To investigate clinical manifestations of juvenile systemic sclerosis (SSc; scleroderma), including disease characteristics and patient quality of life, using the multinational Childhood Arthritis and Rheumatology Research Alliance (CARRA) Legacy Registry. Methods: Patients with juvenile SSc were prospectively enrolled between 2010 and 2013. The diagnosis of juvenile SSc was determined by the enrolling pediatric rheumatologist, with the requirement for disease onset prior to age 18 years. Collected data included demographics, disease characteristics, medication exposure, and quality of life metrics. Results: In total, 64 patients with juvenile SSc were enrolled a median of 3.6 years after disease onset, which occurred at a median age of 10.3 years. The most common organ manifestations were dermatologic and vascular, followed by musculoskeletal, gastrointestinal, and pulmonary; in 38{\%} of patients, ≥4 organ systems were affected. Patients with juvenile SSc had significantly more disability at enrollment compared with CARRA Legacy Registry patients with juvenile idiopathic arthritis, dermatomyositis, or systemic lupus erythematosus. Although physician-reported measures correlated most closely with arthritis, dermatologic manifestations, and pulmonary manifestations, poor patient-reported measures were associated with gastrointestinal involvement. During >50 person-years of follow-up, most organ manifestations remained stable, and no mortality or development of new solid organ involvement after enrollment was reported. Conclusion: In the first multicenter prospective cohort of patients with juvenile SSc in North America, the disease burden was high: multiorgan manifestations were common, and functional disability was greater than that observed in patients with other childhood-onset rheumatic diseases. Gastrointestinal involvement had the greatest impact on quality of life.",
author = "{and the Childhood Arthritis and Rheumatology Research Alliance Registry Investigators} and Stevens, {Brandi E.} and Torok, {Kathryn S.} and Li, {Suzanne C.} and Nicole Hershey and Megan Curran and Higgins, {Gloria C.} and Moore, {Katharine F.} and {Egla Rabinovich}, C. and Samuel Dodson and Stevens, {Anne M.} and L. Abramson and E. Anderson and M. Andrew and N. Battle and M. Becker and H. Benham and T. Beukelman and J. Birmingham and P. Blier and A. Brown and H. Brunner and A. Cabrera and D. Canter and D. Carlton and B. Caruso and L. Ceracchio and E. Chalom and J. Chang and P. Charpentier and K. Clark and J. Dean and F. Dedeoglu and B. Feldman and P. Ferguson and M. Fox and K. Francis and M. Gervasini and D. Goldsmith and G. Gorton and B. Gottlieb and T. Graham and T. Griffin and H. Grosbein and S. Guppy and H. Haftel and D. Helfrich and A. Hillard and Hollister, {J. R.} and J. Hsu and Jerath, {Rita S}",
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T1 - Clinical Characteristics and Factors Associated With Disability and Impaired Quality of Life in Children With Juvenile Systemic Sclerosis

T2 - Results From the Childhood Arthritis and Rheumatology Research Alliance Legacy Registry

AU - and the Childhood Arthritis and Rheumatology Research Alliance Registry Investigators

AU - Stevens, Brandi E.

AU - Torok, Kathryn S.

AU - Li, Suzanne C.

AU - Hershey, Nicole

AU - Curran, Megan

AU - Higgins, Gloria C.

AU - Moore, Katharine F.

AU - Egla Rabinovich, C.

AU - Dodson, Samuel

AU - Stevens, Anne M.

AU - Abramson, L.

AU - Anderson, E.

AU - Andrew, M.

AU - Battle, N.

AU - Becker, M.

AU - Benham, H.

AU - Beukelman, T.

AU - Birmingham, J.

AU - Blier, P.

AU - Brown, A.

AU - Brunner, H.

AU - Cabrera, A.

AU - Canter, D.

AU - Carlton, D.

AU - Caruso, B.

AU - Ceracchio, L.

AU - Chalom, E.

AU - Chang, J.

AU - Charpentier, P.

AU - Clark, K.

AU - Dean, J.

AU - Dedeoglu, F.

AU - Feldman, B.

AU - Ferguson, P.

AU - Fox, M.

AU - Francis, K.

AU - Gervasini, M.

AU - Goldsmith, D.

AU - Gorton, G.

AU - Gottlieb, B.

AU - Graham, T.

AU - Griffin, T.

AU - Grosbein, H.

AU - Guppy, S.

AU - Haftel, H.

AU - Helfrich, D.

AU - Hillard, A.

AU - Hollister, J. R.

AU - Hsu, J.

AU - Jerath, Rita S

PY - 2018/12/1

Y1 - 2018/12/1

N2 - Objective: To investigate clinical manifestations of juvenile systemic sclerosis (SSc; scleroderma), including disease characteristics and patient quality of life, using the multinational Childhood Arthritis and Rheumatology Research Alliance (CARRA) Legacy Registry. Methods: Patients with juvenile SSc were prospectively enrolled between 2010 and 2013. The diagnosis of juvenile SSc was determined by the enrolling pediatric rheumatologist, with the requirement for disease onset prior to age 18 years. Collected data included demographics, disease characteristics, medication exposure, and quality of life metrics. Results: In total, 64 patients with juvenile SSc were enrolled a median of 3.6 years after disease onset, which occurred at a median age of 10.3 years. The most common organ manifestations were dermatologic and vascular, followed by musculoskeletal, gastrointestinal, and pulmonary; in 38% of patients, ≥4 organ systems were affected. Patients with juvenile SSc had significantly more disability at enrollment compared with CARRA Legacy Registry patients with juvenile idiopathic arthritis, dermatomyositis, or systemic lupus erythematosus. Although physician-reported measures correlated most closely with arthritis, dermatologic manifestations, and pulmonary manifestations, poor patient-reported measures were associated with gastrointestinal involvement. During >50 person-years of follow-up, most organ manifestations remained stable, and no mortality or development of new solid organ involvement after enrollment was reported. Conclusion: In the first multicenter prospective cohort of patients with juvenile SSc in North America, the disease burden was high: multiorgan manifestations were common, and functional disability was greater than that observed in patients with other childhood-onset rheumatic diseases. Gastrointestinal involvement had the greatest impact on quality of life.

AB - Objective: To investigate clinical manifestations of juvenile systemic sclerosis (SSc; scleroderma), including disease characteristics and patient quality of life, using the multinational Childhood Arthritis and Rheumatology Research Alliance (CARRA) Legacy Registry. Methods: Patients with juvenile SSc were prospectively enrolled between 2010 and 2013. The diagnosis of juvenile SSc was determined by the enrolling pediatric rheumatologist, with the requirement for disease onset prior to age 18 years. Collected data included demographics, disease characteristics, medication exposure, and quality of life metrics. Results: In total, 64 patients with juvenile SSc were enrolled a median of 3.6 years after disease onset, which occurred at a median age of 10.3 years. The most common organ manifestations were dermatologic and vascular, followed by musculoskeletal, gastrointestinal, and pulmonary; in 38% of patients, ≥4 organ systems were affected. Patients with juvenile SSc had significantly more disability at enrollment compared with CARRA Legacy Registry patients with juvenile idiopathic arthritis, dermatomyositis, or systemic lupus erythematosus. Although physician-reported measures correlated most closely with arthritis, dermatologic manifestations, and pulmonary manifestations, poor patient-reported measures were associated with gastrointestinal involvement. During >50 person-years of follow-up, most organ manifestations remained stable, and no mortality or development of new solid organ involvement after enrollment was reported. Conclusion: In the first multicenter prospective cohort of patients with juvenile SSc in North America, the disease burden was high: multiorgan manifestations were common, and functional disability was greater than that observed in patients with other childhood-onset rheumatic diseases. Gastrointestinal involvement had the greatest impact on quality of life.

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U2 - 10.1002/acr.23547

DO - 10.1002/acr.23547

M3 - Article

C2 - 29457372

AN - SCOPUS:85050078257

VL - 70

SP - 1806

EP - 1813

JO - Arthritis Care and Research

JF - Arthritis Care and Research

SN - 2151-464X

IS - 12

ER -