Impact of Juvenile Localized Scleroderma on Longitudinal Quality of Life

Vidya Sivaraman¹, Kathryn S. Torok², Melissa Moore-Clingenpeel³, Fatma Dedeoglu⁴, Polly Ferguson⁵, Elena Pope⁶, Thomas G. Mason II⁷, Mara L Becker⁸, Gloria Higgins⁹, C. Egla Rabinovich¹⁰, Sandy Hong¹¹, Maria Ibarra¹², Ronald M. Laxer¹³, Robert Fuhlbrigge¹⁴, Katie Stewart¹⁵, Marilynn Punaro¹⁶ and Suzanne Li¹⁷, ¹Nationwide Children’s Hospital and The Ohio State University, Columbus, OH, ²Pediatric Rheumatology, University of Pittsburgh Med Ctr, Pittsburgh, PA, ³Research, Nationwide Children's Hospital, Dublin, OH, ⁴Rheumatology, Boston Children's Hospital, Boston, MA, ⁵Pediatrics, University of Iowa, Iowa City, IA, ⁶Section of Dermatology, The Hospital for Sick Children, Hospital for Sick Children, Toronto, ON, Canada, ⁷Division of Rheumatology - Department of Medicine, Mayo Clinic Rochester, Rochester, MN, ⁸Rheumatology, Children's Mercy Kansas City, Kansas City, MO, ⁹Pediatrics and Rheumatology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, ¹⁰Duke University Medical Center, Durham, NJ, ¹¹Pediatrics-Rheumatology, U of Iowa Children's Hosp, Iowa City, IA, ¹²Children's Mercy Hospital, Kansas City, MO, Kansas City, MO, ¹³Div of Rheumatology, The Hospital for Sick Children, Toronto, ON, Canada, ¹⁴Children's Hospital Colorado, Aurora, CO, ¹⁵UT Southwestern, Dallas, TX, ¹⁶Texas Scottish Rite Hospital for Children, Dallas, TX, ¹⁷Pediatrics, Hackensack University Medical Center, Hackensack, NJ

Meeting: 2018 ACR/ARHP Annual Meeting

Keywords: Pediatric rheumatology, Quality of life and scleroderma

Session Information

Date: Monday, October 22, 2018

Title: Pediatric Rheumatology – Clinical Poster II: Autoinflammatory Disorders, Scleroderma, and Miscellaneous

Session Type: ACR Poster Session B

Session Time: 9:00AM-11:00AM

Background/Purpose:

Juvenile localized scleroderma (jLS) is a chronic autoimmune disease, with cutaneous and extra-cutaneous manifestations (ECM) requiring long-term immunosuppressive therapy. Few studies have evaluated the effect of jLS on health-related quality of life (HRQoL). We describe parental HRQoL findings in a multi-center prospective jLS study.

Methods:

A prospective, pilot study of 50 jLS subjects beginning immunosuppressive therapy and followed for 1 year was conducted in 10 Childhood Arthritis and Rheumatology Research Alliance centers. Clinical assessments, treatment effects, and HRQoL measures (including Peds QL^TM Family Impact (PedsQL^TM FI) reports and ACR functional class) were collected at study visits. Domains of Peds QL^TM FI include effect of child’s disease on parental Physical, Emotional, Social and Cognitive Functioning, the family’s Daily Activities, and Family Relationships. The Worry module relates to parental worry about treatment and disease related issues affecting the child and family. Peds QL^TM FI scores were analyzed for association with patient characteristics, with change in domain scores over time analyzed by mixed effects models. Dunnett post-hoc tests were used to compare follow-up scores against baseline.

Results:

Twenty-five subjects completed parental Family Impact QOL data at 0, 6, and 12 month visits. Most were female (68%), Caucasian (88%), had linear subtype (60%), and ECM (84%), and experienced at least one treatment-related adverse effect (72%). Seventeen percent of subjects had ACR global functional > class 1. At all visits, the lowest scores were found in the Peds QL^TM FI domains of Worry and Emotional Functioning (Table). Overall, scores for Emotional Functioning, Daily Activities, and Family Relationships improved from 0 to 12 months (p = 0.015- 0.035, Table). Parents of female subjects were more likely to report higher Physical Functioning and lower (worse) Worry scores over time (p <0.0001, 0.0264, respectively). Parents of subjects with ACR class 2 or higher reported lower Daily Activities scores, with improvement in scores over time (p<0.0001).

Conclusion:

Our study supports the hypothesis that jLS impacts HRQoL. Parents of female subjects were found to worry more about disease issues and treatment, while poorer subject global function impacted family daily activities. Over time, scores improved for parental Emotional Functioning, family Daily Activities, and Family Relationships, possibly related to treatment. Head involvement and ECM were not specifically associated with poorer parental HRQoL scores. Future studies should evaluate the potential impact of jLS on parental and family HRQoL, and how these relate to patient HRQoL to improve long-term management of this disease.

Table. Summary of PedsQL^TM Family Impact Scores at Three Study Timepoints

	0 months (study entry)		6 months		12 months
DOMAINS*	Mean	SD	Mean	SD	Mean	SD	p-value
Physical Functioning	72.0	24.3	81.2	17.7	86.7	14.9	0.08
Emotional Functioning	64.4	26.8	76.0	18.4	80.9	20.3	0.04
Worry	54.6	22.0	61.1	20.1	65.4	17.6	0.10
Daily Activities	83.7	26.0	88.4	15.8	95.7	9.4	0.02
Family Relationships	84.6	17.6	87.6	14.1	95.2	8.9	0.02

*Scores range from 100 (no issues) to 0 (almost always has issues). P-values refer to change over time

Disclosure: V. Sivaraman, None; K. S. Torok, None; M. Moore-Clingenpeel, None; F. Dedeoglu, Novartis, 9; P. Ferguson, None; E. Pope, None; T. G. Mason II, None; M. L. Becker, None; G. Higgins, None; C. E. Rabinovich, None; S. Hong, None; M. Ibarra, None; R. M. Laxer, None; R. Fuhlbrigge, None; K. Stewart, None; M. Punaro, None; S. Li, None.

To cite this abstract in AMA style:

Sivaraman V, Torok KS, Moore-Clingenpeel M, Dedeoglu F, Ferguson P, Pope E, Mason TG II, Becker ML, Higgins G, Rabinovich CE, Hong S, Ibarra M, Laxer RM, Fuhlbrigge R, Stewart K, Punaro M, Li S. Impact of Juvenile Localized Scleroderma on Longitudinal Quality of Life [abstract]. Arthritis Rheumatol. 2018; 70 (suppl 9). https://acrabstracts.org/abstract/impact-of-juvenile-localized-scleroderma-on-longitudinal-quality-of-life/. Accessed .

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