Quality of Life Evaluation in New Onset Juvenile Dermatomyositis Patients from the Printo Trial

Andressa Guariento¹, Gabriella Giancane², Elena Fueri¹, Francesco Zulian³, Angelo Ravelli⁴, Bo Magnusson³, Tadej Avcin³, Fabrizia Corona³, Valeria Gerloni¹, Claudia Bracaglia¹, Rolando Cimaz¹, Antonella Meini², Silvana Martino³, Anne Pagnier¹, Michel Rodiere³, Christine Soler³, Valda Stanevicha³, Rebecca ten Cate⁵, Jelena Vojinovic³, Simona Angioloni¹, Luca Villa¹, Michele Pesce¹, Irene Gregorini¹, Chiara Pallotti¹, Alberto Martini⁶, Angela Pistorio³ and Nicola Ruperto³, ¹Istituto Giannina Gaslini - Pediatria II, Reumatologia - PRINTO, Genova, Italy, ²Pediatria II, Reumatologia - PRINTO, Istituto Giannina Gaslini - Pediatria II, Reumatologia - PRINTO, Genova, Italy, ³Istituto Giannina Gaslini - Pediatria II, Reumatologia - PRINTO, Genoa, Italy, ⁴University of Genova, IRCCS Istituto Giannina Gaslini, Genova, Italy, ⁵Pediatric Rheumatology, Leiden University Medical Center, Leiden, Netherlands, ⁶Istituto Giannina Gaslini, Genoa, Italy

Meeting: 2017 ACR/ARHP Annual Meeting

Date of first publication: September 18, 2017

Keywords: juvenile dermatomyositis, Pediatric rheumatology, quality of life and therapy

Session Information

Date: Tuesday, November 7, 2017

Title: Patient Outcomes, Preferences, and Attitudes Poster III

Session Type: ACR Poster Session C

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

Background/Purpose: Juvenile dermatomyositis (JDM) is the most common clinical pediatric idiopathic inflammatory myopathy and it may severely compromise the quality of life of affected patients. Early adequate immunosuppressive treatment can improve the quality of life in JDM as in other rheumatic diseases like juvenile idiopathic arthritis (JIA).¹

Objective: To evaluate the HRQoL change over time in children with new-onset JDM patients enrolled in the PRINTO trial².

Methods: In the PRINTO JDM trial, children aged 18 years old or younger with newly diagnosed and untreated probable or definite JDM were enrolled. All patients received three daily pulses of intravenous methylprednisolone at onset, and then they were randomized to one of the following three different treatment groups: prednisone (PDN), prednisone plus ciclosporin (PDN+CSA) and prednisone plus methotrexate (PDN+MTX).

In our study, we considered patients who had a complete HRQoL assessment at onset through the Child Health Questionnaire (CHQ). We compared quality of life among the responders (PRINTO20-50-70) belonging to the three treatment groups, and between responder and not responder patients. Moreover we compared the affected patients to healthy children.

Results: Out of a total of 139 patients enrolled in the PRINTO JDM trial, 129 (92.8%) were retained for the analysis (41.9% males and 58.1% females; median age 7.4 years). At baseline, patients with JDM showed poorer scores in quality of life (PhS = 15.6 and PsS=40.0) than healthy children. In particular, “Physical Summary score” (PhS) turned out to be significantly lower than 2 standard deviations with respect to the mean value of the healthy children with no difference among the 3 treatment groups (PDN, PDN+MTX, PDN+CSA). Less compromised scores were observed for the PsychoSocial summary Scale, with values between 1 and 2 standard deviations below the mean values of healthy children. We found similar values between «responder 20» and «not responders». A statistically significant improvement was observed after 6 months of treatment both in the PhS and in the PsS subscales (P<0.0001) irrespective of the treatment group, and in all items of the CHQ with the exception of the GBE (Global behavior parameter). A significant improvement over time was observed regardless of the treatment group and the level of response (PRINTO 20, 50, 70). “Responder” patients showed PhS values significantly higher than “not responder”; on the contrary, PsS values did not reveal a significant difference between “responders” and “not responders”, despite an improvement over time.

Conclusion: Children with new-onset JDM, treated with PDN alone or in combination with other therapies, showed a significant improvement in quality of life during a two-year follow-up. According to the “PRINTO 20-50-70” criteria, “responder” patients showed a statistically significant improvement in the PhS score compared to the “not responders”.

References:

¹Ce´spedes-Cruz A et al. Ann Rheum Dis 2008;67:309–314.

²Ruperto N et al. Lancet 2016;387:671–78.

Disclosure: A. Guariento, None; G. Giancane, None; E. Fueri, None; F. Zulian, None; A. Ravelli, None; B. Magnusson, None; T. Avcin, None; F. Corona, None; V. Gerloni, None; C. Bracaglia, None; R. Cimaz, None; A. Meini, None; S. Martino, None; A. Pagnier, None; M. Rodiere, None; C. Soler, None; V. Stanevicha, None; R. ten Cate, None; J. Vojinovic, AbbVie, 8; S. Angioloni, None; L. Villa, None; M. Pesce, None; I. Gregorini, None; C. Pallotti, None; A. Martini, GASLINI Hospital, 3,Astellas, AstraZeneca, Bristol-Myers Squibb, Italfarmaco, and MedImmune, 8,AbbVie Inc., AstraZeneca, Bristol-Myers Squibb, Janssen Biologics B.V., Eli Lilly and Co., “Francesco Angelini”, GlaxoSmithKline, Italfarmaco, Novartis, Pfizer, Roche, Sanofi Aventis, Schwarz Biosciences GmbH, Xoma, and Wyeth Pharmaceuticals, 2; A. Pistorio, None; N. Ruperto, GASLINI Hospital, 3,Astellas, AstraZeneca, Bristol-Myers Squibb, Italfarmaco, Janssen Biologics B.V., MedImmune, Roche, and Wyeth/Pfizer, 8.

To cite this abstract in AMA style:

Guariento A, Giancane G, Fueri E, Zulian F, Ravelli A, Magnusson B, Avcin T, Corona F, Gerloni V, Bracaglia C, Cimaz R, Meini A, Martino S, Pagnier A, Rodiere M, Soler C, Stanevicha V, ten Cate R, Vojinovic J, Angioloni S, Villa L, Pesce M, Gregorini I, Pallotti C, Martini A, Pistorio A, Ruperto N. Quality of Life Evaluation in New Onset Juvenile Dermatomyositis Patients from the Printo Trial [abstract]. Arthritis Rheumatol. 2017; 69 (suppl 10). https://acrabstracts.org/abstract/quality-of-life-evaluation-in-new-onset-juvenile-dermatomyositis-patients-from-the-printo-trial/. Accessed .

« Back to 2017 ACR/ARHP Annual Meeting

ACR Meeting Abstracts - https://acrabstracts.org/abstract/quality-of-life-evaluation-in-new-onset-juvenile-dermatomyositis-patients-from-the-printo-trial/