Presentation of juvenile systemic sclerosis and difference to adult patients

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Presentation of juvenile systemic sclerosis and difference to adult patients Ivan Foeldvari, MD Hamburg Centre for Peadiatric and Adolescence Rheumatology, Germany Centre for Treatment of Scleroderma and Uveitis in Childhood and Adolescence Teaching Unit of the Asklepios Campus of the Semmelweis Medical School, Budapest, Hungary www.sklerodermie.org www.uveitis-kindesalter.de www.orphan-diseases-in-pediatric-rheumatology. de

Hamburger Zentrum für Kinder- und Jugendrheumatologie Pediatric and Adolescent Rheumatologist Transtion programm with an associated adult rheumatologist Associated Occupationaltherapiest Associated Physiotherapiest Social worker Study nurses Assoziierted Team Uveitis (Uveitis spesialised ophthalmologist) Temporomandibular joint involvement (Orthodentist) Pediatric orthopedic specialist Pediatric dermatologist,... Outpatient Painteam Paediatrician specialised on chronic pain and his team Paediatric Psychiatrist Occupational therapy team

Cooperations/ Participation Hamburger Elterninitiative Rheumakranker Kinder e.v. www.kinderrheuma.de Deutsche Rheumaliga Lupus Erythematodes Selbsthilfegemeinschaft Scleroderma Foundation UK FEDERATION OF EUROPEAN SCLERODERMA ASSOCIATIONS (FESCA) Advisor for pediatric issues Scleroderma Foundation USA EMA /Enpra working group PRES Scleroderma working SCTC juvenile scleroderma working group

Topics of the talk Epidemiology of jssc Classification issues Specific pediatric issues Comparison of the pediatric data to adult population with diffuse subtype

Topics of the talk Epidemiology of jssc Classification issues Specific pediatric issues Comparison of the pediatric data to adult population with diffuse subtype

Epidemiology and demographic of jssc! Data regarding incidence and prevalence is rare! According a cross-sectional study by Herrick et al. ( Arthritis Care 2010;62:213)! Incidence rate 0.27 (95% CI 0.1-0.5) per million children

Estimated Prevalence of jssc using the USA claims data T. Beukelman, F. Xie, I. Foeldvari- JSRD 2018, 3: 189-190

Topics of the talk Epidemiology of jssc Classification issues Specific pediatric issues Comparison of the pediatric data to adult population with diffuse subtype

Proposed classification criteria for juvenile systemic scleroderma Zulian et al. Arthritis Rheum 2007;57:203-12 First and Second International Workshop on Juvenile Scleroderma June 2001 and 2004 Padua, Italy Steering Committee: F. Zulian (Padua), I. Foeldvari (Hamburg), J. Harper (London), A. Peserico (Padua), N. Ruperto (Pavia) Major criteria! Sclerosis* / induration* Definite disease 1 major and 2 minor criteria! Minor criteria! Vascular changes*! Pulmonary involvement*! Gastrointestinal involvement*! Renal involvement*! Cardiovascular involvement*! Musculoskeletal involvement*! Neurologic involvement*! Serology* * Per definition typical for SSc

New Classification of Systemic Sclerosis Arth Rheum 2013,65: 2737-47 The maximum possible score is 19 Patients with a score of 9 are classified as having SSc. The definitions of the items used in the criteria are defined in the publication.

New Classification of Systemic Sclerosis Arth Rheum 2013,65: 2737-47

ACR Abstract - 2015 Performance of the adult Systemic Sclerosis classification in juvenile systemic sclerosis patients. Results from the juvenile systemic sclerosis inception cohorte www.juvenile-scleroderma.com I. Foeldvari 1, J. Klotsche 13, M. Katsicas 2, M. Teresa Terreri 3, R. Cimaz 4, M. Kostic 5, F. Sztajnbok 6, D. Nemcova 7, M. Moll 8, M. Jose Santos 9, T. Avcin 10, J. Brunner 11, S. Nielsen 12, T. Kallinich 13, K. Minden 13, J. Müller 14, M. Janarthanan 15, Y. Uziel 16, A. Giraldo 17, D. Eleftheriou 18, K. Torok 19, N.Helmus 1.

Inception Cohort for juvenile systemic Sclerosis (jssc) " Data of juvenile systemic sclerosis patients are prospectively collected with a standardized assessment including quality of life " The aim of collecting data in patients with recent onset jssc is to learn more about the evolvement of organ involvement and the quality of life in these patients " The project should help to improve the care of these patients throughout the world

Inception Cohort for juvenile systemic Sclerosis (jssc) " Inclusion criteria # Patients diagnosed with juvenile systemic sclerosis ( fulfilling the adult classification criteria) # Developed the first non-raynaud symptom before the age of 16 years # Age at inclusion younger than 18 years of age " Method # Assessment every 6 months according to a standardized protocol # Assessment of organ involvement and patient related outcomes

Results All paediatric patients in the cohort fulfil the adult classification, as the main criteria of the paediatric classification, proximal skin sclerosis from MCP joint, already gives sufficient points to be classified as SSc. The pediatric patients had a mean score of: 15.3 at <6 months (n=13) 15.5 at <12 months( n=19) 17.2 at < 24 months ( n=25) 17.4 at <48 months (n=35). The cluster analysis showed 3 cluster of patients

Topics of the talk Epidemiology of jssc Classification issues Specific pediatric issues Comparison of the pediatric data to adult population with diffuse subtype

Are there clinical differences between male and female patients?

ARE THERE DIFFERENCES IN THE CLINICAL PRESENTATION ACCORDING ANTI-SCL70 POSITIVITY AND ANTI-SCL70 NEGATIVITY?

ARE THERE DIFFERENCES IN THE CLINICAL PRESENTATION ACCORDING THE AGE AT INCLUSION IN THE COHORT?

Is there a change in organ involvement pattern after 12 or 24 months? Results from the Juvenile Scleroderma Incpetion Cohort

Topics of the talk Epidemiology of jssc Classification issues Specific pediatric issues Comparison of the pediatric data to adult population with diffuse subtype

Publications selected for comparison 2. Scalapino K, Arkachaisri T, Lucas M, Fertig N, Helfrich DJ, Londino AV, Jr., et al. Childhood onset systemic sclerosis: classification, clinical and serologic features, and survival in comparison with adult onset disease. J Rheumatol. 2006;33(5):1004-13. 4. Martini G, Foeldvari I, Russo R, Cuttica R, Eberhard A, Ravelli A, et al. Systemic sclerosis in childhood: clinical and immunologic features of 153 patients in an international database. Arthritis Rheum. 2006;54(12):3971-8. 5. Foeldvari I, Zhavania M, Birdi N, Cuttica RJ, de Oliveira SH, Dent PB, et al. Favourable outcome in 135 children with juvenile systemic sclerosis: results of a multi-national survey. Rheumatology (Oxford). 2000;39(5):556-9. 24. Walker UA, Tyndall A, Czirjak L, Denton C, Farge-Bancel D, Kowal- Bielecka O, et al. Clinical risk assessment of organ manifestations in systemic sclerosis: a report from the EULAR Scleroderma Trials And Research group database. Ann Rheum Dis. 2007;66(6):754-63.

Promotion for our project I would like to invite you to participate on the Juvenile Inceptions Cohort Project www.juvenile-scleroderma.com If interested, please contact us: foeldvari@t-online.deor inceptioncohort @kinderrheumatologie.de

Conclusion Pediatric onset jssc has a distinct feature with the around 75% diffuse subtype of patients 15 to 29% of patients with overlap features Rare occurence of renal crisis, less pulmonary hypertension The follow up in the inception cohort showes a stabilisation of the organ involvement pattern, with a significant improvement in patient related outcomes We need more patients with longer follow up with standardized assessment to answer lot of open questions

Thanks for Your interest Looking forward to your questions

New Classification of Systemic Sclerosis Arth Rheum 2013,65: 2737-47