AB1717 GUIDANCE FOR STEM CELL THERAPY FOR JUVENILE SYSTEMIC SCLEROSIS PATIENTS
Authors: I. Foeldvari, K. Torok, J. Silva, P. Horvei, C. P. Denton, F. Roser, T. Constantin, P. Costa Reis, M. Curran, M. Cutolo, J. Henes, B. Hinrichs, K. Fligelstone, S. Li, S. Maillard, P. Moinzadeh, C. Orteu, C. Pain, C. Pilkington, L. Schraven, V. Smith
Journal: Annals of the Rheumatic Diseases (2024)

<h3>Background:</h3> Hemopoetic stem cell transplantation (HSTC) and cellular therapies(CT) are a promising therapeutic options for adult and for juvenile systemic sclerosis (jSSc) patients. Currently, studies regarding efficacy of this procedure are derived mostly from adult patients. As pediatric patients have a unique potential to recover and remodel, the applicability of the adult stem cell transplant criteria to pediatric patients with jSSc is unclear/ unknown and may be too stringent. <h3>Objectives:</h3> To develop guidance for HSCT and CT for patients with jSSc. <h3>Methods:</h3> At a multidisciplinary expert workshop conducted in Hamburg Germany in December 2023, adult and pediatric data were reviewed regarding indications and effectiveness of HSCT and CT. We discussed the extent adult data and criteria can be extrapolated to juvenile patients, and its limitations. In the consensus meeting, we formulated, based on nominal group technique,a guidance regarding HSCT and CT in jSSc patients. <h3>Results:</h3> The following statements were voted by the multidisciplinary committee of experts: 1.All types of jSSc can be considered for a haematopoietic stem cell transplant or CT regardless of disease duration. 17/17 voted yes. 2.All types of juvenile systemic sclerosis (limited cutaneous, diffuse cutaneous, overlap and sine scleroderma) can be considered. 17/17 voted yes. 3.To be considered for HSCT or CT the patient should have: a.Progression of disease or lack of improvement despite treatment with ≥2 disease-modifying antirheumatic drugs (DMARDS). 17/17 voted yes. b.Moderate or severe disease. 17/17 voted yes. c.Been assessed by a multidisciplinary team with expertise in jSSc. 17/17 voted yes. 4.In addition to above, patients with jSSc should have ≥ 1 of the following: a.Moderate or severe skin involvement or progression of skin thickening. 17/17 voted yes. b.Moderate or severe interstitial lung disease, moderate or severe respiratory impairment or progression of interstitial lung disease. 16 voted yes, 1 abstained. c.Moderate or severe myositis/myopathy, or progression of myositis/myopathy. 17/17 voted yes. d.Moderate or severe cardiac involvement or progression of cardiac involvement. 17/17 voted yes. 5.The following are additional considerations: a.Moderate or severe gastrointestinal dysfunction or progression of gastrointestinal dysfunction. 16/16 voted yes. b.Moderate or severe arthritis/arthropathy or progression of arthritis/arthropathy. 14 voted yes, 2 abstained. c.Moderate or severe cutaneous ulceration or progression of cutaneous ulceration. 12 voted yes, 1 voted no, 3 abstained. 6.When considering stem cell transplant or CT (in regards to 4 and 5), greater importance was attributed to progression rather than degree of severity by the expert panel. 16 voted yes. 7.Paediatric patients have a unique potential to recover and remodel, the applicability of the adult HSCT criteria to paediatric patients with jSsc is unclear/unknown and may be too stringent. The expert panel defers the decision on who should be excluded from stem cell transplantation or CT to the local pediatric bone marrow transplant team. 15/15 voted yes. <h3>Conclusion:</h3> We established a guidance for HSCT and CT for jSSc, which will help worldwide to standardize the inclusion criteria and make the results of the future procedures more comparable. We hope that HSCT and CT will have standardized exclusion criteria and transplant protocols to enable data collection, interpretation and improve outcomes and care. <h3>REFERENCES:</h3> <b>NIL.</b> <h3>Acknowledgements:</h3> <b>NIL.</b> <h3>Disclosure of Interests:</h3> Ivan Foeldvari: None declared, Kathryn Torok: None declared, Juliana Silva: None declared, Paulina Horvei Janssen und Boehringer Ingelheim, Janssen, GlaxoSmithKline, Boehringer Ingelheim, Roche, CSL Behring, Corbus, Acceleron, Horizon, Arxx Therapeutics, Lilly, Novartis, Certa, GSK, Abbvie, Horizon, Christopher P Denton: None declared, Franziska Roser: None declared, Tamas Constantin: None declared, Patricia Costa Reis: None declared, Megan Curran: None declared, Maurizio Cutolo: None declared, Jörg Henes: None declared, Bernd Hinrichs: None declared, Kim Fligelstone: None declared, Suzanne Li: None declared, Susan Maillard: None declared, Pia Moinzadeh: None declared, Catherine Orteu: None declared, Clare Pain: None declared, Clarissa Pilkington: None declared, Linda Schraven: None declared, Vanessa Smith: None declared.