ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 2204

Guidance for Stem Cell Therapy for Juvenile Systemic Sclerosis Patients

Ivan Foeldvari1, Kathryn Torok2, Juliana Silva3, Paulina Horvei4, Christopher Denton5, Franziksa Rosser6, Tamas Constantin7, Patricia Costa Reis8, Megan Curran9, Maurizio Cutolo10, Joerg Henes11, Bernd Hinrichs12, Kim Fligelstone13, Suzanne Li14, Susan Maillard3, Pia Moinzadeh15, Catherine Orteu16, Clare Pain17, Clarissa Pilkington18, Linda Schraven19 and vanessa smith20, 1Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany, 2Division of Rheumatology, Scleroderma Center, Department of Pediatrics, UPMC Children’s Hospital of Pittsburgh, Pittsburgh, PA, USA, Pittsburgh, PA, 3Great Ormond Street Hospital, London, United Kingdom, 4Division of Blood and Marrow Transplantation and Cellular Therapies, Department of Pediatrics, UPMC Children’s Hospital of Pittsburgh, Pittsburgh, PA, USA, Pittsburgh, 5University College London, Northwood, United Kingdom, 6Division of Pulmonology Medicine, Department of Pediatrics, UPMC Children’s Hospital of Pittsburgh, Pittsburgh, PA, USA, Pittsburgh, PA, 7Semmelweis University, Budapest, Hungary, 8Hospital de Santa Maria, Lisbon, Portugal, 9Children's Hospital Colorado, Colorado, 10Laboratory of Experimental Rheumatology and Academic Division of Clinical Rheumatology, Department of Internal Medicine, University of Genova, IRCCS Ospedale Policlinico San Martino, Genoa, Italy, Genova, Italy, 11University Hospital Tuebingen, Tuebingen, Germany, 12Children's pulmonology, Asklepios Klinik Nord – Heidberg, Hamburg, Germany, 13FESCA, London, United Kingdom, 14Hackensack University Medical Center, Hackensack, 15University Hospital Cologne, Cologne, Germany, 16Royal Free London NHS Foundation Trust, London, United Kingdom, 17Alder Hey Children’s Foundation NHS Trust, Liverpool, United Kingdom, 18Great Ormond Street Hospital, London, United Kingdom, 19Dutch representative of Fesca, London, United Kingdom, 20Ghent University Hospital, Gent, Belgium

Meeting: ACR Convergence 2024

Keywords: ,

Session Information

Date: Monday, November 18, 2024

Title: Pediatric Rheumatology – Clinical Poster III

Session Type: Poster Session C

Session Time: 10:30AM-12:30PM

Background/Purpose: Hemopoetic stem cell transplantation(HSTC) and cellular therapies(CT) are a promising therapeutic options for juvenile systemic sclerosis(jSSc) 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.

Methods: 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 based on nominal group technique,a guidance regarding HSCT and CT in jSSc was formulated.

Results: 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.

 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,

 c) Moderate or severe cutaneous ulceration or progression of cutaneous ulceration. 12 voted yes.

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.

Conclusion: 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.

Disclosures: I. Foeldvari: Boehringer-Ingelheim, 1, Eli Lilly, 6, miirsubishi, 2; K. Torok: None; J. Silva: None; P. Horvei: None; C. Denton: AbbVie, 2, 5, Acceleron, 2, Arxx Therapeutics, 2, 5, Bayer, 2, Boehringer Ingelheim, 2, 6, Certa, 2, Corbus, 2, CSL Behring, 2, 5, Galapagos, 2, GlaxoSmithKline, 2, 5, 6, Horizon, 2, 5, Inventiva, 2, Janssen, 2, 6, Lilly, 2, Novartis, 2, Roche, 2, Sanofi-Aventis, 2, Servier, 5, Zurabio, 2; F. Rosser: None; T. Constantin: None; P. Costa Reis: None; M. Curran: None; M. Cutolo: AbbVie/Abbott, 5, Amgen, 5, Balcacci, 2, 5, Boehringer-Ingelheim, 2, 5, Bristol-Myers Squibb(BMS), 2, 5, UCB, 5; J. Henes: None; B. Hinrichs: None; K. Fligelstone: None; S. Li: None; S. Maillard: None; P. Moinzadeh: None; C. Orteu: None; C. Pain: None; C. Pilkington: None; L. Schraven: None; v. smith: Argenx, 2, BKC Moving Media Makers, 6, Boehringer-Ingelheim, 2, 5, 6, Janssen, 2, 5, 6, WebMDD Global LLC, 2.

To cite this abstract in AMA style:

Foeldvari I, Torok K, Silva J, Horvei P, Denton C, Rosser F, Constantin T, Costa Reis P, Curran M, Cutolo M, Henes J, Hinrichs B, Fligelstone K, Li S, Maillard S, Moinzadeh P, Orteu C, Pain C, Pilkington C, Schraven L, smith v. Guidance for Stem Cell Therapy for Juvenile Systemic Sclerosis Patients [abstract]. Arthritis Rheumatol. 2024; 76 (suppl 9). https://acrabstracts.org/abstract/guidance-for-stem-cell-therapy-for-juvenile-systemic-sclerosis-patients/. Accessed .

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