Article
Guidance for stem cell therapy for juvenile systemic sclerosis patients
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Authors
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Ivan Foeldvari
- Hamburg Centre for Pediatric and Adolescence Rheumatology, Hamburg
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Kathryn Torok
- University of Pittsburgh and University of Pittsburgh Medical Center Children’s Hospital of Pittsburgh, Pittsburgh
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Juliana Silva
- Great Ormond Street Hospital, London
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Paulina Horvei
- University of Pittsburgh and University of Pittsburgh Medical Center Children’s Hospital of Pittsburgh, Pittsburgh
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Christopher P. Denton
- UCL Division of Medicine, Royal Free London NHS Foundation Trust, London
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Franziska Roser
- University of Pittsburgh and University of Pittsburgh Medical Center Children’s Hospital of Pittsburgh, Pittsburgh
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Tamas Constantin
- Unit of Pediatric Rheumatology, Tűzoltó Street Department, Pediatric Centre, Semmelweis University, Budapest
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Patricia Costa Reis
- Faculdade de Medicina, Universidade de Lisboa, Lisboa
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Megan Curran
- Children’s Hospital Colorado, Colorado
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Maurizio Cutolo
- University of Genoa and IRCCS San Martino Polyclinic Hospital, Genoa
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Jörg Henes
- University Medical Center Tübingen, Tübingen
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Bernd Hinrichs
- Children’s pulmonology, Asklepios Klinik Nord – Heidberg, Hamburg
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Kim Fligelstone
- FESCA, London
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Suzanne Li
- Hackensack University Medical Center, Hackensack
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Susan Maillard
- Great Ormond Street Hospital, London
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Pia Moinzadeh
- University Hospital Cologne, Cologne
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Catherine Orteu
- Royal Free London NHS Foundation Trust, London
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Clare Pain
- Alder Hey Children’s Foundation NHS Trust, Liverpool
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Clarissa Pilkington
- Great Ormond Street Hospital, London
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Linda Schraven
- Dutch representative of Fesca, London
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Vanessa Smith
- Ghent University, Ghent
| Published: | September 18, 2024 |
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Outline
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Introduction: 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.
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, we formulated, based on nominal group technique, a guidance regarding HSCT and CT in jSSc patients.
Results: The following statements were voted by the multidisciplinary committee of experts:
All types of jSSc can be considered for a haematopoietic stem cell transplant or CT regardless of disease duration. 17/17 voted yes.
All types of juvenile systemic sclerosis (limited cutaneous, diffuse cutaneous, overlap and sine scleroderma) can be considered. 17/17 voted yes.
To be considered for HSCT or CT the patient should have: Progression of disease or lack of improvement despite treatment with ≥2 disease-modifying antirheumatic drugs (DMARDS). 17/17 voted yes.
Moderate or severe disease. 17/17 voted yes.
Been assessed by a multidisciplinary team with expertise in jSSc. 17/17 voted yes.
In addition to above, patients with jSSc should have ≥1 of the following: Moderate or severe skin involvement or progression of skin thickening. 17/17 voted yes.
Moderate or severe interstitial lung disease, moderate or severe respiratory impairment or progression of interstitial lung disease. 16 voted yes, 1 abstained.
Moderate or severe myositis/myopathy, or progression of myositis/myopathy. 17/17 voted yes.
Moderate or severe cardiac involvement or progression of cardiac involvement. 17/17 voted yes.
The following are additional considerations: Moderate or severe gastrointestinal dysfunction or progression of gastrointestinal dysfunction. 16/16 voted yes.
Moderate or severe arthritis/arthropathy or progression of arthritis/arthropathy. 14 voted yes, 2 abstained.
Moderate or severe cutaneous ulceration or progression of cutaneous ulceration. 12 voted yes, 1 voted no, 3 abstained.
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.
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.
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.
