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. 2025 Sep 1:keaf416.
doi: 10.1093/rheumatology/keaf416. Online ahead of print.

Allogeneic hematopoietic stem cell transplantation for severe Still's disease: a retrospective report of 3 pediatric patients

Muriel Schmutz  1 , Jade Cognard  2 , Alice Hadchouel  3   4 , Laureline Berteloot  5 , Veronique Hentgen  6 , Christine Pietrement  2 , Laurye-Anne Eveillard  1 , Paul Bastard  1   4 , Martin Castelle  1 , Marwa Chbihi  1   4 , Romain Levy  1   4 , Anne Welfringer-Morin  7 , Marie Pouletty  8 , Pierre Quartier  1   4 , Bénédicte Neven  1   4 , Despina Moshous  1   4 , Benjamin Fournier  1 , Marie Louise Frémond  1   4
Affiliations

Allogeneic hematopoietic stem cell transplantation for severe Still's disease: a retrospective report of 3 pediatric patients

Muriel Schmutz et al. Rheumatology (Oxford). .

Abstract

Objectives: Severe forms of systemic juvenile idiopathic arthritis (sJIA), also called pediatric-onset Still's disease are associated with two major life-threatening complications: macrophage activation syndrome (MAS) and severe lung disease. Patients are usually resistant to conventional synthetic (cs) Disease-Modifying Antirheumatic Drugs (DMARDs), biologic (b) DMARDs, and targeted synthetic (ts) DMARDs. Recently, allogeneic hematopoietic stem cell transplantation (HSCT) has been performed in a small number of patients with refractory and life-threatening disease. We aimed to report outcomes and complications of allogeneic HSCT in patients with severe, refractory sJIA treated at our center.

Methods: We conducted a retrospective, observational, single-center study in a tertiary pediatric immunology care center (Necker Hospital, Paris, France).

Results: We report 3 sJIA patients who underwent allogeneic HSCT at a median age of 3.5 years. All had recurrent MAS; 2 had lung disease and the HLA-DRB1*15 haplotype, associated with severe delayed hypersensitivity to IL-1/IL-6 inhibitors. Donors were matched sibling donors for the first and third patient, and matched unrelated donor for the second patient. They presented multiple post-graft complications: graft-vs-host disease, infections, thrombotic microangiopathy and severe inflammatory complications on previously affected organs, such as skin and lungs. At a median follow-up of 22 months (20-33) after transplantation, they were all in remission with full-donor chimerism and were off immunosuppressive treatment.

Conclusion: Allogeneic HSCT can be an effective salvage therapy in patients with refractory sJIA. However the risk of post-transplant endothelial complications and severe inflammation in previously affected organs, such as joints, skin and lungs deserves particular attention.

Keywords: Still’s disease; allogeneic HSCT; lung disease; macrophage activation syndrome; systemic juvenile idiopathic arthritis.

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