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Case Reports
. 2026 Jan 8;65(1):keaf416.
doi: 10.1093/rheumatology/keaf416.

Allogeneic haematopoietic stem cell transplantation for severe Still's disease: a retrospective report of three paediatric 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
Case Reports

Allogeneic haematopoietic stem cell transplantation for severe Still's disease: a retrospective report of three paediatric patients

Muriel Schmutz et al. Rheumatology (Oxford). .

Abstract

Objectives: Severe forms of systemic JIA (sJIA), also called paediatric-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) DMARDs, biologic (b) DMARDs, and targeted synthetic (ts) DMARDs. Recently, allogeneic haematopoietic 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 centre.

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

Results: We report three sJIA patients who underwent allogeneic HSCT at a median age of 3.5 years. All had recurrent MAS; two 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 JIA.

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