Bone-marrow derived mesenchymal stromal cells infusion in therapy refractory juvenile idiopathic arthritis patients

Joost F Swart, Sytze de Roock, Rutger A J Nievelstein, Ineke C M Slaper-Cortenbach, Jaap J Boelens, Nico M Wulffraat

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

OBJECTIVES: To compare the total number of adverse events (AEs) before and after mesenchymal stromal cell (MSC) infusion in refractory JIA and to evaluate its effectiveness. METHODS: Single-centre Proof of Mechanism Phase Ib, open label intervention study in JIA patients previously failing all biologicals registered for their diagnosis. Six patients received 2 million/kg intravenous infusions of allogeneic bone-marrow derived MSC. In case of ACR-Ped30-response but subsequent loss of response one and maximal two repeated infusions are allowed. RESULTS: Six JIA patients with 9.2 years median disease duration, still active arthritis and damage were included. All had failed methotrexate, corticosteroids and median five different biologicals. MSC were administered twice in three patients. No acute infusion reactions were observed and a lower post-treatment than pre-treatment incidence in AEs was found. The one systemic onset JIA (sJIA) patient had again an evolving macrophage activation syndrome, 9 weeks after tocilizumab discontinuation and 7 weeks post-MSC infusion. Statistically significant decreases were found 8 weeks after one MSC infusion in VAS well-being (75-56), the JADAS-71 (24.5-11.0) and the cJADAS10 (18.0-10.6). CONCLUSION: MSC infusions in six refractory JIA patients were safe, although in sJIA stopping the 'failing' biologic treatment carries a risk of a MAS flare, as the drug might still suppress the systemic features. TRIAL REGISTRATION: Trial register.nl, http://https://www.trialregister.nl, NTR4146.

Original languageEnglish
Pages (from-to)1812-1817
Number of pages6
JournalRheumatology (Oxford, England)
Volume58
Issue number10
Early online date27 Apr 2019
DOIs
Publication statusPublished - 1 Oct 2019

Keywords

  • Juvenile arthritis
  • mesenchymal stromal cells
  • stem cell transplantation
  • therapeutics

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