Investigations of HCT for refractory JIA have employed high dose immunosuppressive drugs, such as CY and ATG with or without low dose TBI. Initial disease response has been observed in approximately two-thirds of patients; however, relapse occurs in about half of the responders. The current report describes a regimen with substantially increased dose of TBI, previously shown to be associated with high rate of disease response in adult patients with refractory MS and severe SSc. We report outcome of the lead patient in this pilot study, who remains in complete remission now, five yr after HCT. This regimen should be considered for patients with JIA refractory to medical therapies, including previous low intensity regimen HCT.
- autologous peripheral blood stem cells
- juvenile idiopathic arthritis
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health