Intense Immunosuppression and Stem-Cell Transplantation for Patients With Severe Rheumatic Autoimmune Disease: A Review

Jacob M. van Laar, MD, Alan Tyndall, MD

Disclosures

Cancer Control. 2003;10(1) 

In This Article

Juvenile Idiopathic Arthritis

Patients with JIA, a heterogeneous disease, experience different outcomes, depending on the subtype. Systemic and polyarticular forms carry the burden of morbidity and mortality, which explains why these subsets in particular have been targeted with SCT.[43] At present, data are available on 15 patients from a single-regimen study in the Netherlands, as part of the 43 patients from the International Registry. In a preliminary report on the first 4 patients,[44] conditioning with cyclophosphamide (200 mg/kg), ATG (20 mg/kg) and low-dose total body irradiation (4 Gy), followed by rein-fusion of T-cell-depleted bone marrow SCT resulted in eight full remissions and two partial remissions. Unfortunately, following transplant, 2 patients died of infection-associated hemophagocytic syndrome known as macrophage activation syndrome. The same fatal complication was also noted in another study,[43] and a possible role of in vivo T-cell depletion has been suggested. This led to modifications of protocols, and no mortality has occurred since.

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