Juvenile Idiopathic Arthritis
The term juvenile idiopathic arthritis (JIA) encompasses a heterogeneous group of pediatric diseases, having joint inflammation as a common feature and, conversely, variable degrees of systemic involvement. JIA is the most common indication to autologous HSCT in pediatric patients, although most of the reports derive from the activity of a single investigation group. The relatively low mortality rate of conventionally treated JIA does not seem at a glance to favor autologous HSCT as a therapeutic strategy. On the other hand, refractory JIA is a severely disabling disease profoundly affecting QoL; moreover, JIA with systemic involvement accounts for most of the disease-related mortality. Relying on these considerations, autografting has been explored as possibly a cost-effective therapeutic strategy in refractory JIA patients. The HSCT procedure per se is characterized by a considerable rate of TRM, with late infectious complications, attributable to long-lasting immunosuppression, being the leading cause. A characteristic complication is the macrophage activation syndrome (MAS); this is a disease-linked phenomenon[160,161] that can arise during the mobilizing phase, where it can be interpreted as an outstanding example of the most general problem of disease flare-up during the mobilizing phase, and after autografting.[158,162] Long-term results can be differently interpreted: obtaining a notable CR rate, although rather low, may be regarded as positive in refractory patients. On the other hand, four deaths within 16 months and a long-term DFS of approximately 40% in children series, do not authorize an overwhelming enthusiasm. By considering the available data regarding HSCT in JIA, the question can be raised as to whether attempts should be addressed to optimize autografting procedure or to improve conventional treatment strategies.
Expert Rev Hematol. 2009;2(6):699-715. © 2009 Expert Reviews Ltd.
Cite this: Autologous Hematopoietic Stem Cell Transplantation in Autoimmune Diseases - Medscape - Dec 01, 2009.