Teng YKO et al. (2005) Long-term followup of health status in patients with severe rheumatoid arthritis after high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation. Arthritis Rheum 52: 2272–2276

High-dose chemotherapy (HDC) followed by autologous hematopoietic stem-cell transplantation (HSCT) is a new treatment strategy for patients with severe rheumatoid arthritis (RA) that is refractory to drug treatment. Although short-term studies had demonstrated significant improvement in signs and symptoms, the long-term impact of treatment had not been explored.

In this small Dutch study, Teng et al. followed eight patients (one male, seven female) who had undergone HDC and HSCT as part of a multicenter phase II trial. All had disease that was refractory to disease-modifying antirheumatic drugs and four had not responded to tumor-necrosis-factor blockade. Patients were assessed every 3 months in the first year, every 6 months in the second year and once more at 5 years.

Good clinical improvement was noted in four patients 6 months after treatment; a further two improved moderately and two did not improve. By 2-year follow-up, all patients had relapsed and were using antirheumatic medications; however, response to these medications was restored. Functionality and health status showed a short-term improvement, but had declined by the 5-year point, although health status remained above that seen at baseline. Analysis of the quality-adjusted life-years gained using conventional RA therapy compared with HDC plus HSCT, confirmed that (when treatment-related mortality is less than 2.8%) HDC plus HSCT is superior to conventional treatment.