Risk taking in patients with rheumatoid arthritis: are the risks of haemopoietic stem cell transplantation acceptable?

Objectives. Autologous haemopoietic stem cell transplantation (HSCT), which carries defined risks of early treatment-related mortality (TRM), has rece ntly been proposed as an experimental therapy for severe rheumatoid arthrit is (RA). The aim of this study was to establish whether the risks of this a pproach are acceptable to patients with RA and whether risk taking related to disease-associated or personal/social parameters. Methods. A standard gamble questionnaire was used to determine the acceptab le risk of mortality for a potentially curative procedure in patients with RA aged <70 yr. Additional data collected included age, sex, duration of RA , number of second-line agents, domestic and workforce information, and sel f-assessed disability. Results. The 53 patients (age range 24-69 yr, 39 female, 14 male, disease d uration 2-43 yr) interviewed were prepared to accept a broad range of treat ment-related mortality in order to be returned to normality off all drugs ( median 5%, range 0-50%). Risk taking was significantly related to degree of disability measured by the disability section of the Health Assessment Que stionnaire (HAQ; P = 0.001)and negatively related to age (P = 0.04), althou gh only HAQ score maintained significance on multivariate analysis. Using l inear regression, we were able to determine that current TRM of autologous HSCT in Australia (3.3%) would be acceptable to patients with HAQ scores of >0.44 (84% of our sample), but allogeneic HSCT (with a TRM of 13.1%) would be acceptable only to severely disabled patients with HAQ scores of >2.45 (4% of our sample), assuming the procedure to be curative. Conclusion. Along with previous studies, these results suggest that, if lon g-term efficacy can be proven, then the risks of autografting may be accept able to most patients with RA, particularly those with significant disabili ty.