Abstract

Background Patients with intractable rheumatoid arthritis (RA) may benefit from treatment with high dose chemotherapy (HDC) followed by rescue with autologous peripheral blood stem cells (SCT). The aim of this study was to establish whether the risks of this approach are acceptable to patients with RA and whether risk taking was associated with disease associated, socio-economic parameters and/or personality. Objectives Methods 45 RA patients with active RA enrolled. Patients received information about the potential benefit of HDC (2/3 of patients good clinical response, 1/3 not), observed toxicities (alopecia, infections and bleedings) and duration of hospitalisation. Cure was assumed not te be a realistic perspective. Patients were asked what the minimal duration of benefit should be, given a transplant related mortality (TRM) of 0.01% and 2%. Furthermore maximal accepted TRM was asked given a duration of benefit of 2 years. Analysis of predictive factors of patients? willingness to accept risk of death by the Mann-Whitney U-test was performed with parameters of disease activity (Swollen Joint Count, Tender Joint Count, VAS, ESR and HAQ), socio-economic parameters, RAQoL and the Life Orientation Test. Results Of the 45 patients, 5 patients were willing to accept TRM in the treatment of RA, 40 patients were not. Of these 5 patients the mean duration of benefit given a TRM of 0.01% was 45 months (range 2‑96). For a TRM of 2% the mean duration was 52 months (range 6‑120). VAS disease activity (P = 0.006), VAS pain (P = 0.021) and HAQ (P = 0.05) were signicantly higher in these patients. Religiosity (P = 0.093) and a higher Ritchie articular index (P = 0.1) showed a trend towards risk taking. Conclusion We considered an approach to investigate risk taking in patients with RA based on a realistic perspective in which the trade off between short term risks and possible long term gain of HDC was investigated. It was found that patient with a chronic disease such as RA in which prevention of morbidity instead of mortality is the major

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