Departments of Rheumatology and
1 Medical Decision Making, Leiden University Medical Center, PO Box 9600, 2300 RC Leiden, The Netherlands
SIR, We read with interest the article by Snowden et al. [1] on risk taking in rheumatoid arthritis (RA) patients in the context of haemopoietic stem cell transplantation (HSCT). HSCT is an experimental treatment for a variety of autoimmune diseases, including RA. With over 200 cases of autologous HSCT registered, preliminary results show the feasibility and potential clinical effectiveness of various protocols (A. Tyndall, personal communication). The treatment-related mortality (TRM) of 9% may be related to patient and/or disease selection. It underscores, however, the hazard of extrapolating TRM from other studies involving HSCT in malignant diseases such as breast carcinoma. In assessing risk/benefit, patients also need to be informed about realistic therapeutic goals. At present, long-term remissions lasting up to 21 months have been reported in RA patients after HSCT [2]. However, persistence of disease activity or relapses occurring within several years after the procedure should caution against unrealistic optimism [3]. Furthermore, given that HSCT is considered optional for patients with severe, refractory disease with a major destructive component, functional capacity as scored by the health assessment questionnaire may not normalize after HSCT [4]. Without doubt, patients will accept lower TRM when temporary improvement of disease activity instead of cure is the optimal achievable goal.
We do not wish to imply, however, that HSCT is not a treatment option for RA patients. We have selected 19 patients with intractable, refractory RA who were considered eligible for HSCT. The patients were informed of the risks associated with HSCT including a TRM arbitrarily set at 5% and of the presumed benefit, long-term improvement obviating the need for disease-modifying anti-rheumatic drugs. Ten patients gave no consent because of the potential of TRM (9/10) or infertility (1/10), nine patients have given informed consent. Seven patients recently received the treatment resulting in significant improvement [>50% according to American College of Rheumatology (ACR) response criteria] of disease activity in six. Although the numbers were small, the groups did not differ in age or health assessment questionnaire, considered predictors of acceptability of risk according to Snowden et al. When asked retrospectively after HSCT how long the duration of good clinical response should be in order to make HSCT worthwhile, the six patients' answers varied from 6 to 24 months. Of course, the answer is greatly influenced by the tolerability of the treatment.
Based on our experiences, we fully support the conclusion of Snowden et al., namely that the decision to proceed with HSCT is a very personal one which must follow extensive explanations and counselling.
References