Rheumatology, Vol 38, 321-324, Copyright © 1999 by British Society for Rheumatology
JA Snowden, I Nivison-Smith, JC Biggs and PM Brooks
OBJECTIVES: Autologous haemopoietic stem cell transplantation (HSCT), which
carries defined risks of early treatment-related mortality (TRM), has
recently been proposed as an experimental therapy for severe rheumatoid
arthritis (RA). The aim of this study was to establish whether the risks of
this approach 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 acceptable 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 self-
assessed disability. RESULTS: The 53 patients (age range 24-69 yr, 39
female, 14 male, disease duration 2-43 yr) interviewed were prepared to
accept a broad range of treatment-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 Questionnaire (HAQ; P = 0.001) and
negatively related to age (P = 0.04), although only HAQ score maintained
significance on multivariate analysis. Using linear 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 long-term efficacy can be
proven, then the risks of autografting may be acceptable to most patients
with RA, particularly those with significant disability.
ORIGINAL PAPERS
Risk taking in patients with rheumatoid arthritis: are the risks of haemopoietic stem cell transplantation acceptable?
Department of Haematology, St Vincent's Hospital, Sydney, Australia.
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