High Dose Chemo and Hematopoietic Stem Cell Transplantation: Treatment Preference in Patients with RA and Rheumatologists

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High Dose Chemotherapy and Hematopoietic Stem Cell Transplantation: A

Study of Treatment Preference in Patients with Rheumatoid Arthritis and

Rheumatologists

ROBERT J. VERBURG, SASKIA D. MAHABALI, ANNE M. STIGGELBOUT, JACOB K.

SONT, and JACOB M. van LAAR

ABSTRACT.

Objective. Patients with intractable rheumatoid arthritis (RA) may

benefit from treatment with high dose chemotherapy followed by rescue

with autologous hematopoietic peripheral blood stem cell transplant

(HSCT). We investigated whether the risks of this approach are

acceptable to patients with RA and rheumatologists and whether risk

taking by patients was associated with disease characteristics,

socioeconomic variables, and/or personality traits.

Methods. A survey in the outpatient clinic was conducted among 2 cohorts

of 45 (cohort A) and 51 (cohort RA patients with active disease.

Patients received information about the potential benefit of HSCT (2/3

chance of a good clinical response, 1/3 no response) and treatment

related morbidity and mortality. Cure was assumed not to be a realistic

perspective. Cohort A was asked to choose between their own disease

state for an indefinite time or HSCT. Nonparametric tests were performed

to evaluate putative predictive factors that led patients to accept

transplant related mortality (TRM): swollen joint count, tender joint

count, visual analog scale (VAS) measures of disease activity and pain,

erythrocyte sedimentation rate, Health Assessment Questionnaire (HAQ),

socioeconomic variables, RA Quality of Life Questionnaire (RAQoL), and

the Life Orientation Test. Cohort B was asked to consider a worst case

scenario with respect to their disease activity. The minimal duration of

benefit was assessed, given a TRM of 0.01% and 2%. To evaluate treatment

preference of physicians, 96 Dutch rheumatologists responded to a

hypothetical clinical case analogous to the interviews with RA patients.

The minimum duration of benefit was assessed, given a TRM of 2% and the

maximal TRM acceptable to rheumatologists if duration of benefit was 2

years in 2/3 patients.

Results. In cohort A, 5 of 45 patients were willing to accept risk of

death. VAS disease activity (p = 0.006), VAS pain (p = 0.021), and HAQ

(p = 0.05) were significantly higher in patients willing to accept risk

of death. Religiosity (p = 0.093), a higher Ritchie Articular Index (p =

0.096), and low quality of life (by RAQoL) (p = 0.133) showed trends

toward risk taking. In cohort B, 22 of 50 patients (44%) were willing to

accept a risk of TRM related to HSCT. For the 22 patients the median

required duration of benefit given a TRM of 2% was 5 years (range 1-15).

Physicians also required a median duration of benefit of 5 years.

Conclusion. We evaluated risk taking in patients with RA and physicians

based on a realistic perspective in which the tradeoff between short

term risks and possible longterm benefit of HSCT was investigated. Based

on current efficacy data for HSCT (2 years improvement in 2/3 patients),

half the patients would accept the current TRM of 2%, based on registry

results. Patients willing to accept TRM had higher VAS disease activity,

VAS pain, and HAQ. Doctors were more willing to accept mortality in the

treatment of RA. (J Rheumatol 2002;29:1653-8)