Efficacy and cost-effectiveness of autologous bone marrow transplantation in metastatic breast cancer. Estimates using decision analysis while awaiting clinical trial results
B. E. Hillner, T. J. Smith and C. E. Desch
Department of Internal Medicine, Medical College of Virginia, Virginia Commonwealth University, Richmond 23298.
OBJECTIVE--To assess the efficacy and cost-effectiveness of standard
chemotherapy and high-dose chemotherapy with autologous bone marrow
transplantation (ABMT) in metastatic breast cancer. DESIGN--Decision
analysis model using a Markov process. SETTING--Response and recurrence
rates from the published literature for standard therapy and from case
series of ABMT. Costs were based on local charges and on adjusted Medicare
data. PATIENTS--Hypothetical cohorts of women with metastatic breast cancer
who had no bone marrow involvement and no comorbid illness.
INTERVENTION--The standard chemotherapy cohort received cyclophosphamide,
doxorubicin, and fluorouracil. The ABMT cohort was treated with intense
induction chemotherapy, then additional high-dose chemotherapy following a
remission, with ABMT support. MAIN OUTCOME MEASURES--Anticipated survival,
incremental cost per year of life, and incremental cost per
quality-adjusted year of life gained using a 5-year time horizon. Rigorous
sensitivity analyses were done, including assessing a benefit "tail" of
normal life expectancy for those free of disease after 5 years.
RESULTS--ABMT was the preferred approach under almost all assumptions, but
the size of the benefit varied greatly. ABMT had a survival benefit of 6.0
months at 5 years at an incremental cost of $115,800 per year of life
saved. If patients who were free of disease after 5 years had normal
survival, the benefit was 18.1 months at an incremental cost of $28,600 per
year. The benefit of ABMT was primarily dependent on whether the recurrence
risk was constant or decreases after a finite period of time.
CONCLUSION--Using reasonable assumptions, ABMT provided a substantial
benefit but at a cost that may be untenable. Decision analysis highlights
the limitations in the currently available data and the assumptions made
for the emotional question of using ABMT in metastatic breast cancer. The
model supports the need for randomized clinical trials.
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