Efficacy and cost-effectiveness of autologous bone marrow transplantation in metastatic breast cancer. Estimates using decision analysis while awaiting clinical trial results.
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.


METHODS
Decision analysis model using a Markov process.


METHODS
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.


METHODS
Hypothetical cohorts of women with metastatic breast cancer who had no bone marrow involvement and no comorbid illness.


METHODS
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.


METHODS
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.


CONCLUSIONS
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.
Hillner
B E
BE
Department of Internal Medicine, Medical College of Virginia, Virginia Commonwealth University, Richmond 23298.
Smith
T J
TJ
Desch
C E
CE
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000191:economics*; D001943:Breast Neoplasms / Q000191:economics* / Q000628:therapy; D002986:Clinical Trials as Topic; D003362:Cost-Benefit Analysis; D003661:Decision Support Techniques; D005260:Female; D006801:Humans; D008390:Markov Chains; D008875:Middle Aged; D009362:Neoplasm Metastasis; D011788:Quality of Life; D012680:Sensitivity and Specificity