Hematopoietic Stem-Cell Transplantation versus Immunosuppressive Therapy in Patients with Adult Acquired Severe Aplastic Anemia: A Cost-Effectiveness Analysis

OBJECTIVE: Controversy remains regarding which therapy to initially select for severe aplastic anemia (SAA) patients aged 35–50. This cost-effectiveness analysis aimed to use the Markov model to compare immunosuppressive therapy (IST) with hematopoietic stem-cell transplantation (HSCT) in age-stratified patients with SAA. METHODS: A cost-effectiveness analysis using a Markov model compared IST with HSCT in age-stratified patients with SAA. Baseline data were derived from a systematic literature review and collected from Huashan Hospital, Fudan University. The primary outcome was an incremental cost-effectiveness ratio (ICER). RESULTS: The HSCT strategy dominated in patients aged 18–35 even though it was $146,970 more expensive than IST, and the ICER of HSCT to IST was $14,054.19/quality-adjusted life-year (QALY), which was less than the willingness-to-pay value of $25,397.57/QALY. The IST strategy dominated in patients aged 35–50, because it was $72,009 less expensive than HSCT and yielded 3.24 QALYs more than HSCT. The model was vigorous in the sensitivity analyses of the key variables tested through the plausible ranges that were acquired from costing sources and previously published literature. CONCLUSION: The preferred induction strategy for patients aged 18–35 with SAA appears to be HSCT, and the preferred strategy for patients aged 35–50 is IST, which minimizes costs while maximizing QALYs.