Cost-utility of pre-emptive plerixafor versus rescue plerixafor in the mobilization of hematopoietic stem cells in multiple myeloma

BACKGROUND: Multiple myeloma (MM) is a hematologic cancer with rising incidence worldwide. Autologous stem cell transplantation (ASCT) is a key treatment for eligible patients, but mobilization failure remains a major obstacle. Plerixafor enhances stem cell mobilization, but its high costs and lack of standardized protocol prevent its widespread use. OBJECTIVE: To evaluate the cost-utility of preemptive versus rescue use of plerixafor for hematopoietic stem cell mobilization inMM patients. METHODS: A Markov model was developed using real-world data from 196 MM patients undergoing stem cell mobilization at two SãoPaulo transplant centers in Brazil. The model compared two strategies: (1) preemptive plerixafor and (2) rescue plerixafor use following mobilization failure. Transition probabilities, utilities, and costs were informed by clinical data and literature. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: The preemptive strategy led to higher rates of successful mobilization and ASCT, resulting in greater quality-adjusted life years (QALYs), but also higher costs. Still, it demonstrated favorable results compared to the rescue approach and passed the Brazilian willingness to pay thresholds of acceptability. CONCLUSION: Preemptive plerixafor passes the cost-utility guidelines to be used in MM patients in Brazil, potentially guiding policy decisions on resource allocation within the national health system.