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

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. To evaluate the cost-utility of preemptive versus rescue use of plerixafor for hematopoietic stem cell mobilization inMM patients. 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. 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. 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.