Safety and Effectiveness of Plerixafor for Peripheral Blood Stem Cell Mobilization in Autologous Stem Cell Transplantation: Results of a Post-Marketing Surveillance Study

Background Plerixafor was approved in Japan in 2016 for peripheral blood stem cell (PBSC) mobilization in autologous stem cell transplantation (A-SCT). Objective Our objective was to evaluate the safety and effectiveness of plerixafor in Japanese patients undergoing A-SCT for various indications in real-world practice. Patients and Methods This post-marketing surveillance study included Japanese patients initiating PBSC mobilization with plerixafor for A-SCT. Safety assessments included the incidence of adverse events (AEs) including serious AEs, adverse drug reactions (ADRs), and laboratory variables. Effectiveness assessments were the proportion of patients with the target CD34+ cell yield (>= 2 x 10(6) cells/kg) <= 4 days after plerixafor administration and the number of days required to reach the target CD34+ cell yield. Results In total, 785 patients were registered, and the safety and effectiveness analysis sets comprised 764 and 717 patients, respectively. ADRs occurred in 12.2% of patients, with gastrointestinal disorders (5.5%), laboratory investigations (4.5%), and blood and lymphatic system disorders (3.0%) being the most common. A total of 71.1% of patients had the target CD34+ cell yield within <= 4 days of treatment, with a mean (standard deviation) of 1.3 (0.7) days to reach the target CD34+ cell yield. Over 80% of patients with a baseline CD34+ cell count >2 cells/mu L had a target CD34+ cell yield within <= 4 days of treatment. Conclusions This large post-marketing surveillance study provided real-world evidence detailing the safety and effectiveness of plerixafor for PBSC mobilization in Japanese patients undergoing A-SCT. Importantly, no new safety concerns were identified, and the safety profile of plerixafor was consistent with the established profile of this drug.