Key clinical point: Plerixafor in combination with granulocyte colony-stimulating factor (G-CSF) and azacitidine appears tolerable when given over 5 days and has encouraging response rates in patients with myelodysplastic syndromes (MDS).
Major finding: Two complete responses and 6 marrow responses were seen for an overall response rate (ORR) of 36% in evaluable patients. The ORR for patients receiving plerixafor (any dose), G-CSF, and azacitidine was 53%. Blast mobilization during treatment correlated with response, with patients that mobilized greater than 2-fold having an ORR of 60% vs. 17% for those that did not ( P = .035).
Study details: The data come from an open-label, phase 1 study of 28 MDS patients treated with plerixafor, G-CSF, and azacitidine with a standard 3 + 3 design with 3 dose escalation cohorts of plerixafor 320 mg/kg/day, 440 mg/kg/day, and 560 mg/kg/day.
Disclosures: The study was supported by the American Society of Hematology Clinical Research Training Institute. Four of the authors reported receiving honoraria from Sanofi and 1 received honoraria from Bristol Myers Squibb.
Source: Huselton E et al. Leuk Lymphoma. 2021 Jan 19. doi: 10.1080/10428194.2021 .