Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC).

ABSTRACT: This study (NCT01288573) investigated plerixafor's safety and efficacy in children with cancer. Stage 1 investigated the dosage, pharmacokinetics (PK), pharmacodynamics (PD), and safety of plerixafor?+?standard mobilization (G-CSF?±?chemotherapy). The stage 2 primary endpoint was successful mobilization (doubling of peripheral blood CD34+ cell count in the 24?h prior to first apheresis) in patients treated with plerixafor?+?standard mobilization vs. standard mobilization alone. In stage 1, three patients per age group (2-<6, 6-<12, and 12-<18 years) were treated at each dose level (160, 240, and 320?µg/kg). Based on PK and PD data, the dose proposed for stage 2 was 240?µg/kg (patients 1-<18 years), in which 45 patients were enrolled (30 plerixafor arm, 15 standard arm). Patient demographics and characteristics were well balanced across treatment arms. More patients in the plerixafor arm (24/30, 80%) met the primary endpoint of successful mobilization than in the standard arm (4/14, 28.6%, p?=?0.0019). Adverse events reported as related to study treatment were mild, and no new safety concerns were identified. Plerixafor?+?standard G-CSF?±?chemotherapy mobilization was generally well tolerated and efficacious when used to mobilize CD34+ cells in pediatric cancer patients.

SUBMITTER: Morland B 

PROVIDER: S-EPMC7452813 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature