Publication details

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)

Authors	MORLAND B. KEPÁK Tomáš DALLORSO S. SEVILLA J. MURPHY D. LUKSCH R. YANIV I. BADER P. ROSSLER J. BISOGNO G. MAECKER-KOLHOFF B. LANG P. T. ZWAAN C. M. SUMERAUER D. KRIVAN G. BERNARD J. LIU Q. Y. DOYLE E. LOCATELLI F.
Year of publication	2020
Type	Article in Periodical
Magazine / Source	Bone Marrow Transplantation
MU Faculty or unit	Faculty of Medicine
Citation
web	https://www.nature.com/articles/s41409-020-0836-2
Doi	http://dx.doi.org/10.1038/s41409-020-0836-2
Keywords	COLONY-STIMULATING FACTOR; NON-HODGKINS-LYMPHOMA; CHEMOKINE RECEPTOR; PROGENITOR CELLS; CHILDREN; ANTAGONIST; EFFICACY; AMD3100
Description	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 mu g/kg). Based on PK and PD data, the dose proposed for stage 2 was 240 mu g/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.