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.

Morland, B., Kepak, T., Dallorso, S., Sevilla, J., Murphy, D., Luksch, R., Yaniv, I., Bader, P., Rossler, J., Bisogno, G., Maecker-Kolhoff, B., Lang, P., Zwaan, C. M., Sumerauer, D., Krivan, G., Bernard, J., Liu, Q., Doyle, E., Locatelli, F., 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), <<BONE MARROW TRANSPLANTATION>>, 2020; 55 (9): 1744-1753. [doi:10.1038/s41409-020-0836-2] [https://hdl.handle.net/10807/229758]

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)

Locatelli, Franco
Ultimo
Writing – Review & Editing
2020

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.
2020
Inglese
Morland, B., Kepak, T., Dallorso, S., Sevilla, J., Murphy, D., Luksch, R., Yaniv, I., Bader, P., Rossler, J., Bisogno, G., Maecker-Kolhoff, B., Lang, P., Zwaan, C. M., Sumerauer, D., Krivan, G., Bernard, J., Liu, Q., Doyle, E., Locatelli, F., 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), <<BONE MARROW TRANSPLANTATION>>, 2020; 55 (9): 1744-1753. [doi:10.1038/s41409-020-0836-2] [https://hdl.handle.net/10807/229758]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/229758
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