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)

Bruce Morland, Tomas Kepak, Sandro Dallorso, Julian Sevilla, Dermot Murphy, Roberto Luksch, Isaac Yaniv, Peter Bader, Jochen Rößler, Gianni Bisogno, Britta Maecker-Kolhoff, Peter Lang, C Michel Zwaan, David Sumerauer, Gergely Kriván, John Bernard, Qianying Liu, Eileen Doyle, Franco Locatelli

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Pages (from-to)1744-1753
Number of pages10
JournalBone Marrow Transplantation
Volume55
Issue number9
DOIs
Publication statusPublished - Sep 2020

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