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)

MORLAND, B., Tomáš KEPÁK, S. DALLORSO, J. SEVILLA, D. MURPHY, R. LUKSCH, I. YANIV, P. BADER, J. ROSSLER, G. BISOGNO, B. MAECKER-KOLHOFF, P. T. LANG, C. M. ZWAAN, D. SUMERAUER, G. KRIVAN, J. BERNARD, Q. Y. LIU, E. DOYLE and F. LOCATELLI. 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. London: Nature Publishing Group, 2020, vol. 55, No 9, p. 1744-1753. ISSN 0268-3369. Available from: https://dx.doi.org/10.1038/s41409-020-0836-2.

Basic information

• Original name: 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. (826 United Kingdom of Great Britain and Northern Ireland, guarantor), Tomáš KEPÁK (203 Czech Republic, belonging to the institution), S. DALLORSO (380 Italy), J. SEVILLA (724 Spain), D. MURPHY (826 United Kingdom of Great Britain and Northern Ireland), R. LUKSCH (380 Italy), I. YANIV (376 Israel), P. BADER (276 Germany), J. ROSSLER (756 Switzerland), G. BISOGNO (380 Italy), B. MAECKER-KOLHOFF (276 Germany), P. T. LANG (276 Germany), C. M. ZWAAN (528 Netherlands), D. SUMERAUER (203 Czech Republic), G. KRIVAN (348 Hungary), J. BERNARD (840 United States of America), Q. Y. LIU (840 United States of America), E. DOYLE (840 United States of America) and F. LOCATELLI (380 Italy).
• Edition: Bone Marrow Transplantation, London, Nature Publishing Group, 2020, 0268-3369.

Other information

• Original language: English
• Type of outcome: Article in a journal
• Field of Study: 30205 Hematology
• Country of publisher: United Kingdom of Great Britain and Northern Ireland
• Confidentiality degree: is not subject to a state or trade secret
• WWW: URL
• Impact factor: Impact factor: 5.483
• RIV identification code: RIV/00216224:14110/20:00116161
• Organization unit: Faculty of Medicine
• Doi: http://dx.doi.org/10.1038/s41409-020-0836-2
• UT WoS: 000517862900001
• Keywords in English: COLONY-STIMULATING FACTOR; NON-HODGKINS-LYMPHOMA; CHEMOKINE RECEPTOR; PROGENITOR CELLS; CHILDREN; ANTAGONIST; EFFICACY; AMD3100
• Tags: 14110321, rivok
• Tags: International impact, Reviewed

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 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.