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.

Birmingham Women's and Children's Hospital Birmingham UK

Department for Women's and Children's Health University of Padua Padua Italy

Division of Pediatric Hematology Oncology Department of Pediatrics Inselspital Bern University Hospital University of Bern Bern Switzerland

Erasmus MC Sophia Children's Hospital Rotterdam Netherlands

Faculty Hospital Motol Prague Czech Republic

Fondazione IRCCS Istituto Nazionale dei Tumori Milan Italy

Hannover Medical School Hannover Germany

Hospital Infantil Universitario Niño Jesus FIB HIUNJ CIBERER Madrid Spain

IRCCS Bambino Gesù Children's Hospital Rome Italy

IRCCS Giannina Gaslini Genova Italy

Mustang Bio Inc Worcester Massachusetts USA

Princess Máxima Center Utrecht Netherlands

Royal Hospital for Children Glasgow UK

Sackler Faculty of Medicine Tel Aviv University Tel Aviv Israel

Sanofi Genzyme Cambridge MA USA

Sanofi US Bridgewater NJ USA

Sapienza University of Rome Rome Italy

Schneider Children's Medical Center of Israel Tel Aviv University Petah Tikva Israel

United St Istvan and St Laszlo Hospital Budapest Hungary

Universitätsklinikum Frankfurt am Main Frankfurt Germany

University Children's Hospital Tübingen Germany

University Hospital Brno and ICRC St Anna University Hospital Masaryk University Brno Czech Republic

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