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, Bruce; Kepak, Tomas; Dallorso, Sandro; Sevilla, Julian; Murphy, Dermot; Luksch, Roberto; Yaniv, Isaac; Bader, Peter; Rössler, Jochen; Bisogno, Gianni; Maecker-Kolhoff, Britta; Lang, Peter; Zwaan, C Michel; Sumerauer, David; Kriván, Gergely; Bernard, John; Liu, Qianying; Doyle, Eileen; Locatelli, Franco (2020). 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, 55(9), pp. 1744-1753. Springer Nature 10.1038/s41409-020-0836-2

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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.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine
04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine > Paediatric Haematology/Oncology

UniBE Contributor:

Rössler, Jochen Karl

Subjects:

600 Technology > 610 Medicine & health

ISSN:

1476-5365

Publisher:

Springer Nature

Language:

English

Submitter:

Jochen Karl Rössler

Date Deposited:

20 May 2020 14:19

Last Modified:

12 Mar 2021 19:26

Publisher DOI:

10.1038/s41409-020-0836-2

PubMed ID:

32127657

BORIS DOI:

10.7892/boris.144043

URI:

https://boris.unibe.ch/id/eprint/144043

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