Salvage treatment with plerixafor in poor mobilizing allogeneic stem cell donors: results of a prospective phase II-trial


We conducted a prospective clinical trial to investigate the safety and efficacy of plerixafor (P) in allogeneic peripheral blood stem cells (PBSC) donors with poor mobilization response to standard-dose granulocyte colony-stimulating factor (G-CSF), defined by <2 × 106 CD34 + cells/kg recipient body-weight (CD34+/kg RBW) after 1st apheresis. A single dose of 240 µg/kg P was injected subcutaneously at 10 p.m. on the day of the 1st apheresis. Thirty-seven allogeneic PBSC donors underwent study treatment. The median CD34+ count in peripheral blood was 15/µl on Day 1 after G-CSF alone, versus 44/µl on Day 2 after G-CSF plus P (p < 0.001). The median yield of CD34+ cells was 1.1 × 108 on Day 1 and 2.8 × 108 on Day 2. In contrast to a median yield of only 1.31 × 106 CD CD34+/kg RBW on Day 1, triggering study inclusion, a median of 3.74 × 106 CD CD34+/kg RBW were collected with G-CSF plus P on Day 2. Of 37 donors, 21 reached the target cell count of >4.5 × 106 CD34+/kg RBW (57%, 95%CI 40–73%). No donor experienced a severe adverse event requiring treatment. In conclusion, P might be considered on a case-by-case basis for healthy allogeneic donors with very poor stem cell mobilization success after G-CSF.

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Fig. 1
Fig. 2: CD34+ cell counts in the peripheral blood and the leukapheresis products before and after mobilization with Plerixafor.
Fig. 3: Adverse event rates reported for mobilization with rhG-CSF only versus rhG-CSF plus Plerixafor.
Fig. 4: Day 30 self-assessment of the mobilization procedure by the donors.


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The authors most of all appreciate the altruistic generosity of the allogeneic PBSC donors. Furthermore they thank the nurses and physicians of the apheresis units at the Department of Transfusion Medicine and CCC Cellex Collection Center GmbH Cologne and Dresden for very skillful medical care of the donors. This work has been funded by research grants from the Deutsche Forschungsgemeinschaft (DFG) within the corresponding research project “SFB 655, B7” and by DKMS gemeinnützige GmbH, Tübingen. We are grateful to the patients and their transplant physicians who agreed to report on the clinical outcomes of the transplantations facilitated within this study: University of Chicago, Chicago (IL), USA, Michael Bishop Universitätsklinik Rostock, Rostock, Germany, Christian Junghanß St. Stephan and St. Ladislaus Hospital-Clinic, Budapest, Hungary, Laszlo Gopsca General Hospital of Thessaloniki “G. Papanikolaou”, Thessaloniki, Greece, Anastasios Manolis University of Arizona, Tucson (AZ), USA, Faiz Anwer University Medical Center Groningen (UMCG), Groningen, Netherlands, Laura Bungener Vrije University Medical Center, Amsterdam, Netherlands, Ellen Meier Erasmus-MC-Daniel Den Hoed Cancer Center, Rotterdam, Netherlands, Jan J. Cornelissen Universitätsklinikum Carl-Gustav-Carus Dresden, Dresden, Germany, Martin Bornhäuser Universitätsklinikum Erlangen, Erlangen, Germany, Wolf Roesler Universitätsklinikum Hamburg Eppendorf, Hamburg, Germany, Nicolaus Kröger Centre Hospitalier Universitaire de Nancy—Hôpitaux de Brabois, France, Marie Thérèse Rubio Karmanos Cancer Center, Detroit Medical Center, Michigan, USA, Voravit Ratanatharathorn L’Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza, Torino, Italy, Benedetto Bruno Radboud Universitair Medisch Centrum, Nijmegen, Netherlands, Nicolaas Schaap Maastricht Universitair Medisch Centrum, Maastricht, Netherlands, Michel van Gelder University of Kansas Hospital, Kansas City (KS), USA, Sunil Abhyankar Barnes Jewish Hospital, St. Louis (MO), USA, Donna J. Fugate Institute of Hematology and Blood Transfusion, Prague, Czech Republik, Irina Kolarikova Fundacion Favaloro, Buenos Aires, Argentina, Gustavo Piccinelli University of Alabama at Birmingham, Birmingham (AL), USA, Lawrence Lamb Universitätsklinikum Frankfurt (Main), Frankfurt/Main, Germany, Gesine Bug Universitätsklinikum Essen, Klinik für Knochenmarktransplantation, Germany, Hellmut Ottinger Universitätsklinikum Mainz, III. Medizinische Klinik, Germany, Eva Wagner.

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Correspondence to Johannes Schetelig.

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Sanofi-Aventis provided plerixafor at no charge and financial support for anxillary research. JS—consultancy honoraria from AstraZeneca, Janssen, Roche, Gilead, Abbvie, Sanofi, Molmed; lecture fees from AstraZeneca, Janssen, Roche, Gilead, Abbvie, Sanofi, Novartis; research support from Genzyme, Sanofi, GSK, Novartis, AKH—consultancy honoraria from Sanofi; lecture fees from Sanofi, Janssen, and Therakos; research support from Sanofi. The remaining authors declared no conflict of interest.

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Hölig, K., Schmidt, H., Hütter, G. et al. Salvage treatment with plerixafor in poor mobilizing allogeneic stem cell donors: results of a prospective phase II-trial. Bone Marrow Transplant (2020).

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