2018
DOI: 10.3324/haematol.2017.187047
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Abstract: Gene therapy for sickle cell disease is limited by the yield of hematopoietic progenitor cells that can be harvested for transduction or gene editing. We therefore performed a phase I dose-escalation study of the hematopoietic progenitor cell mobilizing agent plerixafor to evaluate the efficacy and safety of standard dosing on peripheral blood CD34+ cell mobilization. Of 15 patients enrolled to date, only one was chronically transfused and ten were on hydroxyurea. Of eight patients who achieved a CD34+ cell co… Show more

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Cited by 48 publications
(25 citation statements)
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“…The HGB‐206 study of LentiGlobin in SCD is now evaluating the hypothesis that a period of regular RBC transfusions prior to cell collection, combined with refinements to the gene therapy manufacturing process, may increase transduction of HSPCs and improve engraftment of transduced HSPCs. The study protocol has also been modified to incorporate a new, plerixafor‐only mobilization process that has recently shown acceptable safety in patients with SCD (Cavazzana et al , ; Tisdale et al , ; Boulad et al , ; Lagresle‐Peyrou et al , ), which will allow evaluation of mobilization and apheresis compared to direct BM harvest as a source of HSPCs for SCD gene therapy. Very early, high‐level results appear promising (Kanter et al , ).…”
Section: Discussionmentioning
confidence: 99%
“…The HGB‐206 study of LentiGlobin in SCD is now evaluating the hypothesis that a period of regular RBC transfusions prior to cell collection, combined with refinements to the gene therapy manufacturing process, may increase transduction of HSPCs and improve engraftment of transduced HSPCs. The study protocol has also been modified to incorporate a new, plerixafor‐only mobilization process that has recently shown acceptable safety in patients with SCD (Cavazzana et al , ; Tisdale et al , ; Boulad et al , ; Lagresle‐Peyrou et al , ), which will allow evaluation of mobilization and apheresis compared to direct BM harvest as a source of HSPCs for SCD gene therapy. Very early, high‐level results appear promising (Kanter et al , ).…”
Section: Discussionmentioning
confidence: 99%
“…Results have shown appropriate mobilization of CD34+ cells 6 h after a single dose of Plerixafor and are of higher quality and purity, decreasing the need for multiple bone marrow harvests and the associated stress/pain. Associated with hyper-transfusion therapy, it has become the preferred way of marrow stimulation to yield appropriate hematopoietic stem/progenitor cells in patients with SCD (Boulad et al, 2018;Hsieh and Tisdale, 2018;Lagresle-Peyrou et al, 2018).…”
Section: Allogeneic Bone Marrow Transplantmentioning
confidence: 99%
“…For patients with SCD, however, G-CSF is contraindicated, because it can precipitate fatal vaso-occlusive crises. 90 , 91 Therefore, (off-label) plerixafor has been used as the only mobilization agent 92 94 and results in stem cells that are well suited for transduction and expression of the therapeutic ß-globin. 89 …”
Section: Challenges In Gene Therapy For ß-Hemoglobinopathiesmentioning
confidence: 99%