{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,16]],"date-time":"2026-04-16T01:39:31Z","timestamp":1776303571516,"version":"3.50.1"},"reference-count":19,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2021,11,9]],"date-time":"2021-11-09T00:00:00Z","timestamp":1636416000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Transfusion"],"published-print":{"date-parts":[[2022,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Fanconi anemia (FA) is an inherited disorder characterized clinically by congenital abnormalities, progressive bone marrow failure (BMF), and a predisposition to malignancy. Gene therapy (GT) of FA, via the infusion of gene\u2010corrected peripheral blood (PB) autologous hematopoietic stem cells (HSCs), may constitute a cure for BMF. GT bypasses the donor restrictions and adverse events associated with allogenic HSC transplantation. However, adequate harvesting of PB\u2010HSCs is a crucial determinant of successful engraftment in gene therapy. Harvesting the low numbers of HSCs in patients with FA is particularly challenging.<\/jats:p><\/jats:sec><jats:sec><jats:title>Study design and methods<\/jats:title><jats:p>This open\u2010label phase I\/II trial evaluates the feasibility and safety of co\u2010administration of G\u2010CSF and plerixafor in patients with FA for the mobilization and harvesting of peripheral HSCs, intending to use them in a gene therapy trial. Patients with mutations in the <jats:italic>FANCA<\/jats:italic> gene received two subcutaneous injections of G\u2010CSF (6\u00a0\u03bcg\/kg \u00d7\u20092\/d from D1 to D8. Plerixafor (0.24\u2009mg\/kg\/d) was administered 2\u00a0h before apheresis (from D5 onward).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>CD34<jats:sup>+<\/jats:sup> cells were mobilized for four patients quickly but transiently after the plerixafor injection. One patient had a CD34<jats:sup>+<\/jats:sup> cell count of over 100\/\u03bcl; the mobilization peaked 2\u00a0h after the injection and lasted for more than 9\u00a0h. There were no short\u2010term adverse events associated with the mobilization or harvesting procedures.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Our data in patients with FA show that the mobilization of HSCs with G\u2010CSF and plerixafor is safe and more efficient in younger individuals without BMF.<\/jats:p><\/jats:sec>","DOI":"10.1111\/trf.16721","type":"journal-article","created":{"date-parts":[[2021,11,9]],"date-time":"2021-11-09T15:05:37Z","timestamp":1636470337000},"page":"165-172","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["A new step in understanding stem cell mobilization in patients with Fanconi anemia: A bridge to gene therapy"],"prefix":"10.1111","volume":"62","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8806-011X","authenticated-orcid":false,"given":"Jean\u2010s\u00e9bastien","family":"Diana","sequence":"first","affiliation":[{"name":"Biotherapy Department H\u00f4pital Necker Enfants Malades, Asistance Publique\u2010H\u00f4pitaux de Paris, Universite de Paris  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sandra","family":"Manceau","sequence":"additional","affiliation":[{"name":"Biotherapy Department H\u00f4pital Necker Enfants Malades, Asistance Publique\u2010H\u00f4pitaux de Paris, Universite de Paris  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Thierry","family":"Leblanc","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Unit H\u00f4pital Robert Debr\u00e9, Asistance Publique\u2010H\u00f4pitaux de Paris, Universite de Paris  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alessandra","family":"Magnani","sequence":"additional","affiliation":[{"name":"Biotherapy Department H\u00f4pital Necker Enfants Malades, Asistance Publique\u2010H\u00f4pitaux de Paris, Universite de Paris  Paris 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