Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.

Haematologica
Chantal Lagresle-PeyrouMarina Cavazzana

Abstract

Sickle cell disease is characterized by chronic anemia and vaso-occlusive crises, which eventually lead to multi-organ damage and premature death. Hematopoietic stem cell transplantation is the only curative treatment but it is limited by toxicity and poor availability of HLA-compatible donors. A gene therapy approach based on the autologous transplantation of lentiviral-corrected hematopoietic stem and progenitor cells was shown to be efficacious in one patient. However, alterations of the bone marrow environment and properties of the red blood cells hamper the harvesting and immunoselection of patients' stem cells from bone marrow. The use of Filgrastim to mobilize large numbers of hematopoietic stem and progenitor cells into the circulation has been associated with severe adverse events in sickle cell patients. Thus, broader application of the gene therapy approach requires the development of alternative mobilization methods. We set up a phase I/II clinical trial whose primary objective was to assess the safety of a single injection of Plerixafor in sickle cell patients undergoing red blood cell exchange to decrease the hemoglobin S level to below 30%. The secondary objective was to measure the efficiency of mobilization and...Continue Reading

Citations

Sep 12, 2018·Human Gene Therapy·Marina Cavazzana, Fulvio Mavilio
Oct 26, 2018·Annual Review of Medicine·Stuart H Orkin, Daniel E Bauer
Jul 20, 2019·Human Molecular Genetics·Yasuhiro IkawaMarina Cavazzana
Sep 5, 2019·Current Opinion in Hematology·Yvette C Tanhehco, Monica Bhatia
Jan 7, 2020·Expert Opinion on Drug Metabolism & Toxicology·Miao MiaoGuangdi Li
Feb 9, 2020·British Journal of Haematology·Alexis LeonardAllistair Abraham
Mar 7, 2020·British Journal of Haematology·Amina Nardo-MarinoDavid Rees
Jun 5, 2020·Blood·Mark C Walters
Dec 19, 2019·Expert Opinion on Investigational Drugs·Alessandro MatteLucia De Franceschi
Nov 19, 2019·Frontiers in Pediatrics·Frank J T StaalMarina Cavazzana
Apr 1, 2020·The American Journal of Bioethics : AJOB·Susannah W Lee, Jessica C Ginsberg
Mar 27, 2019·Nature Medicine·Yuxuan WuDaniel E Bauer
Apr 1, 2020·The American Journal of Bioethics : AJOB·Akshay Sharma, Liza-Marie Johnson
Jun 9, 2020·Frontiers in Physiology·Gabriel Salinas Cisneros, Swee L Thein
Mar 13, 2019·Nature Reviews. Drug Discovery·Marina CavazzanaEmmanuelle Six
Dec 6, 2018·Nature Reviews. Drug Discovery·Marilyn J TelenGregory M Vercellotti
Dec 19, 2020·British Journal of Haematology·Thomas A Fox, Claire Booth
Jan 27, 2021·Clinics in Laboratory Medicine·Yan Zheng, Stella T Chou
Dec 25, 2019·Seminars in Cancer Biology·Sara K DanielVenu G Pillarisetty
Feb 6, 2021·Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis·David G Justus, John P Manis
Apr 16, 2021·The Journal of Clinical Investigation·Phillip A DoerflerMitchell J Weiss
Aug 25, 2018·Molecular Therapy. Methods & Clinical Development·Leslie WeberAnnarita Miccio
Jun 8, 2021·HemaSphere·Gabriel Salinas Cisneros, Swee Lay Thein
Jun 7, 2021·Human Genomics·Kariofyllis KaramperisGeorge P Patrinos
May 22, 2020·Immunology Letters·Júlia Teixeira Cottas de Azevedo, Kelen Cristina Ribeiro Malmegrim
Jul 8, 2021·Blood·Allistair A Abraham, John F Tisdale

❮ Previous
Next ❯

Methods Mentioned

BETA
RNA-Seq
flow cytometry
genetic modification
leukapheresis

Clinical Trials Mentioned

NCT02151526
NCT02212535

Software Mentioned

Treestar
Plerixafor
FlowJo

Related Concepts

Related Feeds

Allogenic & Autologous Therapies

Allogenic therapies are generated in large batches from unrelated donor tissues such as bone marrow. In contrast, autologous therapies are manufactures as a single lot from the patient being treated. Here is the latest research on allogenic and autologous therapies.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Anemia

Anemia develops when your blood lacks enough healthy red blood cells. Anemia of inflammation (AI, also called anemia of chronic disease) is a common, typically normocytic, normochromic anemia that is caused by an underlying inflammatory disease. Here is the latest research on anemia.