Update of hematopoietic cell transplantation for sickle cell disease

Current Opinion in Hematology
Mark C Walters

Abstract

Hematopoietic cell transplantation (HCT) is a curative therapy for sickle cell disease (SCD) that is utilized very rarely because of limited allogeneic donor availability, limited healthcare resources needed to expand the treatment to regions in the world where most affected individuals reside, and by a view among SCD experts that HCT lacks the evidential rigor with short and long-term toxicity profiles that together might support its broader application. In this update, recent advances focused on donor selection, reduced toxicity preparation for HCT, and treatment of young adults will be presented. The current status of conventional bone marrow transplantation with a human leukocyte antigen-identical sibling donor is summarized. HCT for SCD is curative in almost all children who have a human leukocyte antigen-matched sibling donor. The future of this therapy will hinge on expanding the number of individuals who might be treated.

References

Sep 20, 1984·The New England Journal of Medicine·F L JohnsonF T Billings
Oct 19, 2001·Bone Marrow Transplantation·K van BesienE Kodish
Aug 22, 2003·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Robert IannoneMark C Walters
Apr 27, 2007·British Journal of Haematology·Julie A PanepintoUNKNOWN Non-Malignant Marrow Disorders Working Committee, Center for International Blood and Marrow Transplant Research
Oct 4, 2007·British Journal of Haematology·Catherine J WuLakshamanan Krishnamurti
Oct 23, 2008·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Lakshmanan KrishnamurtiCatherine J Wu
Oct 14, 2009·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Mark C WaltersUNKNOWN Multicenter Study of Bone Marrow Transplantation for Sickle Cell Disease
Dec 17, 2009·The New England Journal of Medicine·Matthew M HsiehJohn F Tisdale
Feb 1, 2011·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Annalisa RuggeriUNKNOWN New York Blood Center
Feb 22, 2012·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Naynesh R KamaniShalini Shenoy
Jun 5, 2012·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Michael RothRosanna Ricafort
Feb 19, 2013·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Mari H DallasWing Leung
May 23, 2013·Blood·Franco LocatelliUNKNOWN Eurocord and European Blood and Marrow Transplantation (EBMT) group
Jun 19, 2013·Archives de pédiatrie : organe officiel de la Sociéte française de pédiatrie·J BockenmeyerJ-H Dalle
Dec 12, 2013·Journal of Pediatric Hematology/oncology·Prasad Bodas, Seth Rotz
Jul 24, 2014·The New England Journal of Medicine·Loren GragertMartin Maiers
Jul 25, 2014·JAMA : the Journal of the American Medical Association·Matthew M HsiehJohn F Tisdale
Jul 30, 2014·Bone Marrow Transplantation·G LucarelliT T Wakama
Sep 10, 2014·JAMA : the Journal of the American Medical Association·Barbara P YawnJoylene John-Sowah

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Citations

Jan 23, 2016·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Matthew M BoeligWilliam H Peranteau
May 12, 2016·Transfusion·Lynne Neumayr, Elliott Vichinsky
Jun 4, 2016·British Journal of Haematology·Staci D ArnoldLakshmanan Krishnamurti
Feb 11, 2016·World Journal of Clinical Pediatrics·Lorenzo IughettiDonatella Venturelli
Jul 25, 2018·Pediatric Blood & Cancer·Lia Neu PhillipsThomas A Olson
Dec 6, 2018·Nature Reviews. Drug Discovery·Marilyn J TelenGregory M Vercellotti
Jun 14, 2017·Learning Health Systems·Million A TegengeTelba Irony
May 21, 2020·Journal of Pediatric Hematology/oncology·Iman ShaheenBasant Meligy
May 28, 2021·Gene Therapy·Pervinder Sagoo, H Bobby Gaspar
Jun 23, 2021·Biomedical Reports·Stephanie Jyet Quan Loo, Nyet Kui Wong
Jan 18, 2018·Chemical Reviews·Jooyeon ParkHyunjoon Kong
Mar 11, 2020·Molecular Therapy. Methods & Clinical Development·Christopher B ChambersAndrew Wilber
Nov 4, 2017·Cell Stem Cell·Richard A MorganDonald B Kohn

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