Mark C Walters1. 1. Jordan Family Director, Blood and Marrow Transplant Program, Division of Hematology/Oncology/BMT, UCSF Benioff Children's Hospital, Oakland, California, USA.
Abstract
PURPOSE OF REVIEW: 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. RECENT FINDINGS: 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. SUMMARY: 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.
PURPOSE OF REVIEW: 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. RECENT FINDINGS: 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. SUMMARY: 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.
Authors: M Mynarek; C Bettoni da Cunha Riehm; F Brinkmann; K Weißenborn; M Tell-Lüersen; H-G Heuft; B Maecker-Kolhoff; K-W Sykora Journal: Klin Padiatr Date: 2013-04-26 Impact factor: 1.349
Authors: M Bhatia; Z Jin; C Baker; M B Geyer; K Radhakrishnan; E Morris; P Satwani; D George; J Garvin; G Del Toro; W Zuckerman; M T Lee; M Licursi; R Hawks; E Smilow; L A Baxter-Lowe; J Schwartz; M S Cairo Journal: Bone Marrow Transplant Date: 2014-05-05 Impact factor: 5.483
Authors: Robert Iannone; James F Casella; Ephraim J Fuchs; Allen R Chen; Richard J Jones; Ann Woolfrey; Michael Amylon; Keith M Sullivan; Rainer F Storb; Mark C Walters Journal: Biol Blood Marrow Transplant Date: 2003-08 Impact factor: 5.742
Authors: Matthew M Hsieh; Courtney D Fitzhugh; R Patrick Weitzel; Mary E Link; Wynona A Coles; Xiongce Zhao; Griffin P Rodgers; Jonathan D Powell; John F Tisdale Journal: JAMA Date: 2014-07-02 Impact factor: 56.272
Authors: C Brachet; N Azzi; A Demulder; C Devalck; A Gourdin; B Gulbis; A Klein; P Q Le; M Loop; E Sariban; A Ferster Journal: Bone Marrow Transplant Date: 2004-04 Impact factor: 5.483
Authors: M C Walters; K M Sullivan; F Bernaudin; G Souillet; J P Vannier; F L Johnson; C Lenarsky; D Powars; N Bunin; K Ohene-Frempong Journal: Blood Date: 1995-02-15 Impact factor: 22.113
Authors: Holly K Miller; Thomas M Braun; Terri Stillwell; Andrew C Harris; Sung Choi; James Connelly; Daniel Couriel; Steven Goldstein; Carrie L Kitko; John Magenau; Attaphol Pawarode; Pavan Reddy; Mary Riwes; Gregory A Yanik; John E Levine Journal: Biol Blood Marrow Transplant Date: 2016-12-22 Impact factor: 5.742
Authors: Elena Vuelta; José L Ordoñez; David J Sanz; Sandra Ballesteros; Jesús M Hernández-Rivas; Lucía Méndez-Sánchez; Manuel Sánchez-Martín; Ignacio García-Tuñón Journal: Int J Mol Sci Date: 2022-06-07 Impact factor: 6.208