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Organ function indications and potential improvements following curative therapy for sickle cell disease
Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):277-282. doi: 10.1182/hematology.2022000372.ABSTRACTCurative therapies for sickle cell disease include allogeneic hematopoietic stem cell transplantation (HSCT) and gene-modified autologous stem cell transplantation. HSCT has been used for 30 years with success measured by engraftment, symptom control, graft-vs-host disease (GVHD) risk, organ toxicity, and immune reconstitution. While human leukocyte antigen-matched sibling donor (MSD) transplants have excellent outcomes, alternate donor transplants (unrelated/haploidentical) are just beginning to overcome GVHD and...
Source: Hematology ASH Education Program - December 9, 2022 Category: Hematology Authors: Monica L Hulbert Allison A King Shalini Shenoy Source Type: research

Paradoxical protection from atherosclerosis and thrombosis in a mouse model of sickle cell disease
Summary Sickle cell disease (SCD) is associated with vascular complications including premature stroke. The role of atherothrombosis in these vascular complications is unclear. To determine the effect of SCD on atherosclerosis and thrombosis, mice with SCD along with controls were generated by transplantation of bone marrow from mice carrying the homozygous sickle cell mutation (Hbbhβs/hβs) or wild‐type mice (Hbb+/+) into C57BL6/J or apolipoprotein E deficient (Apoe−/−) recipient mice. At the time of sacrifice, 23–28 weeks following bone marrow transplantation, anaemia, reticulocytosis, and splenomegaly were pre...
Source: British Journal of Haematology - April 17, 2013 Category: Hematology Authors: Hui Wang, Wei Luo, Jintao Wang, Chiao Guo, Stephanie L. Wolffe, Julia Wang, Eddy B. Sun, Kori N. Bradley, Andrew D. Campbell, Daniel T. Eitzman Tags: Research Paper Source Type: research

Long-Term Outcome and Evaluation of Organ Function in Pediatric Patients Undergoing Haploidentical and Matched Related Hematopoietic Cell Transplantation for Sickle Cell Disease
We report the long-term outcomes of 22 pediatric patients who underwent related donor HSCT for SCD at St. Jude Children's Research Hospital, either a myeloablative sibling MRD HSCT (n = 14) or reduced-intensity parental haploidentical donor HSCT (n = 8). The median patient age was 11.0 ± 3.9 years in the MRD graft recipients and 9.0 ± 5.0 years in the haploidentical donor graft recipients. The median follow-up was 9.0 ± 2.3 years, with an overall survival (OS) of 93% and a recurrence/graft failure rate of 0%, for the MRD cohort and 7.4 ± 2.4 years, with an OS of 75%, disease-free survival of 38%, and disease recurren...
Source: Biology of Blood and Marrow Transplantation - February 18, 2013 Category: Hematology Authors: Mari H. Dallas, Brandon Triplett, David R. Shook, Christine Hartford, Ashok Srinivasan, Joseph Laver, Russell Ware, Wing Leung Tags: Clinical Research Source Type: research

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