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  1. Article ; Online: Allogeneic haematopoietic stem-cell transplantation versus gene therapy for haemoglobinopathies.

    Olson, Timothy S / Walters, Mark C

    The Lancet. Haematology

    2023  Volume 10, Issue 10, Page(s) e798–e800

    MeSH term(s) Humans ; Hemoglobinopathies/genetics ; Hemoglobinopathies/therapy ; Genetic Therapy ; Hematopoietic Stem Cell Transplantation
    Language English
    Publishing date 2023-10-02
    Publishing country England
    Document type Journal Article
    ISSN 2352-3026
    ISSN (online) 2352-3026
    DOI 10.1016/S2352-3026(23)00246-6
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  2. Article ; Online: Induction of Fetal Hemoglobin by Gene Therapy.

    Walters, Mark C

    The New England journal of medicine

    2021  Volume 384, Issue 3, Page(s) 284–285

    MeSH term(s) Anemia, Sickle Cell/genetics ; Anemia, Sickle Cell/therapy ; CRISPR-Cas Systems ; Fetal Hemoglobin/genetics ; Gene Editing ; Genetic Therapy ; Humans ; beta-Thalassemia/genetics
    Chemical Substances Fetal Hemoglobin (9034-63-3)
    Language English
    Publishing date 2021-01-20
    Publishing country United States
    Document type Editorial ; Comment
    ZDB-ID 207154-x
    ISSN 1533-4406 ; 0028-4793
    ISSN (online) 1533-4406
    ISSN 0028-4793
    DOI 10.1056/NEJMe2034338
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  3. Article ; Online: Sickle marrow: double, double toil and trouble.

    Walters, Mark C

    Blood

    2020  Volume 135, Issue 23, Page(s) 2017–2018

    MeSH term(s) Anemia, Sickle Cell ; Animals ; Blood Transfusion ; Bone Marrow ; Mice ; Neovascularization, Pathologic
    Language English
    Publishing date 2020-06-03
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2020005114
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  4. Article ; Online: Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.

    Lawal, R AdeBisi / Walters, Mark C / Fitzhugh, Courtney D

    Hematology/oncology clinics of North America

    2022  Volume 36, Issue 6, Page(s) 1313–1335

    Abstract: Curative therapies for sickle cell disease (SCD) include allogeneic human leukocyte antigen (HLA)- matched sibling and haploidentical hematopoietic cell transplant (HCT), gene therapy, and gene editing. However, comparative trial data that might ... ...

    Abstract Curative therapies for sickle cell disease (SCD) include allogeneic human leukocyte antigen (HLA)- matched sibling and haploidentical hematopoietic cell transplant (HCT), gene therapy, and gene editing. However, comparative trial data that might facilitate selecting one curative therapy over another are unavailable. New strategies to decrease graft rejection and graft-versus-host disease (GVHD) risks are needed to expand haploidentical HCT. Myeloablative gene therapy and gene editing also has limitations. Herein, we review recent studies on curative therapies for SCD in the past 5 years.
    MeSH term(s) Humans ; Hematopoietic Stem Cell Transplantation ; Transplantation Conditioning ; Graft vs Host Disease/etiology ; Graft vs Host Disease/prevention & control ; Anemia, Sickle Cell/genetics ; Anemia, Sickle Cell/therapy ; Genetic Therapy ; Allografts
    Language English
    Publishing date 2022-11-11
    Publishing country United States
    Document type Journal Article ; Review ; Research Support, N.I.H., Extramural ; Research Support, N.I.H., Intramural
    ZDB-ID 93115-9
    ISSN 1558-1977 ; 0889-8588
    ISSN (online) 1558-1977
    ISSN 0889-8588
    DOI 10.1016/j.hoc.2022.06.007
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  5. Article ; Online: In Reply: Subaxial Cervical Spine Injury Classification Systems.

    Walters, Beverly C / Hadley, Mark N

    Neurosurgery

    2019  Volume 85, Issue 2, Page(s) E404

    MeSH term(s) Humans ; Neck Injuries ; Spinal Injuries
    Language English
    Publishing date 2019-05-22
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 135446-2
    ISSN 1524-4040 ; 0148-396X
    ISSN (online) 1524-4040
    ISSN 0148-396X
    DOI 10.1093/neuros/nyz152
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    Zs.MO 539: Show issues

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  6. Article ; Online: Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing.

    Locatelli, Franco / Corbacioglu, Selim / Hobbs, William / Frangoul, Haydar / Walters, Mark C

    American journal of hematology

    2023  Volume 99, Issue 3, Page(s) 430–438

    Abstract: A growing number of gene therapy- and gene editing-based treatments for patients with sickle cell disease (SCD) are entering clinical trials. These treatments, designed to target the underlying cause of SCD, have the potential to provide functional cures, ...

    Abstract A growing number of gene therapy- and gene editing-based treatments for patients with sickle cell disease (SCD) are entering clinical trials. These treatments, designed to target the underlying cause of SCD, have the potential to provide functional cures, which until now were possible only through allogeneic hematopoietic stem cell transplant. However, as these novel approaches advance from early- to late-stage clinical trials, it is essential to identify physiologically and clinically relevant endpoints that can demonstrate the achievement of a functional cure for SCD. Here, we present an overview of the pathophysiology of SCD and current treatment options, review ongoing SCD clinical trials using gene therapy or gene editing approaches, and identify the most relevant endpoints for demonstrating the attainment of a functional cure for SCD.
    MeSH term(s) Humans ; Gene Editing ; Anemia, Sickle Cell/genetics ; Anemia, Sickle Cell/therapy ; Hematopoietic Stem Cell Transplantation ; Genetic Therapy
    Language English
    Publishing date 2023-11-27
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 196767-8
    ISSN 1096-8652 ; 0361-8609
    ISSN (online) 1096-8652
    ISSN 0361-8609
    DOI 10.1002/ajh.27164
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    Zs.A 1251: Show issues Location:
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  7. Article ; Online: Update of hematopoietic cell transplantation for sickle cell disease.

    Walters, Mark C

    Current opinion in hematology

    2015  Volume 22, Issue 3, Page(s) 227–233

    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 ...

    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.
    MeSH term(s) Anemia, Sickle Cell/therapy ; Hematopoietic Stem Cell Transplantation/methods ; Humans
    Language English
    Publishing date 2015-05
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 1153887-9
    ISSN 1531-7048 ; 1065-6251
    ISSN (online) 1531-7048
    ISSN 1065-6251
    DOI 10.1097/MOH.0000000000000136
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    Zs.A 3703: Show issues Location:
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  8. Article ; Online: The case for the future role of evidence-based medicine in the management of cervical spine injuries, with or without fractures.

    Hadley, Mark N / Walters, Beverly C

    Journal of neurosurgery. Spine

    2019  Volume 31, Issue 4, Page(s) 457–463

    Abstract: The authors believe that the standardized and systematic study of immobilization techniques, diagnostic modalities, medical and surgical treatment strategies, and ultimately outcomes and outcome measurement after cervical spinal trauma and cervical ... ...

    Abstract The authors believe that the standardized and systematic study of immobilization techniques, diagnostic modalities, medical and surgical treatment strategies, and ultimately outcomes and outcome measurement after cervical spinal trauma and cervical spinal fracture injuries, if performed using well-designed medical evidence-based comparative investigations with meaningful follow-up, has both merit and the remarkable potential to identify optimal strategies for assessment, characterization, and clinical management. However, they recognize that there is inherent difficulty in attempting to apply evidence-based medicine (EBM) to identify ideal treatment strategies for individual cervical fracture injuries. First, there is almost no medical evidence reported in the literature for the management of specific isolated cervical fracture subtypes; specific treatment strategies for specific fracture injuries have not been routinely studied in a rigorous, comparative way. One of the vulnerabilities of an evidenced-based scientific review in spinal cord injury (SCI) is the lack of studies in comparative populations and scientific evidence on a given topic or fracture pattern providing level II evidence or higher. Second, many modest fracture injuries are not associated with vascular or neural injury or spinal instability. The application of the science of EBM to the care of patients with traumatic cervical spine injuries and SCIs is invaluable and necessary. The dedicated multispecialty author groups involved in the production and publication of the two iterations of evidence-based guidelines on the management of acute cervical spine and spinal cord injuries have provided strategic guidance in the care of patients with SCIs. This dedicated service to the specialty has been carried out to provide neurosurgical colleagues with a qualitative review of the evidence supporting various aspects of care of these patients. It is important to state and essential to understand that the science of EBM and its rigorous application is important to medicine and to the specialty of neurosurgery. It should be embraced and used to drive and shape investigations of the management and treatment strategies offered patients. It should not be abandoned because it is not convenient or it does not support popular practice bias or patterns. It is the authors' view that the science of EBM is essential and necessary and, furthermore, that it has great potential as clinician scientists treat and study the many variations and complexities of patients who sustain acute cervical spine fracture injuries.
    MeSH term(s) Cervical Vertebrae/injuries ; Disease Management ; Evidence-Based Medicine ; Humans ; Spinal Injuries/therapy
    Language English
    Publishing date 2019-09-20
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 2158643-3
    ISSN 1547-5646 ; 1547-5654
    ISSN (online) 1547-5646
    ISSN 1547-5654
    DOI 10.3171/2019.6.SPINE19652
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    Uk I Zs.135 -Spine-: Show issues Location:
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  9. Article: The case for HLA-identical sibling hematopoietic stem cell transplantation in children with symptomatic sickle cell anemia.

    Fitzhugh, Courtney D / Walters, Mark C

    Blood advances

    2017  Volume 1, Issue 26, Page(s) 2563–2567

    Abstract: This article has a companion Counterpoint by DeBaun and Clayton. ...

    Abstract This article has a companion Counterpoint by DeBaun and Clayton.
    Language English
    Publishing date 2017-12-12
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2876449-3
    ISSN 2473-9537 ; 2473-9529
    ISSN (online) 2473-9537
    ISSN 2473-9529
    DOI 10.1182/bloodadvances.2017007708
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  10. Article ; Online: Enrollment Lessons from a Biological Assignment Study of Marrow Transplantation versus Standard Care for Adolescents and Young Adults with Sickle Cell Disease: Considerations for Future Gene and Cellular Therapy Trials.

    Krishnamurti, Lakshmanan / Neuberg, Donna / Sullivan, Keith M / Smith, Shannon / Eapen, Mary / Walters, Mark C

    Transplantation and cellular therapy

    2022  Volume 29, Issue 4, Page(s) 217–221

    Abstract: We previously conducted a single-arm feasibility study (STRIDE1) of myeloablative bone marrow transplantation (BMT) in adolescents and young adults with sickle cell disease (SCD). The trial identified donors before entry, enrolled well, and found no ... ...

    Abstract We previously conducted a single-arm feasibility study (STRIDE1) of myeloablative bone marrow transplantation (BMT) in adolescents and young adults with sickle cell disease (SCD). The trial identified donors before entry, enrolled well, and found no unexpected regimen-related toxicity. Although many single-arm studies have been published, there are no controlled trials of either BMT or gene therapy in SCD. Therefore, we designed a comparative trial by biological assignment (available donor versus no donor). This multicenter National Institutes of Health-funded study (Blood and Marrow Transplant Clinical Trials Network 1503; STRIDE2) enrolled patients between 2016 and 2021 at 35 sites. Lagging recruitment led to study closure, and here we report the impediments to accrual. The BMT regimen and entry criteria were from STRIDE1, and 2-year survival was the primary endpoint. To minimize selection bias from prior HLA typing, STRIDE2 excluded individuals with previously identified donors. Accrual was stopped at 69% of target (138 enrolled; assigned 28 with donor, 96 with no donor). Barriers to enrollment included lower than expected frequency of HLA-matched related and unrelated donors; loss of enrollees owing to previously identified donors; conventional care arm dissuading some seeking BMT; challenging short-term endpoints in SCD, including incomplete documentation of sickle pain episodes; state Medicaid (primary insurers of SCD) denial of BMT coverage for adult SCD despite the study having secured Coverage with Evidence Development from the Center for Medicare & Medicaid Services; slowed accrual in 2019 to 2021 during the Coronavirus disease 2019 pandemic; and restriction of BMT resourcing for nonmalignant diseases by academic medical (cancer) centers. Social obstacles and access to BMT centers also limited entry, as did practitioner and participant concerns over suitability, cost, and toxicity. Planning for future controlled trials of curative therapy in SCD and other nonmalignant diseases likely will meet these enrollment challenges. Lessons from this trial may aid the development of future comparative studies.
    MeSH term(s) Aged ; United States/epidemiology ; Humans ; Adolescent ; Young Adult ; Bone Marrow Transplantation ; Bone Marrow ; COVID-19 ; Medicare ; Anemia, Sickle Cell/therapy ; Unrelated Donors
    Language English
    Publishing date 2022-10-18
    Publishing country United States
    Document type Multicenter Study ; Journal Article ; Research Support, N.I.H., Extramural
    ZDB-ID 3062231-1
    ISSN 2666-6367
    ISSN (online) 2666-6367
    DOI 10.1016/j.jtct.2022.10.008
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