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  1. Article: PRIMARY STROKE PREVENTION IN CHILDREN WITH SICKLE CELL ANEMIA LIVING IN AFRICA: THE FALSE CHOICE BETWEEN PATIENT-ORIENTED RESEARCH AND HUMANITARIAN SERVICE-PART II.

    Debaun, Michael R

    Transactions of the American Clinical and Climatological Association

    2022  Volume 132, Page(s) 236–248

    Abstract: In the United States, primary stroke prevention in children with sickle cell anemia (SCA) is the standard of care and includes annual transcranial Doppler ultrasound to detect elevated velocities. For children with velocities ≥200 cm/sec, initial monthly ...

    Abstract In the United States, primary stroke prevention in children with sickle cell anemia (SCA) is the standard of care and includes annual transcranial Doppler ultrasound to detect elevated velocities. For children with velocities ≥200 cm/sec, initial monthly blood transfusion therapy for at least a year, followed by the option of hydroxyurea therapy, results in a significant decline in the stroke rate. In Africa, no prior strategy exists for primary stroke prevention because regular blood transfusion therapy is not feasible. We completed a randomized controlled trial for primary stroke prevention using initial low- and moderate-dose hydroxyurea as an alternative to blood transfusion therapy. The trial results demonstrated equal primary stroke prevention efficacy with both doses. After the trial, we provided a state-supported stroke screening service and treatment for over 20,000 children in Kano, Nigeria. We will review the strategies implemented to conduct clinical research and provide humanitarian assistance to children with SCA in Nigeria.
    MeSH term(s) Anemia, Sickle Cell/complications ; Anemia, Sickle Cell/therapy ; Antisickling Agents/therapeutic use ; Child ; Humans ; Hydroxyurea/therapeutic use ; Nigeria ; Stroke/diagnostic imaging ; Stroke/etiology ; Stroke/prevention & control
    Chemical Substances Antisickling Agents ; Hydroxyurea (X6Q56QN5QC)
    Language English
    Publishing date 2022-10-04
    Publishing country United States
    Document type Journal Article ; Randomized Controlled Trial
    ZDB-ID 603823-2
    ISSN 0065-7778
    ISSN 0065-7778
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Sickle cell disease strategies and priorities - Authors' reply.

    Piel, Frédéric B / DeBaun, Michael R / Nnodu, Obiageli

    The Lancet. Haematology

    2023  Volume 10, Issue 10, Page(s) e795

    MeSH term(s) Humans ; Anemia, Sickle Cell/therapy
    Language English
    Publishing date 2023-10-02
    Publishing country England
    Document type Letter ; Comment
    ISSN 2352-3026
    ISSN (online) 2352-3026
    DOI 10.1016/S2352-3026(23)00269-7
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  3. Article ; Online: Increasing access to quality healthcare for children who are incarcerated: American Pediatric Society issue of the year (2023-2024).

    Barnert, Elizabeth / DeBaun, Michael R

    Pediatric research

    2023  Volume 95, Issue 3, Page(s) 610–612

    MeSH term(s) Child ; Humans ; United States ; Quality of Health Care ; Delivery of Health Care ; Prisoners
    Language English
    Publishing date 2023-07-19
    Publishing country United States
    Document type Journal Article
    ZDB-ID 4411-8
    ISSN 1530-0447 ; 0031-3998
    ISSN (online) 1530-0447
    ISSN 0031-3998
    DOI 10.1038/s41390-023-02739-3
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  4. Article ; Online: Initiating adjunct low-dose hydroxyurea therapy for stroke prevention in children with SCA during the COVID-19 pandemic.

    DeBaun, Michael R

    Blood

    2020  Volume 135, Issue 22, Page(s) 1997–1999

    MeSH term(s) Anemia, Sickle Cell/complications ; Anemia, Sickle Cell/therapy ; Antisickling Agents/therapeutic use ; Blood Donors/supply & distribution ; Blood Safety ; Blood Transfusion ; COVID-19 ; Child ; Child, Preschool ; Coronavirus Infections/epidemiology ; Humans ; Hydroxyurea/therapeutic use ; Pandemics ; Pneumonia, Viral/epidemiology ; Risk Factors ; Secondary Prevention ; Stroke/etiology ; Stroke/prevention & control
    Chemical Substances Antisickling Agents ; Hydroxyurea (X6Q56QN5QC)
    Keywords covid19
    Language English
    Publishing date 2020-04-13
    Publishing country United States
    Document type Letter
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2020005992
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  5. Article ; Online: The range of haploidentical transplant protocols in sickle cell disease: all haplos are not created equally.

    Kassim, Adetola A / DeBaun, Michael R

    Hematology. American Society of Hematology. Education Program

    2023  Volume 2023, Issue 1, Page(s) 532–541

    Abstract: The ideal curative therapy for sickle cell disease (SCD) must be applicable across all ages and include individuals with strokes and preexisting heart, lung, and kidney disease. Myeloablative, matched sibling donor hematopoietic stem cell transplant (HCT) ...

    Abstract The ideal curative therapy for sickle cell disease (SCD) must be applicable across all ages and include individuals with strokes and preexisting heart, lung, and kidney disease. Myeloablative, matched sibling donor hematopoietic stem cell transplant (HCT) for children with SCD has shown excellent outcomes over the past 3 decades but has been restricted due to the limited availability of a human leukocyte antigen-matched sibling donor (10%-15%) and increased treatment-related death in adults with myeloablative conditioning. To overcome these 2 significant barriers to curative therapy in SCD, related haploidentical HCT has become an active area of research. The use of related haploidentical donors (first- and second-degree relatives) increases the donor pool to at least 90% of those eligible across the life span. Importantly, most adults, even with strokes or significant comorbidities, can tolerate the nonmyeloablative conditioning regimen without treatment-related death. Since 2013, at least 3 related haploidentical HCT strategies have emerged as potential curative therapies for SCD: (1) a nonmyeloablative, T-cell replete, bone marrow transplant with thiotepa and posttransplant cyclophosphamide with a goal of complete donor chimerism; (2) a nonmyeloablative, in vivo T-cell depletion, using peripheral blood stem cells (PBSCs) with a goal of stable mixed donor-recipient chimerism; and (3) a myeloablative, ex vivo T-cell depletion using PBSCs and advanced-technology graft manipulation, with a goal of complete donor chimerism. We review the similarities, differences, outcomes, and gaps in knowledge with these 3 haploidentical HCT approaches for SCD.
    MeSH term(s) Adult ; Child ; Humans ; Hematopoietic Stem Cell Transplantation/methods ; Bone Marrow Transplantation ; Cyclophosphamide ; Anemia, Sickle Cell/therapy ; Transplantation, Homologous/methods ; Transplantation Conditioning/methods ; Graft vs Host Disease ; Review Literature as Topic
    Chemical Substances Cyclophosphamide (8N3DW7272P)
    Language English
    Publishing date 2023-12-08
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2084287-9
    ISSN 1520-4383 ; 1520-4391
    ISSN (online) 1520-4383
    ISSN 1520-4391
    DOI 10.1182/hematology.2023000486
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  6. Article ; Online: Effect of allogeneic hematopoietic stem cell transplantation on sickle cell disease-related organ complications: A systematic review and meta-analysis.

    Dovern, Elisabeth / Aydin, Mesire / DeBaun, Michael R / Alizade, Komeil / Biemond, Bart J / Nur, Erfan

    American journal of hematology

    2024  

    Abstract: Sickle cell disease (SCD)-related organ complications are a major cause of morbidity and mortality in patients with SCD. We sought to assess whether hematopoietic stem cell transplantation (HSCT) stabilizes, attenuates, or exacerbates organ decline. We ... ...

    Abstract Sickle cell disease (SCD)-related organ complications are a major cause of morbidity and mortality in patients with SCD. We sought to assess whether hematopoietic stem cell transplantation (HSCT) stabilizes, attenuates, or exacerbates organ decline. We performed a systematic review and meta-analysis of trials investigating organ function before and after HSCT in patients with SCD. We searched MEDLINE/PubMed and EMBASE up to September 21, 2023. Continuous data were expressed as standardized mean difference (SMD) and pooled in a weighted inverse-variance random-effects model; binomial data were expressed as risk ratio (RR) using the Mantel-Haenszel random-effects meta-analyses. Of 823 screened studies, 34 were included in this review. Of these, 17 (774 patients, 23.6% adults, 86.3% HLA-identical sibling donor, 56.7% myeloablative conditioning regimen) were included in the meta-analyses. Pulmonary function remained stable. Mean tricuspid regurgitant jet velocity decreased but did not reach statistical significance. In children, estimated glomerular filtration rate decreased (SMD -0.80, p = .01), and the presence of proteinuria increased (RR 2.00, p = <.01), while splenic uptake and phagocytic function improved (RR 0.31, p = <.01; RR 0.23, p = <.01). Cerebral blood flow improved (SMD -1.39, p = <.01), and a low incidence of stroke after transplantation in high-risk patients was found. Retinopathy and avascular osteonecrosis were investigated in only one study, showing no significant changes. While HSCT can improve some SCD-related organ dysfunctions, transplantation-related toxicity may have an adverse effect on others. Future research should focus on identifying individuals with SCD who might benefit most from HSCT and which forms of organ damage are more likely to exacerbate post-transplantation.
    Language English
    Publishing date 2024-03-22
    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.27297
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  7. Article ; Online: Altered VWF:ADAMTS13 homeostasis is a target for therapeutic intervention in sickle cell disease.

    Chaturvedi, Shruti / DeBaun, Michael R

    Proceedings of the National Academy of Sciences of the United States of America

    2022  Volume 119, Issue 40, Page(s) e2213079119

    MeSH term(s) ADAMTS13 Protein ; Anemia, Sickle Cell/genetics ; Anemia, Sickle Cell/therapy ; Homeostasis ; Humans ; von Willebrand Factor
    Chemical Substances von Willebrand Factor ; ADAMTS13 Protein (EC 3.4.24.87) ; ADAMTS13 protein, human (EC 3.4.24.87)
    Language English
    Publishing date 2022-09-28
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 209104-5
    ISSN 1091-6490 ; 0027-8424
    ISSN (online) 1091-6490
    ISSN 0027-8424
    DOI 10.1073/pnas.2213079119
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  8. Article ; Online: Time-sensitive healthcare guidelines for youth with chronic diseases in custody: gaps in care.

    Dickens, Colin / Ramesh, Ahalya / Adanlawo, Temiloluwa / DeBaun, Michael R

    Pediatric research

    2023  

    Abstract: Case study: On May 9th, 2023, a U.S. Border Patrol detained a family of five near Brownsville, TX. During processing, one of the family members, an eight-year-old girl, ADRA, was noted to have sickle cell anemia and a heart disease condition. Five days ... ...

    Abstract Case study: On May 9th, 2023, a U.S. Border Patrol detained a family of five near Brownsville, TX. During processing, one of the family members, an eight-year-old girl, ADRA, was noted to have sickle cell anemia and a heart disease condition. Five days after they arrived at the Donna Facility, on May 14th, ADRA displayed symptoms, including abdominal pain and fever, and tested positive for Influenza A. She was administered medication and transferred to a designated isolation unit at the Harlingen Border Patrol Station. Despite her deteriorating condition and her mother's urgent requests for medical intervention, there were no documented consultations with an on-call physician or considerations for her transfer to a local hospital. On May 17th, ADRA's health critically declined, marked by multiple visits to the medical unit for vomiting and abdominal pain. An ambulance was dispatched only after ADRA experienced a seizure and became unresponsive, Fig. 1. Her subsequent death was deemed a "preventable tragedy" attributed to systemic failures in the Border Patrol's medical care and decision-making processes in a juvenile care monitor's report.
    Language English
    Publishing date 2023-12-09
    Publishing country United States
    Document type Journal Article
    ZDB-ID 4411-8
    ISSN 1530-0447 ; 0031-3998
    ISSN (online) 1530-0447
    ISSN 0031-3998
    DOI 10.1038/s41390-023-02947-x
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  10. Article ; Online: Sexual violence as a precipitator of chronic pain in young adults with sickle cell disease.

    Chopra, Maya / Byrd, Jeannie / Wuichet, Kristin / DeBaun, Michael R

    Blood advances

    2022  Volume 6, Issue 16, Page(s) 4831–4833

    MeSH term(s) Anemia, Sickle Cell/complications ; Chronic Pain/etiology ; Humans ; Sex Offenses ; Young Adult
    Language English
    Publishing date 2022-07-12
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2915908-8
    ISSN 2473-9537 ; 2473-9529
    ISSN (online) 2473-9537
    ISSN 2473-9529
    DOI 10.1182/bloodadvances.2022007600
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