دورية أكاديمية
أعرض في EDS

Global perspectives on cellular therapy for children with sickle cell disease.

التفاصيل البيبلوغرافية
العنوان: Global perspectives on cellular therapy for children with sickle cell disease.
المؤلفون: John TD; Center for Cell and Gene Therapy, Baylor College of Medicine.; Texas Children's Cancer and Hematology Center, Texas Children's Hospital.; Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA., Namazzi R; Department of Paediatrics and Child Health, Makerere University College of Health Sciences, Kampala, Uganda., Chirande L; School of Medicine, The Muhimbili University of Health and Allied Sciences, Dar es Salaam, Tanzania., Tubman VN; Center for Cell and Gene Therapy, Baylor College of Medicine.; Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
المصدر: Current opinion in hematology [Curr Opin Hematol] 2022 Nov 01; Vol. 29 (6), pp. 275-280. Date of Electronic Publication: 2022 Sep 21.
نوع المنشور: Journal Article; Review; Research Support, N.I.H., Extramural
اللغة: English
بيانات الدورية: Publisher: Lippincott Williams And Wilkins Country of Publication: United States NLM ID: 9430802 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1531-7048 (Electronic) Linking ISSN: 10656251 NLM ISO Abbreviation: Curr Opin Hematol Subsets: MEDLINE
أسماء مطبوعة: Publication: Philadelphia Pa : Lippincott Williams And Wilkins
Original Publication: Philadelphia, PA : Current Science, c1993-
مواضيع طبية MeSH: Anemia, Sickle Cell*/complications , Anemia, Sickle Cell*/therapy , Graft vs Host Disease* , Hematopoietic Stem Cell Transplantation*/adverse effects, Africa South of the Sahara/epidemiology ; Child ; Humans ; Quality of Life
مستخلص: Purpose of Review: Low-income and middle-income countries (LMICs), primarily in sub-Saharan Africa (SSA), predominantly experience the burden of sickle cell disease (SCD). High frequency of acute and chronic complications leads to increased utilization of healthcare, which burdens fragile health systems. Mortality for children with limited healthcare access remains alarmingly high. Cellular based therapies such as allogeneic hematopoietic stem cell transplant (HSCT) are increasingly used in resource-rich settings as curative therapy for SCD. Broad access to curative therapies for SCD in SSA would dramatically alter the global impact of the disease.
Recent Findings: Currently, application of cellular based therapies in LMICs is limited by cost, personnel, and availability of HSCT-specific technologies and supportive care. Despite the challenges, HSCT for SCD is moving forward in LMICs. Highly anticipated gene modification therapies have recently proven well tolerated and feasible in clinical trials in resource-rich countries, but access remains extremely limited.
Summary: Translation of curative cellular based therapies for SCD should be prioritized to LMICs where the disease burden and cost of noncurative treatments is high, and long-term quality of life is poor. Focus on thoughtful modifications of current and future therapies to meet the need in LMICs, especially in SSA, will be especially impactful.
(Copyright © 2022 Wolters Kluwer Health, Inc. All rights reserved.)
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معلومات مُعتمدة: K23 HL148548 United States HL NHLBI NIH HHS
تواريخ الأحداث: Date Created: 20221007 Date Completed: 20221011 Latest Revision: 20231102
رمز التحديث: 20240628
مُعرف محوري في PubMed: PMC10107365
DOI: 10.1097/MOH.0000000000000738
PMID: 36206076
قاعدة البيانات: MEDLINE
الوصف
تدمد:1531-7048
DOI:10.1097/MOH.0000000000000738