دورية أكاديمية
أعرض في EDS

Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres.

التفاصيل البيبلوغرافية
العنوان: Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres.
المؤلفون: Alzahrani M; Division of Hematology & Hematopoietic Stem Cell Transplantation, Department of Oncology, King Abdulaziz Medical City, Riyadh, Saudi Arabia., Damlaj M; Division of Hematology & Hematopoietic Stem Cell Transplantation, Department of Oncology, King Abdulaziz Medical City, Riyadh, Saudi Arabia., Jeffries N; Office of Biostatistics Research, National Heart, Lung, and Blood Institute, Bethesda, MD, USA., Alahmari B; Division of Hematology & Hematopoietic Stem Cell Transplantation, Department of Oncology, King Abdulaziz Medical City, Riyadh, Saudi Arabia., Singh A; Division of Hematology/Oncology, University of Illinois Hospital and Health Science Systems, Chicago, IL, USA., Rondelli D; Division of Hematology/Oncology, University of Illinois Hospital and Health Science Systems, Chicago, IL, USA., Tisdale JF; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA., Saraf SL; Division of Hematology/Oncology, University of Illinois Hospital and Health Science Systems, Chicago, IL, USA., Hsieh MM; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA.
المصدر: British journal of haematology [Br J Haematol] 2021 Feb; Vol. 192 (4), pp. 761-768. Date of Electronic Publication: 2021 Feb 03.
نوع المنشور: Clinical Trial; Journal Article; Multicenter Study; Research Support, N.I.H., Extramural
اللغة: English
بيانات الدورية: Publisher: Wiley-Blackwell Country of Publication: England NLM ID: 0372544 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2141 (Electronic) Linking ISSN: 00071048 NLM ISO Abbreviation: Br J Haematol Subsets: MEDLINE
أسماء مطبوعة: Publication: Oxford : Wiley-Blackwell
Original Publication: Oxford : Blackwell Scientific Publications
مواضيع طبية MeSH: HLA Antigens*/immunology , Hematopoietic Stem Cell Transplantation*/adverse effects , Hematopoietic Stem Cell Transplantation*/methods, Anemia, Sickle Cell/*therapy, Adolescent ; Adult ; Alemtuzumab/therapeutic use ; Anemia, Sickle Cell/immunology ; Antineoplastic Agents, Immunological/therapeutic use ; Child ; Female ; Graft Rejection/etiology ; Graft Rejection/immunology ; Graft Survival ; Graft vs Host Disease/etiology ; Graft vs Host Disease/immunology ; Humans ; Immunosuppressive Agents/therapeutic use ; Male ; Middle Aged ; Sirolimus/therapeutic use ; Tissue Donors ; Transplantation, Homologous/adverse effects ; Transplantation, Homologous/methods ; Treatment Outcome ; Young Adult
مستخلص: Non-myeloablative haematopoietic progenitor cell transplantation (HPCT) from matched related donors (MRD) has been increasingly utilized in sickle cell disease (SCD). A total of 122 patients received 300 cGy of total body irradiation (TBI), alemtuzumab, unmanipulated filgrastim-mobilized peripheral blood HPC and sirolimus. The median follow-up was four years; median age at HPCT was 29 years. Median neutrophil and platelet engraftment occurred on day 22 and 19 respectively; 41 patients required no platelet transfusions. Overall and sickle-free survival at one and five years were 93% and 85% respectively. Age, sex, pre-HPCT sickle complications, ferritin and infused HPC numbers were similar between graft failure and engrafted patients. Mean donor myeloid chimaerism at one and five years post HPCT were 84% and 88%, and CD3 was 48% and 53% respectively. Two patients developed grade 1 and 2 skin graft-versus-host disease (GVHD) with no chronic GVHD. Median days of recipients taking immunosuppression were 489; 83% of engrafted patients have discontinued immunosuppression. Haemoglobin, haemolytic parameters and hepatic iron levels improved post HPCT. Pulmonary function testing, hepatic histology and neurovascular imaging remained stable, suggesting cessation of further sickle-related injury. Fourteen patients had children. In this largest group of adult SCD patients, this regimen was highly efficacious, well-tolerated despite compromised organ functions pre HPCT, and without clinically significant GVHD.
(© Published 2020. This article is a U.S Government work and is in the public domain in the USA.)
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معلومات مُعتمدة: Z99 HL999999 United States ImNIH Intramural NIH HHS; HL006007-11 United States HL NHLBI NIH HHS
فهرسة مساهمة: Keywords: alemtuzumab; allogeneic transplantation; matched related donor; sickle cell disease; sirolimus
المشرفين على المادة: 0 (Antineoplastic Agents, Immunological)
0 (HLA Antigens)
0 (Immunosuppressive Agents)
3A189DH42V (Alemtuzumab)
W36ZG6FT64 (Sirolimus)
تواريخ الأحداث: Date Created: 20210203 Date Completed: 20210615 Latest Revision: 20221005
رمز التحديث: 20240628
مُعرف محوري في PubMed: PMC8582053
DOI: 10.1111/bjh.17311
PMID: 33534948
قاعدة البيانات: MEDLINE
الوصف
تدمد:1365-2141
DOI:10.1111/bjh.17311