دورية أكاديمية
Matched related hematopoietic cell transplant for sickle cell disease with alemtuzumab: the Texas Children's Hospital experience.
| العنوان: | Matched related hematopoietic cell transplant for sickle cell disease with alemtuzumab: the Texas Children's Hospital experience. |
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| المؤلفون: | John TD; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA. tami.john@bcm.edu., Friend B; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Yassine K; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Sasa G; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Bhar S; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Salem B; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Omer B; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Craddock J; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Doherty E; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Martinez C; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Heslop HE; Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital, Houston, TX, USA., Krance RA; Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA., Leung K; Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA, USA. |
| المصدر: | Bone marrow transplantation [Bone Marrow Transplant] 2021 Nov; Vol. 56 (11), pp. 2797-2803. Date of Electronic Publication: 2021 Jul 17. |
| نوع المنشور: | Journal Article |
| اللغة: | English |
| بيانات الدورية: | Publisher: Nature Publishing Group Country of Publication: England NLM ID: 8702459 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5365 (Electronic) Linking ISSN: 02683369 NLM ISO Abbreviation: Bone Marrow Transplant Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: <2003->: London : Nature Publishing Group Original Publication: Basingstoke, Hampshire : Scientific & Medical Division, Macmillan Press, c1986- |
| مواضيع طبية MeSH: | Anemia, Sickle Cell*/therapy , Graft vs Host Disease*/drug therapy , Hematopoietic Stem Cell Transplantation*, Aged ; Alemtuzumab/therapeutic use ; Hospitals ; Humans ; Retrospective Studies ; Texas ; Transplantation Conditioning ; Unrelated Donors |
| مستخلص: | Serotherapeutic agents facilitate engraftment and prevent graft-versus-host disease (GVHD) following hematopoietic stem cell transplant. Anti-thymocyte globulin is generally added to conditioning chemotherapy for matched related donor transplant (MRD-HCT) for sickle cell disease (SCD). Alemtuzumab, however, is appealing due to its broad lymphocyte killing that may achieve very low rejection and GVHD rates. To assess the impact of alemtuzumab in MRD-HCT for SCD, we retrospectively reviewed transplant-related outcomes and markers of immunity in 38 consecutive patients at Texas Children's Hospital having received myeloablative conditioning with alemtuzumab. Median follow-up was 4.8 years (range: 0.2-17). All patients engrafted. Donor chimerism was mixed in 47.1% of patients at ≥2-years. Donor chimerism <50% was uncommon (n = 2). One patient with low myeloid chimerism (19%) had sickle-related hemolysis at 10-years. Incidence of acute GVHD grade II-IV (5.3%) and extensive chronic GVHD (2.8%) was very low. Five-year event-free survival (EFS) and composite chronic GVHD-EFS were excellent at 94.7% (95% CI: 80.3, 98.6) and 89.2% (95% CI: 73.7, 95.8), respectively. Infections did not contribute to mortality although cytomegalovirus reactivation occurred commonly in the first 3 months after transplant. Our data suggest potential for alemtuzumab in myeloablative transplant for children with SCD although further evaluation in older patients and with unrelated donors is warranted. (© 2021. The Author(s), under exclusive licence to Springer Nature Limited.) |
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| المشرفين على المادة: | 3A189DH42V (Alemtuzumab) |
| تواريخ الأحداث: | Date Created: 20210718 Date Completed: 20220310 Latest Revision: 20220721 |
| رمز التحديث: | 20231215 |
| DOI: | 10.1038/s41409-021-01415-6 |
| PMID: | 34274957 |
| قاعدة البيانات: | MEDLINE |
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Full Text Finder | تدمد: | 1476-5365 |
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| DOI: | 10.1038/s41409-021-01415-6 |