دورية أكاديمية
Hematopoietic stem cell transplantation for B-thalassemia major with alemtuzumab.
| العنوان: | Hematopoietic stem cell transplantation for B-thalassemia major with alemtuzumab. |
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| المؤلفون: | Sánchez LM; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., George A; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Friend BD; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Bhar S; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Sasa G; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Doherty EE; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Craddock J; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Steffin D; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Salem B; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Yassine K; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Omer B; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Martinez C; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., Leung K; Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA., Krance RA; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA., John TD; Department of Pediatrics, Division of Hematology/Oncology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.; Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA. |
| المصدر: | Pediatric hematology and oncology [Pediatr Hematol Oncol] 2024 May; Vol. 41 (4), pp. 260-272. Date of Electronic Publication: 2023 Dec 22. |
| نوع المنشور: | Journal Article |
| اللغة: | English |
| بيانات الدورية: | Publisher: Informa Healthcare Country of Publication: England NLM ID: 8700164 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1521-0669 (Electronic) Linking ISSN: 08880018 NLM ISO Abbreviation: Pediatr Hematol Oncol Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: 2006- : London : Informa Healthcare Original Publication: Washington, DC : Hemisphere Pub. Corp., c1986- |
| مواضيع طبية MeSH: | beta-Thalassemia*/therapy , beta-Thalassemia*/complications , Hematopoietic Stem Cell Transplantation*/methods , Graft vs Host Disease*/etiology , Graft vs Host Disease*/prevention & control, Humans ; Infant ; Child, Preschool ; Child ; Adolescent ; Alemtuzumab/therapeutic use ; Transplantation, Homologous ; Transplantation Conditioning/methods ; Retrospective Studies |
| مستخلص: | While matched related donor (MRD) allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for transfusion-dependent beta-thalassemia (TDT), the use of alternative sources has increased, resulting in the exploration of novel transplant-conditioning regimens to reduce the contribution of graft-versus-host disease (GVHD) and graft failure (GF) to transplant-related morbidity and mortality. Alemtuzumab is a CD52 monoclonal antibody that has been successfully incorporated into myeloablative conditioning regimens for other hematologic conditions, yet there have been limited studies regarding the use of alemtuzumab in HSCT for TDT. The purpose of this study was to evaluate engraftment, incidence of GVHD, and transplant related morbidity and mortality in patients with TDT who received alemtuzumab in addition to standard busulfan-based conditioning. The primary endpoint was severe GVHD-free, event-free survival (GEFS). Our cohort included 24 patients with a median age of 6.8 years (range 1.5-14.9). Eleven patients received a 10/10 MRD HSCT, eleven 10/10 unrelated donor (UD), and two mismatched UD. All patients achieved primary engraftment. For all patients, 5-year GEFS was 77.4% and 5-year overall survival (OS) was 91%. The 5-year cumulative incidence of GF (attributed to poor graft function) without loss of donor chimerism was 13.8% (95% CI: 4.5, 35.3). We report low rates of significant acute GVHD grade II-IV (12.5%) and chronic GVHD (4.4%). Younger age and MRD were associated with significantly improved GEFS, OS and EFS. Our results show that the use of alemtuzumab promotes stable engraftment, may reduce rates of severe GVHD, and results in acceptable GEFS, OS, and EFS. |
| فهرسة مساهمة: | Keywords: Alemtuzumab; hematopoietic stem cell transplantation; transfusion-dependent beta-thalassemia |
| المشرفين على المادة: | 3A189DH42V (Alemtuzumab) |
| تواريخ الأحداث: | Date Created: 20231222 Date Completed: 20240418 Latest Revision: 20240418 |
| رمز التحديث: | 20240418 |
| DOI: | 10.1080/08880018.2023.2296933 |
| PMID: | 38131101 |
| قاعدة البيانات: | MEDLINE |
PDF full text from Publisher | تدمد: | 1521-0669 |
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| DOI: | 10.1080/08880018.2023.2296933 |