A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation
| Autor: | Shiting Liu, Xiao-qing Feng, Lei Shi, Geyu Chen, Na Li, Lan He, Fuyu Pei, Qingxia Su, Yuelin He, Huaying Liu, Qiujun Liu, Chunfu Li, Xuedong Wu, Wanxia Tan, Yanhua Wang, Yuqiong Ren, Jianyu Liao |
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| Rok vydání: | 2012 |
| Předmět: |
Adult
Male medicine.medical_specialty Transplantation Conditioning Adolescent medicine.medical_treatment Immunology Graft vs Host Disease Hematopoietic stem cell transplantation ThioTEPA Biochemistry Gastroenterology Young Adult Internal medicine medicine Humans Cumulative incidence Child Peripheral Blood Stem Cell Transplantation business.industry Graft Survival beta-Thalassemia Beta thalassemia Infant Cell Biology Hematology Middle Aged medicine.disease Fludarabine Surgery Transplantation surgical procedures operative Treatment Outcome Child Preschool Female business Unrelated Donors Busulfan medicine.drug |
| Zdroj: | Blood. 120(19) |
| ISSN: | 1528-0020 |
| Popis: | We used a novel NF-08-TM transplant protocol based on intravenous busulfan, cyclophosphamide, fludarabine, and thiotepa in 82 consecutive patients with β-thalassemia major (TM), including 52 with allogeneic peripheral blood stem cell transplantation (PBSCT) from unrelated donors (UDs) with well-matched human leukocyte antigens and 30 with hematopoietic stem cell transplantation (HSCT) from matched sibling donors (MSDs). The median age at transplantation was 6.0 years (range, 0.6-15.0 years), and the ratio of male-to-female patients was 56:26. The median follow-up time was 24 months (range, 12-39 months). The estimated 3-year overall survival and TM-free survival were 92.3% and 90.4% in the UD-PBSCT group and 90.0% and 83.3% in the MSD-HSCT group. The cumulative incidences of graft rejection and grades III-IV acute graft-versus-host disease were 1.9% and 9.6%, respectively, in the UD-PBSCT group and 6.9% and 3.6%, respectively, in the MSD-HSCT group. The cumulative incidence of transplant-related mortality was 7.7% in the UD-PBSCT group and 10.0% in the MSD-HSCT group. In conclusion, UD-PBSCTs using the well-tolerated NF-08-TM protocol show similar results to MSD-HSCTs and can be used to treat β-thalassemia patients in the absence of MSDs. |
| Databáze: | OpenAIRE |
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