A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation

• Published in: Blood 2012-11, Vol.120 (19), p.3875-3881
• Main Authors: Li, Chunfu, Wu, Xuedong, Feng, Xiaoqing, He, Yuelin, Liu, Huaying, Pei, Fuyu, Liao, Jianyu, He, Lan, Shi, Lei, Li, Na, Liu, Qiujun, Liu, Shiting, Chen, Geyu, Su, Qingxia, Ren, Yuqiong, Wang, Yanhua, Tan, Wanxia
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Anemias. Hemoglobinopathies; beta-Thalassemia - complications; beta-Thalassemia - mortality; beta-Thalassemia - therapy; Biological and medical sciences; Child; Child, Preschool; Diseases of red blood cells; Female; Graft Survival; Graft vs Host Disease - drug therapy; Graft vs Host Disease - etiology; Hematologic and hematopoietic diseases; Humans; Infant; Male; Medical sciences; Middle Aged; Peripheral Blood Stem Cell Transplantation - adverse effects; Transplantation Conditioning; Treatment Outcome; Unrelated Donors; Young Adult
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2012-03-417998

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.