Vlachos A, Federman N, Reyes-Haley C, Abramson J, Lipton J M
Division of Pediatric Hematology/Oncology and Stem Cell Transplantation, Schneider Children's Hospital, Albert Einstein College of Medicine at the Long Island Jewish Medical Center, New Hyde Park, NY 11040, USA.
Bone Marrow Transplant. 2001 Feb;27(4):381-6. doi: 10.1038/sj.bmt.1702784.
The Diamond Blackfan Anemia (DBA) Registry of North America is a detailed database of patients with DBA from the United States and Canada. To date, 354 patients have been registered. From this database an analysis of the outcome of hematopoietic stem cell transplantation for DBA was undertaken. Of the 20 transplanted patients who met criteria for the diagnosis of DBA, eight underwent an allogeneic HLA-matched sibling hematopoietic stem cell transplant (SCT) and 12 an alternative donor SCT. The median age at transplant for all patients was 6 years 2 months; 3 years 10 months vs 9 years 1 month for HLA-matched sibling and alternative donor SCT, respectively. All of the HLA-matched sibling transplants were done using a non-irradiation-containing regimen, whereas the majority of alternative donor transplants were performed using total body irradiation. The survival for HLA-matched sibling vsalternative donor transplant was 87.5% +/- 11.7% vs 14.1% +/- 12.1% at greater than 5 years from SCT (P = 0.015). The use of HLA-matched sibling SCT should be considered for all patients with suitable donors. However, alternative donor SCT in DBA must be approached cautiously, the potential for severe aplastic anemia (SAA) or hematopoietic malignancy not withstanding.
北美钻石黑范贫血（DBA）登记处是一个来自美国和加拿大的DBA患者详细数据库。迄今为止，已登记354例患者。基于该数据库，对DBA患者造血干细胞移植的结果进行了分析。在20例符合DBA诊断标准的移植患者中，8例接受了 HLA 匹配的同胞异基因造血干细胞移植（SCT），12例接受了替代供体SCT。所有患者移植时的中位年龄为6岁2个月；HLA匹配的同胞和替代供体SCT的移植年龄分别为3岁10个月和9岁1个月。所有HLA匹配的同胞移植均采用不含照射的方案，而大多数替代供体移植采用全身照射。SCT后5年以上，HLA匹配的同胞移植与替代供体移植的生存率分别为87.5%±11.7%和14.1%±12.1%（P = 0.015）。对于所有有合适供体的患者，均应考虑使用HLA匹配的同胞SCT。然而，DBA患者的替代供体SCT必须谨慎进行，尽管存在发生严重再生障碍性贫血（SAA）或血液系统恶性肿瘤的可能性。
