Hamidieh Amir Ali, Alimoghaddam Kamran, Jahani Mohammad, Mousavi Seyyed Asadollah, Iravani Masoud, Bahar Babak, Jalili Mahdi, Jalali Arash, Behfar Maryam, Ghavamzadeh Ardeshir
Hematology, Oncology, and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
Hematol Oncol Stem Cell Ther. 2011;4(3):109-15. doi: 10.5144/1658-3876.2011.109.
Hematopoietic cell transplantation (HCT) is the only therapeutic modality capable of correcting the hematologic manifestations of Fanconi anemia (FA). The development of well-tolerated immunosuppressive conditioning regimens for FA patients undergoing HCT has proven to be a challenging task for hematologists.
Retrospective, patients referred to the hematology, oncology and stem cell transplantation research center.
We analyzed the outcome of 53 FA patients who had undergone HCT between 1992 and 2010. The median age at transplantation was 9 years. Patients received transplants from an HLA-identical sibling (n=39) or matched relative (n=9) and one-antigen locus mismatched other relative/sibling (n=5). All of the patients underwent transplantation with fludarabine and non-fludarabine-based conditioning regimens. No radiation therapy was given.
The median follow-up period for survivors was 13.5 months (range, 3 months-13.5 years). The 3-year overall survival (OS) was 60.6%. The 3-year OS for patients who did or did not receive fludarabine-based preparative regimens for the allograft was 36.4%, and 70%, respectively. However, there were no statistically significant differences in OS rates between these two groups (P=.112). Graft failure occurred in 4 patients (7.5%). All of these 4 patients had received fludarabine-based conditioning regimens. The incidence of acute GVHD after fludarabine-based regimens was 45% versus 79% in non-fludarabine-based regimens (P=.03).
Despite the high incidence of acute GVHD (78.6%) in the non-fludarabine group, which resulted in the death of some patients, the OS rate was significantly better than in fludarabine recipients. Therefore, in spite of the fact that recent studies advocate the fludarabine-based conditioning regimens, we propose to conduct a multicenter, prospective study to evaluate the outcomes of regimens employed in FA patients.
造血细胞移植（HCT）是唯一能够纠正范可尼贫血（FA）血液学表现的治疗方式。事实证明，为接受HCT的FA患者制定耐受性良好的免疫抑制预处理方案，对血液学家而言是一项具有挑战性的任务。
回顾性研究，研究对象为转诊至血液学、肿瘤学和干细胞移植研究中心的患者。
我们分析了1992年至2010年间接受HCT的53例FA患者的治疗结果。移植时的中位年龄为9岁。患者接受了来自人类白细胞抗原（HLA）匹配同胞（n = 39）或匹配亲属（n = 9）以及一个抗原位点不匹配的其他亲属/同胞（n = 5）的移植。所有患者均接受了基于氟达拉滨和非氟达拉滨的预处理方案进行移植。未进行放射治疗。
幸存者的中位随访期为13.5个月（范围：3个月至13.5年）。3年总生存率（OS）为60.6%。接受或未接受基于氟达拉滨的同种异体移植预处理方案的患者，其3年OS分别为36.4%和70%。然而，两组之间的OS率无统计学显著差异（P = 0.112）。4例患者（7.5%）发生移植物失败。这4例患者均接受了基于氟达拉滨的预处理方案。基于氟达拉滨方案后的急性移植物抗宿主病（GVHD）发生率为45%，而基于非氟达拉滨方案的发生率为79%（P = 0.03）。
尽管非氟达拉滨组急性GVHD发生率较高（78.6%），导致部分患者死亡，但其OS率明显优于接受氟达拉滨治疗的患者。因此，尽管近期研究提倡基于氟达拉滨的预处理方案，但我们建议开展一项多中心前瞻性研究，以评估FA患者所采用方案的治疗结果。
