氟达拉滨、坎帕斯和低剂量环磷酰胺（FCC）联合或不联合 TBI 预处理在儿童重型再生障碍性贫血中可获得极好的移植结果。
Fludarabine, Campath, and Low-Dose Cyclophosphamide (FCC) with or without TBI Conditioning Results in Excellent Transplant Outcomes in Children with Severe Aplastic Anemia.
机构信息
Section of Oncology and BMT, Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.
Section of Oncology and BMT, Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.
出版信息
Biol Blood Marrow Transplant. 2020 Oct;26(10):1900-1905. doi: 10.1016/j.bbmt.2020.06.027. Epub 2020 Jul 5.
Various reduced-intensity conditioning regimens are in use for allogeneic hematopoietic cell transplant (HSCT) in patients with idiopathic severe aplastic anemia (SAA). We describe the use of fludarabine, Campath, and low-dose cyclophosphamide (FCC) conditioning in 15 children undergoing related or unrelated donor transplants. Total body irradiation (TBI) of 2 Gy was added for unrelated donor HSCT. At a median follow-up of 2.3 years, the failure-free survival was 100%, with low rates of infection and toxicity. There was no occurrence of grade III to IV acute graft-versus-host disease (GVHD). All patients had full donor myeloid chimerism post-HSCT, even with mixed chimerism in the T cell lineage. The absence of chronic GVHD and long-term stable mixed donor T cell chimerism confirms immune tolerance following FCC (± TBI) conditioned transplantation in children with SAA.
各种减低强度的预处理方案被用于治疗特发性重型再生障碍性贫血（SAA）患者的异基因造血细胞移植（HSCT）。我们描述了在 15 例接受亲缘或非亲缘供者移植的患儿中使用氟达拉滨、坎帕斯和低剂量环磷酰胺（FCC）预处理的情况。非亲缘供者 HSCT 中添加 2Gy 的全身照射（TBI）。中位随访 2.3 年后，无失败生存率为 100%，感染和毒性发生率低。未发生 III 级至 IV 级急性移植物抗宿主病（GVHD）。所有患者在 HSCT 后均有完全供者髓系嵌合体，即使 T 细胞系有混合嵌合体。FCC（±TBI）预处理移植后无慢性 GVHD 和长期稳定的混合供者 T 细胞嵌合体，证实了 SAA 患儿接受 FCC（±TBI）预处理移植后存在免疫耐受。
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