Department of Pediatrics, Columbia University, New York, NY, USA.
Department of Biostatistics, Columbia University, New York, NY, USA.
Leukemia. 2015 Feb;29(2):448-55. doi: 10.1038/leu.2014.194. Epub 2014 Jun 18.
The outcome of children, adolescents and young adults (CAYA) with poor-risk recurrent/refractory lymphoma is dismal (⩽30%). To overcome this poor prognosis, we designed an approach to maximize an allogeneic graft vs lymphoma effect in the setting of low disease burden. We conducted a multi-center prospective study of myeloablative conditioning (MAC) and autologous stem cell transplantation (AutoSCT), followed by a reduced intensity conditioning (RIC) and allogeneic hematopoietic cell transplantation (AlloHCT) in CAYA, with poor-risk refractory or recurrent lymphoma. Conditioning for MAC AutoSCT consisted of carmustine/etoposide/cyclophosphamide, RIC consisted of busulfan/fludarabine. Thirty patients, 16 Hodgkin lymphoma (HL) and 14 non-Hodgkin lymphoma (NHL), with a median age of 16 years and median follow-up of 5years, were enrolled. Twenty-three patients completed both MAC AutoSCT and RIC AlloHCT. Allogeneic donor sources included unrelated cord blood (n=9), unrelated donor (n=8) and matched siblings (n=6). The incidence of transplant-related mortality following RIC AlloHCT was only 12%. In patients with HL and NHL, 10 year EFS was 59.8% and 70% (P=0.613), respectively. In summary, this approach is safe, and long-term EFS with this approach is encouraging considering the poor-risk patient characteristics and the use of unrelated donors for RIC AlloHCT in the majority of cases.
儿童、青少年和年轻成人(CAYA)中患有高危复发性/难治性淋巴瘤的患者的预后较差(⩽30%)。为了克服这种不良预后,我们设计了一种方法,即在疾病负担较低的情况下最大限度地发挥同种异体移植物抗淋巴瘤效应。我们对患有高危难治性或复发性淋巴瘤的 CAYA 患者进行了一项多中心前瞻性研究,即进行清髓性预处理(MAC)和自体干细胞移植(AutoSCT),然后进行减轻强度预处理(RIC)和异基因造血细胞移植(AlloHCT)。MAC AutoSCT 的预处理方案包括卡莫司汀/依托泊苷/环磷酰胺,RIC 方案包括白消安/氟达拉滨。共纳入 30 例患者,其中 16 例霍奇金淋巴瘤(HL)和 14 例非霍奇金淋巴瘤(NHL),中位年龄为 16 岁,中位随访时间为 5 年。23 例患者完成了 MAC AutoSCT 和 RIC AlloHCT。异基因供体来源包括无关脐带血(n=9)、无关供体(n=8)和匹配的同胞(n=6)。RIC AlloHCT 后的移植相关死亡率仅为 12%。在 HL 和 NHL 患者中,10 年 EFS 分别为 59.8%和 70%(P=0.613)。综上所述,考虑到高危患者特征以及在大多数情况下使用无关供体进行 RIC AlloHCT,该方法安全,且长期 EFS 令人鼓舞。