Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol.
Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol.
复制标题
DOI：
10.1136/bmjopen-2020-040467
复制
发表时间：
2020-12-04
期刊：
影响因子：
2.9
通讯作者：
Takahashi S
中科院分区：
文献类型：
--
作者：
Terakura S;Konuma T;Tanaka M;Ozawa Y;Onizuka M;Nanno S;Onishi Y;Aotsuka N;Kondo T;Kawakita T;Kato J;Kobayashi T;Nishida T;Yamaguchi T;Kuwatsuka Y;Takahashi S
与其他替代供体的移植相比，观察到脐带血移植（CBT）后的长期生活质量更好，而CBT后的移植失败和复发仍然是主要问题。为了最大限度地减少CBT后移植失败和复发，在一些回顾性研究中，通过添加高剂量阿糖胞苷（CA）和同时连续使用粒细胞集落刺激因子（G-CSF）来加强预处理，可显著提高CBT后的生存率。为了证实G-CSF + CA联合治疗的效果，除了标准预处理方案环磷酰胺（CY）/全身照射（TBI）外，我们设计了一项随机对照研究，比较了CA/CY/TBI与G-CSF预处理（G-CSF联合条件脐带血移植[G-CONCORD]研究）。这是一项多中心、开放标签、随机III期研究，旨在比较G-CSF+CA/CY/TBI作为CBT与CA/CY/TBI的预处理方案。年龄在16-55岁的急性髓性白血病或骨髓增生异常综合征患者有资格参加。目标样本量为160例，注册期为4年。主要终点是CBT后的2年无病生存率。次要终点是总生存期、复发、非复发死亡率、急性和慢性移植物抗宿主病、植入率、中性粒细胞恢复时间、短期不良事件、感染发生率和死亡原因。本研究在患者登记后，在有G-CSF组与无G-CSF组之间采用基于网络的单次一对一随机化。两组均采用大剂量CA联合CY/TBI预处理。在日本实施《临床试验法》后，研究方案获得了中央审查委员会（名古屋大学认证审查委员会）的批准。本研究的数据手稿将提交给高质量的同行评审医学期刊发表。研究结果将通过在国家/国际会议和同行评审期刊上的演讲传播。UMIN 000029947和jRCT 041180059。
A better long-term quality of life after umbilical cord blood transplantation (CBT) is observed compared with transplants from other alternative donors, whereas graft failure and relapses after CBT are still major issues. To minimise graft failure and relapse after CBT, intensification of conditioning by the addition of high-dose cytosine arabinoside (CA) and concomitant continuous use of granulocyte-colony stimulating factor (G-CSF) are reported to convey a significantly better survival after CBT in some retrospective studies. To confirm the effect of G-CSF plus CA combination, in addition to the standard conditioning regimen, cyclophosphamide (CY)/total body irradiation (TBI), we design a randomised controlled study comparing CA/CY/TBI with versus without G-CSF priming (G-CSF combined conditioned cord blood transplantation [G-CONCORD] study). This is a multicentre, open-label, randomised phase III study that aimed to compare G-CSF+CA/CY/TBI as a conditioning regimen for CBT with CA/CY/TBI. Patients with acute myeloid leukaemia or myelodysplastic syndrome, aged 16–55 years, are eligible. The target sample size is 160 and the registration period is 4 years. The primary endpoint is the 2-year disease-free survival rate after CBT. The secondary endpoints are overall survival, relapse, non-relapse mortality, acute and chronic graft-versus-host disease, engraftment rate, time to neutrophil recovery, short-term adverse events, incidence of infections and causes of death. This study employs a single one-to-one web-based randomisation between the with-G-CSF versus without-G-CSF groups after patient registration. Combination of high-dose CA and CY/TBI in both groups is used for conditioning. The study protocol was approved by the central review board, Nagoya University Certified Review Board, after the enforcement of the Clinical Trials Act in Japan. The manuscripts presenting data from this study will be submitted for publication in quality peer-reviewed medical journals. Study findings will be disseminated via presentations at national/international conferences and peer-reviewed journals. UMIN000029947 and jRCTs041180059.
登录
查看更多内容
DOI：
10.1056/nejmoa1602074
发表时间：
2016-09-08
期刊：
The New England journal of medicine
影响因子：
--
作者：
Milano F;Gooley T;Wood B;Woolfrey A;Flowers ME;Doney K;Witherspoon R;Mielcarek M;Deeg JH;Sorror M;Dahlberg A;Sandmaier BM;Salit R;Petersdorf E;Appelbaum FR;Delaney C
通讯作者：
Delaney C
影响因子：
20.3
作者：
Armand, Philippe;Kim, Haesook T.;Saber, Wael
通讯作者：
Saber, Wael
影响因子：
7.5
作者：
Barker, Juliet N.;Boughan, Kirsten;Politikos, Ioannis
通讯作者：
Politikos, Ioannis
影响因子：
158.5
作者：
Laughlin, MJ;Eapen, M;Horowitz, MM
通讯作者：
Horowitz, MM
影响因子：
20.3
作者：
Greenberg, P;Cox, C;Bennett, J
通讯作者：
Bennett, J