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[在大西部大区儿童急性淋巴细胞白血病中使用博纳吐单抗:给所有人的一个机会]

[Use of blinatumomab in children acute lymphoblastic leukemia in the Grand Ouest interregion: A chance for all].

作者信息

Camuset Margaux, Grain Audrey, Lorton Fleur, Minckes Odile, Jourdain Anne, Millot Frédéric, Pellier Isabelle, Gandemer Virginie, Battisti Fanny Rialland

机构信息

Hémato-immuno-oncologie pédiatrique, CHU de Nantes, 5, allée de l'île-Gloriette, 44093 Nantes cedex 01, France.

Hématologie pédiatrique, AP-HP, CHU Robert-Debré, 48, boulevard Sérurier, 75019 Paris, France.

出版信息

Bull Cancer. 2019 Mar;106(3):206-215. doi: 10.1016/j.bulcan.2018.11.012. Epub 2019 Jan 11.

Abstract

INTRODUCTION

Relapsed/refractory acute lymphoblastic leukemia (ALL) in children has a pejorative prognosis and justifies to be treated by hematopoietic stem cell transplantation (HSCT). A minimal residual disease (MRD) before transplantation is a major part of prognosis. Blinatumomab, a bispecific antibody CD19/CD3, allowed to achieve a cytologic and molecular complete remission in adults with refractory B-precursor ALL. This retrospective study analyses results from a pediatric cohort treated by blinatumomab thanks to an interregional structuring consortium.

PATIENTS AND METHODS

Patients between 0 and 23 years old, from the 7 centers of the french "Grand Ouest" interregional network, treated by blinatumomab for a relapsed or refractory ALL, from January 2015 to January 2018, were included. The efficiency of blinatumomab was assessed in terms of complete remission, minimal residual disease, overall survival, and tolerability of treatment.

RESULTS

Thirteen of 18 patients achieved a complete remission, with negative minimal residual disease for ten of them. Fourteen patients proceeded to stem cell transplantation,. Eight out of 14 patients obtained long term remission after HSCT. As far as tolerance is concerned, no serious adverse event, neurological or psychiatric disorder, was observed.

CONCLUSION

Thanks to an interregional network collaboration, all children with high risk ALL coming from the western french interregion could be treated by blinatumomab. Blinatumomab offered good hematological conditions to undergo HSCT with a good tolerability.

摘要

引言

儿童复发/难治性急性淋巴细胞白血病(ALL)预后不佳,造血干细胞移植(HSCT)是合理的治疗方法。移植前的微小残留病(MRD)是预后的主要因素。双特异性抗体CD19/CD3的博纳吐单抗可使难治性B前体ALL成人患者实现细胞学和分子学完全缓解。这项回顾性研究分析了一个儿科队列的结果,该队列由一个跨地区组织的联盟使用博纳吐单抗进行治疗。

患者与方法

纳入2015年1月至2018年1月期间,来自法国“大西部”跨地区网络7个中心、接受博纳吐单抗治疗复发或难治性ALL的0至23岁患者。从完全缓解、微小残留病、总生存期和治疗耐受性方面评估博纳吐单抗的疗效。

结果

18例患者中有13例实现完全缓解,其中10例微小残留病为阴性。14例患者进行了干细胞移植。14例患者中有8例在HSCT后获得长期缓解。就耐受性而言,未观察到严重不良事件、神经或精神障碍。

结论

通过跨地区网络合作,所有来自法国西部跨地区的高危ALL儿童都可以接受博纳吐单抗治疗。博纳吐单抗为接受HSCT提供了良好的血液学条件,耐受性良好。

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