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儿童实体瘤的自体骨髓移植

Autologous bone marrow transplantation in paediatric solid tumours.

作者信息

Pinkerton R, Philip T, Bouffet E, Lashford L, Kemshead J

出版信息

Clin Haematol. 1986 Feb;15(1):187-203. doi: 10.1016/s0308-2261(86)80011-x.

Abstract

Massive therapy with ABMT is now an established treatment modality in paediatric oncology. The technical aspects and most treatment-related complications have been clarified and many phase II studies have shown encouraging results. In advanced neuroblastoma the poor outlook with conventional chemotherapy has stimulated extensive investigation of forms of massive therapy. Current results from several centres indicate that although the median survival is increased, long-term survival in an unselected group of stage IV patients is unlikely to exceed 30% with current regimens. In the future, management of this disease may involve the use of more intensive induction regimens to improve the quality of remission at the time of ABMT, which remains the single most important prognostic factor. Improved purging procedures involve the possible use of double massive therapy regimens and a combination of immunological and chemical treatments. In other paediatric tumours, the relative rarity and limited indications for ABMT make the evaluation of its role more difficult. Preliminary results in advanced rhabdomyosarcoma and Ewing's sarcoma are none the less encouraging and justify further investigation. The value of purging procedures remains controversial and their assessment has been hampered by the lack of sensitive clonogenic assays to detect residual tumour cells. However, neuroblastoma has provided a useful model for the investigation of physical, immunological and chemical procedures. Massive therapy is expensive, time consuming, and carries a high cost in patient morbidity and stress to the families involved. As with any new treatment, it must be adequately assessed in phase III, randomized studies. The ENSG and SIOP trials are a beginning and the future of massive therapy in the paediatric patient will, we hope, be based on a rigorous and scientific comparison with other treatment modalities.

摘要

大剂量自体骨髓移植(ABMT)治疗现已成为小儿肿瘤学中一种既定的治疗方式。其技术层面以及大多数与治疗相关的并发症已得以阐明,许多II期研究也显示出了令人鼓舞的结果。在晚期神经母细胞瘤中,传统化疗预后不佳促使人们对大剂量治疗形式进行了广泛研究。几个中心目前的结果表明,尽管中位生存期有所延长,但按照当前方案,未经选择的IV期患者的长期生存率不太可能超过30%。未来,这种疾病的治疗可能会采用更强化的诱导方案,以提高进行ABMT时的缓解质量,而缓解质量仍然是最重要的单一预后因素。改进的净化程序可能包括使用双重大剂量治疗方案以及免疫和化学治疗的联合应用。在其他小儿肿瘤中,ABMT相对罕见且适应证有限,这使得评估其作用更加困难。不过,晚期横纹肌肉瘤和尤因肉瘤的初步结果仍然令人鼓舞,值得进一步研究。净化程序的价值仍存在争议,由于缺乏检测残留肿瘤细胞的敏感克隆形成试验,对其评估受到了阻碍。然而,神经母细胞瘤为研究物理、免疫和化学程序提供了一个有用的模型。大剂量治疗费用高昂、耗时,而且给患者带来较高的发病率,并给相关家庭带来压力。与任何新治疗方法一样,必须在III期随机研究中对其进行充分评估。欧洲神经母细胞瘤研究组(ENSG)和国际小儿肿瘤学会(SIOP)的试验只是一个开端,我们希望,小儿患者大剂量治疗的未来将基于与其他治疗方式进行严格且科学的比较。

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