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单药贝伐珠单抗治疗骨髓纤维化:骨髓增殖性疾病研究联盟试验的结果。

Single agent bevacizumab for myelofibrosis: results of the Myeloproliferative Disorders Research Consortium Trial.

机构信息

Mayo Clinic, Scottsdale, AZ, USA.

出版信息

Haematologica. 2013 Sep;98(9):1421-3. doi: 10.3324/haematol.2012.083337. Epub 2013 Jun 28.

Abstract

The myeloproliferative neoplasm, myelofibrosis, is a morbid and frequently fatal illness encompassing primary myelofibrosis, and end-stage essential thrombocythemia and polycythemia. Bevacizumab (15 mg/kg intravenous (i.v.) every 21 days) was tested in a phase II international trial conducted by the Myeloproliferative Disorders Research Consortium. Thirteen patients were enrolled in the first stage of this 2-stage trial. Among the 11 patients who received therapy, only 3 received more than 4 cycles of therapy; none of the patients achieved an objective response. Furthermore, significant toxicity, not directly related to the vascular or gastrointestinal events typically associated with the anti-VEGF monoclonal antibody preparation in other disease states, was observed. Lack of objective responses coupled with toxicity led to the decision to terminate the study early. If future studies incorporate bevacizumab in combination therapy for myelofibrosis, more modest doses should be considered. (clinicaltrials.gov Identifier 00667277).

摘要

骨髓增生性肿瘤,骨髓纤维化,是一种严重且常致命的疾病,包括原发性骨髓纤维化、终末期原发性血小板增多症和红细胞增多症。贝伐珠单抗(15mg/kg 静脉注射(i.v.)每 21 天一次)在由骨髓增生性疾病研究联合会进行的一项 2 期国际试验中进行了测试。该 2 期试验的第一阶段共纳入了 13 名患者。在接受治疗的 11 名患者中,只有 3 名患者接受了超过 4 个周期的治疗;没有患者达到客观缓解。此外,观察到与其他疾病状态中通常与抗 VEGF 单克隆抗体制剂相关的血管或胃肠道事件无关的显著毒性。缺乏客观反应加上毒性导致提前终止研究。如果未来的研究将贝伐珠单抗纳入骨髓纤维化的联合治疗,应考虑更适度的剂量。(临床试验.gov 标识符 00667277)。

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