用于评估临床试验中治疗骨髓增生性肿瘤伴原始细胞增多/转化型（MPN-BP）患者反应的标准：骨髓增生性肿瘤急性髓系白血病后联盟的正式建议。
Proposed criteria for response assessment in patients treated in clinical trials for myeloproliferative neoplasms in blast phase (MPN-BP): formal recommendations from the post-myeloproliferative neoplasm acute myeloid leukemia consortium.
机构信息
Tisch Cancer Institute, Mount Sinai School of Medicine, New York, NY, USA.
出版信息
Leuk Res. 2012 Dec;36(12):1500-4. doi: 10.1016/j.leukres.2012.08.013. Epub 2012 Aug 28.
Abstract
Leukemic transformation (LT) of a myeloproliferative neoplasm (MPN) is associated with a dismal prognosis and no medical therapies have shown a survival improvement in patients with MPN in blast phase (MPN-BP). Effective therapies for the treatment of MPN-BP are a serious unmet need. Consensus response criteria do not exist for the treatment of patients with MPN-BP and this is necessary for the uniformed reporting of treatment response in clinical trials. We have identified relevant MPN and MPN-BP features in order to define treatment response categories that reflect hematological, clinical, pathological, cytogenetic and molecular changes after therapeutic intervention. We plan to validate these proposed response criteria within multi-centered clinical trials.
摘要
白血病转化（LT）的骨髓增殖性肿瘤（MPN）与预后不良相关，且在 blast 期 MPN（MPN-BP）患者中，没有任何医学疗法显示出生存改善。治疗 MPN-BP 的有效疗法是一个严重的未满足需求。对于 MPN-BP 患者的治疗，共识缓解标准并不存在，这对于临床试验中治疗反应的统一报告是必要的。我们已经确定了相关的 MPN 和 MPN-BP 特征，以便定义治疗反应类别，这些类别反映了治疗干预后血液学、临床、病理学、细胞遗传学和分子变化。我们计划在多中心临床试验中验证这些拟议的缓解标准。
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