Despite an increasing desire to use historical cohorts as “synthetic” controls for new drug evaluation, limited data exist regarding the comparability of real-world outcomes to those in clinical trials. Governmental cancer data often lacks details on treatment, response, and molecular characterization of disease sub-groups. The Australasian Leukaemia and Lymphoma Group National Blood Cancer Registry (ALLG NBCR) includes source information on morphology, cytogenetics, flow cytometry, and molecular features linked to treatment received (including transplantation), response to treatment, relapse, and survival outcome. Using data from 942 AML patients enrolled between 2012–2018, we assessed age and disease-matched control and interventional populations from published randomized trials that led to the registration of midostaurin, gemtuzumab ozogamicin, CPX-351, oral azacitidine, and venetoclax. Our analyses highlight important differences in real-world outcomes compared to clinical trial populations, including variations in anthracycline type, cytarabine intensity and scheduling during consolidation, and the frequency of allogeneic hematopoietic cell transplantation in first remission. Although real-world outcomes were comparable to some published studies, notable differences were apparent in others. If historical datasets were used to assess the impact of novel therapies, this work underscores the need to assess diverse datasets to enable geographic differences in treatment outcomes to be accounted for.
尽管人们日益希望将历史队列作为新药评估的"合成"对照组，但关于真实世界结果与临床试验结果可比性的数据仍然有限。政府癌症数据往往缺乏治疗细节、疗效反应以及疾病亚组的分子特征信息。澳大利亚白血病与淋巴瘤组国家血癌登记处（ALLG NBCR）收录了与接受治疗（包括移植）、治疗反应、复发及生存结果相关联的形态学、细胞遗传学、流式细胞术及分子特征等源信息。通过分析2012年至2018年间登记的942例AML患者数据，我们将年龄与疾病匹配的对照组及已发表的随机试验干预人群进行比对，这些试验涉及米哚妥林、吉妥珠单抗奥佐米星、CPX-351、口服阿扎胞苷和维奈托克的注册审批。我们的分析揭示了真实世界结果与临床试验人群间的重要差异，包括蒽环类药物类型、巩固治疗期间阿糖胞苷强度与给药方案，以及首次缓解期异基因造血细胞移植的实施频率等方面。虽然真实世界结果与部分已发表研究具有可比性，但在其他研究中存在显著差异。若采用历史数据集评估新型疗法的效果，本研究强调有必要评估多样化数据集，以充分考虑治疗结果的地域性差异。
肿瘤(癌症)患者之家