GEARBOx - a multisite clinical trial matching tool for children with relapsed or refractory cancer

项目摘要/摘要 尽管治愈率稳步上升，但癌症对许多儿童来说仍然是致命的，特别是那些复发或 难治性疾病。精准医疗为这些孩子带来了巨大的希望，但将儿童与可用的 临床试验仍然是一个挑战，因为缺乏最新的信息和高度复杂的资格标准 基于临床信息以及基因组和免疫表型生物标记物数据。与 白血病和淋巴瘤协会，儿科癌症数据共享(PCDC)团队开发了变速箱 (复发时的基因组资格算法以获得更好的结果)，这是一个临床试验匹配工具，允许临床医生 为复发或难治性急性髓系白血病的患者快速找到潜在的匹配 (AML)。我们现在努力扩大变速箱，以包括更多的肿瘤类型，包括急性 淋巴细胞性白血病、神经母细胞瘤、横纹肌肉瘤、尤文肉瘤和骨肉瘤。另外， 我们将使用新的自动化技术增强临床试验中通常手动提取的资格标准 方法：研究方法。临床医生可获得的有关试验的信息将得到补充，以包括以下关键数据 如何快速有效地让孩子参加配对研究。最后，PCDC团队将与一家 第三方流式细胞仪实验室(血液学)创建自动提取和合并结构化的方法 将免疫表型数据直接输入变速箱工具。这些改进将极大地增强这一过程 为患者找到合适的临床试验，然后让他们参加，从而改善获得关键的 对这些孩子进行精确治疗。

PROJECT SUMMARY / ABSTRACT Despite a steady rise in cure rates, cancer remains deadly for many children, especially those with relapsed or refractory disease. Precision medicine holds great promise for these kids, but matching children to available clinical trials remains a challenge due a lack of up-to-date information and highly complex eligibility criteria based on clinical information and genomic and immunophenotype biomarker data. In collaboration with The Leukemia & Lymphoma Society, the Pediatric Cancer Data Commons (PCDC) team has developed GEARBOx (Genomic Eligibility Algorithm at Relapse for Better Outcomes), a clinical trial matching tool to allow clinicians to quickly find potential matches for their patients with relapsed or refractory acute myelogenous leukemia (AML). We now endeavor to expand GEARBOx to include several more tumor types, including acute lymphoblastic leukemia, neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, and osteosarcoma. Additionally, we will augment the often-manual abstraction of eligibility criteria from clinical trials with new automated methods. The information available to clinicians about trials will be supplemented to include critical data for how to quickly and efficiently enroll a child on a matched study. Finally, the PCDC team will partner with a third-party flow cytometry lab (Hematologics) to create ways to automatically pull and incorporate structured immunophenotype data directly into the GEARBOx tool. These enhancements will greatly enhance the process of finding appropriate clinical trials for patients and then getting them enrolled, thus improving access to critical precision therapy for these children.