Department of Medicine III, University Hospital, Ludwig Maximilian University, Munich, Germany.
German Cancer Consortium, Heidelberg, Germany.
Blood. 2017 Sep 28;130(13):1491-1498. doi: 10.1182/blood-2017-07-737353. Epub 2017 Aug 7.
Follicular lymphoma (FL) is a clinically and molecularly highly heterogeneous disease. Most patients achieve long-lasting remissions and have excellent overall survival (OS) with current treatment. However, ∼20% of patients have early progression of disease and short OS. At present, therapies are not guided by individual risk or disease biology. Reliable tools for patient stratification are urgently needed to avoid overtreatment of low-risk patients and to prioritize alternative approaches in high-risk patients. A rapidly expanding repertoire of promising therapeutic options is available for clinical evaluation; however, the numbers of patients with FL and the resources to conduct adequately powered trials are limited. Recent studies have shown that gene mutations can serve as prognostic and/or predictive biomarkers, in particular when integrated into composite risk models. Before translating these findings into routine clinical practice, however, several challenges loom. We review aspects of "clinicogenetic" risk model development and validation that apply to FL and more generally to other cancers. Finally, we propose a crowdsourcing effort that could expedite the development, validation, refinement, and selection of risk models. A new era of collaboration and harmonization is required if we hope to transition from empiric selection of therapeutics to risk-based, biology-guided treatment of patients with FL.
滤泡性淋巴瘤(FL)是一种临床上和分子上高度异质的疾病。大多数患者通过目前的治疗能够获得持久的缓解和极好的总生存期(OS)。然而,约 20%的患者疾病早期进展且 OS 较短。目前,治疗并非基于个体风险或疾病生物学。迫切需要可靠的患者分层工具,以避免对低危患者过度治疗,并为高危患者优先考虑替代方法。目前有大量有前途的治疗选择可供临床评估;然而,FL 患者的数量以及进行充分效能试验的资源有限。最近的研究表明,基因突变可以作为预后和/或预测生物标志物,特别是当整合到复合风险模型中时。然而,在将这些发现转化为常规临床实践之前,还存在一些挑战。我们回顾了适用于 FL 以及更广泛的其他癌症的“临床遗传学”风险模型开发和验证的各个方面。最后,我们提出了一项众包工作,这可能会加速风险模型的开发、验证、改进和选择。如果我们希望从经验性选择治疗方法过渡到基于风险、生物学指导的 FL 患者治疗,就需要新的协作和协调时代。