Carraway Hetty E, Brunner Andrew M, Lai Catherine E, Luskin Marlise R, Park Jae, Perl Alexander E, Stein Eytan M, Wang Eunice S, Zeidan Amer M, Zeidner Joshua F, Komrokji Rami
Division of Hematologic Oncology and Blood Disorders, Leukemia Program, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH; Department of Medicine, Cleveland Clinic Lerner College of Medicine of Case Western Reserve University, Cleveland, OH.
Leukemia Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA.
Clin Lymphoma Myeloma Leuk. 2025 Aug;25(8):583-589. doi: 10.1016/j.clml.2025.03.005. Epub 2025 Mar 13.
The management of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) have evolved substantially in recent years with the development of targeted therapies and novel nontargeted approaches. However, many questions remain about how to best use current therapies, and there is a large unmet need for effective therapies, particularly for patients with higher-risk MDS, AML, and those with MDS/AML relapsed/refractory (R/R) to prior therapy.
A panel of experts was assembled to discuss current controversies and unanswered questions in the care of patients with MDS and AML. Workshop topics included: molecular testing and new classification systems, clonal hematopoiesis, treatment of MDS (lower-risk and higher-risk), frontline treatment of AML, treatment of special populations, treatment of R/R AML, and novel approaches.
We identified many areas of ongoing controversy in the diagnosis and management of MDS and AML related to classification and risk assessment, treatment selection, sequencing of therapies, and monitoring of responses. Many clinical trials are ongoing to further improve outcomes for patients with MDS and AML, and we noted potential areas of debate related to study design, selection of endpoints, and assessment of responses. The controversies and gaps in knowledge identified by this panel will inform a follow-up conference in 2025 that will employ a modified Delphi method with a goal of developing and publishing formal consensus recommendations that can provide actionable guidance to clinicians in practice.
近年来,随着靶向治疗和新型非靶向方法的发展,骨髓增生异常综合征(MDS)和急性髓系白血病(AML)的治疗有了显著进展。然而,关于如何最佳使用当前疗法仍有许多问题,并且对于有效疗法存在大量未满足的需求,特别是对于高危MDS、AML患者以及对先前治疗复发/难治(R/R)的MDS/AML患者。
召集了一组专家讨论MDS和AML患者护理中当前的争议和未解决的问题。研讨会主题包括:分子检测和新的分类系统、克隆性造血、MDS的治疗(低危和高危)、AML的一线治疗、特殊人群的治疗、R/R AML的治疗以及新方法。
我们确定了MDS和AML诊断与管理中许多持续存在争议的领域,涉及分类和风险评估、治疗选择、治疗顺序以及反应监测。许多临床试验正在进行,以进一步改善MDS和AML患者的预后,并且我们注意到与研究设计、终点选择和反应评估相关的潜在争议领域。该专家小组确定的争议和知识空白将为2025年的后续会议提供信息,该会议将采用改进的德尔菲法,目标是制定并发布正式的共识建议,为临床医生在实践中提供可操作的指导。