Section of Hematology, Department of Internal Medicine, Yale School of Medicine, and Yale Comprehensive Cancer Center, Yale University, New Haven, CT, USA.
Department of Medical Oncology, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA.
Curr Hematol Malig Rep. 2024 Jun;19(3):138-150. doi: 10.1007/s11899-024-00733-y. Epub 2024 Apr 18.
Myelodysplastic syndromes/neoplasms (MDS) represent a diverse group of pathologically distinct diseases with varying prognoses and risks of leukemia progression. This review aims to discuss current treatment options for elderly patients with MDS, focusing on patients ineligible for intensive chemotherapy or allogenic hematopoietic stem cell transplantation (HSCT). The challenges associated with treatment in this population and emerging therapeutic prospects are also explored.
Recent advancements in molecular diagnostics have enhanced risk stratification by incorporating genetic mutations, notably through the molecular International Prognostic Scoring System (IPSS-M). Lower-risk MDS (LR-MDS) treatment ranges from observation to supportive measures and erythropoiesis-stimulating agents (ESAs), with emerging therapies like luspatercept showing promise. High-risk MDS (HR-MDS) is treated with hypomethylating agents (HMAs) or allogenic HSCT, but outcomes remain poor. Elderly MDS patients, often diagnosed after 70, pose challenges in treatment decision-making. The IPSS-M aids risk stratification, guiding therapeutic choices. For LR-MDS, supportive care, ESAs, and novel agents like luspatercept are considered. Treatment of HR-MDS involves HMAs or allogenic HSCT. Emerging treatments, including oral HMAs and novel agents targeting FLT3, and IDH 1/2 mutations, show promise. Future research should refine treatment strategies for this elderly population focusing on quality-of-life improvement.
骨髓增生异常综合征/肿瘤(MDS)是一组具有不同病理特征、预后和白血病进展风险的疾病。本文旨在讨论目前不适合强化化疗或异基因造血干细胞移植(HSCT)的老年 MDS 患者的治疗选择,重点讨论不适合强化化疗或异基因 HSCT 的老年 MDS 患者的治疗选择。还探讨了该人群治疗中存在的挑战和新出现的治疗前景。
分子诊断学的最新进展通过纳入基因突变(特别是通过分子国际预后评分系统(IPSS-M))增强了风险分层。低危 MDS(LR-MDS)的治疗范围从观察到支持性治疗和红细胞生成刺激剂(ESA),新兴疗法如 luspatercept 显示出前景。高危 MDS(HR-MDS)采用低甲基化药物(HMA)或异基因 HSCT 治疗,但结果仍然较差。年龄较大的 MDS 患者,通常在 70 岁以后诊断,在治疗决策中存在挑战。IPSS-M 有助于风险分层,指导治疗选择。对于 LR-MDS,采用支持性护理、ESA 和 luspatercept 等新型药物。HR-MDS 的治疗包括 HMA 或异基因 HSCT。新兴治疗方法,包括口服 HMA 和针对 FLT3 和 IDH1/2 突变的新型药物,显示出前景。未来的研究应针对老年人群,重点改善生活质量,制定更精细的治疗策略。