来那度胺 (LEN) 可在 60-70% 的 del(5q) 骨髓增生异常肿瘤 (MDS) 患者中诱导红细胞输注独立性 (RBC-TI)。目前的建议是继续对有反应的患者使用 LEN，直到失败或进展，这可能会产生毒性，并且医疗系统的成本很高。这项 HARMONY Alliance 研究调查了停止 LEN 而不依赖红细胞输注的 MDS del(5q) 患者的结果。我们招募了 118 名 IPSS-R 低中风险患者。 70 名患者 (59%) 因不耐受而停用 LEN，其中 38 名 (32%) 患者根据医生决定，9 名 (8%) 患者根据自己的决定，1 名 (1%) 患者出于未知原因停用 LEN。停药后中位随访 49 个月后，50/118 名患者失去 RBC-TI，22/30 接受细胞遗传学重新评估的患者失去完全细胞遗传学反应。中位 RBC-TI 持续时间为 56 个月。在多变量分析中，LEN 停用后的 RBC-TI 持续时间与 LEN 治疗前的低输血负担、治疗 ≥ 12 个 LEN 周期、年龄较小以及 LEN 停用时较高的 Hb 水平相关。 48 名患者因失去反应而再次接受 LEN 治疗，28 名患者再次达到 RBC-TI。这些数据表明，在达到 RBC-TI 的 MDS del(5q) 患者中停止 LEN 治疗可以使大部分患者延长 TI 维持时间。© 2024。作者，获得 Springer Nature Limited 的独家许可。

Lenalidomide (LEN) can induce red blood cell-transfusion independence (RBC-TI) in 60-70% of del(5q) myelodysplastic neoplasm (MDS) patients. Current recommendation is to continue LEN in responding patients until failure or progression, with likelihood of toxicity and a high cost for healthcare systems. This HARMONY Alliance study investigated the outcome of MDS del(5q) patients who discontinued LEN while RBC-transfusion independent. We enrolled 118 patients with IPSS-R low-intermediate risk. Seventy patients (59%) discontinued LEN for intolerance, 38 (32%) per their physician decision, nine (8%) per their own decision and one (1%) for unknown reasons. After a median follow-up of 49 months from discontinuation, 50/118 patients lost RBC-TI and 22/30 who underwent cytogenetic re-evaluation lost complete cytogenetic response. The median RBC-TI duration was 56 months. In multivariate analysis, RBC-TI duration after LEN discontinuation correlated with low transfusion burden before LEN therapy, treatment ≥ 12 LEN cycles, younger age and higher Hb level at LEN withdrawal. Forty-eight patients were re-treated with LEN for loss of response and 28 achieved again RBC-TI. These data show that stopping LEN therapy in MDS del(5q) patients who reached RBC-TI allows prolonged maintenance of TI in a large subset of patients.© 2024. The Author(s), under exclusive licence to Springer Nature Limited.