Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary.
National Institute for Hematology and Infectious Diseases, Department of Hematology and Stem Cell Transplantation, South Pest Central Hospital, Budapest, Hungary.
Pathol Oncol Res. 2021 Apr 22;27:613264. doi: 10.3389/pore.2021.613264. eCollection 2021.
In Hungary, the cost of lenalidomide-based therapy is covered only for relapsed multiple myeloma (MM) patients, therefore lenalidomide is typically used in the second-line either as part of a triplet with proteasome inhibitors or as a doublet. Lenalidomide-dexamethasone is a standard treatment approach for relapsed/refractory MM, and according to recent large randomized clinical trials (RCT, the standard arm of POLLUX, ASPIRE, TOURMALINE), the progression-free survival (PFS) is expected to be approximately 18 months. We surveyed ten Hungarian centers treating MM and collected data of 278 patients treated predominantly after 2016. The median age was 65 years, and patients were distributed roughly equally over the 3 international staging system groups, but patients with high risk cytogenetics were underrepresented. 15.8% of the patients reached complete response, 21.6% very good partial response, 40.6% partial response, 10.8% stable disease, and 2.5% progressed on treatment. The median PFS was unexpectedly long, 24 months, however only 9 months in those with high risk cytogenetics. We found interesting differences between centers regarding corticosteroid type (prednisolone, methylprednisolone or dexamethasone) and dosing, and also regarding the choice of anticoagulation, but the outcome of the various centers were not different. Although the higher equivalent steroid dose resulted in more complete responses, the median PFS of those having lower corticosteroid dose and methylprednisolone were not inferior compared to the ones with higher dose dexamethasone. On multivariate analysis high risk cytogenetics and the number of prior lines remained significant independent prognostic factors regarding PFS ( < 0.001 and = 0.005). Our results show that in well-selected patients Lenalidomide-dexamethasone can be a very effective treatment with real-world results that may even outperform those reported in the recent RCTs. This real world information may be more valuable than outdated RCT data when treatment options are discussed with patients.
在匈牙利,来那度胺的治疗费用仅涵盖复发多发性骨髓瘤(MM)患者,因此来那度胺通常作为三联疗法的一部分与蛋白酶体抑制剂联合使用,或作为二联疗法使用。来那度胺-地塞米松是复发/难治性 MM 的标准治疗方法,根据最近的大型随机临床试验(RCT,POLLUX、ASPIRE、TOURMALINE 的标准组),无进展生存期(PFS)预计约为 18 个月。我们调查了十个治疗 MM 的匈牙利中心,并收集了 2016 年后主要接受治疗的 278 名患者的数据。中位年龄为 65 岁,患者在 3 个国际分期系统组中的分布大致相等,但具有高危细胞遗传学特征的患者代表性不足。15.8%的患者达到完全缓解,21.6%非常好的部分缓解,40.6%部分缓解,10.8%疾病稳定,2.5%治疗进展。出乎意料的是,中位 PFS 很长,为 24 个月,但高危细胞遗传学特征的患者仅为 9 个月。我们发现不同中心之间在皮质类固醇类型(泼尼松龙、甲泼尼龙或地塞米松)和剂量、抗凝药物选择方面存在有趣的差异,但各个中心的结果没有差异。尽管较高等效类固醇剂量导致更多的完全缓解,但使用较低皮质类固醇剂量和甲泼尼龙的患者的中位 PFS 并不劣于使用较高剂量地塞米松的患者。多变量分析显示,高危细胞遗传学特征和先前治疗线数仍然是 PFS 的独立预后因素(<0.001 和 =0.005)。我们的研究结果表明,在精心选择的患者中,来那度胺-地塞米松可能是一种非常有效的治疗方法,其真实世界的结果甚至可能优于最近 RCT 报告的结果。在与患者讨论治疗选择时,这种真实世界的信息可能比过时的 RCT 数据更有价值。