Department of Hematology, Nicolaus Copernicus Hospital, Torun, Poland.
Department of Hematology, St. John's Cancer Center, Lublin, Poland.
Eur J Haematol. 2018 Sep;101(3):354-361. doi: 10.1111/ejh.13106. Epub 2018 Jul 12.
Patients with relapsed/refractory multiple myeloma (RRMM) have poor prognosis. Pomalidomide is an immunomodulatory compound that has demonstrated activity in MM patients with disease refractory to lenalidomide and bortezomib.
Participants of clinical trials are highly selected populations; therefore, the aim of this study was to present observations from real practice that might provide important information for practitioners.
We analyzed retrospectively 50 patients treated with pomalidomide in 12 Polish sites between 2014 and 2017. Median age was 63 years, median time since diagnosis 4.5 years and median number of prior regimens 4.
The overall response rate was 39.1%. Median progression-free survival (PFS) and overall survival (OS) were 10.0 and 14.0 months, respectively. Previous treatment with immunomodulatory drugs, bortezomib or stem cell transplant had no impact on PFS and OS. Most frequent grade 3/4 treatment-emergent adverse events were hematologic (neutropenia 24.0%, thrombocytopenia 10.0%, anemia 8.0%). Most common grade 3/4 non-hematologic toxicities were respiratory tract infection (14.0%) and neuropathy (4.0%).
This real-world data have confirmed that pomalidomide is an active drug in RRMM and support results of published clinical trials and other real-world studies.
复发/难治性多发性骨髓瘤（RRMM）患者预后较差。泊马度胺是一种免疫调节化合物，在对来那度胺和硼替佐米耐药的 MM 患者中显示出活性。
临床试验参与者是高度选择的人群；因此，本研究旨在展示来自实际实践的观察结果，这些结果可能为从业者提供重要信息。
我们回顾性分析了 2014 年至 2017 年间在波兰 12 个地点接受泊马度胺治疗的 50 例患者。中位年龄为 63 岁，中位诊断后时间为 4.5 年，中位既往治疗方案数为 4 个。
总缓解率为 39.1%。中位无进展生存期（PFS）和总生存期（OS）分别为 10.0 和 14.0 个月。先前使用免疫调节药物、硼替佐米或干细胞移植对 PFS 和 OS 没有影响。最常见的 3/4 级治疗相关不良事件为血液学毒性（中性粒细胞减少症 24.0%、血小板减少症 10.0%、贫血症 8.0%）。最常见的 3/4 级非血液学毒性为呼吸道感染（14.0%）和周围神经病（4.0%）。
这些真实世界的数据证实，泊马度胺是 RRMM 的一种有效药物，支持已发表的临床试验和其他真实世界研究的结果。
