Division of Hematology/Oncology, Mayo Clinic Scottsdale, Arizona, USA.
Am J Hematol. 2011 Jan;86(1):96-8. doi: 10.1002/ajh.21892.
We have previously reported the benefits of combination therapy with thalidomide and prednisone for the improvement of cytopenias and splenomegaly for patients with myelofibrosis (MF); both primary and those arising from an antecedent polycythemia vera (PV) and essential thrombocythemia (ET). We report here the overall efficacy of three different thalidomide-prednisone based regimens (alone or in combination with either oral cyclophosphamide for three months or continuous weekly etanercept) when assessed by the IWG-MRT response criteria which were developed after completion of these studies. Additionally we report on the durability of response and long-term follow-up. We observed an overall response rate of 28% by IWG-MRT criteria (mostly clinical improvement - 22% anemia, 8% splenomegaly) with a median duration of response of 8.5 months. Toxicities primarily consisted of neuropathy (Grade 3 or higher neuropathy in 6%) and cytopenias (Grade 3 or higher in 20%). After a median follow-up of three years, fourteen patients (28%) had expired from MF at the time of this analysis with median survival of 36 months.
我们之前曾报道过沙利度胺联合泼尼松治疗骨髓纤维化（MF）患者血细胞减少和脾肿大的疗效，这些患者既有原发性的，也有继发于先前真性红细胞增多症（PV）和原发性血小板增多症（ET）的。我们根据国际工作组-骨髓纤维化研究小组（IWG-MRT）反应标准报告了三种不同的基于沙利度胺-泼尼松的方案（单独使用或与连续每周使用依那西普联合使用三个月）的总体疗效，这些标准是在这些研究完成后制定的。此外，我们还报告了反应的持久性和长期随访结果。根据 IWG-MRT 标准，我们观察到总体反应率为 28%（主要为临床改善 - 22%贫血，8%脾肿大），反应持续时间的中位数为 8.5 个月。毒性主要包括周围神经病（3 级或更高级别的神经病占 6%）和血细胞减少症（3 级或更高级别的占 20%）。在中位随访 3 年后，14 名患者（28%）在本次分析时因 MF 而死亡，中位生存时间为 36 个月。
