Jurczyszyn Artur, Castillo Jorge J, Olszewska-Szopa Magdalena, Kumar Lalit, Thibaud Santiago, Richter Joshua, Flicker Kari, Fiala Mark, Vij Ravi, Yi Shuhua, Xu Fang, Silbermann Rebecca, Gaisan Carmen Montes, Ocio Enrique M, Waszczuk-Gajda Anna, Crusoe Edvan De Queiroz, Salomon-Perzyński Aleksander, Hus Iwona, Valls Julio Davila, Gozzetti Alessandro, Czepiel Jacek, Krzanowska Katarzyna, Chappell Aimee, Chellapuram S K, Suska Anna, Vesole David H
Plasma Cell Dyscrasia Center, Department of Hematology Faculty of Medicine, Jagiellonian University Medical College, Cracow, Poland.
Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA.
Clin Lymphoma Myeloma Leuk. 2022 May;22(5):297-304. doi: 10.1016/j.clml.2021.10.007. Epub 2021 Oct 31.
POEMS syndrome, a rare plasma cell disorder, is challenging both in the diagnostic and therapeutic management. We present real word retrospective analysis of 108 cases analyzing clinical features and therapeutic modes. We compare our results with the available literature. This is the first description with such wide use of proteasome inhibitors in first line treatment. POEMS (Polyneuropathy, organomegaly, endocrinopathy, M-protein, skin changes) syndrome is a rare and challenging plasma cell disorder, both in the diagnostic and therapeutic management of the disease. Currently, the literature on POEMS is sparse with most evidence being case reports and small case studies. We present a retrospective real world experience of 108 patients with POEMS. We analyzed the clinical features and therapeutic interventions. Regarding clinical features, our findings demonstrated that skin lesions, thrombocythemia and polycythemia were present less frequently than reported previously. Regarding clinical interventions, this is one of the largest analyses of front line treatment in POEMS and the first one to include frequent utilization of proteasome inhibitors (37%). Bortezomib monotherapy was the most effective therapy achieving complete remission/very good partial remissions (CR/VGPR) in 69% of patients. Thirty percent of patients proceeded to planned autologous stem cell transplant (ASCT) as part of the front-line treatment resulting in statistically superior progression-free (PFS) and overall survival (OS) compared to non-ASCT treated patients (P= .003). In multivariate analysis, anemia, thrombocytopenia, and as age over 60 were associated with a negative impact on patient outcomes.
POEMS综合征是一种罕见的浆细胞疾病，在诊断和治疗管理方面都具有挑战性。我们对108例病例进行了真实世界回顾性分析，分析其临床特征和治疗模式。我们将我们的结果与现有文献进行了比较。这是首次描述在一线治疗中如此广泛地使用蛋白酶体抑制剂。POEMS（多发性神经病、器官肿大、内分泌病、M蛋白、皮肤改变）综合征是一种罕见且具有挑战性的浆细胞疾病，在该疾病的诊断和治疗管理中均是如此。目前，关于POEMS的文献稀少，大多数证据为病例报告和小型病例研究。我们呈现了108例POEMS患者的回顾性真实世界经验。我们分析了临床特征和治疗干预措施。关于临床特征，我们的研究结果表明，皮肤病变、血小板增多症和红细胞增多症的出现频率低于先前报道。关于临床干预措施，这是对POEMS一线治疗进行的最大规模分析之一，也是首次纳入频繁使用蛋白酶体抑制剂（37%）的分析。硼替佐米单药治疗是最有效的治疗方法，69%的患者实现了完全缓解/非常好的部分缓解（CR/VGPR）。30%的患者作为一线治疗的一部分进行了计划中的自体干细胞移植（ASCT），与未接受ASCT治疗的患者相比，其无进展生存期（PFS）和总生存期（OS）在统计学上更优（P = 0.003）。在多变量分析中，贫血、血小板减少症以及年龄超过60岁对患者预后有负面影响。
