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新型药物时代浆细胞白血病患者自体或异基因干细胞移植的疗效

Outcomes with Autologous or Allogeneic Stem Cell Transplantation in Patients with Plasma Cell Leukemia in the Era of Novel Agents.

作者信息

Lemieux Christopher, Johnston Laura J, Lowsky Robert, Muffly Lori S, Craig Juliana K, Shiraz Parveen, Rezvani Andrew, Frank Matthew J, Weng Wen-Kai, Meyer Everett, Shizuru Judith, Arai Sally, Negrin Robert, Miklos David B, Sidana Surbhi

机构信息

Stanford Cancer Institute, Stanford University Medical Center, Stanford, California.

Stanford Cancer Institute, Stanford University Medical Center, Stanford, California.

出版信息

Biol Blood Marrow Transplant. 2020 Dec;26(12):e328-e332. doi: 10.1016/j.bbmt.2020.08.035. Epub 2020 Sep 19.

Abstract

Plasma cell leukemia (PCL) is a rare and very aggressive plasma cell disorder. The optimal treatment approach, including whether to pursue an autologous (auto) or allogeneic (allo) stem cell transplantation (SCT) is not clear, given the lack of clinical trial-based evidence. This single-center retrospective study describes the outcomes of 16 patients with PCL (n = 14 with primary PCL) who underwent either autoSCT (n = 9) or alloSCT (n = 7) for PCL in the era of novel agents, between 2007 and 2019. The median age of the cohort was 58 years. High-risk cytogenetics were found in 50% of the patients. All patients received a proteasome inhibitor and/or immunomodulatory drug-based regimen before transplantation. At the time of transplantation, 10 patients (62%) obtained at least a very good partial response (VGPR). The response after autoSCT (3 months) was at least a VGPR in 6 patients (67%; complete response [CR] in 5). All patients undergoing alloSCT achieved a CR at 3 months. Maintenance therapy was provided to 5 patients (56%) after autoSCT. The median progression-free survival after transplantation was 6 months in the autoSCT group, compared with 18 months in the alloSCT group (P = .09), and median overall survival (OS) after transplantation in the 2 groups was 19 months and 40 months, respectively (P = .41). The median OS from diagnosis was 27 months and 49 months, respectively (P = .50). Of the 11 deaths, 10 patients (91%) died of relapsed disease. AlloSCT was not observed to offer any significant survival advantage over autoSCT in PCL, in agreement with recent reports, and relapse remains the primary cause of death in these patients.

摘要

浆细胞白血病(PCL)是一种罕见且侵袭性很强的浆细胞疾病。鉴于缺乏基于临床试验的证据,包括是否进行自体(auto)或异基因(allo)干细胞移植(SCT)在内的最佳治疗方法尚不清楚。这项单中心回顾性研究描述了2007年至2019年新型药物时代16例PCL患者(n = 14例原发性PCL)接受autoSCT(n = 9)或alloSCT(n = 7)治疗PCL的结果。该队列的中位年龄为58岁。50%的患者存在高危细胞遗传学特征。所有患者在移植前均接受了基于蛋白酶体抑制剂和/或免疫调节药物的方案。移植时,10例患者(62%)至少获得了非常好的部分缓解(VGPR)。autoSCT后(3个月),6例患者(67%)至少获得了VGPR(5例完全缓解[CR])。所有接受alloSCT的患者在3个月时均达到CR。autoSCT后,5例患者(56%)接受了维持治疗。autoSCT组移植后的中位无进展生存期为6个月,而alloSCT组为18个月(P = 0.09),两组移植后的中位总生存期(OS)分别为19个月和40个月(P = 0.41)。从诊断开始的中位OS分别为27个月和49个月(P = 0.50)。在11例死亡病例中,10例患者(91%)死于疾病复发。与最近的报道一致,在PCL中未观察到alloSCT比autoSCT具有任何显著的生存优势,复发仍然是这些患者的主要死亡原因。

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