母细胞性浆细胞样树突状细胞肿瘤的同种异体造血细胞移植：CIBMTR 分析。
Allogeneic Hematopoietic Cell Transplantation for Blastic Plasmacytoid Dendritic Cell Neoplasm: a CIBMTR analysis.
发表日期：2023 Oct 04
作者：
Hemant S Murthy, Mei-Jie Zhang, Karen Chen, Sairah Ahmed, Uday Deotare, Siddhartha Ganguly, Ankit Kansagra, Fotios V Michelis, Taiga Nishihori, Mrinal M Patnaik, Muhammad Bilal Abid, Mahmoud Aljurf, Yasuyuki Arai, Ulrike Bacher, Talha Badar, Sherif M Badawy, Karen Ballen, Minoo Battiwalla, Amer Beitinjaneh, Nelli Bejanyan, Vijaya R Bhatt, Valerie Inez Brown, Rodrigo Martino, Jean-Yves Cahn, Paul Castillo, Jan Cerny, Saurabh Chhabra, Edward A Copelan, Andrew Daly, Bhagirathbhai Dholaria, Miguel Angel Diaz, César O Freytes, Michael R Grunwald, Shahrukh Hashmi, Gerhard C Hildebrandt, Omer Jamy, Jacinth Joseph, Christopher G Kanakry, Nandita Khera, Maxwell M Krem, Yachiyo Kuwatsuka, Hillard M Lazarus, Lazaros J Lekakis, Hongtao Liu, Dipenkumar Modi, Pashna Munshi, Alberto Mussetti, Neil Palmisiano, Sagar S Patel, David A Rizzieri, Sachiko Seo, Mithun Vinod Shah, Akshay Sharma, Melhem M Solh, Scott R Solomon, Matthew L Ulrickson, Celalettin Ustun, Marjolein W M van der Poel, Leo F Verdonck, John L Wagner, Trent P Wang, Baldeep Wirk, Amer M Zeidan, Mark R Litzow, Partow Kebriaei, Christopher S Hourigan, Daniel J Weisdorf, Wael Saber, Mohamed A Kharfan-Dabaja
来源：
Blood Advances
摘要：
母细胞性浆细胞样树突状细胞肿瘤（BPDCN）是一种罕见的血液恶性肿瘤，预后不良，并且被认为无法通过标准常规化疗治愈。小型观察性研究表明，同种异体造血细胞移植 (allo-HCT) 可为 BPDCN 患者提供持久的缓解。我们使用国际血液和骨髓移植研究中心 (CIBMTR) 报告的数据，对 2007 年至 2018 年间接受异基因 HCT 的 78 个中心的 164 名 BPDCN 患者进行了分析。结果：幸存者的中位随访时间为 49 个月（范围 6-121）。 5 年总生存率 (OS)、无病生存率 (DFS)、复发率和不复发率 (NRM) 分别为 51.2%（95% 置信区间 [95%CI]：42.5-59.8%）、44.4%（分别为 95% CI：36.2-52.8%）、32.2%（95% CI：24.7-40.3%）和 23.3%（95% CI：16.9-30.4%）。疾病复发是最常见的死亡原因。在多变量分析中，年龄≥60 岁可预测 OS 较差（风险比 [HR]= 2.16，95% CI 1.35-3.46，p= 0.001）和较高的 NRM [HR= 2.19，95% CI 1.13-4.22，p= 0.02]。 allo-HCT 时的缓解状态（CR2/PIF/复发与 CR1）可预测较差的 OS [HR= 1.87，95% CI 1.14-3.06，p= 0.01] 和 DFS [HR= 1.75，95% CI 1.11- 2.76，p=0.02]。使用清髓性预处理和全身照射 (TBI) 可预测改善 DFS 并降低复发风险。结论：Allo-HCT 可有效为 BPDCN 提供持久缓解和长期生存。年轻化和 CR1 中的同种异体 HCT 预测可提高生存率，而 TBI 清髓性预处理可预测较少复发并提高无病生存期 (DFS)。需要结合异基因 HCT 的新策略来进一步改善结果。版权所有 © 2023 美国血液学会。
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy with a poor prognosis and considered incurable with standard conventional chemotherapy. Small observational studies have shown that allogeneic hematopoietic cell transplantation (allo-HCT) offers durable remissions in patients with BPDCN. We conducted an analysis of 164 patients with BPDCN from 78 centers who underwent allo-HCT between 2007-2018 using data reported to the Center for International Blood and Marrow Transplant Research (CIBMTR). Results: Median follow up of survivors was 49 months (range 6-121). 5-year overall survival (OS), disease-free survival (DFS), relapse, and non-relapse (NRM) rates were 51.2% (95% confidence interval [95%CI]: 42.5-59.8%), 44.4% (95%CI: 36.2-52.8%), 32.2% (95%CI: 24.7-40.3%), and 23.3% (95%CI: 16.9-30.4%), respectively. Disease relapse was the most common cause of death. On multivariate analyses, age ≥60 was predictive for inferior OS (hazard ratio [HR]= 2.16, 95% CI 1.35-3.46, p= 0.001), and higher NRM [HR= 2.19, 95% CI 1.13-4.22, p= 0.02]. Remission status at time of allo-HCT (CR2/PIF/Relapse vs CR1) was predictive of inferior OS [HR= 1.87, 95% CI 1.14-3.06, p= 0.01] and DFS [HR= 1.75, 95% CI 1.11-2.76, p= 0.02]. Use of myeloablative conditioning with total body irradiation (TBI) was predictive for improved DFS and reduced risk of relapse. Conclusion: Allo-HCT is effective in providing durable remissions and long-term survival in BPDCN. Younger age and allo-HCT in CR1 predicted for improved survival, while myeloablative conditioning with TBI predicted for less relapse and improved DFS. Novel strategies incorporating allo-HCT are needed to further improve outcomes.Copyright © 2023 American Society of Hematology.