Lee Catherine J, Kim Soyoung, Tecca Heather R, Bo-Subait Stephanie, Phelan Rachel, Brazauskas Ruta, Buchbinder David, Hamilton Betty K, Battiwalla Minoo, Majhail Navneet S, Lazarus Hillard M, Shaw Peter J, Marks David I, Litzow Mark R, Chhabra Saurabh, Inamoto Yoshihiro, DeFilipp Zachariah, Hildebrandt Gerhard C, Olsson Richard F, Kasow Kimberly A, Liesveld Jane L, Rotz Seth J, Badawy Sherif M, Bhatt Neel S, Yared Jean A, Page Kristin M, Arellano Martha L, Kent Michael, Farhadfar Nosha, Seo Sachiko, Hematti Peiman, Freytes César O, Rovó Alicia, Ganguly Siddhartha, Nathan Sunita, Burns Linda, Shaw Bronwen E, Muffly Lori S
Utah Blood and Marrow Transplant Program, Huntsman Cancer Institute, University of Utah, Salt Lake City, UT.
Center for International Blood and Marrow Transplant Research (CIBMTR), Department of Medicine, and.
Blood Adv. 2020 Mar 24;4(6):983-992. doi: 10.1182/bloodadvances.2019001126.
There is marked paucity of data regarding late effects in adolescents and young adults (AYAs) who undergo myeloablative conditioning (MAC) allogeneic hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML). We evaluated late effects and survival in 826 1-year disease-free survivors of MAC HCT for AYA AML, with an additional focus on comparing late effects based upon MAC type (total body irradiation [TBI] vs high-dose chemotherapy only). The estimated 10-year cumulative incidence of subsequent neoplasms was 4% (95% confidence interval [CI], 2%-6%); 10-year cumulative incidence of nonmalignant late effects included gonadal dysfunction (10%; 95% CI, 8%-13%), cataracts (10%; 95% CI, 7%-13%), avascular necrosis (8%; 95% CI, 5%-10%), diabetes mellitus (5%; 95% CI, 3%-7%), and hypothyroidism (3%; 95% CI, 2%-5%). Receipt of TBI was independently associated with a higher risk of cataracts only (hazard ratio [HR], 4.98; P < .0001) whereas chronic graft-versus-host disease (cGVHD) was associated with an increased risk of cataracts (HR, 3.22; P = .0006), avascular necrosis (HR, 2.49; P = .006), and diabetes mellitus (HR, 3.36; P = .03). Estimated 10-year overall survival and leukemia-free survival were 73% and 70%, respectively, and did not differ on the basis of conditioning type. In conclusion, late effects among survivors of MAC HCT for AYA AML are frequent and are more closely linked to cGVHD than type of conditioning.
关于接受清髓性预处理（MAC）的异基因造血细胞移植（HCT）治疗急性髓系白血病（AML）的青少年和青年（AYA）患者的远期效应，相关数据极为匮乏。我们评估了826例接受MAC HCT治疗AYA AML且无病生存1年的患者的远期效应和生存率，并特别关注基于MAC类型（全身照射[TBI]与单纯高剂量化疗）比较远期效应。后续肿瘤的估计10年累积发病率为4%（95%置信区间[CI]，2%-6%）；非恶性远期效应的10年累积发病率包括性腺功能障碍（10%；95% CI，8%-13%）、白内障（10%；95% CI，7%-13%）、无血管性坏死（8%；95% CI，5%-10%）、糖尿病（5%；95% CI，3%-7%）和甲状腺功能减退（3%；95% CI，2%-5%）。仅接受TBI独立与白内障风险较高相关（风险比[HR]，4.98；P <.0001），而慢性移植物抗宿主病（cGVHD）与白内障风险增加（HR，3.22；P =.0006）、无血管性坏死（HR，2.49；P =.006）和糖尿病（HR，3.36；P =.03）相关。估计10年总生存率和无白血病生存率分别为73%和70%，且在预处理类型方面无差异。总之，接受MAC HCT治疗AYA AML的幸存者中远期效应常见，且与cGVHD的关联比预处理类型更为密切。
