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异基因造血干细胞移植在老年骨髓增生异常综合征患者中的应用未得到充分利用。

Allogeneic Hematopoietic Stem Cell Transplantation Is Underutilized in Older Patients with Myelodysplastic Syndromes.

作者信息

Getta Bartlomiej M, Kishtagari Ashwin, Hilden Patrick, Tallman Martin S, Maloy Molly, Gonzales Patrick, Castro-Malaspina Hugo, Perales Miguel-Angel, Giralt Sergio, Tamari Roni, Klimek Virginia

机构信息

Adult Bone Marrow Transplant Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York.

Department of Medicine, Mount Sinai St. Luke's-West Hospital, New York, New York.

出版信息

Biol Blood Marrow Transplant. 2017 Jul;23(7):1078-1086. doi: 10.1016/j.bbmt.2017.03.020. Epub 2017 Mar 20.

Abstract

Allogeneic hematopoietic stem cell transplantation (HCT) is the only curative treatment for myelodysplastic syndrome (MDS). The proportion of MDS patients referred for transplantation evaluation, those undergoing transplantation, and the reasons for not undergoing transplantation are unknown. In this retrospective analysis, predefined HCT eligibility and indications criteria were applied to 362 unselected patients with newly diagnosed MDS seen by leukemia faculty between 2008 and 2015 at Memorial Sloan Kettering Cancer Center. Two hundred ninety-four patients (81%) were deemed eligible for transplantation and among these, transplantation was considered indicated in 244 (83%). Of these, 158 of 244 (65%) were referred for transplantation evaluation at a median of 3.9 months from diagnosis. Overall 120 of 362 (33%) underwent transplantation at a median of 7.7 months from diagnosis. Metastatic solid-organ malignancy was the major reason for transplantation ineligibility (54%), and death due to MDS, which occurred in 41% of candidates who did not undergo transplantation, was the major reason for not undergoing transplantation. Factors associated with a lower likelihood of referral for transplantation evaluation included age ≥65 (P < .001), ≥2 comorbidities (P = .008), intermediate-1/low risk MDS (P < .001), <5% blasts at diagnosis (overall P < .001), having Medicare/Medicaid health insurance (P < .001), not being married (P = .017), and diagnosis between 2008 and 2011 (P = .035). On multivariate analysis adjusting for all of the previous factors, diagnosis between 2008 and 2011 (P < .001), age ≥65 (P = .001), and <5% blasts at diagnosis (overall P = .031) were associated with a lower likelihood of referral for transplantation evaluation. Factors associated with a lower likelihood of undergoing transplantation included age ≥65 (P < .001), ≥2 comorbidities (P = .003), intermediate-1/low risk MDS (P < .001), <5% blasts (overall P < .001), very low/low/intermediate risk International Prognostic Scoring System-revised karyotype (P = .018), and having Medicare/Medicaid health insurance (P < .001). In multivariate analysis adjusting for all of the previous factors, age ≥65 (P = .021), presence of ≥2 comorbidities (P = .018), and <5% blasts (overall P = .011) were associated with a lower likelihood of undergoing transplantation. The results highlight that transplantation for MDS remains underutilized, particularly for candidates over the age of 65.

摘要

异基因造血干细胞移植(HCT)是骨髓增生异常综合征(MDS)唯一的治愈性治疗方法。转诊进行移植评估的MDS患者比例、接受移植的患者比例以及未接受移植的原因尚不清楚。在这项回顾性分析中,将预先定义的HCT合格标准和适应症标准应用于2008年至2015年期间在纪念斯隆凯特琳癌症中心由白血病科医生诊治的362例未经挑选的新诊断MDS患者。294例患者(81%)被认为符合移植条件,其中244例(83%)被认为有移植指征。在这些患者中,244例中的158例(65%)在诊断后中位3.9个月时被转诊进行移植评估。总体而言,362例中的120例(33%)在诊断后中位7.7个月时接受了移植。转移性实体器官恶性肿瘤是不符合移植条件的主要原因(54%),未接受移植的候选者中有41%因MDS死亡,这是未接受移植的主要原因。与转诊进行移植评估可能性较低相关的因素包括年龄≥65岁(P<0.001)、≥2种合并症(P = 0.008)、中危1/低危MDS(P<0.001)、诊断时原始细胞<5%(总体P<0.001)、拥有医疗保险/医疗补助健康保险(P<0.001)、未婚(P = 0.017)以及2008年至2011年期间诊断(P = 0.035)。在对所有上述因素进行调整的多因素分析中,2008年至2011年期间诊断(P<0.001)、年龄≥65岁(P = 0.001)以及诊断时原始细胞<5%(总体P = 0.031)与转诊进行移植评估的可能性较低相关。与接受移植可能性较低相关的因素包括年龄≥65岁(P<0.001)、≥2种合并症(P = 0.003)、中危1/低危MDS(P<0.001)、原始细胞<5%(总体P<0.001)、极低危/低危/中危国际预后评分系统修订核型(P = 0.018)以及拥有医疗保险/医疗补助健康保险(P<0.001)。在对所有上述因素进行调整的多因素分析中,年龄≥65岁(P = 0.021)、存在≥2种合并症(P = 0.018)以及原始细胞<5%(总体P = 0.011)与接受移植的可能性较低相关。结果表明,MDS的移植治疗仍未得到充分利用,尤其是65岁以上的候选者。

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