Improving Revised International Prognostic Scoring System Pre-Allogeneic Stem Cell Transplantation Does Not Translate Into Better Post-Transplantation Outcomes for Patients with Myelodysplastic Syndromes: A Single-Center Experience
Improving Revised International Prognostic Scoring System Pre-Allogeneic Stem Cell Transplantation Does Not Translate Into Better Post-Transplantation Outcomes for Patients with Myelodysplastic Syndromes: A Single-Center Experience
复制标题
DOI：
10.1016/j.bbmt.2018.02.007
复制
发表时间：
2018-06-01
影响因子：
4.3
通讯作者：
Narayanan, Sujaatha
中科院分区：
文献类型：
--
作者：
Alzahrani, Musa;Power, Maryse;Narayanan, Sujaatha
骨髓增生异常综合征（MDS）患者的自然病史是可变的。修订的国际预后评分（IPSS-R）在实践中通常用于预测MDS患者在诊断时和造血干细胞移植（HSCT）前的结局。然而，目前尚不清楚在异基因HSCT前使用化疗或低甲基化剂的IPSS-R变化对移植后结局的影响。我们评估了HSCT前IPSS-R预后评分的改善是否会导致HSCT后临床结局的改善。次要目标包括研究预后因素对移植后存活率的影响。纳入了1997年2月至2013年4月期间在不列颠哥伦比亚省白血病/BMT项目中接受同种异体HSCT的所有MDS患者。从程序数据库中审查相关信息。根据MDS诊断时和HSCT前的数据计算IPSS-R。将HSCT前IPSS-R改善的患者的结局与IPSS-R稳定或恶化的患者进行比较。使用Kaplan-Meier方法估计总生存期（OS）和无事件生存期（EFS），使用对数秩检验确定P值。使用多变量考克斯比例风险回归模型计算风险比，以研究预后变量对OS和EFS的影响。共纳入138例连续患者。其中62例患者（45%）IPSS-R改善，23例（17%）恶化，41例（30%）保持稳定，12例（9%）未知。改善组、恶化组和稳定组的OS无统计学差异（分别为30%、22%和40%; P= 0.63）。5年时复发和非复发死亡率的累积发生率分别为28.4%（95%置信区间[CI]，21.1 - 36.1）和31.6%（95% CI，23.8 - 39.7）。原始细胞20%的患者复发率为23%（P = .0004）。整个队列的OS为34%，EFS为33%。HSCT前接受清髓性预处理和非清髓性预处理的患者的结局无显著差异（OS分别为34%和39%; P= 0.63，EFS分别为34%和32%; P= 0.86）。IPSS-R改善、恶化或稳定患者的OS无统计学差异。在多变量分析中，只有3个因素与OS相关：诊断时的细胞遗传学风险组，移植时的原始细胞计数，以及是否存在慢性移植物抗宿主病。HSCT前改善IPSS-R并不能转化为更好的生存结局。移植前原始细胞计数对移植后结果具有高度预测性。(C)2018年美国血液和骨髓移植协会。
The natural history of patients with myelodysplastic syndromes (MDS) is variable. The Revised International Prognostic Score (IPSS-R) is commonly used in practice to predict outcomes in patients with MDS at both diagnosis and before hematopoietic stem cell transplantation (HSCT). However, the effect of change in the IPSS-R before allogeneic HSCT with chemotherapy or hypomethylating agents on post-transplantation outcomes is currently unknown. We assessed whether improvement in IPSS-R prognostic score pre-HSCT would result in improvement in clinical outcomes post-HSCT. Secondary goals included studying the effect of prognostic factors on post-transplantation survival. All patients with MDS who underwent allogeneic HSCT at the Leukemia/BMT Program of British Columbia between February 1997 and April 2013 were included. Pertinent information was reviewed from the program database. IPSS-R was calculated based on data from the time of MDS diagnosis and before HSCT Outcomes of patients who had improved IPSS-R pre-HSCT were compared with those with stable or worse IPSS-R. Overall survival (OS) and event-free survival (EFS) were estimated using the Kaplan-Meier method, with P values determined using the log-rank test. Hazard ratios were calculated using multivariable Cox proportional hazards regression models to study the effects of the prognostic variables on OS and EFS. A total of 138 consecutive patients were included. IPSS-R improved in 62 of these patients (45%), worsened in 23 (17%), remained stable in 41 (30%), and was unknown in 12 (9%). OS was not statistically different across the improved, worsened, and stable groups (30% versus 22% versus 40%, respectively; P= .63). The cumulative incidences of relapse and nonrelapse mortality at 5 years were 28.4% (95% confidence interval [CI], 21.1 to 36.1) and 31.6% (95% CI, 23.8 to 39.7), respectively. The rate of relapse was 23% in patients with 20% blasts (P = .0004). In the entire cohort OS was 34% and EFS was 33%. There was no significant difference in outcomes between patients who received myeloablative conditioning and those who received nonmyeloablative conditioning before HSCT (OS, 34% and 39%, respectively; P= .63 and EFS, 34% and 32%, respectively; P= .86). OS was not statistically different among patients with improved, worsened, or stable IPSS-R. On multivariate analysis, only 3 factors were associated with OS: cytogenetic risk group at diagnosis, blast count at transplantation, and the presence or absence of chronic graft-versus-host disease. Improving IPSS-R before HSCT does not translate into better survival outcomes. Blast count pretransplantation was highly predictive of post-transplantation outcomes. (C) 2018 American Society for Blood and Marrow Transplantation.