Allogeneic hematopoietic stem cell transplantation in myelofibrosis:: the 20-year experience of the Gruppo Italiano Trapianto di Midollo Osseo (GITMO)
Allogeneic hematopoietic stem cell transplantation in myelofibrosis:: the 20-year experience of the Gruppo Italiano Trapianto di Midollo Osseo (GITMO)
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DOI：
10.3324/haematol.12828
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发表时间：
2008-10-01
期刊：
影响因子：
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通讯作者：
Papineschi, Federico
中科院分区：
文献类型：
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作者：
Patriarca, Francesca;Bacigalupo, Andrea;Papineschi, Federico
背景异基因造血干细胞移植是治疗骨髓纤维化的一种潜在的治疗方法，尽管它的应用受到移植相关死亡率高的限制。在这项研究中，我们评估了骨髓纤维化患者进行异基因干细胞移植的结果，和预后factors.Design和MethodsOne百例患者在1986年和2006年之间的26个意大利中心移植的影响。我们通过单变量和多变量分析分析了患者的特征和干细胞移植前疾病的临床特征以及移植程序对移植相关死亡率、总生存率和无复发生存率的影响。结果干细胞移植时患者的中位年龄为49岁（范围，21-68）和90%的人有一个中等或高Dupriez评分。48%的患者接受了清髓性预处理方案，78%的患者接受了来自匹配同胞供体的干细胞。移植后第90天的累计植活率为87%（95%CI，0.87-0.97）。移植相关死亡的1年和3年累积发生率分别为35%和43%。干细胞移植后估计的3年总体和无复发生存率分别为42%和35%。在多变量分析中，移植相关死亡率的负预测因素是1995年之前的干细胞移植年份、无关供体和诊断与移植之间的长时间间隔。接受外周血干细胞而不是骨髓作为移植物来源的患者的总体和无复发生存期有延长的趋势（分别为p=0.070和p=0.077）。强度的预处理方案（清髓与降低强度方案）没有显着影响outcome.ConclusionsWe的结论是，骨髓纤维化患者进行异基因干细胞移植的结果显着改善后，1996年由于移植相关的死亡率降低。我们观察到，移植相关死亡率的降低与选择匹配的同胞供体相关，而较长的总生存期与使用外周血作为干细胞来源相关。
BackgroundAllogeneic stem cell transplantation is a potentially curative treatment for myelofibrosis, although its use is limited by a high rate of transplant-related mortality. In this study, we evaluated the outcome of patients with myelofibrosis who underwent allogeneic stem cell transplantation, and the impact of prognostic factors.Design and MethodsOne hundred patients were transplanted in 26 Italian centers between 1986 and 2006. We analyzed the influence of the patients' characteristics and the clnical features of their disease before stem cell transplantation and of transplant procedures on transplant-related mortality, overall survival, and relapse-free survival by means of univariate and multivariate analyses.ResultsThe median age of the patients at the time of stem cell transplantation was 49 years (range, 21-68) and 90% of them had an intermediate or high Dupriez score. Forty-eight percent received a myeloablative conditioning regimen and 78% received stem cells from matched sibling donors. The cumulative incidence of engraftment at day 90 after transplant was 87% (95% CI, 0.87-0.97). The cumulative 1-year and 3-year incidences of transplant-related mortality were 35% and 43%, respectively. The estimated 3-year overall and relapse-free survival rates after stem cell transplantation were 42% and 35%, respectively. In multivariate analysis, negative predictors of transplant-related mortality were year of stem cell transplantation before 1995, unrelated donor, and a long interval between diagnosis and transplantation. There was a trend towards longer overall and relapse-free survival in patients receiving peripheral blood stem cells rather than bone marrow as the source of their graft (p=0.070 and p=0.077, respectively). The intensity of the conditioning regimen (myeloablative versus reduced intensity regimens) did not significantly influence the outcome.ConclusionsWe conclude that the outcome of myelofibrosis patients who underwent allogeneic stem cell transplantation significantly improved after 1996 due to the reduction in transplant-related mortality. We observed that a reduction in transplant-related mortality was associated with the choice of a matched sibling donor, whereas longer overall survival was associated with the use of peripheral blood as the source of stem cells.