Ribera Josep-María, Oriol Albert, Bethencourt Concepción, Parody Ricardo, Hernández-Rivas Jesús-María, Moreno María-José, del Potro Eloy, Torm Mar, Rivas Concepción, Besalduch Joan, Sanz Miguel-Angel, Ortega Juan-José
Servicio de Hematología Clínica, Institut Català d'Oncologia-Hospital Universitari Germans Trias i Pujol, C/ Canyet S/N, 08916 Badalona, Spain.
Haematologica. 2005 Oct;90(10):1346-56.
The optimal post-remission therapy for adults with high-risk acute lymphoblastic leukemia (ALL) is not well established. This multicenter randomized trial by the Spanish PETHEMA Group was addressed to compare three options of post-remission therapy in adults with high-risk ALL: chemotherapy, allogeneic stem cell transplantation (SCT) and autologous SCT.
A total of 222 valid high-risk ALL patients entered the trial. All received a standard five-drug/five-week induction course. Patients in complete remission with an HLA-identical family donor were assigned to allogeneic SCT (n=84) and the remaining were randomized to autologous SCT (n=50) or to delayed intensification followed by maintenance chemotherapy up to 2 years in complete remission (n=48).
Overall, 183 patients achieved complete remission (82%). With a median follow-up of 70 months, the median disease-free survival and overall survival were 17 and 23 months, respectively. The 5-year disease-free survival and overall survival were 35% (95% CI, 30%-41%) and 34% (95% CI, 28%-39%), respectively. Patients allocated to the chemotherapy, allogeneic and autologous SCT were comparable in the main pre-treatment ALL characteristics and the rate of response to therapy. Intention-to-treat analysis showed no differences between patients according to whether they had or did not have a donor in disease-free survival (39%, 95% CI 30-48% vs. 33%, 95% CI 23-41%) and overall survival (44%, 95% CI 35-52% vs. 35%, 95% CI 25-44%), as well as for autologous SCT vs. chemotherapy comparisons (disease-free survival: 40%, 95% CI 28-52% vs. 51%, 95% CI 37-67%; overall survival: 43%, 95% CI 29-58% vs. 52%, 95% CI 39-65%). No differences were observed when the analysis was made on the basis of the treatment actually performed.
This study failed to prove that, when a family donor is available, allogeneic SCT produces a better outcome than autologous SCT or chemotherapy in adults with high-risk ALL.
高危成人急性淋巴细胞白血病（ALL）缓解后的最佳治疗方案尚未明确。西班牙PETHEMA组开展的这项多中心随机试验旨在比较高危成人ALL缓解后治疗的三种方案：化疗、异基因干细胞移植（SCT）和自体SCT。
共有222例有效的高危ALL患者进入试验。所有患者均接受了标准的五药/五周诱导疗程。有HLA配型相合的家族供者且完全缓解的患者被分配接受异基因SCT（n = 84），其余患者被随机分配接受自体SCT（n = 50）或延迟强化治疗，随后进行维持化疗直至完全缓解2年（n = 48）。
总体而言，183例患者实现完全缓解（82%）。中位随访70个月，无病生存期和总生存期的中位数分别为17个月和23个月。5年无病生存率和总生存率分别为35%（95%CI，30% - 41%）和34%（95%CI，28% - 39%）。分配至化疗、异基因和自体SCT组的患者在主要的ALL预处理特征和治疗反应率方面具有可比性。意向性分析显示，无论有无供者，患者在无病生存期（39%，95%CI 30 - 48%对33%，95%CI 23 - 41%）和总生存期（44%，95%CI 35 - 52%对35%，95%CI 25 - 44%）方面无差异，自体SCT与化疗比较也无差异（无病生存期：40%，95%CI 28 - 52%对51%，95%CI 37 - 67%；总生存期：43%，95%CI 29 - 58%对52%，95%CI 39 - 65%）。基于实际进行的治疗进行分析时未观察到差异。
本研究未能证明，在有家族供者的情况下，高危成人ALL患者接受异基因SCT比自体SCT或化疗能产生更好的疗效。
