Leukemia/Bone Marrow Transplantation Program of British Columbia, Division of Hematology, Department of Internal Medicine, University of British Columbia, Vancouver, British Columbia, Canada.
Leuk Lymphoma. 2012 Jul;53(7):1321-6. doi: 10.3109/10428194.2011.652108. Epub 2012 Mar 5.
Sudden blast phase (SBP) is a rare event that occurs in an unpredictable fashion amongst patients with chronic myeloid leukemia (CML) who otherwise appear to be responding satisfactorily to imatinib (IM) treatment. We investigated the incidence, clinical characteristics, treatment outcome and long-term follow-up of 213 patients with chronic phase CML treated with IM according to the European LeukemiaNet guidelines. Nine patients, eight of whom received IM as first-line therapy, developed SBP (4.2% of the total). They tended to have low or intermediate risk Sokal scores at diagnosis, a predominance of the lymphoid phenotype and a short interval from "optimal" response to the development of BP. Five of the nine patients with SBP are alive in complete molecular remission; however, all of them underwent allogeneic hematopoietic stem cell transplant. The cumulative incidence of SBP for the patients who received IM as first-line therapy was 5.9% and the 2-year overall survival of the nine patients who developed SBP was 56%. Despite the improved outcome for patients with SBP receiving tyrosine kinase inhibitors (TKIs) and transplant, many of these patients are not salvaged with these therapies. This illustrates the need to develop predictive models to identify patients early whose response to TKI therapy will not be durable and hopefully prevent the transformation to advanced disease.
急变期（SBP）是一种罕见事件，在接受伊马替尼（IM）治疗的慢性髓性白血病（CML）患者中以不可预测的方式发生，这些患者在其他方面似乎对治疗反应良好。我们根据欧洲白血病网络指南调查了 213 例接受 IM 治疗的慢性期 CML 患者的发生率、临床特征、治疗结果和长期随访情况。9 例患者（占总数的 4.2%）发生了 SBP，其中 8 例患者在一线接受了 IM 治疗。他们在诊断时的 Sokal 评分往往较低或中等，以淋巴样表型为主，从“最佳”反应到 BP 发展的时间间隔较短。9 例 SBP 患者中有 5 例处于完全分子缓解，但均接受了异基因造血干细胞移植。一线接受 IM 治疗的患者中 SBP 的累积发生率为 5.9%，发生 SBP 的 9 例患者的 2 年总生存率为 56%。尽管接受酪氨酸激酶抑制剂（TKI）和移植的 SBP 患者的预后得到了改善，但这些患者中的许多人无法通过这些治疗挽救。这表明需要开发预测模型，以便早期识别出对 TKI 治疗反应不会持久的患者，并希望预防疾病进展。
