[小儿急性淋巴细胞白血病]。

Harefuah Pub Date : 2023-01-01
Sarah Elitzur, Shai Izraeli
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引用次数: 0

摘要

急性淋巴细胞白血病(ALL)是最常见的儿童恶性肿瘤,约占儿童癌症的25%。近几十年来,存活率有了显著提高,从20世纪60年代的约10%提高到今天的90%以上。这一巨大成就是通过协作性随机临床试验、高效、适应风险的多药化疗方案、有效的中枢神经系统预防策略和改进的支持性护理的逐步发展而取得的。最近,我们对ALL的遗传基础的了解已经大大增强,并且已经开发了通过一系列最小残留疾病测量来评估治疗反应的精确方法。某些患者亚群具有遗传上可靶向的病变,如费城阳性ALL,其预后通过联合酪氨酸激酶抑制剂和化疗显着改善,或“费城样”ALL的特定患者亚群。尽管在治疗儿童ALL方面取得了巨大进展,但仍存在重大挑战。化疗的急性副作用可能危及生命,长期副作用往往损害幸存者的生活质量。复发或难治性ALL患者的生存率仍然很低。这导致了新的基于免疫的疗法被引入到复发/难治性B-ALL的治疗中:blinatumomab,一种CD19双特异性t细胞结合剂;inotuzumab-一种cd22免疫毒素和CD19-CAR(嵌合抗原受体)T细胞。与化疗相比,这些方式已证明缓解率提高,毒性降低。在不久的将来,免疫疗法在治疗新诊断和复发患者中的作用将得到更精确的定义。
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[PEDIATRIC ACUTE LYMPHOBLASTIC LEUKEMIA].

Introduction: Acute lymphoblastic leukemia (ALL) is the most common childhood malignancy, constituting approximately 25% of childhood cancers. In recent decades, survival rates have improved dramatically, from approximately 10% in the 1960's to over 90% today. This tremendous achievement has been accomplished through collaborative randomized clinical trials, with progressive evolution of highly efficient, risk-adapted multi-agent chemotherapeutic regimens, effective central nervous system prophylactic strategies and improved supportive care. Recently, our understanding of the genetic basis of ALL has been greatly enhanced, and precise methods for treatment response assessment with serial measurements of minimal residual disease have been developed. Certain patient subgroups have genetically targetable lesions, such as Philadelphia-positive ALL, whose outcomes have been dramatically improved by combined tyrosine kinase inhibitors and chemotherapy, or specific patient subsets of "Philadelphia-like" ALL. Despite the great progress in curing childhood ALL, significant challenges still remain. Acute adverse effects of chemotherapy may be life-threatening, and long-term side effects often impair survivors' quality of life. Survival rates in patients with relapsed or refractory ALL remain poor. This led to the introduction of novel immune-based therapies into the treatment of relapsed/refractory B-ALL: blinatumomab, a CD19 bi-specific T-cell engager; inotuzumab- a CD22-immunotoxin, and CD19-CAR (chimeric antigen receptor) T cells. These modalities have demonstrated improved remission rates with reduced toxicity compared to chemotherapy. The role of immunotherapy in the treatment of newly-diagnosed and relapsed patients will be more precisely defined in the near future.

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