Luca Pagliaro, Sai-Juan Chen, Daniel Herranz, Cristina Mecucci, Christine J Harrison, Charles G Mullighan, Ming Zhang, Zhu Chen, Nicolas Boissel, Stuart S Winter, Giovanni Roti
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引用次数: 0
摘要
急性淋巴细胞白血病(ALL)是一种以未成熟淋巴细胞失控增殖为特征的血液恶性肿瘤。过去几十年来,人们在了解急性淋巴细胞白血病的生物学特性方面取得了重大进展,从而在诊断、治疗和监测方面取得了显著进步。自化疗出现以来,ALL 已成为检验适用于一般癌症的创新方法的平台。例如,全息医学的出现使人们对 ALL 的分子和遗传特征有了更深入的了解。基因组剖析技术的创新确定了驱动 ALL 的特定基因改变和突变,激发了新疗法的灵感。酪氨酸激酶抑制剂和免疫疗法等靶向药物已在亚组患者中显示出良好疗效,同时将不良反应降至最低。此外,嵌合抗原受体T细胞疗法的开发是ALL治疗领域的一大突破,它能带来显著的疗效,并有可能长期缓解病情。进步不仅限于治疗方式。可测量的残留疾病监测和体内外药物反应谱筛查可以更早地发现疾病复发并识别特殊反应者,从而使临床医生能够针对不同患者调整治疗策略。数十年的支持性和预防性护理改善了治疗相关并发症的管理,提高了 ALL 患者的生活质量。
Acute lymphoblastic leukaemia (ALL) is a haematological malignancy characterized by the uncontrolled proliferation of immature lymphoid cells. Over past decades, significant progress has been made in understanding the biology of ALL, resulting in remarkable improvements in its diagnosis, treatment and monitoring. Since the advent of chemotherapy, ALL has been the platform to test for innovative approaches applicable to cancer in general. For example, the advent of omics medicine has led to a deeper understanding of the molecular and genetic features that underpin ALL. Innovations in genomic profiling techniques have identified specific genetic alterations and mutations that drive ALL, inspiring new therapies. Targeted agents, such as tyrosine kinase inhibitors and immunotherapies, have shown promising results in subgroups of patients while minimizing adverse effects. Furthermore, the development of chimeric antigen receptor T cell therapy represents a breakthrough in ALL treatment, resulting in remarkable responses and potential long-term remissions. Advances are not limited to treatment modalities alone. Measurable residual disease monitoring and ex vivo drug response profiling screening have provided earlier detection of disease relapse and identification of exceptional responders, enabling clinicians to adjust treatment strategies for individual patients. Decades of supportive and prophylactic care have improved the management of treatment-related complications, enhancing the quality of life for patients with ALL.
期刊介绍:
Nature Reviews Disease Primers, a part of the Nature Reviews journal portfolio, features sections on epidemiology, mechanisms, diagnosis, management, and patient quality of life. The editorial team commissions top researchers — comprising basic scientists and clinical researchers — to write the Primers, which are designed for use by early career researchers, medical students and principal investigators. Each Primer concludes with an Outlook section, highlighting future research directions. Covered medical specialties include Cardiology, Dermatology, Ear, Nose and Throat, Emergency Medicine, Endocrinology, Gastroenterology, Genetic Conditions, Gynaecology and Obstetrics, Hepatology, Haematology, Infectious Diseases, Maxillofacial and Oral Medicine, Nephrology, Neurology, Nutrition, Oncology, Ophthalmology, Orthopaedics, Psychiatry, Respiratory Medicine, Rheumatology, Sleep Medicine, and Urology.