1. 概述

血液肿瘤是指来源于造血细胞的恶性疾病，可累及骨髓、血液及全身各个脏器和组织。临床常见的血液肿瘤主要包括各种类型的白血病、多发性骨髓瘤、淋巴瘤、骨髓增生异常综合征、骨髓增殖性肿瘤等，其发病率和死亡率均居前列，是严重危害人类健康的重大疾病。近年来，血液肿瘤在精准诊断、预后分层、免疫治疗和靶向治疗等方面取得了长足进展，尤其是嵌合抗原受体T细胞免疫疗法，在国内飞速发展，展现出了令人鼓舞的疗效和安全性，有助于改善患者的生存并提高生活质量。本文针对2024年度血液肿瘤领域中优秀的临床与基础研究进行概述，对学科十大前沿进展进行了总结，并展望了血液肿瘤的未来学科发展方向。

4. 本学科发展趋势与对策

4.1. 未来5年发展的战略需求

血液肿瘤领域在未来5年内的发展战略需求包括加强精准医疗、深化免疫疗法研究和促进转化医学的发展。通过在这些关键领域的努力，我们有望进一步提高血液肿瘤的治疗效果，为患者提供更好的治疗方案。精准医疗是指根据个体患者的基因组信息和其他相关因素，为其量身定制的医疗方案。在血液肿瘤领域，精准医疗可以帮助医生更好地预测患者的病情发展和治疗效果，从而选择最佳的治疗方案。未来5年内，我们需要进一步加强对血液肿瘤相关基因的研究，建立更完善的基因组数据库，并将精准医疗应用到临床实践中。在血液肿瘤领域，免疫疗法已经取得了一些重要的突破，如CAR-T细胞疗法。然而，目前的免疫疗法仍然存在一些限制，如治疗效果的不稳定性和副作用的发生。我们需要进一步研究免疫疗法的机制，寻找更有效的治疗靶点，并改进治疗方案，以提高治疗效果和减少副作用。在血液肿瘤领域，转化医学的发展可以帮助我们更好地理解疾病的发生机制，发现新的治疗靶点，并加速新药的研发和临床试验，我们需要加强基础研究与临床实践的紧密合作，促进转化医学的发展，并加强对新药的评估和监管，以确保其安全性和有效性。

近年来，新的免疫药物、分子靶向药物及新的技术不断涌现，它们将基础科学前沿最新研究成果和新型工程技术相结合，进一步推进创新型治疗进展。但由于创新型治疗相当一部分源自欧美国家，往往价格高昂，很难广泛惠及中国患者，因此需要大力支持一系列国产原研创新型治疗的研发上市，同时加强支付体系的创新、以及医保政策、医院考核政策等的优化，多端发力支持创新治疗的发展应用。

4.2. 未来5年重点发展方向

血液肿瘤未来5年的重点发展方向包括分子生物学研究、免疫治疗和精准医疗。通过这些研究的不断深入，相信将有更多的突破和进展，为血液肿瘤的早期诊断和个体化治疗提供更好的方法和手段。

（1）分子生物学研究是血液肿瘤研究的重要方向之一。通过对血液肿瘤相关基因的研究，可以深入了解疾病的发生机制和进展过程，为疾病的早期诊断和治疗提供依据。未来5年，分子生物学研究将继续深入，重点关注血液肿瘤相关基因的突变、表达和调控机制，以及与肿瘤发展相关的信号通路和分子靶点的发现。

（2）近年来，免疫治疗在血液肿瘤的巨大成功引发了我们对系统性免疫治疗方案的深入思考。未来5年，免疫治疗领域将继续成为血液肿瘤治疗的重要发展方向之一。研究人员将进一步研究免疫治疗的机制，发展更有效的免疫治疗方法，并探索免疫治疗与其他治疗手段的联合应用，以提高治疗效果。其中，最重要的是优化免疫治疗的方案，构建最优的整体治疗策略。包括了选择合适的联合使用策略，减少肿瘤负荷的同时维持患者免疫储备，协同激活抗肿瘤免疫治疗反应的同时减少毒副反应，维持持久疗效的同时减少患者经济负担，进而建立优化的整体治疗方案。而这些工作都有赖于我们将免疫治疗机制探索与临床试验验证相结合，在具备充分理论依据的基础上逐步优化治疗方案，建立整体化、系统性的治疗方案，为血液肿瘤患者提供更好的治疗选择。

（3）精准医疗是一种根据患者的个体差异，为其提供个性化的预防、诊断和治疗方案的方法。在血液肿瘤的治疗中，精准医疗已经得到了广泛应用。未来5年，精准医疗将继续成为血液肿瘤治疗的重要方向之一。研究人员将通过对患者基因组、转录组和蛋白组的研究，发现与血液肿瘤相关的分子标志物，并开发相应的靶向治疗方法，以实现对患者的精准治疗。

4.3. 未来5年发展对策

（1）加强早期筛查和预防。早期筛查和预防是降低血液肿瘤发病率和死亡率的重要手段。在未来5年，我们应该加强对高风险人群的筛查和监测，例如家族遗传史、染色体异常等。同时，开展相关的健康教育，提高公众对血液肿瘤的认知，推广健康生活方式，如戒烟、戒酒、合理膳食等，以降低患病风险。

（2）推广精准医疗。精准医疗是未来血液肿瘤治疗的重要方向。通过基因测序和分子诊断技术，可以更好地了解患者的疾病特征，为个体化治疗提供依据。我们应该加大对精准医疗的研究和应用，不断优化治疗方案，提高疗效和安全性。同时，加强医疗机构之间的协作和信息共享，推动精准医疗的普及和应用。

（3）加强科研和创新。科研和创新是血液肿瘤未来发展的重要驱动力。我们要加大对血液肿瘤的基础和临床研究投入，推动新技术、新药物的研发和应用。同时，加强学术交流和合作，促进科研成果的转化。此外，加强对血液肿瘤发病机制的研究，寻找新的治疗靶点和策略，为患者提供更有效的治疗手段。

【主编】

王建祥中国医学科学院血液病医院

李建勇南京医科大学第一附属医院、江苏省人民医院

邱录贵中国医学科学院血液病医院

纪春岩山东大学齐鲁医院

【副主编】

魏辉中国医学科学院血液病医院

王迎中国医学科学院血液病医院

徐卫南京医科大学第一附属医院、江苏省人民医院

朱华渊南京医科大学第一附属医院、江苏省人民医院

安刚中国医学科学院血液病医院

易树华中国医学科学院血液病医院

叶静静山东大学齐鲁医院

章静茹山东大学齐鲁医院

【编委】（按姓氏拼音排序）

纪敏山东大学齐鲁医院

李艳中国医学科学院血液病医院

卢菲山东大学齐鲁医院

缪祎南京医科大学第一附属医院、江苏省人民医院

邱少伟中国医学科学院血液病医院

王婷玉中国医学科学院血液病医院

夏奕南京医科大学第一附属医院、江苏省人民医院

徐燕中国医学科学院血液病医院

★

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