Division of Haematology, Mayo Clinic, Rochester, MN, USA.
National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Lancet Haematol. 2022 May;9(5):e374-e384. doi: 10.1016/S2352-3026(22)00045-X.
Remarkable improvements in outcomes for many haematological malignancies have been driven primarily by a proliferation of novel therapeutics over the past two decades. Targeted agents, immune and cellular therapies, and combination regimens have adverse event profiles distinct from conventional finite cytotoxic chemotherapies. In 2018, a Commission comprising patient advocates, clinicians, clinical investigators, regulators, biostatisticians, and pharmacists representing a broad range of academic and clinical cancer expertise examined issues of adverse event evaluation in the context of both newer and existing therapies for haematological cancers. The Commission proposed immediate actions and long-term solutions in the current processes in adverse event assessment, patient-reported outcomes in haematological malignancies, toxicities in cellular therapies, long-term toxicity and survivorship in haematological malignancies, issues in regulatory approval from an international perspective, and toxicity reporting in haematological malignancies and the real-world setting. In this follow-up report, the Commission describes progress that has been made in these areas since the initial report.
在过去的二十年中，新型治疗药物的大量涌现主要推动了许多血液恶性肿瘤治疗效果的显著改善。靶向药物、免疫和细胞疗法以及联合治疗方案具有与传统有限细胞毒性化疗不同的不良事件特征。2018 年，一个由患者权益倡导者、临床医生、临床研究人员、监管机构、生物统计学家和药剂师组成的委员会，代表广泛的学术和临床癌症专业知识，检查了血液癌症新疗法和现有疗法背景下不良事件评估的问题。委员会在当前的不良事件评估、血液恶性肿瘤患者报告结果、细胞疗法毒性、血液恶性肿瘤长期毒性和生存、国际视角下的监管批准问题以及血液恶性肿瘤和真实世界环境中的毒性报告等方面提出了即刻行动和长期解决方案。在本后续报告中，委员会描述了自最初报告以来在这些领域取得的进展。
