美国卫生部宣布自闭症三项行动 对乙酰氨基酚将启动安全标签变更
2025年9月22日，美国总统特朗普与美国卫生与公众服务部部长 Robert F. Kennedy Jr. 在首都华盛顿的白宫 Roosevelt 室宣布三项应对自闭症的行动。卫生部称，美国自闭症谱系障碍患病情况自2000年以来上升近400%，目前每31名儿童中就有1名受影响。
第一项行动针对治疗。食品药品管理局当天发布《联邦公报》通知，说明将更新药物 leucovorin 的标签，用于脑叶酸缺乏症，该病症与自闭症有关联。卫生部称，这是首个获食品药品管理局认可、用于脑叶酸缺乏并伴有自闭症症状儿童的治疗路径。标签更新后，各州医疗补助计划可与联邦医疗保险和医疗补助服务中心合作，将该药用于自闭症相关适应症；国立卫生研究院将开展确证性试验与安全性研究。卫生部同时说明，leucovorin 不是自闭症的治愈药物，可能只对一部分儿童的言语相关缺陷有改善作用，须在严密医疗监督下使用。
第二项行动针对对乙酰氨基酚。食品药品管理局将发出医生通知，并启动对乙酰氨基酚（泰诺等同类产品）安全标签变更程序，卫生部将开展全国公共宣传。卫生部称，食品药品管理局此举是回应此前临床与实验室研究提示的可能性：孕期使用对乙酰氨基酚与不良神经发育结局之间可能有关联。卫生部同时写明，也有相反研究显示无关联，孕期未经治疗的发热对母亲与胎儿都有风险；在文献相互冲突、缺乏明确因果证据的情况下，卫生部希望临床医生在孕期确需治疗发热与疼痛时运用最佳判断，使用最低有效剂量与最短疗程，并指出对乙酰氨基酚往往是孕期唯一可用的退热止痛选择，其他替代药物有明确的不良影响记录。
第三项行动是研究投入。国立卫生研究院当天公布「自闭症数据科学计划」入选名单，共13个项目、总金额超过5,000万美元，将综合大规模生物、临床与行为数据，并结合环境、营养、医疗与社会因素研究自闭症成因。
部长 Kennedy 说，家庭长期以来得不到答案与选择，今天的行动打开了首个获认可的治疗路径之门，并把潜在风险告知医生与家庭，政府将遵循科学、恢复信任。食品药品管理局局长 Marty Makary 说，越来越多证据提示部分自闭症儿童存在脑内叶酸缺乏，这一问题可以用 leucovorin 治疗，医生应立即能为合适的儿童使用这一选项。
（来源：美国卫生与公众服务部）
HHS Announces Three Autism Actions as FDA Begins Acetaminophen Safety Label Change
On September 22, 2025, President Donald J. Trump and U.S. Health and Human Services Secretary Robert F. Kennedy Jr. announced three actions on autism from the Roosevelt Room of the White House in Washington. HHS said autism spectrum disorder has surged nearly 400 percent since 2000 and now affects 1 in 31 American children.
First, the Food and Drug Administration published a Federal Register notice outlining a label update for leucovorin for cerebral folate deficiency, which has been associated with autism. HHS said the action establishes the first FDA-recognized therapeutic pathway for children with cerebral folate deficiency and autistic symptoms. After the label update, state Medicaid programs will be able to cover leucovorin for the autism indication in partnership with the Centers for Medicare & Medicaid Services, and the National Institutes of Health will launch confirmatory trials and safety studies. HHS noted that leucovorin is not a cure for autism and may only improve speech-related deficits for a subset of children, and must be given under close medical supervision.
Second, the FDA will issue a physician notice and begin the process to initiate a safety label change for acetaminophen (Tylenol and similar products), and HHS will launch a nationwide public service campaign. HHS said the FDA is responding to prior clinical and laboratory studies that suggest a potential association between acetaminophen use during pregnancy and adverse neurodevelopmental outcomes. HHS also stated that there are contrary studies showing no association, and that untreated fever in pregnancy carries risks for both mother and fetus. Given the conflicting literature and lack of clear causal evidence, HHS said it wants clinicians to exercise their best judgment, prescribing the lowest effective dose for the shortest duration when treatment is required, and noted that acetaminophen is often the only tool for fever and pain in pregnancy, as alternatives such as NSAIDs have well documented adverse effects.
Third, NIH announced the recipients of the Autism Data Science Initiative, funding 13 projects totaling more than $50 million, integrating large-scale biological, clinical and behavioral data with environmental, nutritional, medical and social factors.
Secretary Kennedy said families had been left without answers or options, and that the actions open the door to the first FDA-recognized treatment pathway, inform doctors and families about potential risks, and invest in research. FDA Commissioner Dr. Marty Makary said a growing body of evidence suggests some children with autism are folate deficient within the brain, a problem that can be treated with leucovorin, and that physicians should immediately have the option available for candidate children.
(Source: U.S. Department of Health and Human Services)
来源：美国卫生与公众服务部
发布：GSM·美国（发布链接）
图片来源：图：2025年9月22日，卫生与公众服务部长 Robert F. Kennedy Jr. 在白宫 Roosevelt 室就自闭症行动发表讲话，特朗普在旁。白宫官方照片，公共领域，经 Wikimedia Commons。 / Photo: HHS Secretary Robert F. Kennedy Jr. gives remarks on the autism actions in the Roosevelt Room of the White House on September 22, 2025, with President Donald Trump standing by. Official White House photo, public domain, via Wikimedia Commons.
GSM Global · 本篇英文版 English edition of this story
