如果您正在考虑使用阿尔茨海默氏症药物 Leqembi，需要了解哪些信息

医疗保险将承担疾病早期患者的大部分费用，但 Leqembi 存在安全风险，只能适度减缓认知能力下降。

What to Know if You’re Considering the Alzheimer’s Drug Leqembi

Medicare will cover much of the cost for patients in the early stages of the disease, but Leqembi has safety risks and can only modestly slow cognitive decline.

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周四，美国食品和药物管理局完全批准了 Leqembi 药物用于治疗阿尔茨海默病早期患者，医疗保险表示将承担这种每年 26,500 美元的药物费用的 80%。这两个联邦机构的决定将大大增加获得该药物的机会，但也给患者及其家人带来了困境。

有许多因素需要权衡，包括财务和医疗因素。 以下是一些关键问题的答案：

On Thursday, the Food and Drug Administration gave full approvalto the drug Leqembi for patients who are in the early stages of Alzheimer’s disease, and Medicare said it would cover 80 percent of the cost of the $26,500-per-year medication. The decisions by the two federal agencies will vastly increase access to the drug but also present a dilemma for patients and their families.

There are many factors, both financial and medical, to weigh. Here are answers to some crucial questions:

该药物的效果如何？

Leqembi 不能治愈阿尔茨海默氏症，并且该药物不能改善患者的记忆或认知能力。它也不能阻止疾病恶化。 Leqembi 可以做的是适度减缓处于疾病早期阶段的患者的认知能力下降。 一项大型临床试验的数据表明，该药物可能会在 18 个月的（使用）时间内 使这些患者的病情减缓约 5 个月。

药物对患者日常生活的影响可能会有很大差异。 对于某些人来说，Leqembi 可能意味着病人在额外几个月能够（自己）遵循食谱（做饭）、平衡支票簿（管理家庭收支）、或在没有帮助的情况下完成其他活动。 对于其他人来说，影响可能要微妙得多，几乎不易察觉。

How well does the drug work?

Leqembi is not a cure for Alzheimer’s, and the drug doesn’t improve patients’ memories or cognitive abilities. It also does not stop the disease from getting worse. What Leqembi can do is modestly slow down cognitive decline in patients who are in the early stages of the disease. Data from a large clinical trial suggested that the drug may slow decline by about five months over a period of 18 months for those patients.

How the drug might affect a patient’s daily life is likely to vary widely. For some people, Leqembi might mean several additional months of being able to follow a recipe, balance a checkbook or accomplish other activities without help. For others, the impact might be much more subtle and barely noticeable.

服用有风险吗？

是的。 该药物可导致大脑肿胀或出血，通常是轻度或中度、并可自行消退，但也可能很严重，在极少数情况下可能致命。 FDA 非常担心这些副作用，因此要求在药物标签上添加“黑框警告”（最紧急级别），称该药物可能导致“严重且危及生命的事件”。

风险较高的患者包括那些服用血液稀释剂的患者、那些脑部发生过四次以上微观出血的患者、以及那些患有阿尔茨海默病相关基因突变（称为 APOE4）的患者——尤其是如果他们有两个突变拷贝。 这些病人和他们的医生应该考虑增加的安全风险是否超过他们对可能适度减缓认知能力下降的药物的渴望。

该药物的一项大型临床试验发现，接受 Leqembi 治疗的患者中有近 13% 出现脑肿胀，但大多数病例为轻度或中度。 接受安慰剂的患者中只有不到 2% 出现过这种肿胀。 大多数脑肿胀不会引起任何症状，通常会在几个月内消退。

接受 Leqembi 治疗的患者中约有 17% 出现脑出血，而接受安慰剂治疗的患者中这一比例为 9%。 研究称，脑出血最常见的症状是头晕。

Are there risks from taking it?

Yes. The drug can cause swelling or bleeding in the brain that is often mild or moderate and resolves on its own but can be serious and in very rare cases can be fatal. The F.D.A. was so concerned about these side effects that it is requiring a “black-box warning” — the most urgent level — on the drug’s label, saying that the medication can cause “serious and life-threatening events.”

Patients who are at higher risk include those on blood thinners, those who have had more than four microscopic bleeds in the brain and those with an Alzheimer’s-linked gene mutation called APOE4 — especially if they have two copies of the mutation. They, along with their doctors, should consider whether the increased safety risk outweighs their desire for a medication that might modestly slow cognitive decline.

A large clinical trial of the drug found that nearly 13 percent of patients receiving Leqembi experienced brain swelling, but most of those cases were mild or moderate. Less than 2 percent of patients receiving the placebo experienced such swelling. Most brain swelling did not cause any symptoms and generally resolved within a few months.

About 17 percent of the patients receiving Leqembi experienced brain bleeding, compared with 9 percent of patients receiving the placebo. The most common symptom from brain bleeds was dizziness, the study said.

谁有资格服用 Leqembi？

Leqembi——每两周在医生办公室或诊所通过静脉注射给药——将适用于被诊断患有早期阿尔茨海默氏症的人和患有阿尔茨海默氏症前期疾病（称为轻度认知障碍）的人。 美国约有 150 万人符合这一描述。 另外 500 万患有阿尔茨海默氏症的人将不符合 Leqembi 的资格，因为他们的疾病进展得太严重。

FDA 要求的药物标签指示医生在未进行测试以确认患者具有阿尔茨海默病的特征之一的情况下，不要对患者进行治疗。这些特征是：大脑中淀粉样蛋白的堆积，而Leqembi 会攻击这种蛋白。 淀粉样蛋白水平可以通过 PET 扫描、脊椎穿刺、或新推出的血液检测来评估。

Who is eligible to take Leqembi?

Leqembi — which is administered by intravenous infusions in a doctor’s office or clinic every two weeks — will be available for people diagnosed as having early-stage Alzheimer’s and for those with a pre-Alzheimer’s condition called mild cognitive impairment. About 1.5 million people in the United States fit that description. An additional 5 million who have Alzheimer’s will not qualify for Leqembi because their disease has progressed too far.

The F.D.A.-required label on the drug instructs doctors not to treat patients without testing to confirm that they have one of the hallmarks of Alzheimer’s: a buildup in the brain of the protein amyloid, which Leqembi attacks. Amyloid levels can be assessed with PET scans, spinal taps or newly available blood tests.

需要支付多少钱？

大多数患者都达到了参加医疗保险的年龄，医疗保险表示将支付该药每年 26,500 美元费用的 80%。 患者将需要支付约 6,600 美元的自付款，这可能使许多人无法承担该药物的经济负担。 许多医疗保险患者拥有的补充私人保险政策可能涵盖部分或全部自付费用。

然而，可能还有数万美元的额外费用，包括输液和定期脑部扫描的就诊费用。 一些阿尔茨海默病专家估计，服用 Leqembi 的总费用每年可能高达 90,000 美元左右。 如果覆盖率达到 80%，治疗可能会让患者每年承担 18,000 美元的自付费用。

How much will we have to pay?

Most patients will be old enough for Medicare, which has said it will pay for 80 percent of the $26,500 annual cost of the drug. Patients would be left with about $6,600 in co-payments, which may put the drug out of financial reach for many. Some or all of that co-payment might be covered by the supplemental private insurance policies that many Medicare patients have.

There are potentially tens of thousands of dollars of additional costs, however — including medical visits for the infusions and regular brain scans. Some Alzheimer’s experts have estimated that the total cost of taking Leqembi could run to about $90,000 a year. With 80 percent coverage, treatment could potentially leave patients saddled with $18,000 per year in out-of-pocket costs.

病人及其家人应该如何决定？

和你的医生谈谈。 如果你的医生不熟悉阿尔茨海默氏症的治疗，请考虑与专家交谈。最重要的是与医学专家讨论决定，他们会针对你的具体情况仔细解释风险。

要了解这些风险，请要求进行基因检测以确定您是否患有 APOE4 基因突变。 携带该突变两个拷贝的人（约占阿尔茨海默病患者的 15%）出现脑肿胀和出血的风险特别高。

还有其他因素需要考虑。 每两周去诊所输液会不会造成负担？

而且，重要的是，你和你的家人如何看待你当前的认知状况以及它如何影响你的生活？ 由于 Leqembi 适用于症状较轻的人，因此有些人可能不太愿意冒安全风险，但其他人可能认为尝试一种可以让他们在这个轻微阶段保持更长时间的药物特别重要。

How should people and their families decide?

Talk to your doctor. If your doctor is not well-versed in Alzheimer’s treatments, consider talking with a specialist. The most important thing is to discuss the decision with medical experts who will carefully explain the risks for your specific situation.

To learn those risks, ask for genetic testing to determine if you have the APOE4 gene mutation. People with two copies of that mutation — about 15 percent of Alzheimer’s patients — are at especially high risk of brain swelling and bleeding.

There are other factors to consider too. Would going to a clinic for a drug infusion every two weeks pose a burden?

And, importantly, how do you and your family perceive your current cognitive condition and how it affects your life? Because Leqembi is for people with mild symptoms, some people might be less inclined to take safety risks, but others might consider it especially important to try a drug that might keep them at this mild stage a bit longer.

Pam Belluck is a health and science writer whose honors include sharing a Pulitzer Prize and winning the Victor Cohn Prize for Excellence in Medical Science Reporting. She is the author of “Island Practice,” a book about an unusual doctor. More about Pam Belluck