2026年6月27日 / 美国东部时间上午8:00 / 哥伦比亚广播公司新闻（CBS News）
作者：克里··布林 新闻编辑
克里··布林是CBSNews.com的新闻编辑。她毕业于纽约大学亚瑟·L·卡特新闻学院，此前曾在NBC新闻的《今日数字》（TODAY Digital）工作。她的报道领域包括时事、突发新闻以及药物使用相关议题。
查看完整简历
当米歇尔·威廉姆斯最初出现高血压症状时，她并未太在意。
她带着两个年幼的孩子，新冠疫情正肆虐，而她和丈夫正在翻新最近买下的一栋农舍。压力导致血压升高似乎合情合理。她的医生建议做一些轻微的生活方式调整，但并未表现出警觉。
其他症状则缓慢恶化。威廉姆斯的脚肿得厉害，不得不换穿更大尺码的鞋子。她感到疲惫不堪，越来越频繁的如厕次数让她无法安睡。她的血压持续升高，脚踝开始肿胀，随后蔓延至腿部。最终，她的医生将她转诊给了肾脏科医生。
威廉姆斯表示，2023年6月的那次就诊并不顺利。她打印并带去了自己的病历，但接诊的医师助理对此毫无兴趣。这位助理建议她努力控制血压，但完全不愿了解血压居高不下的原因，威廉姆斯说。她被告知六个月后再来复诊。
“我回到车里时，心情极度低落。肯定是哪里出了问题，我就是没被认真对待，”威廉姆斯说，“我真的觉得等不了六个月。”
米歇尔·威廉姆斯与丈夫和孩子。 米歇尔·威廉姆斯 摄
“请帮帮我”
2023年7月，威廉姆斯主动转诊至克利夫兰诊所——她的岳父曾在此接受过肾脏移植手术。她提交了自己的医疗记录，并附上一封“近乎恳切的信，写着‘请帮帮我，肯定哪里不对劲’”。几周后，她被安排了为期两天的全套检查。
完成一连串检查后，威廉姆斯和丈夫驱车两小时回家。刚开进自家车道，她的电话就响了。接诊的肾脏科医生对她的血液检查结果感到担忧，希望为她做骨髓和肾脏活检。
“那时候真的很吓人，对吧？”威廉姆斯说，“所以我们收拾了几样东西，立刻掉头回去了。”
接下来的24小时内，威廉姆斯完成了两项活检。她还回答了数百个问题，帮助医生排除可能的诊断。活检恢复后，她回到了家中。
检查显示她的尿液和骨髓存在异常，最终被诊断为多发性骨髓瘤——一种可能引发肾脏问题的血癌。在近六个月的时间里，她接受了化疗和免疫治疗。但威廉姆斯的症状依旧没有缓解。
“这让我不禁思考：如果我们正在治疗根本病因，但她却没有好转，那我们还在关注什么，又遗漏了什么？”克利夫兰诊所的肾脏科医生贾格米特·丁格拉说道，“她没有达到我们预期的治疗反应，这让我很不安。所以我们继续排查。”
米歇尔·威廉姆斯与丈夫。 米歇尔·威廉姆斯 摄
什么是IC-MPGN？
第二轮活检显示，威廉姆斯的骨髓健康，但肾脏中堆积了复杂的免疫沉积物。在排除所有其他可能性后，丁格拉最终确诊威廉姆斯患有IC-MPGN——一种与免疫系统相关的肾脏疾病。
爱荷华大学的肾脏科医生、研究员兼教授卡拉·内斯特表示，这种疾病是“最罕见的”肾脏病症之一。未参与威廉姆斯治疗的内斯特估计，每百万人口中仅有1至4人被诊断出IC-MPGN。
内斯特称，大多数IC-MPGN病例的病因不明。患者最常出现的症状包括肿胀和疲劳，正如威廉姆斯的经历。她说，大多数患者直到“症状负担相当严重”时才会得到确诊。
在威廉姆斯被确诊时，尚无针对IC-MPGN的特效治疗方法。内斯特表示，直到2025年，标准治疗方案包括类固醇、免疫抑制剂甚至化疗。这些治疗能缓解部分症状，但无法延缓疾病进展。患者被告知，从确诊IC-MPGN到发展为终末期肾病，大约还有10年时间。即便接受肾脏移植也只是推迟不可避免的结果，因为根本病因并未得到治疗。
“我们没能解决问题。坦白说，预后非常糟糕，”内斯特说。
在临床试验中找到“新生”
威廉姆斯感到走投无路。丁格拉开了多种药物，但要么毫无效果，要么让她身体不适。
“有很长一段时间，我整个人都昏昏沉沉的，”威廉姆斯回忆道，“明明知道可选方案不多，但我还是觉得必须坚持下去。”
2024年11月，她接到了一个意外电话。丁格拉了解到佩塞他卡普（pegcetacoplan）——一种仍在临床试验阶段的药物，显示出治疗IC-MPGN的真正潜力。威廉姆斯有些紧张，但还是同意加入试验。她于2025年2月入组。情况很快出现转机。
“很快我们就看到了变化。我又变回了那个精力充沛的自己。感觉重获了自由，”威廉姆斯说。
六个月后，该药物成为首款获美国食品药品监督管理局（FDA）批准用于治疗IC-MPGN的药物。这款注射剂商品名为Empaveli，丁格拉称其为“患者的游戏规则改变者”。
“我们突然就能停止使用所有那些乱七八糟的其他疗法了。我们终于可以通过靶向治疗让患者达到缓解，而且……不再使用那些原本效果就不佳的药物，”内斯特说道，她补充道，该药物已获FDA批准用于12岁以上的成人和儿童患者。
服用该药物一年多后，威廉姆斯的状况显著好转，丁格拉说。她现在仍需每两个月复诊一次进行监测，但如果病情保持稳定，复诊间隔会逐渐拉长，丁格拉表示。日常生活也恢复了常态：肿胀消失了，服用的药物也大幅减少。
“我不再纠结于吃什么、能不能睡够觉了，”威廉姆斯说，“这真的是一场变革。我终于又找回了自己。”
编辑：妮可·布朗·周
A mom “didn’t feel taken seriously” even as her symptoms worsened. Then tests found a rare disease.
June 27, 2026 / 8:00 AM EDT / CBS News
By Kerry Breen News Editor
Kerry Breen is a news editor at CBSNews.com. A graduate of New York University’s Arthur L. Carter School of Journalism, she previously worked at NBC News’ TODAY Digital. She covers current events, breaking news and issues including substance use.
Read Full Bio
When Michelle Williams started experiencing high blood pressure, she didn’t pay it much attention.
She had two small kids, the COVID-19 pandemic was raging, and she and her husband were in the middle of renovating a farmhouse they had recently purchased. It made sense stress would spike her blood pressure. Her doctor recommended some minor lifestyle changes, but didn’t seem alarmed.
Other symptoms escalated slowly. Williams’ feet became so swollen she had to buy larger shoes. She was fatigued, but more and more frequent bathroom visits kept her from sleeping. Her blood pressure kept rising. Her ankles started swelling, then her legs. Finally, her doctor referred her to a nephrologist, or kidney specialist.
That visit, in June 2023, didn’t go well, Williams said. The physician assistant she saw did not show interest in the medical records she had printed out and brought, she said. The PA advised her to work to lower her blood pressure, but showed no interest in understanding why it was so high, Williams said. She was told to return in six months.
“I got to my car, and I was just depressed. Something was wrong. I didn’t feel taken seriously,” Williams said. “I didn’t really feel like I had six months to wait.”
Michelle Williams with her husband and children. Michelle Williams
“Please help me”
In July 2023, Williams referred herself to the Cleveland Clinic, where her father-in-law had once undergone a kidney transplant. She sent over her medical documentation, along with a “kind of impassioned letter about ‘Please help me, something’s wrong.’” Several weeks later, she was scheduled for two full days of appointments.
After a barrage of exams, Williams and her husband made the two-hour drive home. As they pulled into their driveway, her phone rang. The nephrologist she had seen was alarmed by her blood work, and wanted to biopsy her bone marrow and kidney.
“That was kind of scary, right?” Williams said. “So we gathered up a few things and turned around and went back.”
Williams had both biopsies done over the next 24 hours. She also answered hundreds of questions meant to help doctors eliminate possible diagnoses. After recovering from the biopsies, she returned home.
Tests showed abnormalities in her urine and bone marrow, leading to a diagnosis of multiple myeloma, a form of blood cancer than can cause kidney problems. For nearly six months, she underwent chemotherapy and immunotherapy. Still, Williams’ symptoms persisted.
“It really got me thinking: If we are treating the underlying cause and she’s not improving, then what else are we looking at and what else are we missing?” said Dr. Jagmeet Dhingra, a nephrologist at the Cleveland Clinic. “It didn’t sit right that she wasn’t showing the response we wanted to see. So we kept looking.”
Michelle Williams and her husband. Michelle Williams
What is IC‑MPGN?
Another round of biopsies showed Williams’ bone marrow was healthy, but complex immune deposits were building up in her kidneys. After ruling out all other possibilities, Dhingra finally diagnosed Williams with IC‑MPGN, a kidney disease related to the immune system.
The disease is “one of the rarest” kidney conditions, said Dr. Carla Nester, a nephrologist, researcher and professor at the University of Iowa. Nester, who was not involved in Williams’ care, estimated that only about one to four people per million are diagnosed with IC-MPGN.
In most cases, IC-MPGN has no known cause, Nester said. Patients most often notice symptoms like swelling and fatigue, as Williams did. Most people don’t get diagnosed until they have “quite a significant burden of symptoms,” she said.
At the time of Williams’ diagnosis, there was no specific treatment for IC-MPGN available. Nester said that until 2025, standard treatment included steroids, immunosuppressants and even chemotherapy. They helped some symptoms, but didn’t slow the progression of the disease, Nester said. Patients were told they had about 10 years between diagnosis with IC-MPGN and end-stage kidney disease. Even a kidney transplant only delayed the inevitable, because the underlying condition wasn’t being treated.
“We were not fixing the problem. It was a horrible prognosis, frankly,” Nester said.
Finding “freedom” in a clinical trial
Williams felt trapped. Dhingra was prescribing multiple medications, but they either made no difference or left her feeling unwell.
“For quite a while, I was just in a kind of fog,” Williams recalled. “Knowing there wasn’t a lot of options, it just felt like I had to make it work.”
Then she received a surprising call in November 2024. Dhingra had learned about pegcetacoplan, a medication still in clinical trials, that was showing potential as a real treatment for IC-MPGN. Williams was nervous, but agreed to join the trial. She was enrolled in February 2025. Things took a turn immediately.
“Right away, we saw change. I was back to my feisty self. I felt like I had freedom back,” Williams said.
Six months later, it became the first medication to be approved by the FDA for the treatment of IC-MPGN. The injectable medication is sold under the brand name Empaveli and is a “game-changer for patients,” Dhingra said.
“We were suddenly able to stop using all that other crazy stuff we were doing. We were suddenly able to put patients in remission on targeted therapy, and … stop using the other things that weren’t working well anyway,” said Nester, who noted the medication is FDA-approved for adults and children over the age of 12.
After over a year on the medication, Williams is doing significantly better, Dhingra said. She still sees him every other month for monitoring, but those visits will become more spaced out if her condition stays stable, Dhingra said. Day-to-day life is much more normal: Her swelling is gone and she is on far less medication.
“I don’t have that paranoia over me anymore about what I eat, and getting enough sleep,” Williams said. “It’s kind of been a game changer. I just feel back to myself again.”
Edited by Nicole Brown Chau
发表回复