The importance of listening to people with primary biliary cholangitis
Ingo van Thiel is a patient representative for a German organisation for people with liver diseases, Deutsche Leberhilfe e.V. (German Liver Aid). He explains the enormous value of listening carefully to what the PBC community says about its unmet needs—even a single word can have the power to improve patients’ lives
“We can never completely understand what it means to be in a patient’s skin. But I think sometimes, standing between doctors and patients, we get close. Because we listen.”
In his 23 years of experience as a patient advisor and representative, Ingo has seen how a patient organisation’s broad perspective of a disease gives insight into the diversity of experiences that patients with liver disease have. “Patients tell us more than they sometimes tell their doctors, because a doctor’s time is limited. Speaking with doctors and with a lot of patients saves us from thinking that everybody feels the exact same thing and has the exact same problems.
“There are overlaps, definitely, in people’s experiences. Fatigue or itch are huge problems in PBC, but even those problems may not be identical—they may feel different from patient to patient. And they may cause different problems because the patient has a different job or different social surroundings.”
Patients may sometimes believe, mistakenly, that what has happened to them will also happen in the exact same way to everybody else with the same diagnosis, Ingo says. This can be unhelpful if, for example, a patient tells another, “I lost my job, so you will lose yours too”, or says, “I have this side effect, so you will have it too”. At times, such misunderstandings can undermine the valuable support that patients can—and very much do—give each other. Ingo believes that the best approach for patients, and for everybody involved in their care, is to look beyond their own experience and be aware that the same diagnosis may look or feel different for other patients.
“Talking with as many people as possible—patients with other patients, patients with patient representatives, with doctors, also with industry—is key, because the more people talk with each other, the more they communicate, the broader the horizon becomes for everyone.”
There is no better example of the communicative approach Ingo describes than the initiative to change the name of PBC from primary biliary cirrhosis to primary biliary cholangitis. The change was to just a single word but it has made a world of difference to the patient community.
The initiative for change was led by patient organisations who had seen the negative effects that came from describing the condition as “primary biliary cirrhosis”. Cirrhosis is the end stage of PBC but it is not present in the beginning and can often be avoided by timely treatment. However, patients were hearing that word and focusing on it, not understanding that cirrhosis might happen if they did not have treatment for the condition. In Germany, Leberhilfe pushed hard for doctors to always stress that point, but still, when patients heard the word, sometimes it was “like a black curtain came down” so that they did not process further explanations from their doctor. Many left their appointment thinking that they were going to die, Ingo says.
“I told one woman that having PBC did not have to mean she had cirrhosis and asked if her liver had been checked for it. I’d had this moment in many helpline calls, and usually the reaction was surprise and relief. Not here. She was so angry! She said, ‘I thought I was a dead woman walking for seven years. Now, I hear for the first time that I might not even be that sick. Why has nobody told me?’”
Doctors were well aware of the problem but there was never any agreement on a better name. Also, while most patients hated the cirrhosis word, the PBC abbreviation was popular and well known. A breakthrough came during an international meeting in London in 2014, when Ingo cited a doctor’s idea (which he had heard years before), to exchange the word “cirrhosis” for “cholestasis” or “cholangitis”. While this idea was not new at all and had been previously disregarded, as Ingo recalls, this time its merits were fully understood. The approach was enthusiastically embraced by an influential doctor at the meeting, creating the opportunity for change.
Robert Mitchell-Thain of the PBC Foundation quickly created an online survey for patients, patient representatives and doctors to collect the community’s views. Patients were asked about their feelings towards the name of PBC, whether they had experienced stigma because of it (assumptions about drinking, for example), the impact it had on getting life insurance and the difficulties it caused when explaining the condition to others.
“Within a few weeks he had more than 700 responses,” Ingo says—an incredible outcome given that PBC is a rare disease. “There was a huge majority saying ‘Change the name. Fix that one word. Do it!’”
Robert and Ingo painstakingly prepared a presentation to pitch the proposal at a major conference on PBC. After an “interesting but quite dry scientific session” it was their turn to present the case for changing the name. When Robert began to speak, “you could have heard a pin drop”, Ingo recounts. “Robert told the story of his mother (a PBC patient and the founder of the PBC Foundation). He brought in humour. He brought in data from the survey. He brought in emotion.”
Robert’s powerful speech had a strong effect on the audience. The attendees were persuaded not to keep looking for a perfect name (in scientific terms) and to accept a small change that would make a real difference to patients. Within a year, the name change had been approved and the disease was officially renamed “primary biliary cholangitis”. At a stroke, the conversations around the disease began to change, especially during a patient’s initial diagnosis.
The change of name was a remarkable success story, Ingo says. There are, however, many unmet needs that remain for people with PBC. He hopes that in time these will be resolved through the continued cooperative efforts of patients, patient representatives, doctors, scientists and industry.
First, while treatment options for PBC have improved, more needs to be done to improve their efficacy, Ingo says. “The basic medicine for PBC (ursodeoxycholic acid, UDCA) has been around since the 1980s. This works fine for most patients, but some studies say that up to a third of patients may not benefit enough from it.” He also notes that patients sometimes experience gastrointestinal issues as a side effect.
Second, he hopes one day to see the development of a treatment to address the cause of PBC—which is still unknown. “We know a lot about what happens in PBC, but the big why question, is still, I believe, open for PBC and all autoimmune diseases. Getting to the root of that could be a huge game-changer.”
Third, he says that another big challenge is turning the tide in how symptoms are managed in PBC. “We have a few dedicated doctors and specialists who are really into symptom treatment, but that approach is only slowly seeping into the larger community of hepatologists.” He believes that doctors may focus on test results and may not heed what the patient says they are actually experiencing in their daily lives. In his experience, patients can feel like a “train wreck” even when their liver values are fine.
In PBC, symptoms such as fatigue and itch may appear long before the liver is seriously damaged, Ingo says. “Some symptoms get even worse with advanced disease, but you can have all that trouble while your liver is still looking pretty good. Many people feel exhausted in an inexplicable way that their relatives or co-workers don’t understand”.
“Full-blown PBC fatigue basically makes you unable to function. It’s a huge challenge. It’s also a social issue, because fatigue does not really show and not all hepatologists take the symptom burden equally seriously.”
Middle-aged women make up a large part of PBC patients and, unfortunately, when they experience such a non-specific symptom, they sometimes encounter prejudice from doctors—even some PBC specialists. “It’s usually male doctors mansplaining to female PBC patients that their symptoms are either not real or due to menopause and not caused by PBC,” Ingo says. “I wish these doctors would allow themselves to be enlightened more about the symptom burden.”
In reality, the fatigue people feel in PBC is completely different to everyday tiredness, Ingo explains. “One hepatologist told me about a woman who went shopping and came home with her bags full of frozen food. She said, ‘I came home and I was so tired, I just wanted to sit down on the sofa for a moment. I woke up hours later and everything was thawed.’”
Fatigue may be “just as bad” after a liver transplant, Ingo says. “Men, especially, may suffer from even worse fatigue afterwards. You would hope for some of your old energy to return after a liver transplant, but not so much, or not at all, in PBC. Liver transplant can completely resolve the itch caused by PBC, but fatigue continues even after the old liver has gone.”
Although it may not yet be possible to identify the root cause of a patient’s fatigue, it is sometimes possible to alleviate it, Ingo says. Sometimes improvement comes through trying different things, rather than through a one-size-fits-all treatment or approach.
For example, a patient with fatigue may be “itching like hell, which could keep them awake at night”. Different medications could be tried to alleviate the itch, Ingo explains, which might improve the person’s sleep and therefore improve their fatigue a little. Other patients with fatigue might benefit from suggestions about nutrition or hydration or advice about other health problems. “You sometimes hear a patient’s tone of voice change when you go through these things, because even though there is no general fix, there are at least options they can try. They can actively do something for themselves.”
Ingo believes that taking care of the mind through self-empowerment strategies is an important approach that can help patients. “It can help patients deal with their very real symptom burden better. That doesn’t mean the symptoms are imagined, but when you know you can do something, it can change your attitude and give you back a feeling of control.”
Itch can present an enormous challenge for patients, and sometimes there is no medication that will improve it, Ingo says. In extreme cases, people might even require a liver transplant to resolve it.
“Itch can be experienced as even worse than pain, especially when none of the medications work. Itch can make people scratch themselves until they bleed—even make people suicidal in extreme cases.”
There are unseen consequences to PBC that can have far-reaching implications for patients’ personal relationships, Ingo explains. Sjögren’s syndrome, which may affect patients with PBC, can cause dry eyes, dry mouth and vaginal dryness too. “The impact on people’s sex life is something which is really in the shadows but is probably a much bigger thing than is talked about. If your mucous membranes are dry, that can also affect intimate parts and make a lot of things very painful, so it can affect partnerships.”
Similarly, fatigue and brain-fog can interfere with a patient’s sex-life and relationships, as can itching. “If you’re scratching yourself so hard that your whole skin is looking raw or bloody, that can also create a lot of shame and a feeling of not wanting to show your body, even to your partner.” Sometimes, people may just withdraw from their partners. In such cases, sometimes people don’t realise that the changes in their sexual behaviour are because of their disease, and they find other reasons to explain the situation, such as “It is what it is” or “Maybe I just don’t feel it like I used to”, Ingo explains.
“The fact that PBC may cause problems on a sexual level should be proactively communicated by patient organisations and doctors, as a part of the information package and in a non-interfering way,” Ingo says. “And if patients have questions, they should be given further helpful suggestions about how to address these issues, be it offering medical help for dry mucosa, advising partnership counselling for better communication or finding alternative ways of intimacy they might like to try in their relationship.
“Waiting for patients to cross their own shame barriers and openly address potential sexual issues is probably not the right approach. I think this is something where patient organisations could possibly be more, let’s say, discreetly proactive.”
“These issues around shame and relationships are probably a hugely underestimated area of unmet need.”
The social burden
“Of the thousands of PBC patients I’ve talked with in the last 23 years, I recall only two or three who did not complain of being wrongly accused of having alcohol problems.”
In Ingo’s experience, a major obstacle for patients looking for a diagnosis is that the signs of the disease may be wrongly understood as the result of drinking too much alcohol. “That is a challenge because alcohol problems affect a huge community of people and their reaction of protest can be the same as that of someone who really does not drink, so it’s not always easy for a doctor to tell,” he says. “Still, there are a lot of doctors who know how to ask and look further instead of pigeon-holing the patient as somebody who is drinking too much.”
Stigma is another obstacle that people with PBC encounter. “People assume that PBC patients must have an alcohol problem, or they think of viral hepatitis (and they have no idea what that is either) and say, ‘Oh, you must have a drug problem.’”
“People assume you’ve done something wrong, so that’s why you have a liver problem.”
Deutsche Leberhilfe e.V. is a German patient organisation with about 1,600 paying members who have various liver diseases. Founded by liver patients in 1987, it has grown into a professional patient organisation that is nationally and internationally active. Its focus is on raising awareness of liver diseases, providing patients with accessible and accurate information, improving patient–doctor relationships and representing patients’ interests at a political level.
It offers support to patients over the phone, through its website and by email. It also provides free brochures and leaflets about different topics related to liver disease and publishes a quarterly members’ magazine with in-depth coverage of relevant developments. Its areas of interest include a wide range of frequent and rare liver diseases, including primary biliary cholangitis (PBC).
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