麻豆传媒

Primary biliary cholangitis (PBC) can deliver a significant blow to patients' quality of life, especially if they are not appropriately diagnosed and treated.

"PBC is one of those liver diseases where you can have cirrhosis and liver failure if you don't do anything about it in a matter of 10 to 15 years," said Lucy Mathew, NP, of Cedars Sinai in Los Angeles. "Now, if you do diagnose early and if you treat them correctly and treat them optimally, then you can slow down the progression to the point that I've had people in their 70s or even 80s with PBC who are doing very well."

Early diagnosis is challenging when many patients present without symptoms, which means a clinician needs to have tested a patient's alkaline phosphatase and noticed its elevation to refer them to a hepatologist.

"The two scenarios [that should raise clinical suspicion] are if you have an elevated alkaline phosphatase that persists for 6 months or more, and the second thing, of course, would be itching," David N. Assis, MD, of Yale Liver Clinics and Yale New Haven Hospital in New Haven, Connecticut, said. "Anybody with itching should have their liver tests checked. I think that's a good public service announcement."

PBC is most common among middle-age women, but prompt diagnosis becomes even more important with younger patients, suggested Ehud Zigmond, MD, director of the Center of Liver Diseases at Sheba Medical Center in Israel.

"When you get the disease at a very young age, the disease is more aggressive," Zigmond said. "For a 20-year-old, I would really push for complete normalization of all liver enzymes," as opposed to settling for the standard treatment response marker of having alkaline phosphatase levels at less than 1.67 times the upper limit of normal. "When you treat a 20-year-old or a 70-year-old, we have to remember that for a 20-year-old, you have to keep the liver healthy for another 80 years," he added.

Mathew agreed, noting that "the more normalization of alkaline phosphatase you can get, the better." The new arrival of additional medications beyond ursodeoxycholic acid (UDCA), the first-line treatment, to reduce alkaline phosphatase gives clinicians more options in achieving that normalization.

Robert S. Brown Jr., MD, MPH, chief of the division of gastroenterology and hepatology at Weill Cornell Medicine in New York City, said UDCA can take up to a year to get maximal benefit, but "as more data have pushed to say the goal should be normalized," he considers adding another of the recently available agents if a patient's alkaline phosphatase hasn't normalized by then.

However, research on quality-of-life outcomes in patients with PBC suggests that effectively managing the condition requires more than simply watching lab values. Studies have shown that symptoms play a substantial role in patients' quality of life, yet are never even asked about their symptoms.

Driving home the importance of asking patients about symptoms, comments from patients living with PBC the negative impact of fatigue on their lives, as well as the value of having a supportive physician or medical team.

Quality-of-Life Research in PBC Patients

One of the to assess quality of life in patients with PBC included 276 British patients, mostly women, who had worse scores on overall quality of life compared with controls (P<0.001), driven by lower energy levels (P<0.0001) and emotional reactions (P<0.005). However, patients who had been treated with UDCA for at least 6 months had better energy scores (P<0.001) and emotional reactions (P<0.002) versus those not taking UDCA or taking it for less than 6 months.

Notably, patients' quality of life was associated with individual symptoms, but not with liver labs, bilirubin levels, stage of disease, or even duration of disease. Rather, worse overall quality of life was linked to pruritus (P<0.02), jaundice (P<0.01), fatigue (P<0.0001), splenomegaly (P<0.001), and osteopenia (P<0.0001).

Little had changed for British patients with PBC by of 2,240 patients similarly found fatigue to negatively affect quality of life. Again, patients treated with UDCA reported a better quality of life, though that finding was interestingly independent of their response to it. Most importantly, however, patients who underwent transplant reported a substantially lower quality of life, leading the authors to emphasize "the importance of timely, effective treatment to reduce the need for transplant."

While fatigue appears to have the greatest influence on quality of life, itching can also play a big role. A found that 81% of patients reported pruritus, and the 37% reporting clinically significant pruritus had worse overall quality of life. Though patients with clinically significant itch were more likely to get treatment for their itch, a third had never received treatment for it. The authors noted that these findings "highlight the pervasive impact of pruritus in patients with PBC, as well as some shortcomings of the medical community's current response."

Other recent research suggested that patients' quality of life may be affected by cultural or geographic factors. A looked at quality of life for 515 PBC patients (90.5% women) in the U.K., Spain, Japan, and Italy, finding worse fatigue among British patients after controlling for age and clinical factors compared with patients in the other countries (P<0.05). British and Japanese patients also had worse itching than Spanish and Italian patients.

Patient Support

Patients have said that connecting with an online PBC group, such as the and the American Liver Foundation's , can offer support and information.

"Recognition and early treatment are critical to trying to get patients' quality of life improved, and there are very good patient advocacy groups for those that have PBC," said Brett E. Fortune, MD, medical director of the liver transplant program at Montefiore Medical Center in New York City. "It's not a death sentence. It's definitely treatable."

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