Clinical Pearls for the Management of ALGS


Experts in pediatric hepatology share advice for the optimal management of Alagille syndrome.

William F. Balistreri, MD: Our listeners include gastroenterologists, hepatologists, and other health care personnel. What advice would you give them? Obviously, recognize the disease, screen for the disease, and then treat the disease. Let’s start with Phil. Any words of advice for our listeners?

Philip Rosenthal, MD: IBAT inhibitors are approved, and we should consider utilizing them earlier in the therapy of individuals who have significant cholestasis and itching in particular. That’s what they’re approved for. As we get more familiar with the use of these drugs and we get more data, we’ll have a better understanding of who the appropriate candidates are. I’m hopeful that there will be much more data looking at the genetic differences of these individuals and whether we might be able to tweak who the ideal candidate would be to receive these drugs.

William F. Balistreri, MD: We’ll get some guidelines from the pediatric GI [gastrointestinal] society [North American Society for Pediatric Gastroenterology, Hepatology and Nutrition; NASPGHAN], AASLD [American Association for the Study of Liver Diseases], and so on. Jennifer, any parting comments or words of advice for our listeners?

Jennifer M. Vittorio, MD: Before we talk about some of the management, which we have reviewed, the first thing I’d bring up and encourage people to look for is the symptoms themselves. Sometimes, if you’re sitting in the office, you have to be not only direct but sometimes creative in the way you ask these questions. If you simply ask, “Is your child itchy?” you may miss something. Sometimes, rather than asking whether they’re itchy, I focus on something like how they’re sleeping, whether they’re waking up at night, what’s causing that. In some of the more severe cases, parents will tell you that when they wake up in the morning, they notice that their child has been scratching so much that there’s blood on the sheets or the pillow case and things like that.

We’ve alluded how itchiness is subjective. Are there more objective ways that we could be measuring this? There are some tools out there. But you need the wherewithal to know that pruritus is a known complication of Alagille syndrome and to make sure you’re asking the appropriate screening questions in a way that encourages response. That’s the first step. Then management-wise, we’re going to have a lot of more interesting conversations in the months to come. Don’t be afraid to utilize a lot of your colleagues who may be seeing these patients. As hepatologists, we sometimes forget that we often see several of these patients. Depending on where you’re practicing, that may or may not be the case, but the resources are out there.

William F. Balistreri, MD: Thank you, Jennifer. Ryan, do you have any words of wisdom?

Ryan T. Fischer, MD: These are great. I’m probably the least wise person in any of these conferences, but that gives me a great opportunity to listen and learn. That’s the big thing that I apply to this Alagille population. These children are so rewarding. We love the ones we get to take care of. They’re some of the sweetest ones. It can break your heart with the type of itching they go through and when you see excoriations on them. They can crack you up when they come in for a clinic visit. They’re wonderfully complex. I recommend that when you think about Alagille, you embrace the entirety of it. Trying to be a medical home for them is key, but with that comes great responsibility. You have to manage a lot of spinning plates and be aware of a lot of things that could happen. But at the same time, it’s so worth it. That’s my big takeaway.

William F. Balistreri, MD: Amen. Embrace the challenge. Maintain that holy curiosity. It can be extraordinarily rewarding when we have success. Reggie, final words?

Regino P. Gonzalez-Peralta, MD: I’d summarize it in 3 bullet points, the famous NASPGHAN message point. First, Alagille is a chronic multisystem disease that may be difficult to treat and is associated with complications. Second, families need to understand that we’re here to advocate for them, and as the captains of the ship, we should be most able to help them navigate the journey. Third, the future is bright with the development of these new IBAT inhibitors to treat the very disturbing symptom of pruritus and perhaps avoid the progression of liver disease.

William F. Balistreri, MD: Well stated, Reggie. Thank you. This has been a terrific discussion. The 1 conclusion I’d also draw is that we need to get together again in 6 or 8 months to see how this field is evolving. It’s moving that rapidly. I sincerely want to thank all of you for this very informative discussion and thank all of you who are watching this HCPLive® Peer Exchange. If you enjoyed this content, please subscribe to the e-newsletters to receive upcoming news about other Peer Exchanges. Hopefully we can all get together again. Thank you all.

Transcript Edited for Clarity

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