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One rare diagnosis
Two extraordinary stories
Featuring Emmy and Abby

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Young adults living with cholestatic pruritus (itch) in Alagille syndrome

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These are Emma’s and Abby’s stories
Not all patients taking LIVMARLI, and their caregivers, will have the same experiences.

Emma:

I’m Emma. I’m 19, and I’m from Washington State. I was diagnosed with Alagille syndrome very soon after birth. I had the symptoms of jaundice, prominent facial features, and low bile duct count. When I was about four or five years old, I realized I had Alagille syndrome. That meant for me that I had a lifelong illness that I had to manage.

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My cholestatic pruritus (itch)
Emma

Emma:

One of the main symptoms for Alagille syndrome is cholestatic pruritus. My itch was pretty severe. It’s like bugs under your skin almost. It’s just something you constantly have to soothe and you just can never soothe it. Daily life for me would be very disrupted. I would have to itch before I even went into public. I managed the itch by scratching, or if it was a lot, I would take ice baths and lotion stuff, but they’re temporary relief. It was hard to maintain friendships, relationships with people along with schooling. It was very hard to focus because my main focus was just the itching. It’s very sensory, overwhelming.

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Normalizing cholestatic pruritus (itch)

Emma:

The itching just became my everyday life. No matter how severe it was or how bad it was that was just my life. I just lived with it. Looking back, I’m like, that was not normal. I got on the clinical trial for LIVMARLI when I was 12. I’m 19 now, so that was seven years. I’ve been taking it every day since then.

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Meet Abby

Abby:

I’m Abby. I’m 26 years old and I’m from Huntsville, Alabama. I was 11 months old when I was diagnosed with Alagille syndrome. The symptoms that led me to being diagnosed was I was itching from the time that I was able to start to itch. I was not growing, and I was jaundiced.I believe at the age of 10 and 11, I started to understand what Alagille was and what it meant for the rest of my life. I knew that I was always going to have something wrong with my liver, and I knew that I was always going to have to go to doctor’s appointments and take medication, and those are just some of the symptoms that stood out to me.

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My cholestatic pruritus (itch)
Abby

Abby:

My itching would start bright and early in the morning and progressively get worse throughout the day. Some of the medications, physicians were offering me were cholestyramine and hydroxyzine. They had me on rifampin at one point, and none of those helped. Then at around almost two years old, I had a biliary diversion and I lived with that for my entire life, until I was 19. Even with the diversion, the itching was still there. I still scratched my feet on thecarpet.

The palms of my hands would itch. My legs would itch, so I would still have to try to use other methods to help my itching. It was very difficult to go to school and have social interactions and do things. As a kid with the pruritus, you feel very alone a lot of the time.

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Normalizing cholestatic pruritis (itch)

Abby:

Looking back, the pruritus has always been normalized for me. It was just something I had to live with.

I have a really good support system, always have, but I wish I had physicians advocating a little bit more for me because the pruritus is a real thing and it is very debilitating. It is awful.

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Speaking up about cholestatic pruritus (itch)

Abby:

I encourage anyone that your pruritus affects your day-to-day life, and that you have normalized to talk to your physician and let them know how it affects you and see where you can go from there.

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Growing up with cholestatic pruritus (itch)
Emma and Abby

Emma:

Hello.

Abby:

Hi.

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Mirum brought Emma and Abby together to discuss living with cholestatic pruritus (itch) in Alagille syndrome.

This was a rare opportunity for 2 patients to connect on both the shared and unique parts of their experiences.

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Living day to day with cholestatic pruritus (Itch)

Emma:

At what point did you realize how bad the itching was? Did you find that out or someone else had to point that out?

Abby:

I mean, I always knew it was bad. I always knew it was bad. It was no secret. I feel like whenmy husband and I went to a doctor’s appointment maybe two years back, and he asked the doctor if there was anything that would help with the itching, I think that’s really when it was like, “Oh, OK. It really is like that noticeable and that bad.”

Emma:

Yeah. I feel the same way. I knew it was bad, but I think it was me seeing the reactions of other people. We’d go in public, my mom would get so strict. She’s like, “Stop itching. Stop itching.” She’d whisper it to me. I’m like, “I didn’t even know I was doing it.” Or, at school, I remember I liked this one boy, and I’d be talking to him. He’s like, “What are you doing?” I find out I’m doing this the whole time, while I’m talking to him.

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Early attempts to treat cholestatic pruritus (itch)

Emma:

At what point did the itching get so bad that you’re like, “I need to get something like treatment?”

Abby:

Well, my parents made that decision for me early in life, and I had the diversion done, but then when I was 19, I got it reversed. After the reversal,I knew that it was going to be tough because even going from one thing to another, your body has to have time to adjust. I tried several different things, like the allergy medications you can take. Nothing helped me sleep all the way through the night if I was having a bad day. I think I remember we used an anti-itch lotion, and I don’t remember it helping at all.

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Treatment with LIVMARLI
Emma and Abby

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LIVMARLI is an FDA-approved medicine for cholestatic pruritus (itch) patients with Alagille syndrome who are 3 months of age and older.

Abby:

How did you hear about LIVMARLI? How old were you? How did that all come about?

Emma:

I was about like fifth grade. My mom’s always been a big advocate, so she’s always on the hunt for something. She found about LIVMARLI. I got in the clinical trial. I was in that for about a month before I got on the medicine. It only took a week or two, and I started seeing results. What about you? How did you hear about LIVMARLI?

Abby:

A physician reached out to me to educate me about LIVMARLI, and then the physician got in contact with my doctor after I said I was willing to do it and then I was able to start on it. I’m very glad he reached out to me. If he didn’t, I don’t think I would be in the place I am today. I would still be itching all the time.

Emma:

I don’t know what my life would be like. I don’t really want to go back to the itching.

Abby:

That’s how I feel too. I feel like I lived my whole life with it, so I don’t want to go back.

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Noticing improvements in cholestatic pruritus (itch)

Emma:

When I started taking LIVMARLI, for the first couple weeks, I noticed myself I didn’t need as much time itching, or I could focus in class. That was a big one, where I wasn’t constantly fidgeting.

Abby:

I started noticing being able to sleep better and stay asleep all night long. Before, sometimes I would wake up in the middle of the night and have to scratch or itch my feet. I think it took about three or four weeks before I could actually sleep throughout the night.

Emma: I think it was the conversations I had with people, my mom especially, I’d be talking to her, she’s like, “You haven’t itched once in the last five minutes.” I’m like, “I haven’t?” I’ve just not been doing it, sitting perfectly still.

Abby:

I think mine was more at work because I sit in a cube all day. So I would get antsy and you’d want to itch, but when I was sitting at work for all day long and haven’t itched a single time, I was like, “Wow.” I felt better. I was able to sleep, and I wasn’t so groggy in the mornings because I was able to sleep because I’m not itching as much.

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LIVMARLI is an FDA-approved medicine for cholestatic pruritus (itch) patients with Alagille syndrome who are 3 months of age and older.

Abby:

My physician told me of several different side effects while taking LIVMARLI, including stomach pain, diarrhea, vomiting. I only experienced stomach pain one time, and it subsided after eating a larger meal.

Why do you think it is important to take LIVMARLI every day?

Emma:

It helps me manage my itching, and it just lets me go about my daily life without worrying about the itching as much. What about you?

Abby:

I want to live mylife like this. I love to travel. I love to go do things, and it’s very important to me to manage my pruritus and make sure my itching is at bay.

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Living with Alagille syndrome
as an adult and looking ahead

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Why it’s important to talk about cholestatic pruritus (itch)

Emma:

Why do you think it’s so important for us to talk about the pruritus?

Abby:

Because I think that a lot of people struggle with it, and I think that a lot of people feel like there’s no way out of it, and they have no hope. I think it’s really important to talk about your experiences and how you’ve dealt with it, and then where you are now so people can understand there are opportunities out there and there is hope.

Emma:

There’s a lot of unknowns when I was growing up. I didn’t know what was going to happen. I didn’t know what to do, so seeing someone else’s experience is a nice guide, a shining light in the dark.

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INDICATION

LIVMARLI is a prescription medicine used to treat cholestatic pruritus (itch) in patients who are 3 months of age and older with Alagille syndrome.

It is not known if LIVMARLI is safe and effective in children with Alagille syndrome who are under 3 months of age. It is not known if LIVMARLI is safe and effective in adults who are 65 years of age and older.

IMPORTANT SAFETY INFORMATION
What are the possible side effects of LIVMARLI?

LIVMARLI can cause serious side effects, including:

Liver injury. Changes in certain liver tests are common in patients but may worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your health care provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your health care provider right away if you get any signs or symptoms of liver problems, including:

nausea or vomiting
your skin or the white part of your eye turns yellow
dark or brown urine
pain on the right side of your stomach (abdomen)
fullness, bloating, or fluid in your stomach area (ascites)
loss of appetite
bleeding or bruising more easily than normal, including vomiting blood

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain during treatment. Diarrhea can also cause the loss of too much body fluid (severe dehydration). Your health care provider may advise you to monitor for new or worsening stomach problems, including stomach pain, diarrhea, blood in your stool, or vomiting

Tell your health care provider right away if you have any new or worsening signs or symptoms of stomach and intestinal problems, including:

diarrhea

more frequent bowel movements than usual
stools that are black, tarry, or sticky, or have blood or mucous
severe stomach-area pain or tenderness
vomiting
urinating less often than usual
dizziness
headache

A condition called Fat-Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your health care provider should do blood tests before starting and during treatment, and may monitor for bone fractures and bleeding, which are common side effects

Tell your health care provider about all medicines that you take, as LIVMARLI may interact with other medicines. If you take a medicine that lowers cholesterol by binding bile acids, such as cholestyramine, colesevelam, or colestipol, take it at least 4 hours before or 4 hours after you take LIVMARLI.

Your health care provider may change your dose, or temporarily or permanently stop treatment if you have certain side effects.

LIVMARLI is taken by mouth, 1 time each day, 30 minutes before a meal in the morning. Be sure to use the provided oral dosing dispenser to accurately measure the dose of medicine.

These are not all of the possible side effects of LIVMARLI. For more information, ask your health care provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

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Visit LIVMARLI.com/support-and-resources for helpful information about living with cholestatic pruritus (itch) in Alagille syndrome.

Alagille Syndrome

Cholestatic Pruritus in Young Adulthood: Meet Emma and Abby

From Infancy to Adulthood: Signs and Symptoms of Alagille Syndrome

What People with ALGS Wish Their Loved Ones Understood

Normalizing the Itch: Pruritus and Alagille Syndrome

The Mental and Emotional Toll of Alagille Syndrome

Tips for Managing Alagille Syndrome in Older Patients

How to Prepare Your Family for an Appointment When Seeking Support For Alagille Syndrome

Get Acquainted With ALGS, A Lesser-Known Rare Liver Disease

What is Alagille Syndrome With Cholestatic Pruritus?

10 Tips for Cholestatic Pruritus in Alagille Syndrome

Feed for Alagille Syndrome