In a 6-month clinical study called MARCH-PFIC, 93 patients with progressive familial intrahepatic cholestasis (PFIC) were treated with either LIVMARLI or placebo* to find out if it helped reduce levels of cholestatic pruritus (itch) and bile acids in the blood (known as serum bile acids, or sBAs). After 6 months, patients had the option to enter a long-term follow-up study (called MARCH-ON).
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Early improvements Improvements in cholestatic pruritus were seen as early as 2 weeks after starting LIVMARLI. -
Relief that lasts Relief from cholestatic pruritus lasted through 2 years for those who stayed on LIVMARLI in the long-term study. -
Reductions in sBA levels About 5 in 10 patients had their sBA levels reduced by at least 75%†
- *The MARCH-PFIC study was a multicenter, double-blind study, meaning the study was run in many locations, and patients and doctors were not aware which patients received LIVMARLI and which received placebo. After 26 weeks, for patients who chose to remain on treatment, all patients took only LIVMARLI. Placebo looked and tasted like a “real” treatment, but did not have any active medicine.
- †Compared with less than 1 in 10 people (7%) who received placebo.
The MARCH-PFIC study
Patients were included if they were between 1 and 18 years old, diagnosed with progressive familial intrahepatic cholestasis (PFIC), cholestatic pruritus (itch) lasting at least 6 months, and an average morning cholestatic pruritus severity score of ≥1.5 as measured by the Itch Reported Outcome (Observer) (ItchRO[Obs]) tool.
The ItchRO scale
In the clinical study, patients kept an ongoing log of their itch using the Itch Reported Outcome (ItchRO) tool. This tool was used to measure itch severity over time based on a 5-point scale:
0
Not itchy at all
1
A little bit itchy
2
Somewhat itchy
3
Very itchy
4
Extremely itchy
Defining “improvement”
Changes in ItchRO score by 1 point or more represent noticeable differences in the itch; for example, a change from 3 to 2 means that a patient went from feeling very itchy to feeling somewhat itchy.
Talk with your doctor about what to expect and how to manage any side effects.
Gastrointestinal (GI) events like diarrhea or stomach pain are common as your body adjusts to LIVMARLI. Some patients may experience fat-soluble vitamin (FSV) deficiency while taking LIVMARLI. Other common side effects include liver test abnormalities, bone fractures, and rectal bleeding. Learn more about safety with LIVMARLI.
Only included patients with PFIC2.
14 patients received LIVMARLI; 17 patients received placebo
Included patients with PFIC2 as well as patients with PFIC1, PFIC3, PFIC4, or PFIC6.
33 patients received LIVMARLI; 31 patients received placebo
Other patients with less common or more variable forms of PFIC were also studied.
During the first 6 months of treatment, patients taking LIVMARLI in the PFIC2 group saw significant improvements in cholestatic pruritus (average 1.7-point reduction in ItchRO score) compared with those who took placebo (average less than 0.6-point reduction in ItchRO score).
with PFIC experienced noticeably less cholestatic pruritus within 6 months of starting treatment with LIVMARLI.‡
with PFIC who were treated with LIVMARLI had significantly more days with little to no cholestatic pruritus‡ (had an ItchRO score of 0 or 1).
Half of the patients in the All-PFIC group who took LIVMARLI had a
or achieved sBA levels less than 102 µmol/L.§
sBA refers to the level of bile acid in a person's blood. High levels of bile acids in the body are associated with cholestatic pruritus and liver damage.
Lasting Results
Those who stayed on LIVMARLI in the long-term study felt relief from cholestatic pruritus through 2 years.
- ‡Compared with around 3 in 10 patients with PFIC who received placebo.
- §Compared with 7% of patients who received placebo.
—Stuart, a real person living with PFIC
Not all patients taking LIVMARLI and their caregivers will have the same experience.
We want to celebrate your successes with you. If you’ve noticed improvements in cholestatic pruritus in PFIC since starting LIVMARLI, get in touch!
Visit mylivmarlistory.com to share your experience.
An exploratory analysis was performed to see if there were any other benefits associated with improvements in cholestatic pruritus in the clinical studies. Of 59 patients included in the analysis, 37 patients (63%) experienced significant improvements in cholestatic pruritus with LIVMARLI (had at least a 1-point decrease in ItchRO score) and 22 patients (37%) did not. Patients who experienced improvements in cholestatic pruritus also saw improvements in the level of bilirubin in their blood as well as improvements in their growth.‖
How LIVMARLI affected bilirubin
Bilirubin is a part of bile that forms when your body breaks down red blood cells. If bile is not flowing properly, bilirubin can build up in the blood.
Doctors can measure bilirubin to detect, monitor, and understand the severity of liver injury or disease.
2x reduction in bilirubin
Those who saw improvements in cholestatic pruritus with LIVMARLI also had more than twice the reduction in bilirubin compared with those who did not, on average.
100% of patients
(12 out of 12) who started with normal total bilirubin levels kept those levels normal after 26 weeks vs half (1 out of 2) who did not see improvements in cholestatic pruritus.¶#
56% of patients
(14 out of 25) who started with high bilirubin levels saw those levels normalize after 26 weeks of treatment with LIVMARLI vs 5% of patients (1 out of 19) who did not see improvements in their cholestatic pruritus.#
How LIVMARLI affected growth (height and weight)
Children with conditions that cause cholestatic pruritus can sometimes experience poor growth (shorter stature and decreased weight).
Results from MARCH-PFIC and MARCH-ON showed that patients who experienced improvements in cholestatic pruritus were also more likely to grow in height and gain weight over time.
Height and weight were measured as a Z-score, which shows how someone compares with the average for their age and sex.
People who felt cholestatic pruritus relief with LIVMARLI started
growing taller in just 1 month and gaining weight in 6 weeks.
Improvements in height and weight continued for more than a year.**††
- ‖"Improvements" defined as an improvement in ItchRO score of 1 or more.
- ¶Based on average total bilirubin value at Weeks 18, 22, and 26.
- #Normal bilirubin defined as levels ≤1.2 mg/dL; high bilirubin defined as levels >1.2 mg/dL as measured by a blood test.
- **From extended observation of patients taking LIVMARLI after completing a placebo-controlled study.
- ††At 70 weeks, 12 patients in the PFIC2 group and 23 patients in the All-PFIC group who experienced cholestatic pruritus improvements also had increases in height and weight. Height and weight were measured as a Z-score, which shows how someone compares with the average for their age and sex.
LIVMARLI is the only ileal bile acid transporter (IBAT) inhibitor available as a tablet or a liquid (oral solution) medicine.
