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IDENTIFYING PATIENTS

Your patients’ outcomes start with your treatment decision.

In Alagille syndrome, it’s important to diagnose early and accurately.1 To do so, look for clinical features from 3 of the following 7 clinical criteria.2*†

Hepatic criteria
HEPATIC
  • Cholestasis
  • Jaundice
  • Hepatomegaly
Cardiac criteria
CARDIAC
  • Pulmonary stenosis
  • Tetralogy of Fallot
Facial criteria
FACIAL
  • Prominent forehead
  • Pointed chin
  • Deep‐set eyes
  • Bulbous tipped nose
Ocular criteria
OCULAR
  • Posterior embryotoxon
  • Optic disk drusen
Skeletal criteria
SKELETAL
  • Butterfly vertebrae
  • Pathological fractures
Renal criteria
RENAL
  • Renal dysplasia
  • Renal tubular acidosis
Vascular criteria
VASCULAR
  • Intracranial bleeding
  • Central nervous system (CNS) vascular malformations

*Absent of a molecular diagnosis or family history.

†Does not represent all possible clinical features.

From Diagnosis to Patient Identification

5-Year-Old Patient

Living With a Bothersome Itch That Won’t Quit

Medical History

  • Diagnosis: Diagnosed with Alagille syndrome at 1 year old, following identification of 4 (of 7) clinical criteria: jaundice, mild hepatomegaly, renal dysplasia, and posterior embryotoxon
    • Confirmatory genetic testing revealed a mutation in NOTCH2
  • Surgical interventions: None
  • Prior medication: Topical steroids
  • Current medication: Ursodiol

Physical Examination

  • Cholestatic pruritus:
    • CSS: 1 (rubbing or mild scratching when undistracted)
    • ItchRO(Obs) score: 2 (moderate itch)
  • Isolated patches of skin are red and inflamed from scratching
  • Despite current treatment, patient’s mother notes that scratching is persistent and with a diurnal pattern, occurring most often in the morning

Lab Results

  • sBA: 250 µmol/L (reference range: 0.0 to 10.0 µmol/L3,4)
5-year-old patient

Do you see patients like this in your practice?

Find out if LIVMARLI could help

7-Year-Old Patient

Still Scratching Despite Current Treatment

Medical History

  • Diagnosis: Diagnosed with Alagille syndrome at 6 months old, following identification of 4 (of 7) clinical criteria: jaundice, hepatomegaly, characteristic facial features, and butterfly vertebrae
    • Confirmatory genetic testing revealed a mutation in JAG1
    • Initially misdiagnosed as biliary atresia
  • Surgical interventions: Kasai procedure
  • Prior medication: Antihistamines
  • Current medication: Ursodiol

Physical Examination

  • Cholestatic pruritus:
    • CSS: 2 (active scratching without abrasions)
    • ItchRO(Pt) score: 2 (moderate itch)
  • Large areas of inflamed skin
  • Scars from old scratch wounds
  • Sleep disturbances
  • Patient’s mother is concerned that continued scratching will lead to open wounds
  • Often wears long-sleeved clothing to hide scratches and wounds

Lab Results

  • sBA: 325 µmol/L (reference range: 0.0 to 10.0 µmol/L3,4)
7-year-old patient

Do you see patients like this in your practice?

Find out if LIVMARLI could help

18-Month-Old Patient

Suffering From Abrasions and Bleeding Despite SOC Treatments

Medical History

  • Diagnosis: Diagnosed with Alagille syndrome at 6 months old, following identification of 3 (of 7) clinical criteria: pulmonary stenosis, posterior embryotoxon, and jaundice
    • Confirmatory genetic testing revealed a mutation in JAG1
  • Surgical interventions: None
  • Prior medication: Antihistamines, topical steroids
  • Current medication: Ursodiol, rifampicin

Physical Examination

  • Cholestatic pruritus:
    • CSS: 4 (cutaneous mutilations, hemorrhage, scarring)
    • ItchRO(Obs) score: 3 (severe itch)
  • Fussiness and irritability
  • Significant sleep disturbances
  • Numerous abrasions and large areas of red, inflamed skin
  • Patient’s father notes that bedsheets are regularly bloodied due to open wounds from scratching

Lab Results

  • sBA: 350 µmol/L (reference range: 0.0 to 10.0 µmol/L3,4)
18-month-old patient

Do you see patients like this in your practice?

Find out if LIVMARLI could help

Root Out
Excess Bile

Learn how LIVMARLI—the first FDA-approved treatment for cholestatic pruritus in Alagille syndrome—battles bile acid buildup.5

See How LIVMARLI Works

Encourage patients to download the Itch✓ app to help them track symptom patterns over time and generate customized reports to share at appointments.

Check Out the Itch✓ App

Mirum Access Plus assists both you and your patients at every turn, helping you navigate the payer approval process—and beyond—with ease.

Learn More About
Mirum Access Plus

CSS=Clinician Scratch Scale; ItchRO(Obs)=Itch Reported Outcome (Observer); ItchRO(Pt)=Itch Reported Outcome (Patient); JAG1=jagged canonical Notch ligand 1; NOTCH2=neurogenic locus notch homolog protein 2; sBA=serum bile acid; SOC=standard of care.

References: 1. Kamath BM, Baker A, Houwen R, Todorova L, Kerkar N. Systematic review: the epidemiology, natural history, and burden of Alagille syndrome. J Pediatr Gastroenterol Nutr. 2018;67(2):148-156. doi:10.1097/MPG.0000000000001958 2. Ayoub MD, Kamath BM. Alagille syndrome: diagnostic challenges and advances in management. Diagnostics (Basel). 2020;10(11):907. doi:10.3390/diagnostics10110907 3. Bile acids. Labcorp.com. Accessed May 23, 2023. https://www.labcorp.com/tests/010330/bile-acids 4. Jahnel J, Zöhrer E, Scharnagl H, Erwa W, Fauler G, Stojakovic T. Reference ranges of serum bile acids in children and adolescents. Clin Chem Lab Med. 2015;53(11):1807-1813. doi:10.1515/cclm-2014-1273 5. LIVMARLI® (maralixibat) oral solution. Prescribing Information. Mirum Pharmaceuticals, Inc.

INDICATION

LIVMARLI is indicated for the treatment of cholestatic pruritus in patients who are 3 months of age and older with Alagille syndrome.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

LIVMARLI is contraindicated in patients with prior or active hepatic decompensation events (eg, variceal hemorrhage, ascites, or hepatic encephalopathy).

WARNINGS AND PRECAUTIONS

Hepatotoxicity: LIVMARLI treatment is associated with a potential for drug-induced liver injury. In the Alagille syndrome trial, treatment-emergent elevations of liver tests or worsening of liver tests occurred.

Obtain baseline liver tests and monitor during treatment. Liver-related adverse reactions and physical signs of hepatic decompensation should also be monitored. Dose reduction or treatment interruption may be considered if abnormalities occur in the absence of other causes. For persistent or recurrent liver test abnormalities, consider treatment discontinuation. Permanently discontinue LIVMARLI if a patient experiences the following: persistent or recurrent liver test abnormalities, clinical hepatitis upon rechallenge, or a hepatic decompensation event.

Gastrointestinal (GI) Adverse Reactions: Diarrhea and abdominal pain were reported as the most common adverse reactions. Monitor for dehydration and treat promptly. Consider reducing the dosage or interrupting dosing if a patient experiences persistent diarrhea or has diarrhea with bloody stool, vomiting, dehydration requiring treatment, or fever.

Fat-Soluble Vitamin (FSV) Deficiency: Patients can have FSV deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may adversely affect absorption of FSVs. If bone fractures or bleeding occur, consider interrupting LIVMARLI and supplement with FSVs. LIVMARLI can be restarted once FSV deficiency is corrected and maintained at corrected levels.

Risk of Propylene Glycol Toxicity (Pediatric Patients Less Than 5 Years of Age): Total daily intake of propylene glycol should be considered for managing the risk of propylene glycol toxicity. Monitor patients for signs of propylene glycol toxicity. Discontinue LIVMARLI if toxicity is suspected.

ADVERSE REACTIONS

The most common adverse reactions are diarrhea, abdominal pain, vomiting, FSV deficiency, liver test abnormalities, and bone fractures.

DRUG INTERACTIONS

Administer bile acid binding resins at least 4 hours before or 4 hours after administration of LIVMARLI.
A decrease in the absorption of OATP2B1 substrates (eg, statins) due to OATP2B1 inhibition by LIVMARLI in the GI tract cannot be ruled out. Consider monitoring the drug effects of OATP2B1 substrates as needed.

DOSING INFORMATION

LIVMARLI should be taken 30 minutes before a meal. The provided oral dosing dispenser must be used to accurately measure the dose. Any remaining LIVMARLI should be discarded 100 days after first opening the bottle.

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CSS=Clinician Scratch Scale; ItchRO(Obs)=Itch Reported Outcome (Observer); ItchRO(Pt)=Itch Reported Outcome (Patient); JAG1=jagged canonical Notch ligand 1; NOTCH2=neurogenic locus notch homolog protein 2; sBA=serum bile acid; SOC=standard of care.

References: 1. Kamath BM, Baker A, Houwen R, Todorova L, Kerkar N. Systematic review: the epidemiology, natural history, and burden of Alagille syndrome. J Pediatr Gastroenterol Nutr. 2018;67(2):148-156. doi:10.1097/MPG.0000000000001958 2. Ayoub MD, Kamath BM. Alagille syndrome: diagnostic challenges and advances in management. Diagnostics (Basel). 2020;10(11):907. doi:10.3390/diagnostics10110907 3. Bile acids. Labcorp.com. Accessed May 23, 2023. https://www.labcorp.com/tests/010330/bile-acids 4. Jahnel J, Zöhrer E, Scharnagl H, Erwa W, Fauler G, Stojakovic T. Reference ranges of serum bile acids in children and adolescents. Clin Chem Lab Med. 2015;53(11):1807-1813. doi:10.1515/cclm-2014-1273 5. LIVMARLI® (maralixibat) oral solution. Prescribing Information. Mirum Pharmaceuticals, Inc.

INDICATION

LIVMARLI is indicated for the treatment of cholestatic pruritus in patients who are 3 months of age and older with Alagille syndrome.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

LIVMARLI is contraindicated in patients with prior or active hepatic decompensation events (eg, variceal hemorrhage, ascites, or hepatic encephalopathy).

WARNINGS AND PRECAUTIONS

Hepatotoxicity: LIVMARLI treatment is associated with a potential for drug-induced liver injury. In the Alagille syndrome trial, treatment-emergent elevations of liver tests or worsening of liver tests occurred.

Obtain baseline liver tests and monitor during treatment. Liver-related adverse reactions and physical signs of hepatic decompensation should also be monitored. Dose reduction or treatment interruption may be considered if abnormalities occur in the absence of other causes. For persistent or recurrent liver test abnormalities, consider treatment discontinuation. Permanently discontinue LIVMARLI if a patient experiences the following: persistent or recurrent liver test abnormalities, clinical hepatitis upon rechallenge, or a hepatic decompensation event.

Gastrointestinal (GI) Adverse Reactions: Diarrhea and abdominal pain were reported as the most common adverse reactions. Monitor for dehydration and treat promptly. Consider reducing the dosage or interrupting dosing if a patient experiences persistent diarrhea or has diarrhea with bloody stool, vomiting, dehydration requiring treatment, or fever.

Fat-Soluble Vitamin (FSV) Deficiency: Patients can have FSV deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may adversely affect absorption of FSVs. If bone fractures or bleeding occur, consider interrupting LIVMARLI and supplement with FSVs. LIVMARLI can be restarted once FSV deficiency is corrected and maintained at corrected levels.

Risk of Propylene Glycol Toxicity (Pediatric Patients Less Than 5 Years of Age): Total daily intake of propylene glycol should be considered for managing the risk of propylene glycol toxicity. Monitor patients for signs of propylene glycol toxicity. Discontinue LIVMARLI if toxicity is suspected.

ADVERSE REACTIONS

The most common adverse reactions are diarrhea, abdominal pain, vomiting, FSV deficiency, liver test abnormalities, and bone fractures.

DRUG INTERACTIONS

Administer bile acid binding resins at least 4 hours before or 4 hours after administration of LIVMARLI.
A decrease in the absorption of OATP2B1 substrates (eg, statins) due to OATP2B1 inhibition by LIVMARLI in the GI tract cannot be ruled out. Consider monitoring the drug effects of OATP2B1 substrates as needed.

DOSING INFORMATION

LIVMARLI should be taken 30 minutes before a meal. The provided oral dosing dispenser must be used to accurately measure the dose. Any remaining LIVMARLI should be discarded 100 days after first opening the bottle.