• About
  • Masthead
  • License Content
  • Advertise
  • Submit Press Release
  • RSS/Email List
  • Write for us
  • Contact us
2 Minute Medicine
No Result
View All Result

No products in the cart.

SUBSCRIBE
  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • AccountLog-in/out
    • Subscribe
    • Sign-in
    • My account
2 Minute Medicine
  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • AccountLog-in/out
    • Subscribe
    • Sign-in
    • My account
SUBSCRIBE
2 Minute Medicine
Subscribe
Home All Specialties Chronic Disease

Maralixibat appears safe and efficacious for patients with Alagille syndrome and cholestatic pruritus

byNeel MistryandTeddy Guo
November 9, 2021
in Chronic Disease, Gastroenterology, Pediatrics
Reading Time: 3 mins read
0
Share on FacebookShare on Twitter

1. Switching to placebo after week 18 resulted in greater elevations in serum bile acid levels and pruritus compared to patients who remained on maralixibat.

2. Maralixibat was well-tolerated with few self-limiting adverse events that were mild-to-moderate in severity. 

Evidence Rating Level: 1 (Excellent)

Study Rundown: Alagille syndrome is a rare autosomal-dominant genetic condition marked by an inability to drain bile. The resultant cholestasis may progress to liver damage which manifests symptomatically as jaundice, chronic pruritus, and xanthomas. Currently, there is no available pharmacotherapy for this disease with most patients ultimately needing a liver transplant. Apical sodium-dependent bile acid transporter (ASBT) inhibitors, such as maralixibat, are known to disrupt the enterohepatic circulation of bile and may be used to prevent cholestasis. This randomized controlled trial aimed to evaluate the safety and efficacy of maralixibat in patients with Alagille syndrome. The primary endpoint was a change in mean serum bile acid (sBA) levels during the randomized withdrawal period (RWD), while key secondary endpoints included changes in Clinician Scratch Scale (CSS) and Clinical Xanthoma Scale (CXS) score. According to study results, patients assigned to the placebo group had a significant increase in sBA levels and pruritus compared to those who remained on maralixibat. In addition, those on maralixibat showed a progressive decrease in sBA levels over time. Although this study was well conducted, it had a small sample size, impacting the validity of results.

Click to read the study in The Lancet

Relevant Reading: Outcomes of Childhood Cholestasis in Alagille Syndrome: Results of a Multicenter Observational Study

In-depth [randomized controlled trial]: Between Oct 28, 2014, and Aug 14, 2015, 36 patients were assessed for eligibility. Included were those 1-18 years of age with three times the normal sBA levels and uncontrollable pruritus. Enrolled patients (n=31) were given maralixibat 380 ug/kg daily for 18 weeks and assigned to continue maralixibat or placebo for 4 weeks (weeks 19-22; randomized withdrawal period [RWD]). All patients were subsequently given maralixibat to week 48. Mean age among those enrolled was 5.4 years (standard deviation [SD] 4.25) and the majority (66%; 19 of 29) of RWD follow-ups were male. Regarding the primary endpoint, patients who switched to placebo after week 18 showed increased sBA (94 umol/L, 95% confidence interval [CI] 23 to 164) and pruritus (1.7 points, 95% CI 1.2 to 2.2) compared to those who remained on maralixibat. Furthermore, a progressive improvement in both markers was noted for patients on maralixibat from baseline to week 48 (sBA: -96 umol/L, 95% CI -162 to -31 and pruritus: -1.6 points, 95% CI -2.1 to -1.1). Of the 15 participants who remained on maralixibat, the reductions in sBA persisted to week 204. No serious adverse events were noted. Overall, findings from this study suggest that maralixibat contains good safety and efficacy for management of cholestasis in patients with Alagille syndrome, although further studies are needed to confirm this. 

RELATED REPORTS

Expectant management of patent ductus arteriosus noninferior to early ibuprofen use

SAR-CoV-2 infection during pregnancy may lead to adverse neurodevelopmental outcomes in male offspring

Lebrikizumab effective in treating moderate-to-severe atopic dermatitis

Image: PD 

©2021 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

Tags: Alagille syndromebile acidcholestasischolestatic pruritusileal bile acid transporter (IBAT) inhibitorliver failureMaralixibatpediatric GIpediatrics
Previous Post

Low-to-Moderate caffeine intake during pregnancy is not associated with increased risk of maternal cardiometabolic complications

Next Post

Inhaled and intranasal ciclesonide for the treatment of COVID-19 in adult outpatients

RelatedReports

Wide international variation in survival for periviable neonates
Cardiology

Expectant management of patent ductus arteriosus noninferior to early ibuprofen use

March 30, 2023
Paternal factors associated with short interpregnancy interval
Infectious Disease

SAR-CoV-2 infection during pregnancy may lead to adverse neurodevelopmental outcomes in male offspring

March 29, 2023
Pimecrolimus effective first-line treatment for atopic dermatitis
Chronic Disease

Lebrikizumab effective in treating moderate-to-severe atopic dermatitis

March 29, 2023
Nivolumab may be effective in platinum-resistant ovarian cancer
Obstetrics

Immediate postpartum opioid administration not associated with adverse infant outcomes

March 28, 2023
Next Post
Novel coronavirus identified from patients with pneumonia in Wuhan, China

Inhaled and intranasal ciclesonide for the treatment of COVID-19 in adult outpatients

Novel coronavirus identified from patients with pneumonia in Wuhan, China

Immune thrombocytopenia and thrombosis mortality predictors following SARS-CoV-2 vaccine

#VisualAbstract: Internal mammary node irradiation improves survival for patients with centromedially located node-positive breast cancer

#VisualAbstract: Low-dose computed tomography improves the detection of secondary primary lung cancers compared to chest radiographs for head and neck cancer survivors

License Our Award-Winning Physician-Written Medical News and Visual Abstracts

2 Minute Medicine is the leading authoritative medical news licensing service, and the only with reports written by practicing doctors.

LICENSE CONTENT

2MM+ Premium Access

No ads & unlimited access to all current reports, over 9000 searchable archived reports, visual abstracts, Weekly Rewinds, and the online edition of The Classics Series™ textbook.

Subscription Options
2 Minute Medicine

2 Minute Medicine® is an award winning, physician-run, expert medical media company. Our content is curated, written and edited by practicing health professionals who have clinical and scientific expertise in their field of reporting. Our editorial management team is comprised of highly-trained MD physicians. Join numerous brands, companies, and hospitals who trust our licensed content.

Recent Reports

  • Metformin use may decrease risk of osteoarthritis development
  • Intensive blood pressure lowering by non-physician healthcare providers significantly reduces the risk of cardiovascular disease
  • Expectant management of patent ductus arteriosus noninferior to early ibuprofen use
License Content
Terms of Use | Disclaimer
Cookie Policy
Privacy Statement (EU)
Disclaimer

© 2021 2 Minute Medicine, Inc. - Physician-written medical news.

  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • Account
    • Subscribe
    • Sign-in
    • My account

© 2021 2 Minute Medicine, Inc. - Physician-written medical news.

Want more physician-written
medical news?

Join over 10 million yearly readers and numerous companies. For healthcare professionals
and the public.

Subscribe for free today!

Subscription options