UDCA Treatment Response in Primary Biliary Cholangitis in the United States
Author(s)
Robert G. Gish, MD1, Robert G. Wong, MD2, Yi Pan, PhD3, Mihail Samnaliev, PhD3, Diane Ito, MA3, Chong H Kim, MPH, MS, PhD4.
1Robert G. Gish Consultants, La Jolla, CA, USA, 2Stanford University School of Medicine, Palo Alto, CA, USA, 3Stratevi, Santa Monica, CA, USA, 4Gilead Sciences, Foster City, CA, USA.
1Robert G. Gish Consultants, La Jolla, CA, USA, 2Stanford University School of Medicine, Palo Alto, CA, USA, 3Stratevi, Santa Monica, CA, USA, 4Gilead Sciences, Foster City, CA, USA.
OBJECTIVES: Primary biliary cholangitis (PBC) is a progressive, chronic inflammatory liver disease associated with substantial clinical and economic burden. Many patients have inadequate response to ursodeoxycholic acid (UDCA), the recommended first-line treatment for PBC. However, evidence from clinical trials on UDCA response rates varies widely, limiting efforts to identify and assess the population of patients with PBC who may require second-line therapies. We evaluated UDCA response rates and predictors of response among individuals with PBC using a large integrated claims and EHR dataset from the United States.
METHODS: Komodo’s Healthcare Map from September 1, 2017 to September 30, 2023 was used to identify PBC patients who initiated UDCA (index date), had continuous insurance coverage within 12 months pre-index, and baseline ALP >=1.67×ULN. Patients prescribed obeticholic acid or fibrates at baseline were excluded. UDCA response at 12 months (+/-6 months) was assessed using a composite endpoint (alkaline phosphatase [ALP] <1.67×ULN, >=15% ALP reduction, and total bilirubin [TB] <=1.0×ULN) and an ALP normalization endpoint (ALP <= ULN). Patients’ characteristics were compared between responders and non-responders.
RESULTS: Of 726 patients, 500 (69%) met the composite response endpoint and 281 (39%) met the ALP response endpoint. Compared to non-responders, composite responders were older (62.1 vs 57.6), more often female (84.6% vs 74.8%), White (55.8% vs 41.2%), Medicare-insured (47.6% vs 37.6%), had lower comorbidity (CCI: 2.8 vs 3.3), were less likely to have autoimmune hepatitis (13.8% vs 19.9%) or cirrhosis (19.2% vs 37.2%), and had lower baseline ALP (408 vs 579) and TB (1.1 vs 1.8) (all p<.05).
CONCLUSIONS: Although 69% of patients with PBC met the composite response endpoint, the majority did not achieve ALP normalization. This highlights the need for effective second-line treatment options. Several demographic and clinical predictors of response emerged suggesting opportunities to tailor therapies to specific patient subgroups.
METHODS: Komodo’s Healthcare Map from September 1, 2017 to September 30, 2023 was used to identify PBC patients who initiated UDCA (index date), had continuous insurance coverage within 12 months pre-index, and baseline ALP >=1.67×ULN. Patients prescribed obeticholic acid or fibrates at baseline were excluded. UDCA response at 12 months (+/-6 months) was assessed using a composite endpoint (alkaline phosphatase [ALP] <1.67×ULN, >=15% ALP reduction, and total bilirubin [TB] <=1.0×ULN) and an ALP normalization endpoint (ALP <= ULN). Patients’ characteristics were compared between responders and non-responders.
RESULTS: Of 726 patients, 500 (69%) met the composite response endpoint and 281 (39%) met the ALP response endpoint. Compared to non-responders, composite responders were older (62.1 vs 57.6), more often female (84.6% vs 74.8%), White (55.8% vs 41.2%), Medicare-insured (47.6% vs 37.6%), had lower comorbidity (CCI: 2.8 vs 3.3), were less likely to have autoimmune hepatitis (13.8% vs 19.9%) or cirrhosis (19.2% vs 37.2%), and had lower baseline ALP (408 vs 579) and TB (1.1 vs 1.8) (all p<.05).
CONCLUSIONS: Although 69% of patients with PBC met the composite response endpoint, the majority did not achieve ALP normalization. This highlights the need for effective second-line treatment options. Several demographic and clinical predictors of response emerged suggesting opportunities to tailor therapies to specific patient subgroups.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO256
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)