Healthcare Resource Utilization Associated With Acute Pancreatitis in Patients With Familial Chylomicronemia Syndrome Treated With Olezarsen: A Post Hoc Analysis of Balance Study Data
Author(s)
Asia Sikora Kessler, PhD1, Montserrat Vera-Llonch, MD, MPH, MSc1, Veronica J. Alexander, PhD1, Catrina Richards, MSc2, Quratul Ann, MSc, BSc2, Catalina Mejía Herrera, MSc, Bachelor in Statistics3, Spyros Paparrodopoulos, MSc, BSc4, Shuting Xia, MS1, Sotirios Tsimikas, MD5, Seth J. Baum, MD, MASPC, FACC, FAHA, FNLA6.
1Ionis Pharmaceuticals, Inc, Carlsbad, CA, USA, 2IQVIA Ltd, London, United Kingdom, 3IQVIA Ltd, Frankfurt, Germany, 4IQVIA Ltd, Athens, Greece, 5Vascular Medicine Program, Sulpizio Cardiovascular Center, University of California, San Diego, La Jolla, CA, USA, 6Flourish Research, Boca Raton, FL, USA.
1Ionis Pharmaceuticals, Inc, Carlsbad, CA, USA, 2IQVIA Ltd, London, United Kingdom, 3IQVIA Ltd, Frankfurt, Germany, 4IQVIA Ltd, Athens, Greece, 5Vascular Medicine Program, Sulpizio Cardiovascular Center, University of California, San Diego, La Jolla, CA, USA, 6Flourish Research, Boca Raton, FL, USA.
OBJECTIVES: The randomized, placebo-controlled, phase 3 Balance study assessed olezarsen versus placebo in 66 patients with familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized by severe hypertriglyceridemia and a high risk of acute pancreatitis. This analysis evaluated adjudicated pancreatitis (AP)- and AP- or abdominal pain-related healthcare resource utilization (HCRU) among patients from the Balance study.
METHODS: This was a post hoc analysis of the Balance study to assess hospitalizations, inpatient days, emergency room (ER) visits, intensive care unit (ICU) admissions, and length of hospital stay (LOS) in each olezarsen dose group (50 or 80 mg every 4 weeks) and the pooled group (50 mg and 80 mg) versus placebo over a 53-week treatment period. Negative binomial regression models were used to estimate annualized adjusted least-squares mean rates (LSMRs) and mean rate ratios (MRRs) with 95% confidence intervals. ANCOVA was used to compare inpatient days.
RESULTS: Two patients in the pooled olezarsen group (one in each dose group) were hospitalized for AP. The yearly LSMRs of AP-related hospitalizations were significantly lower in the pooled olezarsen group than in the placebo group (0.04 vs 0.36; MRR, 0.12; P=0.014), with fewer inpatient days (−4.36 days, P=0.0274). The annualized ICU admission rate in the pooled olezarsen group was 50% lower (relative risk: 0.50 [95% CI: 0.30, 0.83]) versus placebo. ER visits were rare in all groups. AP-related LOS was longer in the placebo group (12.95 days) than in the pooled olezarsen group (5.29 days). Similar results were observed for AP- or abdominal pain-related HCRU.
CONCLUSIONS: In the Balance study, olezarsen was associated with fewer AP- and AP- or abdominal pain-related hospitalizations and reduced inpatient days versus placebo in patients with FCS, suggesting that healthcare costs may be lowered with this treatment. Long-term data from clinical practice is expected to provide additional supportive evidence.
METHODS: This was a post hoc analysis of the Balance study to assess hospitalizations, inpatient days, emergency room (ER) visits, intensive care unit (ICU) admissions, and length of hospital stay (LOS) in each olezarsen dose group (50 or 80 mg every 4 weeks) and the pooled group (50 mg and 80 mg) versus placebo over a 53-week treatment period. Negative binomial regression models were used to estimate annualized adjusted least-squares mean rates (LSMRs) and mean rate ratios (MRRs) with 95% confidence intervals. ANCOVA was used to compare inpatient days.
RESULTS: Two patients in the pooled olezarsen group (one in each dose group) were hospitalized for AP. The yearly LSMRs of AP-related hospitalizations were significantly lower in the pooled olezarsen group than in the placebo group (0.04 vs 0.36; MRR, 0.12; P=0.014), with fewer inpatient days (−4.36 days, P=0.0274). The annualized ICU admission rate in the pooled olezarsen group was 50% lower (relative risk: 0.50 [95% CI: 0.30, 0.83]) versus placebo. ER visits were rare in all groups. AP-related LOS was longer in the placebo group (12.95 days) than in the pooled olezarsen group (5.29 days). Similar results were observed for AP- or abdominal pain-related HCRU.
CONCLUSIONS: In the Balance study, olezarsen was associated with fewer AP- and AP- or abdominal pain-related hospitalizations and reduced inpatient days versus placebo in patients with FCS, suggesting that healthcare costs may be lowered with this treatment. Long-term data from clinical practice is expected to provide additional supportive evidence.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
RWD94
Topic
Clinical Outcomes, Economic Evaluation, Real World Data & Information Systems
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Rare & Orphan Diseases