TEPROTUMUMAB VERSUS INTRAVENOUS METHYLPREDNISOLONE ALONE, AND IN COMBINATION WITH MYCOPHENOLATE MOFETIL, FOR MODERATE-TO-SEVERE ACTIVE THYROID EYE DISEASE: A MATCHING-ADJUSTED INDIRECT COMPARISON
Author(s)
Alexandra Eddy, MSc1, Galin Spicer, MD1, Louisa Pericleous, PhD1, Christina Giannopoulou, MSc2;
1Amgen Inc, Thousand Oaks, CA, USA, 2Amgen (Europe) GmbH, Rotkreuz, Switzerland
1Amgen Inc, Thousand Oaks, CA, USA, 2Amgen (Europe) GmbH, Rotkreuz, Switzerland
OBJECTIVES: Teprotumumab has demonstrated efficacy in thyroid eye disease (TED), a complex, autoimmune condition. However, in the absence of head-to-head data, matching-adjusted indirect comparisons (MAIC) were conducted to assess the relative effectiveness of teprotumumab versus intravenous methylprednisolone (IVMP), and in combination with mycophenolate mofetil (MMF), in moderate-to-severe active TED.
METHODS: Randomized controlled trials (RCTs) reporting mean change from baseline (CFB) in proptosis (mm) and diplopia response rate (reduction of ≥1 grade) for teprotumumab and comparators were identified via systematic literature review. Pooled treatment effects for IVMP were derived from seven studies on proptosis and four on diplopia. For IVMP+MMF, one study was available for proptosis, with none identified for diplopia. A scenario considering both RCTs and real-world evidence (RCT+RWE) for IVMP included ten studies for proptosis and six for diplopia. For teprotumumab, individual patient-level data from the phase 2 trial (NCT01868997) and phase 3 OPTIC (NCT03298867) were pooled. Covariates informed by UK clinicians and included in the analysis were smoking, baseline diplopia, baseline proptosis, and radioiodine therapy (RIT) for IVMP, and smoking, baseline diplopia, age, sex, and RIT for IVMP+MMF.
RESULTS: In the base case (RCTs), teprotumumab was associated with greater CFB in proptosis versus IVMP (-2.41 mm, 95% confidence interval [CI]: -3.22, -1.60) and IVMP+MMF (-3.06 mm, 95% CI:-4.35, -1.77). Teprotumumab showed higher odds of diplopia response versus IVMP (odds ratio: 2.09, 95% CI: 0.93, 4.68). In the RCT+RWE scenario, results for teprotumumab versus IVMP showed greater CFB in proptosis (-2.20 mm, 95% CI: -3.04, -1.36) and higher odds of diplopia response (odds ratio: 2.88, 95% CI: 1.17,7.10).
CONCLUSIONS: Results show teprotumumab demonstrated a clinical benefit versus IVMP and IVMP+MMF for patients with moderate-to-severe active TED. By incorporating more recently published evidence, these findings align with two previous indirect comparisons and strengthen the evidence supporting teprotumumab’s efficacy in TED.
METHODS: Randomized controlled trials (RCTs) reporting mean change from baseline (CFB) in proptosis (mm) and diplopia response rate (reduction of ≥1 grade) for teprotumumab and comparators were identified via systematic literature review. Pooled treatment effects for IVMP were derived from seven studies on proptosis and four on diplopia. For IVMP+MMF, one study was available for proptosis, with none identified for diplopia. A scenario considering both RCTs and real-world evidence (RCT+RWE) for IVMP included ten studies for proptosis and six for diplopia. For teprotumumab, individual patient-level data from the phase 2 trial (NCT01868997) and phase 3 OPTIC (NCT03298867) were pooled. Covariates informed by UK clinicians and included in the analysis were smoking, baseline diplopia, baseline proptosis, and radioiodine therapy (RIT) for IVMP, and smoking, baseline diplopia, age, sex, and RIT for IVMP+MMF.
RESULTS: In the base case (RCTs), teprotumumab was associated with greater CFB in proptosis versus IVMP (-2.41 mm, 95% confidence interval [CI]: -3.22, -1.60) and IVMP+MMF (-3.06 mm, 95% CI:-4.35, -1.77). Teprotumumab showed higher odds of diplopia response versus IVMP (odds ratio: 2.09, 95% CI: 0.93, 4.68). In the RCT+RWE scenario, results for teprotumumab versus IVMP showed greater CFB in proptosis (-2.20 mm, 95% CI: -3.04, -1.36) and higher odds of diplopia response (odds ratio: 2.88, 95% CI: 1.17,7.10).
CONCLUSIONS: Results show teprotumumab demonstrated a clinical benefit versus IVMP and IVMP+MMF for patients with moderate-to-severe active TED. By incorporating more recently published evidence, these findings align with two previous indirect comparisons and strengthen the evidence supporting teprotumumab’s efficacy in TED.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
CO111
Topic
Clinical Outcomes
Topic Subcategory
Comparative Effectiveness or Efficacy
Disease
SDC: Diabetes/Endocrine/Metabolic Disorders (including obesity), SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)