Thyroid Eye Disease
Conditions
Keywords
Thyroid Eye Disease, Teprotumumab, TEPEZZA®
Brief summary
The main objective of this study is to assess the number of participants with proptosis response as of last intravenous infusion of teprotumumab.
Detailed description
A retrospective cohort will be followed retrospectively for endpoints at the only facility providing treatment of teprotumumab in mainland China. Eligible participants' data were collected retrospectively over a period of approximately 3 years. The target population is patients with Thyroid Eye Disease (TED) that have received teprotumumab treatment in BOAO Pilot Zone. While the primary objective is to assess the proportion of patients with proptosis response as of the last intravenous infusion of teprotumumab, the secondary objectives are to describe baseline characteristics of patients at teprotumumab initiation, and the teprotumumab use at follow-up, to describe real-world effectiveness of clinical outcomes of interest, to describe patient-reported outcomes and to describe real-world safety of teprotumumab.
Interventions
DRUGTeprotumumab
Participants received teprotumumab as an intravenous (IV) infusion.
Sponsors
Amgen
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 120 Years
Healthy volunteers
No
Inclusion criteria
* Receipt of at least 1 infusion of teprotumumab for treatment of the TED. * Obtained study specific informed consent form (ICF), if required.
Exclusion criteria
* Documentation of non-Chinese ethnicity.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Proptosis Response as of the Last IV Infusion Teprotumumab | From baseline up to approximately 3 years | Proptosis response is defined as having a ≥ 2 mm reduction in proptosis measurement from baseline in the more severely affected eye without deterioration (≥ 2 mm increase) of proptosis in the fellow eye following the last infusion. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Characteristics of Participants at Teprotumumab Initiation: Body Mass Index at Index Date | From baseline up to approximately 3 years | — |
| Clinical Characteristics of Participants at Teprotumumab Initiation: Smoking Status | From baseline up to approximately 3 years | — |
| Clinical Characteristics of Participants at Teprotumumab Initiation: Disease Duration | From baseline up to approximately 3 years | — |
| Clinical Characteristics of Participants at Teprotumumab Initiation: TED Activity | From baseline up to approximately 3 years | — |
| Clinical Characteristics of Participants at Teprotumumab Initiation: TED Severity | From baseline up to approximately 3 years | — |
| Clinical Characteristics of Participants at Teprotumumab Initiation: Eye symptoms | From baseline up to approximately 3 years | — |
| Clinical Characteristics of Participants at Teprotumumab Initiation: Clinical Activity Score | From baseline up to approximately 3 years | — |
| Clinical Characteristics of Participants at Teprotumumab Initiation: Computed Tomography (CT)/Magnetic Resonance Imaging (MRI) Measurements | From baseline up to approximately 3 years | — |
| Clinical Characteristics of Participants at Teprotumumab Initiation: Quality of Life | From baseline up to approximately 3 years | — |
| Demographics of Participants at Teprotumumab Initiation | From baseline up to approximately 3 years | Demographics include age in years and sex (male, female, unknown). |
| Teprotumumab Use | From baseline up to approximately 3 years | Teprotumumab use includes dose and dosing schedule, total number of infusions and duration, dose reduction and modification (including causes), treatment interruption and causes, time interval from interruption to re-initiation, and subsequent re-initiation treatment (if any), treatment discontinuation (including causes). |
| Proptosis Measurements of Participants | From baseline up to approximately 3 years | Proptosis measurements include all available proptosis measurements by eye for each participant and the change in proptosis measurement between baseline and the last infusion by eye. |
| Clinical Activity Scores (CAS) of Participants | From baseline up to approximately 3 years | CAS will include all available measures of CAS items by eye for each participant and the number of participants achieving CAS value of 0 or 1 as of the last infusion among patients with active TED at baseline. |
| Overall Response as of the Last Infusion Among Participants with Active TED | From baseline up to approximately 3 years | Overall response is defined as having ≥ 2-point reduction in CAS AND ≥ 2 mm reduction in proptosis from baseline in the most severely affected eye, provided there is no corresponding deterioration (≥ 2-point increase in CAS or ≥ 2 mm increase in proptosis) in the fellow eye following the last infusion. Active TED is defined as CAS≥3 on a 7-point scale at baseline. |
| Diplopia Measurements for Participants | From baseline up to approximately 3 years | Diplopia measurements will include all available measures of diplopia for each participant, the binocular diplopia response as of the last infusion defined as having baseline binocular diplopia grade \> 0 and a reduction of ≥ 1 grade between baseline and the last infusion, and complete diplopia response defined as having baseline binocular diplopia grade \> 0 and achieving grade of 0 as of the last infusion. |
| Quality of Life (QoL) as Reported by Participants | From baseline up to approximately 3 years | All available participant-reported QoL for each participant, overall and at subscale level, and the change in QoL between baseline and the last infusion, overall and at subscale level. |
| Incidence of Adverse Events (AEs) | From baseline up to approximately 3 years | The incidence of AEs between the administration of the first dose and 30 days after the last dose of teprotumumab, including: * Any AE * Specific AEs, inclusive of infusion-related reactions, hyperglycemia, hearing impairment, and new onset or exacerbation of IBD * Serious adverse event (SAE) * AE leading to treatment discontinuation * AE leading to death. |
| Status of Specific AEs up to 6 Months After Last Infusion of Teprotumumab | From baseline up to approximately 3 years | Specific AEs include infusion-related reactions, hyperglycemia, hearing impairment, and new onset or exacerbation of IBD. |
| Number of Participants with Relevant Medical History at Teprotumumab Initiation | From baseline up to approximately 3 years | Medical history includes history of compressive optic neuropathy, hyperglycemia, inflammatory bowel disease (IBD), infusion reactions, and hearing impairment; therapeutic history for thyroid disease (Graves' disease or others) before teprotumumab administration; therapeutic history for TED before teprotumumab administration and complications if any; comorbidities; concomitant treatments; lab tests. |
Countries
China
Outcome results
None listed