Interstitial Lung Diseases, Systemic Sclerosis Associated Interstitial Lung Disease
Conditions
Brief summary
This post-approval registry study is planned to generate data to address remaining questions on long-term effectiveness and to better characterize longer term beneficial effects of Nintedanib in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD) in terms of survival, quality of life, pattern of disease progression as well as effectiveness and safety in the subgroup of patients with pulmonary hypertension.
Interventions
DRUGOfev®
Ofev®
Sponsors
Boehringer Ingelheim
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Enrolled in the EUSTAR registry * Provided consent at their site to have their data included in the EUSTAR registry for a broad range of research studies, and agree to visit the site every 12 months * Have a diagnosis of ILD defined by radiological findings on High Resolution Computed Tomography (HRCT) and/or X-ray that is documented in the electronic Case Report Form (eCRF) by the treating physician
Exclusion criteria
* Women with Systemic Sclerosis Interstitial Lung Disease (SSc-ILD) who are pregnant, or breastfeeding will be excluded since Ofev® is contraindicated for women who are pregnant due to safety concerns * SSc-ILD patients aged \<18 years: only adults will be included in the study since Ofev® is approved for treatment of SSc-ILD in adults * Patients with a previous Hematopoietic Stem Cell Transplantation (HSCT)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to absolute Forced Vital Capacity (FVC) decline (% predicted) ≥5%, lung transplantation (indicating end stage ILD), or death | up to 10 years |
| Time to absolute FVC decline (% predicted) ≥10%, lung transplantation (indicating end-stage ILD), or deat | up to 10 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to relative (ml) FVC decline ≥5% | up to 10 years | — |
| Time to relative (ml) FVC decline ≥10% | up to 10 years | — |
| Time to lung transplantation (indicating end-stage ILD) | up to 10 years | — |
| Time to death | up to 10 years | — |
| Change from baseline in FVC (% predicted) | at baseline, up to 10 years | — |
| Change from baseline in FVC (ml) | at baseline, up to 10 years | — |
| Lung specific Quality of Life (QoL) measured as change from baseline of total, impact, and symptom scores using the Living with Pulmonary Fibrosis (L-PF) questionnaire, measured as change of baseline at years 1, 2, 3,….10 | at baseline, up to 10 years | The L-PF patient reported outcome measure (PROM) is a questionnaire designed for patients with fibrosing ILDs and developed with those patients to investigate their level of symptoms and QoL. The L-PF consists of two modules, symptoms and impacts, and five scales: symptoms total, dyspnea, cough, fatigue, impacts total, and Total L-PF score. Items in both modules have response options on a five-option numeric rating score with an anchor of 0 Not at all to 4 Extremely. Overall scores range from 0 to 100, with higher numbers indicating a greater impairment. |
| Time to worsening of Pulmonary Hypertension (PH) in the sub-population of patients with PH at baseline | up to 10 years | Worsening of PH is defined as a composite outcome of the following: 1. time to all-cause death; 2. time to non-planned PH-related hospitalization; or 3) time to PH-related deterioration identified by at least one of the following parameters: i. increase in World Health organization functional class, ii. deterioration in exercise testing; or iii. Signs or symptoms of right-sided heart failure |
| Time to absolute (% predicted) FVC decline ≥5% | up to 10 years | — |
| Final outcome for the major bleeding events (i.e., recovered or fatal) overall | up to 10 years | — |
| Proportion of patients with major bleeding that had a pre-existing risk for bleeding | up to 10 years | — |
| Incidence rate of gastrointestinal perforations in exposed vs unexposed treatment groups | up to 10 years | — |
| Final outcome (i.e., recovered or fatal) for gastrointestinal perforation events overall | up to 10 years | — |
| Proportion of patients with gastrointestinal perforations that had a pre-existing risk for gastrointestinal perforations | up to 10 years | — |
| Incidence rate of thromboembolism (arterial or venous) in exposed vs unexposed treatment groups | up to 10 years | — |
| Final outcome (i.e., recovered or fatal) for thromboembolic events overall | up to 10 years | — |
| Proportion of patients with thromboembolism that had a pre-existing risk for thromboembolism | up to 10 years | — |
| Incidence rate of major bleeding (defined as those requiring intervention or hospitalization) in exposed vs unexposed treatment groups | up to 10 years | — |
| Time to absolute (% predicted) FVC decline ≥10% | up to 10 years | — |
Countries
Switzerland
Outcome results
None listed