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Table 1 Objectives and endpoints

From: Investigating significant health trends in progressive fibrosing interstitial lung disease (INSIGHTS-ILD): rationale, aims and design of a nationwide prospective registry

 

Objectives

Endpoints

Primary

To compare various PPF groups and treatment strategies in terms of characteristics and outcomes (lung function, survival, quality of life, other)

Time to progression of disease (any one of the following):

 Decrease of FVC > 10% predicted within one year

 Decrease Dlco > 15% predicted plus initiation of LTOT or permanent increase of oxygen flow when LTOT is established within one year

 Decrease of 6-MWD > 50 m within one year

 Hospitalization due to respiratory decompensation

 Death of any cause

 Change of treatment strategy (stop of anti-inflammatory or antifibrotic therapy, initiation of new anti-inflammatory or antifibrotic therapy or a combination of both)

Secondary

To assess drug utilization

 Treatment strategy (immunosuppression, antifibrotic therapy)

 Dose and dosing schedule

 Duration of treatment (persistence)

 Switches between treatments

Phases without drug treatment (with reasons)

 Reasons for drug discontinuation

 

1. To determine risk factors for progression of disease

2. To determine factors for treatment success or failure, respectively, defined as clinical, functional, and radiographic stabilization (i.e. absence of deterioration in each of the categories) and change of treatment strategy (i.e. stop/start of antifibrotic therapy; stop/start of immunosuppressive therapy)

 Survival

 Clinical symptoms

  Dyspnea

  Cough

 Lung function

  Annual DLCO decline

  Annual FVC decline

 6-min walk distance

 QoL scores over time

 Radiographic course: fibrosis on HRCT

 Therapy escalation

 Clinical events (exacerbations, hospitalisations)

Analyses stratified according to different diagnosis groups

 

3. To determine potential biomarkers, which are routinely measured in many centers, to differentiate inflammatory driven from fibrosis driven progress

Laboratory parameters:

 CRP

 LDH

 Differential blood cell count (lymphocytes, neutrophils, eosinophils, monocytes),

 BAL differential cell count (as available)

 

To assess safety

Adverse Events

Adverse Drug Reactions