Professor Anne Holland
Patient reported outcome measures (PROMs) in ILD are readily accessible at low cost and should be used more in research and clinical practice, respiratory physicians have heard.
TSANZ president Professor Anne Holland told the TSANZSRS 2024 meeting that the corollary is that when patient reported outcomes are measured, clinicians have to take action on the results.
She said PROMs were not just checklists to be ticked off in the clinic and then put away for comparison at subsequent visits.
“So if you’re measuring something that tells you that a patient is having substantial distress for instance, and may have a diagnosis of depression if we looked into it further, we have to be willing to act on that.”
“They are actually really useful tools in that patients are trying to tell us about how they feel and we need to be paying attention to that.”
Professor Holland, Head of Respiratory Research at Monash University and Alfred Health in Melbourne, said PROMs alone will not improve patient outcomes.
“They’re not a treatment so it is what we do with the information that matters both in trials and clinical practice. We use them to understand the burden of disease for our patients. They’re very useful for documenting treatment responses. But hopefully they also prompt us to develop better interventions because we certainly need that and to deliver patient centred care.”
She said patient reported outcome measures mattered because the burden of symptoms such as breathlessness, cough, fatigue, depression and chest pain was very high
“We also understand from our patients that [their disease] places a strain on their relationships and reduces their independence. It can cause financial stress, social isolation, anxiety, panic, and certainly frustration and anger.”
“It also matters because many patients will unfortunately feel worse over time.”
She said symptoms such as dyspnoea have also been shown to be associated with poorer outcomes.
Unfortunately, enthusiasm for the use of PROMs has waned – possibly because most phase three clinical trials for ILD pharmacotherapy have not been able to demonstrate an improvement in PROMs.
“So of course, the question then is, is it because the treatment is not making any difference which is certainly possible as it’s a treatment that is designed to prevent progression of fibrosis? Or is it that we actually don’t yet have the right tools that can pick up small and important changes in these measures?”
Professor Holland said in contrast, trials of pulmonary rehabilitation have been able to demonstrate fairly substantial changes in measures such as health-related quality of life and dyspnoea.
“It is worth pointing out though here that most of these patient reported outcome measures used in pulmonary rehab trials have not been disease specific or have been developed in other lung diseases such as COPD and co-opted for use in this space.”
She said previous work on developing a progressive fibrosing ILD-specific PROM [link here] had initially identified seven key signs/symptoms and 43 proximal and distal impacts for consideration – a complexity which did not bode well for a short and simple PROM.
It was however able to propose a final model of the three most relevant symptoms – shortness of breath on exertion, fatigue and cough – and three relevant impacts included effects on physical functioning, activities of daily living and emotional wellbeing.
Professor Holland said some ideal characteristics of a PROM including language and cultural validation still required more work in ILD.
“And often we don’t know what a clinically significant change is…we know that in ILD that might be different for improvement and deterioration, and sometimes we don’t have that information.”
She said developing a new PROM was not for the faint hearted and for now, the options were the 34-item SGRQ-1 (an IPF-specific version of the SGRQ), the 15-item K-BILD and the 44-item L-PF [link here].
Only the K-BILD and L-PF were concordant with FDA/EMA guidance on PROMs.
Professor Holland said a recent 2024 study [link here] of the effects of nintedanib on symptoms in patients with PPF using the L-PF had shown nintedanib reduced worsening of dyspnoea, fatigue and cough and the impacts of ILD over 52 weeks in patients with PPF.
“So this is, I think, a very encouraging development because we haven’t seen anything as clear as this in terms of change in symptoms and impacts with any of our pharmacotherapy up until this point, so bodes well for the future.”