Publication of an international consensus on the definition of combined pulmonary fibrosis and emphysema (CPFE) and its recognition as a distinct syndrome could spur the development of the first treatments for the condition, it is hoped.
Syndrome of Combined Pulmonary Fibrosis and Emphysema: An Official ATS/ERS/JRS/ALAT Research Statement (link) has identified that patients with CPFE “are predominantly male, with a history of smoking, severe dyspnea, relatively preserved airflow rates and lung volumes on spirometry, severely impaired DlCO, exertional hypoxemia, frequent pulmonary hypertension, and a dismal prognosis”.
It offers a research definition and classification criteria and “proposes that studies on CPFE include a comprehensive description of radiologic and, when available, pathological patterns,” to help standardise research in the field so that it can address current knowledge gaps such as the pathogenetic mechanisms in CPFE and its natural history, as well as improving methods of early diagnosis and treatment.
Dr Nazia Chaudhuri, a respiratory consultant with expertise in Interstitial Lung Disease (ILD), and Chair of British Thoracic Society ILD and Rare Lung Disease Specialist Advisory Group, told the limbic that the move will allow clinical researchers and the pharma industry to define CPFE for clinical trials to find new therapies and improve understanding of disease pathogenesis.
With no approved therapies for patients with CPFE, “often the only treatment we can offer is palliative care,” she said. As such, the prognosis for the condition remains very poor.
“Patients can develop cancer because of their smoking history and they’re also at high risk of hypertension, so often they die very soon after they are diagnosed,” she said.
Another issue is that patients with CPFE may also not be eligible for access to antifibrotic therapy.
“As clinicians we’re all aware of the combination of pulmonary fibrosis and emphysema in our patients, but treating it is a problem … patients do not have access to antifibrotics if their FVC is over 80%, and this is the group of patients that will have an FVC of over 80%,” she said.
The lack of specific treatments for the condition stems in part from the exclusion of patients with CPFE in clinical trials, because the disease is so heterogeneous and the CT features vary from patient-to-patient. Also, they’ve been excluded from trials of IPF because they have emphysema, “so these patients are very much at a disadvantage”, Dr Chaudhuri noted.
“Now that there’s a standardised definition, the hope is that pharma will include patients with CPFE in research trials, as currently they tend to concentrate on IPF alone, and it will also allow better comparison of data from different studies, she said.”
It also might encourage more research into treatments for pulmonary hypertension in CPFE, as currently there is no approved therapy for pulmonary hypertension in those with lung disease, she noted.
The statement is published in the American Journal of Respiratory and Critical Care Medicine.