Rare Disease Highlight: Idiopathic Pulmonary Fibrosis

Idiopathic pulmonary fibrosis (IPF) is a subtype of interstitial lung disease (ILD) characterized by an age-related chronic and progressive inflammation and fibrosis in the lungs. [1] The cause of IPF is unknown and occurs primarily in older male adults, with the average age at diagnosis being 66 years and the incidence increasing with age. [1, 2] Therefore, this indicates the disease mostly affects the elderly population.

As part of the differential diagnostic process, a physical examination and patient history review will be performed first. Notably, IPF should be included in the differential diagnosis for adults presenting with breathing discomfort following an activity that does not normally lead to such discomfort, persistent dry cough, or Velcro-like crackles identified during a lung examination with a stethoscope. Imaging of the chest is also helpful in narrowing the differential diagnosis. Depending on the imaging data, disorders known to cause or be associated with ILD should be ruled out. Signs, symptoms, and factors associated with autoimmune conditions should also be investigated. If results from an examination of lung tissue removed from the body (lung biopsy) intend to aid treatment decision-making, a biopsy may be considered if the clinical and imaging data are unsuccessful in diagnosing the patient. [1]

Two FDA-approved medications are available for treatment of IPF: nintedanib (Ofev) and pirfenidone (Esbriet). Both medications have shown some efficacy in reducing severe respiratory events (i.e., acute exacerbations and hospitalizations), however, these treatments have limitations. [1] According to a recent analysis of the cost-effectiveness of Ofev and Esbriet, both are currently too expensive to be considered cost-effective for the treatment of IPF. [3] Potential safety concerns may also limit their use. Antiacid therapy (i.e., proton pump inhibitors and histamine H-2 receptor antagonists) is also recommended for IPF management. Additionally, nonpharmacologic options, including supplemental oxygen, lung rehabilitation comprising of education and exercises, and lung transplantation, are available. Such interventions aim to help patients live healthier and more normal lives. [1] Although lung transplantation improves survival of patients with IPF, the average survival of IPF patients after transplantation is lower than patients with other lung conditions. Specifically, only 66% of IPF patients survive more than 3 years after transplantation and only 53% survive more than 5 years. [4]

Despite available therapies and management strategies for IPF, the morbidity and mortality remain high. [1] Individuals with IPF typically suffer progressively debilitating symptoms including cough, shortness of breath, fatigue, and weight loss, which all negatively impact their daily life. Compared to the general population and patients with other lung conditions, the perceived physical and mental health over time based on patient assessment, or health-related quality of life (HRQoL), is substantially worse in patients with moderate IPF. HRQoL is further affected by age and disease severity, which is important because IPF is most often observed in older individuals. [5] IPF patients are also at increased risk for coronary artery disease, lung cancer, and high blood pressure in the lungs, which are life-threatening and emphasize the clinical severity of IPF. [1, 5, 6] Thus, new treatment interventions for IPF are required to address the high unmet medical need in this patient population.


  • Lederer, D.J. and F.J. Martinez, Idiopathic Pulmonary Fibrosis. New England Journal of Medicine, 2018. 378(19): p. 1811-1823.
  • Jo, H.E., et al., Idiopathic Pulmonary Fibrosis and the Elderly: Diagnosis and Management Considerations. Drugs Aging, 2016. 33(5): p. 321-34.
  • Dempsey, T.M., et al., Cost-effectiveness of the anti-fibrotics for the treatment of idiopathic pulmonary fibrosis in the United States. BMC Pulmonary Medicine, 2022. 22(1): p. 18.
  • Laporta Hernandez, R., et al., Lung Transplantation in Idiopathic Pulmonary Fibrosis. Med Sci (Basel), 2018. 6(3).
  • Cox, I.A., et al., Health-related quality of life of patients with idiopathic pulmonary fibrosis: a systematic review and meta-analysis. European Respiratory Review, 2020. 29(158): p. 200154.
  • Balestro, E., et al., Idiopathic Pulmonary Fibrosis and Lung Transplantation: When it is Feasible. Medicina, 2019. 55(10): p. 702.

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