Idiopathic pulmonary fibrosis: challenges and opportunities for the clinician and investigator.
Idiopathic pulmonary fibrosis (IPF) is a relentlessly progressive and typically fatal interstitial lung disease. Besides its grave natural history and prognosis, three aspects of IPF challenge clinicians and investigators: (1) recent changes in the conceptual framework and definition of IPF complicate interpretation of prior clinical investigations; (2) while most patients with suspected IPF do not undergo open-lung biopsy, clinical definitions that do not include biopsy criteria have not been validated prospectively; and (3) available treatments have not been shown to be effective. To optimize clinical care and facilitate clinical investigation, a major goal of IPF research should be to develop validated sets of clinical diagnostic and prognostic criteria. Studies have shown the diagnostic value of high-resolution CT scans and identified important prognostic variables; many of these observations await prospective validation. While previous therapeutic studies have been limited by small sample sizes, lack of a placebo control group, and insufficient attention to patient-centered outcomes, the recent study of interferon gamma-1b demonstrated the feasibility of a large-scale, multicenter clinical trial in IPF. In this article, we discuss how overcoming challenges in IPF research will enable future investigators to conduct well-designed observational studies and clinical trials, whose meaningful results will advance our understanding of IPF, its management, and its impact on patients' lives.
Swigris
Jeffrey J
JJ
Division of Pulmonary and Critical Care Medicine, Stanford University, Medical Center, Stanford, CA 94305-5236, USA. jswigris@stanford.edu
Kuschner
Ware G
WG
Kelsey
Jennifer L
JL
Gould
Michael K
MK
D002986:Clinical Trials as Topic; D006801:Humans; D018579:Patient Selection; D011379:Prognosis; D011658:Pulmonary Fibrosis / Q000000981:diagnostic imaging / Q000401:mortality / Q000628:therapy; D009626:Terminology as Topic; D014057:Tomography, X-Ray Computed