Pulmonary Fibrosis Foundation Launches National PF Patient Registry, Enrolls First Patient

The Pulmonary Fibrosis Foundation (PFF) recently announced the launch of the PFF Patient Registry, which will comprise the foundation’s first comprehensive collection of data from pulmonary fibrosis (PF) patients across America.

The first patient was enrolled last week, kicking off patient enrollment in the registry, which will be conducted at participating PFF Care Center Network (CCN) sites. The CCN is a growing national network composed of 40 medical center sites in 27 states. These centers are using a multidisciplinary, collaborative approach to deliver comprehensive care to people living with PF while offering access to important support services for patients and their families, and are focused on improving the lives of people living with PF. The overall goal for the CCN and the Patient Registry is to improve understanding of interstitial lung diseases leading to improved treatment and care of patients.

“The PFF Patient Registry is an important research tool that will help us unlock the mysteries of this devastating and fatal disease. People with nearly all types of PF will be able to participate, and in a secure, confidential manner,” Patti Tuomey, EdD, PFF president and CEO, said in a press release. “The Foundation is so grateful to the medical teams at the PFF Care Center Network sites who are enrolling patients, and especially to patients who are participating and all of the families, friends, and donors who are endorsing this Registry.”

The PFF Patient Registry will provide researchers with medical data to help them gain a better understanding of how different forms of PF progress, to determine who is affected by pulmonary fibrosis, and to assess how patients respond to various treatments. Patients participating in the Patient Registry will be able to do so through their regularly scheduled medical appointments. The registry also maintains a biorepository where patients can elect to donate blood samples. Data collected by the registry will help inform best care practices and identify potential treatment targets, while also creating a potential pool of patients available to participate in clinical trials.

Data, CT images, and biosamples provided by registry participants will be used by researchers to learn more about the causes of pulmonary fibrosis, and help with development of methods to make faster diagnoses, to identify better treatments, and to discover which treatments are best for particular patients.

“Information collected through the PFF Patient Registry will help facilitate ongoing research necessary to better understand how we can help people living with multiple types of pulmonary fibrosis, including other diseases that often include pulmonary fibrosis, such as rheumatoid-arthritis associated pulmonary fibrosis,” said Kevin Flaherty, M.D., MS, medical director of the Destination Program in Pulmonary Fibrosis at the University of Michigan and Steering Committee chairman of the PFF Care Center Network and PFF Patient Registry. “This project provides an incredible opportunity to advance progress toward a cure for pulmonary fibrosis.”

The PFF hosted a webinar, “Update on Research in Pulmonary Fibrosis and the PFF Patient Registry and the PFF Care Center Network,” on April 13, addressing different ways in which the PF community and the PFF are working to discover the causes of PF and find new treatments, and medical and patient perspectives on the future of research in pulmonary fibrosis.

Presenters at the Webinar were Flaherty; PFF Chief Medical Officer Gregory P. Cosgrove, M.D.; and PFF Senior Medical Advisor Associate David J. Lederer, M.D., MS, an associate professor of medicine and epidemiology (in pediatrics) at Columbia University Medical Center in New York whose research focuses on the related areas of enhancing the understanding of the causes of lung fibrosis in humans, and improving outcomes after lung transplantation. For more information, visit http://pulmonaryfibrosis.org/life-with-pf/pff-educational-resources/webinars

The establishment of the PFF Patient Registry has been partly enabled by the support from founding partner Genentech and visionary partner Boehringer Ingelheim, as well as from the Chicago Community Trust, the Rattner Family Foundation, the McQuaid Family Foundation, and Biogen. Additional donations will support expansion of enhancements to this research tool.

“The generous investments funders have made to date have allowed us to establish the PFF Patient Registry and enroll the first of at least 2,000 patients,” Tuomey said. “Further funding will be important in order for us to fully realize research opportunities through Registry data.”

The PFF Summit 2017: From Bench to Bedside, its fourth biennial international healthcare conference, will be held in November 2017.

Pulmonary Fibrosis

According to the PFF, idiopathic pulmonary fibrosis (IPF) is a devastating progressive disease in which lung tissue becomes thickened, stiff, and scarred over time. The development of the scar tissue is called fibrosis. The word pulmonary means lung, and the word fibrosis means scar tissue – similar to scars you may have on your skin from an old injury or surgery. So in its simplest sense, pulmonary fibrosis means scarring in the lungs.

As the lung tissue becomes scarred and grows thicker, the lungs lose their ability to transfer oxygen into the bloodstream. As a result, the brain and other organs don’t receive enough oxygen, and low oxygen levels (and the stiff scar tissue itself) can cause shortness of breath — particularly when walking and exercising — and cough, which are the most common symptoms of pulmonary fibrosis. Symptoms may be mild, but as the disease progresses, patients may become breathless while taking part in everyday activities, even eating.

Due to a lack of oxygen in the blood, some people with IPF may also have clubbing of the fingertips — a thickening of the flesh under the fingernails, causing the nails to curve downward. This is not specific to IPF and occurs in other diseases of the lungs, heart, and liver, and can also be present at birth.

Pulmonary fibrosis isn’t just one disease, but a family of more than 200 different lung diseases that look very much alike and fall into an even larger group of diseases called “interstitial lung diseases.” In some cases, doctors can determine a cause of the fibrosis, but in many instances, there is no known cause. In that case, (and when certain pathologic or radiographic criteria are met), the disease is called IPF. There is no cure for IPF, and presently, there are two FDA-approved treatments for the condition in the U.S.

About 10 to 15 percent of those with an idiopathic form of pulmonary fibrosis have another family member afflicted by the disease. This is called familial pulmonary fibrosis (FPF) or familial interstitial pneumonia (FIP). A number of genes and genetic variants have been identified that are associated with PF development, but frequently genetic tests are not performed when pulmonary fibrosis is diagnosed, as the significance of these genetic abnormalities is only beginning to be understood.

Every individual diagnosed with pulmonary fibrosis has a unique experience with the disease, since there is no “standard” or expected clinical course. Some patients remain in stable condition for extended periods of time; others may experience a rapid progression of symptoms; while others may experience a stepwise deterioration over time, fluctuating between periods of stability and worsening symptoms. Accordingly, PF treatment strategies are highly individualized, based upon the patient’s medical history and other conditions.

Pulmonary fibrosis is now the leading indication for lung transplantation in the U.S. The PFF says pulmonary fibrosis accounted for nearly half of all lung transplants performed in 2013. Transplantation can improve both longevity and quality of life in patients who have no other significant health problems. In the past, it was uncommon for anyone over the age of 65 to receive transplants, but as surgical techniques and outcomes have improved, more centers are performing transplants in older people.

PFF acknowledges that the fight for improved treatments and an eventual cure for pulmonary fibrosis will be a long one, with future partnerships and ongoing donor support needed for it to succeed.

The PFF, a nonprofit resource for pulmonary fibrosis patients, their families, and the medical community, has a four-star rating from Charity Navigator and is a Better Business Bureau-accredited charity. For more information about the PFF Care Center Network and the PFF Patient Registry, visit http://pulmonaryfibrosis.org.