COPD: Chronic Obstructive Pulmonary Disease; HRCT: High-resolution Computed Tomography; IC: Informal Caregiver; IPF: Idiopathic Pulmonary Fibrosis; P3F: Participation Program for Pulmonary Fibrosis; P4F: Patient Participation Program for Pulmonary Fibrosis; PCORI: Patient Centered Outcomes Research Institute; PF: Pulmonary Fibrosis

Pulmonary fibrosis (PF) refers to a condition in which the lung parenchyma is diffusely scarred. This scarring leaves the lungs stiff and malfunctioning, restricted from filling to capacity and unable to transfer normal amounts of oxygen into the bloodstream. There are a number of known causes of PF, including connective tissue diseases (e.g., rheumatoid arthritis) and environmental or occupational exposures (e.g., asbestos); however, most commonly, the cause of PF is unknown. And, among all PF cases whose causes are unknown, the most common entity is idiopathic pulmonary fibrosis (IPF), a specific diagnosis, rendered only when a particular pattern of scarring is identified on high-resolution computed tomography (HRCT) images or in surgical lung biopsy specimens [1].

In 2014, PF, regardless of its cause, is not curable, and universally reliably effective therapeutic agents are lacking. Over the last ten years, amazing progress has been made in deciphering the pathogenesis of PF and thus pinpointing a number of promising targets at which to take aim with novel therapies. The enthusiasm that these discoveries have conjured is tempered somewhat by the vexing observation that the incidence of PF (at least in the U.S.) appears to be on the rise [2,3].

Whatever its cause, PF is a potentially life-shortening and, inarguably, a life-altering condition, insinuating itself into patients’ lives with activity-limiting shortness of breath, nagging cough and relentless fatigue [4,5]. Most patients with PF will need supplemental oxygen at some point in the course of their disease. It is not surprising that quality of life among patients with PF is poor compared with people in the general population [6,7]. Despite the practice of prescribing supplemental oxygen for many patients with PF, very little is known about whether and how supplemental oxygen benefits them [8]; justification lies in scientific rationale and extension of limited and often surprisingly conflicting data from the Chronic Obstructive Pulmonary Disease (COPD) literature. Clearly, this is a topic ripe for additional research.

Our team-the P₃F or Participation Program for Pulmonary Fibrosis (www.pulmonaryfibrosisresearch.org)-was recently awarded funding from PCORI (the Patient-Centered Outcomes Research Institute) to take aim at this issue. In an effort to capture potential subjects, we have created and maintain a secure contact registry of PF patients and informal caregivers (ICs) of PF patients who are willing to be contacted about our-and other investigators’ research opportunities. Here, we describe the registry and the first 102 enrollees.

Approval for the P3F Contact Registry was granted by the National Jewish Health Institutional Review Board (HS#2789), and it is registered on ClinicalTrials.gov (NCT01935726).

Any English literate PF patient (or IC of a PF patient) over the age of 18 may enroll. Consent and enrollment forms can be obtained, completed and submitted by enrollees in any of three ways: 1) Complete the forms (in PDF or HTML format) and submit online 8 at

https://dccweb.njhealth.org/sec/P3F_Swigris/Forms/PF_Registry_ PDF_Sept_2013.pdf or

https://dccweb.njhealth.org/sec/P3F_Swigris/Forms/PF_Registry_ HTML_Sept_2013.html respectively;

2) Download a PDF file of the forms at

https://dccweb.njhealth.org/sec/P3F_Swigris/Forms/PF_Registry_ Paper_Sept_2013.pdf and mail the completed forms to the P3F Coordinating Center; or

3) Call or e-mail the P3F (contact information at www. pulmonary fibrosis research.org) and request to have a hardcopy of the forms mailed to them in a packet that includes a pre-paid/-addressed envelope for returning the completed forms.

The enrollment form asks for the following information (all self-report): demographic data, contact information, data on when and how the PF diagnosis was made, supplemental oxygen use, and whether the enrollee authorizes the P3F to contact them about future research studies for which they may qualify. Registrants must sign the form either electronically or by hand to have their data stored in the Registry database; without a signature, the form is not processed. Other than the signature field, any question on the form may be left blank. Forms submitted electronically, including HTML and PDF formats, are processed by Cardiff TeleForm™ Verification software with human verification. Those submitted in paper format are entered into the database manually by qualified P3F staff. The fidelity of submitted information is not checked (e.g., we do not collect data to confirm diagnosis or clinical status). The database is HIPPA-compliant and housed on a secure server managed by the Data Coordinating.

Center (DCC) at National Jewish Health. We have funding to maintain the registry for three years but anticipate it will long outlive our funding. We have developed a protocol to allow other investigators access to group-level, de-identified data from the registry upon request. In addition, we have devised procedures to make registrants aware of other investigators’ studies after protocols for those studies have been vetted by the P3F review board. Once registrants are informed of such studies, they are free to contact these other investigators to learn more about their studies and to enroll if they so desire; the investigators would need to confirm the PF diagnosis themselves if they require greater robustness than self-report. For this manuscript, summary statistics for the first 102 enrollees were generated and tabulated. Student’s t test was used to test for significant differences between continuous variables. We considered p<0.05 to represent statistical significance. All statistics were run using SAS, version 9.3 (SAS Institute, Inc.; Cary, NC).

Twenty-three registrants mailed in hard copy consent/enrollment forms; the other 79 enrolled online. Among the 90 patient registrants, 22 mailed in hard copy forms, and 68 enrolled online. Patients who mailed in their forms were older than patients who enrolled online (68.6 ± 9.0 vs. 64.4 ± 8.4 years, t=2.05, p=0.04).

Characteristics of the 102 enrollees in the P3F contact registry are displayed in Table 1. Eighty-six patient enrollees gave their state of residence: they hail from 30 different states; 10 are from California, and six are from each of Colorado, Texas, Virginia and Washington. Among the 85 patients who gave their year of diagnosis, 67 were diagnosed in 2009 or later. The majority of patient enrollees report their PF as being idiopathic in etiology. Six of the IC enrollees are men and six are women.

Table 1: First 102 enrollees in P3F contact registry.

What makes the P3F registry unique is its inextricable link to our website, which offers PF patients, ICs and providers a wealth of disease related information that is at once trustworthy and patient-friendly, yet detailed enough to be useful to practitioners caring for PF patients. It also provides PF patients and ICs a safe online environment, where experiences are shared and practical, patient-to-patient information is exchanged. The registry, itself, is special, because it is inclusive: any patient with PF regardless of cause is welcome to enroll; ICs are also encouraged to enroll, so they can be contacted about planned future projects; enrollment is simple and can be accomplished by any one of several means; and it offers other PF researchers a database of potential subjects for their projects. We are acutely aware that many patients do not have access to a PF center of excellence and are uninformed about advances in the PF field and research opportunities. The P3F affords many of them the first opportunity to actively participate in their medical care by receiving immediate and accurate disease-related information, and the contact registry allows them the chance to play a role in elevating the PF field to a new level of understanding by participating in important research investigations.

This study was funded by PCORI. PCORI played no role in study design, development, data collection or in the generation of this manuscript.