Card14 Research — Tel Aviv

From the Editor…

The following article from The Jerusalem Post is frequently referenced by PRP patients and caregivers. It was written by Judy Siegel-Itzkovich and published on June 17, 2012. Please note that the gene CARD14 is not the cause of pityriasis rubra pilaris, but rather the “genetic basis” for our disease.

Dr. Jouni Uitto, professor and chair of the department of dermatology and cutaneous biology, Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia was a member of the Tel Aviv research team and is a good friend of the PRP community.

Tel Aviv scientists discover origin of pityriasis rubra pilaris

Only now, almost 200 years after it was first described, Israeli and other researchers have discovered the genetic basis for the severe skin disease pityriasis rubra pilaris (PRP), which affects people of all backgrounds around the world.

After more than a decade, the lead researchers at Tel Aviv’s Sourasky Medical Center finally identified mutations in the gene CARD14 that codes for a very important regulator of inflammation in the skin. The mutations associated with PRP lead to increased inflammatory activity within the skin, due to abnormal function of CARD14. The prevalence of the disease may be as many as one in 5,000 people.

The team – headed by dermatology department chief Prof. Eli Sprecher   and including Dana Fuchs, a doctoral student in his lab – published their paper on Sunday in the American Journal of Human Genetics. “The discovery of the PRP gene was a long and chaotic journey, with many dead ends and false routes; at some point, we were unsure whether all the participating families had the same disease,” they said in the paper.

PRP used to be considered an immunological skin disorder, but it is in fact due to a genetic defect in the epidermis, the outer layer of the skin. Also known as Devergie’s disease, it causes broad swatches of reddish- orange, scaly skin patches on the body and face, itching, serious flaking and thickened bumps around hair follicles in the scalp.

Although most patients are over age 50, it may attack people of any age and any race or nationality, with men and women equally affected.

The CARD14 protein is strongly expressed in the epidermis, said Sprecher: “This is really shifting the focus of our attention from the immune system to the skin. The skin is often perceived as a passive victim of abnormal immunological activation in inflammatory skin diseases like PRP and psoriasis. In fact, it may play a more important, and even perhaps primordial role.”

“More importantly, mutations in CARD14 are found in only a portion of PRP patients but were also recently found by a US group in a subset of psoriasis patients. No matter how you look at the data, these two sets of facts actually demarcate a new medical entity comprising individuals affected by two diseases once thought to be separate conditions, but in fact caused by the very same genetic defect,” he continued.

The discovery was made thanks to the intensive study of four affected families, three in the US and one in Holland, who suffer from the skin disease. But the disease affects Israelis as well as other people around the world.

This is not the first time that genetics is redefining disease classification in dermatology, but it may be one of the first discoveries that is likely to have major therapeutic implications, Sprecher added. “Indeed, the mechanism found to be defective in our patients suggests that they may respond better than others to drugs known to interfere with the pathway found to be hyperactive in our studies.”

Once the gene involvement is known and we know the molecular path involved in it, we can better know what medications to treat it. There are side effects involved, but it’s worth doing it,” said Sprecher.

Among the possible medications are injections of tumor necrosis tactor (TNF) antagonists and interleukin 1 antagonists.

The research team also included Ofer Sarig of Sourasky Medical Center, Noam Shomron of Tel Aviv University, Maurice van Steensel of Maastricht University, Jouni Uitto of Thomas Jefferson University, Philip Fleckman of the University of Washington and Gabriele Richard at GenDx.

Sprecher told The Jerusalem Post on Sunday that some of the people who in the past were thought to have PRP actually had a different skin disease – a type of psoriasis – caused by the same gene mutation. But not all those with psoriasis have this gene defect, he said. A sign of this was that some patients reacted positively to phototherapy (light treatment), while others’ conditions worsened with it.

Card14 Research — Tel Aviv

Phases of Clinical Research

From the Editor…

To our knowledge, no clinical trials are currently underway regarding pityriasis rubra pilaris and any pharmaceutical in the pipeline. However, there is ongoing clinical and genetic research at Thomas Jefferson UnIversity.

We need to foster more research. Perhaps the PRP community could ask PRP-savvy dermatologists to consider independent, case studies regarding the efficacy of treatment options, e.g., retinoids versus immunosuppressants versus biologicals.

The Food and Drug Administration (FDA) has described five phases of a clinical trial of a drug based on the study’s characteristics, such as the objective and number of participants. Perhaps the PRP community should adopt the spirit of “Phase 2″ and become advocates of studies that gather preliminary data on effectiveness”. There are two possible titles for such research:

(1)  PRP Efficacy Research
(2) What-works-and-what-doesn’t-work Research

For the record, here are the FDA’s five phases of clinical research.

Phase 0
Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies)

Phase 1
Studies that are usually conducted with healthy volunteers and that emphasize safety. The goal is to find out what the drug’s most frequent and serious adverse events are and, often, how the drug is metabolized and excreted.

Phase 2
Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition). For example, participants receiving the drug may be compared to similar participants receiving a different treatment, usually an inactive substance, called a placebo, or a different drug. Safety continues to be evaluated, and short-term adverse events are studied.

Phase 3
Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4
Studies occurring after FDA has approved a drug for marketing. These including postmarket requirement and commitment studies that are required of or agreed to by the study sponsor. These studies gather additional information about a drug’s safety, efficacy, or optimal use.

06.01.09  Phases of Clinical Research

How to Get Involved in Research — GARD

Editor’s Note: The Genetic and Rare Disease Information Center offers the following tutorial. If you follow all the links and search for research related to pityriasis rubra pilaris, you will find no current research.

Reports about clinical research study results are often in the news, but it can be difficult to find out about studies that are in progress. If you or a loved one has a rare condition, you may be interested in participating in a research study. This guide can help you find current research studies.

Where can I learn about clinical research studies?

A good place to start is on the National Institutes of Health (NIH) resource page, The Basics: NIH Clinical Research Trials and You. This article will help you understand the different types of studies and the process of informed consent. It also explains what to consider as you look for a study. More information on participating in research can be found at the following sites:

✽      GARD Video Tutorial: How to Get Involved in Research

✽ About Clinical Studies

✽ Clinical Trials

How can I find a clinical research study for a rare disease?

The following websites are a great place to look for studies. Start your search using the name of your condition. If you don’t find studies, try using a different name for your condition. You can also search using words that describe the condition, such as “inflammatory skin” or “genetic neuromuscular.”  If you aren’t sure which search terms to use, try using words that you see when you read about your condition. You can also search for a type of treatment, like “stem cell” or “gene therapy.”

✽ is an NIH website that has information on publicly and privately funded clinical studies in the United States and other countries.

✽      The NIH Clinical Research Study website will help you find research opportunities at the NIH in Bethesda, Maryland.

✽      The Rare Diseases Clinical Research Network (RDCRN) is made up of 22 research consortia to advance medical research on rare diseases. This network of physician scientists and their multidisciplinary teams work with patient advocacy groups to study more than 200 rare diseases at sites across the United States. This initiative is funded by the National Center for Advancing Translational Sciences (NCATS) at the NIH.

✽      NIH Genetic Modification Clinical Research Information System (GeMCRIS) is a searchable database of human gene transfer trials registered with the NIH. You can search the database by medical condition.

✽      Orphanet is a European database for rare diseases and is a resource for learning about clinical trials and research studies.

✽      CenterWatch is a searchable database of domestic and international clinical trials and research center profiles.

In addition to these sites, we suggest that you contact advocacy groups to learn more about research. Check the GARD disease pages to see if organizations are listed for your condition. If you cannot find studies after searching these sites, please contact a GARD Information Specialist.

Will I have to travel to participate in a clinical research study?
Some studies require participants to travel to a research center for evaluation or treatment. Other studies involve completing surveys or collecting a sample that can be sent through the mail.

See our guide called Help with Travel Costs for resources that offer financial assistance for transportation and lodging.

How can I learn about future studies?

You can sign up to receive alerts about new research studies at many of the resources listed above. Another option is to join a patient registry where you can provide your contact information and details about your health. Patient registries help researchers plan new studies and find people who might want to participate. They are important for research because it can be difficult for researchers to find participants with rare diseases.

Where can I find a patient registry for a rare disease?

Start your search by contacting support and advocacy groups. Check the GARD disease pages to see if any organizations are listed for your condition. The following tools can help you find patient registries.

✽      GenomeConnect is a patient registry that offers a secure place for you to privately share your genetic test results and health information with researchers, clinicians, and the scientific community. The registry aims to speed discovery regarding genetic changes and health.

✽      The Rare Diseases Clinical Research Network (RDCRN) has a contact registry to keep you informed about RDCRN research opportunities as they become available. You can also receive updates on the progress of current research projects.

✽     The Coordination of Rare Diseases at Sanford (CoRDS) registry is a centralized international patient registry for all rare diseases. The goal of the CoRDS registry is to connect patients and researchers to help advance treatments and cures for rare diseases.

✽      The Registry of Patient Registries was created by the Agency for Healthcare Research and Quality to pool information about registry opportunities.

✽     ResearchMatch is a free national research registry designed to bring together patients, healthy volunteers and researchers. ResearchMatch was developed by major academic institutions across the country and is funded by NCATS at the NIH.


06.01.08  How to Get Involved in Research — GARD

About Clinical Studies — GARD

06.01.07  About Clinical Studies — GARD

What Is a Clinical Study?

A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies. includes both interventional and observational studies.

Clinical Trials

In a clinical trial, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants’ behavior, such as diet. Clinical trials may compare a new medical approach to a standard one that is already available, to a placebo that contains no active ingredients, or to no intervention. Some clinical trials compare interventions that are already available to each other. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants. For example, investigators may give a drug or treatment to participants who have high blood pressure to see whether their blood pressure decreases.

Clinical trials used in drug development are sometimes described by phase. These phases are defined by the Food and Drug Administration (FDA).

Some people who are not eligible to participate in a clinical trial may be able to get experimental drugs or devices outside of a clinical trial through expanded access. See more information on expanded access from the National Library of Medicine.

Observational Studies

In an observational study, investigators assess health outcomes in groups of participants according to a research plan or protocol. Participants may receive interventions (which can include medical products such as drugs or devices) or procedures as part of their routine medical care, but participants are not assigned to specific interventions by the investigator (as in a clinical trial). For example, investigators may observe a group of older adults to learn more about the effects of different lifestyles on cardiac health.


Who Conducts Clinical Studies?

Every clinical study is led by a principal investigator, who is often a medical doctor. Clinical studies also have a research team that may include doctors, nurses, social workers, and other health care professionals.

Clinical studies can be sponsored, or funded, by pharmaceutical companies, academic medical centers, voluntary groups, and other organizations, in addition to Federal agencies such as the National Institutes of Health, the U.S. Department of Defense, and the U.S. Department of Veterans Affairs. Doctors, other health care providers, and other individuals can also sponsor clinical research.


Where Are Clinical Studies Conducted?

Clinical studies can take place in many locations, including hospitals, universities, doctors’ offices, and community clinics. The location depends on who is conducting the study.


How Long Do Clinical Studies Last?

The length of a clinical study varies, depending on what is being studied. Participants are told how long the study will last before they enroll.


Reasons for Conducting Clinical Studies

In general, clinical studies are designed to add to medical knowledge related to the treatment, diagnosis, and prevention of diseases or conditions. Some common reasons for conducting clinical studies include:

•Evaluating one or more interventions (for example, drugs, medical devices, approaches to surgery or radiation therapy) for treating a disease, syndrome, or condition

•Finding ways to prevent the initial development or recurrence of a disease or condition. These can include medicines, vaccines, or lifestyle changes, among other approaches.

•Evaluating one or more interventions aimed at identifying or diagnosing a particular disease or condition

•Examining methods for identifying a condition or the risk factors for that condition

•Exploring and measuring ways to improve the comfort and quality of life through supportive care for people with a chronic illness


Participating in Clinical Studies

A clinical study is conducted according to a research plan known as the protocol. The protocol is designed to answer specific research questions and safeguard the health of participants. It contains the following information:

•The reason for conducting the study

•Who may participate in the study (the eligibility criteria)

•The number of participants needed

•The schedule of tests, procedures, or drugs and their dosages

•The length of the study

•What information will be gathered about the participants

Who Can Participate in a Clinical Study?

Clinical studies have standards outlining who can participate. These standards are called eligibility criteria and are listed in the protocol. Some research studies seek participants who have the illnesses or conditions that will be studied, other studies are looking for healthy participants, and some studies are limited to a predetermined group of people who are asked by researchers to enroll.

Eligibility. The factors that allow someone to participate in a clinical study are called inclusion criteria, and the factors that disqualify someone from participating are called exclusion criteria. They are based on characteristics such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.

             How Are Participants Protected?

Informed consent is a process used by researchers to provide potential and enrolled participants with information about a clinical study. This information helps people decide whether they want to enroll or continue to participate in the study. The informed consent process is intended to protect participants and should provide enough information for a person to understand the risks of, potential benefits of, and alternatives to the study. In addition to the informed consent document, the process may involve recruitment materials, verbal instructions, question-and-answer sessions, and activities to measure participant understanding. In general, a person must sign an informed consent document before joining a study to show that he or she was given information on the risks, potential benefits, and alternatives and that he or she understands it. Signing the document and providing consent is not a contract. Participants may withdraw from a study at any time, even if the study is not over. See the Questions to Ask section on this page for questions to ask a health care provider or researcher about participating in a clinical study.

             Institutional review boards.

Each federally supported or conducted clinical study and each study of a drug, biological product, or medical device regulated by FDA must be reviewed, approved, and monitored by an institutional review board (IRB). An IRB is made up of doctors, researchers, and members of the community. Its role is to make sure that the study is ethical and that the rights and welfare of participants are protected. This includes making sure that research risks are minimized and are reasonable in relation to any potential benefits, among other responsibilities. The IRB also reviews the informed consent document.

In addition to being monitored by an IRB, some clinical studies are also monitored by data monitoring committees (also called data safety and monitoring boards).

Various Federal agencies, including the Office of Human Subjects Research Protection and FDA, have the authority to determine whether sponsors of certain clinical studies are adequately protecting research participants.

             Relationship to Usual Health Care

Typically, participants continue to see their usual health care providers while enrolled in a clinical study. While most clinical studies provide participants with medical products or interventions related to the illness or condition being studied, they do not provide extended or complete health care. By having his or her usual health care provider work with the research team, a participant can make sure that the study protocol will not conflict with other medications or treatments that he or she receives.

             Considerations for Participation

Participating in a clinical study contributes to medical knowledge. The results of these studies can make a difference in the care of future patients by providing information about the benefits and risks of therapeutic, preventative, or diagnostic products or interventions.

Clinical trials provide the basis for the development and marketing of new drugs, biological products, and medical devices. Sometimes, the safety and the effectiveness of the experimental approach or use may not be fully known at the time of the trial. Some trials may provide participants with the prospect of receiving direct medical benefits, while others do not. Most trials involve some risk of harm or injury to the participant, although it may not be greater than the risks related to routine medical care or disease progression. (For trials approved by IRBs, the IRB has decided that the risks of participation have been minimized and are reasonable in relation to anticipated benefits.) Many trials require participants to undergo additional procedures, tests, and assessments based on the study protocol. These requirements will be described in the informed consent document. A potential participant should also discuss these issues with members of the research team and with his or her usual health care provider.

             Questions to Ask

Anyone interested in participating in a clinical study should know as much as possible about the study and feel comfortable asking the research team questions about the study, the related procedures, and any expenses. The following questions may be helpful during such a discussion. Answers to some of these questions are provided in the informed consent document. Many of the questions are specific to clinical trials, but some also apply to observational studies.

What is being studied?

•Why do researchers believe the intervention being tested might be effective? Why might it not be effective? Has it been tested before?

•What are the possible interventions that I might receive during the trial?

•How will it be determined which interventions I receive (for example, by chance)?

•Who will know which intervention I receive during the trial? Will I know? Will members of the research team know?

•How do the possible risks, side effects, and benefits of this trial compare with those of my current treatment?

What will I have to do?

What tests and procedures are involved?

How often will I have to visit the hospital or clinic?

Will hospitalization be required?

How long will the study last?

Who will pay for my participation?

Will I be reimbursed for other expenses?

•What type of long-term follow-up care is part of this trial?

•If I benefit from the intervention, will I be allowed to continue receiving it after the trial ends?

Will results of the study be provided to me?

Who will oversee my medical care while I am participating in the trial?

•What are my options if I am injured during the study?

In search of PRP Patients

06.01.06  In search of PRP Patients

There are an estimated 800 Americans with PRP. Unfortunately, we have only been able to identify 188 (23.5%) who are currently “active”. This is simply unacceptable.

While the number of patients served by dermatology patient organizations for psoriasis and eczema is measured in the millions, we measure the PRP patient population in the hundreds.

Teaching Hospitals

The journey will begin with the 500-plus dermatology departments in teaching hospitals and Veteran Administration Medical Centers in the U.S.. We will confirm whether or not they have one or more patient with “active” PRP. As applicable, we will reach out by email, fax and snail mail to key contacts:

✴Department chair

✴Head of the faculty

✴Head of the residence program

✴Chief resident

✴Dermatology clinic director

Initially, we are looking for a “Yes” or “No” answer. If the answer is Yes, then the followup question is “How many are active?”

Private Clinics

According to Harris Williams & Company research, there are an estimated 7,800 dermatology practices in the U.S. of which 48 percent have three or more. The initial goal will be to research the most cost-efficient method of identifying these locations. The battle cry:

“Someone’s gotta have a list.”

American Academy of Dermatology

The AAD has a ca[tiveds audience of healthcare professionals and a myriad of communication channels. While the PRP Alliance is only one dermatology patient organization, we have access to the AAD president. Remember the “Open Door” policy? Who knows what we can accomplish if we just try.

State, Regional and Local AAD affiliated Societies

The AAD could open doors with the 90-plus dermatologist societies under it’s organizational umbrella. We want to ask their members if they have one or more “active” PRP patients.

Other Dermatology-related Societies & Associations

No stone left unturned. The following will be targeted:

✴Society for Pediatric Dermatology  (link)

✴Society of Dermatology Physician Assistants Dermatology Nurses Association (link)

✴American Society of Dermatopathology (link)

✴Society for Investigative Dermatology (link)

✴Woman’s Dermatologic Association (link)

Finding “Active” PRP Patients

06.01.05  Finding “Active” PRP Patients

by Bill McCue,
PRP Patient Advocate

Nicola Galt and I attended the Coalition of Skin Diseases Annual Luncheon at the Marriott Marquis in San Francisco on Saturday, March 21, 2015. Dr.Mark Lebwohl, incoming  president of the American Academy of Dermatology, spoke briefly and pledged to have an open door for patient support groups. His comment took me by surprise. Since May, 2013, I had been trying, quite unsuccessfully, to penetrate the AAD “non-member” shell. I was inspired to take action.

✴PRPA incorporated as a nonprofit in Texas

✴IRS grants PRPA 501(c)(3) tax-exempt status

✴PRPA joins Coalition of Skin Diseases (CSD)

✴I made an 18-month commitment to two AAD workgroups: Patient Reported Outcome Measures and Inflammatory Skin Disease/Itch Measures

✴PRPA joins the National Organization of Rare Disorders (NORD) and

✴PRPA participates in the American Academy of Dermatology Association’s Legislative Conference.

If Dr. Lebwohl hadn’t made his “open door” comment in San Francisco, I might still be just thinking about incorporating as a nonprofit corporation in Texas.

But, he did.


For those new to the world of PRP, Pityriasis rubra pilaris is an über rare skin disease. With an oft-quoted prevalence rate of one in 400,000.

The search for “active” PRPers began in ernest on November 6, 2013. The 2014 PRP Worldwide Census sought to identify PRP patients who were either “active” or “in remission.” It was our hope that identifying the dermatologists who diagnosed and/or treated the PRP patient might eventually lead me to other PRPers who were patients of the PRP savvy.

We also made an attempt to reach out to 92 state, regional and local “societies” that fall under the AAD umbrella. That was a major disappointment. Perhaps PRP was simply too rare to spark any response.

To date, we have been able to identify 1,622 patients with PRP. Sadly, only 292 (18%) are classified as “active” Data has been acquired from the following sources:

✴2014 PRP Worldwide Census

✴Email-by-email harvesting of data from 30,000 documents in the PRP (email) Support Group archive

✴The gathering of data from over 20,000 posts and comments in the PRP Facebook Support

✴2015 Core Data Census

Published in On the Road…October 2015

What is a Patient Registry?

06.01.04  Patient registry?

Source: EveryLife Foundation, Community Congress, Webinar, Sanford Research

Clinical Development for Rare Diseases: A Primer for Rare Disease Patients and Advocates

March 29, 2017

• A registry is a collection of information about individuals, usually focused around a specific diagnosis or condition. – NIH

• Contact registries collect basic emographic and contact data

– Not the same as registering for an email list

– It’s important to collect data which may be used as inclusion/exclusion criteria (birth date, geographic location, etc.)

What is a natural history study?

• Natural history is the natural course of the disease from the time immediately prior to its inception, progressing through its pre-symptomatic phase and different clinical stages to the point where the disease has ended without external intervention – FDA R01 Natural History Study RFA

– Focuses on progression

– Foundation of drug development/clinical trials

 What is the difference between a patient registry and a natural history study?

• Developing a patient registry around one rare disease does not mean that the natural history of the disease is being captured

• Natural history studies are the foundation of drug discovery/clinical trials; however patient registries are the foundation of natural history studies

• Both natural history studies and registries are important for rare diseases

– Collect information to engage community, educate public and researchers

Importance of patient registries and a natural history studies?

• Define your population

• Identify questions in which the answers would be important to researchers, physicians or patients and families

• Creating a patient registry is a great way to foster community (support group)

– A means to develop new relationships – Gives participants a voice

– Remove sense of isolation

– Identity

• A registry and natural history study creates hope for the community

• Patient registries and natural history studies create awareness

– Drive research

• Information collected will hopefully lead to publication of materials more accessible to physicians

• If physicians have more resources available they can make better decisions and better diagnose patients


• A rare disease diagnosis often means the patients are the experts

• Patient registries and natural history studies can help your organization attract the attention of researchers

• Researchers are often introduced to rare disease research by chance

• It’s best to be prepared for when an expert is found

Operational Challenges

• Patients are often motivated, but it is difficult to identify them

• Establishing partnerships with organizations • Trust is a key issue

• May need to recruit globally in order to accrue adequate patient populations (especially in ultra-rare cases)

• Data entry can be time consuming

• Critical to ensure patients aren’t lost to follow-up


• Patient registries and natural history studies present unique opportunities to foster community, educate, promote awareness and research

– Benefits outweigh the challenges

• Patientregistriesarethephonebook

• Natural history studies are the encyclopedia

• CoRDS is both

Austin Letcher

Sr. Research Associate, CoRDS 605.312.6423

About CoRDS

Mission: To accelerate research into rare diseases Goal: To establish an international rare disease patient registry for all rare diseases

• Established by David Pearce, PhD in 2010

• Create resource of contact information and clinical data on individuals diagnosed with any rare disease to enable a comparative analysis across diseases

• Connect researchers to participants interested in participating in research

• Partner with patient advocacy groups to create disease specific registries and natural history studies

• IRB approval

In Search of PRP Research

06.01.03  Finding research

In Search of PRP Research

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The truth be told, we really have no way of knowing what PRP-related research is currently underway or in the pipeline. That said, we do plan to find out.

We start the search for PRP research with the nation’s 555 teaching hospitals . Each institution has a dermatology department, faculty and clinicians. We intend to gather the following information:

1.    Gather basic information

✴Name of institution: University of Texas Southwestern (UTSW)

✴Department: Department of Dermatology

✴Address: 5323 Harry Hines Blvd., Dallas, Texas 75390

✴Phone: 214-648-3493

✴Fax: 214-648-5553

✴Other contact options: n/a

2.    Confirm leadership

✴Kim Yancey, MD, Chair, Department of Dermatology

✴Ponciano Cruz, Director, Residency Program

✴Benjamin Chong, MD, Chief, Dermatology Clinic, Parkland Memorial Hospital

3.    Confirm area hospitals served

✴UT Southwestern

✴Parkland Memorial Hospital

✴Dallas Veterans Affairs Medical Center

✴Children’s Health℠ .

4.    Confirm PRP-related experience of faculty/clinicians

✴Clay Cockerell, MD, Dermatopathologist

✴Arturo Dominquez, MD (2):  BM-8765, DK-6543

✴Heather Wickless, MD (1)

5.    Confirm PRP Research Status

✴No PRP-related research at this time (04/24/2017)

6.    Confirm online access to PRP-related

✴No PRP-related documents available on this website at this time (04/24/2017)

Path Forward

The collection of the above data is straightforward.

1.     Identify PRP patients currently under treatment at a teaching hospital in the U.S.

The PRP Alliance database reports 652 PRP patients who have confirmed U.S residency. The number being treated at one of the 555 teaching hospitals is not known. There are 594 PRP patients who have not confirmed whether or not they are U.S. residents. A portion of these will, in fact, be U.S. residents. The PRP Alliance will help these PRP patients and caregivers gather the information we seek.

2.     Augment these efforts with volunteers.

3.     As a last resort, the PRP Alliance will seek funding to collect the remaining data.

Anticipated Outcome

✴Identify previously unknown “active” PRP patients currently being treated by clinicians at teaching hospitals

✴Identify teaching hospitals and specific dermatologists with experience treating PRP

✴Advocate the referral of future “active” PRP patients to the National Institutes of Health, Genetic and Rare Disease Information Center. The PRP Alliance and the PRP Facebook Support Group are listed under “Support Organizations”