Community Call Recording – A Unique Adult Experience December 28, 2015 by Kelly Helm Guest Speaker Dine Watson Historically, our adult community has sometimes felt neglected because of the attention that pediatric patients receive. NKI is working hard to offer more and better opportunities for our adult population. This webinar recording highlights the unique experience that adult Nephrotic Syndrome patients have while juggling their health and day-to-day life. Washington Post contributor and adult FSGS patient Bernadine Watson is a guest host. Watch A Unique Adult Experience here: https://www.youtube.com/watch?v=j-1d4NciOmo
Strength in Numbers – completing the picture one story at a time December 15, 2015 by Kylie Karley Puzzles can be frustrating! The more complicated the picture, the harder the puzzle. And nothing is more frustrating than missing puzzle pieces! Nephrotic Syndrome is an especially complicated puzzle. Why do some patients respond to steroids while others don’t? What role to our genes play in how these diseases progress? How come some people get FSGS as children while others are diagnosed as adults? Like puzzle pieces, patients with Nephrotic Syndrome are each unique and vitally important. We need every single piece of the puzzle to make the picture complete. Each patient story gives nephrotic syndrome researchers good information that will help us understand these diseases. Researchers have set up a variety of studies and databases to collect and organize these patient stories. NephCure Kidney International supports all of these efforts because each adds to the big picture. Our goal with this newsletter, is to help you understand the different efforts, what makes them unique, and how you can get involved. Cure Glomerulonephropathy (CureGN) The Midwest Pediatric Nephrology Consortium The NephCure Accelerating Cures Network The NephCure Kidney Network Patient Registry The Nephrotic Syndrome Study Network (NEPTUNE) *These efforts are all US-based. For more information about similar international projects, see the UK-based National Registry of Rare Kidney Diseases (RaDaR) and INSIGHT (Insight Into Nephrotic Syndrome: Investigating Genes, Health and Therapeutics), a Canadian study of pediatric nephrotic syndrome. For frequently asked questions about getting involved with one of these projects, click here. Thank you to Hailey Desmond, Corinna Bowers, Tina Mainieri, Dr. Laura Mariani, Dr. Larry Greenbaum, and Dr. Jonathan Hogan for their help with creating this “Strength In Numbers” content.
It All Adds Up – A Doctor Explains “Strength in Numbers” December 15, 2015 by Kylie Karley We recently caught up with Dr. Jonathan Hogan from University of Pennsylvania who explained why he thinks it is important for patients to add their voices to consortia studies and patient registries. See below for our Q&A with Dr. Hogan! Dr. Jonathan Hogan from the University of Pennsylvania Q: How would you define consortia? JH: Consortia are groups of institutions, businesses and individuals that come together to achieve a common goal. In the case of health and medicine, consortia are often formed to promote the awareness, understanding, research and treatment of certain diseases. Q: There are several groups of researchers building this consortium model (and patient registries )- why is it important for all of these researchers to be collecting patient data? JH: Obtaining data is the most important step in research. Without data, researchers cannot answer questions that they have about the diseases they are studying. Patient registries are one of the most effective ways of obtaining data. This is particularly true for rarer diseases, for which finding patients and obtaining the data on how their diseases has affected them and responded to treatment (for example) can be very challenging. Q: Why should patients care about the consortia that are happening? JH: As the theme of this article suggests, there is strength in numbers. This statement is true for many reasons. The larger the amount of affected patients, the larger the effect on public health. This draws attention to the disease and the importance of getting behind research. With more patient data, more questions can be asked and answered about these diseases. There is only so much attention and resources (money) to be spread around in disease research. The more “visible” the disease is (through participation in research studies, registries, fundraising, advertising their disease and lobbying lawmakers), the more attention (and money) will be devoted to it. Q: How can patients get the most out of their experience while participating in a consortium? JH: Being engaged with the consortium allows patients to get the most out of their experience. In the age of the internet and social media, there are more options than ever for patients to get involved. This may include participating in patient registries, advertising about their disease and need for research through word of mouth and social media, and attending events in person that focus on their disease. Support groups also are a great way for patients to interact with other members of the community who are affected by these diseases and care about making a difference. Q: In the big picture, can you briefly describe how participating in consortia contributes to new therapies and treatment options? JH: Participating in consortia contributes to new therapies and treatment options by providing more data to both ask and answer questions for a specific disease. Without patients contributing their information and being aware of state-of-the-art research for a disease, it is impossible to move fields forward in the understanding and treatment of these disorders. Consortia provide a common arena where patients and researchers can convene to accomplish these goals. This is particularly important for rare diseases, for which finding enough patients to treat with new therapies is very challenging. Q: What do you see for the future of this “big data” being collected in consortium? JH: There is enormous potential in the collection of “big data”. Larger numbers of patients contributing their information allows for the use of novel approaches to the understanding and treatment of diseases. In the age of the internet and social media, consortia have a unique opportunity to collect and analyze data from patients worldwide.
The Unique Adult Experience December 15, 2015 by Kylie Karley Living Your Best Life with Nephrotic Syndrome By Bernadine Watson Contributor to Washington Post, FSGS patient, wife, mother CLICK HERE to watch “The Unique Adult Experience” Webinar held last week and hosted by Bernadine In 1984, I was thirty-three years old and dealing with all of the stresses of being a single, working mother of an adolescent boy. During a routine physical, my doctor added another worry to my plate. “You have excess protein in your urine” she announced at the end of our visit, sounding somewhat surprised. She had been my doctor a while and I was always very healthy. “I suggest you see a nephrologist,” she continued. “A small amount of protein in the urine is normal, but the amount you have could be a sign of kidney disease.” Kidney disease, I thought to myself. But I’m only in my thirties and feel fine. I had every reason to feel a bit fatigued, given my responsibilities, but I didn’t believe I could have a serious illness. As far as I knew kidney disease didn’t run in my family. Most important, I didn’t have time for kidney or any kind of disease. Within a few weeks, however, I saw a nephrologist and was diagnosed with a kidney disease I had never heard of and could hardly pronounce: Focal Segmental Glomerulosclerosis or FSGS as it’s called by those in the know. Thirty-one years have passed since my diagnosis. During that time I’ve had what one of my doctors calls “a pretty good dose” of FSGS. I’ve had two kidney transplants, spent five years on dialysis and suffered the pain, complications, inconvenience and sometimes embarrassment associated with having the disease. I know the terrible leg cramps, fatigue and nausea that can accompany dialysis; and the awful swelling, weight gain, bone loss and anxiety that can result from taking anti-rejection medications. There have been times when the surgeries, pills, scars and doctors’ appointments have seemed unbearable. The truth is, FSGS has been a constant companion for nearly half of my life. How in the world can you live your best life when you have a disease that can make you miserable and has actually tried to kill you? Honestly, there are days when I know I won’t be at my best. I may not feel well physically or may not be emotionally ready to deal with the world. On these days, I just do my best to muddle through. However, I find that I have fewer “bad” days since I’ve learned to make peace with FSGS. I’ve decided I want to live as well as I can, despite the fact that I have a chronic illness. Over the years, I’ve developed a few strategies for dealing with the physical, mental and emotional challenges of this disease. There is no magic involved but on most days these strategies help me do pretty well. 1. Put Yourself First. This may take a while to learn and may sound impractical or even selfish. We all have jobs, families and other responsibilities that come with everyday life. Many of us put others’ needs above our own, even when we don’t feel well. I believe, however, that I am no good to anyone if I am always worn out or sick. Putting yourself first means, asking for help from those around you; turning down request from others if you have too much on your plate or are not feeling your best; taking time off from work if and when you have the opportunity; avoiding people and situations that cause you stress and finding quiet time for yourself. You’ll be amazed at how much better you’ll feel if can do even a few of these things. 2. Take Full Responsibility For Your Health Try handling your own health care like you would handle care for any other loved one, like our mother or our child. Come to medical appointments on time and prepared to discuss issues that concern you. You and your nephrologist should be on the same team and have the same goal: keeping you as healthy as possible. Act like a member of the team. Ask any questions you have about your treatment. Ask for help in accessing any specialist you need to help you deal with this disease. Over the years, I’ve asked for and received referrals to nutritionists, orthopedists, physical therapists, mental health counselors and a psychiatrist—all to treat conditions related to FSGS. Make sure you always have enough of your medications on hand and take them as prescribed. Be insistent with you medical team about responding to your requests for refills, since you, not they, will be the one who has to bear the consequences of missing doses of your medications. Take the time to understand the side effects of your medicines and get help from your doctor in managing these effects. Don’t suffer if you don’t have to. 3. Take Good Care of Yourself Taking good care of yourself is always a good idea, but especially if you have a chronic illness. One of my favorite things to do for myself is to take naps. Naps help boost my mood and combat fatigue caused by my disease and some of the medications I have to take. While I realize that physical activity isn’t everyone’s favorite thing, try to find exercises that you enjoy or at least can tolerate. I’ve found exercise to be a necessity, not a luxury. While I’ve always been physically active, since being diagnosed with FSGS, I’ve become more intentional about my fitness routine. I love yoga and find that it has made me stronger and lifts my spirits. I also lift weights and ride a bike to support my heart and bones. Walking is a terrific activity that is easy and has many benefits. Exercise will help you look and feel better and can help counter some of the weight gain caused by transplant medications. Of course watching what and how much you eat is critical. I struggle with this all the time. Prednisone makes me ravenous and sometimes I find myself eating something before I even realize what it is. Fortunately, exercise helps. So does reminding myself that my diet and weight have a direct impact on health. 4. Enjoy Your Life Living with Nephrotic Syndrome is tough and can take a significant physical and emotional toll. Try to remember that this disease is something that happened to your body. You are not the disease and you did nothing to deserve it. Find ways to enjoy your life and do the things you like to do. Listen to music, read books, go to the movies, volunteer at your church or in the community, get dressed up and go out with your friends. I find that doing the things I love reminds me that I am still me, in-spite of FSGS. I want to live my best life!
The NephCure Kidney Network Patient Registry (NKN) December 15, 2015 by Kylie Karley GOAL: To collect patient-reported information from NS patients all over the world in a centralized location that will be easily accessible for researchers. By encouraging patients to join and share their health information themselves, the NKN tries to empower the patient community and engage them meaningfully in the research process. The NKN can also help connect patients to clinical research opportunities that they may be interested in. WHO CAN PARTICIPATE: Any patient with a Primary Nephrotic Syndrome condition can participate, from anywhere in the world. In fact, the NKN has participants from 33 countries! COMMITMENT: Participants spend 30-45 minutes after consenting to share their information answering surveys online about their medical history, their preferences about research, how they feel, and their family background. Participants are then asked to update their information every 6 months, and can choose to receive information about research studies that they may be interested in. WHY IT IS IMPORTANT: The NKN is a useful tool in connecting patients to research studies, thus allowing research to happen faster. The information provided by patients in the NKN can also influence what research is conducted, making sure that patients’ have a say in the process. Finally, the NKN is a patient-powered research network, and encourages patients and their families to pay attention to research and be more involved. We are currently close to reaching 500 participants in the NKN- register today and help us meet that goal by the end of the year!
NephCure Accelerating Cures Institute (NACI) December 15, 2015 by Kylie Karley GOAL: To create a thorough database of clinical information (Electronic Health Records) from all patients that suffer from a primary NS condition. The information in this database can be used to find the best standards of care and the best treatment options for patients living with these diseases. It will also speed up the development of new treatments. WHO CAN PARTICIPATE: Any patient seen at a NACI site can consent to share their electronic health records. Currently, there are NACI sites in Ann Arbor, MI, Charlotte, NC, and Los Angeles, CA. Over the next few years, NACI will expand to include 30 sites globally. Check here for updates about new sites. If you are not near a NACI site, being a part of the NephCure Kidney Network is a step you can take to help reach the same goal! COMMITMENT: Once patients agree to share their electronic health records with NACI, the NACI team does the rest! The commitment for patients is minimum. WHY IT IS IMPORTANT: NACI is a very inclusive study – any patient with a Primary NS condition can share their information. By having patients share their electronic health records, doctors and researchers have access to a very detailed story of how the disease has progressed over time. They will be able to see which treatments work best for different types of patients. This information can be used to create research projects that are meaningful to patients. The information collected through NACI will also help us count the number of people living with a Primary NS condition and speed up the drug development process.
Midwest Pediatric Nephrology Consortium (MWPNC) December 15, 2015 by Kylie Karley GOAL: To promote high quality standards of care in pediatric nephrology by encouraging doctors to work together and think about and discuss research that will benefit patients. MWPNC also works to encourage younger doctors to be involved in research and high quality patient care. WHO CAN PARTICIPATE: Doctors from 63 medical centers across the country (not just the Midwest, despite the name!) participate in this network to ensure high quality pediatric care. See if your doctor is part of the network by clicking here. There are many MWPNC sponsored studies seeking patient volunteers that can potentially benefit the care and treatment of NS patients including : Randomized Trial Comparing Rituximab Against Mycophenolate Mofetil in Children Wtih Refractory Nephrotic Syndrome (RAMP) Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome MWPNC also sponsors a patient registry specific to pediatric nephrology patients, called Childhood Nephrotic Syndrome Observational Study (CNOS). CNOS collects electronic health records from patients that agree to share them and will help advance the knowledge of how NS progresses in children. COMMITMENT: Doctors commit to meeting twice a year for 2 days at each meeting to specifically discuss pediatric nephrology research. Commitment for patients varies depending on the opportunity that they choose. Each study asks for a different commitment, which may include regular visits to a medical center, taking a medicine that is being tested for effectiveness, and additional bloodwork or other procedures. CNOS asks for participants to share their electronic health information and spend time, either in person or over the phone, answering additional questions about your health and how you feel. WHY IT IS IMPORTANT: As a result of the MWPNC collaboration, 32 articles have been published, with 9 of them being published this past year. MWPNC has sponsored and published studies about vitamin D, blood clots, acute kidney injury and patient reported outcomes in children with nephrotic syndrome. The collaborative effort of the MWPNC ensures that nephrologists are aware of the most current research, and that opportunities are available for doctors to work together to find answers to questions that will help make better treatment and better patient care become a reality.
Cure GN (Cure Glomerulonephropathy) December 15, 2015 by Kylie Karley GOAL: CureGN is a large multi-center, long-term observational study of patients with Nephrotic Syndrome and other glomerular diseases, which aims to improve the diagnosis, treatment and outcomes of patients affected by these diseases. This study, which began patient enrollment from 64 clinical sites in December, 2014, will follow 2,400 patients with Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), membranous nephropathy (MN), or IgA Nephropathy/Henoch Schonlein Purpura. WHO CAN PARTICIPATE: Adults and children seen at a CureGN site (click here for list) with one of the four diseases (FSGS, MCD, MN or IgA Nephropathy/HSP) diagnosed by kidney biopsy may be eligible. The kidney biopsy must have happened within the past 5 years. COMMITMENT: Participants spend an extra 15-20 minutes at their normal nephrologist appointments and answer questions about health history and how they are feeling. Participants are seen at least once per year in-person and additional study visits can occur by phone. For each in-person visit, participants are asked to give a blood and urine sample. WHY IT IS IMPORTANT: CureGN is currently the largest network of clinical sites enrolling patients in a single observational study for Nephrotic Syndrome. CureGN plans to follow these patients for many years to understand long term impacts of the disease and what treatments are effective.
FAQ’s about the Consortia Research Networks December 15, 2015 by Kylie Karley Q: Other than providing data to researchers to fuel breakthroughs, how does my participation in one of these networks benefit me in the short term? A: An added benefit of donating your data to research is that the process enables you to get organized and take control of your health and disease. By tracking and recording your information in an organized way, you can become more empowered and feel more confident in the decisions that you make. Another perk of being a part of a consortia is that you gain access to leading researchers who are at the forefront of the field. Q: How do I know if I am participating in one of these research networks? A: To participate, you should have gone through an informed consent process, in which you reviewed a document (possibly with a study coordinator) that explained the risks, possible benefits, and requirements for participation. After the consent process, you likely answered many questions about your Nephrotic Syndrome history, and you may have even given a blood sample! Sometimes, you answer these questions at the end of your normal appointment, or you might go to an appointment just to answer these questions. If none of this sounds familiar to you, click here to learn about consortia opportunities and a site near you, or register with the NephCure Kidney Network at www.nephcurekidneynetwork.org. Q: I’ll participate, but I want to remain anonymous. Is that possible? A: Privacy is extremely important, and many steps are taken to ensure that your privacy is protected. All research studies have specific sets of rules that are followed to protect your privacy, and these rules are set forth and reviewed by an Institutional (or Independent) Review Board (IRB). You can learn more about IRBs here, and you can also talk to the study coordinator to learn exactly what steps they will take to ensure your privacy. Q: I’m not sure I have time to do this- how long does it take? A: All efforts are made to ensure that it is relatively easy for patients to participate in these opportunities. Many times, enrolling in a consortia can be part of your regular appointment- adding 15 -30 minutes to your normal appointments. Sometimes, you can answer questions over the phone to fulfill your commitment. Some opportunities require minimal input from you after the consent process, such as NACI. Other opportunities can be completed on your own time, such as the NKN. Whatever your time restriction is, there is definitely an opportunity for you to contribute to finding better treatments and cures. Q: Is there a cost to participate? A: There is no cost- researchers just need your participation! Q: Why is it important for me to do this? A: Nephrotic Syndrome is very complicated, and is different for every person. Researchers need to have the most complete collection of information possible to be able to draw accurate conclusions that will benefit the patient community. Participating in one of these opportunities will fuel breakthroughs in the research process that can ultimately lead to better treatments or cures.
The Unique Adult Experience Webinar December 1, 2015 by Kylie Karley “As far as I knew, kidney disease didn’t run in my family. Most important, I didn’t have time for kidney or any other kind of disease.” Dine Watson, author of the Washington Post article “Kidney disease? I was only 33 years old, and I felt fine” will be featured on our next webinar, The Unique Adult Experience. Diagnosed with FSGS in 1984, Dine’s story will resonate with all adults living with Nephrotic Syndrome and the related diseases. During this webinar, Dine will offer insight about all aspects of living as an adult with kidney disease, and also how to hope and how to find a community. Register now to reserve your spot! Hope to see you there! Questions? Contact Chelsey at 610-540-0186 ext. 19 or cfix@nephcure.org