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The Study of Neurofibroma Growth in Adults with NF1Frequently Asked Questions About the Tumor Growth StudyWhat is this study about?We invite you to join a research study of neurofibromatosis type 1 (NF1) in adults at the National Institutes of Health (NIH). We are trying to answer a simple question: in adults, how fast (or how slow) do skin neurofibromas grow? By answering this question, we hope to lay the groundwork for future trials of new drugs to control the growth of neurofibromas. We also hope to find genes that control the growth of neurofibromas in NF1. In 1990, scientists found that mutations (or changes) in the gene called NF1 cause neurofibromatosis type 1. Since that time doctors and scientists have learned a lot about the disease. However, many questions about NF1 still remain. Some questions may come to your mind. How many skin tumors will I have? How fast will they grow and how large will they become? With this study, we are trying to learn how fast or slow neurofibromas grow, and how often they appear. We also are looking for the genes that control the growth rate of the tumors. The results of this study may help us design future trials to test new drugs to treat the tumors. If we can find genes that control the growth rate of the tumors, we hope to use those genes to predict the approximate number of tumors a person with NF1 might have. Why should I join the study, or, what's in it for me?In NF1, there is a long history of cooperation between patients and doctors. Much progress has been made as a result of patients and doctors working together. People join research studies for many reasons. Three common reasons include:
New information, treatments and in the future, a cure, for neurofibromatosis type 1 will only come from people with NF1 and doctors working together. Animal models of the disease (like mice) are helpful up to a point, but there is no replacement for studying the disease in a human being. What we are trying to learn with the study?There are three goals in this study. We hope to understand: 1) How fast (or slow) dermal neurofibromas (skin tumors) grow in NF1, 2) How often new tumors appear, and 3) What genes are involved in the growth of neurofibromas. Ultimately we hope that we can use this information to design trials to test new medicines in NF1 and use genes to predict the number of tumors a person with NF1 might have. What we are NOT trying to do?
What is the approach of the study?We are using three different ways to take pictures of small areas of skin of people with NF1. From the pictures we can make very precise measurements of the number and size of the tumors. Over time, we can track how much the tumors change in size, and how many new tumors appear. By doing this we will be able to more accurately say how fast the tumors grow, and how often they appear. These measurements will be important in any future study of a medication to control the growth rate of the tumors. We are also trying a new approach to find genes that may control the growth of the tumors. Our method looks at how many copies of a gene are made in a cell (called gene expression). We think that big differences in the level of some genes might make a difference in the seriousness of the disease. Who can participate?To be in our study, a person must:
What is involved in the study?After we determine if you are able to join the study, we will ask your help in collecting your medical records. We will help arrange travel for you (and family members, in some cases) for a 1-day visit to NIH in Bethesda, Maryland. We will ask your help in contacting your parents to enroll in the study. There will be some forms for you to sign. At the NIH, we will ask you questions about your medical history and do a physical exam (like in any doctor's office). In addition, we will draw some blood, take some pictures of your skin using several different cameras and remove a neurofibroma and a small piece of normal skin.We may also remove a cherry hemangioma from the skin. There will be time to discuss the genetics of neurofibromatosis type 1 and answer your questions. We can help you find doctors near you for follow-up.At the end of your visit we will discuss our findings with you and make arrangements with you for the return visits. We will send you a letter summarizing your visit to you. What about my privacy?Your medical information will be stored in a password-protected database and in a locked filing cabinet. Although we may share medical information with our colleagues, your name will not be used. Instead a code will be used that is unrelated to your name. The "key" to the code will be stored in a locked drawer in the office of the principal investigator (Dr. Stewart). What is the cost of the study?The visit and all testing are free and there is no cost to you, your family or your insurance company for this study. What about travel?We will pay your travel expenses, subject to NIH regulations. If you live locally (near the NIH) we cover your expenses to come to NIH for the first and follow-up visits. If you live further away, we will pay for your transportation to NIH (plane, car, train, etc), hotel, meals and expenses. Travel must be arranged by NIH. Will I get paid for joining the study?No, participants in the study do not receive any compensation other than travel and lodging expenses (subject to NIH regulations). There may be no benefit to you but hopefully the results from the study will benefit families with neurofibromatosis type 1 in the future. Is there a way I can find out how my participation in this study made a difference?Yes. Watch this website for updates on publications and talks given by Dr. Stewart and Sarah Coombes as a result of this study. Rules on patient privacy and confidentiality make direct contact with study participants via mass e-mail difficult. We also welcome suggestions on how to make joining and participating in the study easier for patients and families. How do I enroll?You may contact either Sarah Coombes (genetic counselor) at coombessl@mail.nih.gov or Dr. Douglas Stewart (principal investigator) at drstewart@mail.nih.gov. Top of page Last Updated: April 6, 2009 |
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