The NF Registry is delivering on its promise to be a valid and useful tool for both patients and researchers. This is the conclusion of an analysis published in the journal PLOS One in June 2017. Not only are thousands of patients from all over the world contributing their data to the Registry, it is being actively used, and appreciated, by researchers working on all forms of NF.
The authors of this peer-reviewed article include staff members of the Children’s Tumor Foundation (CTF) as well as outside experts, including a statistician and three prominent clinicians. You can read the full publication on line at no charge by clicking here.
The NF Registry is currently the largest reported cohort of people with NF at over 8,000 participants, and the only one designed to be available generally to interested investigators. The analysis used a subset of data to examine how well it matched with patterns seen in physician-entered data in terms of symptoms and patterns. It compared the proportions of people in categories (age, ancestral background, gender, geographical location) with expected proportions. Two overall conclusions emerged from the information.
One, that patients are reliable in reporting their symptoms. Therefore, this Registry is valid as a contact registry. This is something CTF has always believed, and now we can point to our results. For example, the occurrence of plexiform tumors was reported by 35% of people with NF1 in the NF Registry, which is consistent with reported of about one-third. Most other symptoms followed similar patterns.
Two, researchers are eager to use this service. Over 18 different studies and clinical trials have used the Registry to email information about studies to people who fit certain trial or study criteria. When recruitments were sent, 35.5% (mean 38.2, range 27–66%) of emails were opened. In contrast, bulk mailing services report an average opening rate of 22% for health-related mass emailings. Investigators have reported that they are pleased with the results of recruitment through the Registry and requests for use are on the increase. Observational trials, especially those for development of patient-reported outcomes, were the most common recruitments. However, we anticipate that the number of interventional (treatment) trials will grow and the NF Registry will be of increasing value as both the number of registrants and the number of trials expand.
The Foundation is committed to continuously improve the quality of our patient-entered data, so we took this opportunity to clarify certain questions, add items suggested by patients, and add a glossary and photographs of major manifestations. If you’ve already filled out the Registry questionnaires, now would be a great time to log back in to www.nfregistry.org and answer some additional questions.
Characterization and utilization of an international neurofibromatosis web-based, patient–entered registry: An observational study. By Mindell Seidlin, Robert Holzman, Pamela Knight, Bruce Korf, Vanessa Rangel Miller, David Viskochil, and Annette Bakker.