I was hired by the MPN Research Foundation in May 2016 to spearhead the development and launch of the first ever patient driven registry. At the time I was working for another disease foundation managing research projects to find a treatment or cure for an ultra orphan disease that affects less than 100 people in the US. While not life threatening, the disease (Usher Syndrome type 3) causes progressive loss of hearing and vision over many years until the patients rely on cochlear implants or tactile sign language to communicate and are completely blind. In working with the patients afflicted by Usher syndrome, I began to understand the disconnect between the patient experience and the scientific process being undertaken in the labs and universities around the world. In cases where a disease progresses slowly or where there may be triggering events that cause progression, are there ways that patients can help the scientific process along?
In the case of MPNs, the answer is a resounding “yes”! While factors such as age, lifestyle, diet and environment have not been conclusively proven to contribute to progression, the general consensus is that tracking these data along with the symptoms and treatment of a group of patients over time (a longitudinal study) can be helpful to research community. Every day decisions are made in pharmaceutical companies, university laboratories and research foundations like the MPNRF on where to allocate resources and personnel. Why shouldn’t the patient community have a greater say in this process? What we needed was a tool to gather individual MPN patient experiences, aggregate the data of many patients and share meaningful outtakes with researchers to focus their efforts and move the process of drug discovery and approval along more quickly.
Through the recently launched registry myMPN the MPN Research Foundation provides patients with an opportunity to share information about their day to day experience with an MPN with researchers. First, an introductory survey collects basic demographic, diagnosis and treatment information for the patient. The “How do you feel today” survey records symptom data in real time and is available to be taken as often as a participant wants. So if a patient feels great one day, they can report that but then when they feel not so great the next day, they can complete a new HDYFT survey. The “Health Event” survey is intended to be used when someone has (as the name suggests) an event such as a change in diagnosis, medication change, hospitalization, thrombotic or bleeding event to report. As participants go through some questions, they will be able to see how their responses compare to others in myMPN.
When a user first logs into myMPN, they are asked to set up their privacy of their account. At that point they will find videos of three patients who volunteered to be video-taped talking about their experience with their disease, their feelings about myMPN and the privacy settings they selected in their myMPN profile. Patients can start with the privacy settings that these “guides” have shared or they can go through each privacy setting individually to select their choices. The privacy settings are fully customizable so that patients control how, if at all, the data they enter into myMPN is shared with the research community.
The registry tool was designed with long term data collection and natural history study objectives in mind but also has some great functionality and convenience. Aggregated with data from others, a single patient’s experience can make a meaningful difference in the research community! For the user, it will become a repository for medical records as well as a journal or diary where patients can record how they are dealing with fatigue, depression, itching and other common symptoms from day to day. When it comes time for a doctor visit, myMPN offers participants the ability to download their records and survey responses to take with them to the appointment. In the year ahead, enhancements to myMPN’s platform will even enable patients to securely upload medical records and test results to their registry profile.
I am humbled by the MPN patient community’s response since I began working on myMPN! We never had to look far for volunteers willing to help with the design, beta testing and development of communication materials. Our team of beta-testers went through the surveys, some taking different perspectives to try out all the question variants, and provided us with feedback and suggestions of modifications. Prior to our full launch this month in conjunction with Blood Cancer Awareness Month, we had over 60 patients who wanted to get a head start and had already populated the database with valuable responses.
Now for the most exciting news – since our full launch this month (an email was sent out the MPN Research Foundation mailing list on September 7th), we had already gained more than 100 new participants in the registry in the first 2 days! We will have to set a new goal for the year because we’ve already met our previous goal! The average age of participants, so far, is 60 years old with 57% female 41% male (2 percent declined to answer). We have 157 completed introductory surveys and 140 completed How do you feel today surveys. 234 health events were reported. Here is a breakdown of the patients who completed the first survey by diagnosis:
46% Essential Thrombocythemia
41% Polycythemia Vera
4% Myeloproliferative Disease (not specified)
We’ll be producing more statistics and reports from the system to share with the MPN patient community over time but we’re very excited at these early results! Stay tuned for more information on our website, in future newsletters and on social media about how the registry data is making a difference in research.
How can you help? If you haven’t already done so, please join us on myMPN (www.mympn.org) to make your data – your experience with an MPN- count! If you have already set up a profile, be sure to complete your surveys and keep coming back regularly to tell us How do you feel today? and report any recent health events. And please be sure to share with us any feedback you have and pass the word along to other patients you know who may not already be participating in myMPN!