Patient Registry Provides Unique Insight Into Myeloproliferative Neoplasms

To date, much of the existing information about myeloproliferative neoplasms (MPNs) has come from clinical trials. Even a simple internet search for more about this rare group of blood cancers and how they impact patients turns up little information.

The MPN Research Foundation (MPNRF)’s myMPN Patient Registry aims to bridge that gap with data from patients themselves — and a new analysis of the registry, presented at the American Association for Cancer Research (AACR)’s annual meeting in Atlanta, has led to some exciting new discoveries, said lead author Robyn M. Scherber, M.D., M.P.H.

In an interview with CURE, Scherber, assistant professor of medicine in the Department of Hematology and Oncology at UT Health San Antonio MD Anderson Cancer Center, and co-author Michelle Woehrle, the executive director of the MPN Research Foundation, explained what this new analysis has taught researchers about this family of diseases that affect the bone marrow and lead to an overproduction of white blood cells, red blood cells and platelets.

Of the 744 patients that have participated in the registry since its inception in September 2017, 62% were female and the mean age was 61 years old. Patients were diagnosed with essential thrombocythemia (38%), polycythemia vera (36%), myelofibrosis (23%) or an alternative MPN diagnosis (3%).

Since the study began a year ago, 2,100 disease-related events were reported to the registry, including 825 blood draws, 144 bone marrow biopsies and 77 transfusions. Ten patients reported that their disease has transformed from one type into another. To provide data on their symptoms, 400 patients completed 675 symptom assessments — many patients completing two or more.

This data is valuable to researchers for many reasons, said Scherber. “We have been able to see changes in symptom burden over time as well as with various therapies. We have also been able to track many disease-related events that will allow us to understand some of the risk factors and outcomes in patients with these events,” she added.

The analysis also uncovered some findings that Scherber and her team hope to present on at a future conference. “We have recently discovered an exciting association between symptom burden and disease driver mutations by evaluating this registry data,” she said. “We have also noted that symptoms vary highly by therapy, which we are investigating further.”

Scherber noted that there are limitations to registries like this one, most notably the fact that patients are submitting information through the computer and are all most likely more educated and have the financial means to have computer access. Additionally, patient-reported data may not be as accurate as found in medical records. “However,” Scherber noted, “so far the data from the registry very closely aligns with previously published data and appears to be very accurate.”

Woehrle added that the MPNRF hopes to eventually put a system in place that would help with accuracy. “It is our goal to validate the patient reported data with patient-supplied medical records. We are working towards perfecting the system, even as we are collecting useful data now.”

With this data analysis ongoing, the future goals of the registry include exploring the variables related to disease progression and transformation and to develop a compendium medical record and specimen registry.

“The MPNRF would like to eventually notate who in the registry has a sample stored in a lab already, or possibly help them bank blood somewhere in order to help understand who in the registry progresses and who does not,” explained Scherber. “By looking at their blood or other samples that have been collected, we may be able to better identify who progresses with the disease and specifically develop interventions for them. Ultimately, this may mean longer, healthier, and more productive lives for patients.”

Ideally, said Woehrle, the information collected by the registry will play a role in patient care going forward. “We are eager to ensure that the patient perspective is included with all decisions being made around patient care,” she said. “The FDA has been increasingly open to considering this kind of information and we wanted to ensure that those living with MPN aren’t at the back of the queue when it comes to collecting and sharing information that they provide about their lives.”

Scherber agreed, adding that “it is our goal that the registry will ultimately lead to better outcomes for MPN patients, and potentially help us solve major disease issues, including what we can do to help eliminate disease progression and improve the quality of life for MPN patients.”