Vienna, 14.09.2023. Anyone suffering from a rare disease has usually gone through a long process to get to their diagnosis. Once the diagnosis is established, additional obstacles often await: The medical expertise in the area of rare diseases is limited to very few people; there are not many people affected, making it difficult to exchange experiences; and information is hard to come by. This is contrasted by the patient's need to find out as much as possible about their disease, it's progression and potential treatments, since uncertainty is especially high after being diagnosed with a rare disease.
This is also the case for patients suffering from MPNs (myeloproliferative neoplasms): As a study by the Gallup institute on behalf of Austrian pharmaceutical company AOP Orphan Pharmaceuticals GmbH (AOP Health) shows that support in the form of information is of particular importance to those affected: 84 percent view clarification by physicians as important, while just over three quarters of those surveyed find self-help groups particularly important.
Peter Löffelhardt, president of the Global MPN Scientific Foundation and a sufferer of MPN himself: “When they receive a diagnosis, patients are anxious – I myself was too at first. They're not familiar with the disease, don't know what it means, are given conflicting information, and are sometimes referred from one place to the next. This leads to the desire to research for themselves, to find specialists, to seek interaction with other people who are also affected and to become as much of an expert as possible with regard to the disease. But with rare diseases especially, the question of how to access this information quickly comes up.”
Two thirds of patients do their own research
As the study by the Gallup institute shows that two thirds of patients suffering from MPN have already researched what treatment options are available even before choosing one for themselves. The internet was the first point of reference for all patients; in addition, they also turned to physicians (82 percent), lectures and events (53 percent), self-help groups (47 percent) and media and websites of pharmaceutical companies (41 percent). A little under one third (29 percent) had conversations with other affected patients.
Haifa Kathrin Al-Ali, internist at the University Hospital Halle (Saale) and director of the Krukenberg Cancer Center Halle: “Competent, informed patients are incredibly important. With rare diseases in particular, it's essential that patients become what you might call managers of their own disease. But this is no easy task, which is why assistance is required when it comes to where to find information, how to evaluate it, what to discuss with your own physician and where and how you can exchange experiences with other people affected by the disease. This is why I consider it extremely important to provide patients with support in this area.”
Checklists & Round Table
To mark the occasion of MPN Awareness Day, pharma company AOP Orphan Pharmaceuticals GmbH (AOP Health), in cooperation with physicians and psychologists, has put together practical tips for those affected along with a guide for talking to physicians. Furthermore, there is a Round Table taking place on 14 September 2023 all about how people affected can become managers of their own disease. “As specialists in the area of rare diseases, we maintain close contact with patient organizations and those who treat patients, who consistently report that patients especially appreciate practical tips. We always strive to meet these needs to the absolute best of our ability. The checklists and the event represent the initial steps, which we hope will meet a certain need”, explains Marion Rottenberg, International Patient Advocacy Lead at AOP Health.
What is MPN?
The term refers to a group of malignant diseases of the bone marrow in which too many red and white blood cells and/or platelets are formed. Myeloproliferative neoplasms are rare chronic diseases. Each year, around 6 new cases per 100,000 inhabitants are diagnosed. The majority of patients with this disease have a virtually normal life expectancy if the disease is managed properly and adequately. If left untreated, MPNs can lead to serious complications and can severely limit the quality of life of those affected.
Sources:
*Swerdlow SH et al. WHO classification of tumours of haematopoetic and lymphoid tissue. International Agency of Research on Cancer 2017; 4th revised edition.
About the study:
For the study, the Gallup institute interviewed 26 patients with polycythaemia vera using CAWI – Computer Assisted Web Interviews. The study was completed in April 2022.