By Marios Georgiou
October 8, 2024
The voices of health care professionals are hugely beneficial to helping increase the public’s understanding of the benefits and applications of plasma-derived medicinal products (PDMPs). In this blog post, you’ll hear from a doctor and nurse who treat patients that rely on PDMPs.
Dr. Silvia Sánchez-Ramón and infusion nurse Laura Rohe, who herself lives with primary immunodeficiency, have devoted their careers to caring for patients. Despite living and working on different continents, their experiences in supporting the patient community is similar. They have both witnessed firsthand how plasma-derived medicines save and improve lives, and their clinical work offers valuable insights into the broader impact of these treatments.
The Role of PDMPs
Plasma-derived medicinal products (PDMPs) are essential in treating rare and chronic diseases, which are often challenging to diagnose, manage, and treat. Common plasma protein deficiencies include primary Immunodeficiency Disorders (PID), von Willebrand disease (VWD), Alpha-1 Antitrypsin Deficiency (AATD), Hereditary Angioedema (HAE), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), and other diseases associated with absence or malfunction of specific proteins. Additionally, many emergency health care providers also use PDMPs in everyday settings, such as surgery, burn care, and more.
Medical specialists play a crucial role in several aspects of patient care. Firstly, they are often the key to helping patients and families receive an accurate and timely diagnosis. Secondly, with their in-depth knowledge of rare plasma protein deficiencies, these specialists can recognize subtle symptoms that might be overlooked by general practitioners. Thirdly, they know which tests to order and how to interpret the results. However, only a relatively small number of specialists in each country have the advanced knowledge and experience to treat these rare diseases.
Following a patient’s diagnosis, specialists are essential in developing treatment plans that work for their patients. These medical experts know the intricacies of using PDMPs, including the correct dosage and frequency of treatment. They prescribe specific types of PDMPs that best suit the clinical needs of each patient. In addition, many specialists often contribute to research in the field, spurring a better understanding of plasma protein deficiencies.
It's important to note that PDMPs are single-source biological products, meaning there are no generic alternatives – and making it even more imperative that patients have reliable access and availability to their treatments.
Provider Perspectives on the Value of Plasma-derived Medicines
Dr. Silvia Sánchez-Ramón is the Head of the Immunology Department at Hospital Universitario Clínico San Carlos in Spain, and also serves as an Associate Professor of Immunology at the Complutense University of Madrid's School of Medicine. She is an expert in immunoglobulins, which are sourced from plasma to treat patients with primary and secondary immunodeficiency to supplement missing or malfunctioning antibodies and prevent occurrence of severe infections.
At the 2024 International Plasma Protein Congress (IPPC) in Athens she emphasized the importance of treatment with immunoglobulins (IG), stating: “For patients with primary immunodeficiencies and many secondary immunodeficiencies, such as leukaemia, lymphoma and melanoma, IG is a life-saving therapy that is crucial for their survival.”
Dr. Sánchez-Ramón also noted that the demand for these therapies is growing, explaining, “There is a significant need for plasma collection to support this expanding group of patients.” Additionally, she shared a study she conducted highlighting the economic impact of immunoglobulin replacement therapy in secondary immunodeficiency to hematological cancer.
“Immunoglobulins treatment was able to reduce the number of days patients were hospitalized for infections by half and eliminated the need for intensive care unit admissions. While immunoglobulin treatment may be considered expensive, the study shows that it is actually more cost-effective compared to the expenses incurred from infections and hospital stays.”
Overall, the findings suggest that not treating patients with plasma-derived medicines can lead to higher costs due to untreated conditions.
Across the Atlantic Ocean, Laura Rohe, RN, BSN, IgCN, is a U.S.-based infusion nurse working in allergy, asthma, and immunology for almost three decades.
In a recent conversation with PPTA President and CEO Anita Brikman, Laura shared her personal commitment to educating her patients about the vital role of the plasma-derived medications they receive. “Educating my patients on the type of medication they are receiving and where it comes from is very important,” she explained. She clarifies to her patients that their medication provides essential proteins from plasma that their bodies either cannot produce or have, but do not function properly.
“This replacement therapy is lifesaving, as they need these immunoglobulin antibodies to protect them from life-threatening infections.”
Laura encourages those considering plasma donation to go for it, highlighting its life-saving potential. “For primary immune deficiencies, immunoglobulin is the standard of care,” she says. “You can't make it in a laboratory — we rely on the thousands of donors who provide us with the circulating antibodies that keep us healthy.” Her patients often express gratitude for receiving treatment and having a diagnosis.
“Many of my patients feel thankful to be on therapy and to finally have a diagnosis, whether it is primary immune deficiency or hereditary angioedema.”
Plasma-derived medicines truly change lives by extending life expectancy, improving quality of life, and preventing serious complications. Their importance is recognized globally, with both the World Health Organization (WHO) and the European Medicines Agency (EMA) including them among the most critical medicines for patients. A sincere thank you to plasma donors for making these life-saving treatments possible.
If you are interested in learning more about donating plasma, visit plasmaweek.org. Or, if you are a health care provider who wants to share your story, click here.