By Jennifer Fulginiti
October 7, 2024
International Plasma Awareness Week is finally here for 2024! PPTA is excited to shine a light on patients around the globe who rely on plasma-derived medicines to live healthy lives. On the first day of #IPAW2024, we are taking a deeper look into these patients' conditions and the importance of continuous availability and access to the medications that work best for them.
Plasma is the single largest component of human blood. It contains water, salts, enzymes, antibodies, and other proteins. Some people are born with conditions that affect the quality or quantity of proteins in their plasma, which can lead to the diagnosis of chronic and rare diseases. Many times, the only treatment for these conditions is medicines derived from donated plasma. Plasma-derived medicines are also lifesaving in many everyday conditions, such as pregnancies with incompatibilities between the mother and child, treating rabies and tetanus, and even major surgeries and transplants.
Treatment for many rare diseases is not one-size-fits-all, and patients often rely on specific medications to improve their quality of life. These medicines—especially plasma-derived ones—are often not interchangeable. That is why it is important to advocate for patients to lawmakers to ensure every patient can receive the medication that works best for them.
Below are stories from patients whose lives have been bettered thanks to the generosity of plasma donors. Read them to learn more about their conditions and how plasma-derived medicines help them live healthy and fulfilling lives.
“IVIG saved my quality of life; it has given me a reason to keep trying.” — Dave, living with Chronic Inflammatory Demyelinating Polyneuropathy
In 2011, Dave’s legs went out from under him in a parking lot. He laid there in shock, eventually crawling to his car in embarrassment. He feared what was happening to him and what his future would be.
A specialist diagnosed Dave with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), an autoimmune disease that robs him of strength in his arms and legs. When the doctor told him that there was a treatment, Dave felt like he could breathe again. He began receiving intravenous immunoglobulin (IVIG) every three weeks, and slowly began to improve.
Today, Dave still has some rough days. But a curb or a step or holding his arm up to brush his teeth are no longer obstacles. He leads a wonderful, active life with a beautiful wife and three amazing daughters. He has work that keeps him challenged, and even goes to the gym. These may not seem like heroic accomplishments, but for Dave, they are everything. From crawling through a parking lot to walking on a treadmill, IVIG not only saved Dave’s quality of life, but it also gave him a reason to keep trying.
"Today, I got to send my kids to school with a lot less worry about germs.” — Erica, mother of two children living with X-linked agammaglobulinemia
Erica is a single mother of two boys, both of whom live with X-linked agammaglobulinemia (XLA), a genetic disorder that makes it hard to fight infections. Her oldest son was diagnosed at 2 ½ years old, and the first two years were rough. He was constantly sick and had ear infections back-to-back, with three surgeries to relieve some of his ear problems. He had staph infections, with a couple of surgeries to relieve the pain that they caused. There were many hospital stays.
As a parent, sending your kids to school is already stressful, but imagine sending a kid without a working immune system. Both of Erica’s kids rely on gammaglobulin, or antibody replacement, every three weeks, and will continue to do so for the rest of their lives. Erica says that thanks to plasma protein therapies, it’s possible for them to go to school, on vacation, play at the park, go to public swimming pools, enjoy birthday parties with friends, go out to eat, and everything else another mother wouldn't have to think twice about doing.
“Today, I can get outside and be active. My dreams are limitless. I don’t have to count myself out.” — Christa, living with Chronic Inflammatory Demyelinating Polyneuropathy
Christa lives with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). With this disease, your body destroys the protective layer around your nerves, disrupting the neuron communication with your brain, making it hard to pick up a pencil or even walk a short distance. Since 2013, Christa has used intravenous immunoglobulin (IVIG), a plasma protein therapy. Before her diagnosis, she had no idea what this disease was or that this form of treatment existed.
Without plasma-derived medicine, Christa said she wouldn’t be able to dress or bathe, cook for herself, work, or go to school for her public health degree, because she wouldn’t be able to type or have the energy to make it through the day. Being diagnosed with CIDP at a young age with a disease that she will have her entire life, plasma medicines are going to allow her to have a family and future. When Christa thinks about what she wants to do, she no longer counts herself out and plans to live her life to the fullest.
“I have learned to take nothing for granted, live life to the fullest and enjoy making memories with my family and friends.” — Michelle, living with Hypogammaglobulinemia & Idiopathic Thrombocytopenic Purpura
Michelle was diagnosed with Idiopathic Thrombocytopenic Purpura (ITP), a disorder that causes low platelets and bleeding problems. When she received life-saving intravenous immunoglobulin (IVIG), she had six platelets left, was bleeding from the inside, and covered in countless scary bruises.
Michelle also discovered that she has hypogammaglobulinemia, a primary immunodeficiency that was present prior to her ITP diagnosis, and the cause of her ITP. She had a series of severe sinus infections that antibiotics did not help. The illness attacked her immune system so severely that it caused ITP.
Michelle receives intravenous immunoglobulin every four weeks, in six-hour infusions, to help her to fight for every day.
“Today, I marched in the school band at a football game.” — Sam, living with Chronic Inflammatory Demyelinating Polyneuropathy
At 10 years old, Sam was diagnosed with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), a rare disorder of the peripheral nerves characterized by gradually increasing sensory loss and weakness. He has had periods of difficulties and needs regular infusions of intravenous immunoglobulin (IVIG).
CIDP gradually became business as usual for him and his family. He managed to keep high grades in school and keep up with a good social life, since IVIG makes his strength and ability to do activities much easier. He is free to go to school, play in the school's band, and do plenty of moderate physical activities, which keep him engaged and active.
"Thanks to plasma protein therapies, I am living with this condition rather than just suffering with it." — John, living with Hereditary Angioedema
John is in his 30s and living with Hereditary Angioedema (HAE), a rare genetic disorder characterized by recurrent episodes of severe swelling. He is happy to say that he lives with this condition rather than just suffering from it thanks to C1-esterase inhibitor plasma therapy.
Before having access to the therapy, John describes his life as dominated by emergency room visits and nights spent on the bathroom floor. HAE patients carry a 30 percent mortality rate when untreated. For John, this meant every night when he went to bed, there was a chance that he could suffer laryngeal swelling in his sleep, and not wake up.
Thanks to C1-esterase inhibitor plasma therapy, he doesn’t have to live with that fear any longer. John can work, travel with my family, and enjoy many aspects of life that he once thought to be out of reach as a chronically ill patient. Watching the tremendous improvement from treatment in HAE-affected family members has given John hope for the future — for himself, and his whole family.
If you enjoyed reading these stories, consider sharing your own here.
There’s no better time than IPAW to get involved! You can help in many ways, including donating plasma, getting the word out about the uniqueness of plasma-derived medicines, and encouraging local lawmakers to take legislative actions to benefit patients. Visit www.plasmaweek.org to get started.
PPTA is proud to work in partnership with patient organizations. If you or someone you know is looking for patient resources, visit the below websites:
Europe
- European Hemophilia Consortium
- European Patient Organisation for Dysimmune and Inflammatory Neuropathies (EPODIN)
- Immunodeficiency UK
- ITP Support Association
- Orphanet
International
- GBS/CIDP Foundation International
- Guillain-Barré Syndrome Support Group
- HAEI - International Patient Organization for C1 Inhibitor Deficiencies
- International Patient Organization for Primary Immunodeficiencies (IPOPI)
- Jeffrey Modell Foundation (JMF)
- World Federation of Hemophilia (WFH)
North America
- Alpha-1 Canada
- Alpha-1 Foundation
- Committee of Ten Thousand
- Hemophilia Federation of America (HFA)
- U.S. Hereditary Angioedema Association
- Immune Deficiency Foundation (IDF)
- National Bleeding Disorders Foundation
- Platelet Disorder Support Association (PDSA)
- National Organization for Rare Disorders (NORD)