Taking Back Control From CAD: How One Patient Came Back to Her Active Lifestyle After Years with a Rare Blood Disorder
(BPT) - Article is sponsored and developed by Sanofi. Nancy and Dr. Bai were compensated by Sanofi. This is not medical advice.
At 84 years old, Nancy has always been described as an active, high-energy person. With her husband, two sons, and two grandsons, her family often spends quality time hiking, fishing, and cheering on their favorite baseball team. But a few years ago, Nancy was diagnosed with a rare blood disorder that threatened the active lifestyle she cherished.
Living with Cold Agglutinin Disease (CAD)
After knee replacement surgery in 2016, Nancy’s bloodwork showed she lost an abnormally large volume of her body’s hemoglobin, a protein found in red blood cells.1 She was referred to a hematologist who diagnosed her with cold agglutinin disease (CAD), a rare autoimmune hemolytic anemia in which the body mistakenly attacks and destroys red blood cells, also known as hemolysis.2
According to her test results, Nancy’s body was destroying red blood cells faster than replacing them. Since red blood cells are vital for carrying oxygen to organs and tissues, she quickly developed symptoms.1
Nancy experienced sudden, overwhelming fatigue during simple daily activities like cooking, cleaning, and grocery shopping. “I found that I couldn't even climb stairs anymore,” she said, and she no longer had the energy to help care for her friends and family.
According to Dr. Wei Bai, board certified oncologist and hematologist, fatigue is one of the most common symptoms for patients with CAD, which can significantly impact their quality of life. Other symptoms may include weakness, shortness of breath, light-headedness, chest pain, and bluish color or discomfort in the hands and feet.3
A Treatment to Take Back Control
Nancy began working with Dr. Bai in 2021, before an approved treatment for CAD was available.4 They tried a variety of tactics like cold avoidance and related medications, but nothing successfully maintained her hemoglobin levels. Despite growing frustrated with fatigue that continued to interfere with her daily life, she never stopped fighting. “My life wasn’t fun anymore. I wanted to get it back,” she said.
New hope for Nancy came in 2022 with the U.S. Food and Drug Administration (FDA) approval of Enjaymo® (sutimlimab-jome), the first and only medication for adults with CAD.4,5
Understanding the Research Behind Enjaymo
Enjaymo is a prescription medicine used to treat hemolysis in adults living with CAD. Its FDA approval is supported by findings from two Phase 3 studies: a single-arm, open-label study in patients with CAD with a recent history of blood transfusion (within the past 6 months), and a double-blind, placebo-controlled clinical trial of adults with CAD without a recent history of blood transfusion (within the past 6 months).4,5 It is recommended that Enjaymo should not be taken by patients with known hypersensitivity to sutimlimab-jome or any of the inactive ingredients.
In one Phase 3 study, Enjaymo demonstrated fast and lasting improvement in hemoglobin levels. The trial met its composite primary endpoint – 73% of patients (16/22) showed an increase in hemoglobin of at least 1.5g/dL from baseline to Week 26 compared to 15% (3/20) on placebo, as well as remained transfusion-free from Week 5 through 26, and required no additional CAD treatment during the same time period. Additional data showed a mean increase from baseline hemoglobin levels. Further, average bilirubin levels from baseline to Week 26 returned to normal among 17 patients taking Enjaymo.4,5
Patients taking Enjaymo also reported a significant improvement in the symptoms and impact of fatigue as measured by The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) at 6 months. In the clinical trial, patients on Enjaymo had an average 11-point improvement from baseline (improving from an average baseline score of 31.67 points to an average score at treatment assessment of 42.5 points) vs a 2-point improvement in patients on placebo. Higher scores on the survey meant a person felt less fatigue. The overall safety results were consistent with the safety profile of Enjaymo from the pivotal study.4,5
Nancy’s Comeback from CAD with Enjaymo
Dr. Bai reviewed Nancy’s history to ensure she had or received the right vaccinations before starting Enjaymo. Together, they discussed the potential side effects of Enjaymo, including increase in blood pressure, urinary tract infection, respiratory tract infection, bacterial infection, swelling in lower legs or hands, joint pain, headache, nausea, runny nose, bluish color to the lips and skin, dizziness, feeling tired or weak, cough, changes in color or sensation in the fingers and toes (Raynaud's phenomenon).5
Dr. Bai also advised Nancy on the infusions for Enjaymo, which are weekly for the first two weeks, then biweekly thereafter.5 She looks forward to her infusions as a time to relax, read a book, and take a few moments for herself away from daily responsibilities.
Since starting Enjaymo, Nancy’s hemoglobin has risen significantly (almost 4 g/dL) to normal levels (11.5 to 16 g/dL for women1) and she hasn’t needed other treatments to manage her CAD. Today, Nancy reports significantly less fatigue than prior to starting Enjaymo. Once again, she has enough energy for grocery shopping, cooking, and caring for her loved ones. “I’m living the life I want with my husband, kids, grandkids, and friends,” she said, “I look forward to going to Alaska next year to fish for salmon and halibut with my family.”
Patient experiences on Enjaymo may vary so please talk to your doctor when making treatment decisions. To learn more, visit Enjaymo.com.
Important Safety Information and Indication
IMPORTANT SAFETY INFORMATION
Do not receive ENJAYMO if you are allergic to sutimlimab-jome or any of the ingredients in ENJAYMO.
ENJAYMO can cause serious side effects, including:
- Serious Infections: ENJAYMO is a prescription medicine that affects your immune system. ENJAYMO can lower the ability of your immune system to fight infections. People who take ENJAYMO may have an increased risk of getting infections caused by certain kinds of bacteria such as Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae. These infections may be serious or life-threatening. Some infections may quickly become life-threatening or cause death if not recognized and treated early.
- You need to receive vaccinations against infections caused by certain kinds of bacteria at least 2 weeks before your first dose of ENJAYMO. You may need to have additional vaccinations during treatment.
- If your healthcare provider decides that urgent treatment with ENJAYMO is needed, you should receive vaccinations as soon as possible.
- Vaccinations may reduce the risk of these infections, but do not prevent all infections. Call your healthcare provider or get medical help right away if you get any new signs and symptoms of an infection, including:
- severe headache with stiff neck or back
- pain during urination or urinating more often than usual
- cough or difficulty breathing
- flu-like symptoms
- pain, redness, or swelling of the skin
- Infusion-related reactions: Treatment with ENJAYMO may cause infusion-related reactions, including allergic reactions that may be serious or life-threatening. Your healthcare provider may slow down or stop your ENJAYMO infusion if you have an infusion-related reaction and will treat your symptoms if needed. Tell your healthcare provider right away if you develop symptoms during your ENJAYMO infusion that may mean you are having an infusion-related reaction, including:
- shortness of breath
- decrease in blood pressure
- chest discomfort
- rapid heartbeat
- injection site reaction
- itchy skin
- Risk of autoimmune disease: ENJAYMO may increase your risk for developing an autoimmune disease such as systemic lupus erythematosus (SLE). Tell your healthcare provider and get medical help if you develop any symptoms of SLE, including:
- joint pain or swelling
- rash on the cheeks and nose
- unexplained fever
- If you have CAD and you stop receiving ENJAYMO, your healthcare provider should monitor you closely for the return of your symptoms after you stop ENJAYMO. Stopping ENJAYMO may cause the breakdown of your red blood cells due to CAD return. Symptoms or problems that can happen due to red blood cell breakdown include:
- shortness of breath
- rapid heart rate
- blood in your urine or dark urine
The most common side effects of ENJAYMO include:
- increase in blood pressure
- urinary tract infection
- respiratory tract infection
- bacterial infection
- swelling in lower legs or hands
- joint pain
- runny nose
- bluish color to the lips and skin
- feeling tired or weak
- changes in color or sensation in the fingers and toes (Raynaud’s phenomenon)
These are not all the possible side effects of ENJAYMO. Call your doctor for medical advice about side effects.
Before receiving ENJAYMO, tell your healthcare provider about all of your medical conditions, including if you:
- have a fever or infection, including a history of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C.
- have an autoimmune disease such as systemic lupus erythematosus (SLE), also known as lupus.
- are pregnant or plan to become pregnant. It is not known if ENJAYMO will harm your unborn baby.
- are breastfeeding or plan to breastfeed. It is not known if ENJAYMO passes into your breast milk.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.
ENJAYMO® is a prescription medicine used to treat the breakdown of red blood cells (hemolysis) in adults with cold agglutinin disease (CAD).
It is not known if ENJAYMO is safe and effective in children.
- Farid Y, Bowman NS, Lecat P. Biochemistry, Hemoglobin Synthesis. StatPearls. 2022.
- Collie JC. Cold agglutinin disease. Practice Essentials, Pathophysiology, Etiology. Medscape. Updated February 7, 2022. https://emedicine.medscape.com/article/135327-overview.
- Joly F, Schmitt LA, Watson PAM, et al. JMIR Form Res. 2022;6(7):e34248.
- Röth A, Berentsen S, Barcellini W, et al. Blood. 2022;140(9):980-991.
- Enjamyo (sutimlimab-jome) [package insert]. Sanofi; 2023.
© 2023 Genzyme Corporation. All rights reserved.
ENJAYMO and Sanofi are registered trademarks of Sanofi or an affiliate.
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