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Navigating a rare blood disorder is not easy, but for those with cold agglutinin disease (CAD), there is hope to take back control. Hear about Duffy’s comeback from CAD. The information conveyed in this article is not medical advice.
Throughout her entire life, Duffy has remained very active. Whether it was participating in half marathons, volunteering at her local hospital, or baking for her grandchildren, Duffy was always on the move. One day in November 2017, she began walking up a flight of stairs and started to feel breathless – this is when she knew there was something seriously wrong.
Charting Unknown Territory
Based on her blood tests, her physician thought she may have lymphoma or liver cancer and immediately ordered a battery of tests to try and diagnose the problem. Duffy was concerned and afraid. “I thought doctors had all the answers, so it frightened me that we were in the unknown,” she said.
While waiting for blood test results and hopefully answers, Duffy and her family went on vacation to Utah, where the altitude was 11,000 feet above sea level. To her surprise, she was so cold, and couldn’t breathe and was gasping for air, which was terrifying to her.
Understanding Cold Agglutinin Disease (CAD)
Shortly after that trip, she was diagnosed with cold agglutinin disease (CAD), a rare autoimmune disease in which the body attacks and destroys its own red blood cells (the destruction of red blood cells is called hemolysis).1,2 Initially, Duffy’s reaction to her diagnosis was one of deep fear. At the time there was little information about this condition available online. She quickly learned that in addition to fatigue and irregular heartbeat, CAD can also cause other symptoms including weakness, shortness of breath, light-headedness, chest pain, and a bluish color or discomfort in the hands and feet.3,4
Given that CAD is a rare disorder,1 sometimes hematologists and other physicians may not be aware of this disease and how to treat it. After finding a hematologist who specialized in treating people with CAD, Duffy learned about ENJAYMO® (sutimlimab-jome), a breakthrough treatment that has been proven to decrease the need for red blood cell transfusion due to hemolysis in adults with CAD.5
The doctor told Duffy that the most common side effects of ENJAYMO are respiratory tract infection, viral infection, diarrhea, indigestion, cough, joint pain, joint inflammation (arthritis), and swelling of the lower legs, ankles, and feet.5 The doctor also explained that she would need at least two hours of monitoring after her first infusion and then at least one hour for subsequent treatments, and that vaccinations against infections caused by certain types of bacteria may be needed before beginning treatment.5
At first, Duffy was hesitant to begin the treatment, but after discussing options with her doctor and family members, she realized that for her, the benefits outweighed the cost by trying ENJAYMO. It seemed to her like a viable option that showed real promise. ENJAYMO works by targeting C1s, a component of the first protein in the classical complement pathway, where Duffy's immune system was going haywire from this disease, and it has shown improvement in hemoglobin levels in clinical trials for some people living with CAD.5 Duffy and her doctor filled out an enrollment form to be connected to ENJAYMO Patient Solutions, to access education and support for patients. Her case manager helped her navigate the necessary insurance approvals so she could start taking ENJAYMO.
ENJAYMO’s Role in a CAD Comeback
The CARDINAL clinical study evaluated the safety and efficacy of ENJAYMO in 24 patients with CAD with a history of transfusion during the 6 months prior to starting the study. 54% of the patients taking ENJAYMO (13/24) met all three of the following criteria: increases in levels of hemoglobin (the protein in red blood cells that carries oxygen) from the start of the study (an increase of ≥ 2 g/dL or hemoglobin ≥ 12 g/dL at treatment assessment, from a baseline hemoglobin level of 8.6g/dL), did not receive transfusions from Week 5 until the end of the 6-month study (Average transfusions at baseline: 2 within the last 6 months; 2 within the last 12 months), and had no additional treatment for CAD beyond what was permitted in the study from Week 5 until the end of the study. Of the 54% of patients who met all three criteria, looking at each criteria separately, 63% of patients (15/24) had increases in hemoglobin levels from the start of the study (an increase of ≥ 2 g/dL or hemoglobin ≥ 12 g/dL at treatment assessment), 71% (17/24) did not receive transfusions from Week 5 until the end of the 6-month study, and 92% (22/24) had no additional treatment for CAD beyond what was permitted in the study. Patient experiences on ENJAYMO may vary so please talk to your doctor when making treatment decisions. To learn more about ENJAYMO and clinical trial data results, please visit ENJAYMO.com.
Charting Duffy’s CAD Comeback
Duffy considers the day she made the decision to begin ENJAYMO as one of great celebration and the beginning of her comeback from CAD. She started receiving ENJAYMO infusions at her doctor’s office, and now Duffy is able to receive infusions at home from a visiting nurse to make her treatment more convenient.
With ongoing ENJAYMO treatment every two weeks, she is now able to continue being active with her hemolysis better managed.5 Since starting ENJAYMO, Duffy’s hemoglobin numbers increased within a few weeks,5 which has positively impacted her anemia. She can focus more on her family, volunteering, and hobbies she had previously paused due to her diagnosis. She is now an advocate for the CAD patient community and encourages others to be their own advocates when it comes to managing CAD.
ENJAYMO is a prescription medicine used to decrease the need for red blood cell transfusion due to 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.
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 a part of 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 meningitides, 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
- rapid heartbeat
- 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 return of your symptoms after you stop ENJAYMO. Stopping ENJAYMO may cause the breakdown of your red blood cells due to CAD to 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:
- respiratory tract infection
- viral infection
- joint pain
- joint inflammation (arthritis)
- swelling of the lower legs, ankles, and feet
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.
- Collie J C. Cold agglutinin disease. Practice Essentials, Pathophysiology, Etiology. Medscape. February 7, 2022. Accessed September 20, 2022. https://emedicine.medscape.com/article/135327-overview
- Mullins M, Jiang X, Bylsma LC, et al. Cold agglutinin disease burden: a longitudinal analysis of anemia, medications, transfusions, and health care utilization. Blood Adv. 2017;1(13):839-848. Published 2017 May 19. doi:10.1182/bloodadvances.2017004390
- Badireddy M, Baradhi KM. Chronic Anemia. StatPearls. StatPearls Publishing; 2022.
- Das S, Maiti A. Acrocyanosis: an overview. Indian J Dermatol. 2013;58(6):417-420. doi:10.4103/0019-5154.119946
- Enjamyo (sutimlimab-jome) [package insert]. Sanofi; 2022.
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