February saw some amazing news for patients with cold agglutinin disease (CAD). The US Drug and Food Administration (FDA) approved sutimlimab (Enjaymo™) as the first drug treatment for patients with our disease. According to Sanofi, its new drug is administered to decrease the need for red blood cell transfusion due to hemolysis in adults with CAD.

I was elated last year when I learned that there was a drug being fast-tracked by the FDA for CAD. According to The New England Journal of Medicine, “cold agglutinin disease is a rare autoimmune hemolytic anemia characterized by hemolysis that is caused by activation of the classic complement pathway. This drug sutimlimab, a humanized monoclonal antibody, selectively targets the C1s protein, a C1 complex serine protease responsible for activating this pathway.” 

I have been lucky thus far. My current treatment has been nothing more than folate supplementation because my body for the most part has been compensating for the hemolysis. There are spontaneous exacerbations and I usually feel the results of it immediately. And because of this I then try to figure out the trigger and try avoiding it in the future. 

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It’s been a short year and a half since my diagnosis while there have been so many others that have had this disease for 20-plus years. As I look forward, I wonder what my future will hold. I find it helpful to learn from others with my disease. In speaking with some of my CAD counterparts in our Facebook support group, I learned that many have gone through years of treatments through trial and error.

Read about experimental treatments for CAD

Some have had their spleens removed but to no avail. The problem is as your liver and spleen work to filter out the remnants of the dead red blood cells, they become overburdened,  which can cause the spleen to enlarge resulting in pain. Sometimes the only way to eliminate the pain is to have a splenectomy. It seems to me that this is not a treatment but a way to alleviate some of the symptoms of this disease.

Another treatment that was often used first was corticosteroids, but research has shown that they did little to treat the hemolysis and are no longer recommended. Of course, there are transfusions, but these are used only when it is medically necessary for emergency situations. Transfusions, I learned, have a short life in the body of a patient with CAD, as we hemolyze our red blood cells and are destroyed so will the transfused ones. 

I am thankful that I have these other patients to go to for insight without which I would surely be using the internet for guidance, which I know is often a bad idea. There are so many treatments out there that are antiquated but still I search in hopes to find something. 

However, now with the news of a drug meant for CAD patients, I can relax because I have an option meant for me specifically. As with all medicines, they are not perfect and I may have side effects but still I am grateful that there is an option. That said, the cost is a concern. I found that before rebates at wholesale it would cost approximately $10,000 per treatment and treatment is every 2 weeks. This does cause concern but I am still thankful that it has been approved. 

There are so many rare diseases that need funding and research to get to the bottom of what triggers it and then to a cure. There is perhaps a light at the end of the tunnel, when you have investors, you have attention, when you have attention, you get action, and that action creates competition. Competition is what creates cures and manageable healthcare costs. As for now, we are still too far away from that for CAD, but I am happy that we are not forgotten and that there are people out there doing research and funding for people like me.