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If you’re living with polycythemia vera, myelofibrosis, or essential thrombocythemia, you may have never heard of your condition before being diagnosed—which makes sense. Collectively known as myeloproliferative neoplasms, or MPNs, these rare blood cancers are, well, very rare. About 20,000 Americans are diagnosed with an MPN each year. (For some context, in 2024, an estimated 313,510 people in the US will learn that they have breast cancer.)
No matter how many people share your diagnosis, receiving any type of serious health news can set off an emotional earthquake that leaves you to sort through complicated feelings. But finding out that you have a rare disease may be even scarier because you likely don’t have a blueprint of what having it means for your future. It can also be incredibly isolating if you don’t know anyone going through the same thing, Raajit K. Rampal, MD, PhD, the director of Memorial Sloan Kettering’s Myeloproliferative Neoplasms Program, tells SELF.
Thankfully, you don’t have to face your diagnosis alone. Expert care teams and advocacy groups can help provide treatment and support. And learning more about rare blood cancers can help you better understand what you may face so you can mentally prepare. Here are some helpful things to remember as you start your research and talk to doctors about your options.
1. You may not experience any symptoms.
MPNs are caused by mutations in the stem cells found in your marrow, a.k.a. the soft, spongy tissue in the center of each bone. If you recall from science class, stem cells hang out there and develop into blood cells and platelets. If mutations (a change in a cell's genetic makeup) make this process go awry, it can cause issues that ultimately lead to blood cancers or other disorders. For example, polycythemia vera occurs when there are too many red blood cells, while essential thrombocythemia can crop up when the body produces too many platelets, which normally help with clotting.
Often, people have no idea their body isn’t doing what it should be because they don’t have any symptoms. They only receive a diagnosis when they have routine lab work done and the complete blood count—a measure of red blood cells, white blood cells and platelets—comes back abnormal, Dr. Rampal says. (This is one of the many reasons you should always get an annual physical exam.) If someone with a rare blood cancer does have symptoms, they’re often random and nonspecific. Depending on the exact disease, you may experience:
- Fatigue
- Intense itching
- Unexpected, rapid weight loss
- Drenching night sweats
- Bone or muscle pain
- Abdominal discomfort and abnormal feelings of fullness
- Bruising
- Recurring infections
Definitely bring up anything concerning or uncomfortable you’re experiencing with your doctor so they can help you manage the symptoms.
2. Your cancer may not be treated immediately.
Most of the time, if you are diagnosed with cancer, doctors begin treatment right away. But that’s not usually the case with MPNs. “When someone is diagnosed, sometimes all we do is say, ‘We’re going to watch your blood counts,’” Dr. Rampal says. Your doctor may tell you to take a daily aspirin and make lifestyle changes, like doing regular physical activity and quitting smoking, to reduce the risk of blood clots, which are a common complication of these cancers.
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Not needing to undergo treatment may seem shocking since this is cancer, after all. But rare blood cancers occur on a spectrum. In some cases, the disease may not progress for years, so doctors may simply suggest regular monitoring, Nicole Lamanna, MD, a Columbia University professor and doctor specializing in blood cancers, tells SELF. If tests eventually show progression, you start experiencing symptoms, or your organs are affected, your healthcare provider may recommend starting treatment.
There are a few different options out there, and which one is right for you really depends on what’s going on with your health. For example, Dr. Rampal explains that if you’re making too many of a particular type of blood cell, your doctor may suggest a medication that shrinks the cancer and helps bring your numbers down. If the medication works, many people can eventually return to being monitored without treatment, Dr. Lamanna says. In some cases, though, a person may stay on one treatment until it stops working and then try something new.
On the other hand, if your organs are inflamed (with myelofibrosis, the spleen can balloon as it tries to filter the abnormal blood cells), your doctor may suggest treating you with a Janus kinase (JAK) inhibitor. Some people with more advanced myelofibrosis may need a bone marrow transplant to prevent their disease from progressing to leukemia, Dr. Rampal says. Despite the name, a transplant doesn’t involve going under the knife. The process begins with chemotherapy to wipe out the ‘sick’ bone marrow, and then it is replaced with an infusion of stem cells, most often from a donor. “These cells set up shop in the bone marrow, repopulate it with healthy cells, and make healthy blood cell counts,” Dr. Lamanna explains. In a few months, the person has a “completely new blood system,” she adds.
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3. Clinical trials may be an option.
Thanks to ongoing research and FDA approvals for new, targeted drugs, Dr. Lamanna says the future is very hopeful for blood cancer patients. But there is still work to be done. “We don’t have great answers for all these diseases,” Dr. Rampal says. “The only way we make progress is by doing clinical trials.”
If you are looking for a new treatment option, it can be helpful to look into trials online and ask your doctor what types you should consider. For example, Phase I trials look at a brand new drug that has not yet been FDA-approved. “These are usually done in patients who’ve gotten every standard option of therapy, and their options are running out,” Dr. Lamanna says. However, with Phase IV, researchers have an FDA-approved drug that’s already on the market and are seeking additional information about it, including things like risks, benefits, and best use, she explains. “These trials are great for somebody less heavily treated or perhaps relapsed after one treatment.”
Dr. Rampal adds that navigating all of this takes courage, commitment, and the right mindset, noting that it’s important for your doctor to explain all the potential benefits and risks. “The new drug may not work or have some side effects. The decision [to join a clinical trial] needs to be made very carefully,” Dr. Rampal says.
4. Advocacy groups can be super helpful.
Although these diseases are rare, you’re not alone. Ask your doctor or look online for advocacy groups, patient support groups, and research societies focused on rare blood diseases or your specific cancer.
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“There’s a community of people who can be a resource, sharing experiences, knowledge, and wisdom,” Dr. Rampal says. Plus, they’ll understand exactly what you’re going through, which can be emotionally beneficial. Dr. Lamanna says advocacy groups can also help you find a specialist or get a free consultation. Even if the nearest specialist is hours away, it may be worth getting their opinion, whether in person or through telemedicine. A doctor specializing in a particular type of rare blood cancer may be able to confirm your diagnosis and explain the stage of your cancer more accurately and will know about all the best treatments available, Dr. Lamanna adds.
Rampal agrees. “Even if you have a good doctor, it’s a good idea to get an opinion from an expert in the field to be sure you’re on the right track,” he says. “Experts tend to be aware of the most updated data and clinically relevant novel developments” and, in turn, can inform you of the most current information to get the best outcome.
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