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.