Can Myeloproliferative Neoplasms Be Cured?

Graft-versus-host disease (GVHD). This is a complication that happens after allogeneic stem cell transplantation where the patient receives healthy stem cells from a donor. GVHD occurs when the donated stem cell rejects the body its being transplanted into because it sees it as 'foreign' and subsequently instigates an immune response that can differ in severity (mild, moderate, or severe).

Patients with graft-versus-host-disease may experience any of the following symptoms depending on the affected organs:

• Nausea
• Vomiting
• Abdominal cramps
• Skin rash
• Loss of appetite
• Diarrhea
• Skin burning
• Redness of the skin
• Jaundice (yellowing of the skin which means the liver is affected)
• Mouth dryness
• Mouth sores 
• Throat ulcerations
• Tooth decay
• Gum disease
• Itchy skin
• Loss of hair
• Loss of appetite
• Unexplained weight loss
• Shortness of breath
• Wheezing
• Fatigue
• Muscle weakness
• Stiffness in the joints

Nevertheless, GVHD symptoms can be controlled by prescribing immunosuppressive medications.

Reduced blood cell counts. The first step to bone marrow transplantation for myeloproliferative neoplasms patients is to undergo chemotherapy to eliminate all diseased existing blood cells to lead the way for newly transplanted stem cells to produce healthier blood cells. However, this process can take some time (a few weeks) which subjects patients to the following risks:

• Anemia reduced red blood cells count which leads to general tiredness, fatigue, and shortness of breath. A quick solution to this problem is for patients to take blood transfusions.
• Uncontrolled bleeding reduced platelet counts lead to a lack of blood clotting which in turn leads to excessive bleeding. The solution to this is for patients to take platelet transfusions.
• Infections reduced white blood cell count means that your immune system is unable to fend off itself against foreign threats like (bacteria, viruses, or fungi) which in turn leads to infections. If the patient is prescribed immunosuppressants as well, this carries a higher risk of infection. To protect yourself, you'll need to be in a germ-free environment whether you're in a hospital or at home.

Chemotherapy medication side effects. These can include short and long-term side effects, and they are:

• Nausea
• Loss of appetite
• Vomiting
• Hair loss
• Mouth ulcers
• Fatigue
• Skin rashes
• Infertility (only happens at high doses of chemotherapy)

Myeloproliferative Neoplasms Treatment Options

"Myeloproliferative Neoplasms (MPNs) is kind of the overarching title of these diseases and the therapy really depends on what the condition specifically is," Dr. Adrienne Philips, a Hematologist/Medical Oncologist, at Weill-Cornell Medicine, explained. Aside from bone marrow transplantation, various other treatment options help in managing the symptoms and controlling the disease. But before your doctor prescribes an MPN medicine for you, they will consider the following:

• Your symptoms
• Your age 
• Your overall health
• Results of your blood count tests 
• If you have any underlying medical conditions
• Any genetic mutations present in your cancer cells

Myeloproliferative neoplasms treatment options include the following:

• Watchful waiting this is usually prescribed when the patient doesn't exhibit any symptoms. So, they don't receive any treatment but are closely monitored for any changes.
• Surgery an operation like 'splenectomy: the removal of the spleen can be done to alleviate symptoms of MPN.
• Chemotherapy Chemical drugs that are given to stop the growth of abnormal MPN cells. However, they can also affect surrounding healthy cells.
• Radiation therapy Subjecting high-energy radiation onto MPN or the spleen in some cases.
• Immunotherapy Using certain medications to boost the body's ability to fight off MPN or to inhibit specific proteins (ex. Interferon alpha).
• Targeted therapy a type of medication that functions in inhibiting the growth of MPN cells. They can recognize specific features on these cells and stop their growth (they're more specific than chemotherapeutic drugs) (ex. JAK inhibitors).

Clinical Trials and Prospects for MPN

Clinical trials for MPN are a type of research study that's controlled by a team of healthcare professionals in order to improve the care of patients who suffer from myeloproliferative neoplasms by finding an MPN cure, or an MPN treatment with better efficacy and fewer side effects. 

The advantage of joining a clinical trial is that you get to gain access to new treatments that are not available which may serve in accomplishing the following:

• Improving your quality of life
• Increasing your survival
• Prolonging remission
• Finding a better medication

Nonetheless, you should also take into consideration that even though the tested drugs in any clinical trial go through extensive pre-clinical testing on lab animals, there's no guarantee of their effect on you. So, you should always consult your doctor about the risks and benefits before deciding to enroll in a clinical trial.

You can talk to your doctor to find out about the latest clinical trials for MPN treatment or use the SurvivorNet clinical trial search tool and find more information on what you're looking for.

Survivorship With Myeloproliferative Neoplasms (MPN)

Since living with myeloproliferative neoplasms can be rather challenging, here are some recommended steps to follow if you've been diagnosed with MPN, these include the following:

Seek support. This can be from a myeloproliferative neoplasms support group, a trusted institution like the MPN Research Foundation, or friends, family, and loved ones. This can be very empowering and encourage you to a better state of mental well-being.

Find specialized healthcare professionals. Since it's a rare disorder, you should find a specialist in this area. 

Become more educated about the disease. When you know everything there's to know about your disease, you can make more informed decisions about what to ask your doctor and weigh the risks and benefits of your treatment options, and more.

Financial stability. After you've received a diagnosis of myeloproliferative neoplasms (MPNs), you should research your financial options and keep them in order. Questions to ask yourself to prepare are:

• Do I have insurance?
• Will my insurance cover my treatment?
• How can I get help with the costs?

Get information about clinical trials. Learning about clinical trials is a great way to keep your options open. Whether or not this is suitable for you, is a conversation you should have with your doctor.

Patient stories. By reading other patient stories, you gain insights into the do's and don'ts and become knowledgeable about the journeys of patients that were before you.

Regular checkups. Maintaining regular checkups will allow your doctor to better monitor your condition and stay ahead of any new developments.

Mental health. It's important to note that your well-being isn't just physical, it's mental as well. Thus, maintaining good mental health and a positive attitude go a long way in your recovery. 

Dr. Ghaith Abu-Zeinah, an instructor in Medicine at Weill Cornell Medical College and an Assistant Attending Physician at the New York-Presbyterian Hospital, explains to SurvivorNet, "I noticed that when patients become active participants in their own treatment and in this community of MPS, I think it really helps, overcome some of the mental burdens that come with having these diseases".

Questions to Ask Your Doctor

• Am I eligible to receive bone marrow transplantation?
• What treatment option do you recommend for me?
• What are the risks and benefits associated with the recommended treatment option?
• Am I a good candidate to enroll in a myeloproliferative neoplasms clinical trial?
• What are the best practices I should follow besides taking my MPN treatment to improve my quality of life?

The Bottom Line

MPNs treatments are incurable and often complex which require a lot of time from the patient. However, with proper care, it’s possible to live a normal life after treatment and experience remission from myeloproliferative neoplasm.

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