Myelofibrosis FAQs
What are myeloproliferative neoplasms (MPNs)?
Myeloproliferative neoplasms (MPNs) are rare diseases of the bone marrow and blood that happen when something goes wrong in the blood cell production process and the body produces too many red blood cells, white blood cells, and platelets.
“Myelo” refers to the bone marrow, “proliferative” refers to the rapid growth of blood cells, and “neoplasm” refers to the abnormal and uncontrolled growth.
What is myelofibrosis?
Myelofibrosis is a type of myeloproliferative neoplasm (MPN). It’s a rare cancer that occurs in the bone marrow (myelo) and causes scarring (fibrosis). Myelofibrosis usually gets worse over time and affects the way your body makes healthy blood cells.
What is the cause of myelofibrosis?
With myelofibrosis, there are changes (mutations) in the DNA of bone marrow stem cells that cause your bone marrow (the spongy tissue inside your bones that makes blood cells) to make too many abnormal blood cells. As these abnormal cells build up, it causes inflammation that then leads to scar tissue. When scar tissue continues to form, it prevents the bone marrow from making enough healthy blood cells, resulting in lower red blood cell counts, platelet counts, and white blood cell counts.
What is primary myelofibrosis?
Primary myelofibrosis is myelofibrosis that occurs on its own without any previous blood disorder.
What is secondary myelofibrosis?
Secondary myelofibrosis is myelofibrosis that develops from another blood disorder.
Can myelofibrosis be cured?
Allogeneic stem cell transplantation (ASCT) is the only potential cure for myelofibrosis. However, ASCT is not always an available option for people based on age, disease course, or other health problems.
The goal of treatment for myelofibrosis is to help manage symptoms, improve quality of life, prevent complications, and control or slow down the disease.
What are some myelofibrosis symptoms?
As normal blood cell production decreases, you may experience the following signs and symptoms:
- Easy bruising or bleeding
- Pain or fullness below the ribs on the left side
- Tiredness, weakness, shortness of breath
- Itching
- Bone pain
- Increased risk of infection
These are not all of the possible myelofibrosis symptoms. It’s important to talk to your doctor about what you’re experiencing.
Why does myelofibrosis cause splenomegaly?
Because myelofibrosis causes a drop in blood cell counts, sometimes other parts of your body, like your spleen, try to help raise your counts by taking on the task of making them. As your spleen works harder to make more blood cells, it may start to grow in size. This is called splenomegaly. Symptoms of splenomegaly can include early feelings of fullness when eating, abdominal discomfort, and pain under the ribs on the left side.
What are signs that myelofibrosis is progressing?
Myelofibrosis is a progressive disease, which means it can get worse over time. If your blood cell counts are continuing to drop, if you’re noticing any new symptoms (like feeling more tired/weaker than usual or bruising easily/bleeding more), or if any of your current symptoms are getting worse, it could be a sign that your disease is progressing.
These are not all the signs that myelofibrosis is progressing. It’s important to talk to your doctor about what you’re experiencing.
What are cytopenias?
A cytopenia is a term that means low blood cell counts. Many people with myelofibrosis will have low blood cell counts, especially low red blood cell counts, which is called anemia, and low platelet counts, which is called thrombocytopenia.
What do I need to know about low blood cell counts?
Low red blood cell count (anemia): Because red blood cells carry oxygen throughout your body, anemia can cause symptoms like fatigue or tiredness, shortness of breath, and pale skin.
Low white blood cell count (leukopenia): White blood cells help fight infection, so when the body doesn’t make enough, it can weaken the immune system and increase the risk of getting sick.
Low platelet count (thrombocytopenia): With thrombocytopenia, blood isn’t able to clot like it should, which can cause bleeding or easy bruising.
Should I be keeping track of my symptoms?
Yes. Myelofibrosis is a progressive disease, which means it gets worse over time. Keeping track of your symptoms can help you spot important changes that might signal your myelofibrosis is progressing. It also helps you have more informed conversations with your doctor about your myelofibrosis.
Download our symptom tracker to help you record your symptoms and how often they happen.
What should I do if my symptoms are getting worse?
If you notice any changes in symptoms, even small ones, don’t wait until your next appointment; reach out and talk to your doctor.
What types of healthcare providers treat myelofibrosis?
- Oncologist (specializes in cancer)
- Hematologist (specializes in blood conditions)
- Hematologist-oncologist (specializes in blood cancer)
- MPN specialist (specializes in myeloproliferative neoplasms); myelofibrosis is a type of MPN
VONJO FAQS
What is VONJO?
VONJO is a prescription medicine used to treat adults with certain types of myelofibrosis who have a platelet count below 50,000 per microliter. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Who is VONJO for?
VONJO is for people who have started to see their symptoms change, which may be a sign that their myelofibrosis is progressing.
VONJO is used to treat adults with certain types of myelofibrosis who have a platelet count below 50,000 per microliter.
How does VONJO work?
In the body, there are many pathways that drive myelofibrosis. VONJO works on more than one of these pathways, including one that may help make new blood cells, one that may help reduce inflammation, and one that may help control iron levels.
How may VONJO help me?
When your growing spleen becomes harder to manage, VONJO may help shrink it. In a study, more people had their spleen get smaller with VONJO compared to people on best available therapy, including ruxolitinib, hydroxyurea, and monitoring without treatment.
What are the possible side effects of VONJO?
With VONJO, the most common side effects that occurred in more than 2 out of 10 people (20%) in a study were:
- Diarrhea
- Nausea
- Low platelet counts (thrombocytopenia) or low red blood cell counts (anemia)
- Swelling of ankles, legs, and feet
These are not all the side effects with VONJO. Please read the Important Safety Information below.
How do I take VONJO?
The recommended starting dose is two 100-mg capsules, which are taken twice each day—with or without food.
- VONJO capsules should be swallowed whole, not chewed, opened, or broken
- You should take VONJO at the same time every day
It’s important to take VONJO for as long as it’s prescribed to you. Do not change your dose or stop taking VONJO without talking with your doctor.
What should I do if I miss a dose of VONJO?
If you miss a dose of VONJO, skip the dose and just take your next dose at your regularly scheduled time. Do not take 2 doses at the same time to make up for the missed dose.
Are there any medicines I shouldn’t take while taking VONJO?
Do not use VONJO if you are taking other medications that are strong CYP3A4 inhibitors or inducers.
Be sure to talk to your doctor about all the medications you are taking.
What should I tell my doctor before taking VONJO?
Don’t forget to tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements and remedies. Taking VONJO with certain other medicines may affect the amount of VONJO or the other medicines in your blood and may increase your risk of side effects or affect how well VONJO works.
Especially tell your doctor if you take hormonal contraceptives (birth control). Hormonal birth control methods, except for intrauterine systems containing levonorgestrel, may not work during treatment with VONJO and for 30 days after your last dose. Talk to your healthcare provider about birth control methods that may be right for you during treatment with VONJO.
You should also tell your doctor about all your medical conditions, including if you:
- Have active bleeding, have had severe bleeding, or plan to have surgery or noninvasive procedures. You should stop taking VONJO 7 days before any planned surgery or invasive procedures
- Have diarrhea or commonly have loose stools
- Have had a blood clot, heart attack, other heart problems, or stroke
- Have a history of low blood levels of potassium. It is important that you get blood tests done during treatment with VONJO to monitor your body salts (electrolytes)
- Smoke or were a smoker in the past
- Have had any other cancers
- Have an infection
- Have nausea or vomiting
- Have liver or kidney problems
- Are pregnant or plan to become pregnant. It is not known if VONJO will harm your unborn baby
- Are breastfeeding or plan to breastfeed. It is not known if VONJO passes into your breast milk. You should not breastfeed during treatment and for 2 weeks after your last dose of VONJO. Talk to your healthcare provider about the best way to feed your baby during this time
- Plan to father a child—VONJO may affect fertility in males
Will my healthcare provider monitor me while I’m taking VONJO?
Your doctor will run some tests before and during treatment with VONJO:
- A complete blood count (CBC) helps track how you’re responding to treatment and if there are changes in your blood cell counts
- Coagulation testing will see how your blood is clotting and check your risk of bleeding before you start taking VONJO
- A baseline electrocardiogram (ECG) will check how your heart is working
Does VONJO have a patient support program?
VONJO Connect™ can provide information about healthcare coverage options and financial assistance that may be available to help you pay for VONJO.
