Myelofibrosis is a type of bone marrow cancer that leads to the formation of fibrous tissue in the bone marrow and results in scarring, which impairs the normal production of blood cells. The exact cause of myelofibrosis is unknown, but it is believed to be caused by genetic mutations in the blood cells.
Jakafi (ruxolitinib) is a type of Janus kinases (JAKs) inhibitor that is used to treat myelofibrosis. Jakafi works by blocking the activity of JAKs proteins, which regulate the action of the immune system and the production of blood cells. By blocking JAKs, Jakafi can help to relieve symptoms, and it can also help to slow down the progression of myelofibrosis.
It’s essential to realize that Jakafi is not a cure for myelofibrosis and does not work equally for everyone.
More information about myelofibrosis
Symptoms of myelofibrosis can include:
- Anemia (a low level of red blood cells)
- Easy bleeding or bruising
- Night sweats
- Bone pains
- Weight loss
Treatment options for myelofibrosis depend on the severity of the symptoms and the patient’s overall health. They include:
- Medications such as Jakafi to relieve symptoms
- Transfusions to treat anemia
- Radiation therapy
- Stem cell transplantation
- Bone marrow transplantation
There are also several clinical trials of new treatments currently being conducted.
What are the available medications?
There is currently no cure for myelofibrosis. Medications used to treat myelofibrosis aim to both relieve the symptoms and slow down the progression of the disease. The primary medications that may be prescribed are JAK or tyrosine kinase inhibitors: These drugs target specific proteins involved in developing myelofibrosis, and may help relieve symptoms.
Some other palliative treatments may be prescribed that are not specifically for myelofibrosis but which target one specific symptom. They can reduce symptoms but produce fewer long-term benefits, including:
- Anagrelide: This drug can reduce the number of platelets in the blood, which can help to prevent or treat blood clots.
- Corticosteroids: These drugs can help reduce inflammation and relieve symptoms such as fatigue, night sweats, and pain.
- Androgens: These drugs can stimulate the production of red blood cells and may help relieve symptoms of anemia.
What is the best treatment for myelofibrosis?
For patients who cannot undergo stem cell transplantation or for whom bone marrow transplantation is not recommended due to age or health status, Jakafi is currently the most favored option of all JAK inhibitors.
Another alternative is tyrosine kinase inhibitors such as Gleevec (imatinib) and Sprycel (dasatinib), which block the activity of specific proteins called tyrosine kinases. These play a role in regulating cell growth and division.
What’s ahead for the treatment of myelofibrosis?
Several new treatments for myelofibrosis are being researched and developed. Some of the most promising new therapies include stem cell transplantation, when a patient’s damaged bone marrow cells are replaced with healthy stem cells, which can help to restore normal blood cell production. Stem cell transplantation has the potential to cure myelofibrosis in some patients, particularly if the transplant is successful and the patient does not experience any complications. However, stem cell transplantation is a complex and aggressive treatment that carries a risk of serious side effects, so it is not suitable for all patients with myelofibrosis. Usually, it is only used for younger patients with advanced disease who are otherwise healthy. Stem cell transplantation requires a period of intensive monitoring and care.
Jakafi, on the other hand, does not cure myelofibrosis, but can help control the disease’s symptoms and slow down its progression. It works by targeting specific proteins involved in the development of myelofibrosis, and can help to reduce the size of the spleen and improve symptoms such as fatigue, night sweats, and bone pain.
Whether a patient is offered stem cell transplantation, JAK inhibitors, or tyrosine kinase inhibitors will depend on a variety of factors, including the patient’s age, overall health status, and the severity of their myelofibrosis.
Not all patients with myelofibrosis will be eligible for stem cell transplantation, and not all patients will benefit from JAK inhibitors or tyrosine kinase inhibitors. The best course of treatment will depend on a number of factors and will be determined on a case-by-case basis.
In summary, stem cell transplantation has the potential to cure myelofibrosis, but it is a complex treatment option that is not suitable for all patients. JAK inhibitors, on the other hand, do not cure myelofibrosis, but they can help to control its symptoms and slow down its progression.
Why you should be sourcing Jakafi from IsraelPharm
JAK inhibitors sourced from US retail pharmacies are extremely expensive. If you are not getting any assistance to pay for this treatment, then IsraelPharm can provide you with substantial savings. The following table compares average retail prices of Jakafi from US outlets with our standard prices for 56 tablets:
|US retail (per 56 tablets)
|IsraelPharm (per 56 tablets)
How well does treating myelofibrosis with Jakafi work to relieve symptoms and extend a patient’s life span?
Myelofibrosis is a chronic and progressive blood cancer that can lead to severe complications if left untreated. The average life expectancy for someone with myelofibrosis without treatment can vary, depending on several factors, such as the stage of the disease, the severity of symptoms, and the presence of other medical conditions. In some cases, myelofibrosis can be fatal, especially if it progresses to a more advanced stage or if serious complications develop.
The treatments for myelofibrosis aim to relieve symptoms and slow down the progression of the disease. They can be effective in extending life expectancy for some patients. However, it’s important to keep in mind that the prognosis for myelofibrosis can vary widely from person to person, and some patients may have a more favorable prognosis than others.
In myelofibrosis, the dysregulation of JAK activity contributes to the development and progression of the disease, leading to the primary symptoms as well as an increased risk of serious complications, such as bleeding and infections. By blocking the activity of Janus kinases, JAKs inhibitors can help to restore normal regulation and relieve symptoms, as well as slow down the progression of the disease.
It’s important to note that the dysregulation of JAKs activity in myelofibrosis is a complex process that is not fully understood, and there is ongoing research aimed at gaining a deeper understanding of the underlying mechanisms and the role of JAKs in the development and progression of the disease. Nevertheless, Jakafi has been shown to be effective in relieving symptoms and slowing down the progression of myelofibrosis in some patients, making them an important treatment option for individuals with this disease.
What is the primary cause of myelofibrosis?
In a healthy person, the activity of Janus kinases is tightly regulated, ensuring that the immune system and blood cell production are functioning normally. However, in certain diseases, including myelofibrosis, the activity of JAKs can become dysregulated, leading to an altered regulation of the immune system and blood cell production, and contributing to the development and progression of the disease.