Myelofibrosis

Treatment Options

Treatment advances in the past decade have brought new hope to people who are diagnosed with myelofibrosis. The introduction of the first targeted therapy for myelofibrosis in 2011 was a significant step in treating it, and research is ongoing to find more therapies.

Myelofibrosis is treated like a chronic condition that will progress. With only one potentially curative therapy, the goals of treatment often focus on managing symptoms of the disease, improving blood counts, prolonging life and reducing the likelihood of transformation to acute myeloid leukemia.

Symptoms can vary widely among patients, so treatment is tailored to each person. A risk group is assigned at diagnosis. According to the Dynamic International Prognostic Scoring System Plus (DIPSS Plus), four risk groups, ranging from low to high, are considered. In general, patients in the low-risk group, who may or may not have symptoms, may be treated with watchful waiting, a clinical trial or targeted therapy. Patients in the high-risk group may receive a stem cell transplant, targeted therapy or a clinical trial.

Your doctor will take into account your risk group, symptoms, age and general health to create your unique treatment plan. Your desired quality of life is also important, so talk with your doctor about how the suggested therapies and potential side effects may best fit in with your expectations.

Watchful waiting may be a treatment approach that is considered if you have slow-growing disease, or if you have very mild or no symptoms. This option, typically for people who are in the lower risk groups, offers the possibility of avoiding the side effects of treatment as long as possible and, hopefully, without affecting the outcome. It is important to make and keep regular checkups to look for signs and symptoms because treatment should begin as soon as the disease progresses or symptoms appear.

Following are the most common treatment options for myelofibrosis. They may be used alone or in combination and may change as necessary.

Stem Cell Transplantation

An allogeneic stem cell transplant is the only treatment that offers a potential cure for myelofibrosis. You may qualify for a transplant depending on the severity of your disease, your age and your overall health. It may be considered risky for some older patients who have other health problems, so you are encouraged to find a doctor and a transplant center with extensive experience in stem cell transplantation. This may require traveling if you are not located near a transplant center. Some treatment centers offer assistance with travel and temporary lodging during treatment. Talk with your health care team at the transplant center and advocacy organizations to learn about the resources available to assist you.

An allogenic transplant uses stem cells donated by a family member or an unrelated donor identified through a national or international registry. To reduce the risk of Graft-versus-Host Disease (GvHD), a serious condition in which the transplanted T-cells attack the patient’s skin, liver and gastrointestinal tract, it is important that the patient’s and donor’s tissues match as closely as possible.

The potential for cure with allogeneic transplantation comes from donor stem cells working directly against the cancer through the graft-versus-tumor effect (also called graft-versus-leukemia or graft-versus-cancer-cell). For the donor stem cells to be effective, transplant recipients must first receive a conditioning treatment, consisting of chemotherapy and possibly radiation therapy, to weaken their own stem cells and allow the donor stem cells to replace them and start fighting the cancer.

A reduced-intensity conditioning treatment is often used for patients with myelofibrosis to spare the toxicity of very high doses of chemotherapy. This procedure uses milder doses of chemotherapy and radiation therapy for conditioning prior to the transplant. This approach depends entirely on the anti-cancer effect of the new immune system transplanted into the patient. High dose or myeloablative conditioning treatments are often reserved for transplant candidates in excellent health and with very active cancer that may be eliminated by the additional chemotherapy.

The allogeneic stem cell transplantation process has four distinct phases:

  1. Donor identification and evaluation, which includes tissue typing of related and unrelated donors, selecting the best match and requesting a thorough health evaluation of the donor as well as availability for the transplant time frame.
  2. Conditioning, which includes chemotherapy and radiation therapy.
  3. Stem cell transfusion, during which the harvested donor stem cells are infused into the recipient’s body intravenously (in the vein).
  4. Recovery and engraftment, in which healthy cells begin to grow. There are typically signs of this in the first 30 days, but enlarged spleen and scar tissue in the bone marrow, common in patients with myelofibrosis, can delay the engraftment process compared to transplant for other diseases. The patient will be at risk for infection while the weakened immune system recovers. The number of red cells, white cells and platelets will continue to be monitored until they are back to safe levels.

Innovations over the years in methods of stem cell collection, patient and donor selection, delivery of treatment and post-transplantation drug therapies have continued to improve the effectiveness and safety of stem cell transplantation. Before choosing to have an allogeneic stem cell transplant, talk with your doctor about the benefits and risks, potential short- and long-term side effects, late effects and the length of time you may need help from a caregiver.

Clinical Trials

These medical research studies may offer access to leading-edge therapies not yet FDA-approved. They may be your best first treatment option depending on your diagnosis. Progress continues to be made treating myelofibrosis. Treatments for mutations other than the JAK2 mutation in myelofibrosis are being researched. Ask your doctor if you should consider a trial as a first-line treatment or at any other time during treatment (see Clinical Trials).

Drug Therapy

Targeted therapy uses drugs or other substances to identify and attack specific mutations found within affected cancer cells. Unlike chemotherapy, which attacks healthy cells as well as cancer cells, targeted therapy is intended to affect primarily cancer cells with fewer off-target side effects.

Patients with certain gene mutations may qualify for targeted therapy (see Genetic Testing). For myelofibrosis, the current targeted therapies are given orally as a pill. Their use is typically restricted to patients with only mild anemia or thrombocytopenia because they can worsen already low blood counts. Both have been shown to decrease spleen size and improve symptoms in some patients with myelofibrosis.

Kinase inhibitors block a type of enzyme called a kinase. Certain kinases are more active in some types of cancer cells and blocking them may help keep the cancer cells from growing. The kinase inhibitors currently FDA-approved for myelofibrosis hinder the JAK1 and/or JAK2 enzymes involved with blood cell production.

Chemotherapy uses drugs to kill rapidly growing cells throughout the body. It may be given into a vein (intravenously or IV) or taken orally as a pill. It is often used in high doses to prepare for a stem cell transplant. In some cases, it is used to treat symptoms of anemia or to reduce high platelet and white blood cell counts.

Radiation Therapy

High-energy radiation to destroy cancer cells and shrink tumors may be given to prepare for an allogeneic stem cell transplant, reduce the size of the spleen, decrease bone pain or shrink tumors that have developed outside of the marrow.

Surgery

Although this treatment is not commonly used to treat myelofibrosis, it may be used to remove the spleen (splenectomy) if it becomes very large and is causing anemia or lowering platelet counts.

 
Common Drug Therapies As of 10/15/20
fedratinib (Inrebic)
ruxolitinib (Jakafi)

 

Donor Stem Cells Can Save Lives

At any given moment, thousands of people need lifesaving blood stem cell transplants but have no available donor. Organizations such as Be The Match (operated by the National Marrow Donor Program) have created registries of millions of potential donors. Minority donors are especially needed. Learn more at www.bethematch.org

Additional Resources

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