FAQs

This section answers questions commonly asked by newly diagnosed patients or their family members.

Who is the best doctor for my diagnosis?

To make sure you get the best care and treatment, it is important to find a doctor with expertise in treating your specific disease. Specialists in bone marrow failure diseases such as aplastic anemia, MDS, and PNH are hematologists (doctors specializing in blood diseases) and oncologists (doctors specializing in cancer).

To find an expert, you might want to call the hematology/oncology department at a teaching hospital affiliated with a local university. They can give you the name of a doctor with expertise in your disease. Doctors at teaching hospitals are most familiar with rare diseases. They work in a research and teaching setting and are up-to-date on both the standard therapies and new investigational treatments.

Though AA&MDSIF does not refer specialists, patients and families can contact the AA&MDSIF patient educator, at (800) 747-2820 or clark@aamds.org for more information on finding a doctor and getting second opinions.

When contacting a specialist, you might ask the following questions:

• Does the specialist accept your health insurance?
• Is the doctor an expert in treating your suspected or diagnosed disease?
• Is the physician's hospital or treatment center accredited and experienced in the treatment of bone marrow failure diseases?
• Is the physician able to discuss all standard treatment options for your disease, including clinical trial options?
• Are you comfortable with the style and level of communication with the physician and other members of the health care team?
• How often will you need to visit the physician, and is transportation and housing an issue?

What are the treatments for aplastic anemia?

The main goal of aplastic anemia treatment is to increase the number of healthy cells in your blood (blood count). When your blood counts go up you are less likely to need blood from a donor (transfusion), your quality of life becomes better and your symptoms are not as bad.

Your doctor will look at several issues to find the best treatment plan for you. These include how severe your symptoms are, your age, other conditions or diseases you have, and whether someone is willing and able to donate matching bone marrow to you (preferably a family member).

There are several treatments and treatment approaches your doctor may consider. These include: * Supportive therapies help manage the symptoms of your aplastic anemia.

Their goal is to increase blood counts and treat infections. Supportive therapy treatments typically include blood transfusions and antibiotics, and may include growth factors or iron chelation for some patients.

• Immunosuppressive therapy lowers your body's immune response. This therapy uses medicines to keep the immune system from attacking the bone marrow stem cells. ATG (antithymocyte globulin) and cyclosporine are the medicines typically used.
• Bone marrow transplantation replaces your unhealthy blood-forming stem cells with healthy ones from a donor.
• Clinical trials, also called research studies, may also be an option for patients who do not have success with the other treatment options.

What are the treatments for MDS?

The main goal of MDS treatment is to increase the number of healthy cells in your blood (blood count). When your blood counts go up you are less likely to need blood from a donor (transfusion), your quality of life becomes better and your symptoms are not as bad.

Your doctor will look at several issues to find the best treatment plan for you. These include your symptoms, your age, the subtype of MDS you have, your disease risk score, and other conditions or diseases you have. He may also look at whether someone is willing and able to donate matching bone marrow to you (preferably a family member).

There are a number of general approaches used in the treatment of MDS depending on the subtype of MDS you have and the severity of your case. These may be divided into the following:

• Wait and watch, also called “watchful waiting” is an approach your doctor might suggest if your blood counts aren't too low and you symptoms aren't too bad.
• Supportive therapies helps you manage the symptoms of your MDS. Their goal is to increase blood counts, treat infections, and treat iron overload. Supportive therapy treatments typically include blood transfusions and antibiotics, and may include growth factors or iron chelation for some patients.
• Immunosuppressive therapy can lower your body's immune response and is appropriate for patients with certain types of MDS. This therapy uses medicines to keep the immune system from attacking the bone marrow. These medicines include ATG (antithymocyte globulin) and cyclosporine.
• Drug therapies approved specifically to treat MDS stop abnormal cells from growing and stimulate the growth of healthy bone marrow cells. These therapies currently include lenalidomide (Revlimid), decitabine (Dacogen) and azacitidine (Vidaza).
• Chemotherapy treats MDS by using drugs that kill abnormal cells.
• Bone marrow/stem cell transplantation replaces your unhealthy blood-forming stem cells with healthy ones from a donor.

Clinical trials, also called research studies, may also be an option for patients who do not have success with the other treatment options.

What are the treatments for PNH?

PNH is considered chronic. That means it lasts for a long time. The only known cure is a bone marrow transplant. Other treatments are designed to ease symptoms and prevent problems.

There are several treatments and treatment approaches your doctor may consider. These include the following:

• Wait and watch, also called “watchful waiting,” is an approach your doctor might suggest if your blood counts aren't too low and you symptoms aren't too bad.
• Supportive therapies help you manage the symptoms of your PNH. Their primary goal in PNH patients is to increase blood counts. Supportive therapy treatments typically include blood transfusions, and may include growth factors. For PNH patients this might also include taking extra iron (iron therapy).
• Blood thinners (anticoagulants) may be used on some patients to help reduce the chance of getting blood clots.
• Immunosuppressive therapy can lower your body's immune response and is appropriate for PNH patients who may also have aplastic anemia. This therapy uses medicines to keep the immune system from attacking the bone marrow. Antithymocyte globulin (ATG) and cyclosporine are the medicines typically used.
• Eculizumab (Soliris®) is the only drug approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Evaluation Agency (EMEA) to treat PNH. It works by making your complement system (a part of your immune system) less active. This reduces hemolysis, the destruction of red blood cells.
• Bone marrow/stem cell transplantation replaces your unhealthy blood forming stem cells with healthy ones.
• Clinical trials, also called research studies, may also be an option for patients who do not have success with the other treatment options.

How long do I have to live?

Upon first diagnosis, many people want to know what to expect and how long they may have to live. This is called a prognosis. This is not an easy question to answer. Each person is unique and each person’s disease is a bit different. The course of a bone marrow failure disease varies a lot from person to person. You may have only very mild symptoms. Or, you may have severe symptoms and need medicines or blood transfusions.

Whether you are an aplastic anemia, MDS or PNH patient it is important to talk with your doctor about your prognosis. This may be hard to hear, but getting a prognosis will give you key information you need to make treatment decisions

and plan for the future.

Below are some prognosis guidelines based on the most current data available:

Aplastic Anemia

Today, with standard treatments, about 8 out of 10 aplastic anemia patients get better. Standard treatments include immunosuppressive therapy with antithymocyte globulin (ATG) with cyclosporine, or a bone marrow transplant. The chance for recovery depends on many factors, including how severe your case is and how you respond to treatment.

MDS

There are many different subtypes of MDS. These subtypes are based on tests of the blood and bone marrow. Identifying your MDS subtype is one of the key factors doctors use to both decide on the best treatment approach and make a prognosis (an educated guess about the likely course of your disease and how long your are likely to live).

To figure out a prognosis for a given patient, doctors will also use a prognostic scoring system. The most common one used today is the International Prognostic Scoring System, or IPSS for short. This system looks at three things:

1. Chromosome changes in the bone marrow cells

2. Number of low blood counts you have

3. Percentage of young white blood cells (blasts) in your bone marrow.

4. The number of cytogenetic changes you have in bone marrow cells (abnormal gene changes

This prognostic scoring system tells your doctor how severe your disease is and how likely it is that your MDS might become acute myeloid leukemia (AML). It also gives your doctor a general idea about and how long you are likely to live.

With current treatments, patients with lower-risk types of some MDS can live for 5 years or even longer. Patients with higher-risk MDS that becomes acute myeloid leukemia (AML) are likely to have a shorter life span. About 30 out of 100 MDS patients will develop AML.

PNH

Many people with PNH live for decades. People with PNH who develop blood clots in key parts of the body or develop MDS (myelodysplastic syndromes) or AML (acute myeloid leukemia), may have a shorter life span.

New treatments are becoming available that are helping PNH patients live longer. You may have seen older literature saying that PNH patients live an average of 15 to 20 years after diagnosis. More recent research shows that lifespan has been steadily climbing over the past 20 years. It is even possible that people with PNH will have a lifespan that is normal compared with people their age. Fortunately MDS and AML are rare in people with PNH.

Is aplastic anemia, MDS or PNH inherited?

Most cases of these diseases are not inherited. In other words, they are can not be passed down through the genes from parent to child. Most cases of aplastic anemia, MDS and PNH are considered to be “acquired” and the cause is not typically known. In very rare cases there are inherited bone marrow failure syndromes that can increase the chances of getting aplastic anemia or MDS.

Should I get a second opinion?

Even if you're happy with your doctor and healthcare team, it's OK to get a second, third, or even fourth opinion. Getting a second opinion will not offend your doctor. In fact, most healthcare providers appreciate and encourage another point of view. And it's your right.

Seeking a second opinion can help you and your family with making difficult decisions about your treatment. Be sure to contact your health insurance company to determine if the healthcare provider is covered under your plan, so you will know what your out of pocket expenses will be. While most insurance companies will cover a second opinion, it is a good idea to check with your insurance company before seeing the new healthcare provider. You should be aware of and keep track of your out of pocket expenses.

What questions should I be asking my medical team?

You likely have many questions for your healthcare team. It is important to keep an ongoing list of your questions between appointments. Be sure to take your list of questions with you and keep asking until you get an answer you understand. Don’t be afraid to ask why your doctor is running a test, scheduling a procedure, or changing a medication. Here are a few questions to consider:

About your disease:

• What subtype of my disease do I have?
• How severe is my disease?
• What is my prognosis?
• What have other people with a similar disease and treatment gone through?

About treatment:

• What are all my treatment options?
• What treatment option do you recommend for me? Why?
• Will I be getting more than one treatment?
• How does this treatment work?
• How likely am I to get better with the treatment?
• How often is this treatment given?
• Has this treatment been used a lot or is it a new or experimental treatment?
• When can I expect the treatment to start working? When/how will I know if it is working?
• What are some of the possible side effects I should be aware of? Are there long-term side effects?

How will having a bone marrow failure disease affect my life?

While it can be difficult to imagine in the days and months following diagnosis, many patients develop strategies for successfully coping with the physical and emotional aspects of living with aplastic anemia, MDS or PNH and lead happy, fulfilling lives. Making small changes in daily activities can make a big impact on how you feel. Fatigue is a big issue for many patients, so listen to your body and rest when you need to. Talk to your doctor about ways you can decrease and manage fatigue. With a bit of patience, planning, and flexibility, many patients find ways to work, travel, exercise, and do the things they loved before diagnosis. On our website you can search for tips on managing fatigue, diet and nutrition, exercise, emotional health and more.

Is there a special diet I should be on that would help me feel better? There is no specific diet that makes aplastic anemia, MDS, or PNH better or worse. Experts recommend you eat a well-balanced diet. Your doctor can help you find the best eating plan for you. You may also want to talk to a dietitian, nutritionist, or other member of your healthcare team. Some insurance plans cover nutrition counseling.

Make sure to check with your doctor before taking any medicines, supplements, vitamins, or herbs. They may interact with your medicines and prevent them from working or increase the risk of side effects.

If your white blood cell count is very low, your doctor may ask you to avoid certain foods that can make you sick. This is called a neutropenic diet. This diet is controversial because research has not proven its value. This is because most infections in patients come from the billions of bacteria that normally live in the mouth, intestines and skin. Nevertheless, you should use common sense and avoid foods that might cause unnecessary infection, and follow these guidelines:

• Avoid eating raw meats and fish
• Drink only beverages that have been pasteurized to kill germs. Milk you buy from the store is fine to drink
• Stay away from buffets
• Don’t eat leftovers

These are common questions patients ask about dealing with health insurance or medical records:

What is Medicare Part D and how can I find out if I am eligible?

Medicare coverage is divided into four parts.

• Medicare Part A covers hospital and inpatient care.
• Medicare Part B covers doctor visits and other outpatient care. Under Parts A and B, you can get care from just about any hospital or doctor you choose.
• Medicare Part C gives you an option to sign up for private managed care plans that cover all Medicare services and sometimes provides extra benefits, but these plans limit the providers you can see.
• Medicare Part D is a separate program that just covers prescription drugs that might be used at home.

Anyone eligible to Medicare Part A (whether actually enrolled or not) or who is currently enrolled in Medicare Part B may join Medicare Part D to get help paying prescription drug costs. Enrollment is voluntary except for people who also receive benefits from Medicaid (Medi-Cal in California).

How do I get my medical records from a doctor?

If you want a copy of your medical records, you must submit a written request to the hospital or the doctor's office. The law requires that patients provide a written authorization for a doctor to disclose medical records or the information contained in them. Some hospitals have their own form that needs to be completed. Patients may receive copies of their medical records, however the originals must remain in the physician's or hospital's file.