Newly Diagnosed

There is no specific diet that makes aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… , 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 white blood cell: Cells in the body that fight disease and infection by attacking and killing germs. There are several types of white blood cells including neutrophils, eosinophils, basophils, lymphocytes and monocytes. Each type of cell fights a different kind of germ. Also called WBC, leukocyte. count is very low, your doctor may ask you to avoid certain foods that can make you sick. This is called a neutropenic diet neutropenic diet: (noo-truh-PEE-nik) A diet for a patient with very low white blood cell count. A neutropenic diet avoids the use of certain foods that can may contain germs, such as raw meats, aged cheeses, fermented drinks, and unwashed fruits and vegetables. . This diet is controversial because research has not proven its value. That’s because most infections in patients commonly come from normal bacteria that live in everyone’s mouth, skin and intestines. Nevertheless, always use common sense and avoid foods known to cause unnecessary infection:

• Avoid eating raw meats and fish fish: See fluorescence in situ hybridization.
• Drink only beverages that have been pasteurized to kill germs, like milk you buy at the store
• Stay away from buffets
• Don’t eat leftovers

While it can be difficult to imagine at first, many patients lead happy, fulfilling lives by developing strategies for successfully coping with the physical and emotional aspects of living with aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… , MDS or PNH.

Making small changes in daily activities can have 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.

Visit the Caring for Yourself page for tips on managing fatigue, diet and nutrition, exercise, emotional health and more.

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 with you and keep asking your questions until you get the answers you understand. Don’t be afraid to ask why your doctor is running a test, scheduling a procedure or changing a medication. Here are some sample 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?

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 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 cover a second opinion, it is a good idea to check with your insurance company before seeing the new healthcare provider. You should be always keep track of your out-of-pocket expenses, along with your other medical records.

These diseases can not be passed down through the genes from parent to child. Most cases of aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… , MDS and PNH are considered “acquired” and the cause is not typically known. In very rare cases, inherited bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… syndromes can increase the chances of getting aplastic anemia or MDS.

How long do I have to live?

This is most people’s first question, along with “what can I expect to happen.” The doctor’s answer is called a prognosis (an educated guess about the likely course of your disease and how long you might live). Because each person is unique, and each person’s disease is different, a prognosis can be difficult to make. Also, how a disease progresses over time is unpredictable, and less is known about rare diseases than common diseases.

Whether you are an aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… , MDS or PNH patient, you need to talk to your doctor about your prognosis. This may be hard to hear, but getting a prognosis will give you key information about the treatment decisions you need to make so you can plan for the future.

Prognosis guidelines based on current data

Aplastic Anemia

With standard treatments, about 8 out of 10 aplastic anemia patients get better. Standard treatments include immunosuppressive therapy immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… with antithymocyte globulin antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. (ATG) with cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. and promacta, or a bone marrow transplant bone marrow transplant: A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due… . 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, which are identified by testing the blood and bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. . Your MDS subtype is a key factor in a doctor’s decision about your treatment and your prognosis (an educated guess about the likely course of your disease and how long you might live).

To figure out a prognosis for a given patient, doctors also use a prognostic scoring system. The most common one used today is the International Prognostic Scoring System International Prognostic Scoring System: A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS) case is progressing and helps predict what may happen with the patient's MDS in the future. Also called IPSS. -R, or IPSS-R for short. This system looks at three things:

• Number of low blood counts you have
• Percentage of young white blood cells (blasts) in bone marrow cells
• Number of cytogenetic changes (abnormal gene changes) in bone marrow cells

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 acute myeloid leukemia: (uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become… (AML). It also gives your doctor a general idea about how long you might 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. Most patients with AML need treatment soon after diagnosis because the disease often progresses fast. The initial goal is to put the patient into remission. The long-term goal is to cure the disease, although this is not always possible.

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.

Treatments available for PNH are helping people with PNH to live longer. Older research you may have come across state that PNH patients live an average of 15 to 20 years after diagnosis. More recent research shows that the PNH lifespan has climbed over the past 20 years. It’s possible that PNH patients – who rarely develop MDS or AML – will soon have a lifespan that is normal compared with people their own age.

It is always best to talk with your treating doctor about your prognosis. Remember, each patient is different.

PNH is considered a chronic disease meaning that it lasts a long time. The only potential cure is a bone marrow transplant (BMT) bone marrow transplant (BMT): A procedure where bone marrow stem cells are collected from marrow inside the donor's hipbone and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells. . However, a BMT carries many risks and is not an option for many people. Other treatments are designed to ease symptoms and prevent problems. These may include:

• Wait and watch:  Also called “watchful waiting,” your doctor might decide to do nothing but monitor your blood counts if they aren't too low and your symptoms aren't too bad.
• Supportive Care Supportive Care: Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive… :  Consist of therapies to help manage the symptoms of your PNH. They work to increase blood counts. Treatment is typically blood transfusions blood transfusions: A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some… and may include growth factors growth factors: Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts. Red… or taking extra iron (iron therapy).
• Blood thinners (anticoagulants) may be used on some patients to help reduce the chance of    having blood clots.
• Immunosuppressive therapy Immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… :  Lowers your body's immune response and is appropriate for PNH patients who also have aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… . This therapy uses medicines to keep the immune system from attacking the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. . Antithymocyte globulin Antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. (ATG) and cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. are the medicines typically used.
• Eculizumab Eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks. … (Soliris ®) was the first 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 complement system: A group of proteins that move freely in the bloodstream. These proteins support (complement) the work of white blood cells by fighting infections. less active and reduces hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. Soliris ® is approved for the treatment of patients with PNH in nearly 50 countries worldwide.
• Ravulizumab-cwvz Ravulizumab-cwvz: Ravulizumab-cwvz (Ultomiris®) is a drug approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat PNH. ULTOMIRIS® is a long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade.  ULTOMIRIS® is a prescription medicine called a…  (Ultomiris®) is a drug approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat PNH. ULTOMIRIS® is a long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade.
• Bone marrow/stem cell transplantation (BMT/SCT):  A procedure that replace your unhealthy blood-forming stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… with healthy ones from a matched donor. BMT is the only potential cure for PNH. Unfortunately, BMT is an “imperfect cure,” carrying many risks and potential long-term side effects. For many people a BMT is not a good option. When considering BMT, be sure to talk with your doctor about its potential impact on your long-term survival and quality of life when compared with other treatment options.
• Clinical trials Clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… :  Also called research studies, they may be an option for patients who do not have success with other treatment options.

MDS treatment is designed 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 may have. He may also consider whether someone is willing and able to donate matching bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. to you (preferably a family member).

There are a number of general approaches used to treat MDS depending on the subtype and severity of your MDS. These may include:

• Wait and watch:  Also called “watchful waiting,” your doctor might decide to do nothing but monitor you if your blood counts aren't too low and your symptoms aren't too bad.
• Supportive care Supportive care: Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive… :  These therapies help you manage your MDS symptoms. They work to increase blood counts, treat infections and treat iron overload iron overload: A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated. and typically include blood transfusions blood transfusions: A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some… and antibiotics antibiotics: The most common white blood cells are called neutrophils. They fight infection. Patients who don't have enough healthy neutrophils are said to have neutropenia. Because of their low white count, they may get infections easily and have trouble getting rid of infections. If you have neutropenia,… , and for some patients, may also include growth factors growth factors: Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts. Red… or iron chelation iron chelation: Iron chelation therapy is the main treatment used when you have a condition called iron overload. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of red blood cell transfusions. Basics Red blood cells contain iron. Each time you get a… .
• Immunosuppressive therapy Immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… :  Consists of medicines to keep the immune system from attacking the bone marrow, which can lower your body's immune response and is appropriate for patients with certain types of MDS. These medicines include ATG (antithymocyte globulin) and cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. .
• Drug therapies:  Approved specifically to treat MDS, they work to stop abnormal cells from growing and stimulate the growth of healthy bone marrow cells. These therapies include lenalidomide lenalidomide: Lenalidomide is a capsule that is taken by mouth. It is approved for treating low-risk, transfusion-dependent myelodysplastic syndrome (MDS) patients with an abnormality of chromosome 5q. It is currently in clinical trials to test its efficacy with a broader range of MDS patients. For some MDS… (Revlimid), decitaibine (Dacogen) and azacitidine azacitidine: It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Azacitidine is approved by the U… (Vidaza).
• Chemotherapy Chemotherapy: (kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with… :  Treats MDS by using drugs that kill abnormal cells.
• Bone marrow/stem cell transplantation (BMT/SCT): A procedure that replaces your unhealthy blood-forming stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… with healthy ones from a matched donor. BMT is the only potential cure for MDS. Unfortunately, BMT is an “imperfect cure,” carrying many risks and potential long-term side effects. For many people a BMT is not a good option. When considering BMT, be sure to talk with your doctor about its potential impact on your long-term survival and quality of life when compared with other treatment options.
• Clinical trials Clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… , also called research studies, may also be an option for patients who do not have success with the other treatment options.

Aplastic anemia Aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… treatment is designed 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 bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. to you (preferably a family member).

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

• Supportive care Supportive care: Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive… : Consists of therapies to help manage aplastic anemia symptoms. Aimed at increasing blood counts and treat infections, these treatments can include blood transfusions blood transfusions: A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some… and antibiotics antibiotics: The most common white blood cells are called neutrophils. They fight infection. Patients who don't have enough healthy neutrophils are said to have neutropenia. Because of their low white count, they may get infections easily and have trouble getting rid of infections. If you have neutropenia,… and for certain patients, growth factors growth factors: Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts. Red… or iron chelation iron chelation: Iron chelation therapy is the main treatment used when you have a condition called iron overload. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of red blood cell transfusions. Basics Red blood cells contain iron. Each time you get a… .
• Immunosuppressive therapy Immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… :  Works to lower your body's immune response. It uses medicines that keep the immune system from attacking your bone marrow stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… . ATG (antithymocyte globulin) and cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. are the medicines typically used. Recently, and drug called eltrombopag eltrombopag: What are the possible side effects of eltrombopag (Promacta)? Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat. Stop using eltrombopag and call your doctor at once if you have: … (Promacta) has started being used as part of immunosuppressive therapy for aplastic anemia. This drug is a platelet platelet: The smallest type of blood cell. Platelets help the blood to clot and stop bleeding. Also called a thrombocyte. growth factor growth factor: A substance made by the body that stimulates the bone marrow to produce blood cells. Some growth factors are man-made in the laboratory and used for treating low blood counts. These include red blood cell growth factors called erythropoietin (EPO) and darbepoetin, and white blood cell growth… , but seems to stimulate growth of red cells and white cells too.
• Bone marrow transplantation (BMT):  A procedure that replaces your unhealthy blood-forming stem cells with healthy ones from a matched bone marrow donor. BMT is the only potential cure for aplastic anemia. Unfortunately, BMT is an “imperfect cure,” carrying many risks and potential long-term side effects. For many people a BMT is not a good option. When considering BMT, be sure to talk with your doctor about its potential impact on your long-term survival and quality of life when compared with other treatment options.
• Clinical trials Clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… :  Also called research studies, they may be an option for patients who do not have success with the other treatment options.

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 bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… diseases such as aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… , MDS and PNH are hematologists (doctors specializing in blood diseases) and oncologists (doctors specializing in cancer).

While AAMDSIF does not recommend specific specialists, our knowledgeable information specialists can help you identify a few physicians with deep expertise in your disease that you can choose from. To speak with an information specialist you can call (800) 747-2820 or email help@aamds.org.

To find an expert, you can also call the hematology/oncology department at any 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 typically most familiar with rare diseases. They work in  research and are up-to-date on both standard therapies and new investigational treatments.

When contacting a specialist, consider asking 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 clinical trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a… 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 going to be an issue?