Pillar 2: Learn About Your Disease and Treatment Options

Remember, knowledge is a powerful part of self-advocacy.

Bone marrow failure diseases are complex. You need to learn all you can about your condition and the available treatments. The more you know, the better off you are to make informed choices about your care.

Staying well informed helps you be a powerful player on your healthcare team. It allows you to educate family and friends, get more out of office visits, make choices about your treatment, and keep an eye on your care.

Today, it's easier than ever to learn about your disease and treatment options. 

Great places to find disease and treatment information:

  • Request a patient information packet from AA&MDSIF. You can order a free information packet from AA&MDSIF right on our Web site, or you can call us at (800) 747-2820.
  • Go to the disease sections of our Web site. Our Web site has a wealth of information on aplastic anemia, MDS and PNH.
  • Watch videos on the Online Learning Center. You'll find video presentations, interviews with medical experts, and interactive learning modules. You can even take part in a live webinar.
  • Talk to your healthcare providers. Ask questions and request materials. But don't rely on your healthcare providers as your only source of information.
  • Talk to the AA&MDSIF patient educator. Call us at (800) 747-2820, option 1.
  • Attend our patient and family conferences. AA&MDSIF patient conferences are great places to learn more about your disease. You can hear from leading medical experts, meet and speak with other patients, and pick up materials.
  • Visit the National Library of Medicine Web site. You'll find reliable and detailed information on many diseases. They have a simple search tool on the main page. From this site you can also use MedLinePlus, a Web site for patients and their families and friends maintained by the National Institutes of Health.
  • Gather and organize. Once you've gathered all the information you can on treatment options, organize it. Then share what you've learned with healthcare providers, family, and friends. Learn more by reviewing Pillar 4.

Find Trustworthy and Reliable Information on the Internet

As you know, there is a lot of information on the Internet. You'll want to find accurate and current information. Use these tips for finding good and reliable information on the Web:

  • .gov identifies a government agency
  • .edu identifies an educational institution
  • .org identifies nonprofit organizations (e.g., scientific, advocacy groups, foundations)
  • .com identifies commercial Web sites (e.g., businesses, pharmaceutical companies, sometimes hospitals)
  • Call the Patient Advocate Foundation at (800) 532-5274 to see if they can help with fees. Ask about their Co-Pay Relief program. Be sure to visit their Web site.
  • Learn about patient assistance programs from drug makers. Go to RxAssist.
  • Contact the Chronic Disease Fund at (972) 608-7141, or toll-free at (877) 968-7233. This nonprofit organization provides financial help to seriously ill Americans who have insurance, but cannot afford their medicines.
  • Contact the Leukemia & Lymphoma Society (LLS) Patient Financial Aid Program. Call (800) 955-4572 to locate a chapter near you. The program provides a limited amount of financial assistance to help patients with significant financial need who are under a doctor's care for a blood cancer disease.
  • Search state insurance and co-pay assistance programs. Go to the Families USA Program Locator. This Web site includes listings of health insurance assistance programs by state. These programs handle many questions about Medicare, Medicaid, and private insurance and may be able to refer you to low-cost or free health care (which may include prescription drug assistance).
  • Join a clinical trial. Find out if there are any free or low-cost clinical trials to participate in. If trial-related costs are of concern to you, the study doctor or nurse can answer questions about what is covered and what costs you or your insurance will be expected to pay. Visit Clinicaltrials.gov.
  • MedlinePlus: This Web site offers a wealth of information about health topics, drugs, and supplements. It also provides videos and interactive tutorials. The Web site is maintained by the National Institutes of Health. 
  • Drugs@FDA:  This Web site, developed by the U.S. Food and Drug Administration (FDA), provides detailed information about all FDA approved drug products. You can easily search by generic or brand name. 
  • Guides Help Patients Find Their Best Treatment Options:  Prepared by Carolyn Clancy, M.D., Director of the Agency for Health Research and Quality (AHRQ), this brief, easy-to-understand advice column is designed to help consumers navigate the health care system.
  • 20 Tips to Help Prevent Medical Errors: from AHRQ
  • Visit the AA&MDSIF Patient Resources page. Here you will find a variety of financial resources that can help with insurance co-pays, medication costs and more.

Is it clear who sponsors the Web site?

Look for Web sites that end in .gov, .edu, or .org.

Is there a way to contact the Web site sponsor?

Trustworthy Web sites have contact information. Many even have toll-free numbers to call.

Is there an editorial board that reviews the information?

Click on the "About Us" page of a Web site to see if there is an editorial board that reviews the information before putting it online. Government Web sites may not have this page.

Is the information current?

New research findings can make a difference in making medically smart choices, so you'll want to look carefully to find out when the Web site was last updated. Older information isn't useless, however. Many Web sites provide older articles so readers can get a historical view of the information.

Could the claims be too good to be true?

Be careful of claims that any one remedy will offer dramatic cures. Talk with your healthcare team about treatment claims.

Learn more

Read about finding reliable online information by using this resource page on our Web site.

Learn About your Treatment Options

If you have a rare disease, it's important to be well informed -- even before you visit the doctor -- so you know the right questions to ask.

--Mikkael Sekeres, MD, hematologist / oncologist

When the time comes to make a decision about treatment, use reliable information, along with input from your healthcare providers, family, friends, and others. Weigh the pros and cons of each treatment. Think about how well it might work, side effects, and costs. Consider how it might affect your lifestyle.

Then work with healthcare providers to develop a care plan you want to do and feel you can do. Remember: you have the right to choose or refuse treatment.

In the months and years to come, you will want to keep track of how well your care plan is working. Note your symptoms and side effects. Review the plan with healthcare providers regularly to see if it can be improved.

When you visit your doctor, it is important to take a list of questions to ask your doctor about treatment. Use this list of questions to consider for your next doctor's visit.

Remember - always talk to your doctor before you stop any medicine. If side effects are tough to handle, your doctor may be able to prescribe a medicine to lower the side effects.

Need help paying for healthcare provider visits or treatments?

It's scary to be diagnosed with a rare disease. Education eases the fear factor. It gives the patient empowerment. You realize: the more I understand this disease, the better I can deal with it.

--Stephen, aplastic anemia and PNH patient

What if your health insurance doesn't cover seeing specialists or certain treatments? Read below to find out where you can get help.

Thinking of participating in a clinical trial?

If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. These studies can offer good treatment options for some people with bone marrow failure diseases like aplastic anemia, MDS, and PNH.

Clinical trials are used to test new treatments before they're made available to the public. If the new treatment proves to be better than the current treatment, the new treatment can then be approved for general use by the FDA (U.S. Food and Drug Administration).

Learn more about clinical trials.

Learn More