- What is the Aplastic Anemia and MDS International Foundation?
The Aplastic Anemia and MDS International Foundation (AAMDSIF) is dedicated to assisting and supporting people who have bone marrow disorders, including aplastic anemia, myelodysplastic syndrome and paroxysmal nocturnal hemoglobinuria. Unlike typical non-profits and advocacy groups that have a singular focus, AAMDSIF concentrates on these three different diseases and related bone marrow failure illnesses that cover the whole spectrum of bone marrow disorders, as they are all interlocked, and patients are likely to progress from one to the other.
The world’s leading nonprofit organization for people living with bone marrow failure diseases, the Foundation provides answers, support and hope to thousands of patients and their families around the world. As a patient-centered organization, AAMDSIF serves patients and loved ones throughout the three phases of bone marrow failure diseases: the life changing phase of diagnosis, the life-threatening phase of treatment and the lifelong phase of living with a chronic disease. The Foundation provides patients access to the most updated information on these diseases, along with a wealth of helpful informational resources, support services and caring communities. A medical advisory board composed of hematologists and oncologists from major hospitals in the U.S. and around the world review and update all resource material, as well as the website, ensuring all information is accurate and current.
The organization was started 37 years ago by a father whose son died from aplastic anemia, a typical outcome at that time. Great progress has been made since then, as today there are treatments for all three major bone marrow disorders, and many people are successfully living with these diseases. Much of this progress is the result of research grants given out by AAMDSIF to encourage young researchers in oncology and hematology to focus their ongoing clinical research efforts on bone marrow disorders. Very few rare diseases have effective treatments, but these three conditions each have viable therapy options, proving the validity of the Foundation’s strategy to support young researchers early in their careers.
- What do bone marrow disorders do to the body and what are the symptoms patients typically face?
The symptoms of bone marrow diseases vary depending on the specific disorder, and they can be different from person to person. Some people may have severe symptoms, while others have mild or no symptoms at all. Depending on the specific disorder, low blood counts—either for red blood cells, white blood cells and/or platelets—may correlate with the following symptoms:
Low red blood cell levels (anemia)
Red blood cells carry oxygen to cells, so a low level can make patients feel tired and rundown. They may also have a decreased appetite, headaches, dizziness and pale skin.
Low white blood cell levels (leukopenia)
White blood cells fight infections, so patients with low counts may be sick more often, get more infections and also take longer to recover. Common symptoms include: mouth sores and sinus, throat, skin, urinary tract and lung infections.
Low platelet levels (thrombocytopenia)
Platelets help the blood clot to stop bleeding. Low levels may cause: bruising or bleeding easily, bleeding gums, nose bleed, and bleeding under the skin, most often on the lower leg.
The long-term outlook for patients with bone marrow disorders is rapidly improving. Recently, therapies and advances were developed that reduce bone marrow transplant side effects and that make the procedure available to older patients who were once ruled out. Additionally, a 100% donor match is no longer required for success, and there is much more awareness about the need for bone marrow donors.
- What are some of the biggest challenges patients face who have bone marrow disorders?
There are many difficulties that go along with these disorders. By the time patients are diagnosed, they are usually experiencing low energy that may progress to exhaustion. It is an invisible disability, so people will not notice that a person has this condition. While patients may not want others to know about their disease, the reality is they may not be able to function normally, either for work or in relationships, creating psychological challenges.
Hospitalization is required for many of the treatments for bone marrow disorders, such as transfusion and transplantation, creating disruption in the patient’s life. Treatment may also require intensive support from caregivers, especially if a patient receives a transplant, and this can be very taxing on the relationship between the caregiver and the patient. The high level of care needed after transplantation can also present a huge impediment for patients living alone.
- What resources does AAMDSIF offer patients?
The AAMDSIF takes great pride in providing a high level of personal assistance to patients. The phone is always answered by knowledgeable, compassionate staff who are there to offer immediate support, helping patients understand their condition and educating them about the important questions to ask their doctors. The organization also facilitates referrals to connect patients with the best experts in the field to treat their disease.
Other support is available. There is information about each bone marrow disorder on the organization’s website, or patients may call and request detailed printed information at no cost. Additionally, there is a volunteer network of patients, so patients can be matched with others who can offer advice and support. Because these disorders are so rare, most patients have never talked to anyone who has the same disease they do, so they often feel lonely and isolated. Connecting with others battling the same illness is an incredible gift.
Patient and family conferences are normally held in regional locations across the country each year, but they are currently virtual due to COVID-19. These conferences are free to attend and include presentations by leading medical experts in rare blood cancers and bone marrow failure diseases. In addition to being able to meet these specialists, patients can also connect with one another at the conferences, bonding and sharing their experiences.
Frequent webinars are also held featuring international experts, and hundreds of past webinars are available on the organization’s website. Additionally, support groups for each disease and all treatment stages are easily accessible online, providing an important lifeline that helps patients cope and adapt to their situation.
- What outreach activities does the AAMDSIF use to connect with patients and families?
The organization is very active on all major social media channels, including: Facebook, Twitter, YouTube, LinkedIn and Instagram. Anyone visiting the website is encouraged to provide an email address, as bulletins with updated information, newsletters and patient stories are sent out on a regular basis. Additionally, a special effort is made to reach out to caregivers and family members, as they are often the first ones to come to the AAMDSIF website. They receive the same information and personal support provided to patients, and there is also a caregivers support group.
- What volunteer opportunities are available with the AAMDSIF? How can people support the organization?
The AAMDSIF provides all of its vast support services free of charge to patients, so the organization greatly appreciates financial support, including cash donations and fundraisers. Regional fundraising walks organized by volunteers are held in Texas; California; Washington, D.C.; New York; and Michigan. These events provide an uplifting and exciting way to spread awareness about bone marrow failure diseases, as patients, families, friends, colleagues and neighbors typically participate. The organization provides assistance to anyone planning a walk. Other volunteers hold their own events, such as raffles and Facebook birthday fundraisers.
Additionally, people can support patients by becoming a bone marrow donor or placing themselves on the list of people willing to donate. These generous people may eventually be matched with someone in need, saving a life.
- Where can patients get the most current information on new advances in bone marrow disorders?
The AAMDSIF is the best place to find the most up-to-date information about bone marrow diseases, advances, treatments and clinical trials. The Foundation’s website and social media channels always have the most current information and breaking news.
The website also serves as a vehicle for patients to find a clinical trial that might be right for them. Clinical research is at the heart of all advances that have been made for bone marrow diseases, and the AAMDSIF is proud to play an important role in connecting patients to appropriate trials, providing them the opportunity to participate in advancing the knowledge base for bone marrow treatments.
- What closing thoughts should readers remember?
Great progress can be made in treating bone marrow disorders when everybody works together. The AAMDSIF is dedicated to advocacy and looks forward to collaborating with other non-profits, payers, pharmaceutical companies and government entities to increase funding for research and to develop innovative solutions that reduce treatment costs to patients.
The organization has tremendous resources and highly knowledgeable, compassionate staff who can help patients during all phases of bone marrow failure diseases. We encourage patients and their loved ones to utilize AAMDSIF’s resources, as the Foundation can provide hope and healing to support them on their treatment journey.