Moderate, Severe, and Very Severe Aplastic Anemia: How are treatment decisions made? | Aplastic Anemia and MDS International Foundation

Moderate, Severe, and Very Severe Aplastic Anemia: How are treatment decisions made?

Dr.Townsley is a staff clinical investigator in the Hematology Branch of the National Heart, Lung, and Blood Institute (NHLBI) at the Clinical Center of the National Institutes of Health (NIH) in Bethesda, Maryland. Her research interests include the pathophysiology of marrow failure syndromes and the use of eltrombopag, a blood growth factor to treat aplastic anemia. Dr. Townsley is the principal investigator for multiple trials using immunosuppression and eltrombopag to treat aplastic anemia and myelodysplastic syndromes. Here, Dr. Townsley speaks about the classification of aplastic anemia and the treatment decision making process that accompanies each category.

Are treatments for each category aplastic anemia (moderate, severe, and very severe) also category-specific or are they decided more on a case-by-case basis?

That’s correct—an aplastic anemia diagnosis is categorized in one of these three groups. The severe and very severe categories always require treatment. Usually the type of treatment is dependent on age, but once the cytopenias (low blood counts) are severe or very severe, some kind of treatment must occur. Moderate aplastic anemia means the blood count criteria do not qualify as severe and treatment is not always required – there can be a ‘watch and wait’ approach. Treatment is usually indicated based on the need for transfusions.  So if patients who have moderate aplastic anemia need transfusions, some sort of treatment is usually recommended. The type of treatment is not standard and could vary, depending on the patient. There are different treatment options that usually involve some sort of immunosuppression, as basic as cyclosporine alone, but also eltrombopag is being tested at the NIH for moderate aplastic anemia.

Does the advent of the newly approved drug eltrombopag (Promacta®) affect treatment decisions?

It does, but at this point it is only approved for patients with refractory aplastic anemia. This is when there is no response or success from earlier immunosuppressive treatment. It should be distinguished from recurrent or relapsed disease, where there is a response, but the aplastic anemia returns. In our trials here at NIH, roughly half of refractory patients responded to eltrombopag. Now we have an option for those who failed one or even several rounds of immunosuppression. But it also means we have a potential therapy that will be interesting to explore for upfront disease – people who are newly diagnosed, and even those with only moderate aplastic anemia. This is currently being investigated in clinical trials at the NIH.

Have you found it challenging to help patients understand that this is not “just anemia”?

Actually what is more challenging is making people understand that what they have is not leukemia. We find that many patients come to us thinking they have a leukemia-like cancer. This may be because they are referred from a doctor’s office where they’re in an environment with other cancer patients and even may have been told that what they have is something akin to a leukemic cancer. Many doctors have rarely seen aplastic anemia, and it is more common to see pancytopenia associated to leukemia. So often patients have been told they may have leukemia even before bone marrow biopsy results are in. So for patients with severe aplastic anemia, we want them to understand that, while not leukemia, it is a very serious disease, but is very treatable.

For patients with moderate aplastic anemia who may not be transfusion-dependent and may feel fine even though they have a low platelet count -- it can be more difficult to get them to understand there are risks and that they need monitoring and perhaps treatment.  This is most often the case with younger patients – adolescents or young adults. 

Have you found it challenging to help patients understand that this is not “just anemia”?

Actually what is more challenging is making people understand that what they have is not leukemia. We find that many patients come to us thinking they have a leukemia-like cancer. This may be because they are referred from a doctor’s office where they’re in an environment with other cancer patients and even may have been told that what they have is something akin to a leukemic cancer. Many doctors have rarely seen aplastic anemia, and it is more common to see pancytopenia associated to leukemia. So often patients have been told they may have leukemia even before bone marrow biopsy results are in. So for patients with severe aplastic anemia, we want them to understand that, while not leukemia, it is a very serious disease, but is very treatable.

For patients with moderate aplastic anemia who may not be transfusion-dependent and may feel fine even though they have a low platelet count -- it can be more difficult to get them to understand there are risks and that they need monitoring and perhaps treatment.  This is most often the case with younger patients – adolescents or young adults.
Interviewee: 

Danielle Townsley, MD, MSc

Position / Title: 
Staff Clinician and Primary Investigator
Institution: 
National Heart Lung and Blood Institute

Dr. Danielle Townsley is a Staff Clinician and Primary Investigator in the Hematology Branch of the National Heart Lung and Blood Institute (NHLBI) at the Clinical Center of the National Institutes of Health in Bethesda, Maryland. She received a Bachelor’s Degree at the University of California, San Diego in 1999 and a Master’s Degree in Epidemiology from the University of Edinburgh, Scotland in 2002. She completed her MD in 2006 and a residency in Internal Medicine at George Washington University in 2009.  In 2011, she completed her hematology fellowship at the NIH where she trained under the mentorship of Drs. Neal Young and Phillip Scheinberg.

Currently she conducts her research in the laboratory of Dr. Neal Young, Chief of the Hematology Branch of NHLBI. Dr. Townsley’s research interests include the pathophysiology of marrow failure syndromes and the use of eltrombopag, a blood growth factor to treat aplastic anemia. She also studies familial causes of bone marrow failure, such as telomere diseases or dyskeratosis congenita, and the impact of inherited and acquired gene mutations on the pathophysiology of bone marrow failure. She is the principle investigator for multiple trials that utilize eltrombopag for aplastic anemia and myelodysplastic syndromes, as well as clinical trials administering the sex hormone, danazol for patients with telomere disorders.

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