Aplastic Anemia and MDS International Foundation: 2018 Scientific Symposium

At the 2018 Aplastic Anemia and MDS International Foundation’s (AAMDSIF) International Bone Marrow Failure Disease Scientific Symposium of March 2018, Dr. David Margolis from the Medical College of Wisconsin, and Dr. Danielle Townsley of MedImmune, discuss treatment Options for aplastic anemia. During their talk the two discuss precision medicine, eltrombopag, and bone marrow transplantation.


Aplastic anemia is a serious blood disorder that damages stem cells, causing the body’s bone marrow stops making enough new blood cells. The condition can either develop over time or appear suddenly, and there is no specific age range that is more at risk than another. To learn more about aplastic anemia, click here.


Dr. Margolis starts off the lecture  with good news, stating that treatment options “keep on improving.” Margolis’s emphasis for the talk was to focus on precision when talking to a patient and their family in finding the right regimen of treatment.


Dr. Townsley defines the aspects needed to develop an effective mode of treatment, stating that it is a combination of things that make up a patient, from their results in the laboratory, to their patient history, and their family history.


Recent data out of Japan has illuminated some confines for what kind of patient responds to transplant therapy. There are new definitive studies showing how and what kind of patient can benefit from PNH clones. The doctors taught all these innovations optimistically, listing off one after another.


Eltrombopag is a prescription medicine used for people with low blood platelet counts when other medicines or surgery to remove the spleen have not worked. The doctors detailed the lengths at which this medication is promising a better mode of treatment for patients. Dr. Townsley also notes the indication that eltrombopag has not been shown to mutate cells as previously feared, which could cause aplastic anemia to mutate into myelodysplasia .


“Yes. We’ve now moved on from non-transplant therapies which are improving, to transplant therapies, which are improving,” Dr. Margolis explains.


The doctors claim that transplant therapies are no longer just an option for children as up to 75 to 80 percent of adult non-related transplants have been successful.

Essentially, the exciting aspect of the talk contains the idea that patients can have a wider variety of care, indicating a greater personalization of treatment of their aplastic anemia.

This sentiment is highlighted for a final time in the closing statements from Dr. Townsley,

“we have so many options for patients. For physicians to be able to say we have all these different things we can offer… We just need to figure out which order, who we offer what. It’s a great place to be.”


Both doctors highly encourage people living with aplastic anemia to participate in clinical research to continue the effort in learning more about the disease in a more individualistic way. Donating one’s time and body to efforts such as these can be instrumental in ensuring new treatments that can be more personalized and varied for every patient.


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