Frontline Eltrombopag for Severe Aplastic Anemia - Episode 7
Phillip Scheinberg, MD: Supportive care has been a very important element of management of patients with severe and very severe aplastic anemia. Now, let me tell you 2 sides of this. Remember that when patients are treated with immunosuppressive therapy, for example, they take 2 to 3 months to respond. Even among those patients who are going to respond, they take 2 or 3 months to respond. So the first 8 weeks, actually, they haven’t yet responded, even those who will eventually become really good responders.
So you have to support these patients very aggressively with platelet transfusions, red cell transfusions. You have to follow them very carefully. You have to monitor their cyclosporine. You have to monitor for toxicities. You have to keep them well and alive without complications to give them time to respond to these regimens. With fever and neutropenia, you have to be very prompt. You have to bring them to the hospital. So nobody is going to respond the next day. You have to be aware of that. Supportive care is critical, especially in that phase. Now, if somebody responds and they don’t require transfusions, the role of supportive care starts to become less important. But in the beginning, it’s critical that you have a very systematic and close approach to these patients with blood banking, the infectious disease consultants, and the ward.
The second facet of this discussion is the analysis we conducted at NIH [National Institutes of Health], where we looked at those patients who failed immunosuppressive therapy and were, in other words, refractory; historically they remained pancytopenic for 6 months during the 1980s and thereafter. What we did notice was that, although survival in the 1980s was in merely 10% to 15% of patients who failed initial immunosuppressive therapy, in the 1990s, we began to see an increase: About 30% to 40% were experiencing survival; and in 2002, the trend continued, with a survival benefit of 50% to 60%.
So the interpretation of that, in part, is that we have done a lot better in supporting patients for longer. Even those patients who did not respond to immunosuppressive still required blood and platelet transfusions and still required antibiotics. All of that improved. The antifungals improved. Our ability to support patients even with very severe forms of pancytopenia has actually improved. So this is a testament to how far we have come in terms of supporting patients, not only waiting for the time for their response but also supporting them if they were to get a second round of immunosuppression. We can keep them out of trouble, or even if they had gone to transplantation, we can also keep them out of trouble in terms of these complications by supporting them.
Supportive care is a series of things that involves blood banking, safer blood products, filtering, antibiotics, and antifungals, and all that kind of stuff. So if supportive care were a drug, it would be a very successful drug. The thing is, it’s kind of a mixture of several things, and it’s not 1 thing, and nobody kind of owns it. It’s a bunch of different things that are benefiting patients with not only aplastic anemia but also other diseases like AML [acute myeloid leukemia] induction and transplant, etc.
So remember, this is a disease in which the marrow fails. People become very, very dependent on supportive care. Aplastic anemia is probably one of the diseases that stress the system in terms of supportive care or even more so than other diseases. That’s why it’s important that patients are treated in a scenario, in a facility where all of this has been provided to them so they can actually benefit.
I have had patients—this is kind of interesting, this analysis, when we pulled the records from patients who actually did not respond to immunosuppression many years ago—and I didn’t think many of them would be alive. And when we went back, several of them were because they were maintained on all forms of supportive care, and after a year or 2, their bone marrow started to get a little bit better, and they started to improve with some growth factor. Sometimes they got androgen, all kinds of different [therapies]. And maybe time alone just helped them. But again, they were maintained during this time period, so I think supportive care is a major component to the management of these patients.
Transcript Edited for Clarity