Considerations for the use of transfusion therapy alongside hypomethylating agents while managing myelodysplastic syndrome.
Amer Zeidan, MBBS, MHS: A large number of patients with MDS [myelodysplastic syndrome] will need blood transfusions. Anemia is the most common symptom and manifestation of MDS. If you look at all the patients with MDS, more than 80% will require blood transfusions. The data suggest that more than 40% will require frequent blood transfusions, or have transfusion dependence. Many patients will require blood transfusions. For platelet transfusions, the estimates vary, but especially for patients with high-risk MDS, when their platelet count is quite low, they often also need blood transfusions. Those are some of the reasons why we bring the patients to the clinic frequently, in addition to the hypomethylating agents that require intravenous or subcutaneous administration. This and the need for transfusions are the 2 most common reasons why patients have to come frequently to the clinics.
Bart Scott, MD, MS: When I see a patient with newly diagnosed MDS, some are already transfusion dependent. They needed red blood cells, and they needed platelets. If they are of good health, of intermediate or higher risk disease, and they are interested in receiving treatment, we would sometimes consider a hypomethylating therapy for them. Not all patients with higher risk disease are transfusion dependent. One of the interesting aspects of hypomethylating agents, in general, is that patients can feel worse before they start to feel better. What we find is that patients who receive hypomethylating therapy, both azacitidine and decitabine, generally have their counts worsen during the first 2 cycles. It’s important to be upfront and honest about that with patients so that they don’t lose their confidence in you. It’s also important that you have adherence to compliance.
On average, the median time to response to azacitidine is about 4 months, and the median time to response to decitabine is about 2 months. For azacitidine, the majority of patients who respond will respond by 6 cycles, which would be 6 months. For decitabine, the majority of patient who respond will respond by 4 cycles, which would be 4 months. If patients stop early, many haven’t received enough therapy to get a clinical benefit, and it is expected that the counts would get worse. If they're not transfusion dependent, then they could become transfusion dependent. If they are transfusion dependent, this could worsen. I try to encourage patients to complete a full duration of therapy with either the azacitidine or decitabine before we say that it doesn’t work. One of the biggest issues with both of these agents is that the drug is stopped too soon.