I think cost-effectiveness analyses are a really interesting area of research. However, there certainly is more focus on a healthcare system perspective rather than for an individual patient. But however, in a setting where we have limited financial resources in our healthcare system, really optimizing how we use those resources is an important area of research. So the background for this was the randomized ETAL-1 trial, which was a trial conducted in Germany where patients with intermediate-risk AML who were 18 to 59 years of age and had newly diagnosed AML were randomized to either proceeding to an allogeneic stem cell transplant in first remission or to continue on with consolidation chemotherapy...
I think cost-effectiveness analyses are a really interesting area of research. However, there certainly is more focus on a healthcare system perspective rather than for an individual patient. But however, in a setting where we have limited financial resources in our healthcare system, really optimizing how we use those resources is an important area of research. So the background for this was the randomized ETAL-1 trial, which was a trial conducted in Germany where patients with intermediate-risk AML who were 18 to 59 years of age and had newly diagnosed AML were randomized to either proceeding to an allogeneic stem cell transplant in first remission or to continue on with consolidation chemotherapy. And there’s like, in the trial, they could show that the disease-free survival is better with an upfront transplant, but there was no difference in overall survival. And given that transplant comes with significant toxicity, including sometimes lethal side effects from the transplant itself, and is a very costly undertaking, it is really unclear what is the optimal treatment strategy for patients with intermediate-risk AML. So with this context, we conducted a cost-effectiveness analysis that used a partitioned survival analysis model. And what we could show there is that really proceeding to an allogeneic bone marrow transplant in first remission is really a more cost-effective strategy than waiting for patients to relapse and then doing the transplant at the time of relapse. So I think that is largely driven by the fact that yes, for intermediate-risk patients, most patients ultimately do require a bone marrow transplant because they are at such a high risk of relapse that it is really, in our analysis, it was just more cost-effective to proceed with a bone marrow transplant right from the beginning where outcomes tend to also be better than and there are higher chances of actually getting a patient to a transplant compared to a transplant at the time of relapse. However, it’s just a cost-effectiveness analysis, it should not guide clinical decision making but in a context where it is clinical equipoise and cost-effective consideration may play a role, I think this will provide some additional rationale to proceed to a bone marrow transplant early in the disease course in this context. But certain limitations also apply to the study, one being that this was conducted from a US healthcare system perspective, and you cannot really comment on other healthcare systems, for example, UK-based or other European countries. So as really like from a US healthcare system perspective, and only in the context of younger patients with intermediate-risk newly diagnosed AML.
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