Acute myeloid leukemia in the elderly: what constitutes treatment value?

Abstract

Treatment options for patients with acute myeloid leukemia (AML), who are unfit for induction chemotherapy are unsatisfactory. Overall survival (OS) superiority has not been demonstrated in randomized controlled trials (RCT) in this population, challenging the value of available therapies. We sought to assess the relative value of approved therapies using value-assessment tools. Clinical, safety, quality-of-life (QOL), supportive care, and resource utilization outcomes data were abstracted from RCTs and examined using value-assessment frameworks. Three RCTs, one each of azacitidine, decitabine, and low-dose cytarabine were identified. OS was not statistically significant and secondary outcomes including response rates, rates of transfusion independence, the frequency of hospitalizations and changes in QOL were reported differently across trials. Value-assessment tools considered OS as the primary efficacy endpoint without consideration to response rates. The NCCN Evidence Blocks^TM were most successful in considering secondary endpoints. With the move toward value-based care, understanding how these value tools apply to AML patients is critical.

Keywords:

• Acute myeloid leukemia
• elderly
• induction treatment
• value

Note

Potential conflict of interest

Disclosure forms provided by the authors are available with the full text of this article online at https://doi.org/10.1080/10428194.2018.1520992.

Notes

1 The US FDA has not provided guidance on the use of the odds ratio to support drug approvals or labeling claims.