Formulary Perspectives in MDS and AML: Novel Treatment Options for High-Risk Subtypes

At the virtual Great Debates and Updates in Oncology Pharmacy meeting, Christopher Fausel, PharmD, MHA, BCOP, Precision Genomics Oncology Pharmacist, Indiana University Simon Comprehensive Cancer Center, Indianapolis, Indiana, gave a presentation on novel treatment options for high-risk subtypes of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) from the formulary perspective.

Dr Fausel described the effect of complex disease pathophysiology in high-risk MDS and AML subsets, including relapsed/refractory AML and secondary AML, on formulary decision-making; evaluated the evolving treatment paradigm in this patient population using pharmacodynamic data from clinical trials; and summarized pharmacy-based approaches for transitioning high-risk AML treatment to the outpatient setting.

Per revised International Prognostic Scoring System (IPSS-R), high-risk MDS is defined as a risk score greater than 4.5 and bone marrow blasts greater than or equal to 10%. Classic treatment options for high-risk MDS include hypomethylating agents (azacytidine, decitabine, decitabine/cedazuridine), immunosuppressive therapy (antithymocyte globulin and cyclosporine), intensive chemotherapy, and allogeneic hematopoietic stem cell transplant.

Historical treatment strategies for AML include cytarabine or daunorubicin in the induction phase, cytarabine or autologous or allogeneic stem cell transplant in the consolidation phase, and reinduction chemotherapy, autologous/allogeneic stem cell transplant, clinical trial, or palliation in the relapse phase.

Since 2017, 12 agents have made it to the market for the treatment of AML. Of these, Dr Fausel highlighted gemtuzumab ozogamicin, the liposomal formulation daunorubcin/cytarabine, enasidenib, ivosidenib, gilteritinib, and venetoclax.

Dr Fausel outlined a few considerations for pharmacists, including MDS/AML involves a highly specialized patient population, specific training and expertise is required, supportive care is critical to the survival of these patients, risks of bleeding and infection inform all drug-therapy decision-making, and transitions of care.

Pharmacists can reduce medication errors by recommending newer, simplified chemotherapeutic regimens; ensure genomic/biomarker testing and recommend therapy that are target specific genetic factors to avoid ineffective treatments; monitor/mitigate adverse events; provide patient education on toxicity, treatment regimen logistics, and supportive care; and establish treatment standard to drive formulary decision-making.—Janelle Bradley