Targeted Treatment for IDH2-Mutated AML

A 48-Year-Old Male With Chemo-Refractory AML

• A healthy 48-year-old man visited his PCP for flu-like symptoms lasting more than 2 weeks. He is married, with 3 school-age children and is an avid golf and tennis player.
• PE: mild petechiae on lower extremities; otherwise unremarkable
• Labs:
  • WBC, 65,000 (90% blasts)
  • Hb, 8.5 g/dL
  • Platelets 65,000/mL
  • ANC 2.5/mm³
  • LDH, 392 U/L
• Bone marrow biopsy:
  • 50% blasts
  • Cytogenetics; +8
  • NGS;IDH2(R140Q) mutation
• Liver and cardiac workup, WNL
• The patient received 7+3 induction chemotherapy and subsequently reinduction without achieving a remission
• The patient was then started on enasidenib
• He achieved stable disease after 2 cycles of therapy
• After 3 cycles, peripheral blasts, 15%; ANC, 1.1/mm³
  • 2 weeks later, patient reports dyspnea on exertion and mild swelling
  • PE notable for rales bilaterally
  • Chest X-ray shows bilateral diffuse pulmonary infiltrates
  • Additionally, indirect bilirubin, 1.9 mg/dl
  • Patient was started on dexamethasone 10 mg bid and antibiotics; pulmonary symptoms resolved in 1 week
• Bone marrow biopsy after 6 cycles shows morphologic CR, 2% blasts by FC; NGS shows persistence of mutantIDH2
• Patient referred for allogeneic transplant