Targeted Treatment for IDH2-Mutated AML

August 8, 2018

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