Roswell Park-Led Study Shows Promise for New Targeted Combination in People With Relapsed AML - Scorecard - MDSpire
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Roswell Park-Led Study Shows Promise for New Targeted Combination in People With Relapsed AML

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  • August 26, 2026

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Clinical Scorecard: Roswell Park-Led Study Shows Promise for New Targeted Combination in People With Relapsed AML

At a Glance

CategoryDetail
ConditionAcute Myeloid Leukemia (AML)
Key MechanismsCombination therapy using ziftomenib, venetoclax, and azacitidine.
Target PopulationPatients with relapsed or refractory NPM1-mutated AML.
Care SettingClinical trial setting.

Key Highlights

  • Nearly 90% response rate in previously untreated patients.
  • 70% achieved complete or near-complete remission.
  • Responses occurred quickly, within about four weeks.
  • Treatment was generally well tolerated with low rates of serious side effects.
  • Study published in the journal Blood.

Guideline-Based Recommendations

Diagnosis

  • Focus on patients with NPM1-mutated AML.

Management

  • Ziftomenib in combination with venetoclax and azacitidine remains investigational for relapsed AML.

Monitoring & Follow-up

  • Monitor for response rates and side effects during treatment.

Risks

  • Limited treatment options for aggressive leukemia.

Patient & Prescribing Data

Patients with relapsed or refractory AML.

Ziftomenib is approved as a single agent; combination therapy remains investigational.

Clinical Best Practices

  • Utilize highly sensitive testing to detect measurable cancer cells.
  • Support ongoing studies of ziftomenib in combination therapies.

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