Condition

Acute myeloid leukemia

Clinical trials and treatment information for Acute myeloid leukemia

204K

People Affected

Active Trials

123K

New Cases/Year

130K

Deaths/Year

Treatment Rankings

Ranked by evidence volume first. Medicine has spent centuries ranking things by vibes. This is an improvement.

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT)

90% Effectiveness• 95% Confidence• 20% Safety• 100 trials• 100K participants

HIGH EvidenceModerate ValueDose: Varies by preparative regimen and stem cell source

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

6-12 months for full immune recovery

Duration

Initial hospitalization 1-2 months, intensive outpatient 6-12 months, lifetime follow-up

Response Rate

Remission Rate

Number Needed to Harm (NNH)

Common Side Effects:

Graft-versus-host disease (GVHD): 50%

Infections (bacterial, viral, fungal): 85%

Mucositis: 75%

Organ toxicity (liver, kidney, lung): 40%

Relapse: 35%

Non-relapse mortality: 20%

Annual Cost of Care

Drug Cost

$50,000

Monitoring

$500,000

Side Effects

$150,000

Total Annual

$700,000

Cost-Effectiveness

MODERATE

QALYs Gained

ICER

$120,000/QALY

Cost per Remission

$1,400,000

Treatment Outcomes

Primary Outcomes

Overall Survival (OS) at 2 Years2-year OS (high-risk AML, without allo-HSCT): 30%

+83.3% (+25 percentage points)

Relapse-Free Survival (RFS) at 2 Years2-year RFS (high-risk AML, without allo-HSCT): 20%

+175% (+35 percentage points)

Cumulative Incidence of Relapse (CIR) at 2 Years2-year CIR (high-risk AML, without allo-HSCT): 75%

-53.33% (-40 percentage points)

MRD Negativity Rate Post-TransplantMRD negativity rate pre-transplant (in CR): 40%

+75% (+30 percentage points)

Secondary Benefits

Non-Relapse Mortality (NRM) at 1 Year1-year NRM (historic/higher risk groups): 30%

-33.33% (-10 percentage points)

Quality of Life (QoL) - EORTC QLQ-C30 Global Health StatusEORTC QLQ-C30 Global Health Status score (pre-transplant): 50 points (0-100 scale)

+30% (+15 points)

Cumulative Incidence of Chronic GVHD (cGVHD) at 2 Years2-year cumulative incidence of chronic GVHD (overall): 50%

-20% (-10 percentage points)

Common Side Effects

Graft-versus-host disease (GVHD)

+50%

Infections (bacterial, viral, fungal)

+85%

Mucositis

+75%

Clinical Trial Phases:

Phase 4

Cytarabine + Daunorubicin ("7+3" Induction Chemotherapy)

85% Effectiveness• 98% Confidence• 25% Safety• 22 trials• 50K participants

HIGH EvidenceExcellent ValueDose: Cytarabine 100-200 mg/m² daily for 7 days; Daunorubicin 45-60 mg/m² daily for 3 days

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

3-4 weeks

Duration

Induction (7+3 days) then 3-4 consolidation cycles (total 4-6 months)

Response Rate

70%

Remission Rate

70%

Common Side Effects:

Myelosuppression (neutropenia, thrombocytopenia, anemia): 100%

Infection/Febrile neutropenia: 60%

Nausea/Vomiting: 70%

Mucositis: 40%

Diarrhea: 40%

Cardiotoxicity (Daunorubicin): 10%

Alopecia: 85%

Annual Cost of Care

Drug Cost

$3,000

Monitoring

$200,000

Side Effects

$75,000

Total Annual

$278,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

2.5

Cost per Remission

$397,143

Cost per Responder

$397,143

Treatment Outcomes

Primary Outcomes

Bone Marrow BlastsTypical at diagnosis: 60-80% of nucleated cells

-95% (Reduced to <5% of nucleated cells (e.g., from 70% to 2%))

Peripheral Blood BlastsTypical at diagnosis: 30-60% of white blood cells

-100% (Reduced to 0% of white blood cells (e.g., from 45% to 0%))

White Blood Cell CountTypical at diagnosis: >50,000/uL (often 70,000-150,000/uL)

-90% (Reduced from leukocytosis to normal range (<10,000/uL))

Secondary Benefits

ECOG Performance StatusAverage at diagnosis: 2-3 (on a scale of 0=fully active to 5=dead)

-50% (Improved by 1-2 points (e.g., from 2.5 to 1.0))

EORTC QLQ-C30 Global Health Status ScoreAverage at diagnosis: 45-55/100 (lower score indicates worse QoL)

+40% (Increased by 15-25 points (e.g., from 50 to 70))

Hemoglobin LevelTypical at diagnosis: 8.0-9.5 g/dL

+40% (Increased by 3-5 g/dL (e.g., from 9.0 to 13.0 g/dL))

Common Side Effects

Myelosuppression (neutropenia, thrombocytopenia, anemia)

+100%

Infection/Febrile neutropenia

+60%

Nausea/Vomiting

+70%

Clinical Trial Phases:

Phase 3Phase 4

Venetoclax + Azacitidine

80% Effectiveness• 90% Confidence• 35% Safety• 253 trials• 2K participants

HIGH EvidencePoor ValueDose: Venetoclax 400 mg daily; Azacitidine 75 mg/m² daily for 7 days of a 28-day cycle

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

1 month

Duration

Continuous until disease progression or unacceptable toxicity

Response Rate

66.4%

Remission Rate

66.4%

Common Side Effects:

Neutropenia: 65%

Thrombocytopenia: 45%

Anemia: 45%

Nausea: 45%

Diarrhea: 45%

Febrile neutropenia: 35%

Fatigue: 35%

Tumor Lysis Syndrome: 3%

Annual Cost of Care

Drug Cost

$216,000

Monitoring

$75,000

Side Effects

$45,000

Total Annual

$336,000

Cost-Effectiveness

POOR

QALYs Gained

ICER

$331,000/QALY

Cost per Remission

$506,024

Cost per Responder

$506,024

Treatment Outcomes

Primary Outcomes

Bone Marrow Blasts60% of nucleated cells

-95% (-57% (from 60% to 3%))

Peripheral Blood Blasts30% of white blood cells

-100% (-30% (from 30% to 0%))

Absolute Neutrophil Count (ANC)0.2 x 10^9/L

+650% (+1.3 x 10^9/L (from 0.2 to 1.5 x 10^9/L))

Platelet Count30 x 10^9/L

+300% (+90 x 10^9/L (from 30 to 120 x 10^9/L))

Secondary Benefits

Red Blood Cell Transfusion Units per 28 days4 units/28 days

-100% (-4 units/28 days)

Platelet Transfusion Units per 28 days3 units/28 days

-100% (-3 units/28 days)

FACIT-Fatigue Scale Score (0-52, higher=less fatigue)20/52

+75% (+15 points (from 20 to 35))

Common Side Effects

Neutropenia

+65%

Thrombocytopenia

+45%

Anemia

+45%

Clinical Trial Phases:

Phase 3Phase 4

CPX-351 (Liposomal Cytarabine and Daunorubicin)

78% Effectiveness• 90% Confidence• 25% Safety• 43 trials• 1K participants

HIGH EvidencePoor ValueDose: Cytarabine 100 mg/m² and Daunorubicin 44 mg/m² IV on days 1, 3, 5

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

3-4 weeks

Duration

Induction (days 1,3,5) then 2 consolidation cycles (total 3-5 months)

Response Rate

47.7%

Remission Rate

47.7%

Number Needed to Treat (NNT)

Common Side Effects:

Febrile neutropenia: 75%

Hemorrhage: 60%

Nausea: 50%

Diarrhea: 50%

Fatigue: 40%

Edema: 35%

Myelosuppression: 100%

Annual Cost of Care

Drug Cost

$250,000

Monitoring

$150,000

Side Effects

$75,000

Total Annual

$475,000

Cost-Effectiveness

POOR

QALYs Gained

2.5

ICER

$233,000/QALY

Cost per Remission

$995,807

Cost per Responder

$995,807

Treatment Outcomes

Primary Outcomes

Overall Survival5.95 months

+60.67% (+3.61 months)

Complete Remission (CR) or CR with Incomplete Hematologic Recovery (CRi) Rate33.3% of patients

+43.24% (+14.4 percentage points)

Event-Free Survival (EFS)1.39 months

+82.01% (+1.14 months)

Secondary Benefits

Hematopoietic Stem Cell Transplantation (HSCT) Rate25% of patients

+36% (+9 percentage points)

60-Day Mortality Rate21.2% of patients

-35.38% (-7.5 percentage points)

Duration of Complete Remission (CR) or CR with Incomplete Hematologic Recovery (CRi)8.75 months

+17.14% (+1.50 months)

Common Side Effects

Febrile neutropenia

+75%

Hemorrhage

+60%

Nausea

+50%

Clinical Trial Phases:

Phase 3Phase 4

Midostaurin + Chemotherapy

77% Effectiveness• 90% Confidence• 30% Safety• 23 trials• 1.5K participants

HIGH EvidencePoor ValueDose: Midostaurin 50 mg orally twice daily, Days 8-21 of induction/consolidation, then continuously

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

Starts with chemotherapy, effects seen with prolonged remission

Duration

Concurrent with induction/consolidation, then maintenance for up to 12 months

Response Rate

65%

Remission Rate

65%

Number Needed to Treat (NNT)

Common Side Effects:

Nausea/Vomiting: 80%

Diarrhea: 50%

Headache: 25%

Febrile neutropenia: 85%

Annual Cost of Care

Drug Cost

$185,000

Monitoring

$150,000

Side Effects

$75,000

Total Annual

$410,000

Cost-Effectiveness

POOR

QALYs Gained

2.7

ICER

$179,000/QALY

Cost per Remission

$630,769

Cost per Responder

$630,769

Treatment Outcomes

Primary Outcomes

Overall Survival (Median)25.6 months (chemotherapy alone)

+191.8% (+49.1 months)

Event-Free Survival (Median)3.0 months (chemotherapy alone)

+173.3% (+5.2 months)

Complete Remission (CR) Rate51.4% (chemotherapy alone)

+14.6% (+7.5%)

Duration of Complete Remission (Median)8.2 months (chemotherapy alone)

+36.6% (+3.0 months)

Secondary Benefits

60-day Mortality Rate8.6% (chemotherapy alone)

-24.4% (-2.1%)

Complete Remission or Incomplete Recovery (CR+CRi) Rate58.7% (chemotherapy alone)

+12.9% (+7.6%)

Common Side Effects

Nausea/Vomiting

+80%

Diarrhea

+50%

Headache

+25%

Clinical Trial Phases:

Phase 3Phase 4

Gemtuzumab Ozogamicin (GO) + Chemotherapy

76% Effectiveness• 88% Confidence• 30% Safety• 21 trials• 5K participants

HIGH EvidenceGood ValueDose: 3 mg/m² IV on Days 1, 4, 7 of induction (fractionated), or single dose

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

Starts with chemotherapy, effects seen with improved EFS/OS

Duration

Given during induction and/or consolidation

Response Rate

70%

Remission Rate

70%

Number Needed to Treat (NNT)

Common Side Effects:

Myelosuppression: 100%

Febrile neutropenia: 65%

Veno-occlusive disease (VOD)/SOS: 7%

Infusion-related reactions: 25%

Hemorrhage: 45%

Nausea/Vomiting: 45%

Annual Cost of Care

Drug Cost

$65,000

Monitoring

$150,000

Side Effects

$75,000

Total Annual

$290,000

Cost-Effectiveness

GOOD

QALYs Gained

2.7

ICER

$50,000/QALY

Cost per Remission

$414,286

Cost per Responder

$414,286

Treatment Outcomes

Primary Outcomes

Bone Marrow BlastsAverage ~70% at diagnosis

-99% (-69% absolute (e.g., from 70% to 1%))

Overall Survival (Median)18 months (chemotherapy alone)

+33.3% (+6 months)

Event-Free Survival (Median)8 months (chemotherapy alone)

+50% (+4 months)

Complete Remission (CR) + CRi Rate60% (chemotherapy alone)

+16.7% (+10 percentage points (from 60% to 70%))

Secondary Benefits

Minimal Residual Disease (MRD) Negativity Rate (post-consolidation)25% (chemotherapy alone)

+60% (+15 percentage points (from 25% to 40%))

Relapse-Free Survival (Median)10 months (chemotherapy alone)

+60% (+6 months)

Transfusion Independence Rate (at 100 days post-induction)50% (chemotherapy alone)

+30% (+15 percentage points (from 50% to 65%))

Common Side Effects

Myelosuppression

+100%

Febrile neutropenia

+65%

Veno-occlusive disease (VOD)/SOS

+7%

Clinical Trial Phases:

Phase 3Phase 4

Gilteritinib

70% Effectiveness• 85% Confidence• 45% Safety• 58 trials• 700 participants

HIGH EvidencePoor ValueDose: Gilteritinib 120 mg orally once daily

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

1-2 months

Duration

Continuous until disease progression or unacceptable toxicity

Response Rate

34%

Remission Rate

34%

Number Needed to Treat (NNT)

Common Side Effects:

Transaminitis: 45%

Myalgia/arthralgia: 35%

Fatigue: 35%

Diarrhea/Constipation: 35%

Febrile neutropenia: 35%

QT prolongation: 7%

Annual Cost of Care

Drug Cost

$330,000

Monitoring

$50,000

Side Effects

$25,000

Total Annual

$405,000

Cost-Effectiveness

POOR

QALYs Gained

0.7

ICER

$629,000/QALY

Cost per Remission

$1,191,176

Cost per Responder

$1,191,176

Treatment Outcomes

Primary Outcomes

Overall Survival (OS)5.6 months

+66.1% (+3.7 months)

Composite Complete Remission (CRc) Rate15.3% of patients

+122.2% (+18.7 percentage points)

Duration of Remission (DoR) in CR/CRh Patients1.3 months

+253.8% (+3.3 months)

Hematopoietic Stem Cell Transplantation (HSCT) Rate15.3% of patients

+69.9% (+10.7 percentage points)

Secondary Benefits

Measurable Residual Disease (MRD) Negativity Rate in CR/CRh Patients12.3% of CR/CRh patients

+158.5% (+19.5 percentage points)

Red Blood Cell Transfusion Independence Rate14.5% of patients

+114.5% (+16.6 percentage points)

Platelet Transfusion Independence Rate14.8% of patients

+155.4% (+23.0 percentage points)

Common Side Effects

Transaminitis

+45%

Myalgia/arthralgia

+35%

Fatigue

+35%

Clinical Trial Phases:

Phase 3Phase 4