Condition

Acute lymphoid leukemia

Clinical trials and treatment information for Acute lymphoid leukemia

387K

People Affected

Active Trials

135K

New Cases/Year

71K

Deaths/Year

Treatment Rankings

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

Tisagenlecleucel (CAR T-cell Therapy)

92% Effectiveness• 90% Confidence• 25% Safety• 6 trials• 2K participants

MODERATE EvidencePoor ValueDose: 0.2 to 5 x 10^6 CAR-positive viable T cells/kg (for patients up to 50 kg) or 1 to 2.5 x 10^8 CAR-positive viable T cells (for patients over 50 kg)

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Comparative Safety Scale(Higher is safer)

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DangerousModerateSafe

Time to Effect

2-4 weeks

Duration

Single infusion, followed by long-term monitoring

Response Rate

82%

Remission Rate

82%

Common Side Effects:

Cytokine Release Syndrome (CRS): 77%

Neurologic toxicities (ICANS): 47%

B-cell aplasia: 90%

Infection: 43%

Fever: 85%

Annual Cost of Care

Drug Cost

$475,000

Monitoring

$150,000

Side Effects

$250,000

Total Annual

$875,000

Cost-Effectiveness

POOR

QALYs Gained

3.5

ICER

$600,000/QALY

Cost per Remission

$1,067,073

Cost per Responder

$1,067,073

Treatment Outcomes

Primary Outcomes

Complete Remission (CR/CRi) RateExpected CR/CRi rate for relapsed/refractory B-cell ALL with standard salvage therapy: ~25%

+228% (Increase in CR/CRi rate by 57 percentage points (from ~25% to 82%))

Minimal Residual Disease (MRD) Negativity Rate (among CR/CRi patients)Expected MRD negativity rate among CR/CRi relapsed/refractory B-cell ALL patients with prior therapies: ~10%

+730% (Increase in MRD negativity rate by 73 percentage points (from ~10% to 83%))

12-Month Event-Free Survival (EFS) RateExpected 12-month EFS rate for relapsed/refractory B-cell ALL with standard salvage therapy: ~10%

+400% (Increase in 12-month EFS rate by 40 percentage points (from ~10% to 50%))

Secondary Benefits

24-Month Overall Survival (OS) RateExpected 24-month OS rate for relapsed/refractory B-cell ALL with standard salvage therapy: ~15%

+186.67% (Increase in 24-month OS rate by 28 percentage points (from ~15% to 43%))

Karnofsky Performance Status (KPS)KPS: 70 (indicating able to care for self but unable to do normal activity or work, pre-treatment)

+28.57% (+20 points on KPS (from 70 to 90, indicating normal activity with some effort, minor signs of disease))

Patient-Reported Quality of Life (PedsQL Total Score)PedsQL Total Score: 50 (on a 0-100 scale, lower scores indicate worse QoL, pre-treatment)

+40% (+20 points on PedsQL Total Score (from 50 to 70))

Common Side Effects

Cytokine Release Syndrome (CRS)

+77%

Neurologic toxicities (ICANS)

+47%

B-cell aplasia

+90%

Clinical Trial Phases:

Phase 2Phase 3

Blinatumomab

85% Effectiveness• 88% Confidence• 40% Safety• 127 trials• 7K participants

HIGH EvidencePoor ValueDose: 9 mcg/day for week 1, then 28 mcg/day continuous IV infusion

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Comparative Safety Scale(Higher is safer)

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DangerousModerateSafe

Time to Effect

2-4 weeks

Duration

Continuous IV infusion for 28-day cycles (2-4 cycles)

Response Rate

60%

Remission Rate

45%

Common Side Effects:

Cytokine Release Syndrome (CRS): 10%

Neurologic toxicities: 15%

Fever: 40%

Headache: 25%

Neutropenia: 20%

Annual Cost of Care

Drug Cost

$200,000

Monitoring

$30,000

Side Effects

$40,000

Total Annual

$270,000

Cost-Effectiveness

POOR

QALYs Gained

1.2

ICER

$200,000/QALY

Cost per Remission

$600,000

Cost per Responder

$450,000

Treatment Outcomes

Primary Outcomes

Bone Marrow BlastsMedian 75% blasts (relapsed/refractory ALL)

-95% (-71.25 percentage points (to 3.75% from 75%))

Minimal Residual Disease (MRD) LevelMedian 0.1% blast equivalent (for MRD-positive patients)

-99.9% (-0.0999% blast equivalent (achieving <0.001%))

Complete Remission (CR/CRi) Rate0% (for patients not in remission)

+43% (+43 percentage points achievement of CR/CRi)

Secondary Benefits

Median Overall Survival4.0 months (historical control for R/R ALL)

+90% (+3.6 months)

Median Event-Free Survival2.0 months (historical control for R/R ALL)

+195% (+3.9 months)

Health-Related Quality of Life (FACT-Leu)FACT-Leu score: 70/136 (moderate impairment)

+10% (+7 points)

Common Side Effects

Cytokine Release Syndrome (CRS)

+10%

Neurologic toxicities

+15%

Fever

+40%

Clinical Trial Phases:

Phase 2Phase 3

Inotuzumab Ozogamicin

80% Effectiveness• 85% Confidence• 35% Safety• 50 trials• 5K participants

HIGH EvidencePoor ValueDose: 0.8 mg/m² (cycle 1), then 0.5 mg/m² (subsequent cycles) on days 1, 8, 15

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Comparative Safety Scale(Higher is safer)

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DangerousModerateSafe

Time to Effect

3-6 weeks

Duration

IV infusion for 21-28 day cycles (2-4 cycles)

Response Rate

80%

Remission Rate

60%

Common Side Effects:

Veno-occlusive disease (VOD)/Sinusoidal obstruction syndrome (SOS): 11%

Hepatotoxicity: 15%

Myelosuppression: 50%

Fever: 30%

Nausea: 25%

Annual Cost of Care

Drug Cost

$150,000

Monitoring

$25,000

Side Effects

$35,000

Total Annual

$210,000

Cost-Effectiveness

POOR

QALYs Gained

ICER

$300,000/QALY

Cost per Remission

$350,000

Cost per Responder

$262,500

Treatment Outcomes

Primary Outcomes

Maximum Tolerated Dose (MTD) of Bosutinib All Phase I Participants

Number of Participants With a Major Hematologic Response

Number of Participants Who Received First Line Chemotherapy According to National Trial or Treatment Guideline27 Participants

-96% (-26.0 Participants)

Number of Participants According to Number of Lines of Salvage Therapy6 Participants

-83% (-5.0 Participants)

Number of Participants According to Prior Hematopoietic Stem Cell Transplant (HSCT)10 Participants

+50% (+5.0 Participants)

Number of Participants According to Type of Conditioning Regimen for Each HSCT7 Participants

-86% (-6.0 Participants)

Number of Participants Who Were Treated Previously With Blinatumomab4 Participants

+500% (+20.0 Participants)

Number of Participants Treated With Chimeric Antigen Receptor (CAR) T-Cell Therapies

MTD of Inotuzumab Ozogamicin With CVP for Patients With Relapsed or Refractory CD22+ Acute Leukemia

Secondary Benefits

Duration of Response

Overall Survival (OS)

Total Duration of Treatment With Inotuzumab Ozogamicin

Common Side Effects

Veno-occlusive disease (VOD)/Sinusoidal obstruction syndrome (SOS)

+11%

Hepatotoxicity

+15%

Myelosuppression

+50%

Clinical Trial Phases:

Phase 2Phase 3

Multi-agent Chemotherapy (e.g., Hyper-CVAD regimen)

75% Effectiveness• 95% Confidence• 25% Safety• 1 trials• 100K participants

HIGH EvidenceModerate ValueDose: Highly protocol-specific, involves multiple drugs (e.g., Cyclophosphamide, Vincristine, Doxorubicin, Dexamethasone, Methotrexate, Cytarabine)

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Comparative Safety Scale(Higher is safer)

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DangerousModerateSafe

Time to Effect

4-8 weeks (for remission)

Duration

2-3 years (induction, consolidation, maintenance)

Response Rate

85%

Remission Rate

75%

Common Side Effects:

Myelosuppression (Neutropenia, Anemia, Thrombocytopenia): 90%

Nausea and vomiting: 70%

Fatigue: 80%

Mucositis: 50%

Infection: 60%

Alopecia: 80%

Annual Cost of Care

Drug Cost

$50,000

Monitoring

$75,000

Side Effects

$100,000

Total Annual

$225,000

Cost-Effectiveness

MODERATE

QALYs Gained

2.5

Cost per Remission

$300,000

Cost per Responder

$264,706

Treatment Outcomes

Primary Outcomes

Bone Marrow Blast Percentage70%

-92.86% (-65% (from 70% to <5%))

Peripheral Blood Blasts50,000 cells/uL

-100% (-50,000 cells/uL (to 0 cells/uL))

Leukocyte Count (WBC)100,000 cells/uL

-99% (-99,000 cells/uL (to 1,000 cells/uL))

Serum Lactate Dehydrogenase (LDH)800 U/L

-87.5% (-700 U/L (to 100 U/L))

Secondary Benefits

ECOG Performance Status2/5

-100% (-2 points (from 2 to 0))

FACIT-Fatigue Scale Score20/52

+80% (+16 points (from 20 to 36))

Hemoglobin Level8.0 g/dL

+50% (+4.0 g/dL (from 8.0 to 12.0 g/dL))

Common Side Effects

Myelosuppression (Neutropenia, Anemia, Thrombocytopenia)

+90%

Nausea and vomiting

+70%

Fatigue

+80%

Clinical Trial Phases:

Phase 3Phase 4

Dasatinib (for Ph+ ALL)

88% Effectiveness• 90% Confidence• 55% Safety• 35 trials• 10K participants

HIGH EvidencePoor ValueDose: 100 mg orally once daily

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DangerousModerateSafe

Time to Effect

4-8 weeks (for molecular response)

Duration

Long-term (years, often combined with chemotherapy)

Response Rate

90%

Remission Rate

85%

Common Side Effects:

Myelosuppression: 70%

Fluid retention (pleural effusion): 20%

Diarrhea: 30%

Headache: 20%

Rash: 15%

Annual Cost of Care

Drug Cost

$150,000

Monitoring

$15,000

Side Effects

$20,000

Total Annual

$185,000

Cost-Effectiveness

POOR

QALYs Gained

1.8

ICER

$180,000/QALY

Cost per Remission

$217,647

Cost per Responder

$205,556

Treatment Outcomes

Primary Outcomes

Number of Participants With Morphological Complete Response Rate

Number of Participants With Complete Measurable Residual Disease (MRD) Response Rate20 Participants

-60% (-12.0 Participants)

Secondary Benefits

Number of Participants With Adverse Events

Overall Survival

Event-free Survival

Common Side Effects

Myelosuppression

+70%

Fluid retention (pleural effusion)

+20%

Diarrhea

+30%

Clinical Trial Phases:

Phase 3Phase 4

Ponatinib (for Ph+ ALL)

83% Effectiveness• 85% Confidence• 45% Safety• 20 trials• 4K participants

MODERATE EvidencePoor ValueDose: 15 mg to 45 mg orally once daily

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Comparative Safety Scale(Higher is safer)

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DangerousModerateSafe

Time to Effect

8-12 weeks (for molecular response)

Duration

Long-term (years, often combined with chemotherapy)

Response Rate

70%

Remission Rate

55%

Common Side Effects:

Arterial occlusive events: 20%

Hypertension: 60%

Myelosuppression: 50%

Pancreatitis: 10%

Hepatotoxicity: 20%

Fluid retention: 40%

Annual Cost of Care

Drug Cost

$200,000

Monitoring

$20,000

Side Effects

$30,000

Total Annual

$250,000

Cost-Effectiveness

POOR

QALYs Gained

1.5

ICER

$250,000/QALY

Cost per Remission

$454,545

Cost per Responder

$357,143

Treatment Outcomes

Primary Outcomes

BCR-ABL Fusion Transcript Level (MRD)10% International Scale (IS)

-99.9% (Reduction by >3 log (to <0.01% IS))

Bone Marrow Blast Percentage50%

-98% (Reduction to <5% (typically <1% for CR) blasts)

Philadelphia Chromosome-Positive Cells95% of metaphases

-100% (Reduction to 0% Ph+ metaphases)

Secondary Benefits

Absolute Neutrophil Count (ANC)0.5 x 10^9/L

+200% (+1.0 x 10^9/L (to >1.5 x 10^9/L))

Platelet Count50 x 10^9/L

+200% (+100 x 10^9/L (to >150 x 10^9/L))

Common Side Effects

Arterial occlusive events

+20%

Hypertension

+60%

Myelosuppression

+50%

Clinical Trial Phases:

Phase 2Phase 3