Condition

Multiple myeloma

Clinical trials and treatment information for Multiple myeloma

394K
People Affected
150
Active Trials
110K
New Cases/Year
116K
Deaths/Year

Treatment Rankings

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

1
Idecabtagene vicleucel (ide-cel)
95% Effectivenessβ€’ 90% Confidenceβ€’ 20% Safetyβ€’ 28 trialsβ€’ 1K participants
MODERATE EvidencePoor ValueDose: Single intravenous infusion of 150-450 Γ— 10^6 CAR-positive T cells
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Comparative Safety Scale(Higher is safer)

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20
DangerousModerateSafe

Time to Effect

1-3 months

Duration

One-time infusion, followed by long-term monitoring

Response Rate

80%

Remission Rate

40%

Common Side Effects:

Cytokine Release Syndrome (CRS): 85%
Immune effector Cell-Associated Neurotoxicity Syndrome (ICANS): 30%
Myelosuppression (prolonged): 85%
Infections: 40%
Hypogammaglobulinemia: 50%

Annual Cost of Care

Drug Cost

$419,500

Monitoring

$50,000

Side Effects

$70,000

Total Annual

$539,500

Cost-Effectiveness

POOR

QALYs Gained

1.25

ICER

$475,000/QALY

Cost per Remission

$1,348,750

Cost per Responder

$674,375

Treatment Outcomes
Primary Outcomes
M-protein (Monoclonal Protein) Level2.5 g/dL
-96% (-2.4 g/dL)
Bone Marrow Plasma Cell Infiltration70%
-99.5% (-69.65%)
Involved Serum Free Light Chain (sFLC)3000 mg/L
-99% (-2970 mg/L)
Secondary Benefits
EQ-5D-5L Index Score0.55
+30% (+0.165)
Pain Intensity (NRS)7/10 (Numeric Rating Scale)
-57% (-4 points)
Fatigue Severity Scale (FSS) Score6.0/7
-33% (-2.0 points)
Common Side Effects
Cytokine Release Syndrome (CRS)
+85%
Immune effector Cell-Associated Neurotoxicity Syndrome (ICANS)
+30%
Myelosuppression (prolonged)
+85%

Clinical Trial Phases:

Phase 2Phase 3
2
Daratumumab
92% Effectivenessβ€’ 93% Confidenceβ€’ 60% Safetyβ€’ 293 trialsβ€’ 15K participants
HIGH EvidencePoor ValueDose: 16 mg/kg IV or SC weekly for first few weeks, then every 2-4 weeks
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Comparative Safety Scale(Higher is safer)

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60
DangerousModerateSafe

Time to Effect

1-2 months

Duration

Years to lifetime

Response Rate

90%

Remission Rate

60%

Common Side Effects:

Infusion Reactions: 40%
Fatigue: 30%
Nausea/Diarrhea: 25%
Neutropenia: 30%
Upper Respiratory Tract Infection: 30%

Annual Cost of Care

Drug Cost

$180,000

Monitoring

$8,000

Side Effects

$10,000

Total Annual

$198,000

Cost-Effectiveness

POOR

QALYs Gained

2.5

ICER

$180,000/QALY

Cost per Remission

$330,000

Cost per Responder

$220,000

Treatment Outcomes
Primary Outcomes
Serum M-protein concentration5.0 g/dL
-75% (-3.75 g/dL)
Bone Marrow Plasma Cell Infiltration60%
-66% (-40 percentage points)
MRD Negativity Rate (10^-5 sensitivity)0% of patients achieving MRD negativity
+35% (+35 percentage points)
Secondary Benefits
Hemoglobin Level9.5 g/dL
+21% (+2.0 g/dL)
Brief Pain Inventory (BPI) Worst Pain Score6/10
-33% (-2 points on a 0-10 scale)
EQ-5D Utility Score0.75
+13% (+0.10 points)
Common Side Effects
Infusion Reactions
+40%
Fatigue
+30%
Nausea/Diarrhea
+25%

Clinical Trial Phases:

Phase 3Phase 4
3
Carfilzomib
90% Effectivenessβ€’ 92% Confidenceβ€’ 48% Safetyβ€’ 219 trialsβ€’ 10K participants
HIGH EvidencePoor ValueDose: 20-56 mg/mΒ² IV, typically twice weekly in 28-day cycles
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Comparative Safety Scale(Higher is safer)

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48
DangerousModerateSafe

Time to Effect

1-2 months

Duration

6-18 months

Response Rate

85%

Remission Rate

50%

Common Side Effects:

Hypertension: 25%
Cardiac Events (heart failure, ischemia): 10%
Thrombocytopenia: 40%
Fatigue: 40%
Renal Toxicity: 15%

Annual Cost of Care

Drug Cost

$200,000

Monitoring

$10,000

Side Effects

$15,000

Total Annual

$225,000

Cost-Effectiveness

POOR

QALYs Gained

2

ICER

$250,000/QALY

Cost per Remission

$450,000

Cost per Responder

$264,706

Treatment Outcomes
Primary Outcomes
M-protein Reduction2.5 g/dL
-60% (-1.5 g/dL)
Serum Free Light Chain (Involved) Reduction150 mg/L
-70% (-105 mg/L)
Bone Marrow Plasma Cell Infiltration60% of marrow cells
-90% (-54 percentage points)
Pain Intensity (Worst Pain) ScoreBPI pain intensity (worst pain) score: 7/10
-42.86% (-3 points)
Secondary Benefits
Hemoglobin Level9.5 g/dL
+15.79% (+1.5 g/dL)
Fatigue ScorePROMIS Fatigue T-score: 60 (higher is worse)
-11.67% (-7 points)
Estimated Glomerular Filtration Rate (eGFR)45 mL/min/1.73mΒ²
+15.56% (+7 mL/min/1.73mΒ²)
Common Side Effects
Hypertension
+25%
Cardiac Events (heart failure, ischemia)
+10%
Thrombocytopenia
+40%

Clinical Trial Phases:

Phase 3Phase 4
4
Autologous Stem Cell Transplantation (ASCT)
90% Effectivenessβ€’ 94% Confidenceβ€’ 25% Safetyβ€’ 144 trialsβ€’ 10K participants
HIGH EvidenceModerate ValueDose: High-dose Melphalan (e.g., 200 mg/mΒ² IV) single dose
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Comparative Safety Scale(Higher is safer)

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25
DangerousModerateSafe

Time to Effect

3-6 months

Duration

Initial phase 1-2 months, then maintenance for years

Response Rate

85%

Remission Rate

60%

Common Side Effects:

Myelosuppression (severe neutropenia, thrombocytopenia): 90%
Mucositis: 80%
Infections (bacterial, viral, fungal): 60%
Fatigue: 80%
Nausea/Vomiting/Diarrhea: 60%

Annual Cost of Care

Drug Cost

$5,000

Monitoring

$120,000

Side Effects

$25,000

Total Annual

$150,000

Cost-Effectiveness

MODERATE

QALYs Gained

3

ICER

$130,000/QALY

Cost per Remission

$250,000

Cost per Responder

$176,471

Treatment Outcomes
Primary Outcomes
Primary Myeloma Endpoint4 Participants
-75% (-3.0 Participants)
Common Side Effects
Myelosuppression (severe neutropenia, thrombocytopenia)
+90%
Mucositis
+80%
Infections (bacterial, viral, fungal)
+60%

Clinical Trial Phases:

Phase 3Phase 4
5
Bortezomib
88% Effectivenessβ€’ 96% Confidenceβ€’ 50% Safetyβ€’ 591 trialsβ€’ 25K participants
HIGH EvidenceModerate ValueDose: 1.3 mg/mΒ² IV or SC on days 1, 4, 8, 11 of a 21-day cycle
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Comparative Safety Scale(Higher is safer)

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50
DangerousModerateSafe

Time to Effect

1-2 months

Duration

6-18 months

Response Rate

80%

Remission Rate

45%

Common Side Effects:

Peripheral Neuropathy: 60%
Thrombocytopenia: 50%
Fatigue: 40%
Nausea/Diarrhea/Constipation: 40%
Herpes Zoster reactivation: 15%

Annual Cost of Care

Drug Cost

$80,000

Monitoring

$7,500

Side Effects

$12,000

Total Annual

$99,500

Cost-Effectiveness

MODERATE

QALYs Gained

1.8

ICER

$120,000/QALY

Cost per Remission

$221,111

Cost per Responder

$124,375

Treatment Outcomes
Primary Outcomes
Serum M-protein3.5 g/dL
-70% (-2.45 g/dL)
Urine M-protein (Bence Jones protein)1.0 g/24hr
-80% (-0.8 g/24hr)
Bone Marrow Plasma Cells60%
-83.3% (-50 percentage points)
Serum Free Light Chain Ratio (Involved/Uninvolved)50.0
-98% (-49.0)
Secondary Benefits
Pain Score (NRS)7/10
-40% (-2.8 points)
ECOG Performance Status2/5
-50% (-1 point)
EORTC QLQ-C30 Global Health Status Score50/100
+20% (+10 points)
Common Side Effects
Peripheral Neuropathy
+60%
Thrombocytopenia
+50%
Fatigue
+40%

Clinical Trial Phases:

Phase 3Phase 4
6
Lenalidomide
85% Effectivenessβ€’ 95% Confidenceβ€’ 55% Safetyβ€’ 599 trialsβ€’ 20K participants
HIGH EvidenceGood ValueDose: 5-25 mg orally daily for 21 days of a 28-day cycle
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Comparative Safety Scale(Higher is safer)

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55
DangerousModerateSafe

Time to Effect

1-3 months

Duration

Years to lifetime

Response Rate

75%

Remission Rate

40%

Common Side Effects:

Myelosuppression (Neutropenia, Thrombocytopenia): 60%
Fatigue: 50%
Diarrhea/Constipation: 40%
Rash: 30%
Thromboembolism (DVT/PE): 15%

Annual Cost of Care

Drug Cost

$120,000

Monitoring

$7,500

Side Effects

$10,000

Total Annual

$137,500

Cost-Effectiveness

GOOD

QALYs Gained

2

ICER

$100,000/QALY

Cost per Remission

$343,750

Cost per Responder

$183,333

Treatment Outcomes
Primary Outcomes
Number of Patients With Combined Complete Response and Very Good Partial Response
0%
Primary Myeloma Endpoint4 Participants
-75% (-3.0 Participants)
Number of Participants With Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, Deaths
0%
Number of Participants With Clinically Relevant Vital Sign Findings
0%
Number of Participants With Worst Toxicity Grade Hematology Laboratory Tests
0%
Number of Participants With Worst Toxicity Grade Renal and Liver Function Laboratory Tests
0%
Number of Participants With Worst Toxicity Grade Chemistry Laboratory Tests
0%
Secondary Benefits
Number of Patients With Adverse Events (AEs)
0%
Number of Patients With Overall Response
0%
Number of Patients With Stringent Complete Response Rate
0%
Common Side Effects
Myelosuppression (Neutropenia, Thrombocytopenia)
+60%
Fatigue
+50%
Diarrhea/Constipation
+40%

Clinical Trial Phases:

Phase 3Phase 4