Condition

Sickle cell disorders

Clinical trials and treatment information for Sickle cell disorders

7.8M
People Affected
50
Active Trials
300K
New Cases/Year
34K
Deaths/Year

Treatment Rankings

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

1
Hematopoietic Stem Cell Transplantation (HSCT)
95% Effectiveness• 95% Confidence• 30% Safety• 94 trials• 20K participants
HIGH EvidenceGood ValueDose: Conditioning regimens vary (chemotherapy/radiation)
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Comparative Safety Scale(Higher is safer)

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30
DangerousModerateSafe

Time to Effect

6-12 months for engraftment and recovery

Duration

Single procedure, but long recovery and follow-up

Response Rate

90%

Remission Rate

88%

Number Needed to Treat (NNT)

1

Number Needed to Harm (NNH)

7

Common Side Effects:

Graft-versus-host disease (acute/chronic): 50%
Infection (bacterial, viral, fungal): 65%
Organ toxicity (renal, hepatic, pulmonary): 30%
Transplant-related mortality: 10%

Annual Cost of Care

Drug Cost

$42,500

Monitoring

$10,000

Side Effects

$5,000

Total Annual

$57,500

Cost-Effectiveness

GOOD

QALYs Gained

18

ICER

$90,000/QALY

Cost per Remission

$65,341

Cost per Responder

$63,889

Treatment Outcomes
Primary Outcomes
Annual Vaso-occlusive Crisis Rate4 crises/year
-90% (-3.6 crises/year)
Hemoglobin S Percentage of Total Hemoglobin85%
-94.1% (-80% of total hemoglobin)
Hemoglobin Concentration7.5 g/dL
+73.3% (+5.5 g/dL)
Secondary Benefits
Pediatric Quality of Life (PedsQL) Score50/100 points
+40% (+20 points)
Average Chronic Pain Severity (Visual Analog Scale)6/10 points
-83.3% (-5 points)
Annual Hospitalization Days for Sickle Cell Complications6 days/year
-83.3% (-5 days/year)
Common Side Effects
Graft-versus-host disease (acute/chronic)
+50%
Infection (bacterial, viral, fungal)
+65%
Organ toxicity (renal, hepatic, pulmonary)
+30%

Clinical Trial Phases:

Phase 4
2
Exagamglogene autotemcel (Casgevy)
93% Effectiveness• 90% Confidence• 25% Safety• 7 trials• 150 participants
MODERATE EvidencePoor ValueDose: Single infusion of modified cells after myeloablative conditioning
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Comparative Safety Scale(Higher is safer)

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Meth💀
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Chemo☢️
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25
DangerousModerateSafe

Time to Effect

6-12 months for engraftment and recovery

Duration

Single procedure, but long recovery and follow-up

Response Rate

92%

Remission Rate

90%

Number Needed to Treat (NNT)

1

Number Needed to Harm (NNH)

4

Common Side Effects:

Myelosuppression/Febrile neutropenia (from conditioning): 90%
Infection: 70%
Mucositis: 60%
Veno-occlusive disease: 10%

Annual Cost of Care

Drug Cost

$110,000

Monitoring

$15,000

Side Effects

$5,000

Total Annual

$130,000

Cost-Effectiveness

POOR

QALYs Gained

18

ICER

$4,500,000/QALY

Cost per Remission

$144,444

Cost per Responder

$141,304

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+93%
Response Rate
+92%
Remission Rate
+90%
Common Side Effects
Myelosuppression/Febrile neutropenia (from conditioning)
+90%
Infection
+70%
Mucositis
+60%

Clinical Trial Phases:

Phase 3
3
Chronic Blood Transfusion Therapy
88% Effectiveness• 95% Confidence• 55% Safety• 16 trials• 15K participants
HIGH EvidenceGood ValueDose: Typically 10-20 mL/kg every 3-4 weeks
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Comparative Safety Scale(Higher is safer)

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55
DangerousModerateSafe

Time to Effect

Immediate for anemia correction, within weeks for clinical stabilization

Duration

Lifetime

Response Rate

90%

Remission Rate

%

Number Needed to Treat (NNT)

4

Number Needed to Harm (NNH)

2

Common Side Effects:

Iron overload (requiring chelation): 85%
Alloimmunization: 25%
Delayed hemolytic transfusion reactions: 7%
Immediate transfusion reactions: 2%

Annual Cost of Care

Drug Cost

$50,000

Monitoring

$5,000

Side Effects

$3,000

Total Annual

$58,000

Cost-Effectiveness

GOOD

QALYs Gained

0.7

ICER

$80,000/QALY

Cost per Responder

$64,444

Treatment Outcomes
Primary Outcomes
Transcranial Doppler (TCD) Velocity200 cm/s
-25% (-50 cm/s)
Annualized Rate of Vaso-occlusive Crises3.5 crises/year
-70% (-2.45 crises/year)
Hemoglobin S (HbS) Percentage80%
-69% (-55%)
Annualized Rate of Acute Chest Syndrome Episodes0.5 episodes/year
-80% (-0.4 episodes/year)
Secondary Benefits
Quality of Life (PedsQL Sickle Cell Module)PedsQL Sickle Cell Module Score: 60/100
+20% (+12 points)
Hospitalization Days per Year15 days/year
-60% (-9 days/year)
Pain Intensity (Visual Analog Scale)Pain Intensity (VAS): 5/10
-40% (-2 points)
Common Side Effects
Iron overload (requiring chelation)
+85%
Alloimmunization
+25%
Delayed hemolytic transfusion reactions
+7%

Clinical Trial Phases:

Phase 4
4
Hydroxyurea
85% Effectiveness• 98% Confidence• 60% Safety• 150 trials• 50K participants
HIGH EvidenceExcellent ValueDose: 15-35 mg/kg/day, titrated
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Comparative Safety Scale(Higher is safer)

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60
DangerousModerateSafe

Time to Effect

3-6 months

Duration

Lifetime

Response Rate

70%

Remission Rate

%

Number Needed to Treat (NNT)

2

Number Needed to Harm (NNH)

8

Common Side Effects:

Myelosuppression (neutropenia, anemia, thrombocytopenia): 25%
Gastrointestinal upset (nausea, vomiting, diarrhea): 15%
Skin hyperpigmentation: 12%
Leg ulcers: 3%

Annual Cost of Care

Drug Cost

$300

Monitoring

$750

Side Effects

$350

Total Annual

$1,400

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.3

ICER

$15,000/QALY

Cost per Responder

$2,000

Treatment Outcomes
Primary Outcomes
Vaso-occlusive Crises (VOCs) Frequency5 episodes/year
-60% (-3 episodes/year)
Acute Chest Syndrome Frequency0.8 episodes/year
-50% (-0.4 episodes/year)
Hemoglobin F Percentage8%
+300% (+24% (to 32%))
Hemoglobin Levels8.0 g/dL
+12.5% (+1.0 g/dL)
Secondary Benefits
Hospitalization Days per Year18 days/year
-50% (-9 days/year)
Blood Transfusion Frequency3 units/year
-40% (-1.2 units/year)
Quality of Life (PedsQL SCD Module)60 points (out of 100)
+10% (+6 points)
Common Side Effects
Myelosuppression (neutropenia, anemia, thrombocytopenia)
+25%
Gastrointestinal upset (nausea, vomiting, diarrhea)
+15%
Skin hyperpigmentation
+12%

Clinical Trial Phases:

Phase 3Phase 4
5
Voxelotor
68% Effectiveness• 90% Confidence• 70% Safety• 25 trials• 1.5K participants
MODERATE EvidencePoor ValueDose: 1500 mg daily
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Comparative Safety Scale(Higher is safer)

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70
DangerousModerateSafe

Time to Effect

1-2 weeks for Hb, 3-6 months for VOCs

Duration

Lifetime

Response Rate

35%

Remission Rate

%

Number Needed to Treat (NNT)

7

Common Side Effects:

Headache: 25%
Diarrhea: 20%
Abdominal pain: 15%
Nausea: 10%

Annual Cost of Care

Drug Cost

$125,000

Monitoring

$500

Side Effects

$200

Total Annual

$125,700

Cost-Effectiveness

POOR

QALYs Gained

0.18

ICER

$750,000/QALY

Cost per Responder

$359,143

Treatment Outcomes
Primary Outcomes
Hemoglobin (Hb) concentration8.5 g/dL
+12.9% (+1.1 g/dL)
Indirect Bilirubin2.8 mg/dL
-32.1% (-0.9 mg/dL)
Absolute Reticulocyte Count200 x10^9/L
-25% (-50 x10^9/L)
Secondary Benefits
6-Minute Walk Test (6MWT) Distance350 meters
+11.4% (+40 meters)
FACIT-Fatigue Scale ScoreFACIT-Fatigue score: 30/52
+16.7% (+5 points)
Lactate Dehydrogenase (LDH)500 U/L
-20% (-100 U/L)
Common Side Effects
Headache
+25%
Diarrhea
+20%
Abdominal pain
+15%

Clinical Trial Phases:

Phase 3Phase 4
6
Crizanlizumab
65% Effectiveness• 88% Confidence• 65% Safety• 15 trials• 750 participants
MODERATE EvidencePoor ValueDose: 5 mg/kg IV infusion monthly
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Comparative Safety Scale(Higher is safer)

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65
DangerousModerateSafe

Time to Effect

Within weeks to 3 months

Duration

Lifetime (monthly infusions)

Response Rate

28%

Remission Rate

%

Number Needed to Treat (NNT)

4

Common Side Effects:

Nausea: 15%
Arthralgia: 15%
Back pain: 10%
Fever: 10%
Infusion-related reactions: 5%

Annual Cost of Care

Drug Cost

$100,000

Monitoring

$2,000

Side Effects

$300

Total Annual

$102,300

Cost-Effectiveness

POOR

QALYs Gained

0.1

ICER

$550,000/QALY

Cost per Responder

$365,357

Treatment Outcomes
Primary Outcomes
Annualized Rate of Vaso-occlusive Crises2.98 events/year
-45.3% (-1.35 events/year)
Median Time to First Vaso-occlusive Crisis4.07 months
+77.1% (+3.14 months)
Annualized Rate of Hospitalization for Vaso-occlusive Crises2.01 events/year
-33.8% (-0.68 events/year)
Secondary Benefits
Annualized Rate of VOCs Requiring ED Visit or IV Opioids2.00 events/year
-40% (-0.80 events/year)
Plasma Soluble P-selectin Level150 ng/mL
-75% (-112.5 ng/mL)
Common Side Effects
Nausea
+15%
Arthralgia
+15%
Back pain
+10%

Clinical Trial Phases:

Phase 3Phase 4
7
L-Glutamine (Endari)
55% Effectiveness• 80% Confidence• 85% Safety• 7 trials• 400 participants
MODERATE EvidencePoor ValueDose: 10-30 g daily, based on weight
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Comparative Safety Scale(Higher is safer)

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85
DangerousModerateSafe

Time to Effect

3-6 months

Duration

Lifetime

Response Rate

25%

Remission Rate

%

Number Needed to Treat (NNT)

9

Common Side Effects:

Nausea: 8%
Headache: 7%
Abdominal pain: 5%

Annual Cost of Care

Drug Cost

$35,000

Monitoring

$200

Side Effects

$100

Total Annual

$35,300

Cost-Effectiveness

POOR

QALYs Gained

0.07

ICER

$400,000/QALY

Cost per Responder

$141,200

Treatment Outcomes
Primary Outcomes
Frequency of Sickle Cell Crisis (VOCs)4.0 crises/year
-25% (-1.0 crises/year)
Hospitalizations for Sickle Cell Crisis3.0 hospitalizations/year
-33% (-1.0 hospitalizations/year)
Time to First Sickle Cell Crisis84 days
+27.4% (+23 days)
Secondary Benefits
Cumulative Days Hospitalized for Sickle Cell Crisis11.0 days/year
-41% (-4.5 days/year)
Annual Rate of Acute Chest Syndrome (ACS)0.17 events/year
-52.9% (-0.09 events/year)
Cumulative Days of Sickle Cell Crisis25.0 days/year
-40% (-10.0 days/year)
Common Side Effects
Nausea
+8%
Headache
+7%
Abdominal pain
+5%

Clinical Trial Phases:

Phase 3Phase 4