Condition

Thalassemias

Clinical trials and treatment information for Thalassemias

1.3M
People Affected
50
Active Trials
400K
New Cases/Year
11K
Deaths/Year

Treatment Rankings

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

1
Betibeglogene Autotemcel (Beti-cel / Zynteglo)
90% Effectiveness• 85% Confidence• 35% Safety• 5 trials• 300 participants
HIGH EvidencePoor ValueDose: Not applicable (procedure)
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Comparative Safety Scale(Higher is safer)

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35
DangerousModerateSafe

Time to Effect

2-4 weeks for engraftment, 12-18 months for transfusion independence

Duration

One-time treatment (lifelong follow-up)

Response Rate

%

Remission Rate

85%

Common Side Effects:

Neutropenia (from conditioning chemotherapy): 100%
Febrile neutropenia: 85%
Mucositis: 75%
Nausea/vomiting: 65%
Headache: 45%
Veno-occlusive disease (VOD) of the liver: 7%

Annual Cost of Care

Drug Cost

$2,800,000

Monitoring

$1,500

Side Effects

$0

Total Annual

$2,801,500

Cost-Effectiveness

POOR

QALYs Gained

20

ICER

$1,500,000/QALY

Cost per Remission

$3,294,118

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+90%
Remission Rate
+85%
Common Side Effects
Neutropenia (from conditioning chemotherapy)
+100%
Febrile neutropenia
+85%
Mucositis
+75%

Clinical Trial Phases:

Phase 3
2
Hematopoietic Stem Cell Transplantation (HSCT)
85% Effectiveness• 95% Confidence• 35% Safety• 70 trials• 200K participants
HIGH EvidenceExcellent ValueDose: Not applicable (procedure)
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Comparative Safety Scale(Higher is safer)

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35
DangerousModerateSafe

Time to Effect

2-4 weeks for engraftment, 6-12 months for full immune reconstitution

Duration

One-time procedure (lifelong follow-up)

Response Rate

%

Remission Rate

85%

Common Side Effects:

Mucositis: 95%
Infection (bacterial, viral, fungal): 90%
Acute Graft-versus-host disease (GVHD): 50%
Chronic Graft-versus-host disease (GVHD): 30%
Veno-occlusive disease (VOD) of the liver: 10%
Infertility: 70%

Annual Cost of Care

Drug Cost

$0

Monitoring

$1,500

Side Effects

$0

Total Annual

$1,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

15

ICER

$11,595/QALY

Cost per Remission

$411,765

Treatment Outcomes
Primary Outcomes
Transfusion Independence20 units/year (chronic transfusions)
-100% (-20 units/year)
Hemoglobin Level (without transfusions)6.5 g/dL (pre-transfusion average)
+69.23% (+4.5 g/dL)
Serum Ferritin3500 ng/mL
-71.43% (-2500 ng/mL)
Secondary Benefits
Quality of Life (PedsQL Generic Core Scales)65/100
+23.08% (+15 points)
Growth Velocity3 cm/year (below normal for age)
+66.67% (+2 cm/year)
Iron Chelation Therapy Burden (Doses/Week)7 doses/week
-100% (-7 doses/week)
Common Side Effects
Mucositis
+95%
Infection (bacterial, viral, fungal)
+90%
Acute Graft-versus-host disease (GVHD)
+50%

Clinical Trial Phases:

Phase 4
3
Red Blood Cell Transfusions
90% Effectiveness• 100% Confidence• 60% Safety• 24 trials• 1M participants
HIGH EvidenceExcellent ValueDose: 10-15 mL/kg packed red blood cells every 2-4 weeks
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Comparative Safety Scale(Higher is safer)

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60
DangerousModerateSafe

Time to Effect

immediate

Duration

lifetime

Response Rate

100%

Remission Rate

0%

Common Side Effects:

Iron overload: 100%
Febrile non-hemolytic transfusion reaction: 2%
Allergic reaction: 3%
Alloimmunization: 15%
Transfusion-transmitted infections: 0.00005%

Annual Cost of Care

Drug Cost

$20,000

Monitoring

$3,000

Side Effects

$2,000

Total Annual

$25,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

10

Cost per Responder

$25,000

Treatment Outcomes
Primary Outcomes
Hemoglobin (Hb) levels6.5 g/dL
+46.15% (+3.0 g/dL)
Fatigue Severity Scale (FSS) score5.5/7
-45.45% (-2.5 points)
Spleen size below costal margin8 cm below costal margin
-50% (-4 cm below costal margin)
Annual Height Growth Velocity (children)2 cm/year
+150% (+3 cm/year)
Secondary Benefits
Pediatric Quality of Life Inventory (PedsQL) Score55/100
+27.27% (+15 points)
6-Minute Walk Test (6MWT) Distance300 meters
+16.67% (+50 meters)
Reticulocyte Count10%
-70% (-7%)
Common Side Effects
Iron overload
+100%
Febrile non-hemolytic transfusion reaction
+2%
Allergic reaction
+3%

Clinical Trial Phases:

Phase 4
4
Deferasirox
80% Effectiveness• 95% Confidence• 65% Safety• 49 trials• 25K participants
HIGH EvidenceModerate ValueDose: 7-30 mg/kg once daily
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Comparative Safety Scale(Higher is safer)

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65
DangerousModerateSafe

Time to Effect

months to years for clinical benefits

Duration

lifetime

Response Rate

85%

Remission Rate

0%

Common Side Effects:

GI disturbances (nausea, vomiting, diarrhea, abdominal pain): 15%
Skin rash: 7%
Increased serum creatinine: 3%
Increased liver transaminases: 3%
Headache: 5%

Annual Cost of Care

Drug Cost

$10,000

Monitoring

$4,000

Side Effects

$1,000

Total Annual

$15,000

Cost-Effectiveness

MODERATE

QALYs Gained

7

ICER

$112,000/QALY

Cost per Responder

$17,647

Treatment Outcomes
Primary Outcomes
Liver Iron Concentration (LIC)15 mg Fe/g dry weight
-33.3% (-5 mg Fe/g dry weight)
Serum Ferritin3000 ng/mL
-25% (-750 ng/mL)
Cardiac T2* MRI10 ms (impaired)
+25% (+2.5 ms)
Secondary Benefits
Pancreatic T2* MRI12 ms (impaired)
+16.7% (+2 ms)
Alanine Aminotransferase (ALT)70 U/L (elevated)
-21.4% (-15 U/L)
Quality of Life (FACIT-Fatigue Score)25/52 (lower indicates more fatigue)
+12% (+3 points)
Common Side Effects
GI disturbances (nausea, vomiting, diarrhea, abdominal pain)
+15%
Skin rash
+7%
Increased serum creatinine
+3%

Clinical Trial Phases:

Phase 4
5
Luspatercept (Reblozyl)
70% Effectiveness• 90% Confidence• 60% Safety• 4 trials• 700 participants
HIGH EvidencePoor ValueDose: 1.0 mg/kg subcutaneous every 3 weeks
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Comparative Safety Scale(Higher is safer)

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60
DangerousModerateSafe

Time to Effect

weeks to months

Duration

lifetime

Response Rate

21.4%

Remission Rate

0%

Common Side Effects:

Headache: 25%
Bone pain: 25%
Arthralgia: 15%
Fatigue: 15%
Hypertension: 15%
Thromboembolic events (DVT, PE, stroke): 4%

Annual Cost of Care

Drug Cost

$150,000

Monitoring

$2,500

Side Effects

$1,500

Total Annual

$154,000

Cost-Effectiveness

POOR

QALYs Gained

0.1

ICER

$600,000/QALY

Cost per Responder

$719,626

Treatment Outcomes
Primary Outcomes
Red Blood Cell Transfusion Burden (units per 24 weeks)15.0 units per 24 weeks
-21.3% (-3.2 units per 24 weeks)
Length of Transfusion-Free Interval2.0 weeks
+50% (+1.0 week)
Mean Hemoglobin Level Between Transfusions8.5 g/dL
+11.8% (+1.0 g/dL)
Secondary Benefits
FACT-An Fatigue Subscale Score30/52
+16.7% (+5 points)
EQ-5D-5L Health State Index0.65
+12.3% (+0.08)
Serum Ferritin2500 ng/mL
-12% (-300 ng/mL)
Common Side Effects
Headache
+25%
Bone pain
+25%
Arthralgia
+15%

Clinical Trial Phases:

Phase 3Phase 4
6
Hydroxyurea
55% Effectiveness• 80% Confidence• 60% Safety• 33 trials• 10K participants
MODERATE EvidenceExcellent ValueDose: 10-20 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

45%

Remission Rate

0%

Common Side Effects:

Myelosuppression (neutropenia, thrombocytopenia): 25%
GI disturbances (nausea, vomiting, diarrhea): 15%
Skin hyperpigmentation, nail changes: 10%
Hair thinning: 7%

Annual Cost of Care

Drug Cost

$300

Monitoring

$1,000

Side Effects

$200

Total Annual

$1,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

1

ICER

$15,000/QALY

Cost per Responder

$3,333

Treatment Outcomes
Primary Outcomes
Fetal Hemoglobin (HbF)3% of total Hb
+200% (+6% of total Hb)
Total Hemoglobin (Hb)6.5 g/dL
+23.1% (+1.5 g/dL)
Annual Blood Transfusion Requirement10 units/year
-50% (-5 units/year)
Serum Ferritin2500 ng/mL
-20% (-500 ng/mL)
Secondary Benefits
Spleen Length below Costal Margin5 cm
-30% (-1.5 cm)
Health-Related Quality of Life (Generic Score)60 points (out of 100)
+10% (+6 points)
Height-for-Age Z-score-1.5 SD
+33.3% (+0.5 SD)
Common Side Effects
Myelosuppression (neutropenia, thrombocytopenia)
+25%
GI disturbances (nausea, vomiting, diarrhea)
+15%
Skin hyperpigmentation, nail changes
+10%

Clinical Trial Phases:

Phase 4