Betibeglogene Autotemcel (Beti-cel / Zynteglo)

Treatment for Thalassemias

Typical Dosage: Not applicable (procedure)

Effectiveness
90%
Safety Score
35%
Clinical Trials
5
Participants
300

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
35
DangerousModerateSafe
Treatment Details
Dosage Range
Not applicable (procedure)
Time to Effect
2-4 weeks for engraftment, 12-18 months for transfusion independence
Treatment Duration
One-time treatment (lifelong follow-up)
Evidence Quality
HIGH
Confidence Score
85%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$2,800,000
Monitoring:$1,500
Side Effect Mgmt:$0
Total Annual:$2,801,500
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
POOR
ICER
$1,500,000/QALY
QALYs Gained
20
Outcome-Based Costs
Cost per Remission
$3,294,118
Comparison vs Chronic Transfusions + Chelation
Cost Difference
+$2,775,000/year
More expensive
QALY Difference
+17.00 QALYs
Better outcomes
Dominance
No dominance
Betibeglogene Autotemcel (Beti-cel / Zynteglo) Outcomes

for Thalassemias

Efficacy Outcomes
Overall Effectiveness
+90%
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%

Sources:

nih.gov
nih.gov
icer.org

WARNING: LIMITED TRIALS AVAILABLE

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Active Clinical Trials
No active trials currently recruiting for this treatment

No active trials found in ClinicalTrials.gov