DFDA

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

You can search for trials, but you probably can't join any because the 1% Treaty hasn't passed yet. Most trials are severely limited by lack of funding and bureaucratic barriers. Help change this!

Active Clinical Trials
No active trials currently recruiting for this treatment

No active trials found in ClinicalTrials.gov