Exagamglogene autotemcel (Casgevy)
Treatment for Sickle Cell Disorders
Typical Dosage: Single infusion of modified cells after myeloablative conditioning
Effectiveness
93%
Safety Score
25%
Clinical Trials
7
Participants
150
Comparative Safety Scale(Higher is safer)
Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
25
DangerousModerateSafe
Treatment Details
Dosage Range
Single infusion of modified cells after myeloablative conditioning
Time to Effect
6-12 months for engraftment and recovery
Treatment Duration
Single procedure, but long recovery and follow-up
Evidence Quality
MODERATENumber Needed to Treat (NNT)
1(Treat 1 patients to see 1 additional successful outcome)
Number Needed to Harm (NNH)
4(Treat 4 patients to see 1 additional serious adverse event)
Confidence Score
90%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$110,000
Monitoring:$15,000
Side Effect Mgmt:$5,000
Total Annual:$130,000
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
POORICER
$4,500,000/QALY
QALYs Gained
18
Outcome-Based Costs
Cost per Responder
$141,304
Cost per Remission
$144,444
Comparison vs Standard medical management (e.g., hydroxyurea)
Cost Difference
+$55,000/year
More expensive
QALY Difference
+13.00 QALYs
Better outcomes
Dominance
No dominance
Exagamglogene autotemcel (Casgevy) Outcomes
for Sickle Cell Disorders
Efficacy Outcomes
Overall Effectiveness
+93%
Response Rate
+92%
Remission Rate
+90%
Common Side Effects
Myelosuppression/Febrile neutropenia (from conditioning)
+90%
Infection
+70%
Mucositis
+60%
Veno-occlusive disease
+10%
Active Clinical Trials
No active trials currently recruiting for this treatment
No active trials found in ClinicalTrials.gov