DFDA

Luspatercept (Reblozyl)

Treatment for Thalassemias

Typical Dosage: 1.0 mg/kg subcutaneous every 3 weeks

Effectiveness
70%
Safety Score
60%
Clinical Trials
4
Participants
700

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
60
DangerousModerateSafe
Treatment Details
Dosage Range
1.0 mg/kg subcutaneous every 3 weeks
Time to Effect
weeks to months
Treatment Duration
lifetime
Evidence Quality
HIGH
Confidence Score
90%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$150,000
Monitoring:$2,500
Side Effect Mgmt:$1,500
Total Annual:$154,000
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
POOR
ICER
$600,000/QALY
QALYs Gained
0.1
Outcome-Based Costs
Cost per Responder
$719,626
Comparison vs Chronic Transfusions + Chelation
Cost Difference
+$129,000/year
More expensive
QALY Difference
+0.10 QALYs
Better outcomes
Dominance
No dominance
Luspatercept (Reblozyl) Outcomes

for Thalassemias

Efficacy Outcomes
Overall Effectiveness
+70%
Response Rate
+21.4%
Common Side Effects
Headache
+25%
Bone pain
+25%
Arthralgia
+15%
Fatigue
+15%
Hypertension
+15%
Thromboembolic events (DVT, PE, stroke)
+4%

Sources:

nih.gov
nih.gov
researchgate.net

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
3 active trials recruiting for Luspatercept (Reblozyl) in Thalassemias

A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia

NCT06073860RECRUITING
View Study
104 participants
OBSERVATIONAL
Seoul, South Korea +1 more
Started: Mar 25, 2024

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

NCT05664737RECRUITINGPHASE2
View Study
249 participants
INTERVENTIONAL
Halifax, Canada +35 more
Started: Dec 9, 2022

A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India

NCT05891249ACTIVE NOT RECRUITINGPHASE4
View Study
85 participants
INTERVENTIONAL
Ahmedabad, India +8 more
Started: Jun 5, 2023
Completed Clinical Trials
1 completed trial for Luspatercept (Reblozyl) in Thalassemias

Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)

NCT04973280COMPLETED
View Study
187 participants
OBSERVATIONAL
Vienna, Austria +3 more
Started: Jul 26, 2021