Hematopoietic Stem Cell Transplantation (HSCT)

Treatment for Thalassemias

Typical Dosage: Not applicable (procedure)

Effectiveness

85%

Safety Score

35%

Clinical Trials

Participants

200K

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Treatment Details

Dosage Range

Not applicable (procedure)

Time to Effect

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

Treatment Duration

One-time procedure (lifelong follow-up)

Evidence Quality

HIGH

Confidence Score

95%confidence in effectiveness data

Health Economics

Annual Cost of Care

Drug Cost:$0

Monitoring:$1,500

Side Effect Mgmt:$0

Total Annual:$1,500

Cost-Effectiveness Analysis

Cost-Effectiveness Rating

EXCELLENT

ICER

$11,595/QALY

QALYs Gained

Outcome-Based Costs

Cost per Remission

$411,765

Comparison vs Chronic Transfusions + Chelation

Cost Difference

+$325,000/year

More expensive

QALY Difference

+12.00 QALYs

Better outcomes

Dominance

No dominance

Hematopoietic Stem Cell Transplantation (HSCT) Outcomes

for Thalassemias

Efficacy Outcomes

Overall Effectiveness

+85%

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%

Sources:

nih.gov

researchgate.net

nih.gov

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

4 active trials recruiting for Hematopoietic Stem Cell Transplantation (HSCT) in Thalassemias

In-utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Bart's Hydrops Fetalis Syndrome

NCT05797272RECRUITINGNA

View Study

10 participants

INTERVENTIONAL

Shatin, Hong Kong

Started: Oct 1, 2021

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

NCT03653338RECRUITINGPHASE1, PHASE2

View Study

5 participants

INTERVENTIONAL

Pittsburgh, United States

Started: Aug 2, 2018

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

NCT05444894ACTIVE NOT RECRUITINGPHASE1, PHASE2

View Study

9 participants

INTERVENTIONAL

Oakland, United States +7 more

Started: Apr 29, 2022

Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation

NCT07698080NOT YET RECRUITINGPHASE2