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Hematopoietic Stem Cell Transplantation (HSCT)
Treatment for Thalassemias
Typical Dosage: Not applicable (procedure)
Effectiveness
85%
Safety Score
35%
Clinical Trials
70
Participants
200K
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, 6-12 months for full immune reconstitution
Treatment Duration
One-time procedure (lifelong follow-up)
Evidence Quality
HIGHConfidence 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
EXCELLENTICER
$11,595/QALY
QALYs Gained
15
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:
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
5 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
10 participants
INTERVENTIONAL
Shatin, Hong Kong
Started: Oct 1, 2021
Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT
NCT07003269ENROLLING BY INVITATION
30 participants
OBSERVATIONAL
Haikou, China
Started: Sep 1, 2024
Observational Study: Romiplostim for Platelet Recovery in Haploidentical HSCT
NCT07003256ENROLLING BY INVITATION
30 participants
OBSERVATIONAL
Haikou, China
Started: Sep 1, 2024
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
NCT03653338RECRUITINGPHASE1, PHASE2
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
9 participants
INTERVENTIONAL
Oakland, United States +7 more
Started: Apr 29, 2022
Completed Clinical Trials
8 completed trials for Hematopoietic Stem Cell Transplantation (HSCT) in Thalassemias
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
NCT00730314COMPLETEDPHASE1, PHASE2
25 participants
INTERVENTIONAL
Los Angeles, United States
Started: Aug 1, 2008
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
NCT04009525COMPLETEDPHASE4
823 participants
INTERVENTIONAL
Nanning, China
Started: Jul 5, 2019
HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
NCT02435901COMPLETEDPHASE1, PHASE2
29 participants
INTERVENTIONAL
New Hyde Park, United States
Started: Dec 1, 2008
Haploidentical Hematopoietic Stem Cell Transplantation
NCT02165007COMPLETEDPHASE1
7 participants
INTERVENTIONAL
Washington D.C., United States
Started: Jan 1, 2015
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
NCT03513328COMPLETEDPHASE1, PHASE2
6 participants
INTERVENTIONAL
Gainesville, United States
Started: Jun 15, 2018
Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
NCT04390971COMPLETEDNA
3 participants
INTERVENTIONAL
Guiyang, China +2 more
Started: Feb 10, 2023
Post Hematopoietic Stem Cell Transplantation
NCT01610297COMPLETEDPHASE4
27 participants
INTERVENTIONAL
Ankara, Turkey (Türkiye) +5 more
Started: Sep 1, 2013
A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen
NCT00427661COMPLETEDNA
8 participants
INTERVENTIONAL
Pittsburgh, United States
Started: Jun 1, 2002
Showing 20 of 71 total trials