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
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
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:

nih.gov
researchgate.net
nih.gov

WARNING: LIMITED TRIALS AVAILABLE

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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
View Study
10 participants
INTERVENTIONAL
Shatin, Hong Kong
Started: Oct 1, 2021

Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT

NCT07003269ENROLLING BY INVITATION
View Study
30 participants
OBSERVATIONAL
Haikou, China
Started: Sep 1, 2024

Observational Study: Romiplostim for Platelet Recovery in Haploidentical HSCT

NCT07003256ENROLLING BY INVITATION
View Study
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
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
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
View Study
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
View Study
823 participants
INTERVENTIONAL
Nanning, China
Started: Jul 5, 2019

HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

NCT02435901COMPLETEDPHASE1, PHASE2
View Study
29 participants
INTERVENTIONAL
New Hyde Park, United States
Started: Dec 1, 2008

Haploidentical Hematopoietic Stem Cell Transplantation

NCT02165007COMPLETEDPHASE1
View Study
7 participants
INTERVENTIONAL
Washington D.C., United States
Started: Jan 1, 2015

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328COMPLETEDPHASE1, PHASE2
View Study
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
View Study
3 participants
INTERVENTIONAL
Guiyang, China +2 more
Started: Feb 10, 2023

Post Hematopoietic Stem Cell Transplantation

NCT01610297COMPLETEDPHASE4
View Study
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
View Study
8 participants
INTERVENTIONAL
Pittsburgh, United States
Started: Jun 1, 2002
Showing 20 of 70 total trials