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Hematopoietic Stem Cell Transplantation (HSCT)
Treatment for Sickle cell disorders
Typical Dosage: Conditioning regimens vary (chemotherapy/radiation)
Effectiveness
95%
Safety Score
30%
Clinical Trials
94
Participants
20K
Comparative Safety Scale(Higher is safer)
Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
30
DangerousModerateSafe
Treatment Details
Dosage Range
Conditioning regimens vary (chemotherapy/radiation)
Time to Effect
6-12 months for engraftment and recovery
Treatment Duration
Single procedure, but long recovery and follow-up
Evidence Quality
HIGHNumber Needed to Treat (NNT)
1(Treat 1 patients to see 1 additional successful outcome)
Number Needed to Harm (NNH)
7(Treat 7 patients to see 1 additional serious adverse event)
Confidence Score
95%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$42,500
Monitoring:$10,000
Side Effect Mgmt:$5,000
Total Annual:$57,500
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
GOODICER
$90,000/QALY
QALYs Gained
18
Outcome-Based Costs
Cost per Responder
$63,889
Cost per Remission
$65,341
Comparison vs Standard medical management (e.g., hydroxyurea)
Cost Difference
$-17,500/year
Less expensive
QALY Difference
+12.00 QALYs
Better outcomes
Dominance
DOMINATESBetter + cheaper
Hematopoietic Stem Cell Transplantation (HSCT) Outcomes
for Sickle cell disorders
Efficacy Outcomes
Overall Effectiveness
+95%
Response Rate
+90%
Remission Rate
+88%
Common Side Effects
Graft-versus-host disease (acute/chronic)
+50%
Infection (bacterial, viral, fungal)
+65%
Organ toxicity (renal, hepatic, pulmonary)
+30%
Transplant-related mortality
+10%
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
8 active trials recruiting for Hematopoietic Stem Cell Transplantation (HSCT) in Sickle cell disorders
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
NCT03279094RECRUITINGPHASE1
11 participants
INTERVENTIONAL
Duarte, United States
Started: Feb 2, 2018
Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients
NCT06351462NOT YET RECRUITINGNA
220 participants
INTERVENTIONAL
Started: May 1, 2024
TMLI and Alemtuzumab for Treatment of Sickle Cell Disease
NCT05384756ACTIVE NOT RECRUITINGPHASE1
2 participants
INTERVENTIONAL
Duarte, United States
Started: Jul 13, 2022
Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation
NCT04018937ACTIVE NOT RECRUITINGPHASE2
43 participants
INTERVENTIONAL
Birmingham, United States +14 more
Started: Mar 22, 2019
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
131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)
NCT07015684RECRUITINGPHASE1
24 participants
INTERVENTIONAL
New York, United States
Started: Apr 28, 2025
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease
NCT03077542ACTIVE NOT RECRUITINGPHASE1, PHASE2
57 participants
INTERVENTIONAL
Bethesda, United States
Started: Apr 6, 2017
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
NCT05213572RECRUITING
200 participants
OBSERVATIONAL
Bethesda, United States
Started: Mar 24, 2022
Completed Clinical Trials
7 completed trials for Hematopoietic Stem Cell Transplantation (HSCT) in Sickle cell disorders
Hematopoietic Stem Cell Transplant for Sickle Cell Disease
NCT02065596COMPLETEDPHASE1
4 participants
INTERVENTIONAL
Cleveland, United States
Started: Oct 19, 2015
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
Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)
NCT00745420COMPLETEDPHASE2
30 participants
INTERVENTIONAL
Birmingham, United States +18 more
Started: Aug 1, 2008
Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound
NCT00152113COMPLETEDPHASE1
5 participants
INTERVENTIONAL
Memphis, United States
Started: Apr 1, 2005
Effects of Prebiotics on Gut Microbiome in Patients Undergoing HSCT
NCT04629430COMPLETEDNA
40 participants
INTERVENTIONAL
Charlottesville, United States
Started: Feb 19, 2021
HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
NCT02435901COMPLETEDPHASE1, PHASE2
29 participants
INTERVENTIONAL
New Hyde Park, United States
Started: Dec 1, 2008
Induction of Stable Chimerism for Sickle Cell Anemia
NCT00029393COMPLETEDPHASE2
INTERVENTIONAL
Started: Aug 1, 2001
Showing 20 of 96 total trials