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)
Cyanideโ ๏ธ
Meth๐
Cigarettes๐ฌ
Chemoโข๏ธ
Alcohol๐บ
Morphine๐
Antibiotics๐
Tylenol๐
Exercise๐
Water๐ง
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%
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
NCT03279094ACTIVE NOT RECRUITINGPHASE1
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
Effects of Prebiotics on Gut Microbiome in Patients Undergoing HSCT
NCT04629430COMPLETEDNA
40 participants
INTERVENTIONAL
Charlottesville, United States
Started: Feb 19, 2021
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
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 95 total trials