Hydroxyurea
Treatment for Thalassemias
Typical Dosage: 10-20 mg/kg/day, titrated
Effectiveness
55%
Safety Score
60%
Clinical Trials
33
Participants
10K
Comparative Safety Scale(Higher is safer)
Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
60
DangerousModerateSafe
Treatment Details
Dosage Range
10-20 mg/kg/day, titrated
Time to Effect
3-6 months
Treatment Duration
lifetime
Evidence Quality
MODERATEConfidence Score
80%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$300
Monitoring:$1,000
Side Effect Mgmt:$200
Total Annual:$1,500
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
EXCELLENTICER
$15,000/QALY
QALYs Gained
1
Outcome-Based Costs
Cost per Responder
$3,333
Prescription Access Economics
Risk Assessment
Requires Monitoring
YesNeeds lab work/checkups
Hydroxyurea Outcomes
for Thalassemias
Efficacy Outcomes
Overall Effectiveness
+55%
Response Rate
+45%
Common Side Effects
Myelosuppression (neutropenia, thrombocytopenia)
+25%
GI disturbances (nausea, vomiting, diarrhea)
+15%
Skin hyperpigmentation, nail changes
+10%
Hair thinning
+7%
Active Clinical Trials
4 active trials recruiting for Hydroxyurea in Thalassemias
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia
NCT07673302RECRUITINGNA
240 participants
INTERVENTIONAL
Rawalpindi, Pakistan
Started: Jun 20, 2026
Long Term Beta Thalassemia Treatment: Findings From The Extension Period
NCT06490601ACTIVE NOT RECRUITINGPHASE2
30 participants
INTERVENTIONAL
Karachi, Pakistan
Started: Apr 1, 2022
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
NCT07177300RECRUITINGPHASE4
50 participants
INTERVENTIONAL
Cincinnati, United States
Started: Dec 19, 2024
Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia
NCT05357482ACTIVE NOT RECRUITINGPHASE1, PHASE2
40 participants
INTERVENTIONAL
Bethesda, United States
Started: May 12, 2022
Completed Clinical Trials
13 completed trials for Hydroxyurea in Thalassemias
Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major
NCT00336362COMPLETEDNA
26 participants
INTERVENTIONAL
Thessaloniki, Greece
Started: Jul 1, 2006
Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
NCT06239389COMPLETEDPHASE2
24 participants
INTERVENTIONAL
Karachi, Pakistan
Started: Sep 1, 2021
Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia
NCT00809042COMPLETEDPHASE2
120 participants
INTERVENTIONAL
Shiraz, Iran
Started: Jun 1, 2007
Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients
NCT06153784COMPLETEDPHASE2, PHASE3
603 participants
INTERVENTIONAL
Karachi, Pakistan
Started: Jul 7, 2020
Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)
NCT00001958COMPLETEDPHASE2
100 participants
INTERVENTIONAL
Bethesda, United States
Started: Dec 1, 1999
Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia
NCT03651102COMPLETEDPHASE2, PHASE3
654 participants
INTERVENTIONAL
Peshawar, Pakistan
Started: Jan 1, 2018
Efficacy of Combination of Hdroxyurea and Thalidomide Over Hydroxyurea or Thalidomide in the Treatment of Transfusion Dependent Thalassemia in Children
NCT06299670COMPLETEDPHASE4
90 participants
INTERVENTIONAL
Dhaka, Bangladesh
Started: Nov 1, 2023
Clinical Experience of Thalidomide in Thalassemic Patients
NCT05132270COMPLETEDPHASE2, PHASE3
135 participants
INTERVENTIONAL
Karachi, Pakistan
Started: Jan 1, 2020
Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia
NCT03715972COMPLETED
165 participants
OBSERVATIONAL
Los Angeles, United States
Started: Jul 15, 2018
Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia
NCT07292259COMPLETEDPHASE2
100 participants
INTERVENTIONAL
Islamabad, Pakistan +8 more
Started: Jan 1, 2024
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
NCT06606886COMPLETEDPHASE4
96 participants
INTERVENTIONAL
Karachi, Pakistan
Started: Sep 16, 2024
Pharmacokinetics of Oral Hydroxyurea Solution
NCT03763656COMPLETEDPHASE1, PHASE2
33 participants
INTERVENTIONAL
Kingston, Jamaica +5 more
Started: Jan 3, 2019
Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia
NCT04292314COMPLETEDPHASE2, PHASE3
350 participants
INTERVENTIONAL
Banī Suwayf, Egypt +3 more
Started: Nov 1, 2019
Showing 20 of 35 total trials