Hydroxyurea

Treatment for Sickle Cell Disorders

Typical Dosage: 15-35 mg/kg/day, titrated

Effectiveness
85%
Safety Score
60%
Clinical Trials
150
Participants
50K

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
60
DangerousModerateSafe
Treatment Details
Dosage Range
15-35 mg/kg/day, titrated
Time to Effect
3-6 months
Treatment Duration
Lifetime
Evidence Quality
HIGH
Number Needed to Treat (NNT)
2(Treat 2 patients to see 1 additional successful outcome)
Number Needed to Harm (NNH)
8(Treat 8 patients to see 1 additional serious adverse event)
Confidence Score
98%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$300
Monitoring:$750
Side Effect Mgmt:$350
Total Annual:$1,400
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
EXCELLENT
ICER
$15,000/QALY
QALYs Gained
0.3
Outcome-Based Costs
Cost per Responder
$2,000
Hydroxyurea Outcomes

for Sickle Cell Disorders

Efficacy Outcomes
Overall Effectiveness
+85%
Response Rate
+70%
Common Side Effects
Myelosuppression (neutropenia, anemia, thrombocytopenia)
+25%
Gastrointestinal upset (nausea, vomiting, diarrhea)
+15%
Skin hyperpigmentation
+12%
Leg ulcers
+3%

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
6 active trials recruiting for Hydroxyurea in Sickle Cell Disorders

Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

NCT06578507RECRUITINGPHASE2
View Study
50 participants
INTERVENTIONAL
Créteil, France +5 more
Started: Jan 21, 2025

ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study

NCT04707235ACTIVE NOT RECRUITING
View Study
2.09K participants
OBSERVATIONAL
Amiens, France +86 more
Started: Aug 21, 2020

Therapeutic Response Evaluation and Adherence Trial (TREAT)

NCT02286154ACTIVE NOT RECRUITINGNA
View Study
150 participants
INTERVENTIONAL
Cincinnati, United States
Started: Oct 1, 2014

Realizing Effectiveness Across Continents With Hydroxyurea (REACH)

NCT01966731ACTIVE NOT RECRUITINGPHASE1, PHASE2
View Study
635 participants
INTERVENTIONAL
Luanda, Angola +3 more
Started: Jun 1, 2014

Realizing Effectiveness Across Continents With Hydroxyurea

NCT06171217ACTIVE NOT RECRUITINGPHASE2
View Study
810 participants
INTERVENTIONAL
Luanda, Angola +3 more
Started: Oct 27, 2023

Alternative Dosing And Prevention of Transfusions (ADAPT)

NCT05662098ACTIVE NOT RECRUITINGEARLY_PHASE1
View Study
100 participants
INTERVENTIONAL
Jinja, Uganda
Started: Jun 16, 2022
Completed Clinical Trials
11 completed trials for Hydroxyurea in Sickle Cell Disorders

Hydroxyurea Treatment for Adult Sickle Cell Anemia Patients in Kinshasa

NCT05681598COMPLETEDNA
View Study
166 participants
INTERVENTIONAL
Kinshasa, Democratic Republic of the Congo
Started: Aug 30, 2017

Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.

NCT05470270COMPLETEDPHASE2
View Study
33 participants
INTERVENTIONAL
Créteil, France +2 more
Started: Jul 8, 2022

Risk Clinical Stratification of Sickle Cell Disease in Nigeria, Assessment of Efficacy/Safety of Hydroxyurea Treatment

NCT02149537COMPLETEDPHASE4
View Study
53 participants
INTERVENTIONAL
Ibadan, Nigeria
Started: Dec 1, 2014

Mobile-Directly Observed Therapy on Adherence to Hydroxyurea

NCT02844673COMPLETEDPHASE2
View Study
98 participants
INTERVENTIONAL
Dar es Salaam, Tanzania
Started: Apr 28, 2017

Sickle Cell Disease - Stroke Prevention in Nigeria Trial

NCT01801423COMPLETEDNA
View Study
29 participants
INTERVENTIONAL
Kano, Nigeria
Started: Apr 24, 2013

Pharmacokinetics (PK) of Liquid Hydroxyurea in Pediatric Patients With Sickle Cell Anemia

NCT01506544COMPLETEDPHASE1
View Study
39 participants
INTERVENTIONAL
Birmingham, United States +5 more
Started: Dec 1, 2011

Hydroxyurea and Transfusion

NCT03644953COMPLETEDPHASE2
View Study
14 participants
INTERVENTIONAL
Washington D.C., United States
Started: Oct 30, 2018

Hydroxyurea Optimization Through Precision Study

NCT03789591COMPLETEDPHASE3
View Study
104 participants
INTERVENTIONAL
Phoenix, United States +12 more
Started: Jan 17, 2019

Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)

NCT00890396COMPLETED
View Study
163 participants
OBSERVATIONAL
Birmingham, United States +13 more
Started: Sep 1, 2008

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

NCT02225132COMPLETEDPHASE1, PHASE2
View Study
10 participants
INTERVENTIONAL
Bethesda, United States
Started: Aug 23, 2014

Hydroxyurea in Young Children With Sickle Cell Anemia

NCT00519701COMPLETEDNA
View Study
14 participants
INTERVENTIONAL
Durham, United States
Started: Apr 1, 2002
Showing 20 of 150 total trials