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Hydroxyurea for Children and Young Adults With Sickle Cell Disease and Pulmonary Hypertension

This study has been terminated.
(Low subject accrual)
Information provided by (Responsible Party):
Robert I. Liem, Ann & Robert H Lurie Children's Hospital of Chicago Identifier:
First received: July 10, 2006
Last updated: March 28, 2013
Last verified: March 2013
The goal of this study is to test the hypothesis that hydroxyurea is effective for the specific treatment of secondary pulmonary hypertension found on screening in children and young adults with sickle cell disease.

Condition Intervention Phase
Sickle Cell Disease
Pulmonary Hypertension
Drug: Hydroxyurea
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Study of Hydroxyurea for the Treatment of Pulmonary Hypertension in Children and Young Adults With Sickle Cell Disease

Resource links provided by NLM:

Further study details as provided by Ann & Robert H Lurie Children's Hospital of Chicago:

Primary Outcome Measures:
  • Tricuspid Regurgitant Jet Velocity [ Time Frame: 6 and 12 months after HU therapy begins ]
    Primary outcome measure was tricuspid regurgitant jet velocity (TRJV) by echocardiogram after 6 and 12 months of hydroxyurea therapy.

Secondary Outcome Measures:
  • Compliance [ Time Frame: Throughout study ]
    Secondary outcome measures included compliance; laboratory measures of therapy-related toxicity; laboratory biomarkers for hemolysis, oxidative stress and endothelial injury; and quality of life measures by Child Health Questionnaire (CHQ).

Enrollment: 6
Study Start Date: July 2006
Study Completion Date: June 2008
Primary Completion Date: June 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Hydroxyurea Drug: Hydroxyurea
20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.

Detailed Description:

Increasing evidence suggests that pulmonary hypertension, defined by an elevated tricuspid regurgitant jet velocity (TRJV) on echocardiogram, is a major cause of morbidity and mortality in adults with sickle cell disease (SCD). However, both the prevalence and optimal treatment of pulmonary hypertension in children and young adults with SCD are unknown.

We hypothesize that short term therapy with hydroxyurea will decrease TRJV in children and young adults with pulmonary hypertension found on screening. Patients eligible for treatment will have had evidence of pulmonary hypertension on at least 2 screening echocardiograms. Baseline laboratory tests will be obtained and other causes of secondary pulmonary hypertension will be excluded prior to initiation of treatment. Patients will be treated with hydroxyurea according to a standard dose escalation schedule for a total of 12 months. A clinic visit will be required every 2 months and standard screening for toxicity will be performed monthly. There will be an interim analysis of the primary outcome at 6 months following therapy.


Ages Eligible for Study:   10 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age between 10 and 25 years old
  • Sickle cell disease with hemoglobin SS, SC or S-B^0 thalassemia confirmed on hemoglobin electrophoresis
  • Tricuspid regurgitant jet velocity (TRJV) equal to or greater than 2.5 m/sec on 2 baseline Doppler echocardiograms at least 3 months apart

Exclusion Criteria:

  • Patients already being treated with hydroxyurea
  • Patients on a chronic transfusion protocol
  • Patients with evidence of hepatic (alanine aminotransferase [ALT] equal to or greater than 2 SD above normal) or renal dysfunction (creatinine [Cr] equal to or greater than 2 SD above normal)
  • Patients who are pregnant
  • Patients with documented causes of severe pulmonary hypertension other than from SCD
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Please refer to this study by its identifier: NCT00350844

United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614-3394
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Principal Investigator: Robert I Liem, MD Ann & Robert H Lurie Children's Hospital of Chicago
  More Information

Responsible Party: Robert I. Liem, MD, Assistant Professor of Pediatics, NU Feinberg School of Medicine, Ann & Robert H Lurie Children's Hospital of Chicago Identifier: NCT00350844     History of Changes
Other Study ID Numbers: 12735
Study First Received: July 10, 2006
Results First Received: February 18, 2013
Last Updated: March 28, 2013

Keywords provided by Ann & Robert H Lurie Children's Hospital of Chicago:
Sickle Cell Disease
Pulmonary Hypertension

Additional relevant MeSH terms:
Hypertension, Pulmonary
Anemia, Sickle Cell
Vascular Diseases
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors processed this record on May 22, 2017