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Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic Congenital Heart Disease - A Prospective Control Trial

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ClinicalTrials.gov Identifier: NCT02564796
Recruitment Status : Recruiting
First Posted : October 1, 2015
Last Update Posted : July 2, 2019
Sponsor:
Information provided by (Responsible Party):
David Werho, MD, University of California, San Diego

Brief Summary:
Cyanotic congenital cardiac patients require higher hemoglobin concentrations (red blood cell levels) for optimal oxygen delivery to the body. Prophylactic erythropoietin (EPO) and iron can prevent and/or decrease the amount of blood transfusions needed in this population. We seek to investigate if EPO and iron make a clinically significant difference in the number of transfusions given to these patients and the morbidity associated with it.

Condition or disease Intervention/treatment Phase
Cyanotic Congenital Heart Disease Anemia Cyanosis Congenital Heart Disease Drug: Epoetin Alfa and Iron Other: Control Phase 2

Detailed Description:

Congenital heart disease occurs in about 1% of all live births. Cyanotic cardiac lesions in particular are at risk for significant mortality and morbidity because of their reduced ability to provide adequate oxygenation to the body and the brain. Many experts believe that to have adequate oxygen carrying capacity that these infants should ideally have a hemoglobin level greater than 13 g/dL. Many of these patients require blood transfusions prior to surgery to provide adequate oxygenation. The cause for this is likely multifactorial including normal neonatal physiology, frequent lab draws, and co-morbidities. Although rare, the morbidity due to transfusions can be devastating to this population including transmitted infections, transfusion reactions, extra hospitalizations, and antigen sensitization that would complicate heart transplant if needed.

There are centers in the United States that have developed protocols using erythropoietin to minimize blood product transfusions before and after surgery, also referred to as "bloodless surgery". There have been retrospective studies evaluating the success of these protocols, but there are no randomized controlled prospective studies that the investigators have studying the effects of erythropoietin effects in patients with cyanotic heart disease in regards to transfusion prevention.

Congenital cyanotic cardiac patients require higher hemoglobin concentrations for optimal oxygen delivery. Prophylactic erythropoietin can prevent and/or decrease the amount of blood transfusions needed prior to surgery. The researchers seek to investigate if erythropoietin makes a clinically significant difference in the number of transfusions given to these patients and the morbidity associated with it.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic Congenital Heart Disease - A Prospective Randomized Control Trial
Actual Study Start Date : February 2, 2017
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : February 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Placebo Comparator: Control
Group II (non-treatment group): Patients in the treatment group will not receive any extra intervention outside of standard of care. They will receive iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization). They will be followed for 14 weeks.
Other: Control
Patients in the treatment group will not receive any extra intervention outside of standard of care. They will receive iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization). They will be followed for 14 weeks.

Experimental: Epoetin alfa and iron supplements
Group I (treatment group): Patients in the treatment group will receive weekly EPO injections and iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization) They will be followed for 14 weeks.
Drug: Epoetin Alfa and Iron
Patients in the treatment group will receive weekly EPO injections and iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization) They will be followed for 14 weeks.




Primary Outcome Measures :
  1. Number of transfusions needed [ Time Frame: First 4 months of life ]
    Prophylactic erythropoietin can prevent and/or decrease the amount of blood transfusions needed prior to surgery. We seek to investigate if erythropoietin makes a clinically significant difference in the number of transfusions given to these patients and the morbidity associated with it during the period in which the subjects will be active in the study (from baseline to 14 weeks post initial injection). The primary aim will be assessed when all subjects have completed week 14 or discontinue early.


Secondary Outcome Measures :
  1. Oxygen saturation [ Time Frame: First 4 months of life ]
    Often, cyanotic congenital heart defect neonates have prolonged initial hospital stays due to the inability to maintain acceptable oxygen saturations, and transition to adequate oral intake for appropriate weight gain. If the hospital stay is found to be shortened after starting erythropoietin, this may be of clinical and financial significance.

  2. Number of hospitalizations [ Time Frame: First 4 months of life ]
    The number of hospital readmissions (related to failure to thrive or cyanosis) may imply the overall clinical stability of a patient. Because these infants are at high risk for mortality at home, there are multiple reasons why they may be admitted to the hospital including clinically significant anemia which requires blood transfusions, poor weight gain, difficulty feeding, inadequate oxygen saturations, and illnesses. Each admission is stressful to the patient and their families. Having a normal hemoglobin level may have a role in preventing several of these factors, especially regarding failure to thrive or cyanosis.

  3. Weight gain [ Time Frame: First 4 months of life ]
    The secondary outcome of weight gain is appropriate in the setting of infants as this variable has been used to monitor the ability to thrive and meet the body's metabolic demands. It is well established in pediatrics that the neonate and infant should gain 15-30 grams per day for optimal growth. Infants who are cyanotic already have a deficiency in meeting their metabolic demands due to a reduced oxygen carrying capacity. This is further complicated in the instance of anemia. Thus, infants may have an increased ability to optimize weight gain in the setting of normal, stable hemoglobin levels which may be achieved with erythropoietin.

  4. Time to initial discharge [ Time Frame: First 4 months of life ]
    If the hospital stay is found to be shortened after starting erythropoietin, this may be of clinical and financial significance.



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 8 Weeks   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  • Newborns less than 4 weeks old at diagnosis
  • Gestational age >34 weeks
  • Birth weight 2.2-4kg
  • Cyanotic heart disease who have had a surgical shunt or a catheterization intervention that is equivalent to a shunt (patent ductus arteriosus stent, right ventricular outflow tract stent).
  • Baseline hematocrit to be below <40%.
  • Completes at least 1 injection in the study by 8 weeks of age.

Exclusion Criteria

  • Infants diagnosed at greater than 4 weeks of age
  • Gestation <34 weeks
  • Birth weight <2.2 kg or >4kg
  • Hematocrit >40%
  • Newborns with acyanotic heart disease
  • Infants with significant co-morbidities:

    • Renal failure (Creatinine > 2 standard deviations above age adjusted norm)
    • Hepatic failure (elevated AST/ALT levels > 2 standard deviations above age adjusted norm
    • Hemolytic disease
    • Hemoglobinopathies (Sickle-cell disease, Thalassemias)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02564796


Contacts
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Contact: David K Werho, MD 8589665855 dwerho@rchsd.org
Contact: Danica A Griffin, MS 8585761700 ext 227139 dgriffin1@rchsd.org

Locations
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United States, California
Rady Children's Hospital Recruiting
San Diego, California, United States, 92123
Contact: David K Werho, MD    858-966-5855    dwerho@rchsd.org   
Contact: Danica A Griffin, MS    8585761700 ext 227139    dgriffin1@rchsd.org   
Sponsors and Collaborators
University of California, San Diego
Investigators
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Principal Investigator: David K Werho, MD University of California, San Diego/Rady Children's Hospital San Diego

Publications:
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Responsible Party: David Werho, MD, Assistant Clinical Professor, University of California, San Diego
ClinicalTrials.gov Identifier: NCT02564796    
Other Study ID Numbers: 160871
First Posted: October 1, 2015    Key Record Dates
Last Update Posted: July 2, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by David Werho, MD, University of California, San Diego:
Erythropoetin
Cyanotic Heart Disease
Transfusion
Congenital Heart Disease
Additional relevant MeSH terms:
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Heart Diseases
Heart Defects, Congenital
Cyanosis
Cardiovascular Diseases
Cardiovascular Abnormalities
Congenital Abnormalities
Signs and Symptoms
Iron
Epoetin Alfa
Trace Elements
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs
Hematinics