Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic CHD - A Prospective Control Trial

Phase 2TerminatedNCT02564796

University of California, San Diego4 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

Interventions

Epoetin Alfa and IronDRUG

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.

IronDRUG

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.

Eligibility

Sex: ALLMax age: 8 Weeks

Medical Language ↔ Plain English

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)

Outcomes

Primary Outcomes

Number of Transfusions Needed

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.