Safety and Efficacy of Multiple EPO Injections in Children With Cerebral Palsy

Safety and Efficacy of Repetitive Erythropoietin Treatment for Cerebral Palsy

This study is a retrospective review on the safety and efficacy of repetitive erythropoietin injection in children with cerebral palsy

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy
• Intervention / Treatment: Drug: recombinant human erythropoietin

Detailed Description

Cerebral palsy is mostly caused by hypoxic-ischemic encephalopathy from preterm birth, involving inflammatory and apoptotic cellular response. Erythropoietin (EPO) has shown neuroprotective effects via activation of anti-inflammatory, angiogenic, and anti-apoptotic pathways. The safety and feasibility of EPO in children were proven in previous clinical trials. In addition, the efficacy of EPO is actively being investigated and some reports have shown potential benefits in cerebral palsy. To confirm the safety and efficacy of multiple injections with expected effect, retrospective review on medical records was performed.

• Study Type: Observational
• Enrollment (Actual): 164

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Children diagnosed with cerebral palsy who had received recombinant human erythropoietin more than once.

Description

Inclusion Criteria:

• confirmed clinical diagnosis of cerebral palsy based on neuromotor findings,
• less than 18 years of age,
• received recombinant human erythropoietin (rhEPO) more than once.

Exclusion Criteria:

• not responding to phone interview or
• no functional assessments available.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
recombinant human erythropoietin group; cerebral palsy patients who had received erythropoietin from January 2013 to November 2016	Drug: recombinant human erythropoietin; Subcutaneous rhEPO injection; Other Names: rhEPO

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events	telephone interview was conducted to collect information of adverse events from all patients.	interview was performed at an average of 1 year after the completion of rhEPO injections

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change of Gross Motor Function Measure	The change of GMFM score before and after rhEPO injections	The results between the baseline evaluation before rhEPO injection and the last evaluation at an average of 1 year after rhEPO injections were compared

Collaborators and Investigators

• Sponsor: Bundang CHA Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2013
• Primary Completion (Actual): November 30, 2016
• Study Completion (Actual): August 1, 2017

Study Registration Dates

• First Submitted: August 18, 2017
• First Submitted That Met QC Criteria: October 2, 2017
• First Posted (Actual): October 6, 2017

Study Record Updates

• Last Update Posted (Actual): October 10, 2017
• Last Update Submitted That Met QC Criteria: October 7, 2017
• Last Verified: October 1, 2017

More Information

Terms related to this study

• Keywords: Erythropoeitin
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Damage, Chronic; Cerebral Palsy; Hematinics; Epoetin Alfa
• Other Study ID Numbers: Multiple EPO

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No