Erythropoietin in Hemolytic Uremic Syndrome

Effect of Erythropoietin on Red Blood Cell Requirement in Children With Hemolytic Uremic Syndrome: a Randomized Controlled Trial

Sponsors

Lead Sponsor: Hospital General de Niños Pedro de Elizalde

Source Hospital General de Niños Pedro de Elizalde
Brief Summary

This study will evaluate the impact of early administration of erythropoietin in the number of red blood cell transfusions in children with Shiga toxin-producing Escherichia coli hemolytic uremic syndrome (STEC-HUS).

Detailed Description

Introduction:

Anemia in STEC-HUS is treated with red blood cell (RBC) transfusions. It can causes hypervolemia, hyperkalemia, exacerbate the thrombotic state of the disease, transmit infectious agents and trigger antigenic sensitization. Anemia is mainly due to hemolysis, but deficit of erythropoietin synthesis (EPO) may aggravate it. Although recombinant human EPO is frequently used in children with STEC-HUS there is no adequate evidence of its benefit. If it is confirmed that EPO reduce the number of RBC transfusions, its administration could diminish the aforementioned risks and also reduce costs.

Objective:

To determine if EPO administration decreases the number of RBC transfusions and; secondarily, to assess if its levels influence on transfusion requirement.

Methodology:

Randomized, open controlled clinical trial. We will include 28 patients (14 per arm) <18 years with STEC-HUS admitted to our hospital. They will be grouped by simple randomization:(1) One to standard of care (RBC transfusions with hemoglobin ≤7 mg / dl and/or hemodynamic instability) and (2) the other to standard of care plus EPO (50 u / kg subcutaneous three times weekly) and RBC transfusions with hemoglobin ≤7 mg / dl). Serum EPO will be measured by ELISA and together with the clinical and laboratory variables, association with RBC transfusions number will be sought. Written informed consent and assent when appropriate, will be requested prior to enter into the study.

Overall Status Recruiting
Start Date January 1, 2019
Completion Date January 1, 2021
Primary Completion Date January 1, 2021
Phase Phase 4
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of RBC transfusions At the end of the 36 month study recruiting period
Secondary Outcome
Measure Time Frame
Erythropoietin levels At the end of the 36 month study recruiting period
Enrollment 28
Condition
Intervention

Intervention Type: Drug

Intervention Name: erythropoietin

Description: erythropoietin 50 International Units (IU) per kilogram three times weekly by subcutaneous route

Arm Group Label: Erythropoietin

Other Name: EPO

Eligibility

Criteria:

Inclusion Criteria:

- Post diarrheal HUS: Prodrome of enteritis followed by microangiopathic hemolytic anemia, thrombocytopenia and signs of renal damage (increased plasma creatinine, proteinuria, and / or hematuria). Proven STEC infection wiil not be required to enter into the study.

Exclusion Criteria:

- Atypical HUS

- HUS associated with systemic diseases (pneumococcal infection, HIV, Systemic lupus erythematosus) or drugs

- Anemia or known kidney disease

- Previously transfused or treated with erythropoietin

- Contraindications to erythropoietin

Gender: All

Minimum Age: 1 Month

Maximum Age: 18 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Alejandro Balestracci, MD, PhD Principal Investigator Hospital General de Niños Pedro de Elizalde
Overall Contact

Last Name: Alejandro Balestracci, MD, PhD

Phone: 541143632200

Phone Ext.: 3038

Email: [email protected]

Location
Facility: Status: Contact: Contact Backup: HGNPE Alejandro Balestracci 541140345402 [email protected]
Location Countries

Argentina

Verification Date

July 2019

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Hospital General de Niños Pedro de Elizalde

Investigator Full Name: Alejandro Balestracci

Investigator Title: Principal Investigator

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Erythropoietin

Type: Experimental

Description: Erythropoietin plus standard of care (RBC transfusions if Hb ≤7 mg/dl and/or hemodynamic instability)

Label: No Intervention

Type: No Intervention

Description: Standard of care: RBC transfusions if Hb ≤7 mg/dl and/or hemodynamic instability

Patient Data Yes
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Supportive Care

Masking: None (Open Label)

Source: ClinicalTrials.gov