Erythropoietin in Hemolytic Uremic Syndrome

NCT ID: NCT03776851

Last Updated: 2021-01-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-01-01

Study Completion Date

2020-12-30

Brief Summary

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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

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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 after 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.

Conditions

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Hemolytic-Uremic Syndrome Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

NONE

Study Groups

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Erythropoietin

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

Group Type EXPERIMENTAL

erythropoietin

Intervention Type DRUG

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

No Intervention

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

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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erythropoietin

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

Intervention Type DRUG

Other Intervention Names

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EPO

Eligibility Criteria

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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
Minimum Eligible Age

1 Month

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hospital General de Niños Pedro de Elizalde

OTHER

Sponsor Role lead

Responsible Party

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Alejandro Balestracci

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Alejandro Balestracci, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Hospital General de Niños Pedro de Elizalde

Locations

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HGNPE

CABA, , Argentina

Site Status

Countries

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Argentina

References

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Ardissino G, Dacco V, Testa S, Civitillo CF, Tel F, Possenti I, Belingheri M, Castorina P, Bolsa-Ghiringhelli N, Tedeschi S, Paglialonga F, Salardi S, Consonni D, Zoia E, Salice P, Chidini G. Hemoconcentration: a major risk factor for neurological involvement in hemolytic uremic syndrome. Pediatr Nephrol. 2015 Feb;30(2):345-52. doi: 10.1007/s00467-014-2918-0. Epub 2014 Aug 23.

Reference Type BACKGROUND
PMID: 25149851 (View on PubMed)

Scheiring J, Rosales A, Zimmerhackl LB. Clinical practice. Today's understanding of the haemolytic uraemic syndrome. Eur J Pediatr. 2010 Jan;169(1):7-13. doi: 10.1007/s00431-009-1039-4. Epub 2009 Aug 26.

Reference Type BACKGROUND
PMID: 19707787 (View on PubMed)

Exeni R, Donato H, Rendo P, Antonuccio M, Rapetti MC, Grimoldi I, Exeni A, de Galvagni A, Trepacka E, Amore A. Low levels of serum erythropoietin in children with endemic hemolytic uremic syndrome. Pediatr Nephrol. 1998 Apr;12(3):226-30. doi: 10.1007/s004670050443.

Reference Type BACKGROUND
PMID: 9630043 (View on PubMed)

Moore E, Bellomo R. Erythropoietin (EPO) in acute kidney injury. Ann Intensive Care. 2011 Mar 21;1(1):3. doi: 10.1186/2110-5820-1-3.

Reference Type BACKGROUND
PMID: 21906325 (View on PubMed)

Warady BA, Silverstein DM. Management of anemia with erythropoietic-stimulating agents in children with chronic kidney disease. Pediatr Nephrol. 2014 Sep;29(9):1493-505. doi: 10.1007/s00467-013-2557-x. Epub 2013 Sep 5.

Reference Type BACKGROUND
PMID: 24005791 (View on PubMed)

Pape L, Ahlenstiel T, Kreuzer M, Drube J, Froede K, Franke D, Ehrich JH, Haubitz M. Early erythropoietin reduced the need for red blood cell transfusion in childhood hemolytic uremic syndrome: a randomized prospective pilot trial. Pediatr Nephrol. 2009 May;24(5):1061-4. doi: 10.1007/s00467-008-1087-4. Epub 2008 Dec 16.

Reference Type BACKGROUND
PMID: 19085014 (View on PubMed)

Balestracci A, Martin SM, Toledo I, Alvarado C, Wainsztein RE. Early erythropoietin in post-diarrheal hemolytic uremic syndrome: a case-control study. Pediatr Nephrol. 2015 Feb;30(2):339-44. doi: 10.1007/s00467-014-2911-7. Epub 2014 Aug 21.

Reference Type BACKGROUND
PMID: 25138373 (View on PubMed)

Nishiwaki H, Abe Y, Suzuki T, Hasegawa T, Levack WM, Noma H, Ota E. Erythropoiesis-stimulating agents for preventing acute kidney injury. Cochrane Database Syst Rev. 2024 Sep 20;9(9):CD014820. doi: 10.1002/14651858.CD014820.pub2.

Reference Type DERIVED
PMID: 39301879 (View on PubMed)

Balestracci A, Capone MA, Meni Battaglia L, Toledo I, Martin SM, Beaudoin L, Balbaryski J, Gomez L. Erythropoietin in children with hemolytic uremic syndrome: a pilot randomized controlled trial. Pediatr Nephrol. 2022 Oct;37(10):2383-2392. doi: 10.1007/s00467-022-05474-9. Epub 2022 Feb 15.

Reference Type DERIVED
PMID: 35166922 (View on PubMed)

Other Identifiers

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347-HGNPE-2018

Identifier Type: -

Identifier Source: org_study_id

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