Study of Biological Markers in Children With Sickle Cell Disease

NCT04839159 | Unknown

• 1 other identifier: P2012/SCD1
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 3

Brief Summary

Sickle cell disease is associated with significant morbi-mortality hence the interest in an early and targeted care. At present, there is no plasmatic marker able to identify infants at higher risk of developping severe complications later in life. However, recent studies have demonstrated a correlation between certain complications of the disease and biomarkers of the endothelial dysfunction characterizing it. Investigators prospectively followed a cohort of children diagnosed with SCD through the universal neonatal screening using inflammatory and haemostatic plasmatic markers to study their annual evolution. Investigators then will evaluate potential associations between these biological markers and the occurrence of SCD related complications. A secondary objective of this study is to evaluate the repercussions of therapeutic intervention on these markers. .

Conditions

Sickle Cell Disease

Keywords

Sickle cell disease, children, thrombin generation, inflammation, cytokines, adhesion molecules, plasmatic markers, coagulation

Outcome Measures

Primary Outcomes (1)

• Plasmatic levels of IL-6 at 12 months of age

  Measurement of plasmatic levels of IL-6 (fg/mL) by flow cytometric assay

  12 months of age

Secondary Outcomes (96)

• Plasmatic levels of IL-6 at 6 months of age

  6 months of age

• Plasmatic levels of IL-6 at 2 years of age

  2 years of age

• Plasmatic levels of IL-6 at 3 years of age

  3 years of age

• Plasmatic levels of IL-6 at 4 years of age

  4 years of age

• Plasmatic levels of IL-6 before the introduction of any new sickle cell disease treatment as determined by a physician according to the standard of care to which the hospital adheres

  Before the introduction of any new sickle cell disease treatment

Study Arms (1)

SCD Patient

EXPERIMENTAL

Other: Blood sampling

Interventions

Blood sampling OTHER

Blood sampling at the age of 6 and 12 months, 2-3-4 years

SCD Patient

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient aged less than 6 months
• Sickle cell syndrome SS, Sβthal or SC confirmed by hemoglobin electrophoresis
• Subjects legal representatives must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to let participate their child in the study

You may not qualify if:

• Congenital abnormality other than sickle cell disease except for a glucose-6-phosphate-deshydrogenase
• Prematurity
• Initiation of the following therapies before enrollment: chronic transfusion regimen or bone marrow transplantation

Sponsors & Collaborators

• Queen Fabiola Children's University Hospital: lead

Study Sites (3)

CHU Saint Pierre

Brussels, 1000, Belgium

Location

Hôpital Universitaire Des Enfants Reine Fabiola

Brussels, 1020, Belgium

Location

HIS - Site Etterbeek-Ixelles

Brussels, 1050, Belgium

Location

Related Publications (11)

• Ware RE, de Montalembert M, Tshilolo L, Abboud MR. Sickle cell disease. Lancet. 2017 Jul 15;390(10091):311-323. doi: 10.1016/S0140-6736(17)30193-9. Epub 2017 Feb 1.

  PMID: 28159390 BACKGROUND

• Gulbis B, Cotton F, Ferster A, Ketelslegers O, Dresse MF, Ronge-Collard E, Minon JM, Le PQ, Vertongen F. Neonatal haemoglobinopathy screening in Belgium. J Clin Pathol. 2009 Jan;62(1):49-52. doi: 10.1136/jcp.2008.060517.

  PMID: 19103861 BACKGROUND

• Ballas SK, Lieff S, Benjamin LJ, Dampier CD, Heeney MM, Hoppe C, Johnson CS, Rogers ZR, Smith-Whitley K, Wang WC, Telen MJ; Investigators, Comprehensive Sickle Cell Centers. Definitions of the phenotypic manifestations of sickle cell disease. Am J Hematol. 2010 Jan;85(1):6-13. doi: 10.1002/ajh.21550.

  PMID: 19902523 BACKGROUND

• Horan J, Lerner N. Prediction of adverse outcomes in children with sickle cell disease. N Engl J Med. 2000 May 25;342(21):1612-3. doi: 10.1056/NEJM200005253422114. No abstract available.

  PMID: 10841686 BACKGROUND

• Miller ST, Sleeper LA, Pegelow CH, Enos LE, Wang WC, Weiner SJ, Wethers DL, Smith J, Kinney TR. Prediction of adverse outcomes in children with sickle cell disease. N Engl J Med. 2000 Jan 13;342(2):83-9. doi: 10.1056/NEJM200001133420203.

  PMID: 10631276 BACKGROUND

• Quinn CT, Lee NJ, Shull EP, Ahmad N, Rogers ZR, Buchanan GR. Prediction of adverse outcomes in children with sickle cell anemia: a study of the Dallas Newborn Cohort. Blood. 2008 Jan 15;111(2):544-8. doi: 10.1182/blood-2007-07-100719. Epub 2007 Oct 1.

  PMID: 17909076 BACKGROUND

• Gladwin MT, Vichinsky E. Pulmonary complications of sickle cell disease. N Engl J Med. 2008 Nov 20;359(21):2254-65. doi: 10.1056/NEJMra0804411. No abstract available.

  PMID: 19020327 BACKGROUND

• Charrin E, Ofori-Acquah SF, Nader E, Skinner S, Connes P, Pialoux V, Joly P, Martin C. Inflammatory and oxidative stress phenotypes in transgenic sickle cell mice. Blood Cells Mol Dis. 2016 Nov;62:13-21. doi: 10.1016/j.bcmd.2016.10.020. Epub 2016 Oct 28.

  PMID: 27835777 BACKGROUND

• Sarray S, Saleh LR, Lisa Saldanha F, Al-Habboubi HH, Mahdi N, Almawi WY. Serum IL-6, IL-10, and TNFalpha levels in pediatric sickle cell disease patients during vasoocclusive crisis and steady state condition. Cytokine. 2015 Mar;72(1):43-7. doi: 10.1016/j.cyto.2014.11.030. Epub 2015 Jan 5.

  PMID: 25569375 BACKGROUND

• Hoppe C, Klitz W, Noble J, Vigil L, Vichinsky E, Styles L. Distinct HLA associations by stroke subtype in children with sickle cell anemia. Blood. 2003 Apr 1;101(7):2865-9. doi: 10.1182/blood-2002-09-2791. Epub 2002 Nov 27.

  PMID: 12517810 BACKGROUND

• Garrido VT, Proenca-Ferreira R, Dominical VM, Traina F, Bezerra MA, de Mello MR, Colella MP, Araujo AS, Saad ST, Costa FF, Conran N. Elevated plasma levels and platelet-associated expression of the pro-thrombotic and pro-inflammatory protein, TNFSF14 (LIGHT), in sickle cell disease. Br J Haematol. 2012 Sep;158(6):788-97. doi: 10.1111/j.1365-2141.2012.09218.x. Epub 2012 Jul 6.

  PMID: 22775554 BACKGROUND

MeSH Terms

Conditions

Anemia, Sickle Cell; Inflammation; Thrombosis

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Embolism and Thrombosis; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Bushra Zucca, MD

  Queen Fabiola Children's University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 11, 2018
• First Posted: April 9, 2021
• Study Start: May 10, 2012
• Primary Completion: June 30, 2021
• Study Completion: June 30, 2021
• Last Updated: April 9, 2021

Record last verified: 2021-04

Data Sharing

• IPD Sharing: Will not share

Locations

BE (3)