Validation of a Predictive Score of Acute Chest Syndrome

NCT03032055 | Unknown DMC

• 1 other identifier: Presev 2 IRB 00003835
• Study Type: observational
• Target: 800
• Locations: 1 country
• Sites: 1

Brief Summary

Vaso-Occlusive Crisis (VOC), the most common manifestation of sickle cell disease (SCD), is the first cause of death, particularly when complicated by an acute chest syndrome (ACS). The PRESEV score could help the physicians to better manage VOC and could be used for future therapeutic trials. This predictive score of secondary ACS has to be validated in a multicenter international study.

Conditions

Vaso Occlusive Crisis; Acute Chest Syndrome

Keywords

sickle cell desease; vaso occlusive crisis; acute chest syndrome; Validation of predictif score of acute chest syndrome

Outcome Measures

Primary Outcomes (1)

• Primary Outcome Measure of Validation of a Predictive Score of Acute Chest Syndrome

  The primary outcome mesure is the occurrence of an ACS defined by crepitant or bronchial breathing or the association of new radiologic infiltrats and chest pain during the first 15 days of hospitalization for Vaso Occlusive Crisis

  2 years

Secondary Outcomes (1)

• Secondary Outcome Measures: Validation of a Predictive Score of Acute Chest Syndrome

  2 years

Study Arms (2)

VOC

Patients who won't develop a secondary Acute chest syndrome during a vaso occlusive crisis within 15 days after admission. Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate and chest pain or decreased breath sounds . A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults. It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries.

2°ACS

Patients who will develop a secondary acute chest syndrome during a vaso occlusive crisis within 15 days after admission. Secondary Acute chest syndrome is defined by a new auscultatory abnormality (crepitation or bronchial breathing) OR the association of a new radiologic infiltrate AND chest pain or decreased breath sounds . A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults.It is a form of sickle cell crisis. Sickle cell anemia - most common in those of African, Hispanic, and Mediterranean origin - leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries.

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Children (\>2 years) and adults * Male and Female * Homozygous SCD patients * VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine and/or pelvis) that required hospitalization and opioids (level 3), and is not attributable to other causes

You may qualify if:

• Children (\>2 years) and adults
• Male and Female
• Homozygous SCD patients
• VOC admitted at the emergency unit (a severe VOC is defined as pain or tenderness affecting at least one part of the body (e.g. limbs, ribs, sternum, head (skull), spine and/or pelvis) that required hospitalization and opioids (level 3), and is not attributable to other causes.
• Patient has health care insurance (in Europe)
• Written consent given after being informed of the purpose, progress and potential risks

You may not qualify if:

• No inaugural Acute Chest Syndrome
• Homeless patients
• Deprived of their liberty by a court or administrative order or under guardianship
• Unable to understand the purpose and conditions of carrying out the study, unable to give consent

Sponsors & Collaborators

• Soutien aux Actions contre les Maladies du Globule Rouge: lead
• Theravia: collaborator
• Pierre Fabre Laboratories: collaborator

Study Sites (1)

Henri Mondor Hospital

Créteil, 94000, France

RECRUITING

Related Publications (1)

• Kassasseya C, Kene S, Besse-Hammer T, Nzouakou R, Magnang H, Telfer P, Arlet JB, de Luna G, Affo L, Dautheville S, Ngo S, Pelinski Y, Mescam C, Djoumad S, Pham Hung d'Alexandry d'Orengiani AL, Mekontso Dessap A, Michel M, Galacteros F, Audureau E, Guindo A, Habibi A, Khellaf M, Diallo D, Bartolucci P. Validation and Application of a Predictive Score of Acute Chest Syndrome. NEJM Evid. 2026 Jan;5(1):EVIDoa2500074. doi: 10.1056/EVIDoa2500074. Epub 2025 Dec 18.

  PMID: 41410575 DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

serum and DNA in Africa

MeSH Terms

Conditions

Vaso-Occlusive Crises; Acute Chest Syndrome

Condition Hierarchy (Ancestors)

Anemia, Sickle Cell; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lung Diseases; Respiratory Tract Diseases; Respiration Disorders

Study Officials

• BARTOLUCCI Pablo, MD PhD, Pr

  APHP, UPEC, INSERM

  PRINCIPAL INVESTIGATOR

Central Study Contacts

DJOUMAD Sabrina, Master's

CONTACT

33 (1) 49812441; sabrina.djoumad@aphp.fr

BERKENOU Jugurtha, Master's

CONTACT

33 (1) 49814414; jugurtha.berkenou@aphp.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2015
• First Posted: January 26, 2017
• Study Start: January 1, 2016
• Primary Completion: December 31, 2023
• Study Completion: December 31, 2023
• Last Updated: December 13, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)