Validation of a Predictive Score of Acute Chest Syndrome (Presev2)

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.

Study Overview

• Status: Recruiting
• Conditions: Acute Chest Syndrome; Vaso Occlusive Crisis

Detailed Description

International multicentre prospective observational study.

Prediction of an ACS within 15 days after admission by the PRESEV2 score at arrival.

Validation of a Predictive Score of Acute Chest Syndrome (Presev2) associates a categorical pain score of the spine /or pelvis and 3 biological parameters: Reticulocytes, Leucocytes and Hemoglobin.

ACS is defined by crepitant or bronchial breathing or the association of new radiologic infiltrate and chest pain

Inclusion criteria:

• Children (>2 years) and adults
• Male and Female
• Homozygous SCD patients
• The patient can only be included once
• 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

Exclusion criteria:

• 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

STUDY SCHEDULE Screening and inclusion once patients are admitted at the accident and emergency department or medical day unit.

Inclusion visit (day 1) Once admitted at the accident and emergency department, the patient will be informed about the protocol and asked to participate in the study. Informed consent will be obtained according to local regulations by signing the informed consent form. The inclusion and non-inclusion criteria will be verified.

Demographic data, current and previous treatments taken within one month, and medical history will be recorded.

The following parameters of the score will be collected:

Reticulocytes and/or leucocytes counts, urea (mmol/L) and Categorical pain score.

The Score is adjusted with Hydroxyurea treatment (Yes/No) and Hb level (g/dL). Plasmodium falciparum test will be performed only in Africa. Hemoculture if fever (>38°C) is recorded within the 2 first days after admission.

Temperature, Blood pressure, Oxygen saturation, Respiratory rate, Visual analogue score will be recorded.

STUDY DURATION Inclusion period: 2 years Per patient: 15 days Total duration of the study: 2 years

• Study Type: Observational
• Enrollment (Anticipated): 800

Contacts and Locations

• Study Contact: Name: DJOUMAD Sabrina, Master's; Phone Number: 33 (1) 49812441; Email: sabrina.djoumad@aphp.fr
• Study Contact Backup: Name: BERKENOU Jugurtha, Master's; Phone Number: 33 (1) 49814414; Email: jugurtha.berkenou@aphp.fr

Study Locations

• France
  • Creteil, France, 94000
    • Recruiting
    • Henri Mondor Hospital
    • Contact: Pablo BARTOLUCCI, Professor; Phone Number: 33 (1) 49817406; Email: pablo.bartolucci@aphp.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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

Description

Inclusion Criteria:

• Inclusion criteria:
• 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

Exclusion criteria:

• 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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Secondary Outcome Measures: Validation of a Predictive Score of Acute Chest Syndrome	Association of the PRESEV 2 score with the following parameters collected for each patient: The number of days of hospitalization; Blood transfusion requirement; The number of hospitalization in intensive care unit; Death	2 years

Collaborators and Investigators

• Sponsor: Soutien aux Actions contre les Maladies du Globule Rouge
• Collaborators: Pierre Fabre Laboratories; ADDMEDICA SASA
• Investigators: Principal Investigator: BARTOLUCCI Pablo, MD PhD, Pr, APHP, UPEC, INSERM

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2016
• Primary Completion (Anticipated): December 31, 2023
• Study Completion (Anticipated): December 31, 2023

Study Registration Dates

• First Submitted: November 18, 2015
• First Submitted That Met QC Criteria: January 23, 2017
• First Posted (Estimate): January 26, 2017

Study Record Updates

• Last Update Posted (Estimate): December 13, 2022
• Last Update Submitted That Met QC Criteria: December 12, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Keywords: vaso occlusive crisis; sickle cell desease; acute chest syndrome; Validation of predictif score of acute chest syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Respiration Disorders; Lung Diseases; Disease; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell; Syndrome; Acute Chest Syndrome
• Other Study ID Numbers: Presev 2 IRB 00003835

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No