Presev2: Validation of a Predictive Score of Acute Chest Syndrome

Sponsor

Soutien aux Actions contre les Maladies du Globule Rouge (Other)

Overall Status

Unknown status

CT.gov ID

NCT03032055

Collaborator

ADDMEDICA SASA (Industry), Pierre Fabre Laboratories (Industry)

800

Enrollment

Location

Duration (Months)

33.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Vaso Occlusive Crisis Acute Chest Syndrome

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 Design

Study Type:

Observational

Anticipated Enrollment :

800 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Validation of a Predictive Score of Acute Chest Syndrome

Study Start Date :

Jan 1, 2016

Anticipated Primary Completion Date :

Jan 1, 2018

Anticipated Study Completion Date :

Jan 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
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.

Arm

Intervention/Treatment

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.

Outcome Measures

Primary Outcome Measures

Primary Outcome Measure of Validation of a Predictive Score of Acute Chest Syndrome [2 years]
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

Secondary Outcome Measures

Secondary Outcome Measures: Validation of a Predictive Score of Acute Chest Syndrome [2 years]
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

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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