Prognostic Factors of Acute Splenic Sequestration (SSADREPA)

Study of Prognostic Factors of Acute Splenic Sequestration in a Cohort of Sickle Cell Disease (SCD) Children Diagnosed at Birth

Acute splenic sequestration is a frequent and life threatening complication occurring in approximately 10 % of homozygous children. Maximal incidence is between 6 and 18 months.

The investigators formulate the hypothesis that there are clinical, biological and genetic markers predictive of severe complications notably acute splenic sequestration in SCD children. The present research project thus aims at analyzing in a forward-looking way the profile of severity by analysing clinical, biological and genetic characteristics in a multicentric cohort of 60 SCD children

Study Overview

• Status: Completed
• Conditions: Anemia; Drepanocytic
• Intervention / Treatment: Procedure: blood samples and scintigraphy

Detailed Description

A prospective multicentric analysis will be conducted in a cohort of 150 SS or S ß ° children diagnosed at birth, included at 3 -5 months and followed up to the age of 24 months.

Five visits, superimposed to the usual follow-up of SCD children, (Recommendations of the High Authority of Health) at 3 months, 6 months, 12 months, 18 months and 24 months will allow a clinical evaluation and an additional sampling of blood (5 mL) at each visit.

The samples will allow 1.analysis of the red blood cell phenotype (adhesion and deformability) and densities 2. the genetic profile 3.to establish a cell bank, a sera bank and a DNA bank, Spleen function in the cohort will be estimated by spleen scintigraphy, coupled with blood markers (pitted cells, Howell-Jolly bodies counts)

• Study Type: Interventional
• Enrollment (Actual): 58
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Paris, France, 75015
    • Necker Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 months to 6 months (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion criteria :

• Children aged 3 to 6 months
• homozygous (SS) or S beta° sickle cell disease diagnosed by neonatal screening
• With no history of acute splenic sequestration
• Signed parental consent
• Patient covered by national insurance scheme or CMU

Exclusion criteria :

• Children with other SCD genotype
• Children with congenital anatomical asplenia
• Children with previous episode of acute splenic sequestration
• Absence of possible follow-up
• Simultaneous enrolment in another biomedical research

Study Plan

How is the study designed?

Design Details

• Primary Purpose: PREVENTION
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
OTHER: Intervention	Procedure: blood samples and scintigraphy; at 3 months, 6 months, 12 months, 18 months and 24 months will allow a clinical evaluation and an additional sampling of blood of 5 mL at each visit Scintigraphy at 6 months and 18 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Study of prognostic factors of acute splenic sequestration	Complete blood count, reticulocyte count, haemoglobin level, VGM, TGMH, CCMH, hematocrit, % foetal haemoglobin (HbF), red blood cell densities (2 mL); Deformability of red blood cells; Expression study of the cell surface molecule: Phosphatidyl serine, CD 36, READ B-CAM, CD 47, ICAM 4, VLA 4. (Cellulotheque = red blood cells frozen in cryopreservative conditions); Total LDH, Bilirubine (stigmas of hemolysis); Genetic Profil ( DNAtheque)	after three years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Study of hematology parameters	Percentage of Howell Jolly's bodies (0,1 mL); Percentage of pitted cells (0,5 mL fixed in 4 %)formaldehyde; Splenic volume, semi-quantitative measure in scintigraphy; Antibody titers in answer to the antipneumococcic vaccination ( serotheque)	after three years

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Investigators: Principal Investigator: Valentine Brousse, MD, PhD, Assistance Publique - Hôpitaux de Paris

Publications and helpful links

General Publications

• Brousse V, El Hoss S, Bouazza N, Arnaud C, Bernaudin F, Pellegrino B, Guitton C, Odievre-Montanie MH, Mames D, Brouzes C, Picard V, Nguyen-Khoa T, Pereira C, Lapoumeroulie C, Pissard S, Gardner K, Menzel S, Le Van Kim C, Colin-Aronovicz Y, Buffet P, Mohandas N, Elie C, Maier-Redelsperger M, El Nemer W, de Montalembert M. Prognostic factors of disease severity in infants with sickle cell anemia: A comprehensive longitudinal cohort study. Am J Hematol. 2018 Nov;93(11):1411-1419. doi: 10.1002/ajh.25260. Epub 2018 Sep 21.

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 12, 2010
• Primary Completion (ACTUAL): December 22, 2014
• Study Completion (ACTUAL): November 7, 2017

Study Registration Dates

• First Submitted: June 3, 2010
• First Submitted That Met QC Criteria: September 21, 2010
• First Posted (ESTIMATE): September 22, 2010

Study Record Updates

• Last Update Posted (ACTUAL): October 21, 2019
• Last Update Submitted That Met QC Criteria: October 18, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Other Study ID Numbers: P071228; 2009-A00142-55 (OTHER: AFSSAPS)