Clinical trial NCT01207037: Prognostic Factors of Acute Splenic Sequestration
Official title: Study of Prognostic Factors of Acute Splenic Sequestration in a Cohort of Sickle Cell Disease (SCD) Children Diagnosed at Birth
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Anemia; Drepanocytic
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2010-09-22
Enrollment: 58
Sex: ALL
Minimum age (years): 3
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: 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

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)

Eligibility criteria: 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

Primary outcomes:
measure: Study of prognostic factors of acute splenic sequestration
timeFrame: after three years
Secondary outcomes:
measure: Study of hematology parameters
timeFrame: after three years
Other outcomes:
Locations:
city: Paris
country: France
facility: Necker Hospital
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Valentine Brousse, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.