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scientific articles

Prognostic factors of disease severity in infants with sickle cell anemia: A comprehensive longitudinal cohort study

key information

source: American journal of hematology

year: 2018

authors: Brousse V, El Hoss S, Bouazza N, Arnaud C, Bernaudin F, Pellegrino B, Guitton C, Odièvre-Montanié MH, Mames D, Brouzes C, Picard V, Nguyen-Khoa T, Pereira C, Lapouméroulie 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, the Montalembert M


In order to identify very early prognostic factors that can provide insights into subsequent clinical complications, we performed a comprehensive longitudinal multi-center cohort study on 57 infants with sickle cell anemia (55 SS; 2 Sβ°) during the first 2 years of life (ClinicalTrials.gov: NCT01207037). Time to first occurrence of a severe clinical event-acute splenic sequestration (ASS), vaso-occlusive (VOC) event requiring hospitalization, transfusion requirement, conditional/ abnormal cerebral velocities, or death-was used as a composite endpoint. Infants were recruited at a mean age of 4.4 ±1 months. Median follow-up was 19.4 months. During the study period, 38.6% of infants experienced >=1 severe event: 14% ASS, 22.8% >= 1 VOC (median age: 13.4 and 12.8 months, respectively) and 33.3% required transfusion. Of note, 77% of the cohort was hospitalized, with febrile illness being the leading cause for admission.

Univariate analysis of various biomarkers measured at enrollment showed that fetal hemoglobin (HbF) was the strongest prognostic factor of subsequent severe outcome. Other biomarkers measured at enrolment including absolute neutrophil or reticulocyte counts, expression of erythroid adhesion markers, % of dense red cells, cellular deformability or ϒ-globin genetic variants, failed to be associated with severe clinical outcome. Multivariate analysis demonstrated that higher Hb concentration and HbF level are two independent protective factors (adjusted HRs (95% CI) 0.27 (0.11-0.73) and 0.16 (0.06-0.43), respectively). These findings imply that early measurement of HbF and Hb levels can identify infants at high risk for subsequent severe complications, who might maximally benefit from early disease modifying treatments.

organization: Necker-Enfants Malades University Hospital, France; Sorbonne University Paris Cité / Paris Diderot University / INSERM / INTS / GR-Ex Laboratory of Excellence, France; Assistance Publique-Hôpitaux de Paris, France; Paris Descartes University, France; Intercommunal Hospital Center of Créteil, France; Poissy-Saint Germain Hospital, France; Kremlin-Bicêtre University Hospital, France; Armand Trousseau University Hospital, France; Tenon University Hospital, France; Henri Mondor University Hospital, France; King's College London, UK; King's College Hospital NHS Foundation Trust, UK; New York Blood Center, USA

DOI: 10.1002 / ajh.25260

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