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Prognostic factors of disease severity in infants with sickle cell anemia: A comprehensive longitudinal cohort study.
Brousse, Valentine; El Hoss, Sara; Bouazza, Naïm; Arnaud, Cécile; Bernaudin, Francoise; Pellegrino, Béatrice; Guitton, Corinne; Odièvre-Montanié, Marie-Hélène; Mames, David; Brouzes, Chantal; Picard, Véronique; Nguyen-Khoa, Thao; Pereira, Catia; Lapouméroulie, Claudine; Pissard, Serge; Gardner, Kate; Menzel, Stephan; Le Van Kim, Caroline; Colin-Aronovicz, Yves; Buffet, Pierre; Mohandas, Narla; Elie, Caroline; Maier-Redelsperger, Micheline; El Nemer, Wassim; de Montalembert, Mariane.
Afiliação
  • Brousse V; Service de Pédiatrie et Maladies Infectieuses, Hôpital Universitaire Necker-Enfants Malades, Paris, France.
  • El Hoss S; UMR_S 1134 Biologie Intégrée du Globule Rouge, Université Sorbonne Paris Cité/Université Paris Diderot/INSERM/INTS/Laboratoire d'Excellence GR-Ex, Paris, France.
  • Bouazza N; UMR_S 1134 Biologie Intégrée du Globule Rouge, Université Sorbonne Paris Cité/Université Paris Diderot/INSERM/INTS/Laboratoire d'Excellence GR-Ex, Paris, France.
  • Arnaud C; Unité de Recherche Clinique/Centre d'investigation clinique Paris Descartes Necker-Cochin, Assistance Publique-Hôpitaux de Paris, Paris, France.
  • Bernaudin F; Université Paris Descartes, EA7323, Sorbonne Paris Cité, Paris, France.
  • Pellegrino B; Service de Pédiatrie, Centre Hospitalier Intercommunal de Créteil, Créteil, France.
  • Guitton C; Service de Pédiatrie, Centre Hospitalier Intercommunal de Créteil, Créteil, France.
  • Odièvre-Montanié MH; Service de Pediatrie, Centre Hospitalier Poissy-Saint Germain, Poissy, France.
  • Mames D; Service de Pédiatrie, Hôpital Universitaire Kremlin-Bicêtre, Le Kremlin Bicêtre, France.
  • Brouzes C; Service de Pédiatrie, Hôpital Universitaire Armand Trousseau, Paris, France.
  • Picard V; Laboratoire d'Hématologie, Hôpital Universitaire Tenon, Paris, France.
  • Nguyen-Khoa T; Laboratoires d'Hématologie et de Biochimie, Hôpital Universitaire Necker-Enfants Malades, Paris, France.
  • Pereira C; Laboratoire d'Hématologie, Hôpital Universitaire Kremlin Bicêtre, Le Kremlin Bicêtre, France.
  • Lapouméroulie C; Laboratoires d'Hématologie et de Biochimie, Hôpital Universitaire Necker-Enfants Malades, Paris, France.
  • Pissard S; UMR_S 1134 Biologie Intégrée du Globule Rouge, Université Sorbonne Paris Cité/Université Paris Diderot/INSERM/INTS/Laboratoire d'Excellence GR-Ex, Paris, France.
  • Gardner K; UMR_S 1134 Biologie Intégrée du Globule Rouge, Université Sorbonne Paris Cité/Université Paris Diderot/INSERM/INTS/Laboratoire d'Excellence GR-Ex, Paris, France.
  • Menzel S; Laboratoire de Biochimie Génétique, Hôpital Universitaire Henri Mondor, Créteil, France.
  • Le Van Kim C; King's College London, Division of Cancer Studies, London, UK.
  • Colin-Aronovicz Y; King's College Hospital NHS Foundation Trust, London, UK.
  • Buffet P; King's College London, Division of Cancer Studies, London, UK.
  • Mohandas N; UMR_S 1134 Biologie Intégrée du Globule Rouge, Université Sorbonne Paris Cité/Université Paris Diderot/INSERM/INTS/Laboratoire d'Excellence GR-Ex, Paris, France.
  • Elie C; UMR_S 1134 Biologie Intégrée du Globule Rouge, Université Sorbonne Paris Cité/Université Paris Diderot/INSERM/INTS/Laboratoire d'Excellence GR-Ex, Paris, France.
  • Maier-Redelsperger M; UMR_S 1134 Biologie Intégrée du Globule Rouge, Université Sorbonne Paris Cité/Université Paris Diderot/INSERM/INTS/Laboratoire d'Excellence GR-Ex, Paris, France.
  • El Nemer W; Red Cell Physiology Laboratory, New York Blood Center, New York, New York.
  • de Montalembert M; Unité de Recherche Clinique/Centre d'investigation clinique Paris Descartes Necker-Cochin, Assistance Publique-Hôpitaux de Paris, Paris, France.
Am J Hematol ; 93(11): 1411-1419, 2018 11.
Article em En | MEDLINE | ID: mdl-30132969
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 Sß°) 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.
Assuntos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Índice de Gravidade de Doença / Anemia Falciforme Tipo de estudo: Clinical_trials / Etiology_studies / Incidence_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Female / Humans / Infant / Male Idioma: En Ano de publicação: 2018 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Índice de Gravidade de Doença / Anemia Falciforme Tipo de estudo: Clinical_trials / Etiology_studies / Incidence_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Female / Humans / Infant / Male Idioma: En Ano de publicação: 2018 Tipo de documento: Article