Lamiae Grimaldi-Bensouda1, Clémentine Nordon1, Thierry Leblanc2,3, Lucien Abenhaim1, Slimane Allali4, Corinne Armari-Alla5, Claire Berger6, Mary-France Courcoux7, Fanny Fouyssac8, Cécile Guillaumat9, Corinne Guitton10, Philippe Le Moine11, Françoise Mazingue12, Corinne Pondarré13, Caroline Thomas14, Marlène Pasquet15, Yves Perel3,16, Guy Leverger3,7, Nathalie Aladjidi3,16. 1. LASER, Paris, France. 2. Department of Pediatric Hematology, Robert-Debré Hospital, AP-HP, Paris, France. 3. French Reference Center for Rare Diseases and Autoimmune Cytopenias of Childhood (CEREVANCE), Pellegrin Hospital, Bordeaux, France. 4. Department of General Pediatrics, Necker Hospital, AP-HP and Paris Descartes University, Paris, France. 5. Department of Pediatric Hematology, Grenoble University Hospital, Grenoble, France. 6. Department of Pediatric Hematology, Saint-Etienne University Hospital, Saint-Etienne, France. 7. Department of Immunology and Hematology, Armand-Trousseau Hospital, Paris, France. 8. Department of Pediatric Hematology, Nancy University Hospital, Vandoeuvre-les-Nancy, France. 9. Department of General Pediatrics, Sud-Francilien Hospital, Corbeil-Essonnes, France. 10. Department of Pediatric Rheumatology and Hematology, Bicêtre Hospital, Le Kremlin-Bicêtre, France. 11. Department of Pediatrics and Clinical Genetics, Morvan University Hospital, Brest, France. 12. Department of Pediatric Hematology, Lille University Hospital, Lille, France. 13. Department of Pediatrics, Intercommunal Hospital, Creteil, France. 14. Pediatric Intensive Care and Onco-Hematology Units, Nantes University Hospital, Nantes, France. 15. Department of Pediatric Hematology and Immunology, Purpan University Hospital and INSERM U1037, Toulouse, France. 16. Department of Pediatric Hematology, CIC 1401, INSERM CICP, Pellegrin Hospital, Bordeaux, France.
Abstract
OBJECTIVES: Nationwide prospective cohort study exploring (i) the factors associated with treatment initiation (vs. watchful waiting) in children with primary immune thrombocytopenia (ITP) followed in routine clinical practice and (ii) the predictors of chronicity at 12 months. PROCEDURE: Between 2008 and 2013, 23 centers throughout France consecutively included 257 children aged 6 months-18 years and diagnosed with primary ITP over a 5-year period. Data on ITP clinical features along with medical management were collected at baseline and 12 months. Multivariate logistic regressions were used to determine (i) and (ii) as defined above, providing odds ratio (OR) with 95% confidence interval (95% CI). RESULTS: One hundred thirty-seven (53%) children were males, median age was 4.6 years, median platelet count was 7 × 109/l, and 214 (81%) patients initiated medication. Factors independently associated with treatment initiation included platelet counts <10 × 109/l (P < 0.0001) and mucocutaneous bleeding symptoms at baseline (P < 0.001). At 12 months, data were available for 211 (82%) children, of whom 160 (74%) had recovered. Predictors of chronicity included female gender (OR = 2.2; 95% CI = 1.0-4.8), age ≥10 years (OR = 2.6; 95% CI = 1.1-6.0), and platelet counts ≥10 × 109 /l (OR = 3.2; 95% CI = 1.5-6.9). CONCLUSIONS: In routine clinical practice, the decision to apply a watchful waiting strategy seems to be driven by platelet counts even in the absence of bleeding symptoms, resulting in treatment being initiated in more than 80% of the children surveyed. Overall, younger children with ITP showed good prognosis, with lower platelet counts and, to a lesser extent, male gender predicting more favorable outcomes.
OBJECTIVES: Nationwide prospective cohort study exploring (i) the factors associated with treatment initiation (vs. watchful waiting) in children with primary immune thrombocytopenia (ITP) followed in routine clinical practice and (ii) the predictors of chronicity at 12 months. PROCEDURE: Between 2008 and 2013, 23 centers throughout France consecutively included 257 children aged 6 months-18 years and diagnosed with primary ITP over a 5-year period. Data on ITP clinical features along with medical management were collected at baseline and 12 months. Multivariate logistic regressions were used to determine (i) and (ii) as defined above, providing odds ratio (OR) with 95% confidence interval (95% CI). RESULTS: One hundred thirty-seven (53%) children were males, median age was 4.6 years, median platelet count was 7 × 109/l, and 214 (81%) patients initiated medication. Factors independently associated with treatment initiation included platelet counts <10 × 109/l (P < 0.0001) and mucocutaneous bleeding symptoms at baseline (P < 0.001). At 12 months, data were available for 211 (82%) children, of whom 160 (74%) had recovered. Predictors of chronicity included female gender (OR = 2.2; 95% CI = 1.0-4.8), age ≥10 years (OR = 2.6; 95% CI = 1.1-6.0), and platelet counts ≥10 × 109 /l (OR = 3.2; 95% CI = 1.5-6.9). CONCLUSIONS: In routine clinical practice, the decision to apply a watchful waiting strategy seems to be driven by platelet counts even in the absence of bleeding symptoms, resulting in treatment being initiated in more than 80% of the children surveyed. Overall, younger children with ITP showed good prognosis, with lower platelet counts and, to a lesser extent, male gender predicting more favorable outcomes.
Authors: Drew Provan; Donald M Arnold; James B Bussel; Beng H Chong; Nichola Cooper; Terry Gernsheimer; Waleed Ghanima; Bertrand Godeau; Tomás José González-López; John Grainger; Ming Hou; Caroline Kruse; Vickie McDonald; Marc Michel; Adrian C Newland; Sue Pavord; Francesco Rodeghiero; Marie Scully; Yoshiaki Tomiyama; Raymond S Wong; Francesco Zaja; David J Kuter Journal: Blood Adv Date: 2019-11-26