Pediatric refractory chronic immune thrombocytopenia: Identification, patients' characteristics, and outcome - Archive ouverte HAL
Article Dans Une Revue American Journal of Hematology Année : 2024

Pediatric refractory chronic immune thrombocytopenia: Identification, patients' characteristics, and outcome

1 CEREVANCE - Centre de Référence National des Cytopénies Auto-immunes de l'Enfant
2 Centre de recherche du CHU Sainte-Justine / Research Center of the Sainte-Justine University Hospital [Montreal, Canada]
3 UdeM - Université de Montréal
4 CIC Bordeaux
5 CHU Bordeaux - Centre Hospitalier Universitaire de Bordeaux
6 AP-HP Hôpital universitaire Robert-Debré [Paris]
7 CHU Toulouse - Centre Hospitalier Universitaire de Toulouse
8 Hôpital Jeanne de Flandre [Lille]
9 CHU Nantes - Centre Hospitalier Universitaire de Nantes = Nantes University Hospital
10 HCL - Hospices Civils de Lyon
11 Hôpital Arnaud de Villeneuve [CHU Montpellier]
12 Centre Hospitalier Universitaire de Rennes [CHU Rennes] = Rennes University Hospital [Pontchaillou]
13 Service d'Hématologie [CHRU Nancy]
14 CRAN - Centre de Recherche en Automatique de Nancy
15 CHRU Besançon - Centre Hospitalier Régional Universitaire de Besançon
16 Hôpital Bicêtre [AP-HP, Le Kremlin-Bicêtre]
17 Hôpital de Hautepierre [Strasbourg]
18 Hôpital Gatien de Clocheville [Tours]
19 CHU de Poitiers [La Milétrie] - Centre hospitalier universitaire de Poitiers = Poitiers University Hospital
20 CHU Amiens-Picardie
21 CRCI2NA - Centre de Recherche en Cancérologie et Immunologie Intégrée Nantes-Angers
22 CHU Angers - Centre Hospitalier Universitaire d'Angers
23 Hôpital Necker - Enfants Malades [AP-HP]
24 CHUGA - Centre Hospitalier Universitaire [CHU Grenoble]
25 CHRU Brest - Centre Hospitalier Régional Universitaire de Brest
26 CHU Limoges
27 CHU Nice - Centre Hospitalier Universitaire de Nice
28 Institut Jean Godinot [Reims]
29 CHU Reims - Hôpital universitaire Robert Debré [Reims]
30 CHU ST-E - Centre Hospitalier Universitaire de Saint-Etienne [CHU Saint-Etienne]
31 CHU Caen
32 CHU Dijon
33 CHU Clermont-Ferrand
34 CHU Rouen
35 TIMONE - Hôpital de la Timone [CHU - APHM]
36 CHU Trousseau [APHP]
37 SU - Sorbonne Université
38 UPCité - Université Paris Cité
Christophe Piguet
  • Fonction : Auteur
Marianna Deparis
  • Fonction : Auteur
Claire Briandet
  • Fonction : Auteur
Eric Doré
  • Fonction : Auteur
Aude Marie‐Cardine
  • Fonction : Auteur

Résumé

Refractory chronic immune thrombocytopenia (r‐cITP) is one of the most challenging situations in chronic immune thrombocytopenia (cITP). Pediatric r‐cITP is inconsistently defined in literature, contributing to the scarcity of data. Moreover, no evidence is available to guide the choice of treatment. We compared seven definitions of r‐cITP including five pediatric definitions in 886 patients with cITP (median [min‐max] follow‐up 5.3 [1.0–29.3] years). The pediatric definitions identified overlapping groups of various sizes (4%–20%) but with similar characteristics (higher proportion of immunopathological manifestations [IM] and systemic lupus erythematosus [SLE]), suggesting that they adequately captured the population of interest. Based on the 79 patients with r‐cITP (median follow‐up 3.1 [0–18.2] years) according to the CEREVANCE definition (≥3 second‐line treatments), we showed that r‐cITP occurred at a rate of 1.15% new patients per year and did not plateau over time. In multivariate analysis, older age was associated with r‐cITP. One patient (1%) experienced two grade five bleeding events after meeting r‐cITP criteria and while not receiving second‐line treatment. The cumulative incidence of continuous complete remission (CCR) at 2 years after r‐cITP diagnosis was 9%. In this analysis, splenectomy was associated with a higher cumulative incidence of CCR (hazard ratio: 5.43, 95% confidence interval: 1.48–19.84, p = 7.8 × 10 −4 ). In sum, children with cITP may be diagnosed with r‐cITP at any time point of the follow‐up and are at increased risk of IM and SLE. Second‐line treatments seem to be effective for preventing grade 5 bleeding. Splenectomy may be considered to achieve CCR.
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Dates et versions

hal-04653139 , version 1 (18-07-2024)

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Thomas Pincez, Helder Fernandes, Mony Fahd, Marlène Pasquet, Wadih Abou Chahla, et al.. Pediatric refractory chronic immune thrombocytopenia: Identification, patients' characteristics, and outcome. American Journal of Hematology, 2024, 99 (7), pp.1269-1280. ⟨10.1002/ajh.27337⟩. ⟨hal-04653139⟩
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