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Br J Haematol. 2020 Mar 4. doi: 10.1111/bjh.16448. [Epub ahead of print]

Second-line treatment trends and long-term outcomes of 392 children with chronic immune thrombocytopenic purpura: the French experience over the past 25 years.

Author information

1
Pediatric Hematology Unit, CIC1401, INSERM CICP, University Hospital of Bordeaux, Bordeaux, France.
2
Centre de Référence National des Cytopénies Autoimmunes de l'enfant (CEREVANCE), University Hospital of Bordeaux, Bordeaux, France.
3
University of Bordeaux, INSERM U1218, Bordeaux, France.
4
Centre de Recherche Saint Antoine UMR_S 938, Service d'Hématologie Oncologie Pédiatrique, Centre de Référence National des Cytopénies Auto-immunes de l'enfant (CEREVANCE), AP-HP, Hôpital Armand Trousseau, Sorbonne Université, Paris, France.
5
Pediatric Hematology Unit, University Hospital of Toulouse, Toulouse, France.
6
Pediatric Hematology Unit, University Hospital of Nancy, Nancy, France.
7
Pediatric Hematology Unit, University Hospital of Rennes, Rennes, France.
8
Pediatric Hematology Unit, Institute of Pediatric Hematology and Oncology, Claude Bernard University Lyon, Lyon, France.
9
Pediatric Hematology Unit, University Hospital Timone Enfants, Marseille, France.
10
Department of Pediatrics, Hôpital Arnaud de Villeneuve, University Hospital of Montpellier, Montpellier, France.
11
Pediatric Hematology Unit, University Hospital of Nantes, Nantes, France.
12
Pediatric Hematology Unit, University Hospital of Lille, Lille, France.
13
Department of Pediatrics, University Hospital of Bicêtre, Le Kremlin-Bicêtre, France.
14
Pediatric Hematology Unit, University Hospital of Besançon, Besançon, France.
15
Pediatric Hematology Unit, University Hospital of Angers, Angers, France.
16
Pediatric Hematology Unit, University Hospital of Brest, Brest, France.
17
Inserm CIC1401, Bordeaux PharmacoEpi, University of Bordeaux, Bordeaux, France.
18
Hematology Unit, Centre de référeNce National des Cytopénies Auto-Immunes de l'enfant (CEREVANCE), APHP - Hôpital Robert Debré, Paris, France.

Abstract

Childhood chronic immune thrombocytopenic purpura (cITP) is a rare disease. In severe cases, there is no evidence for the optimal therapeutic strategy. Our aim was to describe the real-life management of non-selected children with cITP at diagnosis. Since 2004, patients less than 18 years old with cITP have been enrolled in the national prospective cohort, OBS'CEREVANCE. From 1990 to 2014, in 29 centres, 392 children were diagnosed with cITP. With a median follow-up of six years (2·0-25), 45% did not need second-line therapy, and 55% (n = 217) received one or more second lines, mainly splenectomy (n = 108), hydroxychloroquine (n = 61), rituximab (n = 61) or azathioprine (n = 40). The overall five-year further second-line treatment-free survival was 56% [95% CI 49·5-64.1]. The use of splenectomy significantly decreased over time. Hydroxychloroquine was administered to children with positive antinuclear antibodies, more frequently older and girls, and reached 55% efficacy. None of the patients died. Ten years after the initial diagnosis, 55% of the 56 followed children had achieved complete remission. Children with cITP do not need second-line treatments in 45% of cases. Basing the treatment decision on the pathophysiological pathways is challenging, as illustrated by ITP patients with positive antinuclear antibodies treated with hydroxychloroquine.

KEYWORDS:

children; immune thrombocytopenic purpura; immunosuppressive treatments; observational cohort; splenectomy

PMID:
32130726
DOI:
10.1111/bjh.16448

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