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

dc.rights.licenseopenen_US
dc.contributor.authorDUCASSOU, S.
dc.contributor.authorGOURDONNEAU, A.
dc.contributor.authorFERNANDES, H.
dc.contributor.authorLEVERGER, G.
dc.contributor.authorPASQUET, M.
dc.contributor.authorFOUYSSAC, F.
dc.contributor.authorBAYART, S.
dc.contributor.authorBERTRAND, Y.
dc.contributor.authorMICHEL, G.
dc.contributor.authorJEZIORSKI, E.
dc.contributor.authorTHOMAS, C.
dc.contributor.authorABOUCHALLAH, W.
dc.contributor.authorVIARD, F.
dc.contributor.authorGUITTON, C.
dc.contributor.authorCHEIKH, N.
dc.contributor.authorPELLIER, I.
dc.contributor.authorCARAUSU, L.
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorDROZ-PERROTEAU, Cécile
dc.contributor.authorLEBLANC, T.
dc.contributor.authorALADJIDI, N.
dc.date.accessioned2021-02-08T11:02:40Z
dc.date.available2021-02-08T11:02:40Z
dc.date.issued2020
dc.identifier.issn0007-1048en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/26174
dc.description.abstractEnChildhood 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.
dc.language.isoENen_US
dc.subjectPharmacoEpi-Drugs
dc.title.enSecond-line treatment trends and long-term outcomes of 392 children with chronic immune thrombocytopenic purpura: the French experience over the past 25 years
dc.title.alternativeBr J Haematolen_US
dc.typeArticle de revueen_US
dc.identifier.doi10.1111/bjh.16448en_US
dc.subject.halSciences du Vivant [q-bio]/Santé publique et épidémiologieen_US
dc.identifier.pubmed32130726en_US
bordeaux.journalBritish Journal of Haematologyen_US
bordeaux.page931-942en_US
bordeaux.volume189en_US
bordeaux.hal.laboratoriesBordeaux Population Health Research Center (BPH) - UMR 1219en_US
bordeaux.issue5en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.teamPharmacoEpi-Drugsen_US
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
hal.identifierhal-03134407
hal.version1
hal.date.transferred2021-02-08T11:02:45Z
hal.exporttrue
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