Incidence and risk factors for clinical neurodegenerative Langerhans cell histiocytosis: a longitudinal cohort study

Sébastien Héritier 1 Mohamed-Aziz Barkaoui 2 Jean Miron 3 Caroline Thomas 4 Despina Moshous 5 Anne Lambilliotte 6 Françoise Mazingue 7 Kamila Kebaili 8 Eric Jeziorski 9 Genevieve Plat 10 Nathalie Aladjidi 11 Hélène Pacquement 12 Claire Galambrun 13 Laurence Brugières 14 Guy Leverger 15 Ludovic Mansuy 16 Catherine Paillard 17 Anne Deville 18 Anne Pagnier 19 Anne Lutun 20 Marion Gillibert-Yvert 21 Jean-Louis Stephan 22 Fleur Cohen-Aubart 23 Julien Haroche 23 Isabelle Pellier 24 Frédéric Millot 25 Virginie Gandemer 26 Nadine Martin-Duverneuil 27 Valérie Taly 28, 29 Zofia Hélias-Rodzewicz 30 Jean-François Emile 30 Khê Hoang-Xuan 31 Ahmed Idbaih 32 Jean Donadieu 15
Abstract : Neurodegenerative (ND) complications in Langerhans cell histiocytosis (LCH) are a late-onset but dramatic sequelae for which incidence and risk factors are not well defined. Based on a national prospective registry of paediatric LCH patients, we determined the incidence rate of clinical ND LCH (cND-LCH) and analysed risk factors, taking into account disease extent and molecular characteristics. Among 1897 LCH patients, 36 (1·9%) were diagnosed with a cND-LCH. The 10-year cumulative incidence of cND-LCH was 4·1%. cND-LCH typically affected patients previously treated for a multisystem, risk organ-negative LCH, represented in 69·4% of cND-LCH cases. Pituitary gland, skin and base skull/orbit bone lesions were more frequent (P < 0·001) in cND-LCH patients compared to those without cND-LCH (respectively 86·1% vs. 12·2%, 75·0% vs. 34·2%, and 63·9% vs. 28·4%). The 'cND susceptible patients' (n = 671) i.e., children who had experienced LCH disease with pituitary or skull base or orbit bone involvement, had a 10-year cND risk of 7·8% vs. 0% for patients who did not meet these criteria. Finally, BRAFV 600E status added important information among these cND susceptible patients, with the 10-year cND risk of 33·1% if a BRAFV 600E mutation was present compared to 2·9% if it was absent (P = 0·002).
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Sébastien Héritier, Mohamed-Aziz Barkaoui, Jean Miron, Caroline Thomas, Despina Moshous, et al.. Incidence and risk factors for clinical neurodegenerative Langerhans cell histiocytosis: a longitudinal cohort study. British Journal of Haematology, Wiley, 2018, 183 (4), pp.608-617. ⟨10.1111/bjh.15577⟩. ⟨inserm-02299489⟩

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