Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: a treatment versus no-treatment study

Louis Loschi 1, 2 Raphaël Porcher 3, 4, 5 Fiorenza Barraco 6 Louis Terriou 7 Mohamad Mohty 8 Sophie de Guibert 9 Beatrice Mahe 10 Richard Lemal 11 Pierre-Yves Dumas 12 Gabriel Etienne 12 Fabrice Jardin 2 Bruno Royer 13 Dominique Bordessoule 14 Pierre Simon Rohrlich 15 Luc Mathieu Fornecker 16 Celia Salanoubat 17 Sébastien Maury 18 Jean-Yves Cahn 19 Laure Vincent 20, 21 Thomas Sené 22 Sophie Rigaudeau 23 Stephanie Nguyen 24 Anne-Claire Lepretre 25 Jean-Yves Mary 1, 26 Bernadette Corront 27 Gérard Socié 28, 26 Régis Peffault de Latour 29, 26
Abstract : Intravascular hemolysis in Paroxysmal nocturnal hemoglobinuria (PNH) can effectively be controlled with eculizumab, a humanized monoclonal antibody that binds complement protein C5. We report here a retrospective comparison study between 123 patients treated with eculizumab in the recent period (>2005) and 191 historical controls (from the French registry). Overall survival (OS) at 6 years was 92% (95%CI, 87 to 98) in the eculizumab cohort versus 80% (95%CI 70 to 91) in historical controls diagnosed after 1985 (HR 0.38 [0.15 to 0.94], P = 0.037). There were significantly fewer thrombotic events (TEs) in the group of patients treated with eculizumab (4% [1-10]) as compared to the historical cohort (27% [20-34]). However, we found that TEs may still occur after the initiation of eculizumab treatment and that previous TEs still have a negative impact on survival. Evolutions to myelodysplastic syndrome or acute leukemia were similar in both cohorts. There was less evolution to aplastic anemia in the treatment group. In multivariate analysis, absence of a previous TE and treatment with eculizumab were associated with a better OS. Treatment with eculizumab improves overall survival in classic PNH patients without increasing the risk of clonal evolution.
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Soumis le : mardi 2 octobre 2018 - 11:03:31
Dernière modification le : dimanche 19 mai 2019 - 01:09:44

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Louis Loschi, Raphaël Porcher, Fiorenza Barraco, Louis Terriou, Mohamad Mohty, et al.. Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: a treatment versus no-treatment study. American Journal of Hematology, Wiley, 2016, 91 (4), pp.366 - 370. ⟨10.1002/ajh.24278⟩. ⟨hal-01885662⟩



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