TY - JOUR
T1 - Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria
T2 - A treatment versus no-treatment study
AU - Loschi, Michael
AU - Porcher, Raphael
AU - Barraco, Fiorenza
AU - Terriou, Louis
AU - Mohty, Mohamad
AU - De Guibert, Sophie
AU - Mahe, Beatrice
AU - Lemal, Richard
AU - Dumas, Pierre Yves
AU - Etienne, Gabriel
AU - Jardin, Fabrice
AU - Royer, Bruno
AU - Bordessoule, Dominique
AU - Rohrlich, Pierre Simon
AU - Fornecker, Luc Mathieu
AU - Salanoubat, Celia
AU - Maury, Sebastien
AU - Cahn, Jean Yves
AU - Vincent, Laure
AU - Sene, Thomas
AU - Rigaudeau, Sophie
AU - Nguyen, Stephanie
AU - Lepretre, Anne Claire
AU - Mary, Jean Yves
AU - Corront, Bernadette
AU - Socie, Gerard
AU - Peffault de Latour, Regis
N1 - Publisher Copyright:
© 2016 Wiley Periodicals, Inc.
PY - 2016/4/1
Y1 - 2016/4/1
N2 - 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. Am. J. Hematol. 91:366-370, 2016.
AB - 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. Am. J. Hematol. 91:366-370, 2016.
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U2 - 10.1002/ajh.24278
DO - 10.1002/ajh.24278
M3 - Article
C2 - 26689746
AN - SCOPUS:84961262730
SN - 0361-8609
VL - 91
SP - 366
EP - 370
JO - American Journal of Hematology
JF - American Journal of Hematology
IS - 4
ER -
