TY - JOUR
T1 - CNS involvement and treatment with interferon-α are independent prognostic factors in Erdheim-Chester disease
T2 - A multicenter survival analysis of 53 patients
AU - Arnaud, Laurent
AU - Hervier, Baptiste
AU - Néel, Antoine
AU - Hamidou, Mohamed A.
AU - Kahn, Jean Emmanuel
AU - Wechsler, Bertrand
AU - Pérez-Pastor, Gemma
AU - Blomberg, Bjørn
AU - Fuzibet, Jean Gabriel
AU - Dubourguet, François
AU - Marinho, António
AU - Magnette, Catherine
AU - Noel, Violaine
AU - Pavic, Michel
AU - Casper, Jochen
AU - Beucher, Anne Bérangère
AU - Costedoat-Chalumeau, Nathalie
AU - Aaron, Laurent
AU - Salvatierra, Juan
AU - Graux, Carlos
AU - Cacoub, Patrice
AU - Delcey, Véronique
AU - Dechant, Claudia
AU - Bindi, Pascal
AU - Herbaut, Christiane
AU - Graziani, Giorgio
AU - Amoura, Zahir
AU - Haroche, Julien
PY - 2011/3/10
Y1 - 2011/3/10
N2 - Erdheim-Chester disease (ECD) is a rare form of non-Langerhans histiocytosis, with noncodified therapeutic management and high mortality. No treatment has yet been shown to improve survival in these patients. We conducted a multicenter prospective observational cohort study to assess whether extraskeletal manifestations and interferon-α treatment would influence survival in a large cohort of ECD patients. To achieve this goal, we thoroughly analyzed the clinical presentation of 53 patients with biopsyproven ECD, and we performed a survival analysis using Cox proportional hazard model. Fifty-three patients (39 men and 14 women) with biopsy-proven ECD were followed up between November 1981 and November 2010. Forty-six patients (87%) received interferon-α and/or PEGylated interferon-α. Multivariate survival analysis using Cox proportional hazard model revealed that central nervous system involvement was an independent predictor of death (hazard ratio = 2.51; 95% confidence interval, 1.28-5.52; P = .006) in our cohort. Conversely, treatment with interferon-α was identified as an independent predictor of survival (hazard ratio = 0.32; 95% confidence interval, 0.14-0.70; P = .006). Although definitive confirmation would require a randomized controlled trial, these results suggest that interferon-α improves survival in ECD patients. This may be seen as a significant advance, as it is the first time a treatment is shown to improve survival in this multisystemic disease with high mortality.
AB - Erdheim-Chester disease (ECD) is a rare form of non-Langerhans histiocytosis, with noncodified therapeutic management and high mortality. No treatment has yet been shown to improve survival in these patients. We conducted a multicenter prospective observational cohort study to assess whether extraskeletal manifestations and interferon-α treatment would influence survival in a large cohort of ECD patients. To achieve this goal, we thoroughly analyzed the clinical presentation of 53 patients with biopsyproven ECD, and we performed a survival analysis using Cox proportional hazard model. Fifty-three patients (39 men and 14 women) with biopsy-proven ECD were followed up between November 1981 and November 2010. Forty-six patients (87%) received interferon-α and/or PEGylated interferon-α. Multivariate survival analysis using Cox proportional hazard model revealed that central nervous system involvement was an independent predictor of death (hazard ratio = 2.51; 95% confidence interval, 1.28-5.52; P = .006) in our cohort. Conversely, treatment with interferon-α was identified as an independent predictor of survival (hazard ratio = 0.32; 95% confidence interval, 0.14-0.70; P = .006). Although definitive confirmation would require a randomized controlled trial, these results suggest that interferon-α improves survival in ECD patients. This may be seen as a significant advance, as it is the first time a treatment is shown to improve survival in this multisystemic disease with high mortality.
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U2 - 10.1182/blood-2010-06-294108
DO - 10.1182/blood-2010-06-294108
M3 - Article
C2 - 21239701
AN - SCOPUS:79953124751
VL - 117
SP - 2778
EP - 2782
JO - Blood
JF - Blood
SN - 0006-4971
IS - 10
ER -