CNS involvement and treatment with interferon-α are independent prognostic factors in Erdheim-Chester disease: a multicenter survival analysis of 53 patients.
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 biopsy-proven 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.
Department of Internal Medicine and French Reference Center for Rare Autoimmune and Systemic Diseases, AP-HP, Pitié-Salpêtrière Hôpital, Paris, France.
Laurent
L
Arnaud
Baptiste
B
Hervier
Antoine
A
Néel
Mohamed A
MA
Hamidou
Jean-Emmanuel
JE
Kahn
Bertrand
B
Wechsler
Gemma
G
Pérez-Pastor
Bjørn
B
Blomberg
Jean-Gabriel
JG
Fuzibet
François
F
Dubourguet
António
A
Marinho
Catherine
C
Magnette
Violaine
V
Noel
Michel
M
Pavic
Jochen
J
Casper
Anne-Bérangère
AB
Beucher
Nathalie
N
Costedoat-Chalumeau
Laurent
L
Aaron
Juan
J
Salvatierra
Carlos
C
Graux
Patrice
P
Cacoub
Véronique
V
Delcey
Claudia
C
Dechant
Pascal
P
Bindi
Christiane
C
Herbaut
Giorgio
G
Graziani
Zahir
Z
Amoura
Julien
J
Haroche
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001921:Brain / Q000473:pathology*; D002648:Child; D015331:Cohort Studies; D031249:Erdheim-Chester Disease / Q000188:drug therapy* / Q000401:mortality* / Q000473:pathology*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use*; D016898:Interferon-alpha / Q000627:therapeutic use*; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D016016:Proportional Hazards Models; D055815:Young Adult
D007155:Immunologic Factors; D016898:Interferon-alpha