CNS involvement and treatment with interferon-α are independent prognostic factors in Erdheim-Chester disease: a multicenter survival analysis of 53 patients

被引：286

作者：

Arnaud, Laurent ^{[1
,2
,3
]}

Hervier, Baptiste ^{[1
,2
,4
]}

Neel, Antoine ^{[5
]}

Hamidou, Mohamed A. ^{[5
]}

Kahn, Jean-Emmanuel ^{[6
]}

Wechsler, Bertrand ^{[1
,2
]}

Perez-Pastor, Gemma ^{[7
]}

Blomberg, Bjorn ^{[8
]}

Fuzibet, Jean-Gabriel ^{[9
]}

Dubourguet, Francois ^{[10
]}

Marinho, Antonio ^{[11
]}

Magnette, Catherine ^{[12
]}

Noel, Violaine ^{[13
]}

Pavic, Michel ^{[14
]}

Casper, Jochen ^{[15
]}

Beucher, Anne-Berangere ^{[16
]}

Costedoat-Chalumeau, Nathalie ^{[1
,2
]}

Aaron, Laurent ^{[17
]}

Salvatierra, Juan ^{[18
]}

Graux, Carlos ^{[19
]}

Cacoub, Patrice ^{[1
,2
]}

Delcey, Veronique ^{[20
]}

Dechant, Claudia ^{[21
]}

Bindi, Pascal ^{[22
]}

Herbaut, Christiane ^{[23
]}

Graziani, Giorgio ^{[24
]}

Amoura, Zahir ^{[1
,2
,3
,4
]}

Haroche, Julien ^{[1
,2
,4
]}

机构：

[1] Pitie Salpetriere Hop, AP HP, Dept Internal Med, Paris, France

[2] Pitie Salpetriere Hop, AP HP, French Reference Ctr Rare Autoimmune & Syst Dis, Paris, France

[3] INSERM, UMR, S 945, Paris, France

[4] Univ Paris 06, Paris, France

[5] CHU Hotel Dieu, Dept Internal Med, Nantes, France

[6] Hop Foch, Dept Internal Med, Suresnes, France

[7] La Plana Villarreal Hosp, Dept Dermatol, Castellon de La Plana, Spain

[8] Univ Bergen, Inst Internal Med, Bergen, Norway

[9] CHU, Hop Archet, Dept Internal Med, Nice, France

[10] HIA Val de Grace, Dept Internal Med, Paris, France

[11] Ctr Hosp Porto, Hosp Santo Antonio, Dept Internal Med, Porto, Portugal

[12] Clin Univ Mt Godinne, Dept Geriatr Med, Mt Godinne, Belgium

[13] Hop Robert Debre, CHU Reims, Dept Internal Med, Reims, France

[14] HIA Desgenettes, Dept Internal Med, Lyon, France

[15] Univ Rostock, Dept Hematol & Oncol, Rostock, Germany

[16] CHU Angers, Dept Internal Med, Angers, France

[17] Hop Jacques Coeur, Dept Internal Med, Bourges, France

[18] Univ Hosp Viergen de las Nieves, Dept Rheumatol, Granada, Spain

[19] Catholic Univ Louvain, Clin Univ St Luc, Dept Hematol, Brussels, Belgium

[20] CHU Lariboisiere, Dept Internal Med, Paris, France

[21] Univ Munich, Med Poliklin, Div Rheumatol, D-8000 Munich, Germany

[22] Ctr Hosp Verdun, Dept Med A, Verdun, France

[23] CHU Brugmann, Dept Internal Med & Endocrinol, Brussels, Belgium

[24] Ist Clin Humanitas IRCCS, Dept Internal Med & Nephrol, Rozzano Milano, Italy

来源：

BLOOD | 2011年 / 117卷 / 10期

关键词：

MAGNETIC-RESONANCE; RECEPTOR;

D O I：

10.1182/blood-2010-06-294108

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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 multi-center prospective observational cohort study to assess whether extraskeletal manifestations and interferon-alpha 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-alpha and/or PEGylated interferon-alpha. 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-alpha 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-alpha 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. (Blood. 2011;117(10):2778-2782)

引用

页码：2778 / 2782

页数：5

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