Clinical features, treatment, and outcome of macrophage activation syndrome complicating systemic juvenile idiopathic Arthritis: a multinational, multicenter study of 362 patients

TY - JOUR

T1 - Clinical features, treatment, and outcome of macrophage activation syndrome complicating systemic juvenile idiopathic Arthritis: a multinational, multicenter study of 362 patients

AU - Minoia, Francesca

AU - Davì, Sergio

AU - Horne, AnnaCarin

AU - Demirkaya, Erkan

AU - Bovis, Francesca

AU - Li, Caifeng

AU - Lehmberg, Kai

AU - Weitzman, Sheila

AU - Insalaco, Antonella

AU - Wouters, Carine

AU - Shenoi, Susan

AU - Espada, Graciela

AU - Ozen, Seza

AU - Anton, Jordi

AU - Khubchandani, Raju

AU - Russo, Ricardo

AU - Pal, Priyankar

AU - Kasapcopur, Ozgur

AU - Miettunen, Paivi

AU - Maritsi, Despoina

AU - Merino, Rosa

AU - Shakoory, Bita

AU - Alessio, Maria

AU - Chasnyk, Vyacheslav

AU - Sanner, Helga

AU - Gao, Yi-Jin

AU - Huasong, Zeng

AU - Kitoh, Toshiyuki

AU - Avcin, Tadej

AU - Fischbach, Michel

AU - Frosch, Michael

AU - Grom, Alexei

AU - Huber, Adam

AU - Jelusic, Marija

AU - Sawhney, Sujata

AU - Uziel, Yosef

AU - Ruperto, Nicolino

AU - Martini, Alberto

AU - Cron, Randy Q

AU - Ravelli, Angelo

AU - Pediatric Rheumatology International Trials Organization

N1 - Copyright © 2014 by the American College of Rheumatology.

PY - 2014/11/1

Y1 - 2014/11/1

N2 - OBJECTIVE: To describe the clinical, laboratory, and histopathologic features, current treatment, and outcome of patients with macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (JIA).METHODS: In this multinational, multicenter study, pediatric rheumatologists and hemato-oncologists entered patient data collected retrospectively into a web-based database.RESULTS: A total of 362 patients, 22% of whom had MAS at the onset of systemic JIA, were included in the study by 95 investigators from 33 countries. The most frequent clinical manifestations were fever (96%), hepatomegaly (70%), and splenomegaly (58%). Central nervous system dysfunction and hemorrhages were recorded in 35% and 20% of the patients, respectively. Platelet count and liver transaminase, ferritin, lactate dehydrogenase, triglyceride, and d-dimer levels were the sole laboratory biomarkers showing a percentage change of >50% between the pre-MAS visit and MAS onset. Evidence of macrophage hemophagocytosis was found in 60% of the patients who underwent bone marrow aspiration. MAS occurred most frequently in the setting of active underlying disease, in the absence of a specific trigger. Nearly all patients were given corticosteroids, and 61% received cyclosporine. Biologic medications and etoposide were given to 15% and 12% of the patients, respectively. Approximately one-third of the patients required admission to the intensive care unit (ICU), and the mortality rate was 8%.CONCLUSION: This study provides information on the clinical spectrum and current management of systemic JIA-associated MAS through the analysis of a very large patient sample. MAS remains a serious condition, as a sizeable proportion of patients required admission to the ICU or died.

AB - OBJECTIVE: To describe the clinical, laboratory, and histopathologic features, current treatment, and outcome of patients with macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (JIA).METHODS: In this multinational, multicenter study, pediatric rheumatologists and hemato-oncologists entered patient data collected retrospectively into a web-based database.RESULTS: A total of 362 patients, 22% of whom had MAS at the onset of systemic JIA, were included in the study by 95 investigators from 33 countries. The most frequent clinical manifestations were fever (96%), hepatomegaly (70%), and splenomegaly (58%). Central nervous system dysfunction and hemorrhages were recorded in 35% and 20% of the patients, respectively. Platelet count and liver transaminase, ferritin, lactate dehydrogenase, triglyceride, and d-dimer levels were the sole laboratory biomarkers showing a percentage change of >50% between the pre-MAS visit and MAS onset. Evidence of macrophage hemophagocytosis was found in 60% of the patients who underwent bone marrow aspiration. MAS occurred most frequently in the setting of active underlying disease, in the absence of a specific trigger. Nearly all patients were given corticosteroids, and 61% received cyclosporine. Biologic medications and etoposide were given to 15% and 12% of the patients, respectively. Approximately one-third of the patients required admission to the intensive care unit (ICU), and the mortality rate was 8%.CONCLUSION: This study provides information on the clinical spectrum and current management of systemic JIA-associated MAS through the analysis of a very large patient sample. MAS remains a serious condition, as a sizeable proportion of patients required admission to the ICU or died.

KW - Adrenal Cortex Hormones

KW - Arthritis, Juvenile

KW - Biological Products

KW - Child

KW - Child, Preschool

KW - Cohort Studies

KW - Cyclosporine

KW - Etoposide

KW - Female

KW - Fever

KW - Hepatomegaly

KW - Humans

KW - Intensive Care Units

KW - International Cooperation

KW - Macrophage Activation Syndrome

KW - Male

KW - Prevalence

KW - Retrospective Studies

KW - Splenomegaly

KW - Survival Rate

KW - Treatment Outcome

U2 - 10.1002/art.38802

DO - 10.1002/art.38802

M3 - SCORING: Journal article

C2 - 25077692

VL - 66

SP - 3160

EP - 3169

JO - ARTHRITIS RHEUMATOL

JF - ARTHRITIS RHEUMATOL

SN - 2326-5191

IS - 11

ER -