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Development and Initial Validation of the Macrophage Activation Syndrome/Primary Hemophagocytic Lymphohistiocytosis Score, a Diagnostic Tool that Differentiates Primary Hemophagocytic Lymphohistiocytosis from Macrophage Activation Syndrome

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机构: [1]G Gaslini Inst Children, Genoa, Italy; [2]Univ Genoa, Genoa, Italy; [3]Pediat Hosp Bambino Gesu, Rome, Italy; [4]Univ Med Ctr, Hamburg, Germany; [5]Seattle Childrens Hosp, Seattle, WA USA; [6]Univ Washington, Seattle, WA 98195 USA; [7]Hosp Sick Children, Toronto, ON, Canada; [8]Ricardo Gutierrez Childrens Hosp, Buenos Aires, DF, Argentina; [9]Fudan Univ, Childrens Hosp, Shanghai, Peoples R China; [10]Hosp St Joan de Deu, Barcelona, Spain; [11]Aichi Med Univ, Nagakute, Aichi, Japan; [12]Istanbul Univ, Cerrahpasa Med Sch, Istanbul, Turkey; [13]Oslo Univ Hosp, Rikshosp, Oslo, Norway; [14]La Paz Univ Hosp, Madrid, Spain; [15]Univ Basque Country, Cruces Univ Hosp, BioCruces Hlth Res Inst, Baracaldo, Spain; [16]Univ Naples Federico II, Naples, Italy; [17]Stanford Sch Med, Palo Alto, CA USA; [18]St Petersburg State Pediat Med Univ, St Petersburg, Russia; [19]St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USA; [20]Guangzhou Childrens Hosp, Guangzhou, Guangdong, Peoples R China; [21]Beijing Childrens Hosp, Beijing, Peoples R China; [22]Univ Alabama Birmingham, Birmingham, AL USA; [23]Karolinska Univ Hosp, Stockholm, Sweden; [24]Ist Giannina Gaslini, Pediat Reumatol 2, Via G Gaslini 5, I-16147 Genoa, Italy
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关键词: diagnostic score hemophagocytic syndrome macrophage activation syndrome primary hemophagocytic lymphohistiocytosis

摘要:
Objective To develop and validate a diagnostic score that assists in discriminating primary hemophagocytic lymphohistiocytosis (pHLH) from macrophage activation syndrome (MAS) related to systemic juvenile idiopathic arthritis. Study design The clinical, laboratory, and histopathologic features of 362 patients with MAS and 258 patients with pHLH were collected in a multinational collaborative study. Eighty percent of the population was assessed to develop the score and the remaining 20% constituted the validation sample. Variables that entered the best fitted model of logistic regression were assigned a score, based on their statistical weight. The MAS/HLH (MH) score was made up with the individual scores of selected variables. The cutoff in the MH score that discriminated pHLH from MAS best was calculated by means of receiver operating characteristic curve analysis. Score performance was examined in both developmental and validation samples. Results Six variables composed the MH score: age at onset, neutrophil count, fibrinogen, splenomegaly, platelet count, and hemoglobin. The MH score ranged from 0 to 123, and its median value was 97 (1st-3rd quartile 75123) and 12 (1st-3rd quartile 11-34) in pHLH and MAS, respectively. The probability of a diagnosis of pHLH ranged from < 1% for a score of < 11 to >99% for a score of >= 123. A cutoff value of >= 60 revealed the best performance in discriminating pHLH from MAS. Conclusion The MH score is a powerful tool that may aid practitioners to identify patients who are more likely to have pHLH and, thus, could be prioritized for functional and genetic testing.

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出版当年[2016]版:
大类 | 2 区 医学
小类 | 1 区 儿科
最新[2023]版:
大类 | 2 区 医学
小类 | 1 区 儿科
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出版当年[2015]版:
Q1 PEDIATRICS
最新[2023]版:
Q1 PEDIATRICS

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第一作者机构: [1]G Gaslini Inst Children, Genoa, Italy;
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通讯机构: [1]G Gaslini Inst Children, Genoa, Italy; [24]Ist Giannina Gaslini, Pediat Reumatol 2, Via G Gaslini 5, I-16147 Genoa, Italy
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