哮喘严重程度评分系统（ASSESS）的开发和初步验证
2019/11/12
摘要
背景：量化哮喘严重程度的工具是有限的。
目的：为了开发一种连续的适用于青少年和成人的哮喘严重程度测量方法，即哮喘严重程度评分系统（ASSESS），其中包括哮喘控制，肺功能，药物和急性发作等方面。
方法：使用了严重哮喘研究计划（SARP，NCT01606826）的第3阶段参与者的基线和36个月的纵向数据。确定了量表属性，反应性和最小重要差异（MID）。在SARP 1/2期的参与者中进行外部复制。 在接受肌注曲安奈德进行皮质类固醇反应性试验的受试者和接受生物制剂的受试者中，探索了ASSESS用于检测治疗反应的效用。
结果：ASSESS得分范围为0到20（8.78±3.9；得分越高表示严重程度越严重），并且在5个表型组之间有所不同。测量性质是可以接受的。ASSESS能够反应生活质量的变化，MID为2，对哮喘改善和恶化的预后具有很好的特异性，但敏感性较差。复制分析产生相似的结果，下降2分（改善）与生活质量的改善相关。 曲安奈德治疗后，ASSESS评分降低≥2分（改善）的受试者在肺功能和哮喘控制方面也有较大改善，但这些差异仅限于3、4和5表型。接受生物制剂治疗的受试者也降低了≥2分（ 总体而言）。
结论：ASSESS工具是一种可能在流行病学和临床研究中用于量化单个患者和表型组治疗反应的客观测量方法。但是，验证研究仍待需要。
Development and Initial Validation of the Asthma Severity Scoring System (ASSESS).
Fitzpatrick AM, Szefler SJ, Mauger DT, Phillips BR, Denlinger LC, Moore WC, Sorkness RL, Wenzel SE, Gergen PJ, Bleecker ER, Castro M, Erzurum SC, Fahy JV, Gaston BM, Israel E, Levy BD, Meyers DA, Teague WG, Bacharier LB, Ly NP, Phipatanakul W, Ross KR, Zein J, Jarjour NN.
Abstract
BACKGROUND:Tools for quantification of asthma severity are limited.
OBJECTIVE:To develop a continuous measure of asthma severity, the Asthma Severity Scoring System (ASSESS), for adolescents and adults incorporating domains of asthma control, lung function, medications, and exacerbations.
METHODS:Baseline and 36-month longitudinal data from participants in Phase 3 of the Severe Asthma Research Program (SARP, NCT01606826) were utilized. Scale properties, responsiveness, and a minimal important difference (MID) were determined. External replication was performed in participants enrolled in SARP Phase 1/2. Utility of ASSESS for detecting treatment response was explored in participants undergoing corticosteroid responsiveness testing with intramuscular triamcinolone and participants receiving biologics.
RESULTS:ASSESS scores ranged from 0 to 20 (8.78 ± 3.9; higher scores reflect worse severity) and differed between 5 phenotypic groups. Measurement properties were acceptable. ASSESS was responsive to changes in quality of life with a MID of 2, with good specificity for outcomes of asthma improvement and worsening, but poor sensitivity. Replication analyses yielded similar results, with a 2-point decrease (improvement) associated with improvements in quality of life. Participants with ≥2 point decrease (improvement) in ASSESS scores also had greater improvement in lung function and asthma control after triamcinolone, but these differences were limited to phenotypic clusters 3, 4 and 5. Participants treated with biologics also had ≥2 point decrease (improvement) in ASSESS scores overall.
CONCLUSIONS:The ASSESS tool is an objective measure that may be useful in epidemiologic and clinical research studies for quantification of treatment response in individual patients and in phenotypic groups. However, validation studies are warranted.
背景：量化哮喘严重程度的工具是有限的。
目的：为了开发一种连续的适用于青少年和成人的哮喘严重程度测量方法，即哮喘严重程度评分系统（ASSESS），其中包括哮喘控制，肺功能，药物和急性发作等方面。
方法：使用了严重哮喘研究计划（SARP，NCT01606826）的第3阶段参与者的基线和36个月的纵向数据。确定了量表属性，反应性和最小重要差异（MID）。在SARP 1/2期的参与者中进行外部复制。 在接受肌注曲安奈德进行皮质类固醇反应性试验的受试者和接受生物制剂的受试者中，探索了ASSESS用于检测治疗反应的效用。
结果：ASSESS得分范围为0到20（8.78±3.9；得分越高表示严重程度越严重），并且在5个表型组之间有所不同。测量性质是可以接受的。ASSESS能够反应生活质量的变化，MID为2，对哮喘改善和恶化的预后具有很好的特异性，但敏感性较差。复制分析产生相似的结果，下降2分（改善）与生活质量的改善相关。 曲安奈德治疗后，ASSESS评分降低≥2分（改善）的受试者在肺功能和哮喘控制方面也有较大改善，但这些差异仅限于3、4和5表型。接受生物制剂治疗的受试者也降低了≥2分（ 总体而言）。
结论：ASSESS工具是一种可能在流行病学和临床研究中用于量化单个患者和表型组治疗反应的客观测量方法。但是，验证研究仍待需要。
(中日友好医院呼吸与危重症医学科 王瑞茵 摘译 林江涛 审校)
(J Allergy Clin Immunol. 2019 Oct 8. pii: S0091-6749(19)31254-0. doi: 10.1016/j.jaci.2019.09.018. [Epub ahead of print] )
(J Allergy Clin Immunol. 2019 Oct 8. pii: S0091-6749(19)31254-0. doi: 10.1016/j.jaci.2019.09.018. [Epub ahead of print] )
Development and Initial Validation of the Asthma Severity Scoring System (ASSESS).
Fitzpatrick AM, Szefler SJ, Mauger DT, Phillips BR, Denlinger LC, Moore WC, Sorkness RL, Wenzel SE, Gergen PJ, Bleecker ER, Castro M, Erzurum SC, Fahy JV, Gaston BM, Israel E, Levy BD, Meyers DA, Teague WG, Bacharier LB, Ly NP, Phipatanakul W, Ross KR, Zein J, Jarjour NN.
Abstract
BACKGROUND:Tools for quantification of asthma severity are limited.
OBJECTIVE:To develop a continuous measure of asthma severity, the Asthma Severity Scoring System (ASSESS), for adolescents and adults incorporating domains of asthma control, lung function, medications, and exacerbations.
METHODS:Baseline and 36-month longitudinal data from participants in Phase 3 of the Severe Asthma Research Program (SARP, NCT01606826) were utilized. Scale properties, responsiveness, and a minimal important difference (MID) were determined. External replication was performed in participants enrolled in SARP Phase 1/2. Utility of ASSESS for detecting treatment response was explored in participants undergoing corticosteroid responsiveness testing with intramuscular triamcinolone and participants receiving biologics.
RESULTS:ASSESS scores ranged from 0 to 20 (8.78 ± 3.9; higher scores reflect worse severity) and differed between 5 phenotypic groups. Measurement properties were acceptable. ASSESS was responsive to changes in quality of life with a MID of 2, with good specificity for outcomes of asthma improvement and worsening, but poor sensitivity. Replication analyses yielded similar results, with a 2-point decrease (improvement) associated with improvements in quality of life. Participants with ≥2 point decrease (improvement) in ASSESS scores also had greater improvement in lung function and asthma control after triamcinolone, but these differences were limited to phenotypic clusters 3, 4 and 5. Participants treated with biologics also had ≥2 point decrease (improvement) in ASSESS scores overall.
CONCLUSIONS:The ASSESS tool is an objective measure that may be useful in epidemiologic and clinical research studies for quantification of treatment response in individual patients and in phenotypic groups. However, validation studies are warranted.
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哮喘和过敏的系统生物学和大数据——最新发现和新出现的挑战
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Transgelin-2在癌症和哮喘中的生化和临床意义
