The Mastocytosis Control Test: A Patient-Reported Outcome Measure Assessing Disease Control

IF 8.2 1区医学 Q1 ALLERGY

Journal of Allergy and Clinical Immunology-In Practice Pub Date : 2025-03-01 DOI:10.1016/j.jaip.2024.11.021

Frank Siebenhaar MD , Senan Sofi MD , Sophia Neisinger MD , Cem Akin MD, PhD , Polina Pyatilova MD , Eva Grekowitz MD , Antje Haendel , Tomasz Hawro MD , Lea Kiefer MD , Markus Magerl MD , Martin Metz MD , Marcus Maurer MD , Karsten Weller MD

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引用次数: 0

Abstract

Background

Mastocytosis is characterized by expanding neoplastic mast cells in organs such as the skin, bone marrow, and gastrointestinal tract. The release of mast cell mediators triggers cutaneous, gastrointestinal, and other symptoms. Currently, no validated mastocytosis-specific patient-reported outcome measure (PROM) exists to assess disease control.

Objective

Here, we developed a disease-specific instrument, the Mastocytosis Control Test (MCT), for evaluating and monitoring disease control in patients with nonadvanced disease.

Methods

Six potential MCT items were generated using a combined approach consisting of a literature review, patient interviews, and expert input. Item selection and reduction were performed by impact analysis and interitem correlation, followed by expert reviews and cognitive debriefings. In a validation study, the resulting MCT was tested for validity and reliability by assessing internal consistency, test-retest reliability, convergent validity, known-groups validity, and receiver operating characteristics (ROC) analysis.

Results

Ten patients participated in the item generation phase and 101 in the item reduction and validation phase. The final MCT consisted of 5 items. Our methods showed a valid total score, high internal consistency, and test-retest reliability. Convergent and known-groups validity demonstrated a strong correlation with related anchors. The ROC curve analysis suggested a cutoff value of ≥13 points to identify patients with good disease control.

Conclusions

The MCT is a disease-specific, valid, and reliable PROM for adult patients with nonadvanced disease. It may measure and monitor disease control in routine daily practice and clinical trials.

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肥大细胞增多症控制试验：一种评估疾病控制的病人报告的结果测量方法。

背景：肥大细胞增多症的特点是肿瘤肥大细胞在皮肤、骨髓和胃肠道等器官中扩大。肥大细胞介质的释放引发皮肤、胃肠和其他症状。目前，没有经过验证的肥大细胞增多症特异性患者报告的结果测量（PROM）存在，以评估疾病控制。目的：在这里，我们开发了一种疾病特异性仪器，肥大细胞增多症控制试验（MCT），用于评估和监测非晚期疾病患者的疾病控制。方法：采用文献综述、患者访谈和专家意见相结合的方法生成六个潜在的MCT项目。通过影响分析和项目间关联进行项目选择和缩减，然后进行专家评审和认知汇报。在一项验证研究中，通过评估内部一致性、测试-重测信度、收敛效度、已知组效度和受试者工作特征（ROC）分析来检验结果MCT的效度和信度。结果：10例患者参加了项目生成阶段，101例患者参加了项目减少和验证阶段。最后的MCT包括五个项目。我们的方法显示出有效的总分、较高的内部一致性和重测信度。收敛效度和已知组效度与相关锚点有很强的相关性。ROC曲线分析建议截断值≥13点为疾病控制良好的患者。结论：MCT是一种疾病特异性的、有效的、可靠的非晚期成年患者PROM。可在日常实践和临床试验中测量和监测疾病控制情况。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Allergy and Clinical Immunology-In Practice ALLERGYIMMUNOLOGY-IMMUNOLOGY

CiteScore

11.10

自引率

9.60%

发文量

683

审稿时长

50 days

期刊介绍： JACI: In Practice is an official publication of the American Academy of Allergy, Asthma & Immunology (AAAAI). It is a companion title to The Journal of Allergy and Clinical Immunology, and it aims to provide timely clinical papers, case reports, and management recommendations to clinical allergists and other physicians dealing with allergic and immunologic diseases in their practice. The mission of JACI: In Practice is to offer valid and impactful information that supports evidence-based clinical decisions in the diagnosis and management of asthma, allergies, immunologic conditions, and related diseases. This journal publishes articles on various conditions treated by allergist-immunologists, including food allergy, respiratory disorders (such as asthma, rhinitis, nasal polyps, sinusitis, cough, ABPA, and hypersensitivity pneumonitis), drug allergy, insect sting allergy, anaphylaxis, dermatologic disorders (such as atopic dermatitis, contact dermatitis, urticaria, angioedema, and HAE), immunodeficiency, autoinflammatory syndromes, eosinophilic disorders, and mast cell disorders. The focus of the journal is on providing cutting-edge clinical information that practitioners can use in their everyday practice or to acquire new knowledge and skills for the benefit of their patients. However, mechanistic or translational studies without immediate or near future clinical relevance, as well as animal studies, are not within the scope of the journal.