Baumrin Emily, Cronholm Peter F, Kearney Matthew D, Mengesha Mlka, Cesar Laura G, Keddem Shimrit, Schapira Marilyn M, Lee Stephanie J, Loren Alison W, Gelfand Joel M
Department of Dermatology, University of Pennsylvania, Philadelphia.
Department of Family Medicine and Community Health, University of Pennsylvania, Philadelphia.
JAMA Dermatol. 2025 Mar 1;161(3):281-290. doi: 10.1001/jamadermatol.2024.5380.
Cutaneous chronic graft-vs-host disease (GVHD) is independently associated with morbidity and mortality after allogeneic hematopoietic cell transplant. However, the health-related quality-of-life (HRQOL) domains that are most important to patients are poorly understood.
To perform a concept elicitation study to define HRQOL in cutaneous chronic GVHD from the patient perspective and to compare experiences of patients with epidermal vs sclerotic disease.
DESIGN, SETTING, AND PARTICIPANTS: A single-center qualitative analysis from open-ended, semistructured interviews and free-listing terms conducted between April and September 2023. Participants were 18 years or older with a diagnosis of active cutaneous chronic GVHD, purposefully sampled for epidermal and sclerotic disease features, with ongoing sampling until thematic saturation.
HRQOL domains and codes from patient perspectives of living with cutaneous chronic GVHD were identified by inductive analysis of semistructured interviews. Smith salience index (Smith S) score, a measure of saliency for each list term, was calculated from free-listing terms from deidentified patient interviews.
A total of 31 adults with cutaneous chronic GVHD (median [IQR] age, 61.1 [52.9-68.7] years) participated in interviews; 17 participants (54.8%) were male and 14 (45.2%) were female. Nine participants (29.0%) had epidermal, 13 (41.9%) sclerotic, and 9 (29.0%) a combination of disease types. The study identified 40 codes of importance grouped within 5 HRQOL domains: skin changes and symptoms, social functioning, psychological and emotional functioning, physical functioning, and general health perceptions. The most frequent symptoms were dry skin (n = 20 [65%]), tight skin (n = 19 [61%]), itch (n = 15 [48%]), and discoloration (n = 14 [45%]), which were seen in all disease subtypes. Impairment in social functioning was noted by all participants. Psychological and emotional functioning, including frustration (Smith S score, 0.32) and worry or concern (Smith S score, 0.12), and symptoms including discomfort (Smith S score, 0.20) were the most salient to patients. Individual and environmental factors, such as social comparison, illness comparison with cancer, anatomic location of disease involvement, and disease duration, affected the relationship between skin changes and symptoms and downstream functioning and general health perceptions.
This qualitative analysis demonstrated the direct relationship between cutaneous chronic GVHD and HRQOL domains and identified codes not represented in existing GVHD- and dermatology-specific patient-reported outcome measures. These results can guide patient-reported outcome development and instrument selection for clinical trials and improve clinical decision-making.
皮肤慢性移植物抗宿主病（GVHD）与异基因造血细胞移植后的发病率和死亡率独立相关。然而，对于患者而言最重要的健康相关生活质量（HRQOL）领域却鲜为人知。
开展一项概念引发研究，从患者角度定义皮肤慢性GVHD中的HRQOL，并比较表皮疾病与硬化性疾病患者的经历。
设计、背景和参与者：2023年4月至9月间进行的一项单中心定性分析，采用开放式、半结构化访谈以及自由列举术语的方式。参与者年龄在18岁及以上，诊断为活动性皮肤慢性GVHD，根据表皮和硬化性疾病特征进行有目的抽样，持续抽样直至主题饱和。
通过对半结构化访谈的归纳分析，从患者角度确定了皮肤慢性GVHD患者生活中的HRQOL领域和编码。根据去识别化患者访谈中的自由列举术语计算了史密斯显著性指数（Smith S）得分，该得分用于衡量每个列表术语的显著性。
共有31名患有皮肤慢性GVHD的成年人（年龄中位数[四分位间距]为61.1[52.9 - 68.7]岁）参与了访谈；17名参与者（54.8%）为男性，14名（45.2%）为女性。9名参与者（29.0%）患有表皮疾病，13名（41.9%）患有硬化性疾病，9名（29.0%）患有两种疾病类型的组合。该研究确定了40个重要编码，分为5个HRQOL领域：皮肤变化和症状、社会功能、心理和情感功能、身体功能以及总体健康认知。最常见症状为皮肤干燥（n = 20[65%]）、皮肤紧绷（n = 19[61%]）、瘙痒（n = 15[48%]）和色素沉着（n = 14[45%]），所有疾病亚型均有出现。所有参与者均指出社会功能受损。心理和情感功能，包括沮丧（Smith S得分0.32）和担忧（Smith S得分0.12），以及包括不适（Smith S得分0.
