Treatment satisfaction in erpidermolysis bullosa patients: the impact of demographic and clinical factors

Öz

Aim: Epidermolysis bullosa (EB) is a hereditary disease which causes skin and mucous membrane blistering. While both standard and multidisciplinary care approaches exist for managing EB, the impact of care type on treatment outcomes remains incompletely understood, particularly across different EB subtypes. To compare treatment satisfaction and quality of life outcomes between standard versus multidisciplinary care approaches in pediatric EB patients, and to identify key demographic and clinical factors influencing these outcomes. Material and Methods: This retrospective study evaluated 32 pediatric EB patients (age <16 years) receiving either multidisciplinary care (n = 18) or standard care (n = 14). Multidisciplinary care involved coordinated management by dermatologists, wound care specialists, pain management experts, psychologists and dedicated nurses, while standard care consisted of routine outpatient follow-up. Treatment outcomes were assessed using the validated Epidermolysis Bullosa Quality of Life (EB-QoL) scale at baseline and 6 months. Statistical analysis included repeated measures ANOVA, independent t-tests, and multiple regression analysis, with Levene's test confirming variance homogeneity. Results: While baseline EB-QoL scores were comparable (44.8 ± 8.1 vs 45.2 ± 7.8, p = 0.876), the multidisciplinary care group showed significantly higher scores at 6 months (68.4 ± 9.2 vs 52.3 ± 8.7, p = 0.003). The magnitude of improvement varied by EB subtype, with Simplex patients showing the largest gains (baseline: 60.4 ± 7.2, 6-month: 71.2 ± 8.4) and Dystrophic patients the smallest (baseline: 38.6 ± 6.8, 6-month: 45.3 ± 7.8). Multiple regression analysis identified age (β = 0.324), BMI (β = 0.195), and multidisciplinary care (β = 0.468) as positive predictors of satisfaction, while disease duration (β = -0.286) and comorbidities (β = -0.245) had negative effects. Conclusions: Multidisciplinary approaches to pediatric EB patients benefited from comprehensive care models. These results banner the magnitude of benefit, which relies heavily on skeletal structure. The severity of treatment outcomes was noticeably improved through the effect of structured multidisciplinary care. Each sub-type of EB affliction had improved treatment results but each diverged in the level of gain, which further enhances the need for individual tailored treatment protocols based on EB subtype classification and other parameters.

Anahtar Kelimeler

• Epidermolysis Bullosa
• Pediatric Patients
• Multidisciplinary Care
• Quality of Life
• Psychosocial Support

Epidermolizis bülloza hastalarında tedavi memnuniyeti: demografik ve klinik faktörlerin etkisi

Öz

Amaç: Epidermolizis bülloza (EB), deri ve mukoza zarında büllere neden olan kalıtsal bir hastalıktır. EB'nin yönetiminde hem standart hem de multidisipliner bakım yaklaşımları mevcut olmakla birlikte, bakım türünün tedavi sonuçları üzerindeki etkisi, özellikle farklı EB alt tipleri arasında tam olarak anlaşılamamıştır. Pediatrik EB hastalarında standart bakım ile multidisipliner bakım yaklaşımları arasındaki tedavi memnuniyeti ve yaşam kalitesi sonuçlarını karşılaştırmak ve bu sonuçları etkileyen temel demografik ve klinik faktörleri belirlemek. Gereç ve Yöntem: Bu retrospektif çalışmada multidisipliner bakım (n = 18) veya standart bakım (n = 14) alan 32 pediatrik EB hastası (yaş <16) değerlendirildi. Multidisipliner bakım; dermatologlar, yara bakım uzmanları, ağrı yönetimi uzmanları, psikologlar ve özel hemşirelerden oluşan koordineli bir ekip tarafından sağlanırken, standart bakım rutin poliklinik takibinden oluşmaktaydı. Tedavi sonuçları, başlangıçta ve 6. ayda valide edilmiş Epidermolizis Bülloza Yaşam Kalitesi (EB-QoL) ölçeği kullanılarak değerlendirildi. İstatistiksel analizde tekrarlı ölçümler ANOVA, bağımsız örneklem t-testleri ve çoklu regresyon analizi kullanıldı, varyans homojenliği Levene testi ile doğrulandı. Bulgular: Başlangıç EB-QoL skorları benzerken (44,8 ± 8,1 vs 45,2 ± 7,8, p = 0,876), multidisipliner bakım grubu 6. ayda anlamlı olarak daha yüksek skorlar gösterdi (68,4 ± 9,2 vs 52,3 ± 8,7, p = 0,003). İyileşme düzeyi EB alt tiplerine göre değişkenlik gösterdi; Simpleks hastalar en yüksek artışı (başlangıç: 60,4 ± 7,2, 6.ay: 71,2 ± 8.4), Distrofik hastalar en düşük artışı (başlangıç: 38,6 ± 6,8, 6.ay: 45,3 ± 7,8) gösterdi. Çoklu regresyon analizinde yaş (β = 0,324), VKİ (β = 0,195) ve multidisipliner bakım (β = 0,468) memnuniyetin pozitif belirleyicileri olarak saptanırken, hastalık süresi (β = -0,286) ve komorbiditeler (β = -0,245) negatif etki gösterdi. Sonuçlar: Bulgularımız, yapılandırılmış multidisipliner bakımın tüm EB alt tiplerinde pediatrik hastaların tedavi sonuçlarını önemli ölçüde iyileştirdiğini, ancak faydanın hastalık şiddetine göre değiştiğini göstermektedir. Bu sonuçlar, EB alt tipi ve hasta özelliklerine göre bireyselleştirilmiş tedavi protokolleri içeren kapsamlı bakım programlarının uygulanmasını desteklemektedir.

Anahtar Kelimeler

• Epidermolizis Bülloza
• Pediatrik Hastalar
• Multidisipliner Bakım
• Yaşam Kalitesi
• Psikososyal Destek

Kaynakça

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