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Epidermolizis bülloza hastalarında tedavi memnuniyeti: demografik ve klinik faktörlerin etkisi

Yıl 2025, Cilt: 16 Sayı: 2, 246 - 254, 30.06.2025

Abdulkadir Calavul

https://doi.org/10.18663/tjcl.1624016

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

  • El Hachem M, Zambruno G, Bourdon-Lanoy E, Ciasulli A, Buisson C, Hadj-Rabia S et al. Multicentre consensus recommendations for skin care in inherited epidermolysis bullosa. Orphanet J Rare Dis 2014; 9: 76.
  • Pânzaru MC, Caba L, Florea L, Braha EE, Gorduza EV. Epidermolysis bullosa-a different genetic approach in correlation with genetic heterogeneity. Diagnostics (Basel) 2022; 12: 1325.
  • Fine JD, Bruckner-Tuderman L, Eady RA, Bauer EA, Bauer JW, Has C et al. Inherited epidermolysis bullosa: updated recommendations on diagnosis and classification. J Am Acad Dermatol 2014; 70: 1103-1126.
  • Siprashvili Z, Nguyen NT, Gorell ES, Loutit K, Khuu P, Furukawa LK et al. Safety and wound outcomes following genetically corrected autologous epidermal grafts in patients with recessive dystrophic epidermolysis bullosa. JAMA 2016; 316: 1808-17.
  • Popenhagen MP, Genovese P, Blishen M, Rajapakse D, Diem A, King A et al. Consensus-based guidelines for the provision of palliative and end-of-life care for people living with epidermolysis bullosa. Orphanet J Rare Dis 2023; 18: 268.
  • Has C, Bauer JW, Bodemer C, Bolling MC, Bruckner-Tuderman L, Diem A et al. Consensus reclassification of inherited epidermolysis bullosa and other disorders with skin fragility. Br J Dermatol 2020; 183: 614-27.
  • Jeon K, On HR, Kim SC. Quality of life and economic burden in recessive dystrophic epidermolysis bullosa. Ann Dermatol 2016; 28: 6-14.
  • Bruckner AL, Losow M, Wisk J, Patel N, Reha A, Lagast H et al. The challenges of living with and managing epidermolysis bullosa: insights from patients and caregivers. Orphanet J Rare Dis 2020; 15: 1.
  • Nita M, Pliszczyński J, Kosieradzki M, Fiedor P. Review of the latest methods of epidermolysis bullosa and other chronic wounds treatment including BIOOPA dressing. Dermatol Ther (Heidelb) 2021; 11: 1469-80.
  • Rashidghamat E, McGrath JA. Novel and emerging therapies in the treatment of recessive dystrophic epidermolysis bullosa. Intractable Rare Dis Res 2017; 6: 6-20.
  • Taberna M, Gil Moncayo F, Jané-Salas E, Antonio M, Arribas L, Vilajosana E et al. The Multidisciplinary Team (MDT) Approach and Quality of Care. Front Oncol 2020; 10: 85.
  • Polizzi A, Santonocito S, Patini R, Quinzi V, Mummolo S, Leonardi R et al. Oral alterations in heritable epidermolysis bullosa: a clinical study and literature review. Biomed Res Int 2022; 2022: 6493156.
  • Thien CI, Bessa VR, Miotto IZ, Samorano LP, Rivitti-Machado MC, Oliveira ZNP. Hereditary epidermolysis bullosa: clinical-epidemiological profile of 278 patients at a tertiary hospital in São Paulo, Brazil. Ann Bras Dermatol 2024; 99: 380-90.
  • Retrosi C, Diociaiuti A, De Ranieri C, Corbeddu M, Carnevale C, Giancristoforo S et al. Multidisciplinary care for patients with epidermolysis bullosa from birth to adolescence: experience of one Italian reference center. Ital J Pediatr 2022; 48: 58.
  • Bishnoi A, Manjunath S, Kishore K, De D, Handa S, Murrell DF, Mahajan R. Hindi translation and validation of quality of life score in Indian patients with epidermolysis bullosa. Indian J Dermatol Venereol Leprol 2022; 88: 177-83.
  • Tang JY, Marinkovich MP, Lucas E, Gorell E, Chiou A, Lu Y et al. A systematic literature review of the disease burden in patients with recessive dystrophic epidermolysis bullosa. Orphanet J Rare Dis 2021; 16: 175.
  • Rogers CL, Gibson M, Kern JS, Martin LK, Robertson SJ, Daniel BS et al. A comparison study of outcome measures for epidermolysis bullosa: Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) and the Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB). JAAD Int 2021; 2: 134-52.
  • Akiyama M, Takeichi T, Ikeda S, Ishiko A, Kurosawa M, Murota H et al. Recent advances in clinical research on rare intractable hereditary skin diseases in Japan. Keio J Med 2023; 72: 1-10.
  • Prodinger B, Bauer JW, Laimer M. Translational perspectives to treat epidermolysis bullosa—where do we stand? Exp Dermatol 2020; 29: 1112-1122.
  • Maseda R, Martínez-Santamaría L, Sacedón R, Butta N, de Arriba MDC, García-Barcenilla S et al. Beneficial effect of systemic allogeneic adipose-derived mesenchymal cells on the clinical, inflammatory, and immunologic status of a patient with recessive dystrophic epidermolysis bullosa: a case report. Front Med (Lausanne) 2020; 7: 576558.
  • Behkar A, Garmaroudi G, Nasimi M, Yousefi S, Khosravi H, Kianfar N et al. Assessing quality of life in patients with autoimmune bullous diseases using the Persian version of Treatment of Autoimmune Bullous Disease Quality of Life questionnaire finds similar effects in women as men. Int J Womens Dermatol 2022; 8: e004.
  • Kearney S, Donohoe A, McAuliffe E. Living with epidermolysis bullosa: daily challenges and healthcare needs. Health Expect 2020; 23: 368-376.
  • Pavić J, Krznar M, Čukljek S, Sedić B, Ozimec Vulinec Š, Kovačević I. The association between healthcare satisfaction and social support and stress, depression, and life satisfaction in female caregivers. Int J Environ Res Public Health 2024; 21: 1245.
  • Alheggi A, Alfahhad A, Bukhari A, Bodemer C. Exploring the impact of epidermolysis bullosa on parents and caregivers: a cross-cultural validation of the EB burden of disease questionnaire. Clin Cosmet Investig Dermatol 2024; 17: 1027-32.
  • Menekşe S, Yılmaz A, Seyfettinoğlu F. Single-center experience of surgical treatment of subjects with late-presenting developmental dysplasia of the hip. Genel Tip Derg 2024; 34: 79-85.
  • Martin K, Geuens S, Asche JK, Bodan R, Browne F, Downe A et al. Psychosocial recommendations for the care of children and adults with epidermolysis bullosa and their family: evidence-based guidelines. Orphanet J Rare Dis 2019; 14: 133.
  • Angelis A, Kanavos P, López-Bastida J, Linertová R, Oliva-Moreno J, Serrano-Aguilar P et al. Social/economic costs and health-related quality of life in patients with epidermolysis bullosa in Europe. Eur J Health Econ 2016; 17(Suppl 1): 31-42.
  • Chateau AV, Dlova NC, Mosam A. The impact of epidermolysis bullosa on the family and healthcare practitioners: a scoping review. Int J Dermatol 2023; 62: 1189-97.
  • Sangha N, Pope E, Tawil J, Sauro K, Williams K. Psychosocial impact of epidermolysis bullosa on patients: a qualitative study. Pediatr Dermatol 2021; 38: 931-6.

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

Yıl 2025, Cilt: 16 Sayı: 2, 246 - 254, 30.06.2025

Abdulkadir Calavul

https://doi.org/10.18663/tjcl.1624016

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

Etik Beyan

This study was approved by Eskişehir City Health Practice and Research Center Institutional Ethics Committee with protocol number (Date: 17/10/2024, No: ESH/BAEK 2024/50).

Destekleyen Kurum

The authors received no financial support for the research and/or authorship of this article.

Kaynakça

  • El Hachem M, Zambruno G, Bourdon-Lanoy E, Ciasulli A, Buisson C, Hadj-Rabia S et al. Multicentre consensus recommendations for skin care in inherited epidermolysis bullosa. Orphanet J Rare Dis 2014; 9: 76.
  • Pânzaru MC, Caba L, Florea L, Braha EE, Gorduza EV. Epidermolysis bullosa-a different genetic approach in correlation with genetic heterogeneity. Diagnostics (Basel) 2022; 12: 1325.
  • Fine JD, Bruckner-Tuderman L, Eady RA, Bauer EA, Bauer JW, Has C et al. Inherited epidermolysis bullosa: updated recommendations on diagnosis and classification. J Am Acad Dermatol 2014; 70: 1103-1126.
  • Siprashvili Z, Nguyen NT, Gorell ES, Loutit K, Khuu P, Furukawa LK et al. Safety and wound outcomes following genetically corrected autologous epidermal grafts in patients with recessive dystrophic epidermolysis bullosa. JAMA 2016; 316: 1808-17.
  • Popenhagen MP, Genovese P, Blishen M, Rajapakse D, Diem A, King A et al. Consensus-based guidelines for the provision of palliative and end-of-life care for people living with epidermolysis bullosa. Orphanet J Rare Dis 2023; 18: 268.
  • Has C, Bauer JW, Bodemer C, Bolling MC, Bruckner-Tuderman L, Diem A et al. Consensus reclassification of inherited epidermolysis bullosa and other disorders with skin fragility. Br J Dermatol 2020; 183: 614-27.
  • Jeon K, On HR, Kim SC. Quality of life and economic burden in recessive dystrophic epidermolysis bullosa. Ann Dermatol 2016; 28: 6-14.
  • Bruckner AL, Losow M, Wisk J, Patel N, Reha A, Lagast H et al. The challenges of living with and managing epidermolysis bullosa: insights from patients and caregivers. Orphanet J Rare Dis 2020; 15: 1.
  • Nita M, Pliszczyński J, Kosieradzki M, Fiedor P. Review of the latest methods of epidermolysis bullosa and other chronic wounds treatment including BIOOPA dressing. Dermatol Ther (Heidelb) 2021; 11: 1469-80.
  • Rashidghamat E, McGrath JA. Novel and emerging therapies in the treatment of recessive dystrophic epidermolysis bullosa. Intractable Rare Dis Res 2017; 6: 6-20.
  • Taberna M, Gil Moncayo F, Jané-Salas E, Antonio M, Arribas L, Vilajosana E et al. The Multidisciplinary Team (MDT) Approach and Quality of Care. Front Oncol 2020; 10: 85.
  • Polizzi A, Santonocito S, Patini R, Quinzi V, Mummolo S, Leonardi R et al. Oral alterations in heritable epidermolysis bullosa: a clinical study and literature review. Biomed Res Int 2022; 2022: 6493156.
  • Thien CI, Bessa VR, Miotto IZ, Samorano LP, Rivitti-Machado MC, Oliveira ZNP. Hereditary epidermolysis bullosa: clinical-epidemiological profile of 278 patients at a tertiary hospital in São Paulo, Brazil. Ann Bras Dermatol 2024; 99: 380-90.
  • Retrosi C, Diociaiuti A, De Ranieri C, Corbeddu M, Carnevale C, Giancristoforo S et al. Multidisciplinary care for patients with epidermolysis bullosa from birth to adolescence: experience of one Italian reference center. Ital J Pediatr 2022; 48: 58.
  • Bishnoi A, Manjunath S, Kishore K, De D, Handa S, Murrell DF, Mahajan R. Hindi translation and validation of quality of life score in Indian patients with epidermolysis bullosa. Indian J Dermatol Venereol Leprol 2022; 88: 177-83.
  • Tang JY, Marinkovich MP, Lucas E, Gorell E, Chiou A, Lu Y et al. A systematic literature review of the disease burden in patients with recessive dystrophic epidermolysis bullosa. Orphanet J Rare Dis 2021; 16: 175.
  • Rogers CL, Gibson M, Kern JS, Martin LK, Robertson SJ, Daniel BS et al. A comparison study of outcome measures for epidermolysis bullosa: Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) and the Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB). JAAD Int 2021; 2: 134-52.
  • Akiyama M, Takeichi T, Ikeda S, Ishiko A, Kurosawa M, Murota H et al. Recent advances in clinical research on rare intractable hereditary skin diseases in Japan. Keio J Med 2023; 72: 1-10.
  • Prodinger B, Bauer JW, Laimer M. Translational perspectives to treat epidermolysis bullosa—where do we stand? Exp Dermatol 2020; 29: 1112-1122.
  • Maseda R, Martínez-Santamaría L, Sacedón R, Butta N, de Arriba MDC, García-Barcenilla S et al. Beneficial effect of systemic allogeneic adipose-derived mesenchymal cells on the clinical, inflammatory, and immunologic status of a patient with recessive dystrophic epidermolysis bullosa: a case report. Front Med (Lausanne) 2020; 7: 576558.
  • Behkar A, Garmaroudi G, Nasimi M, Yousefi S, Khosravi H, Kianfar N et al. Assessing quality of life in patients with autoimmune bullous diseases using the Persian version of Treatment of Autoimmune Bullous Disease Quality of Life questionnaire finds similar effects in women as men. Int J Womens Dermatol 2022; 8: e004.
  • Kearney S, Donohoe A, McAuliffe E. Living with epidermolysis bullosa: daily challenges and healthcare needs. Health Expect 2020; 23: 368-376.
  • Pavić J, Krznar M, Čukljek S, Sedić B, Ozimec Vulinec Š, Kovačević I. The association between healthcare satisfaction and social support and stress, depression, and life satisfaction in female caregivers. Int J Environ Res Public Health 2024; 21: 1245.
  • Alheggi A, Alfahhad A, Bukhari A, Bodemer C. Exploring the impact of epidermolysis bullosa on parents and caregivers: a cross-cultural validation of the EB burden of disease questionnaire. Clin Cosmet Investig Dermatol 2024; 17: 1027-32.
  • Menekşe S, Yılmaz A, Seyfettinoğlu F. Single-center experience of surgical treatment of subjects with late-presenting developmental dysplasia of the hip. Genel Tip Derg 2024; 34: 79-85.
  • Martin K, Geuens S, Asche JK, Bodan R, Browne F, Downe A et al. Psychosocial recommendations for the care of children and adults with epidermolysis bullosa and their family: evidence-based guidelines. Orphanet J Rare Dis 2019; 14: 133.
  • Angelis A, Kanavos P, López-Bastida J, Linertová R, Oliva-Moreno J, Serrano-Aguilar P et al. Social/economic costs and health-related quality of life in patients with epidermolysis bullosa in Europe. Eur J Health Econ 2016; 17(Suppl 1): 31-42.
  • Chateau AV, Dlova NC, Mosam A. The impact of epidermolysis bullosa on the family and healthcare practitioners: a scoping review. Int J Dermatol 2023; 62: 1189-97.
  • Sangha N, Pope E, Tawil J, Sauro K, Williams K. Psychosocial impact of epidermolysis bullosa on patients: a qualitative study. Pediatr Dermatol 2021; 38: 931-6.
Toplam 29 adet kaynakça vardır.

Ayrıntılar

Birincil Dil İngilizce
Konular Plastik, Rekonstrüktif ve Estetik Cerrahi
Bölüm Araştırma Makalesi
Yazarlar

Abdulkadir Calavul 0000-0002-6790-128X

Yayımlanma Tarihi 30 Haziran 2025
Gönderilme Tarihi 20 Ocak 2025
Kabul Tarihi 5 Mayıs 2025
Yayımlandığı Sayı Yıl 2025 Cilt: 16 Sayı: 2

Kaynak Göster

APA Calavul, A. (2025). Treatment satisfaction in erpidermolysis bullosa patients: the impact of demographic and clinical factors. Turkish Journal of Clinics and Laboratory, 16(2), 246-254. https://doi.org/10.18663/tjcl.1624016
AMA Calavul A. Treatment satisfaction in erpidermolysis bullosa patients: the impact of demographic and clinical factors. TJCL. Haziran 2025;16(2):246-254. doi:10.18663/tjcl.1624016
Chicago Calavul, Abdulkadir. “Treatment Satisfaction in Erpidermolysis Bullosa Patients: the Impact of Demographic and Clinical Factors”. Turkish Journal of Clinics and Laboratory 16, sy. 2 (Haziran 2025): 246-54. https://doi.org/10.18663/tjcl.1624016.
EndNote Calavul A (01 Haziran 2025) Treatment satisfaction in erpidermolysis bullosa patients: the impact of demographic and clinical factors. Turkish Journal of Clinics and Laboratory 16 2 246–254.
IEEE A. Calavul, “Treatment satisfaction in erpidermolysis bullosa patients: the impact of demographic and clinical factors”, TJCL, c. 16, sy. 2, ss. 246–254, 2025, doi: 10.18663/tjcl.1624016.
ISNAD Calavul, Abdulkadir. “Treatment Satisfaction in Erpidermolysis Bullosa Patients: the Impact of Demographic and Clinical Factors”. Turkish Journal of Clinics and Laboratory 16/2 (Haziran 2025), 246-254. https://doi.org/10.18663/tjcl.1624016.
JAMA Calavul A. Treatment satisfaction in erpidermolysis bullosa patients: the impact of demographic and clinical factors. TJCL. 2025;16:246–254.
MLA Calavul, Abdulkadir. “Treatment Satisfaction in Erpidermolysis Bullosa Patients: the Impact of Demographic and Clinical Factors”. Turkish Journal of Clinics and Laboratory, c. 16, sy. 2, 2025, ss. 246-54, doi:10.18663/tjcl.1624016.
Vancouver Calavul A. Treatment satisfaction in erpidermolysis bullosa patients: the impact of demographic and clinical factors. TJCL. 2025;16(2):246-54.
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