Araştırma Makalesi
BibTex RIS Kaynak Göster

Improving clinical applications of quality of life scores in epidermolysis bullosa: defining clinically significant outcomes in the QOLEB questionnaire

Yıl 2019, Cilt: 2 Sayı: 3, 68 - 75, 29.09.2019
https://doi.org/10.33204/mucosa.598339

Öz

Objective The Quality of Life in Epidermolysis Bullosa (QOLEB) score is an Epidermolysis Bullosa (EB) specific quality of life (QoL) measurement tool. It is a statistically valid and reliable questionnaire which has benefits over generic QoL tools in QoL evaluation in EB. It also has important implications in the evaluation of the clinical efficacy of new interventions in EB. The utility of this score would be increased if the clinical relevance of individual scores and changes in QOLEB scores could be further understood.

Methods In order to achieve this, the minimal clinically important  difference (MCID) was calculated using both anchor-based and distribution-based techniques. Banding techniques were also applied to the QOLEB questionnaire in order to stratify scores into “very mild”, “mild”, “moderate”, “severe” and “very severe” categories.

Results Using these methodologies, the MCID for the QOLEB score was calculated at a 6 point change in QOLEB scores, and the QOLEB bands were calculated as: 0-4 points for ‘very mild’, 5-9 points for ‘mild’, 10-19 points for ‘moderate’, 20-34 points for ‘severe’ and 35-51 points for ‘very severe’ impact on QoL.

Conclusions Calculating the MCID and clinical bands for the QOLEB questionnaire increases the breadth of clinical applications for the QOLEB questionnaire. It now has direct utility in determining the clinical significance of interventions in EB by evaluating changes in QOLEB scores and how they correlate to the MCID and clinical bands.

Kaynakça

  • 1. Frew JW, Martin LK, Nijsten T, Murrell DF. Quality of life evaluation in Epidermolysis Bullosa (EB) through the development of the QOLEB Questionnaire: An EB-Specific quality of life instrument. Br J Dermatol 2009;161:1323-30.
  • 2. Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials 1989;10:407-15.
  • 3. Duipmans JC, Jonkman MF. Interdisciplinary management of epidermolysis bullosa in the public setting: the Netherlands as a model of care. Dermatol Clin 2010;28:383-6.
  • 4. Hongbo Y, Thomas CL, Harrison MA, Salek MA, Finlay AY. Translating the science of quality of life into practice: What do dermatology quality of life index scores mean? J Invest Dermatol 2005;125:659-64.
  • 5. Wagner JE, Ishida-Yamamoto A, McGrath JA, et al. Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. NEJM 2010;363:629-39.
  • 6. Conget P, Rodriguez F, Kramer S, et al. Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa. Cytotherapy 2010;12:429-31.
  • 7. Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidemiology 2003;56:395-407.
  • 8. Metz SM, Wyrwich KW, Babu AN, et al. Validity of patient reported health related quality of life global ratings of change using structural equation modeling. Quality of Life Research 2007;16:1193-202.
  • 9. Barnes ML, Vaidyanathan S, Williamson PA Lipworth BJ. The minimal clinically important difference in allergic rhinitis. Clinical and Experimental Allergy 2009;40:242-50.
  • 10. Crosby RD, Kolotkin RL, Williamsw GR. An integrated method to determine meaningful changed in health related quality of life. J Clin Epidemiol 2004;57:1153-60.
  • 11. Norman GR, Sridhar FG, Guyatt GH, Walter SD. Relation of distribution- and anchor-based approaches in interpretation of changes in health-related quality of life. Med Care 2001;39:1039-47.

Epidermolizis bülloza yaşam kalitesi puanlarının klinik uygulamalarının geliştirilmesi: QOLEB anketinde klinik olarak anlamlı sonuçların tanımlanması

Yıl 2019, Cilt: 2 Sayı: 3, 68 - 75, 29.09.2019
https://doi.org/10.33204/mucosa.598339

Öz

Amaç Epidermolizis Bülloza Yaşam Kalitesi Skorlaması (QOLEB), Epidermolizis Bülloza’ya (EB) spesifik bir yaşam kalitesi (QoL) ölçüm aracıdır. QOLEB, EB’de QoL değerlendirmesinde jenerik QoL araçlarına göre üstünlükleri olan, istatistiksel olarak da geçerli ve güvenilir bir ankettir. Ayrıca, EB’deki yeni müdahalelerin klinik etkinliğinin değerlendirilmesinde önemli etkileri vardır. QOLEB içerisindeki bireysel puanların ve değişikliklerin klinik önemlerinin daha iyi anlaşılması durumunda, bu skorlamanın faydası da arttırılacaktır.

Yöntem Bunu başarmak amacıyla, klinik olarak önemli olan minimum fark (MCID), hem çapa bazlı, hem de dağılım bazlı teknikler kullanılarak hesaplandı. Puanları,“çok hafif”, “hafif”, “orta”, “şiddetli” ve “çok şiddetli” kategorilerine ayırmak için QOLEB anketine bantlama teknikleri de uygulandı. 

Bulgular Bu metodolojileri kullanarak, QOLEB skoru için MCID, QOLEB skorlarında 6 puanlık bir değişiklikle hesaplandı. QOLEB bantları şu şekilde hesaplandı: ‘çok hafif’ için 0-4 puan, ‘hafif’ için 5-9 puan, “orta” için 10-19 puan, “şiddetli” için 20-34 puan ve “çok şiddetli” için 35-51 puan.

Sonuç QOLEB skorlaması için MCID ve klinik bantların hesaplanması, QOLEB anketinin klinik uygulama genişliğini arttırır. Böylece, QOLEB skorlarındaki değişimi ve bunların MCID ve klinik bantlar ile nasıl korelasyon gösterdiğini değerlendirerek EB’deki müdahalelerin klinik önemini belirlemede doğrudan faydalar sağlamaktadır.

Kaynakça

  • 1. Frew JW, Martin LK, Nijsten T, Murrell DF. Quality of life evaluation in Epidermolysis Bullosa (EB) through the development of the QOLEB Questionnaire: An EB-Specific quality of life instrument. Br J Dermatol 2009;161:1323-30.
  • 2. Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials 1989;10:407-15.
  • 3. Duipmans JC, Jonkman MF. Interdisciplinary management of epidermolysis bullosa in the public setting: the Netherlands as a model of care. Dermatol Clin 2010;28:383-6.
  • 4. Hongbo Y, Thomas CL, Harrison MA, Salek MA, Finlay AY. Translating the science of quality of life into practice: What do dermatology quality of life index scores mean? J Invest Dermatol 2005;125:659-64.
  • 5. Wagner JE, Ishida-Yamamoto A, McGrath JA, et al. Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. NEJM 2010;363:629-39.
  • 6. Conget P, Rodriguez F, Kramer S, et al. Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa. Cytotherapy 2010;12:429-31.
  • 7. Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidemiology 2003;56:395-407.
  • 8. Metz SM, Wyrwich KW, Babu AN, et al. Validity of patient reported health related quality of life global ratings of change using structural equation modeling. Quality of Life Research 2007;16:1193-202.
  • 9. Barnes ML, Vaidyanathan S, Williamson PA Lipworth BJ. The minimal clinically important difference in allergic rhinitis. Clinical and Experimental Allergy 2009;40:242-50.
  • 10. Crosby RD, Kolotkin RL, Williamsw GR. An integrated method to determine meaningful changed in health related quality of life. J Clin Epidemiol 2004;57:1153-60.
  • 11. Norman GR, Sridhar FG, Guyatt GH, Walter SD. Relation of distribution- and anchor-based approaches in interpretation of changes in health-related quality of life. Med Care 2001;39:1039-47.
Toplam 11 adet kaynakça vardır.

Ayrıntılar

Birincil Dil İngilizce
Konular Klinik Tıp Bilimleri
Bölüm Original Articles
Yazarlar

John Frew 0000-0001-5042-3632

Dedee F. Murrell Bu kişi benim 0000-0003-2971-0199

Yayımlanma Tarihi 29 Eylül 2019
Yayımlandığı Sayı Yıl 2019 Cilt: 2 Sayı: 3

Kaynak Göster

Vancouver Frew J, Murrell DF. Improving clinical applications of quality of life scores in epidermolysis bullosa: defining clinically significant outcomes in the QOLEB questionnaire. Mucosa. 2019;2(3):68-75.