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Outcomes research in dermatology
This month’s selected commentary
Outcomes research in dermatology Warren R. Heymann, MD Based on the dialogue ‘‘Outcomes research in dermatology’’ between Suephy C. Chen, MD, and Maurice Thew, MD Dialogues in Dermatology, a monthly audio program from the American Academy of Dermatology, contains discussions between dermatologists on timely topics. Commentaries from Dialogues Editorin-Chief Warren R. Heymann, MD, are provided after each discussion as a topic summary and are provided here as a special service to readers of the Journal of the American Academy of Dermatology. ( J Am Acad Dermatol 2008;58:286-7.)
Measure your health by your sympathy with morning and spring. If there is no response in you to the awakening of nature—if the prospect of an early morning walk does not banish sleep, if the warble of the first bluebird does not thrill you—know that the morning and spring of your life are past. Thus you may feel your pulse. Henry David Thoreau
D
ermatologists continue to struggle in accurately quantifying skin disease, its response to treatment, effect on a patient’s quality of life (QOL), its burden on society, and cost effectiveness issues. Although still in its relative infancy, the discipline of outcomes research in dermatology is maturing and will ultimately have a profound effect on our practices. According to Jacobe et al,1 an outcome is defined as one of the possible results that arise from exposure to a causal factor. Outcomes are also referred to as endpoints or variables. Outcomes are ‘‘broadly divided into four categories: physicianbased, patient reported, economic-based, and technology-based’’. To date, there is no consensus regarding the validity and appropriate use of existing health status measures. Indeed, flawed health measurement scales, or their improper use, may be highly problematic. Efforts to improve outcome research in dermatology are underway. There are many health measurement scales in dermatology, such as SCORAD (SCORing Atopic Dermatitis) for atopic dermatitis, SCORTEN for toxic epidermal necrolysis, and PASI (Psoriasis Area and Severity Index) for psoriasis. Indices listed above quantify the physical findings of disease; other measurements, such as Skindex, assess a patient’s QOL.
The statements and opinions expressed in this commentary are those of the Editor-in-Chief of Dialogues in Dermatology.
286
Chren et al2 reported on a survey they named Skindex—a 61-item, self-administered survey with 8 scales, exploring cognitive effects, social effects, depression, fear, embarrassment, anger, physical discomfort, and physical limitations. The authors found their data valid and reproducible.2 The Dermatology Life Quality Index (DLQI) is a simple, 10-question survey addressing how skin disease is interfering with work, study, leisure, and sexual activity. It also asks if the disease or treatment is socially embarrassing. As stated by McCombs and Chen,3 ‘‘The impact of skin disease on patient QOL is often understated. Although few cutaneous diseases are life threatening, virtually all have the potential to impact QOL.’’ Different scales may be used to measure certain patient preferences, such as time tradeoff or willingness-to-pay.3 Data accrued may be valuable for patients, physicians, and health care policymakers. How do dermatologists currently follow their patients? For example, if I am assessing a patient with subacute cutaneous lupus erythematosus (SCLE), I will inquire if there has been a change in the severity of associated arhralgias, or if treatment such as hydroxychloroquine is improving their symptoms. On physical examination, I will try to determine if there has been a diminution of the intensity of erythema or scale. Although this is the time-honored approach to clinical medicine, it is inadequate in conveying vital information about the severity of lupus or its response to treatment. This problem has been addressed by Albrecht et al4 by developing a scoring index, CLASI, for patients with cutaneous lupus erythematosus. The authors define CLASI as consisting of 2 scores; the first summarizes the activity of the disease, whereas the second is a measure of damage done by the disease. ‘‘Activity
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is scored on the basis of erythema, scale/hypertrophy, mucous membrane involvement, acute hair loss, and non-scarring alopecia. Damage is scored in terms of dyspigmentation and scarring, including scarring alopecia. . . .The scores are calculated by simple addition based on the extent of the symptoms.’’ Associated symptoms such as itch, pain, and fatigue are recorded separately on visual 1 to 10 analog scales by the patients.4 Kreuter et al5 studied 10 patients with SCLE in an open study to evaluate the use of mycophenolate sodium as monotherapy in patients with recalcitrant disease. The CLASI was utilized to assess the patients’ progress. Mycophenolate sodium led to a marked improvement of the skin lesions, resulting in a significant decrease of the CLASI from 10.8 6 6.0 at the beginning to 2.9 6 2.6 at the end of therapy. Clinical improvement was confirmed by the use of ultrasonagraphy and colorimetry. Ferraz et al6 had 71 patients with cutaneous lupus lesions assessed by DLQI and SF-36 (a 36-question short form health survey). The authors demonstrated lower DLQI and SF-36 scores for patients with active disease compared to inactive disease, or patients with alopecia compared to those without alopecia.6 These studies underscore the potential for quantification of clinical impressions. Precise data may allow for more rational clinical decisions. The immediate challenge of outcome research in dermatology is to develop reproducible, valid, straightforward, brief scoring systems for use by clinicians, academicians, and health policy administrators. Finlay7 offers the following criteria to be met for any clinical scoring system: 1) the method should be simple enough to use in a busy clinical setting; 2) the method should clearly separate scores derived from the observer and from the patient; 3) the signs chosen to be recorded should be amenable to change and should be unambiguous in their meaning and proven to be so; 4) recording of area of involvement should be based on an assessment of the site of involvement rather than on the virtually impossible task of determining an accurate total percentage involvement; and 5) validity testing including repeatability testing by the same and different observers must be carried out.7
All of us desire the best outcomes for our patients. As outcomes research continues to evolve, the elaboration and refinement of clinically useful health measurements will prove to be valuable tools assuring that our patients receive optimal care. REFERENCES 1. Jacobe HT, Lettenberger JJ, Bergstresser PR. Understanding clinical trial outcomes: design, analysis, and interpretation. Dermatol Ther 2007;20:77-85. 2. Chren MM, Lasek RJ, Quinn LM, Mostow EN, Zyzanski SJ. Skindex, a quality-of-life measure for patients with skin disease: reliability, validity, and responsiveness. J Invest Dermatol 1996; 107:707-13. 3. McCombs K, Chen SC. Patient preference quality of life measures in dermatology. Dermatol Ther 2007;20:102-9. 4. Albrecht J, Taylor L, Berlin JA, Dulay S, Ang G, Fakharzadeh S, et al. The CLASI (Cutaneous Lupus Erythematosus Disease Area and Severity Index): an outcome instrument for cutaneous lupus erythematosus. J Invest Dermatol 2005;125:889-94. 5. Kreuter A, Tomi NS, Weiner SM, Huger M, Altmeyer P, Gambichler T. Mycophenolate sodium for subacute cutaneous lupus erythematosus resistant to standard therapy. Br J Dermatol 2007;156:1321-7. 6. Ferraz LB, Almeida FA, Vasconcellos MR, Faccina AS, Ciconelli RM, Ferraz MB. The impact of lupus erythematosus on the quality of life: the Brazilian-Portuguese version of DLQI. Qual Life Res 2006;15:565-70. 7. Finlay AY. Measurement of disease activity and outcome in atopic dermatitis. Br J Dermatol 1996;135:509-15.
Additional topics from the January 2008 issue of the Dialogues in Dermatology: 1. Dermatology and the law With Noah S. Scheinfeld, MD, interviewed by Gary Brauner, MD 2. Laser therapy for rosacea With Elizabeth I. McBurney, MD, interviewed by Naomi Lawrence, MD Dialogues in Dermatology is published monthly by the American Academy of Dermatology in both audio cassette and CD formats. Corporate and editorial offices: 930 E Woodfield Dr, Schaumburg, IL 60173-4729. 2007 subscription rates: $150 for individuals in the United States, Canada, and Mexico; $200 International. Ó 2008 by the American Academy of Dermatology, Inc. Subscriptions are available by calling toll-free: 866-503-7546 or faxing 847-240-1859. Additional information is available in the Marketplace section of www.aad.org.
Outcomes research in dermatology Warren R. Heymann, MD Based on the dialogue ‘‘Outcomes research in dermatology’’ between Suephy C. Chen, MD, and Maurice Thew, MD Dialogues in Dermatology, a monthly audio program from the American Academy of Dermatology, contains discussions between dermatologists on timely topics. Commentaries from Dialogues Editorin-Chief Warren R. Heymann, MD, are provided after each discussion as a topic summary and are provided here as a special service to readers of the Journal of the American Academy of Dermatology. ( J Am Acad Dermatol 2008;58:286-7.)
Measure your health by your sympathy with morning and spring. If there is no response in you to the awakening of nature—if the prospect of an early morning walk does not banish sleep, if the warble of the first bluebird does not thrill you—know that the morning and spring of your life are past. Thus you may feel your pulse. Henry David Thoreau
D
ermatologists continue to struggle in accurately quantifying skin disease, its response to treatment, effect on a patient’s quality of life (QOL), its burden on society, and cost effectiveness issues. Although still in its relative infancy, the discipline of outcomes research in dermatology is maturing and will ultimately have a profound effect on our practices. According to Jacobe et al,1 an outcome is defined as one of the possible results that arise from exposure to a causal factor. Outcomes are also referred to as endpoints or variables. Outcomes are ‘‘broadly divided into four categories: physicianbased, patient reported, economic-based, and technology-based’’. To date, there is no consensus regarding the validity and appropriate use of existing health status measures. Indeed, flawed health measurement scales, or their improper use, may be highly problematic. Efforts to improve outcome research in dermatology are underway. There are many health measurement scales in dermatology, such as SCORAD (SCORing Atopic Dermatitis) for atopic dermatitis, SCORTEN for toxic epidermal necrolysis, and PASI (Psoriasis Area and Severity Index) for psoriasis. Indices listed above quantify the physical findings of disease; other measurements, such as Skindex, assess a patient’s QOL.
The statements and opinions expressed in this commentary are those of the Editor-in-Chief of Dialogues in Dermatology.
286
Chren et al2 reported on a survey they named Skindex—a 61-item, self-administered survey with 8 scales, exploring cognitive effects, social effects, depression, fear, embarrassment, anger, physical discomfort, and physical limitations. The authors found their data valid and reproducible.2 The Dermatology Life Quality Index (DLQI) is a simple, 10-question survey addressing how skin disease is interfering with work, study, leisure, and sexual activity. It also asks if the disease or treatment is socially embarrassing. As stated by McCombs and Chen,3 ‘‘The impact of skin disease on patient QOL is often understated. Although few cutaneous diseases are life threatening, virtually all have the potential to impact QOL.’’ Different scales may be used to measure certain patient preferences, such as time tradeoff or willingness-to-pay.3 Data accrued may be valuable for patients, physicians, and health care policymakers. How do dermatologists currently follow their patients? For example, if I am assessing a patient with subacute cutaneous lupus erythematosus (SCLE), I will inquire if there has been a change in the severity of associated arhralgias, or if treatment such as hydroxychloroquine is improving their symptoms. On physical examination, I will try to determine if there has been a diminution of the intensity of erythema or scale. Although this is the time-honored approach to clinical medicine, it is inadequate in conveying vital information about the severity of lupus or its response to treatment. This problem has been addressed by Albrecht et al4 by developing a scoring index, CLASI, for patients with cutaneous lupus erythematosus. The authors define CLASI as consisting of 2 scores; the first summarizes the activity of the disease, whereas the second is a measure of damage done by the disease. ‘‘Activity
J AM ACAD DERMATOL
Dialogues in Dermatology 287
VOLUME 58, NUMBER 2
is scored on the basis of erythema, scale/hypertrophy, mucous membrane involvement, acute hair loss, and non-scarring alopecia. Damage is scored in terms of dyspigmentation and scarring, including scarring alopecia. . . .The scores are calculated by simple addition based on the extent of the symptoms.’’ Associated symptoms such as itch, pain, and fatigue are recorded separately on visual 1 to 10 analog scales by the patients.4 Kreuter et al5 studied 10 patients with SCLE in an open study to evaluate the use of mycophenolate sodium as monotherapy in patients with recalcitrant disease. The CLASI was utilized to assess the patients’ progress. Mycophenolate sodium led to a marked improvement of the skin lesions, resulting in a significant decrease of the CLASI from 10.8 6 6.0 at the beginning to 2.9 6 2.6 at the end of therapy. Clinical improvement was confirmed by the use of ultrasonagraphy and colorimetry. Ferraz et al6 had 71 patients with cutaneous lupus lesions assessed by DLQI and SF-36 (a 36-question short form health survey). The authors demonstrated lower DLQI and SF-36 scores for patients with active disease compared to inactive disease, or patients with alopecia compared to those without alopecia.6 These studies underscore the potential for quantification of clinical impressions. Precise data may allow for more rational clinical decisions. The immediate challenge of outcome research in dermatology is to develop reproducible, valid, straightforward, brief scoring systems for use by clinicians, academicians, and health policy administrators. Finlay7 offers the following criteria to be met for any clinical scoring system: 1) the method should be simple enough to use in a busy clinical setting; 2) the method should clearly separate scores derived from the observer and from the patient; 3) the signs chosen to be recorded should be amenable to change and should be unambiguous in their meaning and proven to be so; 4) recording of area of involvement should be based on an assessment of the site of involvement rather than on the virtually impossible task of determining an accurate total percentage involvement; and 5) validity testing including repeatability testing by the same and different observers must be carried out.7
All of us desire the best outcomes for our patients. As outcomes research continues to evolve, the elaboration and refinement of clinically useful health measurements will prove to be valuable tools assuring that our patients receive optimal care. REFERENCES 1. Jacobe HT, Lettenberger JJ, Bergstresser PR. Understanding clinical trial outcomes: design, analysis, and interpretation. Dermatol Ther 2007;20:77-85. 2. Chren MM, Lasek RJ, Quinn LM, Mostow EN, Zyzanski SJ. Skindex, a quality-of-life measure for patients with skin disease: reliability, validity, and responsiveness. J Invest Dermatol 1996; 107:707-13. 3. McCombs K, Chen SC. Patient preference quality of life measures in dermatology. Dermatol Ther 2007;20:102-9. 4. Albrecht J, Taylor L, Berlin JA, Dulay S, Ang G, Fakharzadeh S, et al. The CLASI (Cutaneous Lupus Erythematosus Disease Area and Severity Index): an outcome instrument for cutaneous lupus erythematosus. J Invest Dermatol 2005;125:889-94. 5. Kreuter A, Tomi NS, Weiner SM, Huger M, Altmeyer P, Gambichler T. Mycophenolate sodium for subacute cutaneous lupus erythematosus resistant to standard therapy. Br J Dermatol 2007;156:1321-7. 6. Ferraz LB, Almeida FA, Vasconcellos MR, Faccina AS, Ciconelli RM, Ferraz MB. The impact of lupus erythematosus on the quality of life: the Brazilian-Portuguese version of DLQI. Qual Life Res 2006;15:565-70. 7. Finlay AY. Measurement of disease activity and outcome in atopic dermatitis. Br J Dermatol 1996;135:509-15.
Additional topics from the January 2008 issue of the Dialogues in Dermatology: 1. Dermatology and the law With Noah S. Scheinfeld, MD, interviewed by Gary Brauner, MD 2. Laser therapy for rosacea With Elizabeth I. McBurney, MD, interviewed by Naomi Lawrence, MD Dialogues in Dermatology is published monthly by the American Academy of Dermatology in both audio cassette and CD formats. Corporate and editorial offices: 930 E Woodfield Dr, Schaumburg, IL 60173-4729. 2007 subscription rates: $150 for individuals in the United States, Canada, and Mexico; $200 International. Ó 2008 by the American Academy of Dermatology, Inc. Subscriptions are available by calling toll-free: 866-503-7546 or faxing 847-240-1859. Additional information is available in the Marketplace section of www.aad.org.