Outcome measurements in scleroderma: Results from a Delphi exercise

Hashim Gazi, Janet E. Pope, Philip Clements, Thomas A. Medsger, Richard W. Martin, Peter A. Merkel, Bashar Kahaleh, Frank A. Wollheim, Murray Baron, Mary Ellen Csuka, Paul Emery, Jill F. Belch, Samina Hayat, Edward V. Lally, Joseph H. Korn, László Czirják, Ariane Herrick, Alexander E. Voskuyl, Pius Bruehlmann, Murat InancDaniel E. Furst, Carol Black, Michael H. Ellman, Larry W. Moreland, Naomi F. Rothfield, Vivien Hsu, Maureen Mayes, Kevin M. McKown, Thomas Krieg, James R. Seibold

Research output: Contribution to journalArticle

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Abstract

Objective. To obtain a consensus on the minimal clinically relevant treatment effect in various scleroderma disease outcome measures to be used in future clinical trials. Methods. A Delphi consensus building exercise using a survey was sent out to members of the Scleroderma Clinical Trials Consortium (SCTC). The 65 SCTC members were divided into 2 groups. Group 1 was informed, in a cover letter, of the usual American College of Rheumatology 20% response results in randomized trials using effective biologic treatments for rheumatoid arthritis, while Group 2 was not. The first round of the exercise presented the scleroderma experts with a survey composed of 95 questions/clinical scenarios divided into 8 categories. These included situations where the treatment group improved, or worsened, or where some outcome measures improved, while others worsened. From the responses of this first round, a mean, mode, median, and range of responses for each of the 95 questions was obtained. This information was sent out, in the second round of the Delphi exercise, only to those respondents who answered the first round. The respondent's previous answer and the mean and range from the first round were provided for each question. It gave respondents the option to change any of their initial responses. The median of their responses in the second round was used to calculate the values for the minimal clinically relevant treatment effect. Results. Thirty-two of the 65 SCTC members returned the first round of the Delphi exercise. Twenty-eight members returned the second round. Intraclass correlation coefficients between responses to round 1 and 2 were calculated for the questions. These varied from 0.99 (excellent agreement) to 0.02 (poor agreement). The p value was under 0.09 for 9 questions and under 0.19 for 20 questions. Standard deviations (SD) were calculated and were found to be lesser for each of the questions in round 2 when compared to the SD in responses from round 1, thus indicating a movement towards a consensus by the second round. An average of 33% of the responses were changed by the respondents in the second round of the Delphi exercise to a value closer to the median/average of the first round's responses. A range in required values for the minimal clinically relevant treatment effect for Modified Rodnan skin score is 3 to 7.5 units, Health Assessment Questionnaire Disability Index (HAQ-DI) 0.2 to 0.25 units, HAQ pain 0.2 to 0.3 units, MD global (100 mm visual analog scale) 8 to 13, patient global assessment 10 to 12, and diffusing capacity (percentage predicted) 9 to 10. The scenarios were especially weighted towards overall disease modification, thus organ-specific measures, such as 6 minute walk time (which has been used in many pulmonary artery hypertension trials), forced vital capacity, and a dyspnea rating (which may be important in scleroderma lung trials), were not included in the survey. Conclusion. Our study begins to address the current deficiency in our knowledge of appropriate values for the minimal clinically relevant treatment effect in various scleroderma disease outcome measures. A consensus could be achieved, or at least a range of minimal clinically relevant treatment effect values could be found for several outcome measurements. Of course, this consensus statement will be modified by evidence as it accrues in each consensus area.

Original languageEnglish (US)
Pages (from-to)501-509
Number of pages9
JournalJournal of Rheumatology
Volume34
Issue number3
StatePublished - Mar 1 2007

Fingerprint

Consensus
Clinical Trials
Outcome Assessment (Health Care)
ametantrone
Exercise
Surveys and Questionnaires
Vital Capacity
Therapeutics
Visual Analog Scale
Pulmonary Hypertension
Dyspnea
Pulmonary Artery
Rheumatoid Arthritis
Pain
Lung
Skin
Health

All Science Journal Classification (ASJC) codes

  • Rheumatology
  • Immunology and Allergy
  • Immunology

Cite this

Gazi, H., Pope, J. E., Clements, P., Medsger, T. A., Martin, R. W., Merkel, P. A., ... Seibold, J. R. (2007). Outcome measurements in scleroderma: Results from a Delphi exercise. Journal of Rheumatology, 34(3), 501-509.
Gazi, Hashim ; Pope, Janet E. ; Clements, Philip ; Medsger, Thomas A. ; Martin, Richard W. ; Merkel, Peter A. ; Kahaleh, Bashar ; Wollheim, Frank A. ; Baron, Murray ; Csuka, Mary Ellen ; Emery, Paul ; Belch, Jill F. ; Hayat, Samina ; Lally, Edward V. ; Korn, Joseph H. ; Czirják, László ; Herrick, Ariane ; Voskuyl, Alexander E. ; Bruehlmann, Pius ; Inanc, Murat ; Furst, Daniel E. ; Black, Carol ; Ellman, Michael H. ; Moreland, Larry W. ; Rothfield, Naomi F. ; Hsu, Vivien ; Mayes, Maureen ; McKown, Kevin M. ; Krieg, Thomas ; Seibold, James R. / Outcome measurements in scleroderma : Results from a Delphi exercise. In: Journal of Rheumatology. 2007 ; Vol. 34, No. 3. pp. 501-509.
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abstract = "Objective. To obtain a consensus on the minimal clinically relevant treatment effect in various scleroderma disease outcome measures to be used in future clinical trials. Methods. A Delphi consensus building exercise using a survey was sent out to members of the Scleroderma Clinical Trials Consortium (SCTC). The 65 SCTC members were divided into 2 groups. Group 1 was informed, in a cover letter, of the usual American College of Rheumatology 20{\%} response results in randomized trials using effective biologic treatments for rheumatoid arthritis, while Group 2 was not. The first round of the exercise presented the scleroderma experts with a survey composed of 95 questions/clinical scenarios divided into 8 categories. These included situations where the treatment group improved, or worsened, or where some outcome measures improved, while others worsened. From the responses of this first round, a mean, mode, median, and range of responses for each of the 95 questions was obtained. This information was sent out, in the second round of the Delphi exercise, only to those respondents who answered the first round. The respondent's previous answer and the mean and range from the first round were provided for each question. It gave respondents the option to change any of their initial responses. The median of their responses in the second round was used to calculate the values for the minimal clinically relevant treatment effect. Results. Thirty-two of the 65 SCTC members returned the first round of the Delphi exercise. Twenty-eight members returned the second round. Intraclass correlation coefficients between responses to round 1 and 2 were calculated for the questions. These varied from 0.99 (excellent agreement) to 0.02 (poor agreement). The p value was under 0.09 for 9 questions and under 0.19 for 20 questions. Standard deviations (SD) were calculated and were found to be lesser for each of the questions in round 2 when compared to the SD in responses from round 1, thus indicating a movement towards a consensus by the second round. An average of 33{\%} of the responses were changed by the respondents in the second round of the Delphi exercise to a value closer to the median/average of the first round's responses. A range in required values for the minimal clinically relevant treatment effect for Modified Rodnan skin score is 3 to 7.5 units, Health Assessment Questionnaire Disability Index (HAQ-DI) 0.2 to 0.25 units, HAQ pain 0.2 to 0.3 units, MD global (100 mm visual analog scale) 8 to 13, patient global assessment 10 to 12, and diffusing capacity (percentage predicted) 9 to 10. The scenarios were especially weighted towards overall disease modification, thus organ-specific measures, such as 6 minute walk time (which has been used in many pulmonary artery hypertension trials), forced vital capacity, and a dyspnea rating (which may be important in scleroderma lung trials), were not included in the survey. Conclusion. Our study begins to address the current deficiency in our knowledge of appropriate values for the minimal clinically relevant treatment effect in various scleroderma disease outcome measures. A consensus could be achieved, or at least a range of minimal clinically relevant treatment effect values could be found for several outcome measurements. Of course, this consensus statement will be modified by evidence as it accrues in each consensus area.",
author = "Hashim Gazi and Pope, {Janet E.} and Philip Clements and Medsger, {Thomas A.} and Martin, {Richard W.} and Merkel, {Peter A.} and Bashar Kahaleh and Wollheim, {Frank A.} and Murray Baron and Csuka, {Mary Ellen} and Paul Emery and Belch, {Jill F.} and Samina Hayat and Lally, {Edward V.} and Korn, {Joseph H.} and L{\'a}szl{\'o} Czirj{\'a}k and Ariane Herrick and Voskuyl, {Alexander E.} and Pius Bruehlmann and Murat Inanc and Furst, {Daniel E.} and Carol Black and Ellman, {Michael H.} and Moreland, {Larry W.} and Rothfield, {Naomi F.} and Vivien Hsu and Maureen Mayes and McKown, {Kevin M.} and Thomas Krieg and Seibold, {James R.}",
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Gazi, H, Pope, JE, Clements, P, Medsger, TA, Martin, RW, Merkel, PA, Kahaleh, B, Wollheim, FA, Baron, M, Csuka, ME, Emery, P, Belch, JF, Hayat, S, Lally, EV, Korn, JH, Czirják, L, Herrick, A, Voskuyl, AE, Bruehlmann, P, Inanc, M, Furst, DE, Black, C, Ellman, MH, Moreland, LW, Rothfield, NF, Hsu, V, Mayes, M, McKown, KM, Krieg, T & Seibold, JR 2007, 'Outcome measurements in scleroderma: Results from a Delphi exercise', Journal of Rheumatology, vol. 34, no. 3, pp. 501-509.

Outcome measurements in scleroderma : Results from a Delphi exercise. / Gazi, Hashim; Pope, Janet E.; Clements, Philip; Medsger, Thomas A.; Martin, Richard W.; Merkel, Peter A.; Kahaleh, Bashar; Wollheim, Frank A.; Baron, Murray; Csuka, Mary Ellen; Emery, Paul; Belch, Jill F.; Hayat, Samina; Lally, Edward V.; Korn, Joseph H.; Czirják, László; Herrick, Ariane; Voskuyl, Alexander E.; Bruehlmann, Pius; Inanc, Murat; Furst, Daniel E.; Black, Carol; Ellman, Michael H.; Moreland, Larry W.; Rothfield, Naomi F.; Hsu, Vivien; Mayes, Maureen; McKown, Kevin M.; Krieg, Thomas; Seibold, James R.

In: Journal of Rheumatology, Vol. 34, No. 3, 01.03.2007, p. 501-509.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Outcome measurements in scleroderma

T2 - Results from a Delphi exercise

AU - Gazi, Hashim

AU - Pope, Janet E.

AU - Clements, Philip

AU - Medsger, Thomas A.

AU - Martin, Richard W.

AU - Merkel, Peter A.

AU - Kahaleh, Bashar

AU - Wollheim, Frank A.

AU - Baron, Murray

AU - Csuka, Mary Ellen

AU - Emery, Paul

AU - Belch, Jill F.

AU - Hayat, Samina

AU - Lally, Edward V.

AU - Korn, Joseph H.

AU - Czirják, László

AU - Herrick, Ariane

AU - Voskuyl, Alexander E.

AU - Bruehlmann, Pius

AU - Inanc, Murat

AU - Furst, Daniel E.

AU - Black, Carol

AU - Ellman, Michael H.

AU - Moreland, Larry W.

AU - Rothfield, Naomi F.

AU - Hsu, Vivien

AU - Mayes, Maureen

AU - McKown, Kevin M.

AU - Krieg, Thomas

AU - Seibold, James R.

PY - 2007/3/1

Y1 - 2007/3/1

N2 - Objective. To obtain a consensus on the minimal clinically relevant treatment effect in various scleroderma disease outcome measures to be used in future clinical trials. Methods. A Delphi consensus building exercise using a survey was sent out to members of the Scleroderma Clinical Trials Consortium (SCTC). The 65 SCTC members were divided into 2 groups. Group 1 was informed, in a cover letter, of the usual American College of Rheumatology 20% response results in randomized trials using effective biologic treatments for rheumatoid arthritis, while Group 2 was not. The first round of the exercise presented the scleroderma experts with a survey composed of 95 questions/clinical scenarios divided into 8 categories. These included situations where the treatment group improved, or worsened, or where some outcome measures improved, while others worsened. From the responses of this first round, a mean, mode, median, and range of responses for each of the 95 questions was obtained. This information was sent out, in the second round of the Delphi exercise, only to those respondents who answered the first round. The respondent's previous answer and the mean and range from the first round were provided for each question. It gave respondents the option to change any of their initial responses. The median of their responses in the second round was used to calculate the values for the minimal clinically relevant treatment effect. Results. Thirty-two of the 65 SCTC members returned the first round of the Delphi exercise. Twenty-eight members returned the second round. Intraclass correlation coefficients between responses to round 1 and 2 were calculated for the questions. These varied from 0.99 (excellent agreement) to 0.02 (poor agreement). The p value was under 0.09 for 9 questions and under 0.19 for 20 questions. Standard deviations (SD) were calculated and were found to be lesser for each of the questions in round 2 when compared to the SD in responses from round 1, thus indicating a movement towards a consensus by the second round. An average of 33% of the responses were changed by the respondents in the second round of the Delphi exercise to a value closer to the median/average of the first round's responses. A range in required values for the minimal clinically relevant treatment effect for Modified Rodnan skin score is 3 to 7.5 units, Health Assessment Questionnaire Disability Index (HAQ-DI) 0.2 to 0.25 units, HAQ pain 0.2 to 0.3 units, MD global (100 mm visual analog scale) 8 to 13, patient global assessment 10 to 12, and diffusing capacity (percentage predicted) 9 to 10. The scenarios were especially weighted towards overall disease modification, thus organ-specific measures, such as 6 minute walk time (which has been used in many pulmonary artery hypertension trials), forced vital capacity, and a dyspnea rating (which may be important in scleroderma lung trials), were not included in the survey. Conclusion. Our study begins to address the current deficiency in our knowledge of appropriate values for the minimal clinically relevant treatment effect in various scleroderma disease outcome measures. A consensus could be achieved, or at least a range of minimal clinically relevant treatment effect values could be found for several outcome measurements. Of course, this consensus statement will be modified by evidence as it accrues in each consensus area.

AB - Objective. To obtain a consensus on the minimal clinically relevant treatment effect in various scleroderma disease outcome measures to be used in future clinical trials. Methods. A Delphi consensus building exercise using a survey was sent out to members of the Scleroderma Clinical Trials Consortium (SCTC). The 65 SCTC members were divided into 2 groups. Group 1 was informed, in a cover letter, of the usual American College of Rheumatology 20% response results in randomized trials using effective biologic treatments for rheumatoid arthritis, while Group 2 was not. The first round of the exercise presented the scleroderma experts with a survey composed of 95 questions/clinical scenarios divided into 8 categories. These included situations where the treatment group improved, or worsened, or where some outcome measures improved, while others worsened. From the responses of this first round, a mean, mode, median, and range of responses for each of the 95 questions was obtained. This information was sent out, in the second round of the Delphi exercise, only to those respondents who answered the first round. The respondent's previous answer and the mean and range from the first round were provided for each question. It gave respondents the option to change any of their initial responses. The median of their responses in the second round was used to calculate the values for the minimal clinically relevant treatment effect. Results. Thirty-two of the 65 SCTC members returned the first round of the Delphi exercise. Twenty-eight members returned the second round. Intraclass correlation coefficients between responses to round 1 and 2 were calculated for the questions. These varied from 0.99 (excellent agreement) to 0.02 (poor agreement). The p value was under 0.09 for 9 questions and under 0.19 for 20 questions. Standard deviations (SD) were calculated and were found to be lesser for each of the questions in round 2 when compared to the SD in responses from round 1, thus indicating a movement towards a consensus by the second round. An average of 33% of the responses were changed by the respondents in the second round of the Delphi exercise to a value closer to the median/average of the first round's responses. A range in required values for the minimal clinically relevant treatment effect for Modified Rodnan skin score is 3 to 7.5 units, Health Assessment Questionnaire Disability Index (HAQ-DI) 0.2 to 0.25 units, HAQ pain 0.2 to 0.3 units, MD global (100 mm visual analog scale) 8 to 13, patient global assessment 10 to 12, and diffusing capacity (percentage predicted) 9 to 10. The scenarios were especially weighted towards overall disease modification, thus organ-specific measures, such as 6 minute walk time (which has been used in many pulmonary artery hypertension trials), forced vital capacity, and a dyspnea rating (which may be important in scleroderma lung trials), were not included in the survey. Conclusion. Our study begins to address the current deficiency in our knowledge of appropriate values for the minimal clinically relevant treatment effect in various scleroderma disease outcome measures. A consensus could be achieved, or at least a range of minimal clinically relevant treatment effect values could be found for several outcome measurements. Of course, this consensus statement will be modified by evidence as it accrues in each consensus area.

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Gazi H, Pope JE, Clements P, Medsger TA, Martin RW, Merkel PA et al. Outcome measurements in scleroderma: Results from a Delphi exercise. Journal of Rheumatology. 2007 Mar 1;34(3):501-509.