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Published in: Arthritis Research & Therapy 1/2016

Open Access 01-12-2016 | Review

Progress in defining clinically meaningful changes for clinical trials in nonrenal manifestations of SLE disease activity

Authors: Chan-Bum Choi, Matthew H. Liang, Sang-Cheol Bae

Published in: Arthritis Research & Therapy | Issue 1/2016

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Abstract

Since the 2002 Dusseldorf meeting, one new agent, Benlysta, has been approved by the US Food and Drug Administration for systemic lupus erythematosus. Experiences from the field in conducting trials of all the agents tested during this period have provided valuable practical insights. There has been incremental progress in defining the minimal clinically important difference (MCID) of key disease manifestations and the view is largely that of the health care providers and not that of the person suffering the disease. This basic methodological work on the MCID should improve the efficiency and the clinical relevance of future trials and their design.
Literature
1.
go back to reference Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10:407–15.PubMedCrossRef Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10:407–15.PubMedCrossRef
2.
go back to reference Liang MH. Longitudinal construct validity: establishment of clinical meaning in patient evaluative instruments. Med Care. 2000;38(9 Suppl):Ii84–90.PubMed Liang MH. Longitudinal construct validity: establishment of clinical meaning in patient evaluative instruments. Med Care. 2000;38(9 Suppl):Ii84–90.PubMed
3.
go back to reference Liang MH, Socher SA, Roberts WN, Esdaile JM. Measurement of systemic lupus erythematosus activity in clinical research. Arthritis Rheum. 1988;31:817–25.PubMedCrossRef Liang MH, Socher SA, Roberts WN, Esdaile JM. Measurement of systemic lupus erythematosus activity in clinical research. Arthritis Rheum. 1988;31:817–25.PubMedCrossRef
4.
go back to reference The American College of Rheumatology response criteria for systemic lupus erythematosus clinical trials: measures of overall disease activity. Arthritis Rheum. 2004;50:3418–26. The American College of Rheumatology response criteria for systemic lupus erythematosus clinical trials: measures of overall disease activity. Arthritis Rheum. 2004;50:3418–26.
5.
go back to reference White PD, Grover SA, Kangro HO, Thomas JM, Amess J, Clare AW. The validity and reliability of the fatigue syndrome that follows glandular fever. Psychol Med. 1995;25:917–24.PubMedCrossRef White PD, Grover SA, Kangro HO, Thomas JM, Amess J, Clare AW. The validity and reliability of the fatigue syndrome that follows glandular fever. Psychol Med. 1995;25:917–24.PubMedCrossRef
6.
go back to reference Krupp LB, LaRocca NG, Muir-Nash J, Steinberg AD. The fatigue severity scale. Application to patients with multiple sclerosis and systemic lupus erythematosus. Arch Neurol. 1989;46:1121–3.PubMedCrossRef Krupp LB, LaRocca NG, Muir-Nash J, Steinberg AD. The fatigue severity scale. Application to patients with multiple sclerosis and systemic lupus erythematosus. Arch Neurol. 1989;46:1121–3.PubMedCrossRef
7.
go back to reference Chalder T, Berelowitz G, Pawlikowska T, Watts L, Wessely S, Wright D, et al. Development of a fatigue scale. J Psychosom Res. 1993;37:147–53.PubMedCrossRef Chalder T, Berelowitz G, Pawlikowska T, Watts L, Wessely S, Wright D, et al. Development of a fatigue scale. J Psychosom Res. 1993;37:147–53.PubMedCrossRef
8.
go back to reference Robb-Nicholson LC, Daltroy L, Eaton H, Gall V, Wright E, Hartley LH, et al. Effects of aerobic conditioning in lupus fatigue: a pilot study. Br J Rheumatol. 1989;28:500–5.PubMedCrossRef Robb-Nicholson LC, Daltroy L, Eaton H, Gall V, Wright E, Hartley LH, et al. Effects of aerobic conditioning in lupus fatigue: a pilot study. Br J Rheumatol. 1989;28:500–5.PubMedCrossRef
9.
go back to reference Piper BF, Dibble SL, Dodd MJ, Weiss MC, Slaughter RE, Paul SM. The revised Piper Fatigue Scale: psychometric evaluation in women with breast cancer. Oncol Nurs Forum. 1998;25:677–84.PubMed Piper BF, Dibble SL, Dodd MJ, Weiss MC, Slaughter RE, Paul SM. The revised Piper Fatigue Scale: psychometric evaluation in women with breast cancer. Oncol Nurs Forum. 1998;25:677–84.PubMed
10.
go back to reference Stoll T, Gordon C, Seifert B, Richardson K, Malik J, Bacon PA, et al. Consistency and validity of patient administered assessment of quality of life by the MOS SF-36; its association with disease activity and damage in patients with systemic lupus erythematosus. J Rheumatol. 1997;24:1608–14.PubMed Stoll T, Gordon C, Seifert B, Richardson K, Malik J, Bacon PA, et al. Consistency and validity of patient administered assessment of quality of life by the MOS SF-36; its association with disease activity and damage in patients with systemic lupus erythematosus. J Rheumatol. 1997;24:1608–14.PubMed
11.
go back to reference Austin JS, Maisiak RS, Macrina DM, Heck LW. Health outcome improvements in patients with systemic lupus erythematosus using two telephone counseling interventions. Arthritis Care Res. 1996;9:391–9.PubMedCrossRef Austin JS, Maisiak RS, Macrina DM, Heck LW. Health outcome improvements in patients with systemic lupus erythematosus using two telephone counseling interventions. Arthritis Care Res. 1996;9:391–9.PubMedCrossRef
12.
go back to reference Ware JE, Kosinski M, Keller SD. SF-36 Health survey: manual and interpretation guide. Boston, MA: The Health Institute, New England Medical Center; 1983. Ware JE, Kosinski M, Keller SD. SF-36 Health survey: manual and interpretation guide. Boston, MA: The Health Institute, New England Medical Center; 1983.
13.
go back to reference Tack BB. Fatigue in rheumatoid arthritis. Conditions, strategies, and consequences. Arthritis Care Res. 1990;3:65–70.PubMed Tack BB. Fatigue in rheumatoid arthritis. Conditions, strategies, and consequences. Arthritis Care Res. 1990;3:65–70.PubMed
14.
go back to reference Smets EM, Garssen B, Bonke B, De Haes JC. The Multidimensional Fatigue Inventory (MFI) psychometric qualities of an instrument to assess fatigue. J Psychosom Res. 1995;39:315–25.PubMedCrossRef Smets EM, Garssen B, Bonke B, De Haes JC. The Multidimensional Fatigue Inventory (MFI) psychometric qualities of an instrument to assess fatigue. J Psychosom Res. 1995;39:315–25.PubMedCrossRef
15.
go back to reference Schwartz JE, Jandorf L, Krupp LB. The measurement of fatigue: a new instrument. J Psychosom Res. 1993;37:753–62.PubMedCrossRef Schwartz JE, Jandorf L, Krupp LB. The measurement of fatigue: a new instrument. J Psychosom Res. 1993;37:753–62.PubMedCrossRef
16.
go back to reference Ad Hoc Committee on Systemic Lupus Erythematosus Response Criteria for Fatigue. Measurement of fatigue in systemic lupus erythematosus: a systematic review. Arthritis Rheum. 2007;57:1348–57.CrossRef Ad Hoc Committee on Systemic Lupus Erythematosus Response Criteria for Fatigue. Measurement of fatigue in systemic lupus erythematosus: a systematic review. Arthritis Rheum. 2007;57:1348–57.CrossRef
17.
go back to reference Cleanthous S, Tyagi M, Isenberg DA, Newman SP. What do we know about self-reported fatigue in systemic lupus erythematosus? Lupus. 2012;21:465–76.PubMedCrossRef Cleanthous S, Tyagi M, Isenberg DA, Newman SP. What do we know about self-reported fatigue in systemic lupus erythematosus? Lupus. 2012;21:465–76.PubMedCrossRef
18.
go back to reference Yellen SB, Cella DF, Webster K, Blendowski C, Kaplan E. Measuring fatigue and other anemia-related symptoms with the Functional Assessment of Cancer Therapy (FACT) measurement system. J Pain Symptom Manage. 1997;13:63–74.PubMedCrossRef Yellen SB, Cella DF, Webster K, Blendowski C, Kaplan E. Measuring fatigue and other anemia-related symptoms with the Functional Assessment of Cancer Therapy (FACT) measurement system. J Pain Symptom Manage. 1997;13:63–74.PubMedCrossRef
19.
go back to reference Michielsen HJ, De Vries J, Van Heck GL. Psychometric qualities of a brief self-rated fatigue measure: the Fatigue Assessment Scale. J Psychosom Res. 2003;54:345–52.PubMedCrossRef Michielsen HJ, De Vries J, Van Heck GL. Psychometric qualities of a brief self-rated fatigue measure: the Fatigue Assessment Scale. J Psychosom Res. 2003;54:345–52.PubMedCrossRef
20.
go back to reference Goligher EC, Pouchot J, Brant R, Kherani RB, Avina-Zubieta JA, Lacaille D, et al. Minimal clinically important difference for 7 measures of fatigue in patients with systemic lupus erythematosus. J Rheumatol. 2008;35:635–42.PubMed Goligher EC, Pouchot J, Brant R, Kherani RB, Avina-Zubieta JA, Lacaille D, et al. Minimal clinically important difference for 7 measures of fatigue in patients with systemic lupus erythematosus. J Rheumatol. 2008;35:635–42.PubMed
21.
go back to reference Colangelo KJ, Pope JE, Peschken C. The minimally important difference for patient reported outcomes in systemic lupus erythematosus including the HAQ-DI, pain, fatigue, and SF-36. J Rheumatol. 2009;36:2231–7.PubMedCrossRef Colangelo KJ, Pope JE, Peschken C. The minimally important difference for patient reported outcomes in systemic lupus erythematosus including the HAQ-DI, pain, fatigue, and SF-36. J Rheumatol. 2009;36:2231–7.PubMedCrossRef
22.
go back to reference 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.PubMedPubMedCentralCrossRef 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.PubMedPubMedCentralCrossRef
23.
go back to reference Bonilla-Martinez ZL, Albrecht J, Troxel AB, Taylor L, Okawa J, Dulay S, et al. The cutaneous lupus erythematosus disease area and severity index: a responsive instrument to measure activity and damage in patients with cutaneous lupus erythematosus. Arch Dermatol. 2008;144:173–80.PubMedPubMedCentralCrossRef Bonilla-Martinez ZL, Albrecht J, Troxel AB, Taylor L, Okawa J, Dulay S, et al. The cutaneous lupus erythematosus disease area and severity index: a responsive instrument to measure activity and damage in patients with cutaneous lupus erythematosus. Arch Dermatol. 2008;144:173–80.PubMedPubMedCentralCrossRef
24.
go back to reference Klein R, Moghadam-Kia S, LoMonico J, Okawa J, Coley C, Taylor L, et al. Development of the CLASI as a tool to measure disease severity and responsiveness to therapy in cutaneous lupus erythematosus. Arch Dermatol. 2011;147:203–8.PubMedPubMedCentralCrossRef Klein R, Moghadam-Kia S, LoMonico J, Okawa J, Coley C, Taylor L, et al. Development of the CLASI as a tool to measure disease severity and responsiveness to therapy in cutaneous lupus erythematosus. Arch Dermatol. 2011;147:203–8.PubMedPubMedCentralCrossRef
25.
go back to reference Mikdashi JA, Esdaile JM, Alarcon GS, Crofford L, Fessler BJ, Shanberg L, et al. Proposed response criteria for neurocognitive impairment in systemic lupus erythematosus clinical trials. Lupus. 2007;16:418–25.PubMedCrossRef Mikdashi JA, Esdaile JM, Alarcon GS, Crofford L, Fessler BJ, Shanberg L, et al. Proposed response criteria for neurocognitive impairment in systemic lupus erythematosus clinical trials. Lupus. 2007;16:418–25.PubMedCrossRef
26.
go back to reference Ad Hoc Committee on Lupus Response Criteria: Cognition Sub-committee, Mikdashi JA, Esdaile JM, Alarcon GS, Crofford L, Fessler BJ, et al. Proposed response criteria for neurocognitive impairment in systemic lupus erythematosus clinical trials. Lupus. 2007;16:418–25.CrossRef Ad Hoc Committee on Lupus Response Criteria: Cognition Sub-committee, Mikdashi JA, Esdaile JM, Alarcon GS, Crofford L, Fessler BJ, et al. Proposed response criteria for neurocognitive impairment in systemic lupus erythematosus clinical trials. Lupus. 2007;16:418–25.CrossRef
27.
go back to reference Gladman DD, Urowitz MB, Kagal A, Hallett D. Accurately describing changes in disease activity in systemic lupus erythematosus. J Rheumatol. 2000;27:377–9.PubMed Gladman DD, Urowitz MB, Kagal A, Hallett D. Accurately describing changes in disease activity in systemic lupus erythematosus. J Rheumatol. 2000;27:377–9.PubMed
28.
go back to reference Yee CS, Farewell VT, Isenberg DA, Griffiths B, Teh LS, Bruce IN, et al. The use of Systemic Lupus Erythematosus Disease Activity Index-2000 to define active disease and minimal clinically meaningful change based on data from a large cohort of systemic lupus erythematosus patients. Rheumatology. 2011;50:982–8.PubMedPubMedCentralCrossRef Yee CS, Farewell VT, Isenberg DA, Griffiths B, Teh LS, Bruce IN, et al. The use of Systemic Lupus Erythematosus Disease Activity Index-2000 to define active disease and minimal clinically meaningful change based on data from a large cohort of systemic lupus erythematosus patients. Rheumatology. 2011;50:982–8.PubMedPubMedCentralCrossRef
29.
go back to reference Yee CS, Farewell V, Isenberg DA, Griffiths B, Teh LS, Bruce IN, et al. The BILAG-2004 index is sensitive to change for assessment of SLE disease activity. Rheumatology. 2009;48:691–5.PubMedPubMedCentralCrossRef Yee CS, Farewell V, Isenberg DA, Griffiths B, Teh LS, Bruce IN, et al. The BILAG-2004 index is sensitive to change for assessment of SLE disease activity. Rheumatology. 2009;48:691–5.PubMedPubMedCentralCrossRef
30.
go back to reference Yee CS, Gordon C, Isenberg DA, Griffiths B, Teh LS, Bruce IN, et al. The BILAG-2004 systems tally—a novel way of representing the BILAG-2004 index scores longitudinally. Rheumatology. 2012;51:2099–105.PubMedPubMedCentralCrossRef Yee CS, Gordon C, Isenberg DA, Griffiths B, Teh LS, Bruce IN, et al. The BILAG-2004 systems tally—a novel way of representing the BILAG-2004 index scores longitudinally. Rheumatology. 2012;51:2099–105.PubMedPubMedCentralCrossRef
Metadata
Title
Progress in defining clinically meaningful changes for clinical trials in nonrenal manifestations of SLE disease activity
Authors
Chan-Bum Choi
Matthew H. Liang
Sang-Cheol Bae
Publication date
01-12-2016
Publisher
BioMed Central
Published in
Arthritis Research & Therapy / Issue 1/2016
Electronic ISSN: 1478-6362
DOI
https://doi.org/10.1186/s13075-015-0906-9

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