thalidomide has been researched along with Cirrhosis in 20 studies
Thalidomide: A piperidinyl isoindole originally introduced as a non-barbiturate hypnotic, but withdrawn from the market due to teratogenic effects. It has been reintroduced and used for a number of immunological and inflammatory disorders. Thalidomide displays immunosuppressive and anti-angiogenic activity. It inhibits release of TUMOR NECROSIS FACTOR-ALPHA from monocytes, and modulates other cytokine action.
thalidomide : A racemate comprising equimolar amounts of R- and S-thalidomide.
2-(2,6-dioxopiperidin-3-yl)-1H-isoindole-1,3(2H)-dione : A dicarboximide that is isoindole-1,3(2H)-dione in which the hydrogen attached to the nitrogen is substituted by a 2,6-dioxopiperidin-3-yl group.
Excerpt | Relevance | Reference |
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"We evaluated the antifibrotic effects of pomalidomide in preventive as well as therapeutic treatment regimes using bleomycin-induced dermal fibrosis as a model of early, inflammatory stages of fibrosis and the tight-skin mouse model as a model of later stages of fibrosis with endogenous activation of fibroblasts." | 7.78 | Pomalidomide is effective for prevention and treatment of experimental skin fibrosis. ( Beyer, C; Cedzik, D; Dees, C; Distler, A; Distler, JH; Distler, O; Palumbo-Zerr, K; Schafer, PH; Schett, G; Shankar, SL; Tomcik, M; Weingärtner, S; Zerr, P, 2012) |
"Although thalidomide had no effect on cardiac function, our results suggest that intervention with thalidomide may have beneficial effects in post-MI HF by attenuating collagen accumulation and development of myocardial fibrosis." | 7.73 | Thalidomide attenuates the development of fibrosis during post-infarction myocardial remodelling in rats. ( Attramadal, H; Aukrust, P; Bjørnerheim, R; Frøland, SS; Oie, E; Ueland, T; Vinge, LE; Wang, JE; Yndestad, A, 2006) |
"Thalidomide has shown excellent results in the treatment of multiple myeloma probably due to its anti-angiogenic activity." | 5.31 | Thalidomide in the treatment of myelodysplastic syndrome with fibrosis. ( Apostolidis, P; Dervenoulas, J; Kontopidou, F; Rontogianni, D; Tsirigotis, P; Venetis, E, 2002) |
" There was weak evidence for the improvement of 6MWD using oxygen; dyspnoea using prednisolone, diamorphine, D-pencillamine and colchicine; cough using interferon α and thalidomide; anxiety using diamorphine; fatigue using pulmonary rehabilitation; and QoL using thalidomide and doxycycline." | 4.89 | Interventions to improve symptoms and quality of life of patients with fibrotic interstitial lung disease: a systematic review of the literature. ( Bajwah, S; Higginson, IJ; Koffman, J; Patel, AS; Peacock, JL; Riley, J; Ross, JR; Wells, AU, 2013) |
"Most of the existing studies focus on the early inflammation of rosacea, with few interventions on the later development of fibrosis and the relationship between thalidomide and rosacea." | 4.12 | Thalidomide Attenuates Skin Lesions and Inflammation in Rosacea-Like Mice Induced by Long-Term Exposure of LL-37. ( He, Y; Kang, Y; Liu, H; Wei, Z; Yang, J; Zhang, C; Zhang, Z, 2022) |
"To evaluate the immune regulatory and anti-fibrosis function of thalidomide (Thal) in systemic sclerosis (SSc), we investigated the effects of Thal on: (a) Th17 and Treg cell production; (b) related factors expression; and (c) transforming growth factor (TGF)-β1/Smad3 pathway, using a mouse model of SSc." | 3.96 | Effects of thalidomide on Th17, Treg cells and TGF-β1/Smad3 pathway in a mouse model of systemic sclerosis. ( Lei, L; Li, X; Lu, Y; Tao, Z; Wen, J; Zhao, C; Zheng, L, 2020) |
"Specific inhibition of PDE4 by rolipram and apremilast had potent antifibrotic effects in bleomycin-induced skin fibrosis models, in the topoisomerase I mouse model and in murine sclerodermatous chronic graft-versus-host disease." | 3.85 | Inhibition of phosphodiesterase 4 (PDE4) reduces dermal fibrosis by interfering with the release of interleukin-6 from M2 macrophages. ( Bergmann, C; Beyer, C; Distler, JHW; Kittan, N; Maier, C; Ramming, A; Schett, G; Weinkam, R, 2017) |
"Although others have shown protective effects of thalidomide in disease models involving inflammation, fibrosis and blood vessel maturation, thalidomide was not able to reduce radiation-induced heart damage." | 3.79 | Thalidomide is not able to inhibit radiation-induced heart disease. ( Hoving, S; Seemann, I; Stewart, FA; te Poele, JA; Visser, NL, 2013) |
"We evaluated the antifibrotic effects of pomalidomide in preventive as well as therapeutic treatment regimes using bleomycin-induced dermal fibrosis as a model of early, inflammatory stages of fibrosis and the tight-skin mouse model as a model of later stages of fibrosis with endogenous activation of fibroblasts." | 3.78 | Pomalidomide is effective for prevention and treatment of experimental skin fibrosis. ( Beyer, C; Cedzik, D; Dees, C; Distler, A; Distler, JH; Distler, O; Palumbo-Zerr, K; Schafer, PH; Schett, G; Shankar, SL; Tomcik, M; Weingärtner, S; Zerr, P, 2012) |
"Although thalidomide had no effect on cardiac function, our results suggest that intervention with thalidomide may have beneficial effects in post-MI HF by attenuating collagen accumulation and development of myocardial fibrosis." | 3.73 | Thalidomide attenuates the development of fibrosis during post-infarction myocardial remodelling in rats. ( Attramadal, H; Aukrust, P; Bjørnerheim, R; Frøland, SS; Oie, E; Ueland, T; Vinge, LE; Wang, JE; Yndestad, A, 2006) |
"Treatment with thalidomide markedly stimulated the phosphorylation of AMPKα." | 1.48 | Renal-protective effect of thalidomide in streptozotocin-induced diabetic rats through anti-inflammatory pathway. ( Li, R; Wang, B; Wang, Y; Yang, Y; Zhang, H, 2018) |
"We induced type 1 diabetes using streptozocin in 8-week-old Sprague-Dawley rats, divided them into two groups-a thalidomide treatment group (DM-T, n = 15) and a non-treatment group (DM-N, n = 15)-and compared them with a normal control (n = 10)." | 1.36 | The protective effect of thalidomide on left ventricular function in a rat model of diabetic cardiomyopathy. ( Chang, HJ; Chang, SA; Kim, DH; Kim, HK; Kim, HN; Kim, YJ; Lee, HW; Park, YB; Sohn, DW, 2010) |
"Thalidomide has shown excellent results in the treatment of multiple myeloma probably due to its anti-angiogenic activity." | 1.31 | Thalidomide in the treatment of myelodysplastic syndrome with fibrosis. ( Apostolidis, P; Dervenoulas, J; Kontopidou, F; Rontogianni, D; Tsirigotis, P; Venetis, E, 2002) |
Timeframe | Studies, this research(%) | All Research% |
---|---|---|
pre-1990 | 0 (0.00) | 18.7374 |
1990's | 1 (5.00) | 18.2507 |
2000's | 5 (25.00) | 29.6817 |
2010's | 10 (50.00) | 24.3611 |
2020's | 4 (20.00) | 2.80 |
Authors | Studies |
---|---|
Sloan, SB | 1 |
Kang, Y | 2 |
Zhang, C | 2 |
He, Y | 2 |
Zhang, Z | 2 |
Liu, H | 2 |
Wei, Z | 2 |
Yang, J | 2 |
Lu, Y | 1 |
Zhao, C | 1 |
Lei, L | 1 |
Tao, Z | 1 |
Zheng, L | 1 |
Wen, J | 1 |
Li, X | 1 |
Lu, QK | 1 |
Fan, C | 1 |
Xiang, CG | 1 |
Wu, B | 1 |
Lu, HM | 1 |
Feng, CL | 1 |
Yang, XQ | 1 |
Li, H | 1 |
Tang, W | 1 |
Hsu, VM | 1 |
Denton, CP | 1 |
Domsic, RT | 1 |
Furst, DE | 1 |
Rischmueller, M | 1 |
Stanislav, M | 1 |
Steen, VD | 1 |
Distler, JHW | 2 |
Korish, S | 1 |
Cooper, A | 1 |
Choi, S | 1 |
Schafer, PH | 2 |
Horan, G | 1 |
Hough, DR | 1 |
Zhang, H | 1 |
Yang, Y | 1 |
Wang, Y | 1 |
Wang, B | 1 |
Li, R | 1 |
Hoving, S | 1 |
Seemann, I | 1 |
Visser, NL | 1 |
te Poele, JA | 1 |
Stewart, FA | 2 |
Scharpfenecker, M | 1 |
Floot, B | 1 |
Russell, NS | 1 |
Coppes, RP | 1 |
Bersani-Amado, LE | 1 |
Dantas, JA | 1 |
Damião, MJ | 1 |
Rocha, BA | 1 |
Besson, JC | 1 |
Bastos, RL | 1 |
Silva, LN | 1 |
Bersani-Amado, CA | 1 |
Cuman, RK | 1 |
Maier, C | 1 |
Ramming, A | 1 |
Bergmann, C | 1 |
Weinkam, R | 1 |
Kittan, N | 1 |
Schett, G | 2 |
Beyer, C | 2 |
Fernández-Martínez, E | 1 |
Pérez-Hernández, N | 1 |
Muriel, P | 1 |
Pérez-Alvarez, V | 1 |
Shibayama, M | 1 |
Tsutsumi, V | 1 |
Mittelman, M | 1 |
Oster, HS | 1 |
Hoffman, M | 1 |
Neumann, D | 1 |
Kim, DH | 1 |
Kim, YJ | 1 |
Chang, SA | 1 |
Lee, HW | 1 |
Kim, HN | 1 |
Kim, HK | 1 |
Chang, HJ | 1 |
Sohn, DW | 1 |
Park, YB | 1 |
Weingärtner, S | 1 |
Zerr, P | 1 |
Tomcik, M | 1 |
Palumbo-Zerr, K | 1 |
Distler, A | 1 |
Dees, C | 1 |
Shankar, SL | 1 |
Cedzik, D | 1 |
Distler, O | 1 |
Distler, JH | 1 |
Bajwah, S | 1 |
Ross, JR | 1 |
Peacock, JL | 1 |
Higginson, IJ | 1 |
Wells, AU | 1 |
Patel, AS | 1 |
Koffman, J | 1 |
Riley, J | 1 |
Tsirigotis, P | 1 |
Venetis, E | 1 |
Rontogianni, D | 1 |
Dervenoulas, J | 1 |
Kontopidou, F | 1 |
Apostolidis, P | 1 |
Yndestad, A | 2 |
Vinge, LE | 1 |
Bjørnerheim, R | 1 |
Ueland, T | 2 |
Wang, JE | 1 |
Frøland, SS | 1 |
Attramadal, H | 1 |
Aukrust, P | 2 |
Oie, E | 1 |
Chong, LW | 1 |
Hsu, YC | 1 |
Chiu, YT | 1 |
Yang, KC | 1 |
Huang, YT | 1 |
Damås, JK | 1 |
Øie, E | 1 |
Gullestad, L | 1 |
Rousseau, L | 1 |
Beylot-Barry, M | 1 |
Doutre, MS | 1 |
Beylot, C | 1 |
Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
---|---|---|---|---|---|---|---|
A Phase 2, Proof-Of-Concept, Multicenter, Randomized, Double-Blind, Placebo- Controlled, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Pomalidomide (CC-4047) in Subjects With Systemic Sclerosis With Interst[NCT01559129] | Phase 2 | 23 participants (Actual) | Interventional | 2012-08-09 | Terminated (stopped due to Enrollment was stopped early (see limitations and caveats section).) | ||
A Phase 2, Open Label Single Arm Study for Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis[NCT03529955] | Phase 2 | 8 participants (Actual) | Interventional | 2018-06-12 | Completed | ||
Mean Platelet Volume and Its Relation to Risk Stratification of Myelodysplastic Syndromes[NCT05204953] | 71 participants (Actual) | Observational | 2013-10-31 | Completed | |||
[information is prepared from clinicaltrials.gov, extracted Sep-2024] |
Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). For the analysis of FVC, the baseline value was defined as the average of all values between Screening and Baseline (inclusive), and the average of Weeks 48 and 52 was treated as the Week 52 value, to reduce the total data variability at the key time points. (NCT01559129)
Timeframe: Baseline (defined as the average of all values between Screening and Baseline) and Weeks 48 and 52
Intervention | percent predicted (Mean) |
---|---|
Placebo | -2.8 |
Pomalidomide | -5.2 |
Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 [normal], 1 [mild], 2 [moderate]), or 3 [severe]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51. (NCT01559129)
Timeframe: Baseline and Week 52 (or the Treatment Phase Early Termination visit)
Intervention | units on a scale (Mean) |
---|---|
Placebo | -3.7 |
Pomalidomide | -2.7 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets gastrointestinal (GI) activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms. (NCT01559129)
Timeframe: Baseline and Week 52 (or Treatment Phase Early Termination visit)
Intervention | units on a scale (Mean) |
---|---|
Placebo | 0.00 |
Pomalidomide | 0.1 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 12
Intervention | Participants (Count of Participants) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 1 | 0 | 7 | 2 | 0 | 0 | 0 |
Pomalidomide | 0 | 1 | 1 | 3 | 1 | 0 | 0 | 1 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 156 or the Extension Phase Early Termination visit
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 2 | 2 | 1 | 1 | 0 | 0 |
Pomalidomide | 0 | 1 | 1 | 0 | 0 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 24
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 1 | 0 | 1 | 4 | 3 | 1 | 0 | 0 |
Pomalidomide | 0 | 2 | 1 | 2 | 1 | 0 | 0 | 1 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 52 or at the Treatment Phase Early Termination visit
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 1 | 8 | 3 | 0 | 0 | 0 |
Pomalidomide | 0 | 4 | 1 | 4 | 1 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 64
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 1 | 4 | 1 | 0 | 0 | 0 |
Pomalidomide | 1 | 1 | 0 | 0 | 0 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 76
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 1 | 0 | 1 | 0 | 0 | 0 |
Pomalidomide | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 12
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 1 | 2 | 4 | 2 | 1 | 0 | 0 |
Pomalidomide | 0 | 0 | 2 | 4 | 1 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 156 or at the Extension Phase Early Termination visit
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 1 | 2 | 2 | 1 | 0 | 0 | 0 |
Pomalidomide | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 24
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 1 | 0 | 1 | 4 | 3 | 1 | 0 | 0 |
Pomalidomide | 1 | 1 | 0 | 3 | 2 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 52 or at the Treatment Phase Early Termination visit
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 1 | 7 | 3 | 1 | 0 | 0 |
Pomalidomide | 0 | 3 | 1 | 6 | 0 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 64
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 3 | 2 | 1 | 0 | 0 | 0 |
Pomalidomide | 1 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. (NCT01559129)
Timeframe: Week 76
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 1 | 0 | 1 | 0 | 0 | 0 |
Pomalidomide | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. (NCT01559129)
Timeframe: Week 12
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 2 | 5 | 3 | 0 | 0 | 0 |
Pomalidomide | 0 | 0 | 1 | 5 | 1 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carry very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. (NCT01559129)
Timeframe: Week 156 or the Extension Phase Early Termination visit
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 1 | 1 | 2 | 2 | 0 | 0 | 0 |
Pomalidomide | 0 | 0 | 1 | 0 | 1 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. (NCT01559129)
Timeframe: Week 24
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 1 | 2 | 5 | 2 | 0 | 0 | 0 |
Pomalidomide | 0 | 1 | 1 | 2 | 3 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. (NCT01559129)
Timeframe: Week 52 or at the Treatment Phase Early Termination visit
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 1 | 8 | 2 | 0 | 1 | 0 |
Pomalidomide | 0 | 3 | 1 | 6 | 0 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. (NCT01559129)
Timeframe: Week 64
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 2 | 3 | 1 | 0 | 0 | 0 |
Pomalidomide | 1 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. (NCT01559129)
Timeframe: Week 76
Intervention | Participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Major deterioration | Moderate deterioration | Minor deterioration | No change | Minor improvement | Moderate improvement | Major improvement | Further impairment for other reasons | |
Placebo | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 0 |
Pomalidomide | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 [normal], 1 [mild], 2 [moderate], or 3 [severe]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | ||||
---|---|---|---|---|---|
Week 12 | Week 24 | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | -1.8 | -3.6 | -4.3 | -8.5 | -3.7 |
Pomalidomide | -0.3 | -2.0 | -4.0 | -7.0 | -4.5 |
Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). (NCT01559129)
Timeframe: Baseline (defined as the average of all values between Screening and Baseline) and Weeks 12, 24, 36, 64, 76, and 156
Intervention | percent predicted (Mean) | |||||
---|---|---|---|---|---|---|
Week 12 | Week 24 | Week 36 | Week 64 | Week 76 | Week 156 | |
Placebo | -2.4 | -1.3 | -2.6 | -7.0 | -8.7 | -6.7 |
Pomalidomide | -1.8 | 2.3 | -4.4 | -6.4 | -5.2 | -5.9 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The constipation subscale score is calculated as the average of three questions regarding the frequency of constipation (scored from 0 [no days] to 3 [5-7 days/week] and one question about the presence of stools becoming harder (scored as 0 [No] or 1 [Yes]); the score ranges from 0 to 2.5, where higher scores indicate more frequent symptoms. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | |||||
---|---|---|---|---|---|---|
Week 12 | Week 24 | Week 52/Early Termination | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | -0.1 | -0.2 | 0.0 | 0.0 | 0.0 | -0.2 |
Pomalidomide | 0.2 | 0.3 | 0.3 | 0.1 | 0.0 | 0.1 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The diarrhea subscale score is calculated as the average of one diarrhea question about the frequency of loose stools (on a scale from 0 [none] to 3 [5-7 days/week] and one question about the presence of watery stools (scored as 0 [No] or 1 [Yes]); the score ranges from 0 to 2, where a higher score indicates more frequent symptoms. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | |||||
---|---|---|---|---|---|---|
Week 12 | Week 24 | Week 52/Early Termination | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | 0.4 | 0.1 | 0.3 | 0.1 | 0.0 | 0.3 |
Pomalidomide | -0.2 | -0.3 | 0.0 | 0.5 | -0.5 | -0.8 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The distension/bloating subscale score is calculated as the average of four distension/bloating-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | |||||
---|---|---|---|---|---|---|
Week 12 | Week 24 | Week 52/Early Termination | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | 0.4 | 0.0 | 0.0 | -0.2 | 0.0 | -0.3 |
Pomalidomide | 0.2 | 0.3 | 0.2 | 1.0 | 3.0 | 0.9 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The emotional well-being subscale score is calculated as the average of nine questions regarding the impact of bowel problems on emotional status; the score ranges from 0 to 3, where higher scores indicate more frequent problems. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | |||||
---|---|---|---|---|---|---|
Week 12 | Week 24 | Week 52/Early Termination | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | 0.2 | 0.1 | 0.1 | 0.0 | 0.0 | -0.1 |
Pomalidomide | -0.4 | -0.3 | -0.1 | 0.2 | 0.3 | 0.0 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The fecal soilage subscale score is calculated from one soilage question; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | |||||
---|---|---|---|---|---|---|
Week 12 | Week 24 | Week 52/Early Termination | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | 0.0 | 0.2 | 0.0 | 0.2 | 0.0 | 0.2 |
Pomalidomide | 0.0 | 0.0 | 0.1 | 0.0 | 0.0 | 0.0 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The reflux subscale score is calculated as the average of eight reflux-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | |||||
---|---|---|---|---|---|---|
Week 12 | Week 24 | Week 52/Early Termination | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | 0.1 | -0.1 | -0.1 | -0.2 | -0.5 | 0.0 |
Pomalidomide | -0.1 | 0.0 | 0.1 | 0.2 | -0.2 | 0.1 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The social functioning subscale score is calculated as the average of six questions about how often symptoms interfered with social activities; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | |||||
---|---|---|---|---|---|---|
Week 12 | Week 24 | Week 52/Early termination | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | 0.3 | 0.1 | 0.0 | 0.0 | 0.2 | 0.0 |
Pomalidomide | 0.0 | -0.1 | 0.2 | 0.3 | 0.0 | -0.2 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | units on a scale (Mean) | ||||
---|---|---|---|---|---|
Week 12 | Week 24 | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | 0.2 | 0.1 | 0.0 | -0.1 | 0.0 |
Pomalidomide | -0.1 | -0.1 | 0.4 | 0.5 | 0.0 |
An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A TEAE is any AE that began or worsened on or after the start of study drug through 28 days after the last dose. A treatment-related TEAE is a TEAE which was considered by the investigator to be related to study drug. The severity/intensity of AEs was assessed by the investigator as Mild (asymptomatic or mild symptoms; intervention not indicated), Moderate (symptoms cause moderate discomfort, intervention may be required), or Severe (symptoms cause severe discomfort/pain, requiring medical intervention, inability to perform daily activities). A serious AE is any AE that: - Resulted in death; - Was life-threatening; - Required inpatient hospitalization or prolongation of existing hospitalization - Resulted in persistent or significant disability/incapacity; - Was a congenital anomaly/birth defect; - Constituted an important medical event. (NCT01559129)
Timeframe: From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and pomalidomide.
Intervention | participants (Number) | |||||||
---|---|---|---|---|---|---|---|---|
Any TEAE | Drug-related TEAE | Severe TEAE | Serious TEAE | Serious Drug-related TEAE | TEAE Leading to Drug Interruption | TEAE Leading to Drug Withdrawal | TEAE Leading to Death | |
Extension Phase: Placebo/Pomalidomide | 5 | 3 | 0 | 0 | 0 | 0 | 0 | 0 |
Extension Phase: Pomalidomide/Pomalidomide | 2 | 1 | 0 | 1 | 0 | 0 | 0 | 0 |
Treatment Phase: Placebo | 12 | 8 | 1 | 1 | 0 | 2 | 0 | 0 |
Treatment Phase: Pomalidomide | 9 | 6 | 4 | 4 | 1 | 1 | 4 | 0 |
Oxygen saturation was measured by pulse oximetry. (NCT01559129)
Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Intervention | percent saturation (Mean) | ||||||
---|---|---|---|---|---|---|---|
Baseline | Week 12 | Week 24 | Week 52/Early Termination | Week 64 | Week 76 | Week 156/Early Termination | |
Placebo | 97.5 | 97.1 | 97.6 | 96.8 | 96.3 | 98.5 | 95.8 |
Pomalidomide | 96.8 | 97.4 | 96.5 | 96.8 | 94.0 | 97.0 | 97.5 |
"MMT-8 (Manual Muscle Testing-8) score is a validated tool to assess muscle strength. Calculate the mean change in MMT-8 score at 3 and 6 month(s) compared to baseline in patients with muscle disease.~Units: Units on a scale. Scale goes from 0-150. 150 is perfect strength." (NCT03529955)
Timeframe: Data collected at 3 and 6 months after baseline visit
Intervention | score on a scale (Mean) |
---|---|
MMT-8 Score at 3 Months | 143.3 |
MMT-8 Score at 6 Months | 144.5 |
"Dermatology Life Quality Index (DLQI) is a validated tool to measure quality of life in patients with skin disease. Complete response is defined by a DLQI of zero at 3, and 6 months. Partial response is defined by a decrease of DLQI of at least 5 points at 3, and 6 months compared to baseline. Calculation is performed as the DLQI at 3, and 6 months minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).~Units : Units on a scale from 0-30, higher scores represent worse outcome." (NCT03529955)
Timeframe: Data collected at 3 and 6 months after baseline visit
Intervention | score on a scale (Mean) |
---|---|
DLQI Score at 3 Months | 6.3 |
DLQI Score at 6 Months | 4.2 |
"The durability of response will be measured using the CDASI activity score at 6 months minus CDASI activity score at 3 months. Complete response durability is defined as zero or minus difference between CDASI activity score at 6 months and CDASI activity score at 3 months. Partial response durability is defined as >4 points difference between CDASI activity score at 6 months and CDASI activity score at 3 months. Missing data will be handled using the last observation carried forward approach (LOCF).~CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome." (NCT03529955)
Timeframe: Data collected at 6 months compared to data collected at 3 months
Intervention | score on a scale (Mean) |
---|---|
CDASI Score at 3 Months | 16.9 |
CDASI Score at 6 Months | 14 |
"Cutaneous dermatomyositis disease area and severity index (CDASI) activity score is a validated tool to measure skin disease activity in dermatomyositis. The overall response rate (ORR) includes partial and complete responses. Complete response is defined by a CDASI activity score of zero. Partial response is defined by a decrease of CDASI activity score of at least 4 points. Calculation is performed as the CDASI activity score at 3 month(s) minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).~CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome." (NCT03529955)
Timeframe: Data collected at 3 months after baseline visit
Intervention | Participants (Count of Participants) |
---|---|
Dermatomyositis Patients With Refractory Cutaneous Disease | 7 |
"The proportion of participants experiencing adverse events and serious adverse events was measured over 7 months period (6 months during the study and 1 month follow up) using Common Terminology Criteria for Adverse Events (CTCAE) v5.0.~Grade refers to severity of the AE. The CTCAE displays Grades 1 to 5 with unique clinical descriptions of severity for each AE:~Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age- appropriate instrumental Activity of Daily Living (ADL) Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL Grade 4 Life-threatening consequences; urgent intervention indicated Grade 5 Death related to AE All adverse events subjects experienced were grade 1 or 2 which is mild to moderate in severity." (NCT03529955)
Timeframe: 7 months
Intervention | Participants (Count of Participants) | ||||||||
---|---|---|---|---|---|---|---|---|---|
Headache Grade 1-2 | Nausea Grade 1-2 | Diarrhea Grade 1-2 | Herpes Zoster Grade 1-2 | Influenza Grade 1-2 | Pneumonia Grade 1-2 | Acute sinusitis Grade 1-2 | Hypertension Grade 1-2 | Ocular pressure Grade 1-2 | |
Dermatomyositis Patients With Refractory Cutaneous Disease | 7 | 5 | 4 | 2 | 1 | 1 | 1 | 1 | 1 |
Skin biopsies from lesional skin will be performed before treatment with apremilast and after 3 months of treatment to assess changes in gene expression profiling and immunohistochemistry stain. Gene expression profiling will be analyzed using inferential statistics with a False Discovery Rate (FDR) of < 0.05. (NCT03529955)
Timeframe: Data collected at 3 months after baseline visit
Intervention | Change (Number) | |
---|---|---|
Down regulated genes | Up regulated genes | |
Skin Biopsy at 3 Months Into Apremilast Therapy for Gene Expression Profiling | 123 | 72 |
Skin Biopsy at Baseline for Gene Expression Profiling | 0 | 0 |
Skin biopsies from lesional skin will be performed before treatment with apremilast and after 3 months of treatment to assess changes in immunohistochemistry stain. (NCT03529955)
Timeframe: Data collected at 3 months after baseline visit
Intervention | Percentage of positive cell detection (Mean) | |
---|---|---|
STAT1 | STAT3 | |
Skin Biopsy at 3 Months Into Apremilast Therapy for IHC | 50.1 | 17.4 |
Skin Biopsy at Baseline for IHC | 96.2 | 44.3 |
3 reviews available for thalidomide and Cirrhosis
Article | Year |
---|---|
The lower risk MDS patient at risk of rapid progression.
Topics: Age Factors; alpha Catenin; Antigens, Differentiation; Antineoplastic Agents; Axl Receptor Tyrosine | 2010 |
Interventions to improve symptoms and quality of life of patients with fibrotic interstitial lung disease: a systematic review of the literature.
Topics: Anti-Bacterial Agents; Anti-Inflammatory Agents, Non-Steroidal; Colchicine; Cough; Dyspnea; Exercise | 2013 |
Potential mechanisms of benefit with thalidomide in chronic heart failure.
Topics: Animals; Cardiac Output, Low; Fibrosis; Heart Rate; Humans; Immune System; Immunosuppressive Agents; | 2007 |
1 trial available for thalidomide and Cirrhosis
Article | Year |
---|---|
Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study.
Topics: Adult; Double-Blind Method; Early Termination of Clinical Trials; Female; Fibrosis; Follow-Up Studie | 2018 |
16 other studies available for thalidomide and Cirrhosis
Article | Year |
---|---|
This Month in JAAD Case Reports: April 2022: Apremilast for Morphea.
Topics: Fibrosis; Humans; Scleroderma, Localized; Thalidomide | 2022 |
Thalidomide Attenuates Skin Lesions and Inflammation in Rosacea-Like Mice Induced by Long-Term Exposure of LL-37.
Topics: Animals; Collagen; Fibrosis; Male; Mice; Thalidomide | 2022 |
Thalidomide Attenuates Skin Lesions and Inflammation in Rosacea-Like Mice Induced by Long-Term Exposure of LL-37.
Topics: Animals; Collagen; Fibrosis; Male; Mice; Thalidomide | 2022 |
Thalidomide Attenuates Skin Lesions and Inflammation in Rosacea-Like Mice Induced by Long-Term Exposure of LL-37.
Topics: Animals; Collagen; Fibrosis; Male; Mice; Thalidomide | 2022 |
Thalidomide Attenuates Skin Lesions and Inflammation in Rosacea-Like Mice Induced by Long-Term Exposure of LL-37.
Topics: Animals; Collagen; Fibrosis; Male; Mice; Thalidomide | 2022 |
Effects of thalidomide on Th17, Treg cells and TGF-β1/Smad3 pathway in a mouse model of systemic sclerosis.
Topics: Animals; Cytokines; Disease Models, Animal; Female; Fibrosis; Hydroxyproline; Immunosuppressive Agen | 2020 |
Inhibition of PDE4 by apremilast attenuates skin fibrosis through directly suppressing activation of M1 and T cells.
Topics: Animals; Blotting, Western; Cyclic Nucleotide Phosphodiesterases, Type 4; Female; Fibrosis; Flow Cyt | 2022 |
Renal-protective effect of thalidomide in streptozotocin-induced diabetic rats through anti-inflammatory pathway.
Topics: AMP-Activated Protein Kinases; Animals; Anti-Inflammatory Agents; Cytokines; Cytoprotection; Diabete | 2018 |
Thalidomide is not able to inhibit radiation-induced heart disease.
Topics: Animals; Fibrosis; Heart; Heart Diseases; Male; Mice; Mice, Inbred C57BL; Microvessels; Radiation In | 2013 |
Thalidomide ameliorates inflammation and vascular injury but aggravates tubular damage in the irradiated mouse kidney.
Topics: Angiogenesis Modulating Agents; Animals; Anti-Inflammatory Agents; Female; Fibrosis; Genes, sis; Glo | 2014 |
Involvement of cytokines in the modulation and progression of renal fibrosis induced by unilateral ureteral obstruction in C57BL/6 mice: effects of thalidomide and dexamethasone.
Topics: Animals; Anti-Inflammatory Agents; Cytokines; Dexamethasone; Disease Models, Animal; Disease Progres | 2016 |
Inhibition of phosphodiesterase 4 (PDE4) reduces dermal fibrosis by interfering with the release of interleukin-6 from M2 macrophages.
Topics: Animals; Bleomycin; Cell Differentiation; Collagen; Cyclic Nucleotide Phosphodiesterases, Type 4; Cy | 2017 |
The thalidomide analog 3-phthalimido-3-(3,4-dimethoxyphenyl)-propanoic acid improves the biliary cirrhosis in the rat.
Topics: Animals; Fibrosis; Liver Cirrhosis, Biliary; Liver Function Tests; Male; Necrosis; Rats; Rats, Wista | 2009 |
The protective effect of thalidomide on left ventricular function in a rat model of diabetic cardiomyopathy.
Topics: Analysis of Variance; Animals; Antibiotics, Antineoplastic; Collagen; Diabetes Mellitus, Type 1; Dia | 2010 |
Pomalidomide is effective for prevention and treatment of experimental skin fibrosis.
Topics: Animals; Disease Models, Animal; Fibrosis; Hydroxyproline; Immunosuppressive Agents; Mice; Mice, Inb | 2012 |
Thalidomide in the treatment of myelodysplastic syndrome with fibrosis.
Topics: Aged; Bone Marrow; Female; Fibrosis; Humans; Myelodysplastic Syndromes; Thalidomide; Treatment Outco | 2002 |
Thalidomide attenuates the development of fibrosis during post-infarction myocardial remodelling in rats.
Topics: Animals; Blood Pressure; Cytokines; Disease Models, Animal; Fibrosis; Gene Expression; Hydroxyprolin | 2006 |
Anti-fibrotic effects of thalidomide on hepatic stellate cells and dimethylnitrosamine-intoxicated rats.
Topics: Actins; Animals; Collagen; Dimethylnitrosamine; Fibrosis; I-kappa B Proteins; Immunosuppressive Agen | 2006 |
Cutaneous sarcoidosis successfully treated with low doses of thalidomide.
Topics: Adult; Cicatrix; Female; Fibrosis; Humans; Immunosuppressive Agents; Sarcoidosis; Skin Diseases; Tha | 1998 |