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1.
Pain ; 2021 Apr 08.
Artigo em Inglês | MEDLINE | ID: mdl-33863862

RESUMO

ABSTRACT: Randomized clinical trials have demonstrated the efficacy of opioid analgesics for the treatment of acute and chronic pain conditions, and for some patients, these medications may be the only effective treatment available. Unfortunately, opioid analgesics are also associated with major risks (e.g., opioid use disorder) and adverse outcomes (e.g., respiratory depression and falls). The risks and adverse outcomes associated with opioid analgesics have prompted efforts to reduce their use in the treatment of both acute and chronic pain. This article presents IMMPACT consensus recommendations for the design of opioid sparing clinical trials. The recommendations presented in this article are based on the following definition of an opioid-sparing intervention: any intervention that (1) prevents the initiation of treatment with opioid analgesics, (2) decreases the duration of such treatment, (3) reduces the total dosages of opioids that are prescribed for or used by patients, or (4) reduces opioid-related adverse outcomes (without increasing opioid dosages), all without causing an unacceptable increase in pain. These recommendations are based on the results of a background review, presentations and discussions at an IMMPACT consensus meeting, and iterative drafts of this article modified to accommodate input from the co-authors. We discuss opioid sparing definitions, study objectives, outcome measures, the assessment of opioid-related adverse events, incorporation of adequate pain control in trial design, interpretation of research findings, and future research priorities to inform opioid-sparing trial methods. The considerations and recommendations presented in this article are meant to help guide the design, conduct, analysis, and interpretation of future trials.

2.
Artigo em Inglês | MEDLINE | ID: mdl-33483129

RESUMO

BACKGROUND: Underreporting of harms in randomized controlled trials (RCTs) may lead to incomplete or erroneous assessments of the perceived benefit-to-harm profile of an intervention. To compare benefit with harm in clinical practice and future clinical studies, adverse event (AE) profiles including severity need to be understood. Even though patients report harm symptoms earlier and more frequently than clinicians, rheumatology RCTs currently do not provide a reporting framework from the patient's perspective regarding harms. Our objective for this meta-research project was to identify AEs in order to determine harm clusters and whether these could be self-reported by patients. Our other objective was to examine reported severity grading of the reported harms. METHODS: We considered primary publications of RCTs eligible if they were published between 2008 and 2018 evaluating pharmacological interventions in patients with a rheumatic or musculoskeletal condition and if they were included in Cochrane reviews. We extracted data on harms such as reported AE terms together with severity (if described), and categorized AE- and severity-terms into overall groups. We deemed all AEs with felt components appropriate for patient self-reporting. RESULTS: The literature search identified 187 possible Cochrane reviews, of which 94 were eligible for evaluation, comprising 1,297 articles on individual RCTs. Of these RCTs, 93 pharmacological trials met our inclusion criteria (including 31,023 patients; representing 20,844 accumulated patient years), which reported a total of 21,498 AEs, corresponding to 693 unique reported terms for AEs. We further sub-categorized these terms into 280 harm clusters (i.e., themes). AEs appropriate for patient self-reporting accounted for 58% of the AEs reported. Among the reported AEs, we identified medical terms for all of the 117 harm clusters appropriate for patient reporting and lay language terms for 86%. We intended to include severity grades of the reported AEs, but there was no evidence for systematic reporting of clinician- or patient-reported severity in the primary articles of the 93 trials. However, we identified 33 terms suggesting severity, but severity grading was discernible in only 9%, precluding a breakdown by severity in this systematic review. CONCLUSIONS: Our results support the need for a standardized framework for patients' reporting of harms in rheumatology trials. Reporting of AEs with severity should be included in future reporting of harms, both from the patients' and investigators' perspectives. REGISTRATION: PROSPERO: CRD42018108393.

3.
Semin Arthritis Rheum ; 51(1): 278-284, 2021 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-33412435

RESUMO

Pain is a manifestation of rheumatoid arthritis (RA) that is mediated by inflammatory and non-inflammatory mechanisms and negatively affects quality of life. Recent findings from a Phase 3 clinical trial showed that patients with RA who were treated with a Janus kinase 1 (Jak1) and Janus kinase 2 (Jak2) inhibitor achieved significantly greater improvements in pain than those treated with a tumor necrosis factor blocker; both treatments resulted in similar changes in standard clinical measures and markers of inflammation. These findings suggest that Jak1 and Jak2 inhibition may relieve pain in RA caused by inflammatory and non-inflammatory mechanisms and are consistent with the overarching involvement of the Jak-signal transducer and activator of transcription (Jak/STAT) pathway in mediating the action, expression, and regulation of a multitude of pro- and anti-inflammatory cytokines. In this review, we provide an overview of pain in RA, the underlying importance of cytokines regulated directly or indirectly by the Jak/STAT pathway, and therapeutic targeting of the Jak/STAT pathway in RA. As highlighted herein, multiple cytokines directly or indirectly regulated by the Jak/STAT pathway play important roles in mediating various mechanisms underlying pain in RA. Having a better understanding of these mechanisms may help clinicians make treatment decisions that optimize the control of inflammation and pain.

5.
Semin Arthritis Rheum ; 50(6): 1203-1213, 2020 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-33059293

RESUMO

PURPOSE: Osteoarthritis Research Society International (OARSI) Expert Consensus Guidelines recommend topical non-steroidal anti-inflammatory drugs as first-line medications for osteoarthritis (OA) knee pain, but several voluminous daily applications are required to achieve efficacy. There is a need to develop new and improved topical analgesics with a faster onset, longer duration of action, and the requirement to apply less gel. This trial investigated the safety and efficacy of a new 3.06% diclofenac gel (AMZ001) in subjects with knee OA. METHODS: In total, 444 subjects (AMZ001 twice daily (BID) [n = 121], AMZ001 once daily (QD) + placebo QD [n = 121], placebo BID [n = 121], or Voltaren 1% 4-times daily [n = 81]) were enrolled. All except Voltaren 1% (single-blinded) were applied topically in a double-blind manner for a total of 4-weeks. The primary endpoint was the change from baseline to week 4 in the WOMAC pain sub-score in the target knee. Secondary and exploratory endpoints included additional efficacy measures (WOMAC total score, WOMAC function and stiffness sub-scores, WOMAC pain weight-bearing and non-weight-bearing sub-scores, ICOAP, chair-stand test, OMERACT-OARSI responder rate, PGA, WPAI, EQ-5D, rescue medication use, satisfaction questionnaire) and safety. RESULTS: Treatment with AMZ001 QD was effective at reducing WOMAC pain sub-scores vs placebo (estimated treatment difference [ETD]: -4.61 [95% confidence interval (CI): -9.09, -0.12]; p = 0.0440); however, BID application was not (ETD: -3.76 [95% CI: -8.21, 0.68]; p = 0.0969). For several secondary endpoints, changes from baseline to week 4 conferred nominally statistically significant improvements in favor of AMZ001 vs placebo, including PGA score (AMZ001 BID vs placebo, ETD: -0.61 [95% CI: -1.11, -0.11]; p = 0.0162; AMZ001 QD vs placebo, ETD: -0.63 [95% CI: -1.13, -0.13]; p = 0.0134), WPAI overall work impairment score (AMZ001 QD vs placebo, ETD: -10.44 [95% CI: -20.84, -0.04]; p = 0.0492), and EQ-5D VAS score (AMZ001 BID vs placebo, ETD: 4.70 [95% CI: 0.55, 8.85]; p = 0.0264). Post-hoc analysis excluding 11-14 subjects per group with pain scores that decreased between screening and baseline suggests a consistent effect of both AMZ001 QD (ETD: -5.84 [95% CI: -10.71, -0.97]; p = 0.0189) and BID (ETD: -5.35 [95% CI: -10.16, -0.54]; p = 0.0292) in reducing WOMAC pain sub-scores vs placebo. In general, treatment satisfaction was high, as measured by the satisfaction questionnaire. The frequency and incidence of adverse events (AEs) was greatest in the placebo group. Most AEs (>99%) were of mild or moderate severity. There were no serious AEs. There were no notable effects of any treatment on vital signs, ECGs, physical examination findings, or other laboratory assessments. CONCLUSIONS: Treatment with AMZ001 BID for 4 weeks improved WOMAC pain sub-scores; however, only QD application conferred nominally statistically significant improvements vs placebo. AMZ001 was generally well tolerated.

6.
Lupus Sci Med ; 7(1)2020 06.
Artigo em Inglês | MEDLINE | ID: mdl-32591423

RESUMO

OBJECTIVE: The heterogeneous multisystem manifestations of SLE include fatigue, pain, depression, sleep disturbance and cognitive dysfunction, and underscore the importance of a multidimensional approach when assessing health-related quality of life. The US Food and Drug Administration has emphasised the importance of patient-reported outcomes (PROs) for approval of new medications and Outcome Measures in Rheumatology has mandated demonstration of appropriate measurement properties of selected PRO instruments. METHODS: Published information regarding psychometric properties of the Medical Outcomes Survey Short Form 36 (SF-36), Lupus Quality of Life Questionnaire (LupusQoL) and Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-F), and their suitability as end points in randomised controlled trials (RCTs) and longitudinal observational studies (LOS) were assessed. A search of English-language literature using MEDLINE and EMBASE identified studies related to development and validation of these instruments. Evidence addressed content validity, reliability (internal consistency and test-retest reliability), construct validity (convergent and divergent) and longitudinal responsiveness, including thresholds of meaning and discrimination. RESULTS: All instruments demonstrated strong internal consistency, reliability and appropriate face/content validity, indicating items within each instrument that measure the intended concept. SF-36 and LupusQoL demonstrated test-retest reliability; although not published with FACIT-F in SLE supported by evidence from other rheumatic diseases. All instruments demonstrated convergent validity with other comparable PROs and responsivity to treatment. CONCLUSION: The measurement properties of PRO instruments with published data from RCTs including: SF-36, LupusQoL and FACIT-F indicate their value as secondary end points to support labelling claims in RCTs and LOS evaluating the efficacy of SLE treatments.

7.
J Rheumatol ; 47(9): 1379-1384, 2020 09 01.
Artigo em Inglês | MEDLINE | ID: mdl-32007937

RESUMO

OBJECTIVE: To survey participants with polymyalgia rheumatica (PMR) to evaluate the face validity, acceptability, and domain match of proposed candidate outcome measures. METHODS: A structured, online, anonymous survey was disseminated by patient support groups through their networks and online forums. The candidate outcome measures comprised (1) visual analog scale (VAS) and numerical rating score (NRS) to assess pain; (2) VAS, NRS, and duration to assess stiffness; (3) the modified Health Assessment Questionnaire and Health Assessment Questionnaire Disability Index to assess physical function; and (4) C-reactive protein and erythrocyte sedimentation rate to assess inflammation. Free-text answers were analyzed using descriptive thematic analysis to determine respondents' views of the candidate instruments. RESULTS: Seventy-eight people with PMR from 6 countries (UK, France, USA, Canada, Australia, and New Zealand) participated in the survey. Most respondents agreed candidate instruments were acceptable or "good to go." Free-text analysis identified 5 themes that participants considered inadequately covered by the proposed instruments. These related to (1) the variability, context, and location of pain; (2) the variability of stiffness; (3) fatigue; (4) disability; and (5) the correlation of inflammatory marker levels and severity of symptoms, sometimes reflecting disease activity and other times not. CONCLUSION: Participants reported additional aspects of their experience that are not covered by the proposed instruments, particularly for the experience of stiffness and effect of fatigue. New patient-reported outcome measures are required to increase the relevance of results from clinical trials to patients with PMR.

8.
J Clin Rheumatol ; 2020 Jan 24.
Artigo em Inglês | MEDLINE | ID: mdl-31985721

RESUMO

BACKGROUND/OBJECTIVE: The objective of this prospective cohort study was to understand the positive and negative effects of glucocorticoids (GCs) in patients with systemic lupus erythematosus and myositis from the patients' perspective with the aim of developing a patient-reported outcome measure. METHODS: Included patients were asked to participate in 1 of 5 nominal groups where demographic information and a quality-of-life questionnaire were collected. Patients were asked 2 open-ended questions on (1) benefits and (2) harms related to GC use. We used the Nominal Group Technique, a highly structured consensus method in which responses are generated, shared, and ranked. Descriptive statistics were used to summarize the results. Nominal group sessions took place from April to May 2019. RESULTS: Of 206 patients who were approached, 21 patients participated, 17 with systemic lupus erythematosus and 4 with myositis, predominantly women with more than 10 years of steroid use. The domains ranked highest for GC benefits were disease control (55 votes), fast onset of action (30 votes), increased energy (10 votes), and pain relief (10 votes). The highest-ranked negative effects were bone loss (38 votes) and weight gain (16 votes); psychological effects and damaged internal organs each received 12 votes. CONCLUSIONS: The top-ranked GC effects-both benefits and harms-among patients with systemic rheumatic disease are consistent with the top domains associated with GC use reported with other inflammatory diseases. This study informs the development of a comprehensive patient-reported outcome measure that can be used across inflammatory diseases.

9.
J Pain ; 21(9-10): 931-942, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-31843583

RESUMO

The estimated probability of progressing from phase 3 analgesic clinical trials to regulatory approval is approximately 57%, suggesting that a considerable number of treatments with phase 2 trial results deemed sufficiently successful to progress to phase 3 do not yield positive phase 3 results. Deficiencies in the quality of clinical trial conduct could account for some of this failure. An Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials meeting was convened to identify potential areas for improvement in trial conduct in order to improve assay sensitivity (ie, ability of trials to detect a true treatment effect). We present recommendations based on presentations and discussions at the meeting, literature reviews, and iterative revisions of this article. The recommendations relate to the following areas: 1) study design (ie, to promote feasibility), 2) site selection and staff training, 3) participant selection and training, 4) treatment adherence, 5) data collection, and 6) data and study monitoring. Implementation of these recommendations may improve the quality of clinical trial data and thus the validity and assay sensitivity of clinical trials. Future research regarding the effects of these strategies will help identify the most efficient use of resources for conducting high quality clinical trials. PERSPECTIVE: Every effort should be made to optimize the quality of clinical trial data. This manuscript discusses considerations to improve conduct of pain clinical trials based on research in multiple medical fields and the expert consensus of pain researchers and stakeholders from academia, regulatory agencies, and industry.

10.
J Rheumatol ; 46(9): 1173-1178, 2019 09.
Artigo em Inglês | MEDLINE | ID: mdl-31043547

RESUMO

OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) Safety Working Group objective was to identify harm domains from existing outcome measurements in rheumatology. METHODS: Systematically searching the MEDLINE database on January 24, 2017, we identified full-text articles that could be used for harm outcomes in rheumatology. Domains/items from the identified instruments were described and the content synthesized to provide a preliminary framework for harm outcomes. RESULTS: From 435 possible references, 24 were read in full text and 9 were included: 7 measurement instruments were identified. Investigation of domains/items revealed considerable heterogeneity in the grouping and approach. CONCLUSION: The ideal way to assess harm aspects from the patients' perspective has not yet been ascertained.


Assuntos
Antirreumáticos/uso terapêutico , Ensaios Clínicos como Assunto , Doenças Reumáticas/tratamento farmacológico , Humanos , Avaliação de Resultados em Cuidados de Saúde , Reumatologia
11.
J Rheumatol ; 46(10): 1355-1359, 2019 10.
Artigo em Inglês | MEDLINE | ID: mdl-30936284

RESUMO

OBJECTIVE: Establishing a research agenda on standardizing pain measurement in clinical trials in rheumatic and musculoskeletal diseases (RMD). METHODS: Discussion during a meeting at the Outcome Measures in Rheumatology (OMERACT) 2018, prepared by a systematic review of existing core outcome sets and a patient online survey. RESULTS: Several key questions were debated: Is pain a symptom or a disease? Are pain core (sub)domains consistent across RMD? How to account for pain mechanistic descriptors (e.g., central sensitization) in pain measurement? CONCLUSION: Characterizing and assessing the spectrum of pain experience across RMD in a standardized fashion is the objective of the OMERACT Pain Working Group.


Assuntos
Dor Crônica/diagnóstico , Doenças Musculoesqueléticas/fisiopatologia , Avaliação de Resultados em Cuidados de Saúde/métodos , Medição da Dor/métodos , Doenças Reumáticas/fisiopatologia , Consenso , Inquéritos Epidemiológicos , Humanos , Opinião Pública , Qualidade de Vida , Reumatologia/métodos
12.
J Rheumatol ; 46(8): 1014-1020, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-30770502

RESUMO

OBJECTIVE: To describe the Outcome Measures in Rheumatology (OMERACT) Filter 2.1 methodology for core domain set selection. METHODS: The "OMERACT Way for Core Domain Set selection" framework consists of 3 stages: first, generating candidate domains through literature reviews and qualitative work, then a process of consensus to obtain agreement from those involved, and finally formal voting on the OMERACT Onion. The OMERACT Onion describes the placement of domains in layers/circles: mandatory in all trials/mandatory in specific circumstances (inner circle); important but optional (middle circle); or research agenda (outer circle). Five OMERACT working groups presented their core domain sets for endorsement by the OMERACT community. Tools including a workbook and whiteboard video were created to assist the process. The methods workshop at OMERACT 2018 introduced participants to this framework. RESULTS: The 5 OMERACT working groups achieved consensus on their proposed core domain sets. After the Methodology Workshop training exercise at OMERACT 2018, over 90% of participants voted that they were confident that they understood the process of core domain set selection. CONCLUSION: The methods described in this paper were successfully used by the 5 working groups voting on domains at the OMERACT 2018 meeting, demonstrating the feasibility of the process. In addition, participants at OMERACT 2018 expressed increased confidence and understanding of the core domain set selection process after the training exercise. This methodology will continue to evolve, and we will use innovative technology such as whiteboard videos as a key part of our dissemination and implementation strategy for new methods.


Assuntos
Ensaios Clínicos como Assunto , Avaliação de Resultados em Cuidados de Saúde , Reumatologia , Consenso , Humanos
13.
J Rheumatol ; 46(10): 1406-1408, 2019 10.
Artigo em Inglês | MEDLINE | ID: mdl-30770514

RESUMO

OBJECTIVE: Adaptive trial design was developed initially for oncology to improve trial efficiency. If optimized for rheumatology, it may improve trial efficiency by reducing sample size and time. METHODS: A systematic review assessed design of phase II clinical trials in rheumatoid arthritis. RESULTS: Fifty-six trials were reviewed. Most trials had 4 groups (1 control and 3 intervention), with an average group size of 34 patients. American College of Rheumatology 20 measured at 16 weeks was the most commonly used primary endpoint. CONCLUSION: The next step is to undertake a systematic review of adaptive designs used in early-phase trials in nonrheumatic conditions.


Assuntos
Antirreumáticos/uso terapêutico , Artrite Reumatoide/tratamento farmacológico , Ensaios Clínicos como Assunto , Projetos de Pesquisa , Reumatologia/métodos , Humanos , Avaliação de Resultados em Cuidados de Saúde , Opinião Pública
14.
J Rheumatol ; 46(8): 1021-1027, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-30770515

RESUMO

OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) Filter 2.0 framework was developed in 2014 to aid core outcome set development by describing the full universe of "measurable aspects of health conditions" from which core domains can be selected. This paper provides elaborations and updated concepts (OMERACT Filter 2.1). METHODS: At OMERACT 2018, we discussed challenges in the framework application caused by unclear or ambiguous wording and terms and incompletely developed concepts. RESULTS: The updated OMERACT Filter 2.1 framework makes benefits and harms explicit, clarifies concepts, and improves naming of various terms. CONCLUSION: We expect that the Filter 2.1 framework will improve the process of core set development.


Assuntos
Antirreumáticos/uso terapêutico , Ensaios Clínicos como Assunto , Indicadores Básicos de Saúde , Avaliação de Resultados em Cuidados de Saúde , Doenças Reumáticas/tratamento farmacológico , Humanos , Reumatologia
15.
J Rheumatol ; 46(9): 1168-1172, 2019 09.
Artigo em Inglês | MEDLINE | ID: mdl-30770516

RESUMO

OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) Safety Working Group is identifying core safety domains that matter most to patients with rheumatic disease. METHODS: International focus groups were held with 39 patients with inflammatory arthritis to identify disease-modifying antirheumatic drug (DMARD) experiences and concerns. Themes were identified by pragmatic thematic coding and discussed in small groups by meeting attendees. RESULTS: Patients view DMARD side effects as a continuum and consider the cumulative effect on day-to-day function. Disease and drug experiences, personal factors, and life circumstances influence tolerance of side effects and treatment persistence. CONCLUSION: Patients weigh overall adverse effects and benefits over time in relation to experiences and life circumstances.


Assuntos
Antirreumáticos/efeitos adversos , Artrite Reumatoide/tratamento farmacológico , Adulto , Antirreumáticos/uso terapêutico , Feminino , Grupos Focais , Humanos , Masculino , Satisfação do Paciente , Reumatologia
16.
J Rheumatol ; 46(8): 1028-1035, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-30709952

RESUMO

OBJECTIVE: Outcome Measures in Rheumatology (OMERACT) Filter 2.1 revised the process used for core outcome measurement set selection to add rigor and transparency in decision making. This paper describes OMERACT's methodology for instrument selection. METHODS: We presented instrument selection processes, tools, and reporting templates at OMERACT 2018, introducing the concept of "3 pillars, 4 questions, 7 measurement properties, 1 answer." Truth, discrimination, and feasibility are the 3 original OMERACT pillars. Based on these, we developed 4 signaling questions. We introduced the Summary of Measurement Properties table that summarizes the 7 measurement properties: truth (domain match, construct validity), discrimination [test-retest reliability, longitudinal construct validity (responsiveness), clinical trial discrimination, thresholds of meaning], and feasibility. These properties address a set of standards which, when met, answer the one question: Is there enough evidence to support the use of this instrument in clinical research of the benefits and harms of treatments in the population and study setting described? The OMERACT Filter 2.1 was piloted on 2 instruments by the Psoriatic Arthritis Working Group. RESULTS: The methodology was reviewed in a full plenary session and facilitated breakout groups. Tools to facilitate retention of the process (i.e., "The OMERACT Way") were provided. The 2 instruments were presented, and the recommendation of the working group was endorsed in the first OMERACT Filter 2.1 Instrument Selection votes. CONCLUSION: Instrument selection using OMERACT Filter 2.1 is feasible and is now being implemented.


Assuntos
Antirreumáticos/uso terapêutico , Artrite Psoriásica/tratamento farmacológico , Ensaios Clínicos como Assunto , Avaliação de Resultados em Cuidados de Saúde , Reumatologia/normas , Humanos , Reprodutibilidade dos Testes
17.
J Rheumatol ; 46(10): 1360-1364, 2019 10.
Artigo em Inglês | MEDLINE | ID: mdl-30709960

RESUMO

OBJECTIVE: To report the progress of the Outcome Measures in Rheumatology (OMERACT) Polymyalgia Rheumatica (PMR) Working Group in selecting candidate instruments for a core outcome measurement set. METHODS: A systematic literature review identified outcomes measured and instruments used in PMR studies, and a respondent survey and raw data analysis assessed their domain match and feasibility. RESULTS: Candidate instruments were identified for pain [visual analog scale/numerical rating scale (VAS/NRS)], stiffness (VAS/NRS and duration), and physical function (Health Assessment Questionnaire-Disability Index/modified Health Assessment Questionnaire). Domain match and feasibility assessments were favorable; however, validation in PMR was lacking. CONCLUSION: Further assessment of candidate instruments is required prior to recommending a PMR core outcome measurement set.


Assuntos
Avaliação de Resultados em Cuidados de Saúde/métodos , Polimialgia Reumática/fisiopatologia , Polimialgia Reumática/terapia , Adulto , Idoso , Idoso de 80 Anos ou mais , Sedimentação Sanguínea , Proteína C-Reativa/análise , Estudos de Viabilidade , Feminino , Inquéritos Epidemiológicos , Humanos , Masculino , Pessoa de Meia-Idade , Dor , Medição da Dor , Opinião Pública , Escala Visual Analógica
18.
J Rheumatol ; 46(9): 1179-1182, 2019 09.
Artigo em Inglês | MEDLINE | ID: mdl-30647165

RESUMO

OBJECTIVE: To understand the effects of glucocorticoids (GC), which are of importance to patients. METHODS: The results of 2 literature reviews, a patient survey, and a qualitative study were presented. RESULTS: No validated instrument exists to evaluate GC effect on patients. Survey data revealed skin thinning/bruising, sleep disturbance, and weight gain as the most frequent adverse effects. The qualitative research yielded rich data covering rapid benefits and physical and emotional consequences of GC. CONCLUSION: It was agreed that a patient-reported outcome to measure GC effect was required and a research agenda was developed for this goal.


Assuntos
Glucocorticoides/uso terapêutico , Doenças Reumáticas/tratamento farmacológico , Glucocorticoides/efeitos adversos , Humanos , Medidas de Resultados Relatados pelo Paciente , Resultado do Tratamento
19.
J Rheumatol ; 46(8): 1053-1058, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-30647191

RESUMO

OBJECTIVE: Outcome Measures in Rheumatology (OMERACT) convened a premeeting in 2018 to bring together patients, regulators, researchers, clinicians, and consumers to build upon previous OMERACT drug safety work, with patients fully engaged throughout all phases. METHODS: Day 1 included a brief introduction to the history of OMERACT and methodology, and an overview of current efforts within and outside OMERACT to identify patient-reported medication safety concerns. On Day 2, two working groups presented results; after each, breakout groups were assembled to discuss findings. RESULTS: Five themes pertaining to drug safety measurement emerged. CONCLUSION: Current approaches have failed to include data from the patient's perspective. A better understanding of how individuals with rheumatic diseases view potential benefits and harms of therapies is essential.


Assuntos
Antirreumáticos/uso terapêutico , Satisfação do Paciente , Ensaios Clínicos Controlados Aleatórios como Assunto , Doenças Reumáticas/tratamento farmacológico , Humanos , Medição de Risco , Resultado do Tratamento
20.
Arthritis Rheumatol ; 70(9): 1450-1458, 2018 09.
Artigo em Inglês | MEDLINE | ID: mdl-29648686

RESUMO

OBJECTIVE: Trials of new systemic lupus erythematosus (SLE) treatments are hampered by the lack of effective outcome measures. To address this, we developed a novel Lupus Multivariable Outcome Score (LuMOS) and assessed its performance using data from 2 randomized controlled trials of belimumab in patients with SLE. METHODS: The LuMOS formula was developed by analyzing raw data from 2 pivotal trials, the Study of Belimumab in Subjects with SLE 52-week (BLISS-52) and 76-week (BLISS-76) trials, which are the basis for approval of belimumab. Using the BLISS-76 trial data as the learning data set, we carried out multivariable logistic regression analyses to optimize discrimination of outcomes between patients treated with 10 mg/kg belimumab and patients receiving placebo over the first 52 weeks of follow-up. In addition, the performance of LuMOS was assessed using an independent validation data set from the BLISS-52 trial. RESULTS: The LuMOS model incorporated the following response criteria: a ≥4-point reduction on the Safety of Estrogens in Lupus Erythematosus National Assessment version of the SLE Disease Activity Index, an increase in C4 levels, a decrease in anti-double-stranded DNA titers, and changes in the British Isles Lupus Assessment Group scores for organ system manifestations (no worsening in renal components, and improvements in mucocutaneous components). A decrease in the prednisone dose and increase in C3 levels had very minor impacts on the total LuMOS score. In all analyses of the BLISS-76 and BLISS-52 trial data sets, the mean LuMOS scores were significantly higher (P < 0.0001) in patients treated with 1 mg or 10 mg belimumab compared to placebo. In contrast to the performance of the SLE Responder Index 4 (SRI-4), the LuMOS revealed significant differences between the active treatment group (1 mg belimumab in the BLISS-76 cohort) and placebo group. The effect sizes were significantly much higher with the LuMOS than with the SRI-4. CONCLUSION: The evidenced-based LuMOS outcome scoring system, developed with data from the BLISS-76 trial of belimumab in patients with SLE and validated with data from the BLISS-52 trial, exhibits a superior capacity to discriminate responders from nonresponders when compared to the SRI-4. Use of the LuMOS may improve the efficiency and power of analyses in future lupus trials.


Assuntos
Anticorpos Monoclonais Humanizados/uso terapêutico , Imunossupressores/uso terapêutico , Lúpus Eritematoso Sistêmico/tratamento farmacológico , Avaliação de Resultados em Cuidados de Saúde/métodos , Índice de Gravidade de Doença , Adulto , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Análise Multivariada , Ensaios Clínicos Controlados Aleatórios como Assunto , Resultado do Tratamento
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