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BACKGROUND/PURPOSE: Endoscopic remission is presently recognized as the standard therapeutic target in the treatment of ulcerative colitis (UC). However, achieving histological remission is increasingly viewed as a pivotal objective. This study investigates the effects of attaining completely histological remission on the clinical outcomes for UC patients with a high disease burden who have already reached endoscopic remission. This is the inaugural study to concentrate on this specific patient demographic. METHODS: This retrospective cohort study enrolled moderate-to-severe, biologics-experienced UC patients with completely endoscopic remission (Mayo endoscopic subscore of 0) between June 2017 and October 2023 at Chang Gung Memorial Hospital, Linkou. Patients were classified into histological remission (HR) and non-histological remission (non-HR) groups based on the Nancy index (NI). HR was defined as an NI score of 0, with all other patients categorized as non-HR. The definition of flare-ups was based on both clinical and endoscopic evidence. Comparative analyses focused on baseline characteristics and clinical outcomes at follow-up. RESULTS: A total of 42 patients (HR group: 23, non-HR group: 19) were included. The average follow-up duration was 17.6 months. Baseline characteristics were comparable between the groups. At the end of follow-up, the HR group showed a significantly lower rate of acute flare-ups (26.1% vs. 68.4%, P = 0.006). Although not statistically significant, the HR group also experienced fewer emergency department visits and hospital admissions. CONCLUSIONS: For moderate-to-severe, biologics-experienced UC patients in endoscopic remission, achieving completely histological remission is associated with a substantial reduction in flare-ups, suggesting its potential as a valuable therapeutic target.
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Background: This study aims to explore the clinical value of low disease activity state (LDAS) in the treat-to-target strategy of pediatric systemic lupus erythematosus (pSLE) and find the risk factors for never reaching LDAS. Methods: A total of 272 children with SLE who were diagnosed and followed up in two tertiary hospitals in China during the period from January 2012 to December 2019 were involved in this study, and the clinical presentation, pathology, and treatment were retrospectively studied. Results: The male-to-female ratio was 1:5.2, the age at diagnosis was 11.1 years (IQR, 9.8-13.1 years), the disease duration was 1.0 month (IQR, 0.5-2.0 months), and follow-up was 36.5 months (IQR, 25.7-50.9 months). During follow-up, 230 children achieved LDAS, and 42 were never been in. Male (P = 0.018), mucosal ulcer (P = 0.048), liver function damage (P = 0.026), cardiac effusion (P = 0.034), anemia (P = 0.048), urine red blood cells (P = 0.017), urinary leukocytes (P = 0.032), and endothelial cell proliferation in renal biopsy (P = 0.004)-these indexes have statistical differences between the two groups in the baseline. At baseline, endothelial cell proliferation (P = 0.02) is an independent risk factor for never achieving LDAS by multivariate logistic analysis. During follow-up, non-compliance was a risk factor for never achieving LDAS by comparing between groups. Children with biologics achieved LDAS at a higher rate than children without biologics (P = 0.038). The proportion of organ damage in patients never been in LDAS was significantly higher than that in patients who achieved LDAS (P < 0.001). Conclusion: Endothelial cell proliferation in renal biopsy and non-compliance during follow-up were independent risk factors for never achieving LDAS. At the end of the follow-up, the organ damage in the remission group was similar to that in the LDAS group, indicating that LDAS can be used as a target for pSLE treatment.
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Lúpus Eritematoso Sistêmico , Humanos , Lúpus Eritematoso Sistêmico/complicações , Lúpus Eritematoso Sistêmico/diagnóstico , Masculino , Feminino , Criança , Estudos Retrospectivos , Adolescente , China/epidemiologia , Fatores de Risco , Índice de Gravidade de Doença , Seguimentos , Prognóstico , Resultado do Tratamento , População do Leste AsiáticoRESUMO
BACKGROUND: Crohn's disease and ulcerative colitis (UC) are inflammatory bowel diseases (IBD) characterized by a progressive nature of the disease resulting in subsequent intestinal damage, limited efficacy of current treatments and suboptimal disease management and a significant burden for patients. OBJECTIVES: The IBD-PODCAST study aims to estimate the proportion of Crohn's disease and UC patients with suboptimal disease control (SDC) in a real-world setting. METHODS: A non-interventional and cross-sectional study was conducted across 103 sites in 10 countries (Austria, Belgium, Canada, Germany, Greece, Italy, Portugal, Spain, Turkey, and UK). Criteria for SDC were based on STRIDE-II criteria and adapted by an expert panel. RESULTS: 2185 patients (Crohn's disease: n = 1,108, UC: n = 1077) with a mean (SD) age of 44.0 (14.8) years and mean (SD) disease duration of 12.4 (9.2) years were included (52.2% male). Ileal involvement was present in 39.1% of Crohn's disease patients, 35.3% of UC patients had extensive colitis. 77.3% of Crohn's disease and 65.3% of UC patients were on targeted immunomodulators and, according to STRIDE-II-based treatment phases, 85.6% of Crohn's disease and 85.4% of UC patients were assigned to the long-term treatment phase. SDC was detected in 52.2% of Crohn's disease and 44.3% of UC patients predominantly due to impaired quality of life (QoL), clinically significant extraintestinal manifestations, steroid overuse, signs of active inflammation in UC and Crohn's disease, and active fistulas in Crohn's disease. More than one criterion was seen in 37% of patients with SDC. Opportunities for on-label treatment optimization were observed in 49% of Crohn's disease and 61% of UC patients on advanced therapy. CONCLUSION: The high percentage of SDC in this global, real-world cohort suggests a large disease burden and high unmet medical need in IBD patients. Future analysis should focus on monitoring and responding to SDC in this cohort and on patients' QoL.
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Colite Ulcerativa , Doença de Crohn , Humanos , Masculino , Feminino , Estudos Transversais , Adulto , Doença de Crohn/complicações , Doença de Crohn/terapia , Doença de Crohn/epidemiologia , Doença de Crohn/diagnóstico , Pessoa de Meia-Idade , Colite Ulcerativa/complicações , Colite Ulcerativa/terapia , Colite Ulcerativa/epidemiologia , Colite Ulcerativa/diagnóstico , Colite Ulcerativa/tratamento farmacológico , Qualidade de Vida , Europa (Continente)/epidemiologia , Índice de Gravidade de Doença , Resultado do TratamentoRESUMO
BACKGROUND: Crohn's disease (CD) and ulcerative colitis (UC) are inflammatory bowel diseases (IBD) that contributes in part to irreversible bowel damage and long-term complications, reduced quality of life, invalidity, and economic burden. Suboptimal control of IBD is associated with higher healthcare resource utilization (HCRU), impaired quality of life (QoL), and reduced work productivity. AIMS: The IBD-PODCAST study aimed to assess the proportion of IBD patients with suboptimal control and its associated impact. METHODS: IBD-PODCAST is a cross-sectional, multicenter study that aimed to characterize the CD and UC population with optimal or suboptimal control according to the STRIDE-II criteria and patient- and physician-reported measures. Here we present the results of the Spanish cohort (n = 396). RESULTS: A total of 104/196 (53.1%) CD and 83/200 (41.5%) UC patients were found to have suboptimal disease control. Long-term treatment targets according to STRIDE-II were applied in 172 (87.8%) CD and 181 (90.5%) UC patients. 125 of 172 (72.7%) CD and 74 of 181 (40.9%) UC patients were currently treated with targeted immunomodulators. Patients with CD and UC and suboptimal disease control showed impaired QoL, higher HCRU and direct costs, and also loss of work productivity compared to those with optimal control. CONCLUSION: Despite a high rate of targeted immunomodulator therapy, a substantial proportion of IBD patients show suboptimal disease control according to the STRIDE II criteria. Those patients with suboptimal disease control exhibit impaired QoL, less work productivity, and higher HCRU, suggesting that there is considerable need for better treatment approaches in IBD.
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Colite Ulcerativa , Doença de Crohn , Doenças Inflamatórias Intestinais , Humanos , Qualidade de Vida , Espanha/epidemiologia , Estudos Transversais , Doenças Inflamatórias Intestinais/tratamento farmacológico , Doenças Inflamatórias Intestinais/epidemiologia , Doença de Crohn/diagnóstico , Doença de Crohn/tratamento farmacológico , Colite Ulcerativa/diagnóstico , Colite Ulcerativa/tratamento farmacológico , Fatores Imunológicos/uso terapêuticoRESUMO
Familial Mediterranean fever is the most common monogenic auto-inflammatory disease in the world. It mainly affects people originating from the Mediterranean region. The mutated gene is MEFV, which codes for pyrin. Transmission is autosomal recessive. Patients present with recurrent attacks of fever since childhood associated with abdominal and/or thoracic pain lasting an average of 2-3days and a biological inflammatory syndrome. Other symptoms include arthralgia or arthritis in large joints such as the knees and ankles, myalgia in the lower limbs and pseudo-erysipelas in the ankles. The most serious complication is inflammatory amyloidosis, which can lead to kidney failure. Treatment is based on colchicine, which helps to prevent flares and the onset of renal amyloidosis. This paper proposes national guidelines for the diagnosis, management and follow-up of familial Mediterranean fever in France, where we estimate there are between 5000 and 10,000 patients with the disease at all stages of life. The diagnosis is suspected on the basis of clinical and anamnestic factors and confirmed by genetic analysis. These guidelines also suggest a "treat-to-target" approach to disease management, particularly in case of suspected colchicine resistance - a very rare situation that should remain a diagnosis of elimination, especially after colchicine compliance has been verified. Two special situations are also addressed in these guidelines: kidney failure and pregnancy.
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Amiloidose , Febre Familiar do Mediterrâneo , Insuficiência Renal , Humanos , Criança , Febre Familiar do Mediterrâneo/diagnóstico , Febre Familiar do Mediterrâneo/epidemiologia , Febre Familiar do Mediterrâneo/genética , Colchicina/uso terapêutico , Amiloidose/complicações , Pirina/genética , Insuficiência Renal/complicações , MutaçãoRESUMO
Rheumatoid arthritis (RA) is a chronic inflammatory multisystemic disease of unknown etiology and autoimmune nature that predominantly affects peripheral joints in a symmetrical fashion. Although much progress has been made in understanding the pathophysiology of RA, its etiology remains unknown. Tumor necrosis factor (TNF)-α and interleukin (IL)-6 play the important roles in the pathogenesis and maintenance of inflammation in RA. The presence of anti-citrullinated peptide antibodies aids in the diagnosis in patients with undifferentiated polyarthritis and is associated with a more aggressive RA. The natural history of RA causes joint deformity and disability, as well as reduced life expectancy, both due to increased cardiovascular risk, pulmonary involvement, infections, iatrogenesis or tumors. Early diagnosis and the use of targeted drugs to induce early remission have improved the RA prognosis.
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Artrite Reumatoide , Humanos , Artrite Reumatoide/complicações , Artrite Reumatoide/diagnóstico , Prognóstico , Interleucina-6 , Fator de Necrose Tumoral alfaRESUMO
BACKGROUND: Long-term gout treatment is based on reducing serum urate levels using urate-lowering therapy (ULT). Most guidelines recommend using a lifelong continuation treat-to-target (T2T) strategy, in which ULT is dosed or combined until a serum urate target has been reached and maintained. However, a frequently used alternative strategy in clinical practice is a treat-to-avoid-symptoms (T2S) ULT discontinuation strategy, with the possibility of restarting the medication. This latter strategy aims at an acceptable symptom state, regardless of serum urate levels. High-quality evidence to support either strategy for patients in prolonged remission while using ULT is lacking. METHODS: We developed an investigator-driven pragmatic, open-label, multicentre, randomized, superiority treatment strategy trial (GO TEST Finale). At least 278 gout patients using ULT who are in remission (>12 months, preliminary gout remission criteria) will be randomized 1:1 to a continued T2T strategy (treatment target serum urate < 0.36 mmol/l) or switched to a T2S discontinuation strategy in which ULT is tapered to stop and restarted in case of (persistent or recurrent) flaring. The primary outcome is the between-group difference in the proportion of patients not in remission during the last 6 months of 24 months follow-up and will be analyzed using a two proportion z test. Secondary outcomes are group differences in gout flare incidence, reintroduction or adaptation of ULT, use of anti-inflammatory drugs, serum urate changes, occurrence of adverse events (with a special interest in cardiovascular and renal events), and cost-effectiveness. DISCUSSION: This study will be the first clinical trial comparing two ULT treatment strategies in patients with gout in remission. It will contribute to more specific and unambiguous guideline recommendations and improved cost-effectiveness of long-term gout treatment. It also paves the way (exploratory) to individualized long-term ULT treatment. In this article, we elaborate on some of our trial design choices and their clinical and methodological consequences. TRIAL REGISTRATION: International Clinical Trial Registry Platform (ICTRP) NL9245. Registered on 2 February 2021 (METC Oost-Nederland NL74350.091.20); EudraCT EUCTR2020-005730-15-NL. Registered on 11 January 2021.
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Gota , Humanos , Gota/diagnóstico , Gota/tratamento farmacológico , Supressores da Gota/efeitos adversos , Rim , Estudos Multicêntricos como Assunto , Ensaios Clínicos Controlados Aleatórios como Assunto , Exacerbação dos Sintomas , Ácido Úrico , Ensaios Clínicos Pragmáticos como AssuntoRESUMO
Background: Osteoporosis is a common comorbidity of rheumatoid arthritis (RA). Although RA disease activity has been demonstrated to be associated with bone loss in previous studies, most of them were cross-sectional studies and not in the context of treat-to-target (T2T) strategies. Objectives: This study aimed to evaluate the association of disease activity with bone mineral density (BMD) changes in the context of T2T strategies in a prospective RA cohort. Methods: RA patients were enrolled from a prospective CENTRA cohort of Peking University First Hospital. The follow-ups have been scheduled every 3 to 6 months. BMD was repeated at baseline, 1 year, and then every other year. Demographics, baseline clinical features, laboratory data, and medications at each visit were recorded. Time-adjusted mean disease activity scores were adopted to reflect the overall disease activity during follow-up. The influence of univariable associations between predictors and BMD was investigated using linear regression. Results: A total of 268 patients were included in our analysis. Their mean age was 50 (12.9) years, and 224 (83.6%) were women. The median (IQR) disease duration was 48.7 (107.6) months. Osteoporosis in the lumbar spine was observed in 23.1% of patients and 9.3% in the femoral neck at enrollment. Older age, higher SDAI score, and lower BMI were associated with osteoporosis at baseline. The proportion of patients who achieved DAS28-ESR, CDAI, and SDAI remission or LDA at the end of the first year was 71.5%, 68.8%, and 67.4%, respectively. Reevaluations of BMD at 1 year were applied to 144 patients. Mean decreases of BMDs were 1.75% at the lumbar spine and 1.40% at the femoral neck at 1 year from baseline, respectively. Patients who achieved remission had less yearly bone loss in the lumbar spine (p = 0.036). Female gender was identified as a risk factor in the multiple linear regression analyses, and lower disease activity and bisphosphonates were protective factors of continuous bone loss. Conclusion: Disease activity is associated with bone loss in RA patients in the context of T2T strategies, and those who achieved remission had less yearly bone loss.
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Artrite Reumatoide , Doenças Ósseas Metabólicas , Osteoporose , Artrite Reumatoide/complicações , Artrite Reumatoide/tratamento farmacológico , Densidade Óssea , Estudos de Coortes , Feminino , Humanos , Vértebras Lombares/diagnóstico por imagem , Masculino , Pessoa de Meia-Idade , Osteoporose/tratamento farmacológico , Osteoporose/epidemiologia , Osteoporose/etiologia , Estudos ProspectivosRESUMO
INTRODUCTION: Treat-to-target (T2T) strategy has been the core of rheumatoid arthritis (RA) management for over a decade, although it implementation has varied distinctly in real practices. We report here our investigation of the differences in disease activity and target achievement of two patient cohorts with different T2T implementations. METHODS: Data of the CENTRA (Collaboratively intENsive Treat-to-target in RA) and TARRA (Treat-to-TARget in RA) cohorts were used. The CENTRA cohort is a RA cohort prospectively followed up by a fixed team with tight control, while the TARRA is a longitudinal observational cohort followed up by a rheumatologist with casual control. Patients from the two cohorts were matched 1:3 by propensity score matching. The primary outcome was the Simplified Disease Activity Index (SDAI) at the 1-year follow-up. RESULTS: Included in this analysis were 102 patients from the CENTRA cohort and 271 patients from the TARRA cohort. Both groups were comparable in terms of age, gender, disease course, and seropositivity. At the end of the 1-year follow-up, the SDAI of patients in the CENTRA cohort was significantly lower than that of patients in the TARRA cohort (2.1 vs. 3.4; p < 0.001). A similar result was obtained based on the generalized estimating equation (GEE) model (p = 0.009). In addition, more patients in the CENTRA cohort achieved SDAI-defined remission compared to the TARRA cohort [72 (70.6%) vs. 134 (49.4%); p < 0.001]. CONCLUSION: Patients with RA may benefit more from a tight control T2T strategy with closer follow-up and appropriate education compared with those with a casual T2T strategy.
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BACKGROUND: In Crohn's disease, postoperative endoscopic activity of small bowel lesions outside the scope of ileocolonoscopy has been insufficiently studied. AIMS: We aimed to assess this postoperative activity using capsule endoscopy (CE) and analyze the association between treatment optimization based on CE findings and the long-term course. METHODS: In patients who underwent intestinal resection, we performed CE and assessed the endoscopic activity using the Lewis score within 3 months postoperatively (1st CE) and during follow-up. Postoperative treatments were adjusted according to clinical symptoms or CE findings (severity of 1st CE or worsening of follow-up CEs). Hospitalization, repeat surgery, or endoscopic dilation defined the primary outcome. RESULTS: Among the CE group (N = 48), 85.7% (1st CE) and 79.2% (2nd CE) exhibited endoscopic activities indicating residual or recurrent lesions. Postoperative treatments were adjusted according to clinical symptoms in the non-CE group (N = 57) and clinical symptoms or CE findings in the CE group. Compared to the non-CE group, the CE group had significantly fewer primary outcomes. Patients with treatment adjustments based on CE findings had even lower primary outcome rate. Multivariate analysis identified the CE group as an independent protective factor (hazard ratio = 0.45, 95% confidence interval = 0.20-0.96). Treatment adjustments based on CE findings showed a stronger protective effect (0.30, 0.10-0.75). CONCLUSIONS: Postoperative repeated CE enabled us to assess residual and recurrent lesions accurately before clinical symptoms appeared. The regular assessment of endoscopic activity and subsequent treatment optimization have the potential for improving postoperative course.
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Endoscopia por Cápsula/métodos , Doença de Crohn , Procedimentos Cirúrgicos do Sistema Digestório , Trato Gastrointestinal , Efeitos Adversos de Longa Duração , Complicações Pós-Operatórias , Adulto , Doença de Crohn/epidemiologia , Doença de Crohn/patologia , Doença de Crohn/cirurgia , Procedimentos Cirúrgicos do Sistema Digestório/efeitos adversos , Procedimentos Cirúrgicos do Sistema Digestório/métodos , Feminino , Trato Gastrointestinal/diagnóstico por imagem , Trato Gastrointestinal/cirurgia , Humanos , Japão/epidemiologia , Efeitos Adversos de Longa Duração/diagnóstico , Efeitos Adversos de Longa Duração/terapia , Masculino , Avaliação de Processos e Resultados em Cuidados de Saúde , Gravidade do Paciente , Administração dos Cuidados ao Paciente/métodos , Complicações Pós-Operatórias/diagnóstico , Complicações Pós-Operatórias/epidemiologia , Complicações Pós-Operatórias/terapia , Prevenção Secundária/métodos , TempoRESUMO
BACKGROUND: Adult-onset Still's disease (AOSD) characterized by a high spiking fever, skin rash, arthritis, and leukocytosis. The aim of the present study was considering the long-term outcomes of patients with AOSD who were treated with tight control strategy with disease modifying anti-rheumatic drugs (DMARDs). METHODS: Fifty-six patients with AOSD treated with tight control strategy were included. Four levels of remission were defined. Remission on-treatment was defined as the clinical remission, patient global assessment (PGA) ≤ 1, and prednisolone dose ≤ 5 mg/day for at least 6 months. Remission off-treatment was defined as the clinical remission and PGA ≤ 1 for at least 6 months as well as discontinuation of prednisolone, DMARDs, and biologics. Sustained remission on-treatment was defined as the clinical remission, PGA ≤ 1, and prednisolone dose ≤ 5 mg/day for ≥ 5 years. Sustained remission off-treatment was defined as the clinical remission and PGA ≤ 1 for ≥ 5 years as well as discontinuation of prednisolone, DMARDs, and biologics. RESULTS: Throughout a median follow-up of 47 months, remission on-treatment and off-treatment were obtained in 94.6% and 44.6% of patients, respectively. Sustained remission on-treatment and off-treatment were obtained in 79.2 and 8.3% of patients, respectively. Glucocorticoids (GCs) and DMARDs were discontinued in 66.1% and 48.2% of the patients, respectively. Apart from the older age of the patients in the on-GCs group, no significant differences were observed between the groups. CONCLUSION: Our study showed that using DMARDs with tight control strategy at the presentation of AOSD may control disease activity successfully. Key Points ⢠Using DMARDs with tight control strategy at the presentation of adult-onset Still's disease may control disease activity successfully.
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Antirreumáticos , Doença de Still de Início Tardio , Adulto , Idoso , Antirreumáticos/uso terapêutico , Glucocorticoides/uso terapêutico , Humanos , Prednisolona/uso terapêutico , Indução de Remissão , Doença de Still de Início Tardio/tratamento farmacológicoRESUMO
INTRODUCTION: Treat-to-target (T2T) strategy has greatly improved the prognosis of rheumatoid arthritis (RA). However, the additional benefit of targeting ultrasound (US) remission in addition to clinical remission has been debated. METHODS: RA patients in clinical remission or low disease activity were enrolled. They were assorted into two groups according to the principle of T2T strategy adopted. In clinical group, treatment decision was made with the aim of maintaining DAS28(ESR) ≤ 3.2 only, while in clinical US group, the aim was to attain total power Doppler (PD) US score = 0 in addition to DAS28(ESR) ≤ 3.2. The time-averaged DAS28, flare, and changes of treatment strategy were compared. RESULTS: One hundred ninety-four patients completed 1-year follow-up, with 100 in clinical US and 94 in clinical group. Compared to clinical group, time-averaged DAS28 in clinical US group was significantly lower (1.89 ± 0.51 vs. 2.33 ± 0.71, P < 0.01) with less flare (20.0% vs. 36.2%, P < 0.05). Furthermore, at the end of 1 year, significantly more patients successfully achieved step-down therapy (66.0% vs. 44.7%, P < 0.01) and dramatically fewer patients with step-up therapy in the clinical US group (13.0% vs. 25.5%, P < 0.05) compared to clinical group. In clinical US group, baseline DAS28(ESR) > 2.29, presence of subclinical synovitis, and step-down strategy were independent risk factors for relapse after clinical remission or low disease activity was achieved. CONCLUSIONS: An US-driven T2T in addition to current clinical remission strategy is associated with better control of the disease activity, reduction of relapse, as well as long-term step-down therapy. Step-down strategy should be carefully applied to the patients with baseline DAS28(ESR) over 2.29 and presence of subclinical synovitis even after they have achieved clinical remission or low disease activity. Key Points ⢠Targeting ultrasound remission in addition to current T2T strategy is associated with a better control of RA. ⢠Step-down strategy should be cautiously considered in those with DAS28(ESR) > 2.29 and baseline subclinical synovitis after they have achieved clinical remission or low disease activity.
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Antirreumáticos , Artrite Reumatoide , Sinovite , Antirreumáticos/uso terapêutico , Artrite Reumatoide/diagnóstico por imagem , Artrite Reumatoide/tratamento farmacológico , Humanos , Indução de Remissão , Índice de Gravidade de Doença , Sinovite/diagnóstico por imagem , Sinovite/tratamento farmacológicoRESUMO
Inflammatory bowel disease has become a growing concern worldwide. The chronic and progressive nature of inflammatory bowel disease poses significant challenges to the treatment and management of affected patients, straining health care resources. Therapeutic options and optimal management strategies have evolved dramatically. The treat-to-target strategy has shifted focus toward identifiable and attainable treatment targets and with the ability to optimize tight control. Advancements in our understanding of the pathophysiology led to therapeutic mechanisms that have a more narrowed focus toward gut-specific targets, improving safety profiles.
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Produtos Biológicos/administração & dosagem , Tratamento Conservador/métodos , Imunossupressores/administração & dosagem , Doenças Inflamatórias Intestinais/terapia , Produtos Biológicos/farmacologia , Gerenciamento Clínico , Feminino , Seguimentos , Humanos , Doenças Inflamatórias Intestinais/diagnóstico , Masculino , Seleção de Pacientes , Recidiva , Retratamento , Medição de Risco , Fatores de Tempo , Resultado do TratamentoRESUMO
To reach a Spanish expert consensus on a treat-to-target strategy in osteoporosis, a Delphi Consensus Study has been developed. Most of the experts (59.8%) were rheumatologist with a mean clinical experience of 21.3 years (SD 8.5). Consensus was achieved for 70% of the items. Therapeutic objectives, patient follow-up scheme, treatment failure criteria, and appropriate treatment choice for use in T2T strategy in Spain have been defined. INTRODUCTION: The paper aims to achieve a Spanish expert consensus on a treat-to-target (T2T) strategy in osteoporosis. METHODS: A scientific committee led the project and was involved in expert panel identification and Delphi questionnaire development. Two Delphi rounds were completed. The first-round questionnaire included 24 items and assessed, using a seven-point Likert scale, the experts' wish (W) and prognosis (P) in 5 years for each topic (applicability, therapeutic objectives, patient follow-up, and possible treatment to be prescribed). Items for which there was no consensus in the first round were included in the second round. Consensus was defined as ≥75% agreement (somewhat/mostly/entirely agree) or disagreement (somewhat/mostly/entirely disagree) responses. RESULTS: Of the experts, 112 and 106 completed the first and second rounds, respectively. 59.8% were rheumatologists with a mean clinical experience of 21.3 years (SD 8.5). Consensus was achieved for 70% of the items, and was established regarding the utility of a T2T strategy to define therapeutic objectives, optimal follow-up, and therapeutic algorithm. Participants agreed on the utility of the bone mineral density (BMD) value (T-score >-2.5 SD for spine and >-2.5/-2.0 SD for femoral neck), lack of fractures, and fracture risk (FRAX) as therapeutic objectives. For measuring BMD changes, consensus was achieved on the suitability of hip and femoral neck locations. Experts agreed to consider treatment failure as when a significant BMD gain could not be achieved, or when a new fracture occurs within 2-3 years. There was consensus that all proposed therapies should achieve a therapeutic target through T2T strategy (treatments with the highest consensus scores were denosumab and teriparatide). CONCLUSION: The therapeutic objectives, patient follow-up scheme, treatment failure criteria, and appropriate treatment choice for use in T2T strategy in Spain have been established by a panel of experts. Some aspects nevertheless still require further analysis.
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Conservadores da Densidade Óssea/uso terapêutico , Conduta do Tratamento Medicamentoso/organização & administração , Osteoporose/tratamento farmacológico , Densidade Óssea/efeitos dos fármacos , Conservadores da Densidade Óssea/administração & dosagem , Conservadores da Densidade Óssea/farmacologia , Técnica Delphi , Esquema de Medicação , Humanos , Conduta do Tratamento Medicamentoso/normas , Osteoporose/fisiopatologia , Fraturas por Osteoporose/prevenção & controle , Espanha , Falha de TratamentoRESUMO
The advances in psoriasis management currently allow achieving a good control of the disease. In particular, with the latest developed molecules, available evidence suggests that it is possible to pose an ambitious therapeutic goal, such as a Dermatology Life Quality Index 0/1, a Physician Global Assessment 0/1, or a Psoriasis Area and Severity Index 90/100 response. However, patients often fail to achieve the complete clearance of their cutaneous lesions or the improvement of disease factors that impair their quality of life. To optimize the treatment of psoriasis, it is not enough to define precisely the therapeutic objective, but also to adapt the therapeutic strategy to make the necessary modifications in case of not achieving it at the time point (at the end of the induction phase, or every 3-6 months) to be agreed with the patient (the so-called treat-to-target approach). In the present report, based on the Delphi methodology, 11 dermatologists from the Spanish Psoriasis Group addressed key issues that could be involved in the achievement and maintenance of the therapeutic goals of patients with moderate to severe psoriasis. The document provides 27 consensus statements intended to support clinical decision-making by healthcare professionals for patients who might be candidates to receive biologic therapy.
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Psoríase/terapia , Terapia Biológica , Ensaios Clínicos como Assunto , Fármacos Dermatológicos/uso terapêutico , Humanos , Psoríase/tratamento farmacológico , Psoríase/patologia , Qualidade de Vida , Índice de Gravidade de Doença , Inquéritos e Questionários , Resultado do TratamentoRESUMO
Over the past two decades, the management of juvenile idiopathic arthritis (JIA) has been revolutionized by the increased tendency toward early aggressive interventions and the availability of the novel biologic medications. In 2017, three novel randomized controlled trials have evaluated the effectiveness and tolerability of golimumab and tocilizumab in polyarticular JIA, and shown that methotrexate may increase and prolong the effect of intra-articular corticosteroid injection in children with oligoarthritis. A more rational approach to the management of JIA is being fostered by the recent publication of therapeutic recommendations, consensus treatment plans, and advice for the optimal care. A few months ago, an international consensus effort has led to the development of the recommendations for the treat-to-target in JIA. The application of this strategy in routine care may improve disease outcome. Because the potential of attaining inactive disease in children with JIA has markedly increased, there is an urgent need for randomized controlled trials, analyses of clinical data sets, and expert advice to guide discontinuation of medications once complete disease quiescence has been achieved.
Assuntos
Antirreumáticos/uso terapêutico , Artrite Juvenil/tratamento farmacológico , Anticorpos Monoclonais/uso terapêutico , Anticorpos Monoclonais Humanizados/uso terapêutico , Criança , Glucocorticoides/uso terapêutico , Humanos , Injeções Intra-Articulares , Metotrexato/uso terapêutico , Resultado do TratamentoRESUMO
OBJECTIVES: To evaluate the cost-effectiveness of a Treat-to-Target strategy with certolizumab pegol in patients with rheumatoid arthritis in the context of a pay-for-performance agreement in which medication costs are refunded in case of discontinuation during the first 3 months of treatment. METHODS: The Treat-to-Target strategy consisted of a systematic switch to second-line tumor necrosis factor (TNF)α inhibitor in case of an unmet ACR50 response at 3 months compared to current routine clinical practice. A reference cohort treated first-line with certolizumab pegol according to current practice without systematic switching was considered as the comparator. A decision-tree model was constructed to estimate clinical outcome (health assessment questionnaire-disability index or HAQ-DI score), time spent in ACR50 response (ACR 50), and direct costs of treatment over a 2-year period. HAQ scores were derived from American College of Rheumatology 50 (ACR50) responses. All TNFα inhibitors were assumed to have equivalent efficacy and tolerability. Costs were estimated at 2013 French retail prices (date of the pay-for-performance agreement). RESULTS: The mean duration of an ACR50 response was 1.23 years in the Treat-to-Target strategy certolizumab pegol cohort vs 0.98 years in the reference cohort, resulting in a mean gain in HAQ at 24 months of 0.117. The Treat-to-Target strategy with a mix of TNFα inhibitors as second-line therapy was more expensive than the reference strategy in absolute terms, but this difference was entirely offset by the pay-for-performance agreement. The Treat-to-Target strategy was, thus, cost-neutral over a 2-year period after the payback of CZP cost for patients not achieving the target at 3 months. CONCLUSIONS: In the context of a pay-for-performance agreement, the management of patients with rheumatoid arthritis using a Treat-to-Target strategy with certolizumab pegol in first line is dominant compared to standard use of this drug in the French setting in 2013.
Assuntos
Antirreumáticos/economia , Antirreumáticos/uso terapêutico , Artrite Reumatoide/tratamento farmacológico , Certolizumab Pegol/economia , Certolizumab Pegol/uso terapêutico , Reembolso de Incentivo/economia , Custos e Análise de Custo , Árvores de Decisões , França , Humanos , Revisão da Utilização de Seguros , Modelos Econométricos , Índice de Gravidade de Doença , Fator de Necrose Tumoral alfa/antagonistas & inibidoresRESUMO
The aim of this study was to assess safety and efficacy of ultrasonography (US)-guided intra-articular injections using tumor necrosis factor (TNF) blockers compared to corticosteroids in rheumatoid arthritis (RA) or psoriatic arthritis (PsA) patients, experiencing refractory monoarthritis despite the current systemic therapy. Eighty-two patients were randomized to receive three intra-articular injections monthly of either corticosteroid or TNF blockers. Primary endpoints were the safety and an improvement greater than 20% for visual analogic scales of involved joint pain in patients injected with anti-TNFα. Further clinical, US, and magnetic resonance imaging (MRI) evaluations were considered secondary endpoints. Intra-articular TNF blockers are a safe strategy, determining a significant reduction of patient and physician reported clinical outcomes and US/MRI scores, in RA and PsA patients, when compared to intra-articular injections of corticosteroids. US guidance excluded the possibility to inject the drug in the wrong site, maximizing local effects, reducing systemic effects, and increasing the safety of the procedure. Patients with inflammatory monoarthritis could be successfully treated with US-guided intra-articular TNF blockers that are a safe and well tolerated procedure, to achieve a longstanding clinical and radiological good clinical response and/or disease remission.