RESUMEN
Introduction: Pruritus is the primary symptom of atopic dermatitis (AD). The objective of this study was to develop a patient-reported outcome (PRO) instrument for daily administration in clinical trials to measure AD-related itch in adolescents and adults that would meet the standards described in the US Food and Drug Administration's (FDA's) PRO Guidance.Materials and methods: Six focus groups were conducted with 49 patients with AD (32 adults; 17 adolescents). Three iterative rounds of cognitive debriefing interviews were conducted in 26 patients with AD (17 adults; 9 adolescents) to pretest and refine the instrument.Results: AD-related itching was considered the most bothersome AD symptom by nearly two-thirds of the focus group participants. The items in the initial version of the instrument, named the Atopic Dermatitis Itch Scale (ADIS), were developed to reflect concepts most relevant to the assessment of itching as described during the focus groups. Based on results of the cognitive debriefing interviews, an 8-item final version of the ADIS was created.Conclusion: The ADIS is a content valid PRO instrument addressing the concepts critical to the measurement of AD-related itching. To our knowledge, it is the first instrument developed to assess AD-related itch in patients as young as 12 years following the recommendations of the PRO Guidance.
Asunto(s)
Dermatitis Atópica/patología , Prurito/etiología , Adolescente , Adulto , Anciano , Niño , Dermatitis Atópica/complicaciones , Dermatitis Atópica/psicología , Femenino , Grupos Focales , Humanos , Entrevistas como Asunto , Masculino , Persona de Mediana Edad , Medición de Resultados Informados por el Paciente , Prurito/patología , Índice de Severidad de la Enfermedad , Estados Unidos , Adulto JovenRESUMEN
Mantle cell lymphoma (MCL) is a rare, aggressive, incurable B-cell malignancy. Ibrutinib has been shown to be highly active for patients with relapsed/refractory (R/R) MCL. The RAY trial (MCL3001) was a phase 3, randomized, open-label, multicenter study that compared ibrutinib with temsirolimus in patients with R/R MCL. Active disease is frequently associated with impaired functional status and reduced well-being. Therefore, the current study employed two patient-reported outcome instruments, the Functional Assessment of Cancer Therapy-Lymphoma (FACT-Lym) and the EQ-5D-5L, to assess symptoms, well-being, health status, and health-related quality of life of patients on treatment within the RAY trial. We found that patients on ibrutinib had substantial improvement in FACT-Lym subscale and total scores, and had improvement in EQ-5D-5L utility and VAS scores compared with temsirolimus patients, indicating a superior well-being. These improvements in well-being correlated with clinical response, indicating that better health-related quality of life was associated with decreased disease burden.
Asunto(s)
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Linfoma de Células del Manto/tratamiento farmacológico , Linfoma de Células del Manto/epidemiología , Calidad de Vida , Adenina/análogos & derivados , Adulto , Anciano , Anciano de 80 o más Años , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Resistencia a Antineoplásicos , Femenino , Humanos , Linfoma de Células del Manto/mortalidad , Linfoma de Células del Manto/patología , Masculino , Persona de Mediana Edad , Estadificación de Neoplasias , Piperidinas , Pirazoles/administración & dosificación , Pirimidinas/administración & dosificación , Retratamiento , Sirolimus/administración & dosificación , Sirolimus/análogos & derivados , Resultado del TratamientoRESUMEN
BACKGROUND: Schizophrenia is one of the top 25 leading causes of disability worldwide in 2013. Despite its low prevalence, its health, social, and economic burden has been tremendous, not only for patients but also for families, caregivers, and the wider society. The magnitude of disease burden investigated in an economic burden study is an important source to policymakers in decision making. This study aims to systematically identify studies focusing on the economic burden of schizophrenia, describe the methods and data sources used, and summarize the findings of economic burden of schizophrenia. METHODS: A systematic review was performed for economic burden studies in schizophrenia using four electronic databases (Medline, EMBASE, PsycINFO, and EconLit) from inception to August 31, 2014. RESULTS: A total of 56 articles were included in this review. More than 80% of the studies were conducted in high-income countries. Most studies had undertaken a retrospective- and prevalence-based study design. The bottom-up approach was commonly employed to determine cost, while human capital method was used for indirect cost estimation. Database and literature were the most commonly used data sources in cost estimation in high-income countries, while chart review and interview were the main data sources in low and middle-income countries. Annual costs for the schizophrenia population in the country ranged from US$94 million to US$102 billion. Indirect costs contributed to 50%-85% of the total costs associated with schizophrenia. The economic burden of schizophrenia was estimated to range from 0.02% to 1.65% of the gross domestic product. CONCLUSION: The enormous economic burden in schizophrenia is suggestive of the inadequate provision of health care services to these patients. An informed decision is achievable with the increasing recognition among public and policymakers that schizophrenia is burdensome. This results in better resource allocation and the development of policy-oriented research for this highly disabling yet under-recognized mental health disease.
RESUMEN
BACKGROUND: Schizophrenia results in substantial health care utilization costs. Much of these costs can be attributed to health care use resulting from nonadherence to treatment, relapse, and hospitalization. AIMS OF THE STUDY: The objective of this research is to further estimate the health care resource utilization costs of patients with schizophrenia in the People's Republic of China, Korea, and Malaysia with a specific focus on the reduction in hospitalization costs associated with the use of long-acting, injectable paliperidone palmitate (PP) relative to alternative treatment medications. METHODS: The study focuses exclusively on the estimated reduction in hospitalization days following treatment with PP and the potential associated cost savings. Cost analysis was done using a payer's perspective and only includes direct health care costs associated with hospitalization. Localized cost data were taken from published sources, and health care utilization was estimated based on a clinical study conducted in countries in the Asia-Pacific region. People's Republic of China, Korea, and Malaysia had the highest number of patients enrolled in the clinical study, and thus were chosen for this research. Analysis looked at 12-month and 18-month periods following initial treatment with PP relative to a retrospective 12-month period utilizing alternative treatment medications. RESULTS: Results suggest that reductions in hospital utilization cost over 12 months may occur through the use of PP relative to alternatives-ranging from $1,991 for the People's Republic of China to $6,698 for Korea and $6,716 for Malaysia. CONCLUSION: Given the substantial costs associated with the treatment of schizophrenia both worldwide and in Asia, it is important to fully understand the costs and outcomes associated with various treatment options. In this research, we have specifically analyzed the direct health care cost savings associated with hospital utilization for patients taking PP relative to alternative treatment methods. The results suggest that reductions in hospital utilization cost were associated with PP treatment, likely largely due to increased adherence to treatment.
RESUMEN
OBJECTIVE: To evaluate the efficacy, safety, and impact on hospitalizations of long-acting injectable paliperidone palmitate (PP) treatment, in patients with recent-onset schizophrenia who had not responded satisfactorily to oral antipsychotics. METHODS: In this 18-month, open-label, Phase-IIIb study from Asia-Pacific region, patients (18-50 years) with recent-onset (≤5 years) schizophrenia unsatisfactorily treated with previous oral antipsychotics were initiated on PP 150 mg eq on day 1, 100 mg eq on day 8, followed by flexible once monthly maintenance doses of 50-150 mg eq. The number and duration of hospitalizations were compared using a mirror analysis method between two periods: retrospective (12 months before PP initiation) and prospective (12 and 18 months after PP treatment) periods. RESULTS: A total of 303 out of 521 (58%) patients (mean age, 28.7 years; 65.5% men, 92.5% Asian) completed the study. Positive and Negative Syndrome Scale (PANSS) total score improved significantly from baseline to month 18 (mean [standard deviation, SD] change: -11.3 [21.38], P<0.0001, primary endpoint). Subgroup analysis revealed greater improvements among patients with worse disease severity at baseline: PANSS ≥70 versus <70 (mean [SD] change: -23.1 [24.62] vs -4.7 [15.98], P<0.0001 each). Secondary efficacy endpoints such as Clinical Global Impression of Schizophrenia (CGI-SCH), Medication Satisfaction Questionnaire (MSQ) scores showed significant improvements (P<0.0001) from baseline; 33.3% patients achieved symptom remission. In mirror analyses set (N=474), PP significantly (P<0.0001) reduced mean number of hospitalization days/person/year (12-month: 74.3 vs 19.7; 18-month: 74.3 vs 18.9) as well as percentage of patients requiring hospitalization in past 12 months (12-month: 39.7% vs 24.6%; 18-month: 39.7% vs 25%), and PP treatment increased the proportion of patients not requiring hospitalization (12-month: 60.3% vs 75.4%; 18-month: 60.3% vs 75%) from retrospective to prospective period. Adverse events (≥15%) were extrapyramidal symptoms-related (31.3%), injection-site pain (18.6%), and insomnia (15.2%). CONCLUSION: PP was efficacious and generally tolerable with significant reductions observed in both number of hospitalizations and days spent in hospital.
RESUMEN
This trial was conducted to evaluate the safety and efficacy of the H4 R-antagonist JNJ-39758979 in adult Japanese patients with moderate atopic dermatitis (AD). Eligible patients were randomly assigned to JNJ-39758979 300 mg, 100 mg or placebo once daily for 6 weeks in this phase 2a, double-blind, multicenter, placebo-controlled study. Primary efficacy was assessed via week-6 Eczema Area and Severity Index (EASI) scores. Secondary efficacy assessments included Investigator's Global Assessment (IGA) and patient-reported outcome (PRO) pruritus assessments (Pruritus Categorical Response Scale [PCRS], Pruritus Numeric Rating Scales [PNRS], Pruritus Interference Numeric Rating Scale [PINRS] and Subject's Global Impressions of Change in Pruritus [SGICP]). Eighty-eight of 105 planned patients were randomized before the study was stopped and unblinded for safety reasons. The study did not meet the primary end-point. However, numerical improvements (i.e. decreases) in median EASI were observed with JNJ-39758979 100 mg (-3.7) and 300 mg (-3.0) versus placebo (-1.3) at week 6. Nominally significant improvements across PRO PCRS, PNRS and SGICP assessments were consistently observed, particularly with JNJ-39758979 300 mg. Safety, including adverse events (AE), was comparable between JNJ-39758979 and placebo with the exception of two patients (both receiving JNJ-39758979 300 mg) with serious AE of neutropenia, leading to premature study discontinuation. No deaths were reported. Except for neutropenia, no clinically relevant changes in laboratory values were observed. Although not conclusive, findings suggest H4 R-antagonism may be beneficial for AD, particularly in controlling pruritus. JNJ-39758979 appears to be associated with drug-induced agranulocytosis, likely an off-target effect.
Asunto(s)
Dermatitis Atópica/tratamiento farmacológico , Agonistas de los Receptores Histamínicos/uso terapéutico , Neutropenia/inducido químicamente , Pirimidinas/uso terapéutico , Pirrolidinas/uso terapéutico , Receptores Acoplados a Proteínas G/antagonistas & inhibidores , Adulto , Método Doble Ciego , Terminación Anticipada de los Ensayos Clínicos , Femenino , Agonistas de los Receptores Histamínicos/efectos adversos , Humanos , Japón , Masculino , Prurito/tratamiento farmacológico , Pirimidinas/efectos adversos , Pirrolidinas/efectos adversos , Receptores Histamínicos , Receptores Histamínicos H4 , Índice de Severidad de la Enfermedad , Adulto JovenRESUMEN
BACKGROUND: The risk of case-fatality following hospitalisation for asthma has not been well characterised. We describe trends in 30 day case-fatality following hospitalisation for asthma in adults in Scotland from 1981 to 2009. METHODS: Using the Scottish Morbidity Record Scheme (SMR01) with all asthma hospitalisations for adults (≥18 years) with ICD9 493 and ICD10 J45-J46 in the principal diagnostic position at discharge (1981-2009). These data were linked to mortality data from the General Register Office for Scotland (GROS), with asthma case-fatality defined as death within 30 days of asthma admission (in or out of hospital). Logistic regression was used to explore the impact of age, sex, previous asthma admission (in the 12 months prior to hospitalisation), socioeconomic deprivation, year of admission and co-morbidity on 30-day case-fatality. RESULTS: There were a total of 116,457 asthma hospitalisations; a total of 1000 (0.9%) hospitalisations resulted in a post-admission death (within 30 days of admission). Odds ratios for unadjusted and adjusted case-fatality showed a decreased risk of case-fatality from the mid-1990s onwards when compared to case-fatality in 1981. Advancing age and co-morbid diagnoses of respiratory failure, cancer, renal failure, cor pulmonale, coronary heart disease and respiratory infection were associated with increased likelihood of death. CONCLUSIONS: 30 day case-fatality has declined over the last three decades, comparable to case-fatality reported in other parts of the U.K. This decline may be in part due to improved guidelines, protocols and disease management for asthma over the last 30 years. The likelihood of death 30 days following an asthma admission increased with age group and was associated with respiratory failure, renal failure and cancer.
Asunto(s)
Asma/mortalidad , Adolescente , Adulto , Distribución por Edad , Anciano , Causas de Muerte/tendencias , Comorbilidad , Femenino , Hospitalización/estadística & datos numéricos , Humanos , Masculino , Persona de Mediana Edad , Estudios Retrospectivos , Factores de Riesgo , Escocia/epidemiología , Distribución por Sexo , Adulto JovenRESUMEN
BACKGROUND: The objective of this study was to evaluate the measurement properties of the Psoriasis Symptom Inventory (PSI), an eight-item patient-reported outcome measure for assessing severity of plaque psoriasis symptoms. METHODS: In this prospective, randomized study using data from adults with moderate-to-severe plaque psoriasis, patients completed the PSI, Dermatology Life Quality Index (DLQI), SF-36v2 Acute, and Patient Global Assessment (PtGA). PSI construct validity was assessed using Spearman rank correlations between PSI and DLQI and SF-36; test-retest reliability and sensitivity to change were evaluated using PtGA as an anchor. Daily 24-h and weekly 7-day PSI versions were evaluated. RESULTS: Eight US sites enrolled 143 patients; 139 (97.2%) completed the study. All symptoms (itch, redness, scaling, burning, cracking, stinging, flaking, and pain) were reported across all response options (not at all severe, mild, moderate, severe, very severe). Test-retest reliability was acceptable (intraclass correlation coefficients range = 0.70-0.80). A priori hypotheses of convergent and discriminant validity were confirmed by correlations of PSI with DLQI items and SF-36 domains. The PSI demonstrated good construct validity and was sensitive to within-subject change (p < 0.0001). CONCLUSIONS: The PSI is brief, valid, reproducible, and responsive to change and has the potential to be a useful PRO measure in psoriasis clinical trials.
Asunto(s)
Psoriasis/diagnóstico , Adulto , Anciano , Anciano de 80 o más Años , Femenino , Humanos , Masculino , Persona de Mediana Edad , Evaluación de Resultado en la Atención de Salud , Estudios Prospectivos , Autoinforme , Índice de Severidad de la Enfermedad , Adulto JovenRESUMEN
OBJECTIVE: To develop and assess content validity of the Psoriasis Symptom Inventory (PSI), a patient-reported outcome (PRO) measure of psoriasis symptoms. METHODS: Following initial literature exploration and input from experts, concept elicitation was conducted in two rounds (focus groups and individual interviews) with 59 subjects with mild to severe psoriasis. Transcripts were coded to identify symptom concepts and develop a conceptual framework using ATLAS.ti software. Qualitative content analysis and clinical expert input supported item generation and development of a draft measure. Two rounds of face-to-face cognitive interviews with 40 subjects with moderate to severe psoriasis were conducted to test subject comprehension and content coverage. RESULTS: Concepts of itching, scaling, flaking, tearing/cracking, burning, stinging, pain, bleeding and color of appearance were the most common symptom-related expressions. Saturation of concept was demonstrated. Severity was identified as the most meaningful attribute of psoriasis symptoms. A final 8-item measure was developed to assess patient-perceived symptom severity for itch, pain, burning, stinging, cracking, scaling, flaking and redness. Twenty-four-hour recall and 7-day recall versions were prepared for future quantitative assessment of measurement properties. CONCLUSIONS: The PSI is a short, low burden, patient-reported measure of psoriasis symptom severity with documented evidence of content validity.
Asunto(s)
Evaluación de la Discapacidad , Psoriasis/diagnóstico , Índice de Severidad de la Enfermedad , Perfil de Impacto de Enfermedad , Adulto , Anciano , Femenino , Grupos Focales , Estado de Salud , Humanos , Masculino , Persona de Mediana Edad , Evaluación de Resultado en la Atención de Salud , Psoriasis/etiología , Psoriasis/fisiopatología , Calidad de Vida , Encuestas y Cuestionarios , Adulto JovenRESUMEN
OBJECTIVES: To empirically identify groupings of strength, physical performance, adiposity, and lean mass and test how such groupings of these interrelated measures may relate to disability risk. DESIGN: Prospective Health, Aging and Body Composition Study. SETTING: Two U.S. clinical centers. PARTICIPANTS: One thousand two hundred sixty-three women and 1,221 men. MEASUREMENTS: Weight, strength (knee extension, grip); walking speed, chair stands, dual X-ray absorptiometry (fat and lean mass for total body, arm, and leg; percent fat), and thigh computed tomography scans (muscle area, muscle density). Analyses were stratified according to sex. Factor analysis reduced these variables into a smaller number of components, and proportional hazards models assessed risk of major disability for the components identified. RESULTS: In both sexes, factor analysis reduced the 14 individual variables into three components that explained 76% to 77% of the data variance: Factor 1, an adiposity component, with strong loading by fat mass, weight, and muscle density; Factor 2, a strength and lean body size component with strong loading by lean mass, weight, and strength; Factor 3, a physical performance component with positive loading by walking speed and chair stand performance. Factor 1 (adiposity) and Factor 3 (performance) but not Factor 2 (strength and lean body size) were associated with disability over 6.1 ± 2.6 years. CONCLUSION: The adiposity and physical performance constructs but not the strength and lean body size construct were associated with disability risk, suggesting that adiposity and performance should be considered as risk factors for disability.
Asunto(s)
Actividades Cotidianas , Adiposidad/fisiología , Envejecimiento/fisiología , Evaluación de la Discapacidad , Fuerza Muscular/fisiología , Absorciometría de Fotón , Anciano , Anciano de 80 o más Años , Composición Corporal/fisiología , Peso Corporal , Análisis Factorial , Femenino , Fuerza de la Mano/fisiología , Humanos , Masculino , Estudios Prospectivos , Factores de Riesgo , Muslo/diagnóstico por imagen , Tomografía Computarizada por Rayos X , Estados Unidos , Caminata/fisiologíaRESUMEN
OBJECTIVE: The objective of this study was to develop a patient-reported outcome (PRO) to assess reduced muscle strength in sarcopenia. DESIGN: Qualitative research study. SETTING: University of Arkansas for Medical Sciences. PARTICIPANTS: Subjects with sarcopenia. MEASUREMENTS: Adults aged 55 years and older with sarcopenia (n = 12) attended open-ended, concept elicitation interviews to characterize the functional effects of reduced muscle strength on their lives. The resulting qualitative data were analyzed using a qualitative analysis software program (Atlas.ti [Atlas.ti GmbH, Berlin, Germany]) and a common set of codes was developed to summarize the data. Subsequently, the initial PRO measure was drafted. Cognitive interviews were then conducted with additional sarcopenia subjects (n = 12) to refine the measure. RESULTS: Qualitative interviews identified key concepts (eg, impacts) in the areas of activities of daily living, emotions, social activities, energy, balance, coordination, sleep, and strength. Based on data from the cognitive debriefing interviews (eg, understandability, relevance, suggestions to reword items), the PRO measure development team came to consensus on which items or parts of the instructions to retain, revise, or delete. The final measure included 14 items. CONCLUSION: The final PRO measure, the Age-Related Muscle Loss Questionnaire, can be used in both clinical practice and clinical trial settings to assess functional impacts of reduced muscle strength in sarcopenia.
Asunto(s)
Actividades Cotidianas , Evaluación de Resultado en la Atención de Salud , Sarcopenia/fisiopatología , Anciano , Arkansas , Femenino , Evaluación Geriátrica , Humanos , Entrevistas como Asunto , Masculino , Persona de Mediana Edad , Fuerza Muscular/fisiología , Factores de Riesgo , Programas InformáticosRESUMEN
BACKGROUND: Direct correlation of assessments of a validated composite measure such as the Asthma Control Questionnaire (ACQ) and risk of exacerbation has not been previously demonstrated in a randomized controlled trial. OBJECTIVE: To evaluate the ability of the ACQ score over time to predict risk of a future asthma exacerbation. METHODS: This analysis included data from a 12-week placebo-controlled trial (N = 292) of AMG 317, an IL-4 receptor α antagonist, in patients with moderate to severe atopic asthma. At baseline, patients had an ACQ score ≥1.5. Exacerbations were defined as requirement for systemic corticosteroids. A Cox proportional hazards model was used, with ACQ score as the time-dependent covariate. The analysis was repeated for individual components of the ACQ. RESULTS: Each 1-point increase in ACQ was associated with a 50% increased risk of exacerbation (hazard ratio, 1.50; 95% CI, 1.03-2.20) for the following 2-week period. Evaluation of individual ACQ components also demonstrated a similar trend, though each to a lesser degree than the full composite ACQ. CONCLUSION: Although based on a retrospective analysis, with small number of exacerbations, these findings support the utility of the composite ACQ score measurement to predict risk of future exacerbation in clinical trials and clinical practice. The composite ACQ score measurement was found to be a better predictor of future risk than individual ACQ components.
Asunto(s)
Asma/diagnóstico , Encuestas y Cuestionarios , Adulto , Antiasmáticos/uso terapéutico , Asma/tratamiento farmacológico , Método Doble Ciego , Femenino , Humanos , Masculino , Estudios Multicéntricos como Asunto , Ensayos Clínicos Controlados Aleatorios como Asunto , Estudios RetrospectivosRESUMEN
BACKGROUND: In two previous phase 3 studies, up to 60 weeks of etanercept therapy significantly improved the symptoms of psoriasis and was well tolerated. OBJECTIVE: To evaluate the long-term safety of etanercept in an open-label extension study for up to 72 weeks in patients with moderate-to-severe plaque psoriasis. METHODS: A total of 912 patients received 50 mg subcutaneous etanercept once weekly (OW) for the first 12 weeks of this extension study. Thereafter, eligible patients could maintain the 50 mg QW dose (n = 321) or escalate to 50 mg twice weekly (BIW; n = 591) anytime thereafter based on one of three predetermined criteria. RESULTS: Etanercept was well tolerated during 1056 patient-years of exposure; no difference was observed between the 50 mg QW and 50 mg BIW dosages in rates of adverse events and infections. Improvement in skin disease was maintained throughout the study. Patients who stopped and then restarted etanercept also showed improvement in psoriasis. CONCLUSION: Psoriatic patients continued to benefit from open-label etanercept treatment, both continuous and interrupted therapy, which was generally well tolerated after a combined 2.5 years of experience.
Asunto(s)
Antiinflamatorios no Esteroideos/uso terapéutico , Inmunoglobulina G/uso terapéutico , Psoriasis/tratamiento farmacológico , Receptores del Factor de Necrosis Tumoral/uso terapéutico , Adulto , Anciano , Antiinflamatorios no Esteroideos/administración & dosificación , Antiinflamatorios no Esteroideos/efectos adversos , Relación Dosis-Respuesta a Droga , Esquema de Medicación , Etanercept , Femenino , Humanos , Inmunoglobulina G/administración & dosificación , Inmunoglobulina G/efectos adversos , Estudios Longitudinales , Masculino , Persona de Mediana Edad , Psoriasis/fisiopatología , Receptores del Factor de Necrosis Tumoral/administración & dosificación , Índice de Severidad de la Enfermedad , Factores de TiempoRESUMEN
OBJECTIVE: To evaluate the effects of etanercept treatment on patient-reported outcomes (PRO) in patients with psoriatic arthritis (PsA). METHODS: A 24-week double-blind comparison to placebo was followed by a 48-week open-label phase in which all eligible patients received etanercept. PRO were measured using the Stanford Health Assessment Questionnaire Disability Index (HAQ-DI), the Medical Outcomes Study Short-Form (SF-36), the EQ-5D visual analog scale (VAS), and the American College of Rheumatology (ACR) patient pain assessment. RESULTS: Beginning at Week 4 and continuing through Week 24 of double-blind treatment, patients treated with etanercept had significantly higher mean percentage improvement in HAQ-DI relative to baseline than patients given placebo (53.6% vs 6.4% at Week 24; p < 0.001). After 48 weeks of open-label treatment with etanercept, the mean percentage change from study baseline was 52.8% for the original etanercept group and 46.9% for the original placebo group, with 41.2% of patients overall achieving a HAQ-DI of 0. Mean changes relative to baseline for SF-36 physical component summary scores, EQ-5D VAS, and ACR pain assessment were also significant in the double-blind period for etanercept compared with placebo (p < 0.001 for all 3 measures). Patients taking placebo achieved similar improvements once they began treatment with etanercept in the open-label period. CONCLUSION: Patients with PsA treated with etanercept reported significant improvements in physical function that were almost 10 times the improvement seen with placebo and were maintained for up to 2 years. Almost half of patients treated with etanercept reported no disability by the end of the study.
Asunto(s)
Antirreumáticos/uso terapéutico , Artritis Psoriásica/tratamiento farmacológico , Inmunoglobulina G/uso terapéutico , Evaluación de Resultado en la Atención de Salud , Satisfacción del Paciente , Receptores del Factor de Necrosis Tumoral/uso terapéutico , Adolescente , Adulto , Anciano , Artritis Psoriásica/fisiopatología , Evaluación de la Discapacidad , Método Doble Ciego , Etanercept , Femenino , Estado de Salud , Humanos , Masculino , Persona de Mediana Edad , Dolor/tratamiento farmacológico , Dolor/fisiopatología , Dimensión del Dolor , Calidad de Vida , Índice de Severidad de la Enfermedad , Resultado del Tratamiento , Adulto JovenRESUMEN
BACKGROUND AND OBJECTIVES: The severity of anemia at which to initiate erythropoiesis-stimulating agent (ESA) treatment in nondialysis chronic kidney disease (CKD) patients is unclear. Risk of mortality, hospitalizations, and blood transfusion were compared among nondialysis CKD patients with "early" versus "delayed" ESA initiation. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: A retrospective cohort study was conducted on CKD (estimated GFR <60 ml/min/1.73m(2)) outpatients in the national Veterans Administration who were initiated on ESAs. Patients with ESRD, gastrointestinal bleeding, chemotherapy, or hematologic malignancy were excluded. Patients were characterized as having early [hemoglobin (Hb) 10.0 to 11.0 g/dl] or delayed (Hb 8.0 to 9.9 g/dl) ESA initiation. A propensity score comprising demographic, clinical, and laboratory variables was used to select a 1:1 matched cohort. Cox survival and negative binomial regression were used to compare the matched groups for all-cause mortality, hospitalizations, and blood transfusions. RESULTS: Of 1837 patients who met inclusion criteria, 1410 (77%) were successfully matched. The groups did not differ significantly in 31 characteristics reflecting sociodemographics, comorbidity, healthcare utilization, and renal function. There was no significant difference in mortality with early initiation. Those initiated early had a 17% lower risk of initial hospitalization and a 29% lower risk of transfusion compared with delayed initiation patients. Results did not differ between those with and without pre-ESA transfusion or hospitalization. CONCLUSIONS: In nondialysis CKD, ESA initiation at Hb 10.0 to 11.0 g/dl compared with 8.0 to 9.9 g/dl is associated with reduced risk of blood transfusion and initial hospitalization.
Asunto(s)
Anemia/tratamiento farmacológico , Eritropoyesis/efectos de los fármacos , Hematínicos/administración & dosificación , Enfermedades Renales/tratamiento farmacológico , Anciano , Atención Ambulatoria , Anemia/sangre , Anemia/etiología , Anemia/mortalidad , Biomarcadores/sangre , Transfusión Sanguínea , Distribución de Chi-Cuadrado , Enfermedad Crónica , Esquema de Medicación , Femenino , Hemoglobinas/metabolismo , Hospitalización , Humanos , Estimación de Kaplan-Meier , Enfermedades Renales/sangre , Enfermedades Renales/complicaciones , Enfermedades Renales/mortalidad , Modelos Logísticos , Masculino , Persona de Mediana Edad , Puntaje de Propensión , Modelos de Riesgos Proporcionales , Estudios Retrospectivos , Medición de Riesgo , Factores de Riesgo , Factores de Tiempo , Resultado del TratamientoRESUMEN
OBJECTIVE: Although insulin is the most effective diabetes medication for lowering blood glucose, how insulin is used in clinical practice and how well patients respond to insulin therapy over the course of several years has not been documented. Our objective was to describe glycemic control, side-effects and dose titration over 7 years among persons starting insulin in a health plan that has long used a treatment algorithm similar to the current American Diabetes Association/European Association for the Study of Diabetes (ADA-EASD) algorithm for the management of hyperglycemia. RESEARCH DESIGN AND METHODS: Patients (n = 2417) who initiated insulin therapy between 1 January 1999 and 31 December 2004 were followed for a mean of 49.5 months until 30 June 2007, death, or health plan termination. Mean hemoglobin A1C, number of units of insulin purchased and body weight were assessed on a quarterly basis. The proportion experiencing edema or hypoglycemia was assessed annually. RESULTS: Mean population A1C declined from 9.3 to 7.8% following insulin initiation and remained at that level for 7 years. However, A1C remained above 8% for 40% of patients, half of whom remained above 9.0%. The mean individual coefficient of variation in A1C was 0.12 (inter-quartile range 0.072-0.143). Mean daily insulin dosage started at 55 units and increased to approximately 100 units. Patients gained a mean of 6 lb (2.7 kg) during the first year then gained weight more gradually thereafter. Physicians diagnosed edema in 8-9% of patients annually. Hypoglycemia occurred in fewer than 2% of patients in any given year, with no cases requiring hospitalization. CONCLUSIONS: Insulin lowered mean A1C by about 1.5 percentage points to stable levels, but this required ongoing dosage increases. Nevertheless, many patients remained in poor control. Insulin is effective when used per ADA-EASD guidelines but health plans wishing to optimize diabetes care may need to intensify insulin therapy or consider the use of adjunct therapies in the years after initiation. This study was limited by its observational descriptive design, and its reliance on insulin purchases rather than actual consumption.
Asunto(s)
Diabetes Mellitus Tipo 2/tratamiento farmacológico , Insulina/uso terapéutico , Programas Controlados de Atención en Salud , Adulto , Anciano , Anciano de 80 o más Años , Glucemia/análisis , Femenino , Hemoglobina Glucada/análisis , Humanos , Masculino , Persona de Mediana EdadRESUMEN
OBJECTIVES: To examine the association between strength, function, lean mass, muscle density, and risk of hospitalization. DESIGN: Prospective cohort study. SETTING: Two U.S. clinical centers. PARTICIPANTS: Adults aged 70 to 80 (N=3,011) from the Health, Aging and Body Composition Study. MEASUREMENTS: Measurements were of grip strength, knee extension strength, lean mass, walking speed, and chair stand pace. Thigh computed tomography scans assessed muscle area and density (a proxy for muscle fat infiltration). Hospitalizations were confirmed by local review of medical records. Negative binomial regression models estimated incident rate ratios (IRRs) of hospitalization for race- and sex-specific quartiles of each muscle and function parameter separately. Multivariate models adjusted for age, body mass index, health status, and coexisting medical conditions. RESULTS: During an average 4.7 years of follow-up, 1,678 (55.7%) participants experienced one or more hospitalizations. Participants in the lowest quartile of muscle density were more likely to be subsequently hospitalized (multivariate IRR=1.47, 95% confidence interval (CI)=1.24-1.73) than those in the highest quartile. Similarly, participants with the weakest grip strength were at greater risk of hospitalization (multivariate IRR=1.52, 95% CI=1.30-1.78, Q1 vs. Q4). Comparable results were seen for knee strength, walking pace, and chair stands pace. Lean mass and muscle area were not associated with risk of hospitalization. CONCLUSION: Weak strength, poor function, and low muscle density, but not muscle size or lean mass, were associated with greater risk of hospitalization. Interventions to reduce the disease burden associated with sarcopenia should focus on increasing muscle strength and improving physical function rather than simply increasing lean mass.
Asunto(s)
Composición Corporal/fisiología , Hospitalización , Fuerza Muscular/fisiología , Músculo Esquelético/fisiología , Caminata/fisiología , Absorciometría de Fotón , Anciano , Anciano de 80 o más Años , Femenino , Evaluación Geriátrica , Fuerza de la Mano/fisiología , Humanos , Articulación de la Rodilla/fisiología , Masculino , Medicare , Músculo Esquelético/fisiopatología , Distribución de Poisson , Estudios Prospectivos , Análisis de Regresión , Riesgo , Muslo , Tomografía Computarizada por Rayos X , Torque , Estados UnidosRESUMEN
Objectives. Estimate and compare the proportion of cancer patients with cachexia using different definitions from available clinical data. Methods. Electronic medical records were examined to estimate the proportion of cancer patients with cachexia using 4 definitions: (1) ICD-9 diagnostic code of 799.4 (cachexia), (2) ICD-9 diagnosis of cachexia, anorexia, abnormal weight loss, or feeding difficulties, (3) prescription for megestrol acetate, oxandrolone, somatropin, or dronabinol, and (4) >/=5% weight loss. Patients with cancer of the stomach, pancreas, lung, colon/rectum, head/neck, esophagus, prostate, breast, or liver diagnosed between 1999 and 2004 were followed for cachexia. Results. Of 8541 cancer patients (60% men and 55% Caucasian), cachexia was observed in 2.4% of patients using the cachexia diagnostic code, 5.5% expanded diagnoses, 6.4% prescription medication definition, and 14.7% with >/=5% weight loss. Conclusions. The proportion of patients with cachexia varied considerably depending upon the definition employed, indicating that a standard operational definition is needed.
RESUMEN
OBJECTIVE: To examine self-reported symptoms and functioning in a community-based sample of persons with rheumatoid arthritis who did and did not initiate treatment with biologic agents. METHODS: Data were from annual telephone interviews (1998-2003) with an observational cohort identified through community rheumatologists. Self-reported function and symptoms of subjects who initiated biologic therapy (etanercept or infliximab) and reported consistent use at 2 annual interviews (continuous use; n = 64) were compared at 1 year prior to initiation of therapy (baseline), and years 1 and 2 of therapy to those with no biologic therapy (n = 183) and those who initiated biologic therapy but discontinued use (n = 42). RESULTS: At baseline, subjects taking biologic agents reported significantly worse function and symptoms on all measures except fatigue and pain severity. After 2 years, significant differences in the Health Assessment Questionnaire scores remained, but there were no other significant differences between the nonuser group and the continuous use group. The discontinued use group exhibited significantly greater pain severity and more painful joints than nonusers. Improvements in the number of painful (33.4% versus 16.2%; P = 0.004), and swollen (38.4% versus 18.7%; P = 0.003) joints, and morning stiffness (27.3% versus 10.4%; P = 0.001) were more frequent in the continuous use group than in the nonuser group. CONCLUSION: Results suggest that biologic treatment was initiated based on severe disease. Over approximately 17 months of treatment, differences in some but not all symptoms between the continuous use group and the nonuser group narrowed to statistical nonsignificance.
Asunto(s)
Anticuerpos Monoclonales/uso terapéutico , Antirreumáticos/uso terapéutico , Artritis Reumatoide/tratamiento farmacológico , Inmunoglobulina G/uso terapéutico , Receptores del Factor de Necrosis Tumoral/uso terapéutico , Anciano , Estudios de Cohortes , Investigación Participativa Basada en la Comunidad , Etanercept , Femenino , Estudios de Seguimiento , Humanos , Infliximab , Entrevistas como Asunto , Masculino , Persona de Mediana Edad , Evaluación de Resultado en la Atención de Salud , Índice de Severidad de la Enfermedad , Resultado del TratamientoRESUMEN
OBJECTIVE: The 24-week Etanercept Assessment of Safety and Effectiveness (EASE) study evaluated the effectiveness and tolerability of continuous versus interrupted etanercept treatment in patients with moderate to severe plaque psoriasis. The objective of this analysis was to assess patient-reported outcomes (PROs) and health-care resource utilization (HRU) data from the EASE study. METHODS: Patients received open-label etanercept 50 mg twice weekly for 12 weeks and then received either continued or interrupted (single round of discontinuation and re-treatment with etanercept) etanercept 50 mg once weekly for the second 12 weeks. PROs included the following: 1) the patient global assessments of psoriasis, joint pain, and itching scores; 2) the Dermatology Life Quality Index; 3) the Medical Outcomes Study Short Form 36 vitality domain; 4) the Beck Depression Inventory; 5) the European Quality-of-Life Group Feeling Thermometer; and 6) a patient satisfaction survey. HRU was evaluated using the Economic Implications of Psoriasis patient questionnaire. RESULTS: Continuous treatment with etanercept 50 mg twice weekly for 12 weeks followed by 50 mg once weekly for 12 weeks produced sustained and clinically important improvements in PROs and reductions in HRU. Reductions in some outcome measures after treatment discontinuation at week 12 were observed in the interrupted group; however, most changes did not revert to baseline levels, consistent with some residual clinical effect, and re-treatment produced improvements similar to week 12 levels. CONCLUSIONS: Continuous etanercept treatment provided greater sustained improvements in PROs than interrupted therapy; however, interrupting etanercept therapy, if needed, has predictable and manageable effects.