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PURPOSE: To characterize the significance of patient-level influences, including smoking history, on oncologic outcomes in human papillomavirus (HPV)-mediated oropharyngeal cancer (OPC). MATERIALS AND METHODS: A bi-institutional retrospective cohort study of previously untreated, HPV+ OPC patients who underwent curative treatment from 1/1/2008 to 7/1/2018 was performed. The primary outcome was disease-free survival (DFS) and the primary exposure was ≤10 versus >10-pack-year (PY)-smoking history. RESULTS: Among 953 OPC patients identified, 342 individuals with HPV+ OPC were included. The median patient age was 62â¯years, 33.0% had aâ¯>â¯10-PY-smoking history, 60.2% had AJCC8 stage I disease, and 35.0% underwent primary surgery. The median follow-up was 49â¯months (interquartile range [IQR] 32-75â¯months). Four-year DFS-estimates were similar among patients with ≤10-PY-smoking history (78.0%, 95% CI:71.7%-83.1%) compared to >10-PYs (74.8%; 95% CI:65.2%-82.0%; log-rank:pâ¯=â¯0.53). On univariate analysis, >10-PY-smoking history did not correlate with DFS (hazard ratio[HR]:1.15;95% CI:0.74-1.79) and remained nonsignificant when forced into the multivariable model. On adjusted analyses, stage, treatment paradigm, and age predicted DFS. Neither >10-PYs, nor any other definition of tobacco use (e.g., current smoker orâ¯>â¯20-PYs) was predictive of DFS, overall survival, or disease-specific survival. Conversely, age nonsignificantly and significantly predicted adjusted DFS (adjusted HR[aHR]:1.02,95% CI:0.997-1.05, pâ¯=â¯0.08), overall survival (aHR 1.05; 95% CI: 1.02-1.08; pâ¯=â¯0.002) and disease-specific survival (aHR 1.04;95% CI: 0.99-1.09;pâ¯=â¯0.09). CONCLUSION: Other than age, patient-level influences may not be primary drivers of HPV+ OPC outcomes. Although limited by its modest sample size, our study suggests the significance of smoking has been overstated in this disease. These findings and the emerging literature collectively do not support risk-stratification employing the >10-PY threshold. LEVEL OF EVIDENCE: Level 4.
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Alphapapillomavirus , Neoplasias Orofaríngeas , Infecções por Papillomavirus , Humanos , Pessoa de Meia-Idade , Neoplasias Orofaríngeas/cirurgia , Papillomaviridae , Infecções por Papillomavirus/complicações , Prognóstico , Estudos Retrospectivos , Fumar/efeitos adversos , Fumar/epidemiologiaRESUMO
[This corrects the article on p. 141 in vol. 38, PMID: 32327886.].
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To address knowledge gaps about Turner syndrome (TS) associated disease mechanisms, the Turner Syndrome Society of the United States created the Turner Syndrome Research Registry (TSRR), a patient-powered registry for girls and women with TS. More than 600 participants, parents or guardians completed a 33-item foundational survey that included questions about demographics, medical conditions, psychological conditions, sexuality, hormonal therapy, patient and provider knowledge about TS, and patient satisfaction. The TSRR platform is engineered to allow individuals living with rare conditions and investigators to work side-by-side. The purpose of this article is to introduce the concept, architecture, and currently available content of the TSRR, in anticipation of inviting proposals to utilize registry resources.
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Sistema de Registros , Pesquisa/organização & administração , Síndrome de Turner , Feminino , Humanos , Masculino , Pais , Participação do Paciente , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Declining glycemic control in type 1 diabetes (T1D) during adolescence persists despite treatment advances. Non-adherence, peer relations, diabetes burnout, risk taking, transition to autonomy, family conflict, and poor quality of life (QOL) are recognized barriers. Shared medical appointments (SMAs) in adolescent T1D may offer benefits, but data are limited. Our objective was to determine whether SMAs, with multi-component interventions utilizing multidisciplinary teams, improve glycemic control and psychosocial outcomes in poorly controlled adolescent T1D. METHODS: SMAs focused on self-management, communication skills, goal setting, glucose pattern recognition, and peer/diabetes team support. SMAs included: individual history and physical, labs, surveys, multidisciplinary educational ice breakers, group session, and individual wrap up. Outcomes were QOL, adherence, and retrospective and prospective glycemic control. Three to six subjects and families came to 3 SMAs and 1 individual appointment every 3 months over 9 months. SUBJECTS: A total of 37 English speaking subjects, ages 12-16 yrs, with T1D ≥ 1 year, and hemoglobin A1c (HbA1c) 7.5-11% enrolled. Thirty-two subjects attended 75% of visits, meeting inclusion criteria. RESULTS: HbA1c worsened in the 9 months before study (ΔHbA1c= 0.7 ± 1.2; p < 0.01), but remained stable during study (ΔHbA1c = 0.01 ± 1.2; p > 0.05). There were significant improvements in overall QOL (p = 0.005), school function (p = 0.006), psychosocial function (p = 0.008), barriers (p = 0.02), adherence (p = 0.01), and communication (p = 0.02). Improvements in school function and communication reached clinical significance. CONCLUSION: SMAs are feasible replacements to individual appointments in adolescent T1D, stabilizing glycemic control and improving QOL. Randomized controlled trials with optimizations are needed to further explore and refine this intervention.
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Diabetes Mellitus Tipo 1/tratamento farmacológico , Hiperglicemia/prevenção & controle , Hipoglicemia/prevenção & controle , Educação de Pacientes como Assunto , Grupo Associado , Qualidade de Vida , Autogestão/educação , Adolescente , California , Estudos de Coortes , Terapia Combinada , Diabetes Mellitus Tipo 1/sangue , Diabetes Mellitus Tipo 1/dietoterapia , Dieta para Diabéticos , Resistência a Medicamentos , Estudos de Viabilidade , Feminino , Hemoglobinas Glicadas/análise , Humanos , Hipoglicemiantes/uso terapêutico , Insulina/uso terapêutico , Masculino , Equipe de Assistência ao Paciente , Projetos Piloto , Cooperação e Adesão ao TratamentoRESUMO
BACKGROUND: Transcatheter pulmonary valve replacement (tPVR) is an accepted therapy for treatment of dysfunctional right ventricular outflow tract (RVOT) conduits. At present, the majority of Fallot patients who undergo transannular patch (TAP) repair are not candidates for tPVR due to the large irregular nature of their RVOT. Herein, we describe a novel approach to assessing the RVOT in this group, which may then be used to design, test, and carry out hybrid RVOT modification and transcatheter valve implantation in this population. METHODS: A retrospective analysis of TAP patients who underwent 3D modeling of the RVOT which was then used to develop individualized hybrid procedures designed to modify the RVOT, thereby rendering patients suitable for transcatheter valve implantation. RESULTS: Eight consecutive patients underwent 3D RVOT modeling followed by hybrid implantation of a transcatheter valve via a perventricular approach. A landing zone stent was placed in all and four required additional intravascular geometric remodeling of the RVOT prior to valve implant. Transcatheter valves were successfully implanted in all. There were no instances of valve malposition, embolization, or death. There was one minor procedural complication. No patient had more than trivial pulmonary regurgitation at follow-up. CONCLUSIONS: Using a hybrid approach to remodel the RVOT in TAP patients supported by preprocedural 3D-model planning allows for successful tPVR implantation in this population. A larger cohort and longer follow-up will be needed to determine the ultimate utility of this approach.
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Cateterismo Cardíaco/métodos , Procedimentos Cirúrgicos Cardíacos/efeitos adversos , Implante de Prótese de Valva Cardíaca/métodos , Insuficiência da Valva Pulmonar/terapia , Valva Pulmonar , Tetralogia de Fallot/cirurgia , Adolescente , Adulto , Cateterismo Cardíaco/instrumentação , Criança , Próteses Valvulares Cardíacas , Implante de Prótese de Valva Cardíaca/instrumentação , Humanos , Imageamento Tridimensional , Imageamento por Ressonância Magnética , Pessoa de Meia-Idade , Modelos Anatômicos , Modelos Cardiovasculares , Desenho de Prótese , Valva Pulmonar/diagnóstico por imagem , Insuficiência da Valva Pulmonar/diagnóstico por imagem , Insuficiência da Valva Pulmonar/etiologia , Insuficiência da Valva Pulmonar/fisiopatologia , Radiografia Intervencionista , Estudos Retrospectivos , Stents , Tetralogia de Fallot/diagnóstico , Tomografia Computadorizada por Raios X , Resultado do Tratamento , Adulto JovemRESUMO
Despite significant advances in pharmacology and technology, glycemic targets are difficult to achieve for patients with type 1 diabetes (T1D) and management remains burdensome for patients and their families. Quality improvement (QI) science offers a methodology to identify an aim, evaluate complex contributors to the goal, and test potential interventions to achieve outcomes of interest. Day-to-day management of diabetes is often an iterative process but interventions exist at all care levels: individual patient and family, clinic, and larger population and health system. This article reviews current literature and proposes novel QI interventions for enhancing health outcomes, with attention to essential determinants or drivers of improved glycemic control and patient experience for pediatric T1D in the context of the Chronic Care Model. In-depth consideration of key drivers of successful T1D care, including self-management and integration of technology, are explored, and examples of larger health systems with improved outcomes, including Learning Health Systems are highlighted.
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Diabetes Mellitus Tipo 1/terapia , Adolescente , Glicemia/análise , Criança , Diabetes Mellitus Tipo 1/diagnóstico , Humanos , Educação de Pacientes como Assunto , Melhoria de Qualidade , Autocuidado , Resultado do TratamentoRESUMO
IMPORTANCE: Previous studies assessing the effect of metformin on glycemic control in adolescents with type 1 diabetes have produced inconclusive results. OBJECTIVE: To assess the efficacy and safety of metformin as an adjunct to insulin in treating overweight adolescents with type 1 diabetes. DESIGN, SETTING, AND PARTICIPANTS: Multicenter (26 pediatric endocrinology clinics), double-blind, placebo-controlled randomized clinical trial involving 140 adolescents aged 12.1 to 19.6 years (mean [SD] 15.3 [1.7] years) with mean type 1 diabetes duration 7.0 (3.3) years, mean body mass index (BMI) 94th (4) percentile, mean total daily insulin 1.1 (0.2) U/kg, and mean HbA1c 8.8% (0.7%). INTERVENTIONS: Randomization to receive metformin (n = 71) (≤2000 mg/d) or placebo (n = 69). MAIN OUTCOMES AND MEASURES: Primary outcome was change in HbA1c from baseline to 26 weeks adjusted for baseline HbA1c. Secondary outcomes included change in blinded continuous glucose monitor indices, total daily insulin, BMI, waist circumference, body composition, blood pressure, and lipids. RESULTS: Between October 2013 and February 2014, 140 participants were enrolled. Baseline HbA1c was 8.8% in each group. At 13-week follow-up, reduction in HbA1c was greater with metformin (-0.2%) than placebo (0.1%; mean difference, -0.3% [95% CI, -0.6% to 0.0%]; P = .02). However, this differential effect was not sustained at 26-week follow up when mean change in HbA1c from baseline was 0.2% in each group (mean difference, 0% [95% CI, -0.3% to 0.3%]; P = .92). At 26-week follow-up, total daily insulin per kg of body weight was reduced by at least 25% from baseline among 23% (16) of participants in the metformin group vs 1% (1) of participants in the placebo group (mean difference, 21% [95% CI, 11% to 32%]; P = .003), and 24% (17) of participants in the metformin group and 7% (5) of participants in the placebo group had a reduction in BMI z score of 10% or greater from baseline to 26 weeks (mean difference, 17% [95% CI, 5% to 29%]; P = .01). Gastrointestinal adverse events were reported by more participants in the metformin group than in the placebo group (mean difference, 36% [95% CI, 19% to 51%]; P < .001). CONCLUSIONS AND RELEVANCE: Among overweight adolescents with type 1 diabetes, the addition of metformin to insulin did not improve glycemic control after 6 months. Of multiple secondary end points, findings favored metformin only for insulin dose and measures of adiposity; conversely, use of metformin resulted in an increased risk for gastrointestinal adverse events. These results do not support prescribing metformin to overweight adolescents with type 1 diabetes to improve glycemic control. TRIAL REGISTRATION: clinicaltrials.org Identifier: NCT01881828.
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Diabetes Mellitus Tipo 1/tratamento farmacológico , Hipoglicemiantes/administração & dosagem , Insulina/uso terapêutico , Metformina/administração & dosagem , Obesidade/complicações , Adolescente , Glicemia/análise , Índice de Massa Corporal , Peso Corporal , Criança , Diabetes Mellitus Tipo 1/complicações , Método Duplo-Cego , Quimioterapia Combinada , Feminino , Gastroenteropatias/induzido quimicamente , Hemoglobinas Glicadas/análise , Humanos , Hipoglicemiantes/efeitos adversos , Masculino , Metformina/efeitos adversos , Resultado do Tratamento , Adulto JovemRESUMO
PURPOSE: To describe the characteristics of nail gun-related open-globe injuries. METHODS: Retrospective series of all patients presenting with open globes secondary to nail gun injury from 2000 to 2010. Data were collected on demographics, setting of accident, presenting clinical examination findings, visual acuity, management, surgical procedures needed, and long-term outcomes. RESULTS: Forty-two patients (43 eyes; mean age, 31.6 years; 100% male; 79% Hispanic) suffered open-globe injury from nail gun accidents. Thirty-seven eyes (86%) sustained injury at work. One of 15 (6.7%) patients, on whom data were available, wore protective eyewear during the incident. Entrance wounds were classified into Zone I (n = 24 [56%]), Zone II (n = 12 [28%]), and Zone III (n = 7 [16%]). Six eyes (14%) had retained intraocular foreign bodies. Mean presenting logarithm of the minimum angle of resolution visual acuity was 1.64 ± 0.83, whereas mean final logarithm of the minimum angle of resolution visual acuity was 1.01 ± 0.96 (P = 0.004). Two eyes (4.7%) had no light perception vision at final examination. Seventeen (40%) patients developed a traumatic cataract, and 2 (4.7%) had dislocated lens fragments. Most common findings on presentation included vitreous hemorrhage (n = 30 [70%]) and hyphema (n = 28 [64%]). Two eyes (4.7%) had a retinal detachment at presentation, and 10 (23%) developed a retinal detachment during follow-up visits. Anatomical success was observed in 11 eyes (92%) with a retinal detachment. Three eyes (7.0%) became phthisical or prephthisical, and 1 was enucleated for severe pain. No eyes developed endophthalmitis or sympathetic ophthalmia. CONCLUSION: This is the largest compilation of nail gun-related open-globe injury reported to date. Posterior segment complications, noted in the majority of cases, likely contributed to the overall guarded visual outcomes. Preventative measures for eye protection should be strictly followed while using nail guns.
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Materiais de Construção/efeitos adversos , Corpos Estranhos no Olho/etiologia , Ferimentos Oculares Penetrantes/etiologia , Adulto , Corpos Estranhos no Olho/classificação , Corpos Estranhos no Olho/cirurgia , Ferimentos Oculares Penetrantes/classificação , Ferimentos Oculares Penetrantes/cirurgia , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Acuidade Visual/fisiologia , Adulto JovemRESUMO
OBJECTIVES: To assess utility and accuracy of a gestational age-based screening targeting premature infants to detect congenital hypothyroidism. STUDY DESIGN: A prospective cohort study was conducted in infants <35 weeks' gestational age with clinical outcomes at 2-3 years of age. Patients received newborn screenings at 24 hours and 10-14 days of life. Free T4 (FT4) and thyroid-stimulating hormone (TSH) levels were measured at one month of life and repeated based on algorithm by corrected gestational age. RESULTS: Among infants <35 weeks gestation (n = 938), the incidence of hypothyroidism requiring treatment was 1:58. TSH levels at one month of age was predictive of treatment (AUC 0.96, 95% CI 0.88-1). The optimal TSH threshold of 8 mIU/L (8 µU/ml) increased the specificity to 0.97 and sensitivity to 0.88. Following initiation of treatment for hypothyroidism during NICU hospitalization, 43.8% (n = 7) were diagnosed with permanent congenital hypothyroidism. CONCLUSIONS: Our study supports a gestational age-based screening algorithm for early detection of hypothyroidism in premature infants.
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INTRODUCTION: People enrolled in Medicaid managed care who struggle with diabetes control often have complex medical, behavioral, and social needs. Here the authors report the results of a program designed to partner with primary care teams to address those needs. METHODS: A nonprofit organization partnered with a Medicaid managed care plan and a Federally Qualified Health Center in California to enroll people with A1cs >9% in a 12-month program. The program team included a community health worker, certified diabetes care and education specialist/registered dietitian, behavioral health counselor, and registered nurse. They developed patient-led action plans, connected patients to community resources, and supported behavior changes to improve diabetes control. Baseline assessments of behavioral health conditions and social needs were collected. Monthly A1c values were tracked for participants and a comparison group. RESULTS: Of the 51 people enrolled, 83% had at least 1 behavioral health condition. More than 90% reported at least 1 unmet social need. The average monthly A1c among program participants was 0.699 lower than the comparison group post-enrollment (P = .0008), and the disparity in A1c between Hispanic and non-Hispanic White participants at enrollment declined. DISCUSSION: Participants had high levels of unmet medical, behavioral, and social needs. Addressing these needs resulted in a rapid and sustained improvement in A1c control compared to non-enrollees and a reduction in disparity of control among Hispanic participants. CONCLUSION: By partnering with a primary care team, a program external to Federally Qualified Health Center primary care can improve clinical outcomes for people with complex needs living with diabetes.
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Diabetes Mellitus , Medicaid , Estados Unidos , Humanos , Hemoglobinas Glicadas , Programas de Assistência Gerenciada , Diabetes Mellitus/terapia , EscolaridadeRESUMO
The risk of graft-rejection after allogeneic hematopoietic cell transplantation using conventional cyclophosphamide-based conditioning is increased in patients with bone marrow failure syndromes (BMFS) who are heavily transfused and often HLA-alloimmunized. Fifty-six patients with BMFS underwent fludarabine-based reduced-intensity conditioning and allogeneic peripheral blood progenitor cell (PBPC) transplantation at a single institution. The conditioning regimen consisted of intravenous cyclophosphamide, fludarabine, and equine antithymocyte globulin. Graft-versus-host disease (GVHD) prophylaxis included cyclosporine A alone or in combination with either mycophenolate mofetil or methotrexate. To reduce the risk of graft-rejection/failure, unmanipulated G-CSF mobilized PBPCs obtained from an HLA-identical or single HLA-antigen mismatched relative were transplanted rather than donor bone marrow. Despite a high prevalence of pretransplant HLA-alloimmunization (41%) and a heavy prior transfusion burden, graft-failure did not occur with all patients having sustained donor lympho-hematopoietic engraftment. The cumulative incidence of grade II-IV acute-GVHD and chronic-GVHD was 51.8% and 72%, respectively; with 87.1% surviving at a median follow-up of 4.5 years. A multivariate analysis showed pretransplant alloimmunization and rapid donor T-cell engraftment (≥95% donor by day 30) were both significantly (P < 0.05) associated with the development of chronic-GVHD (adjusted HR 2.13 and 2.99, respectively). These data show fludarabine-based PBPC transplantation overcomes the risk of graft-failure in patients with BMFS, although rapid donor T-cell engraftment associated with this approach appears to increase the risk of chronic-GVHD. (Clinicaltrials.gov identifier: NCT00003838).
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Rejeição de Enxerto/prevenção & controle , Doença Enxerto-Hospedeiro/prevenção & controle , Hemoglobinúria Paroxística/terapia , Transplante de Células-Tronco de Sangue Periférico , Linfócitos T , Condicionamento Pré-Transplante , Adulto , Idoso , Anemia Aplástica , Soro Antilinfocitário/administração & dosagem , Antineoplásicos/administração & dosagem , Doenças da Medula Óssea , Transtornos da Insuficiência da Medula Óssea , Criança , Doença Crônica , Ciclosporina/administração & dosagem , Feminino , Rejeição de Enxerto/etiologia , Rejeição de Enxerto/patologia , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/patologia , Fator Estimulador de Colônias de Granulócitos/administração & dosagem , Hemoglobinúria Paroxística/patologia , Humanos , Imunossupressores/administração & dosagem , Masculino , Pessoa de Meia-Idade , Ácido Micofenólico/administração & dosagem , Ácido Micofenólico/análogos & derivados , Fatores de Risco , Terapia de Salvação , Fatores de Tempo , Transplante Homólogo , Vidarabina/administração & dosagem , Vidarabina/análogos & derivados , Adulto JovemRESUMO
PURPOSE: To describe a case of bilateral optic nerve head drusen (ONHD) in a pediatric patient and the complementary use of advanced noninvasive imaging techniques to confirm this diagnosis. CASE REPORT: A 15-year-old female adolescent with type 1 diabetes and no ocular history was seen at a routine screening without complaints. Visual acuity was 20/20 bilaterally, and visual fields using frequency doubling technology (Zeiss Humphrey Systems, Dublin, CA) were within normal limits. Fundus photography (CX-1 Mydriatic/Non-Mydriatic Hybrid Digital Retinal Camera, Canon, Toyko, Japan) showed slight elevation of the left disc margin, with lack of physiologic cup, elevation of the inferior neuroretinal rim, and no swelling of the nerve fiber layer. Fundus autofluorescence image of the left eye showed hyperfluorescence in the inferior optic disc. Red/green/blue channel separation analysis using the blue channel (Eye-Q software, Canon, Irvine, CA) showed elevation of the nerve fiber layer without obscuration of small vessels surrounding the left optic disc. Embossed technique highlighted the drusen in the left disc. B-scan ultrasound (Eye Cubed, Ellex, Adelaide, Australia) showed a hyperechoic focus on the optic disc with posterior shadowing and corresponding spike on A-scan in both eyes, indicative of bilateral drusen. Optical coherence tomography (RTVue, Optovue, Fremont, CA) showed an elevation in the ONH corresponding to the drusen. CONCLUSIONS: The complementary use of noninvasive imaging modalities such as fundus autofluorescence, red/green/blue separation, B-scan, and optical coherence tomography is important in confirming the diagnosis of ONHD in pediatric patients. They allow us to rule out more serious conditions and avoid unnecessary, costly, and invasive investigative procedures, relieving young patients and their families of potential financial and emotional burdens.
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Angiofluoresceinografia , Drusas do Disco Óptico/diagnóstico , Disco Óptico/patologia , Tomografia de Coerência Óptica/métodos , Adolescente , Feminino , Fundo de Olho , Humanos , Acuidade Visual/fisiologia , Campos Visuais/fisiologiaRESUMO
Background and Objective: Type 1 diabetes, the most common cause of diabetes in pediatrics, is defined by the hyperglycemia that results from the permanent autoimmune damage to the pancreas. The Diabetes Control and Complications Trial (DCCT) demonstrated that strict glycemic control targeting lower HbA1c goals can both delay the onset and progression of its complications that include diabetic neuropathy, nephropathy, retinopathy, and increased cardiovascular events. Our primary objective is to review the literature available regarding the technology applied for the treatment of diabetes, not only aiding patients' quality of life but addressing its effects on hypoglycemia and reduced risk of the long-term complications. It will synthesize the evolution of glucose monitoring devices; the development of insulin: from animal to recombinant engineering, smart insulin in the future; the development of algorithm-driven insulin delivery devices, the closed loop system/artificial pancreas; and the future utilization of technology to support islet cell transplant with the goal of a long-term cure. Emphasis will be made on what is known about the impact on its outcomes in children and adolescents. Methods: A literature search was conducted using PubMed for publications from 1985 to present. Keywords used: type 1 diabetes, children, adolescents, pediatrics, continuous glucose monitoring (CGM), insulin pumps. Referenced articles include other reviews, current care guidelines as supported by cross sectional studies, cohort studies and randomized clinical trials. Key Content and Findings: Understanding the pathophysiology of type 1 diabetes has led to the design of technology that facilitates glucose monitoring and insulin administration in a personalized manner. The current technology has improved outcomes and quality of life by decreasing hypoglycemic events and decreasing risk of long-term metabolic complications. Barriers remain, for children and adults, often driven by patient's preference as well as their understanding of the limitations of what they are wearing. Conclusions: With the progressive evolution of this technology, it is now realistic to lower the burden of diabetes self-management while reducing hypoglycemia and risk of complications that otherwise impact daily life from academics, physical activity, career choices and even life expectancy.
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Individuals regularly struggle to save for retirement. Using a large-scale field experiment ( N = 97 , 149 ) in Mexico, we test the effectiveness of several behavioral interventions relative to existing policy and each other geared toward improving voluntary retirement savings contributions. We find that an intervention framing savings as a way to secure one's family future significantly improves contribution rates. We leverage recursive partitioning techniques and identify that the overall positive treatment effect masks subpopulations where the treatment is even more effective and other groups where the treatment has a significant negative effect, decreasing contribution rates. Accounting for this variation is significant for theoretical and policy development as well as firm profitability. Our work also provides a methodological framework for how to better design, scale, and deploy behavioral interventions to maximize their effectiveness.
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BACKGROUND: Type 1 diabetes and dyslipidemia are known risk factors for cardiovascular disease (CVD), but the relationship between lipid levels in youth with type 1 diabetes and future CVD remains unknown. OBJECTIVE: To characterize lipid levels and CVD risk factors over time in youth with type 1 diabetes. SUBJECTS: The study included adolescents with type 1 diabetes (12-25 yr) with a minimum of 3-yr follow-up. METHODS: A longitudinal prospective, observational study of 46 youth with type 1 diabetes was performed. Fasting lipid profiles, A1C, and body mass index (BMI) were measured every 6 months for at least 3 yr (median 4.2 yr). Low-density lipoprotein (LDL)-cholesterol, total cholesterol (TC), and triglycerides (TG) were divided into categorical variables. RESULTS: At baseline, median age was 14.3 yr, mean diabetes duration was 6.4 ± 3.8 yr, mean A1C was 8.1 ± 1.0%, and median BMI z-score was 0.92. Fifty percent of subjects had LDL levels ≤ 100 mg/dL (≤ 2.6 mmol/L) at study onset. After adjusting for confounding factors, increasing BMI z-score [ß = 0.2, 95% confidence interval (CI ) = 0.03-0.38, p = 0.03] and increasing A1C (ß = 0.18, 95% CI = 0.08-0.29, p = 0.001) were associated with increasing LDL category over time. Non-Hispanic ethnicity (ß = 0.45, 95% CI = 0.12-0.79, p = 0.008) and family history of stroke (ß = 0.38, 95% CI = 0.04-0.72, p = 0.03) were also associated with increasing LDL category. Age, diabetes duration, and tobacco exposure were not related to change in LDL. Increasing A1C was associated with increases in TG (ß = 18.1, 95% CI = 2.3-33.9, p = 0.03), TC (ß = 20.3, 95% CI = 9.0-31.5, p < 0.0001), and LDL (ß = 13.4, 95% CI = 3.17- 23.6, p = 0.01). CONCLUSION: Glycemic control and BMI are modifiable risk factors for dyslipidemia in youth with type 1 diabetes.
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Diabetes Mellitus Tipo 1/metabolismo , Metabolismo dos Lipídeos , Adolescente , Glicemia/metabolismo , Índice de Massa Corporal , Doenças Cardiovasculares/etiologia , Criança , Colesterol/sangue , LDL-Colesterol/sangue , Dislipidemias/prevenção & controle , Feminino , Hemoglobinas Glicadas/metabolismo , Hispânico ou Latino , Humanos , Estudos Longitudinais , Masculino , Estudos Prospectivos , Fatores de Risco , Triglicerídeos/sangue , Adulto JovemRESUMO
OBJECTIVES: Brain MRIs are considered essential in the evaluation of children diagnosed with growth hormone deficiency (GHD), but there is uncertainty about the appropriate cut-off for diagnosis of GHD and little data about the yield of significant abnormal findings in patients with peak growth hormone (GH) of 7-10 ng/mL. We aimed to assess the frequency of pathogenic MRIs and associated risk factors in relation to peak GH concentrations. METHODS: In this retrospective multicenter study, charts of patients diagnosed with GHD who subsequently had a brain MRI were reviewed. MRIs findings were categorized as normal, incidental, of uncertain significance, or pathogenic (pituitary hypoplasia, small stalk and/or ectopic posterior pituitary and tumors). Charges for brain MRIs and sedation were collected. RESULTS: In 499 patients, 68.1% had normal MRIs, 18.2% had incidental findings, 6.6% had uncertain findings, and 7.0% had pathogenic MRIs. Those with peak GH<3 ng/mL had the highest frequency of pathogenic MRIs (23%). Only three of 194 patients (1.5%) with peak GH 7-10 ng/mL had pathogenic MRIs, none of which altered management. Two patients (0.4%) with central hypothyroidism and peak GH<4 ng/mL had craniopharyngioma. CONCLUSIONS: Pathogenic MRIs were uncommon in patients diagnosed with GHD except in the group with peak GH<3 ng/mL. There was a high frequency of incidental findings which often resulted in referrals to neurosurgery and repeat MRIs. Given the high cost of brain MRIs, their routine use in patients diagnosed with isolated GHD, especially patients with peak GH of 7-10 ng/mL, should be reconsidered.
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Encéfalo/diagnóstico por imagem , Hormônio do Crescimento Humano/deficiência , Imageamento por Ressonância Magnética/métodos , Adolescente , Criança , Pré-Escolar , Feminino , Hormônio do Crescimento Humano/sangue , Humanos , Masculino , Estudos RetrospectivosRESUMO
BACKGROUND AND OBJECTIVE: To determine the prevalence of retinal disease among a population in Mwanza, Tanzania, and to identify relevant risk factors for retinal disorders in this cohort. PATIENTS AND METHODS: A cross-sectional population-based study was conducted in Mwanza, Tanzania, among patients older than 18 years. Participants completed a demographics survey and underwent an ophthalmic examination that included fundus photography. RESULTS: Complete data were available for 1,007 (93.8%) of the 1,073 persons examined. The prevalence of vitreoretinal disorders was 22.8% (230/1,007). The leading retinal diseases were age-related macular degeneration (7.0%), hypertensive retinopathy (4.5%), and macular scars (2.7%). CONCLUSION: This study is the first population-based study of retinal disease in Mwanza. The findings reveal a considerable burden of retinal disease in this region, suggesting a need for trained local ophthalmic personnel and resources. [Ophthalmic Surg Lasers Imaging Retina. 2020;51:S17-S25.].
Assuntos
População Negra , Vigilância da População/métodos , Doenças Retinianas/etnologia , Medição de Risco/métodos , Estudos Transversais , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Prevalência , Fatores de Risco , Tanzânia/epidemiologiaRESUMO
OBJECTIVE: To evaluate the role of serial newborn screening of congenital hypothyroidism using thyroxine (T4) in the neonatal intensive care unit (NICU). SUBJECTS: Newborn screen results were reviewed from a single academic NICU during 2007-2016 (n = 6100). Thyroid function levels were reviewed in patients treated for hypothyroidism during that period. Duration of treatment was followed after discharge. RESULTS: Overall incidence of treated hypothyroidism was 1:103 with increasing incidence inversely related to birth weight. Among treated infants (n = 59), initial newborn screen demonstrated sensitivity and specificity of 74.1% and 84.9%, respectively; second screen demonstrated rates of 85.7% and 76.1%, respectively. Based on follow-up data, prevalence of permanent congenital hypothyroidism in our NICU population was 1:870 (n = 7); two patients would have been missed with a single screen. CONCLUSION: Abnormal T4 on newborn screening is common for preterm neonates. Higher rates of permanent congenital hypothyroidism highlight the need for screening beyond the newborn screen.