Development and evaluation of a customized checklist to assess the quality control of disease registry systems of Tehran, the capital of Iran in 2021.

BACKGROUND: Clinical registries facilitate medical research by providing 'real data'. In the past decade, an increasing number of disease registry systems (DRS) have been initiated in Iran. Here, we assessed the quality control (QC) of the data recorded in the DRS established by Shahid Beheshti University of Medical Sciences in Tehran, the capital city of Iran, in 2021. METHODS: The present study was conducted in two consecutive qualitative and quantitative phases and employed a mixed-method design. A checklist containing 23 questions was developed based on a consensus reached following several panel group discussions, whose face content and construct validities were confirmed. Cronbach's alpha was calculated to verify the tool's internal consistency. Overall, the QC of 49 DRS was assessed in six dimensions, including completeness, timeliness, accessibility, validity, comparability, and interpretability. The seventy percent of the mean score was considered a cut-point for desirable domains. RESULTS: The total content validity index (CVI) was obtained as 0.79, which is a reasonable level. Cronbach's alpha coefficients obtained showed acceptable internal consistency for all of the six QC domains. The data recorded in the registries included different aspects of diagnosis/treatment (81.6%) and treatment quality requirements outcomes (12.2%). According to the acceptable quality cut-point, out of 49 evaluated registries, 48(98%), 46(94%), 41(84%), and 38(77.5%), fulfilled desirable quality scores in terms of interpretability, accessibility, completeness, and comparability, however, 36(73.5%) and 32(65.3%) of registries obtained the quality requirement for timeliness and validity, respectively. CONCLUSION: The checklist developed here, containing customized questions to assess six QC domains of DRSs, provided a valid and reliable tool that could be considered as a proof-of-concept for future investigations. The clinical data available in the studied DRSs fulfilled desirable levels in terms of interpretability, accessibility, comparability, and completeness; however, timeliness and validity of these registries needed to be improved.

Herramientas diagnostico-terapéuticas en síndromes mielodisplásicos en Uruguay / Diagnostic and therapeutic tools in myelodysplastic syndromes in Uruguay / Instrumentos diagnóstico-terapêuticos nas síndromes mielodisplásicas no Uruguai

Los Síndromes Mielodisplásicos (SMD) son un grupo heterogéneo de enfermedades mieloides. Esta heterogeneidad en la presentación clínica complejiza el diagnóstico requiriendo diversos estudios complementarios. El tratamiento debe ser individualizado y adaptado al riesgo, desde terapias de soporte hasta intervenciones de alto costo. Para conocer la accesibilidad a las herramientas diagnóstico y terapéuticas se realizó una encuesta online dirigida a los hematólogos que asisten pacientes con SMD en Uruguay en 2016 y 2019. Las encuestas fueron respondidas por 32.5% y 26.6% de los miembros de la Sociedad de Hematología del Uruguay. Más del 90% tienen acceso a estudios histológicos, citogenéticos, FISH y citometría de flujo. La posibilidad de realizar paneles de secuenciación masiva se encuentra restringida a menos de 10% derivando la muestra al exterior, siendo mayor en 2019 en comparación a 2016. Los sistemas de estratificación de riesgo más utilizados son el sistema internacional de puntuación de riesgo (IPSS) y su versión revisada (IPSS-R). La disponibilidad de tratamientos de soporte (transfusiones, eritropoyetina y G-CSF), de azacitidina y del trasplante alogénico de precursores hematopoyéticos es amplia. Existió un aumento en indicación de azacitidina en 2019 con respecto a 2016. Sin embargo, el acceso a decitabina, lenalidomida y fármacos quelantes de hierro es escaso y no se cuenta con ensayos clínicos donde incluir pacientes que fallan o no responden a los tratamientos convencionales. La presente encuesta, realizada en dos períodos, describe la realidad y su evolución en nuestro país en cuanto a accesibilidad a herramientas diagnósticas y terapéuticas extrapolables a otras patologías oncohematológicas. Los datos recabados permitirán plantear estrategias tendientes a mejorar el abordaje diagnóstico-terapéutico de los pacientes con SMD en Uruguay.

Myelodysplastic Syndromes (MDS) constitutes an heterogenous group of hematological malignancies. Reaching an accurate diagnosis, represents in an important number of cases, a major challenge that requires different diagnostic tools. In order to acknowledge the scope of access to those tools in our country, we performed a survey addressed to Uruguayan hematologists who care for MDS patients in their clinical practice. The survey was carried out in 2016 and 2019 among Uruguayan Hematology Society members. Response rate was 32.5% and 26.6% respectively. Access to bone marrow biopsy, cytogenetics, FISH and flow cytometry was accessible to more than 90% of physicians. Less than 10% of respondents were able to request next generation sequencing (NGS) studies and in that case, they have to send them abroad. IPSS and R-IPSS were the most frequently used risk scores. Support treatment such as growth factors and transfusions are widely accessible. Azacytidine and allogenic transplant are available as well. However, access to decitabine, lenalidomide and iron chelating drugs is scarce and there are no clinical trials to include patients who fail or do not respond to conventional treatments. This survey, carried out in two periods, describes the reality and its evolution in our country in terms of accessibility to diagnostic and therapeutic tools that can be extrapolated to other oncohematological pathologies. We were able to get to know our country reality regarding diagnostic and therapeutic tools for MDS patients. This, would represent an important input in order to design health strategies aiming to improve clinical care for our patients.

As Síndromes Mielodisplásicas (SMD) são um grupo heterogêneo de doenças mielóides. Essa heterogeneidade na apresentação clínica torna o diagnóstico mais complexo, exigindo vários estudos complementares. O tratamento deve ser individualizado e adaptado ao risco, desde terapias de suporte até intervenções de alto custo. Para conhecer a acessibilidade de ferramentas diagnósticas e terapêuticas, foi realizada uma pesquisa online dirigida aos hematologistas que atendem pacientes com SMD no Uruguai em 2016 e 2019. As pesquisas foram respondidas por 32,5% e 26,6% dos membros da Sociedad de Hematologia do Uruguai. Mais de 90% têm acesso a estudos histológicos, citogenéticos, FISH e citometria de fluxo. A possibilidade de realização de painéis de sequenciamento massivo está restrita a menos de 10% provenientes da amostra no exterior, sendo maior em 2019 em relação a 2016. Os sistemas de estratificação de risco mais utilizados são o sistema internacional de pontuação de risco (IPSS) e sua versão revisada (IPSS -R). Tratamentos de suporte (transfusões, eritropoietina e G-CSF), azacitidina e transplante alogênico de células-tronco hematopoiéticas estão amplamente disponíveis. Houve aumento da indicação de azacitidina em 2019 em relação a 2016. No entanto, o acesso a decitabina, lenalidomida e quelantes de ferro é escasso e não há ensaios clínicos para incluir pacientes que falham ou não respondem aos tratamentos convencionais. Este inquérito, realizado em dois períodos, descreve a realidade e a sua evolução no nosso país em termos de acessibilidade a instrumentos diagnósticos e terapêuticos que podem ser extrapolados para outras patologias onco-hematológicas. Os dados coletados permitirão propor estratégias destinadas a melhorar a abordagem diagnóstico-terapêutica de pacientes com SMD no Uruguai.

Validation of the Brazilian version of the hepatitis B quality of life evaluation instrument - HBQOL, and its application to patients with chronic hepatitis B in Cascavel - PR

Abstract The objective of this study is to validate the specific questionnaire for Hepatitis B HBQOL (Hepatitis B Quality of Life Instrument, version 1.0) for the Brazilian version, in addition to testing its applicability in patients with hepatitis B under treatment and comparing the quality of life between patients using first-line drugs (tenofovir and entecavir). For the validation, the back-translation technique was used in a sample of 47 patients. Factor analysis was performed between the items in each domain of the questionnaire and the internal consistency was calculated using Cronbach's α coefficient. In assessing the applicability of the validated questionnaire, interviews were carried out with 124 patients. Sociodemographic and treatment data were collected to characterize the sample and perform correlation analyzes. The results demonstrate that the Brazilian version of the questionnaire was successfully validated. In the analysis carried out among the 124 patients, the domains psychological well-being and stigma obtained the highest scores in quality of life and the lowest level of education conferred better results in these two domains. The comparison between tenofovir and entecavir showed no significant difference in patients' quality of life. The use of this validated instrument can make therapeutic decisions more rational

Plano estratégico nacional de resposta ao hiv e sida - pen v (2021 - 2025) / National strategic plan for the response to hiv and aids - pen v (2021 - 2025)

O Plano Estratégico Nacional (PEN) de Resposta ao HIV e SIDA 2021-2025 é o quinto plano de Moçambique. Baseia-se no progresso alcançado até o momento, aborda as causas da epidemia e lacunas identificadas durante os últimos cinco anos e busca ampliar as melhores práticas para garantir que a qualidade e a inovação sustentem a prestação de serviços. O PEN V descreve a estrutura estratégica para uma parceria multissetorial visando acelerar, ainda mais, o progresso na redução das novas infecções pelo HIV e mortes relaccionadas ao SIDA em Moçambique. As províncias desenvolverão planos de implementação provinciais específicos para o contexto local a fim de operacionalizar, com maior detalhe, os objectivos estratégicos e as acções recomendadas. Este processo descentralizado permitirá que as estratégias nacionais para o HIV sejam adaptadas às necessidades e condições específicas das províncias, distritos, municípios e comunidades. Os ministérios, o sector privado e a sociedade civil desenvolverão também os seus próprios planos de implementação sectoriais, todos alinhados com PEN V. O PEN V visa articular um quadro estratégico que irá orientar a visão política e programática da resposta nacional ao HIV e SIDA para os próximos cinco anos (2021-2025); informar as partes interessadas a nível nacional, provincial, distrital, municipal e comunitário sobre as orientações estratégicas, e, por último, servir de enquadramento para coordenar e acompanhar a implementação das acções da resposta nacional ao HIV e SIDA.

An Excel program for calculating statistics presented in Marfak et al.'s article 'Improved RIDIT statistic approach provides more intuitive and informative interpretation of EQ-5D Data'.

The objective is to present and share an Excel program that we have developed to perform statistical analyses based on the Improved RIDIT approach of Marfak et al.'s article 'Improved RIDIT statistic approach provides more intuitive and informative interpretation of EQ-5D Data'.

Comparison of Methods for Estimating Therapy Effects by Indirect Comparisons: A Simulation Study.

Objective. In evidence synthesis, therapeutic options have to be compared despite the lack of head-to-head trials. Indirect comparisons are then widely used, although little is known about their performance in situations where cross-trial differences or effect modification are present. Methods. We contrast the matching adjusted indirect comparison (MAIC), simulated treatment comparison (STC), and the method of Bucher using a simulation study. The different methods are evaluated according to their power and type I error rate as well as with respect to the coverage, bias, and the root mean squared error (RMSE) of the effect estimate for practically relevant scenarios using binary and time-to-event endpoints. In addition, we investigate how the power planned for the head-to-head trials influences the actual power of the indirect comparison. Results. Indirect comparisons are considerably underpowered. None of the methods had substantially superior performance. In situations without cross-trial differences and effect modification, MAIC and Bucher led to similar results, while Bucher has the advantage of preserving the within-study randomization. MAIC and STC could enhance power in some scenarios but at the cost of a potential type I error inflation. Adjusting MAIC and STC for confounders that did not modify the effect led to higher bias and RMSE. Conclusion. The choice of effect modifiers in MAIC and STC influences the precision of the indirect comparison. Therefore, a careful selection of effect modifiers is warranted. In addition, missed differences between trials may lead to low power and partly high bias for all considered methods, and thus, results of indirect comparisons should be interpreted with caution.

[Therapeutic positioning reports: Experience in Spain during the period 2013-2019]. / Informes de posicionamiento terapéutico: experiencia en España en el periodo 2013-2019.

OBJECTIVE: Describe the characteristics of the therapeutic positioning reports (TPRs) published in Spain in the period 2013-2019. DESIGN AND DATA SOURCE: Systematic review of all TPRs published in the website of the Spanish Agency of Medicines and Health Products (AEMPS). SELECTION OF STUDIES: All TPRs published since May 2013, until March 2019 DATA EXTRACTION: The main variables collected were the therapeutic groups assessed, the number of TPRs, the time of elaboration, the existence of restrictions versus the authorized indications and the information on the efficiency of medicines. RESULTS: During the period under review, 214 TPRs were carried out, with an average production time of 8.8 months, almost three times the objective of 3-month initially set. 57% of the TPRs established restrictions of use with respect to the approved indications. 26% of TPRs referred to the existence of economic data, although none included details on the efficiency. 10% of TPRs were updated. CONCLUSIONS: For TPRs to meet their objective of improving the efficiency of the assessment process and the consistency in the decisions on price, reimbursement and financing of medicines by the SNS, the deadlines established for publication must be met, incorporating systematically information on the efficiency of the drugs and including periodic updates with the new information generated.

Questionnaire measuring patient participation in health care: Scale development and psychometric evaluation.

BACKGROUND: Patient participation in treatment and care is often encouraged and is desirable because of its proven positive impact on treatment, quality of care and patient safety. AIMS: To develop an instrument to measure patient participation in health care and to investigate the measurement properties of the Patient Participation Questionnaire (PPQ). METHODS: A literature review was conducted to develop a model of patient participation. The PPQ was constructed consisting of 17 items organized into four subscales. Psychometric evaluation of factor structure, convergent construct validity by hypothesis testing and analyses of internal consistency using Cronbach's alpha were performed on data from a hospitalised mixed group of patients with cardiac disease, pulmonary disease and cancer (N=378 patients). RESULTS: Confirmatory factor analysis did not show a clear model fit, which is why an exploratory factor analysis was performed, suggesting a different four subscale structure consisting of a total of 16 items. The four subscales were labelled Shared decision power, Adapted and individualized knowledge, Collaboration and Human approach. There were strong ceiling effects on all items. Analysis of convergent construct validity showed a moderate correlation (0.59) between the PPQ and another instrument measuring patient participation. Internal consistency for the total PPQ score was high: 0.89. CONCLUSION: In a mixed group of patients with cardiac disease, pulmonary disease and cancer, the PPQ showed promising psychometric properties in terms of factor structure, convergent construct validity and internal consistency. The PPQ may be used to shed light on the experience of patient participation and guide quality improvements.

Re-recognition of pseudogenes: From molecular to clinical applications.

Pseudogenes were initially regarded as "nonfunctional" genomic elements that did not have protein-coding abilities due to several endogenous inactivating mutations. Although pseudogenes are widely expressed in prokaryotes and eukaryotes, for decades, they have been largely ignored and classified as gene "junk" or "relics". With the widespread availability of high-throughput sequencing analysis, especially omics technologies, knowledge concerning pseudogenes has substantially increased. Pseudogenes are evolutionarily conserved and derive primarily from a mutation or retrotransposon, conferring the pseudogene with a "gene repository" role to store and expand genetic information. In contrast to previous notions, pseudogenes have a variety of functions at the DNA, RNA and protein levels for broadly participating in gene regulation to influence the development and progression of certain diseases, especially cancer. Indeed, some pseudogenes have been proven to encode proteins, strongly contradicting their "trash" identification, and have been confirmed to have tissue-specific and disease subtype-specific expression, indicating their own value in disease diagnosis. Moreover, pseudogenes have been correlated with the life expectancy of patients and exhibit great potential for future use in disease treatment, suggesting that they are promising biomarkers and therapeutic targets for clinical applications. In this review, we summarize the natural properties, functions, disease involvement and clinical value of pseudogenes. Although our knowledge of pseudogenes remains nascent, this field deserves more attention and deeper exploration.

The intersection between being a nurse and being a therapist in Mental Health.

OBJECTIVE: to discuss the intersection between being a nurse/being a therapist in the mental health field. METHOD: exploratory, qualitative study based on a self-report interview conducted with ten nurses from a psychiatric hospital in Rio de Janeiro between June and July 2017. Data were analyzed in the light of Travelbee's theory. RESULTS: Nurses consider themselves therapists because of actions performed in mental health care practice, despite the theoretical fragmentation of interpersonal relationships identified in the distance from the promotion of self-knowledge, which is an essential element in the psychotherapeutic process. FINAL CONSIDERATIONS: the strong relationship between being and doing in nursing stands out and favors the development of satisfactory therapeutic relationships with the hospitalized person.

Estimation of Controlled Direct Effects in Longitudinal Mediation Analyses with Latent Variables in Randomized Studies.

In a randomized study with longitudinal data on a mediator and outcome, estimating the direct effect of treatment on the outcome at a particular time requires adjusting for confounding of the association between the outcome and all preceding instances of the mediator. When the confounders are themselves affected by treatment, standard regression adjustment is prone to severe bias. In contrast, G-estimation requires less stringent assumptions than path analysis using SEM to unbiasedly estimate the direct effect even in linear settings. In this article, we propose a G-estimation method to estimate the controlled direct effect of treatment on the outcome, by adapting existing G-estimation methods for time-varying treatments without mediators. The proposed method can accommodate continuous and noncontinuous mediators, and requires no models for the confounders. Unbiased estimation only requires correctly specifying a mean model for either the mediator or the outcome. The method is further extended to settings where the mediator or outcome, or both, are latent, and generalizes existing methods for single measurement occasions of the mediator and outcome to longitudinal data on the mediator and outcome. The methods are utilized to assess the effects of an intervention on physical activity that is possibly mediated by motivation to exercise in a randomized study.

Higher rates of bleeding and use of treatment products among young boys compared to girls with von Willebrand disease.

There are limited observational studies among children diagnosed with von Willebrand Disease (VWD). We analyzed differences in bleeding characteristics by sex and type of VWD using the largest reported surveillance database of children with VWD (n = 2712), ages 2 to 12 years old. We found that the mean ages of first bleed and diagnosis were lowest among children with type 3 VWD. It was even lower among boys than girls among all VWD types, with statistically significant difference among children with type 1 or type 3 VWD. Children with type 3 VWD also reported higher proportions of ever having a bleed compared to other VWD types, with statistically higher proportions of boys compared to girls reporting ever having a bleed with type 1 and type 2 VWD. A similar pattern was observed with the use of treatment product, showing higher usage among type 3 VWD, and among boys than girls with type 1 and type 2 VWD. While there were no differences in life quality or in well-being status by sex, children with type 3 VWD showed a greater need for mobility assistance compared to children with type 1 and type 2 VWD. In an adjusted analysis among children with type 1 VWD, boys showed a significant association of ever bleeding [hazard ratio 1.4; P-value <.001)] compared to girls. Understanding phenotypic bleeding characteristics, well-being status, treatment, and higher risk groups for bleeding among pre-adolescent children with VWD will aid physicians in efforts to educate families about bleeding symptoms.

Shared Decision Making and Treatment for Minority Children With ADHD.

Shared decision making (SDM) involves the patient and family in medical decisions regarding treatment. The purpose of this article is to utilize the 2016 National Survey of Children's Health to explore whether family engagement in SDM increases the odds of treatment for children with ADHD, and more specifically, if the presence of SDM is associated with the reduction of racial and ethnic disparities in treatment. Multivariate logistic regression was used to model the odds of treatment for each racial/ethnic group controlling for sociodemographic and health-related variables. Results indicated that White and Multiracial families engaged in SDM were twice as likely to report treatment for ADHD. Black and Latinx families, however, showed no difference in treatment for ADHD when SDM was present. Based on these findings, we conclude that SDM may be less important for Black and Latinx families when making treatment decisions for children with ADHD.

The intersection between being a nurse and being a therapist in Mental Health / La intersección entre ser enfermero y ser terapeuta de Salud Mental / A interseção entre ser enfermeiro e ser terapeuta em saúde mental

ABSTRACT Objective: to discuss the intersection between being a nurse/being a therapist in the mental health field. Method: exploratory, qualitative study based on a self-report interview conducted with ten nurses from a psychiatric hospital in Rio de Janeiro between June and July 2017. Data were analyzed in the light of Travelbee's theory. Results: Nurses consider themselves therapists because of actions performed in mental health care practice, despite the theoretical fragmentation of interpersonal relationships identified in the distance from the promotion of self-knowledge, which is an essential element in the psychotherapeutic process. Final considerations: the strong relationship between being and doing in nursing stands out and favors the development of satisfactory therapeutic relationships with the hospitalized person.

RESUMEN Objetivo: discutir la intersección entre ser un enfermero/ser un terapeuta en el campo de la salud mental. Método: investigación exploratoria y cualitativa, basada en una entrevista de autoevaluación realizada con diez enfermeras de un hospital psiquiátrico en Rio de Janeiro entre junio y julio de 2017. Los datos se analizaron a la luz de la Teoría de Travelbee. Resultados: Los enfermeros se consideran terapeutas por las acciones desarrolladas en la práctica de la atención de salud mental, a pesar de la fragmentación teórica de la relación interpersonal identificada en la distancia de la promoción del autoconocimiento, que es un elemento esencial en el proceso psicoterapéutico. Consideraciones finales: sobresale la fuerte relación entre el ser y el hacer de enfermería, la cual favorece el desarrollo de relaciones terapéuticas satisfactorias con la persona hospitalizada.

RESUMO Objetivo: discutir a interseção do ser enfermeiro/ser terapeuta no campo da saúde mental. Método: pesquisa exploratória, qualitativa, pautada na entrevista de autorrelato desenvolvida com dez enfermeiras de um hospital psiquiátrico do Rio de Janeiro entre junho e julho de 2017. Os dados foram analisados à luz da teoria de Travelbee. Resultados: As enfermeiras se consideram terapeutas em função das ações desenvolvidas na prática de cuidados em Saúde Mental, apesar da fragmentação teórica do relacionamento interpessoal identificada no distanciamento da promoção de autoconhecimento, que é elemento imprescindível no processo psicoterapêutico. Considerações finais: A forte relação entre ser e fazer da enfermagem se destaca e favorece o desenvolvimento de relações terapêuticas satisfatórias com a pessoa internada.

Tratamento da úlcera plantar devido à hanseníase / Treatment of leprosy-induced plantar ulcers

Introdução: A úlcera plantar por hanseníase é uma lesão no pé resultante da falta de sensibilidade plantar. O objetivo é descrever o tratamento realizado em portadores de úlceras plantares por hanseníase. Métodos: Estudo de prontuários de portadores de úlcera plantar atendidos no Hospital Sarah em Brasília, de 2006 a 2016, quanto ao sexo, idade, etiologia, localização e tratamento. Resultados: Foram atendidos 27 pacientes, 17(62,96%) homens e 10 (37,04%) mulheres, procedentes de Goiás e DF, na faixa etária de 41 a 60 anos (40,74%). Todos necessitaram de um ou mais procedimentos cirúrgicos. Conclusão: Observou-se maior frequência no sexo masculino, grau avançado, localizadas no primeiro artelho. Todos necessitaram de procedimentos cirúrgicos e não cirúrgicos, evoluindo com cicatrização completa da ferida, amputação transtibial em um caso e de artelhos em sete casos, e 90% dos casos apresentaram recorrência da úlcera após um ano.

Introduction: Leprosy-induced plantar ulcers result from a lack of plantar sensitivity. Objective: This study aimed to describe the treatment provided to patients with leprosy-induced plantar ulcers. Methods: We retrospectively reviewed the medical records of patients with plantar ulcers treated at Sarah Hospital in Brasilia from 2006 to 2016 and collected information about sex, age, etiology, location, and treatment. Results: A total of 27 patients (17 [62.96%] men, 10 [37.04%] women; 40.74% were aged 41­60 years) were treated from Goiás and the Federal District. All required ≥1 surgical procedure. Conclusion: A higher frequency of advanced grade was observed in men, primarily on the first toe. All needed surgical and non-surgical procedures and achieved complete wound healing. Transtibial amputation was required in 1 case and toe amputation in 7 cases; 90% patients developed ulcer recurrence after 1 year.

The geography and economics of forgoing medical examinations or therapeutic treatments in Italy during the economic crisis.

BACKGROUND: In Italy, the number of individuals who have forgone medical examinations or treatments for economic reasons is one of the highest in Europe. During the global economic crisis of 2008, the restrictive policies concerning access to healthcare and the quality of these services, which differs widely throughout the country, may have accentuated the territorial differences in unmet needs, thereby penalizing the more disadvantaged segments of the population. The study aimed at evaluating the geographical and socioeconomic differences, in particular the risk of poverty, that influence forgoing healthcare services in Italy. METHODS: Cross-sectional Italian data from the 2004-2015 European Survey on Income and Living Conditions (EU-SILC) were used. Hierarchical logistic models were tested, using as the outcome unmet needs for medical examinations or treatment in the preceding 12 months, and as risk factor the condition of being at risk of poverty. Age, sex, citizenship, educational level, presence of chronic or severely limiting diseases and self-perceived health were used as adjustment factors. Analyses were stratified over three time periods: pre-crisis (2004-2007), initial phase of the crisis (2008-2012) and second phase of the crisis (2013-2015). RESULTS: In Central Italy and particularly in Southern Italy, a marked increase (9.9% in 2013-2015) was seen in the overall rate of unmet needs as well as in that of unmet needs due to economic reasons. The probability of unmet needs was higher, and increased over time, for those at risk of poverty (aOR = 1.54 in 2004-07, aOR = 1.70 in 2008-12, aOR = 2.21 in 2013-15). Individuals with a low educational level, who had a chronic or severely limiting disease, who perceived their health as not good and immigrants had a higher risk of forgoing healthcare. The regions in Southern Italy had a significantly higher probability of unmet needs. CONCLUSIONS: A strong association was found between the probability of forgoing medical examination or treatment and being at risk of poverty. Study results underline the need for healthcare policies aimed at facilitating access to healthcare services, particularly in the South, by developing a progressive mechanism of contribution to healthcare costs proportional to income and by guaranteeing free access to the poor.

Estimating the quality of optimal treatment regimes.

When multiple treatment alternatives are available for a disease, an obvious question is which alternative is most effective for which patient. One may address this question by searching for optimal treatment regimes that specify for each individual the preferable treatment alternative based on that individual's baseline characteristics. When such a regime has been estimated, its quality (in terms of the expected outcome if it was used for treatment assignment of all patients in the population under study) is of obvious interest. Obtaining a good and reliable estimate of this quantity is a key challenge for which so far no satisfactory solution is available. In this paper, we consider for this purpose several estimators of the expected outcome in conjunction with several resampling methods. The latter have been evaluated before within the context of statistical learning to estimate the prediction error of estimated prediction rules. Yet, the results of these evaluations were equivocal, with different best performing methods in different studies, and with near-zero and even negative correlations between true and estimated prediction errors. Moreover, for different reasons, it is not straightforward to extrapolate the findings of these studies to the context of optimal treatment regimes. To address these issues, we set up a new and comprehensive simulation study. In this study, combinations of different estimators with .632+ and out-of-bag bootstrap resampling methods performed best. In addition, the study shed a surprising new light on the previously reported problematic correlations between true and estimated prediction errors in the area of statistical learning.

A multicenter real-world review of 10 kHz SCS outcomes for treatment of chronic trunk and/or limb pain.

Objectives: High-frequency spinal cord stimulation (HF-SCS) at 10 kHz has proven to be efficacious in the treatment of chronic back and leg pain in a randomized, controlled, trial (SENZA-RCT). However, large observational studies have yet to be published. Therefore, we performed a real-world, multicenter, retrospective, review of therapy efficacy in 1660 patients with chronic trunk and/or limb pain. Methods: Data were collected in a real-world environment and retrospectively sourced from a global database. Included patients were trialed and/or permanently implanted with HF-SCS at 10 kHz between April 2014 and January 2018. We evaluated responder rates at 3, 6, and 12 months post-implantation. Response was defined as ≥50% pain relief from baseline. A last visit analysis included responder rate along with overall change in function, sleep, quality of life, and medication intake versus baseline. Results: Eighty-four percent of our HF-SCS-treated patients had both chronic back and leg pain. At least 70% of patients reported response to therapy throughout 12 months of follow-up. This sustained responder rate was corroborated by the last visit value (74.1%). Most patients reported concomitant improvements in function (72.3%), sleep (68.0%), and quality of life (90.3%) at their last visit versus baseline. Thirty-two percent of patients reported decreased medication intake at their last visit. Interpretation: Sustained and effective pain relief was experienced by >70% of our HF-SCS-treated patients, consistent with the findings of a previously published randomized, controlled, trial. Our review provides complementary evidence to support the treatment of chronic back and leg pain with this therapy.