Análise das ações tomadas pela comissão de farmácia e terapêutica do Instituto Dante Pazzanese de Cardiologia em 2021 e 2022 / Analysis of the actions taken by the pharmacy and therapeutics committee of the Instituto Dante Pazzanese de Cardiologia in 2021 and 2022

INTRODUÇÃO E OBJETIVOS: A Comissão de Farmácia e Terapêutica (CFT) é uma instância de caráter consultivo e deliberativo que assessora a Diretoria Geral com objetivo de formular diretrizes e promover uso racional de medicamentos. O objetivo foi mensurar decisões tomadas pela CFT que impactem positivamente para economia de recurso orçamentário sem prejuízo assistencial. MÉTODOS: Estudo descritivo realizado no período de janeiro de 2021 a dezembro de 2022 sobre as ATAS mensais da CFT e sistema de gestão hospitalar eletrônica. Os dados coletados foram: assuntos discutidos, deliberação, valor unitário do medicamento. Foi considerado assuntos com impacto financeiro: substituição da padronização, despadronização, inclusão de medicamento com dosagem de melhor manejo, inclusão de restrição de prescrição para itens de alto valor. Os dados foram coletados em planilha Excel. RESULTADOS E DISCUSSÃO: Foram avaliadas 24 ATAS de CFT, em que foram selecionados temas voltados para farmacoeconomia. Do total, havia 5 pautas (21%) focadas no assunto: despadronização: 2 (40%): metoprolol 100 mg (tem de 50 mg), bosentana 62,5 mg (pertence ao Componente Especializado e o paciente já faz uso domiciliar e passa a ser orientado a trazer de casa), substituição: 1 (20%) sildenafila de 20 mg para 50 mg (preço unit de $12,20 para $0,49), inclusão de dosagem: 1 (20%) alteplase 10 mg ($491,19) sendo que o alteplase 50 mg custa $ 2.356,55 (dose depende do peso e havia desperdício) e inclusão de restrição 1 (20%): levosimendana ($4.900,00). Baseado no consumo anual, fizemos o cálculo de economia: metoprolol e bosentana ($4.373,37), sildenafila ($109.383,11), alteplase ($17.971,22), levosimendana ($ 88.200,00). Essa mudança não deixou o paciente desassistido, pois foi considerado substituições viáveis envolvendo o corpo clínico para adequação de prescrições médicas e apoio da Diretoria Clínica. CONCLUSÃO: Na busca de diminuir gastos com medicamentos sem afetar a assistência do paciente, a Comissão de Farmácia e Terapêutica possibilitou meios para economizar recurso orçamentário, promovendo reuniões extras com alguns setores estratégicos para demonstrar gastos com determinados itens e propor medidas que pudessem minimizar esse impacto financeiro. Houve uma compreensão e aceitação das áreas envolvidas. No montante, o hospital conseguiu economizar R$ 219.927,70. Financiamento e agradecimento: Aos membros da Comissão de Farmácia e Terapêutica que opinaram e sugeriram alternativas terapêuticas plausíveis.

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.

Incidência de falência terapêutica aos regimes de tratamento antirretroviral (tarv) de primeira escolha em crianças menores de 15 anos em moçambique, ano de 2019 / INCIDENCE OF THERAPEUTIC BANKRUPTCY TO SCHEMES OF ANTIRETROVIRAL TREATMENT (ART) OF FIRST CHOICE IN CHILDREN UNDER 15 YEARS IN MOZAMBIQUE, YEAR 2019

Moçambique tem uma das maiores epidemias de HIV em crianças no continente africano. Estimava-se que 150 mil crianças entre 0 a 14 anos viviam com HIV em 20191 . Embora o acesso ao Tratamento Antirretroviral (TARV) ainda constitua um desafio, com apenas 63% das crianças vivendo com HIV em TARV em 2019, o número de crianças em TARV aumentou quase 5 vezes nos últimos 10 anos. Este aumento deveu-se, em parte, a implementação da estratégia Testar e Iniciar, iniciada em 20162 . A implementação desta estratégia foi acompanhada pela expansão da capacidade laboratorial de testagem de Carga Viral (CV) em todo o país, com objectivo de um melhor monitoramento do TARV e diagnóstico precoce de Falência Terapêutica (FT) em pacientes em TARV. Até 2016, a suspeita e identificação de FT em Moçambique foi baseada, principalmente, em critérios clínicos e imunológicos pelo provedor de saúde, com baixa sensibilidade e especificidade quando comparado ao teste de CV capaz de detectar falência virológica, principalmente em crianças3,4. Actualmente, o Ministério da Saúde (MISAU) recomenda dois testes de CV separados por três a seis meses, intercalados por sessões de aconselhamento de adesão, avaliação clínica e imunológica. Quando um paciente tem dois dos seguintes três critérios: (1) resultados consecutivos com CV ≥1,000 cópias/ml; (2) progressão clínica da infecção, expressa principalmente por novas infecções e/ou recorrentes ou tumores oportunistas do estadio III ou IV; (3) ou contagem de células T CD4 persistentemente abaixo de 100 células/ mm3 após 12 meses de TARV, é considerado como tendo FT e uma mudança de tratamento é recomendada5 . Embora o número de crianças em TARV tenha aumentado nos últimos anos, existe pouca ou nenhuma informação sobre a incidência de FT aos regimes de primeira escolha em crianças menores de 15 anos em Moçambique. Desta forma, foi objectivo desta análise estimar a incidência de FT aos regimes de primeira escolha, o Tempo Médio de FT (TMFT) e identificar os regimes terapêuticos com maior incidência de FT entre crianças menores de 15 anos em Moçambique. O conhecimento das taxas de incidência de FT e dos regimes terapêuticos associados a menor incidência pode ajudar os clínicos e fazedores de políticas a escolher e/ou recomendar regimes terapêuticos mais duradoiros em pacientes

Using GRADE evidence to decision frameworks to choose from multiple interventions.

BACKGROUND AND OBJECTIVE: Guideline development groups or other health care decision makers frequently encounter situations that require a simultaneous comparison of multiple interventions. This sometimes becomes apparent either when they identify questions of interest, before they formulate recommendations, or it may surface only when recommendations have already been formulated based on pairwise comparisons. METHODS: Using examples from the World Health Organization, the European Commission, and a professional society, we developed a flexible approach to developing recommendations when a multiple-intervention comparison (MC) is needed using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) evidence to decision (EtD) frameworks. We iteratively refined this approach through user testing and then included a module in GRADE's official software GRADEpro to test the approach in two real and one theoretical guideline recommendations. RESULTS: We found the approach feasible and that all EtD criteria should be considered in an MC approach. We judged that guideline development groups and other decision makers will benefit from the availability of a network meta-analyses (NMA) of intervention effects to support decisions; however, NMA supports only one of many criteria, that is, the balance of health benefits and harms, and is therefore helpful, but not essential to the approach we propose. When similar but not identical comparators are used to address MC, challenges may arise with intransitivity and the relative rankings of interventions. CONCLUSION: We successfully applied the MC approach and software module in generating recommendations across different scenarios and identified challenges. The MC approach allows guideline groups and other decision makers to transparently and critically assess multiple options for a given health question. Application of the approach by others may lead to refinement and allow for better understanding of its impact in developing recommendations and making choices.

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.

The pediatric patient and future fertility: optimizing long-term male reproductive health outcomes.

Globally, male factor infertility accounts for 20%-70% of couples struggling to conceive. Certain male pediatric developmental conditions, such as cryptorchidism, hypospadias, testicular and other childhood cancers, infections, and pediatric varicocele have been associated with future infertility. Early fertility preservation, especially in those with pending chemotherapy or genetic conditions such as Klinefelter syndrome, should be strongly considered in patients expected to experience testicular loss. Although optimal treatment timing may be unknown owing to a paucity of long-term prospective studies, early diagnosis and targeted treatment may optimize fertility potential in adulthood.

Terapias avanzadas / Advanced therapies

No disponible

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.

La UAT: una adaptación del marco de Diálogo Abierto finlandés / The UAT: adaptation of the Finnish Open Dialogue framework

La UAT (Unidad de Atención Temprana) es un dispositivo especializado en el acompañamiento de personas que afrontan por primera vez experiencias que han sido calificadas como psicóticas. El encuadre del trabajo es el marco de Diálogo Abierto, que intentamos adaptar a nuestro contexto en los servicios públicos de salud del área de Alcalá de Henares. En este artículo hablaremos de qué es el Diálogo Abierto, el proceso de adaptación del marco a nuestro contexto, las dificultades y aspectos positivos de esta experiencia, y los resultados que hemos podido observar durante el recorrido de la unidad

The Early Attention Unit is a specialized unit focused on the attention of people who are facing for the first time experiences that have been described as psychotic. The frame of this work is the Open Dialogue approach, which we are trying to adapt to our context in the public health services of Alcalá de Henares. In this article, we will outline the Open Dialogue approach, the process to adapt it to our context, the difficulties and positive aspects of this experience, and the results we have observed during the development of the unit

[National survey of the therapeutic apheresis in Hungary, 2013-2017]. / A terápiás apheresisek számának és indikációjának változása, valamint új eljárások megjelenése Magyarországon 2013 és 2017 között.

Therapeutic apheresis is a treatment option for several subspecialities. It is a relatively expensive intervention, which can only be done by dedicated centers based on specific indications. The Therapeutic Apheresis Committee and the National Health Insurance Fund of Hungary jointly control the number of interventions to be made, the introduction of new diagnoses and the application of new apheresis procedures in Hungary. In this work, we review the therapeutic practice of the period between 2013 and 2017 in Hungary, describing also the new modalities under implementation. Orv Hetil. 2019; 160(19): 727-738.

Healing activities construct the objects of therapy: Medicine's way of seeking truth, organizing forms of reality, regulating patients' bodies, illness and culture?

In this paper, I will explore the concept that healing activities shape the objects of therapy and seek to construct those objects through therapeutic activities. Objects of therapy are the persons, patients, human bodies, diseases, physiological processes and personal suffering-that which clinical medicine constructs through its distinctive formative processes, practices and knowledge. The rationale for choice of philosophical sources namely, Cassirer, Foucault, the anthropological perspective of Good and the sociological account of Frank will be discussed. The claim articulated by Good will be examined and its relationship to culture, illness, medical knowledge, practice, truth, and science. I then focus on Frank's concepts of the patient and the body and how medical knowledge and practices affects it. The concept that the medicalization of the illness narrative silences the patient's voice requiring an ethic of listening will be emphasized, described and further supported by Charon's (2006, Narrative medicine: Honoring the stories of illness. New York, NY: Oxford University Press) and Cassell's (2015, The nature of suffering and the goals of medicine. New York, NY: Oxford University Press) thoughts on narrative of illness in clinical medicine. My position concludes that healing activities construct the objects of therapy: as the medical culture's way of seeking truth; as medicine's way of mediating and organizing forms of reality through culture and symbolic forms; and, as medicine's way of entering the body and constructing the disease. Lastly, I suggest that in spite of the remarkable progress in the control of disease, the failure to address the interpretation of illness meanings is a fundamental flaw in the work of "doctoring." The experience and meanings of illness are at the centre of clinical practice and is a moral, political, ethical and professional obligation. The person is a cultural construct, a complex and culturally shaped way of experiencing self and other, and cultural "work" is required to constitute the person who is the object of medical attention and it also necessitates the ethic of listening.

Boosting treatment stabilization in patients of amphetamine-type stimulant use disorder.

BACKGROUND: To investigate boosting effects on treatment stabilization in the mandatory treatment modality for patients of amphetamine-type stimulant use disorder. METHODS: This is a retrospective follow-up study over a period from January 2013 to December 2018. We analyzed 425 patients of amphetamine-type stimulant use disorder under mandating treatments. Treatment stabilization for a given patient was defined once 4 negative urinalysis had been observed. We developed a dynamic monitoring model of boosting effects informed by the available data, specifically the number of negative urine samples required to reach stabilization, the sum of urinalyses done at the time when the given number of negative urine samples had been observed and who the patient was. To represent the simulated population, a Monte Carlo method was used to generate p-values from 1000 experiments conducted on a computer. RESULTS: In the observed samples, the probability of 4 negative results in urinalysis from 4 outpatient visits was 75.5%. In comparison, the probability for achieving 4th negative results in urinalysis over 4 visits from negative binominal distribution was 57.3%, and from the computer simulation, 49.8%. The observed samples had significantly higher probability of achieving 4 negative results in urinalysis over 4 outpatient visits (p < 0.001). CONCLUSIONS: The mandatory treatment modality boosted treatment stabilization for patients of amphetamine-type stimulant use disorder. The major benefit of using the monitoring model is the ability to monitor boosting effects of stabilization. Results supported the effectiveness of this mandatory treatment modality and can be implemented in deferred-prosecution based treatment modality.

Cost Sharing of Disease-Modifying Treatments (DMTs) as Policy Lever to Improve DMTs' Access in Multiple Sclerosis.

OBJECTIVES: To understand the nonlinear relationship between out-of-pocket (OOP) payments and disease-modifying treatment (DMT) use and adherence, primarily to pinpoint the threshold at which the use of DMTs becomes price sensitive. METHODS: Individuals with more than two multiple sclerosis (MS) diagnoses (International Classification of Diseases, Ninth Revision code 340) were identified from the MarketScan database (2006-2009). Heterogeneity in treatment was normalized by calculating an annual OOP payment as the average OOP payment for purchasing a fixed basket of DMTs at the insurance plan level. A local linear regression with a model-based recursive partitioning algorithm was applied to explore the relationship between OOP and consequently lower DMT use and adherence as measured by days covered by DMT. RESULTS: We identified the inflection points in annual OOP payments as $442 for DMT use and $890 for DMT adherence. For patients with annual OOP payments of more than $442, a $100-increase in OOP payment was associated with a decline of 0.6% in DMT use; for annual OOP payments of more than $890, a $100-increase in OOP payment was associated with two fewer days of DMT treatments. CONCLUSIONS: Although the use of DMTs and DMT adherence appeared unassociated with OOP payment below $442 and $890, respectively, an excessive OOP payment was a barrier to DMT access. This information can inform maximum monthly and yearly payment caps when designing valued-based insurance plans.

Considerations when assessing heterogeneity of treatment effect in patient-centered outcomes research.

When baseline risk of an outcome varies within a population, the effect of a treatment on that outcome will vary on at least one scale (e.g., additive, multiplicative). This treatment effect heterogeneity is of interest in patient-centered outcomes research. Based on a literature review and solicited expert opinion, we assert the following: (1) Treatment effect heterogeneity on the additive scale is most interpretable to health-care providers and patients using effect estimates to guide treatment decision-making; heterogeneity reported on the multiplicative scale may be misleading as to the magnitude or direction of a substantively important interaction. (2) The additive scale may give clues about sufficient-cause interaction, although such interaction is typically not relevant to patients' treatment choices. (3) Statistical modeling need not be conducted on the same scale as results are communicated. (4) Statistical testing is one tool for investigations, provided important subgroups are identified a priori, but test results should be interpreted cautiously given nonequivalence of statistical and clinical significance. (5) Qualitative interactions should be evaluated in a prespecified manner for important subgroups. Principled analytic plans that take into account the purpose of investigation of treatment effect heterogeneity are likely to yield more useful results for guiding treatment decisions.

Transitioning patients with immune thrombocytopenia to second-line therapy: Challenges and best practices.

In patients with immune thrombocytopenia who do not adequately respond to first-line therapy, there is no clear consensus on which second-line therapy to initiate and when. This situation leads to suboptimal approaches, including prolonged exposure to treatments that are not intended for long-term use (eg, corticosteroids) and overuse of off-label therapies (eg, rituximab) while approved, more efficacious options exist. These approaches may not only fail to address symptoms and burden of disease, but may also worsen health-related quality of life. A better understanding of available second-line treatments may ensure best use of therapeutic options and thereby optimize patient outcomes.