Cost of Patients With Hemophilia A and High-Titer Inhibitors in Colombia.

BACKGROUND: In Colombia, hemophilia is considered a high-cost disease, and hemophilia A with high-titer inhibitors may be responsible for a significant economic pressure on the Colombian health system. OBJECTIVES: To estimate the direct cost of care for patients with hemophilia A with high-titer inhibitors in Colombia, from the perspective of the health system. METHODS: A cost-of-illness study was carried out using standard case methodology, which was designed based on literature review and validation by expert consensus. Scenarios were established for adults and children, including cases of prophylaxis, immune tolerance induction, bleeding, and surgery. The frequencies were taken from the official report for Colombia, issued by the High-Cost Account 2017 (reported 2018). The prices were obtained from the list of regulated medicines in the country. The cost estimate is presented with a range of values by weight (between 10 kg and 90 kg). RESULTS: The total estimated cost per year for Colombia was US $44 905 252 (between US $32 260 497 and US $58 202 393). The average cost per year calculated for a patient was US $498 947 (between US $358 450 and US $646 693). A total of 99.8% of the estimated cost was directly related to the cost of the coagulation factors and bypassing agents. CONCLUSIONS: Hemophilia A with high-titer inhibitors is a disease that generates significant pressure on the Colombian health system, mainly linked to the cost of factors and bypassing agents.

Haemophilia care in Latin America: Assessment and perspectives.

INTRODUCTION: The study is the first application of the Principles of Haemophilia Care for Europe (PHCE) in other regions of the world, specifically in Latin America. OBJECTIVE: To identify strengths in the care of haemophilia, and the aspects that should be improved. METHODS: The information was obtained through a questionnaire designed according to the PHCE and answered by specialists in mid-2016. The countries included were as follows: Argentina, Brazil, Chile, Colombia, Costa Rica, El Salvador, Mexico, Panama, Dominican Republic and Venezuela. RESULTS: In most countries, there is a central organization for haemophilia care supported by local groups. The existence of a national registry of people with haemophilia (PWH) was verified in eight countries. Centres of integrated care are located in large cities. In the majority of countries, there was no evidence of the participation of multiple actors in the decision-making. The supply of factor concentrates presents constraints, although it is reported as adequate in half of the countries. In most countries, home treatment is available under special conditions. In most countries, there are restrictions on the use of prophylaxis. The coordination of specialized and emergency services depends on each centre. Unrestricted treatment of inhibitors is performed in most countries. In all countries, there are human resources training programmes; however, clinical and health services researches are not widely developed. CONCLUSION: The study identifies the initial situation of principles of care, as well as the alternatives that must be implemented to achieve improvements in the quality of life of PWH in the region.

Costo efectividad de las pruebas para estratificar riesgo en el tratamiento de la leucemia mieloide aguda (LMA) en población pediátrica / Cost-effectiveness of risk stratification tests for the treatment of acute myeloid leukemia in pediatric patients

RESUMEN Objetivo Estimar la razón de costo-efectividad de las pruebas para estratificación del riesgo en pacientes pediátricos con Leucemia Mieloide Aguda (LMA). Métodos Se construyó un árbol de decisión con años de vida ganados como medida de efectividad. Los costos fueron estimados desde la perspectiva del sistema de salud colombiano. En los costos de la estratificación se incluyeron los costos del tratamiento consecuente con ella. Los precios de medicamentos fueron tomados del SISMED 2008 y el valor monetario de los procedimientos se extrajo del manual tarifario del ISS 2001 adicionando el 30 %. Todos los costos se expresaron en pesos colombianos del 2010 y el producto interno per-cápita de ese año fue empleado como umbral de costo efectividad. Se condujeron análisis de sensibilidad univariados y probabilísticos. Resultados La razón de costo-efectividad incremental de las pruebas de estratificación a todos los pacientes, fue de $8 559 944. Los resultados son sensibles a las probabilidades de recaída, supervivencia al trasplante y efectos secundarios. Conclusión Las pruebas para estratificación del riesgo en LMA son costo-efectivas dentro del sistema de salud colombiano.(AU)

ABSTRACT Objective To estimate the cost-effectiveness of risk-stratification tests for the treatment of acute myeloid leukemia (AML) in pediatric patients. Methods A decision-tree model was built using Life Years Gained as a measure of effectiveness. Costs were estimated considering the perspective of the Colombian health system. Stratification costs included treatment costs based on said stratification. Drug prices were taken from SISMED (Drug Price Information System) 2008 and the monetary value of the procedures was extracted from the ISS 2001 rate manual, plus 30%. All costs were expressed in Colombian pesos for 2010 and the gross domestic product per capita of the same year was used as a cost-effective threshold. Univariate and probabilistic sensitivity analyzes were performed. Results Risk stratification tests have an incremental cost-effectiveness ratio of COP 8,559,944. These results are sensitive to changes in probabilities of relapse, transplant survival and side effects. Conclusion Risk stratification tests for AML treatment in pediatric patients are cost-effective in the context of the Colombian health care system.(AU)

[Cost-effectiveness of risk stratification tests for the treatment of acute myeloid leukemia in pediatric patients]. / Costo efectividad de las pruebas para estratificar riesgo en el tratamiento de la leucemia mieloide aguda (LMA) en población pediátrica.

OBJECTIVE: To estimate the cost-effectiveness of risk-stratification tests for the treatment of acute myeloid leukemia (AML) in pediatric patients. METHODS: A decision-tree model was built using Life Years Gained as a measure of effectiveness. Costs were estimated considering the perspective of the Colombian health system. Stratification costs included treatment costs based on said stratification. Drug prices were taken from SISMED (Drug Price Information System) 2008 and the monetary value of the procedures was extracted from the ISS 2001 rate manual, plus 30%. All costs were expressed in Colombian pesos for 2010 and the gross domestic product per capita of the same year was used as a cost-effective threshold. Univariate and probabilistic sensitivity analyzes were performed. RESULTS: Risk stratification tests have an incremental cost-effectiveness ratio of COP 8,559,944. These results are sensitive to changes in probabilities of relapse, transplant survival and side effects. CONCLUSION: Risk stratification tests for AML treatment in pediatric patients are cost-effective in the context of the Colombian health care system.

OBJETIVO: Estimar la razón de costo-efectividad de las pruebas para estratificación del riesgo en pacientes pediátricos con Leucemia Mieloide Aguda (LMA). MÉTODOS: Se construyó un árbol de decisión con años de vida ganados como medida de efectividad. Los costos fueron estimados desde la perspectiva del sistema de salud colombiano. En los costos de la estratificación se incluyeron los costos del tratamiento consecuente con ella. Los precios de medicamentos fueron tomados del SISMED 2008 y el valor monetario de los procedimientos se extrajo del manual tarifario del ISS 2001 adicionando el 30 %. Todos los costos se expresaron en pesos colombianos del 2010 y el producto interno per-cápita de ese año fue empleado como umbral de costo efectividad. Se condujeron análisis de sensibilidad univariados y probabilísticos. RESULTADOS: La razón de costo-efectividad incremental de las pruebas de estratificación a todos los pacientes, fue de $8 559 944. Los resultados son sensibles a las probabilidades de recaída, supervivencia al trasplante y efectos secundarios. CONCLUSIÓN: Las pruebas para estratificación del riesgo en LMA son costo-efectivas dentro del sistema de salud colombiano.

Guía de atención integral para la detección oportuna, diagnóstico, tratamiento y seguimiento de leucemia linfoide aguda en niños, niñas y adolescentes / Comprehensive guide for early detection, diagnosis, treatment and monitoring of acute leukemia lymphocytic leukaemiaid children and teens

La leucemia linfoide aguda (LLA) es el cáncer más frecuente en niños. A nivel mundial se estima que cerca del 30% corresponde a la totalidad de las neoplasias malignas que se presentan en niños y, de estas, más del 75% son LLA. En Colombia no existe aún un registro nacional de cáncer que nos permita evaluar exactamente la carga de la enfermedad por esta patología; sin embargo, se estima que se presentan aproximadamente 2.080 casos nuevos por año de cáncer en pacientes pediátricos y de estos 500 corresponden a LLA. Con el objetivo de disminuir la mortalidad y mejorar la oportunidad en el diagnóstico y en el tratamiento de los niños afectados por esta patología, se desarrolló esta Guía de práctica clínica (GPC) como producto del trabajo durante meses de un grupo organizado y motivado de médicos especialistas en el área (epidemiólogos, economistas, entre otros), quienes elaboraron recomendaciones para las diferentes etapas tanto para la prevención, sospecha, diagnóstico, tratamiento, seguimiento de los niños y adolescentes con diagnóstico de LLA.

Acute lymphocytic leukaemia (ALL) is the most common cancer in children. Globally, it is estimated that nearly 30% correspond to the total of malignant tumours in children, and 75% of them are ALL. There is no national cancer registry in Colombia that would allow us to precisely assess the burden of the disease for this condition; however, calculations have shown that there are 2,080 new paediatric cancer cases every year, and of those 500 correspond to ALL. With the goal of reducing mortality and improving the chances for a diagnosis and treatment of children affected by this condition, the present clinical practice guide (CPG) was developed after a work of months carried out by an organised and motivated group of physicians specialised in the field (epidemiologists and economists, among others), who formulated recommendations for the different stages: prevention, suspicion, diagnosis, treatment and follow-up of children and teens diagnosed with ALL.

Costo-efectividad de los tratamientos de consolidación para la leucemia mieloide aguda en niños en riesgo alto en el sistema de salud colombiano / Cost-effectiveness of consolidation treatments for acute myeloid leukemia in high-risk pediatric patients within the Colombian healthcare system

Introducción. La leucemia mieloide aguda representa alrededor del 20 % de las leucemias en menores de 18 años. Actualmente, solo existen dos alternativas de tratamiento de consolidación: la quimioterapia y el trasplante con progenitores hematopoyéticos. Objeti vo. Evaluar el costo-efectividad del trasplante alogénico con progenitores hematopoyéticos de donantes emparentados o no emparentados, en comparación con la quimioterapia de consolidación en niños de alto riesgo con leucemia mieloide aguda. Materiales y métodos. Se construyó un árbol de decisiones utilizando los años de vida ganados como resultado. Los costos y probabilidades se extrajeron de estudios y reportes que se encuentran en la literatura científica. El umbral de costo-efectividad fue tres veces el producto interno bruto per cápita de 2010. Se hicieron análisis de sensibilidad univariados y probabilísticos, así como una curva de aceptabilidad. Resultados. Al comparar el trasplante de donante emparentado o no emparentado con los ciclos de quimioterapia, se obtuvieron tasas de costo-efectividad incremental de COP$ 9´226.421 (USD$ 4.820) y COP$ 6´544.116 (USD$ 3.419), respectivamente, cifras estas inferiores al producto interno bruto per cápita: COP$ 12´047.418 (USD$ 6.294). El trasplante resultó ser costo-efectivo en 70 % de las simulaciones y con mayor probabilidad de serlo cuando había disposición a pagar cantidades superiores a COP$ 7´200.000 (USD$ 3.762). Conclusión. El trasplante alogénico (emparentado o no) en Colombia resultó ser costo-efectivo frente al tratamiento de consolidación en niños de alto riesgo con leucemia mieloide aguda.

Introduction: Acute myeloid leukemia represents about 20% of leukemias in minors under 18 years old. At present, there are only two consolidation treatment alternatives: Chemotherapy and stem-cell transplantation. Objective: To evaluate the cost-effectiveness of unrelated and related hematopoietic stem cell transplantations, versus chemotherapy consolidation in pediatric patients with high-risk acute myeloid leukemia. Materials and methods: A decision tree was constructed with life-years gained as the outcome. Costs and probabilities were extracted from the literature. Probabilistic sensitivity analyses and acceptability curves were computed. The cost-effectiveness threshold was three times the 2010 per capita gross domestic product. Results: When compared to consolidation chemotherapy cycles, related and unrelated hematopoietic stem-cell transplantation had incremental cost-effectiveness ratios of COP$ 9,226,421 (USD$ 4,820) and COP$ 6,544,116 (USD$ 3,419) respectively, which are lower than the per capita gross domestic product (COP$ 12,047,418, USD$ 6,294). Transplant proved to be cost-effective in 70% of the simulations and had a higher probability of the willingness to pay being over than COP$ 7,200,000 (USD$ 3,762). Conclusion: In Colombia, related and unrelated hematopoietic stem-cell transplants are cost-effective alternatives to consolidation treatment for high-risk acute myeloid leukemia in pediatric patients.

Evaluating the Development, Implementation and Dissemination of a Multisite Card Study in the WWAMI Region Practice and Research Network.

BACKGROUND: Practice-based research networks (PBRNs) promote the conduct of research in real-world settings by engaging primary care clinicians as champion research collaborators. Card studies are brief surveys administered to patients or clinicians at the point of care. The objective of this paper is to describe the design and evaluation of a card study methodology that the WWAMI Region Practice and Research Network (WPRN) used to develop research partnerships across multiple member sites. METHODS: We used a collaborative model to develop, implement and disseminate the results of a network-wide card study to assess patient preferences for weight loss in primary care. After the card study data collection was completed, we conducted individual and focus group interviews and a brief survey of participating practice champions. RESULTS: Increased research engagement and personal and professional developments were the primary motivators for participating in the development of the card study. Increasing research activity at practices and learning information about patients were motivators for implementing the study. Their participation resulted in champions reporting increased confidence in collaborating on research projects as well as the development of new clinical services for patients. DISCUSSION: This collaborative model positively influenced research capacity in the WPRN and may be a useful strategy for helping PBRNs conducted translational research.

[Cost-effectiveness of consolidation treatments for acute myeloid leukemia in high-risk pediatric patients within the Colombian healthcare system]. / Costo-efectividad de los tratamientos de consolidación para la leucemia mieloide aguda en niños en riesgo alto en el sistema de salud colombiano.

INTRODUCTION: Acute myeloid leukemia represents about 20% of leukemias in minors under 18 years old. At present, there are only two consolidation treatment alternatives: Chemotherapy and stem-cell transplantation. OBJECTIVE: To evaluate the cost-effectiveness of unrelated and related hematopoietic stem cell transplantations, versus chemotherapy consolidation in pediatric patients with high-risk acute myeloid leukemia. MATERIALS AND METHODS: A decision tree was constructed with life-years gained as the outcome. Costs and probabilities were extracted from the literature. Probabilistic sensitivity analyses and acceptability curves were computed. The cost-effectiveness threshold was three times the 2010 per capita gross domestic product. RESULTS: When compared to consolidation chemotherapy cycles, related and unrelated hematopoietic stem-cell transplantation had incremental cost-effectiveness ratios of COP$ 9,226,421 (USD$ 4,820) and COP$ 6,544,116 (USD$ 3,419) respectively, which are lower than the per capita gross domestic product (COP$ 12,047,418, USD$ 6,294). Transplant proved to be cost-effective in 70% of the simulations and had a higher probability of the willingness to pay being over than COP$ 7,200,000 (USD$ 3,762). CONCLUSION: In Colombia, related and unrelated hematopoietic stem-cell transplants are cost-effective alternatives to consolidation treatment for high-risk acute myeloid leukemia in pediatric patients.

Efectividad y seguridad de los regímenes de tratamiento que incluyen inmunoglobulina antitimocítica comparados con ciclofosfamida, ciclosporina, danazol y terapia de soporte / Effectiveness and safety of treatment regimens including antithymocyte immunoglobulin compared to cyclophosphamide, cyclosporine, danazol and supportive therapy

Introducción: La anemia aplásica es una enfermedad poco común que afecta a 2 de cada 1´000.000 de personas anualmente con igual distribución en hombres y mujeres. Se caracteriza por la sustitución del tejido hematopoyético de la medula ósea por grasa causando una pancitopenia periférica, con diferentes niveles de gravedad, originando un síndrome anémico, hemorragias e infecciones graves que pueden llevar a desenlaces fatales de no recibir tratamiento oportuno. La primera opción de tratamiento en pacientes no compatibles para trasplante de progenitores hematopoyeticos es la terapia inmunosupresora. La evidencia actual sugiere la efectividad del tratamiento con inmunoglobulina antitimocítica en los esquemas de tratamiento. Objetivo: Examinar los beneficios y riesgos del uso de la inmunoglobulina antitimocítica en pacientes con anemia aplásica no hereditaria severa a muy severa. Metodología: Se realizó una búsqueda sistemática de estudios clinicos incluyendo utilizando las bases de datos MEDLINE (In-Process & Other Non-Indexed Citations y Daily Update) EMBASE, Cochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects ­ DARE, LILACS y la revisión de publicaciones obtenidas por otros medios comparando el uso de inmunoglobulina antitimocitica en pacientes con anemia aplasica no hereditaria, no candidatos a trasplante de prognitores hematopoyéticos con otros tratamientos inmunosupresores, considerando los resultados en cuanto a tasa de respuesta, sobrevida, recaída y transformación clonal. Resultados: Se obtuvieron 103 publicaciones, 24 fueron tamizadas para valoración de los criterios de inclusión. Tres cumplieron con estos y se evaluaron con las herramientas de la colaboración Cochrane. Todas las referencias reportaron alto riesgo de sesgo. Un ensayo clinico controlado no reportó diferencias significativas para ninguno de los desenlaces evaluados comparando CTX más CsA frente a ATGr más CsA. Uno de los estudios de cohorte reportó una efectividad de 77.8% y sobrevida a 5 años fue de 74.1%. La segunda cohorte reportó respuesta completa (17.7%), respuesta parcial (37.9%) y tasa de respuesta global (55.6%). La recaida fue de 3.2%, la transformacion clonal 0.8% y la sobrevida a 5 años fue 74.7%. Conclusiones: La evidencia sobre la efectividad y seguridad de la inmunoglobulina antitimocitica es limitada y de baja calidad. Con los hallazgos obtenidos en esta revision no es posible determinar la superioridad de esta tecnología frente a otras opciones de tratamiento disponibles.(AU)

Relationship between English language use and preferences for involvement in medical care among Hispanic women.

OBJECTIVE: To assess how English language use by Hispanic women affects their preferences for participating in decision making and information seeking regarding medical care. METHODS: The study included 235 Hispanic women aged 35-61 years participating in a larger multicenter study, the Ethnicity, Needs, and Decisions of Women (ENDOW) Project. Participants were recruited from community settings and primary care public health clinics. Bilingual (English and Spanish speaking) interviewers asked participants questions about demographic characteristics, health status, reproductive history, menopausal status, access to healthcare, experience with hormone replacement therapy (HRT) and hysterectomy, outcome expectations about HRT and hysterectomy, medical decision making, and social support. Using univariate and multivariate analyses, we assessed the relationships between the participants' preferences for participating in decision making and information seeking, their language use, and other covariates of interest. RESULTS: Overall, the participants expressed a strong desire for information about and participating in medical decisions. However, they expressed a lower preference for participating in decisions related to use of HRT compared with the desire for engaging in decision involving invasive medical procedures (hysterectomy and cholecystectomy) and high blood pressure management. Increased use of English language was significantly associated with preferences for participating in medical care decision making, in general (p < 0.001), and with information seeking (p = 0.044). Decreased use of English language was associated with a lower desire for participating in medical care decision making. CONCLUSIONS: Increased use of English language may influence Hispanic women's preferences for participating in medical decisions and their information-seeking behavior.