ABSTRACT
La profilaxis en el tratamiento de la hemofilia ha sido crucial en la mejoría del pronóstico y calidad de vida en las personas con hemofilia (PCH). A pesar de ello, no está globalmente implementado y no ha sido ejecutado satisfactoriamente en Latinoamérica, donde es difícil evaluar la situación, y el manejo de las PCH no se ajusta a los estándares ideales. El grupo GLAITH (Grupo Latino Americano para el Impulso del Tratamiento de la Hemofilia) discutió el problema a través de una encuesta entre sus integrantes. Los hallazgos fueron discutidos en Bogotá en mayo del 2013 en donde los participantes definieron los puntos esenciales a comunicar en un llamado a la acción. Las proporciones de casos de hemofilia A reportados fueron entre 75 y 90% y entre 10 y 25%, los de hemofilia B. La hemofilia grave representó entre el 26 y el 55% de los casos. Un alto porcentaje de PCH tiene artropatía hemofílica. La atención de PCH varía en cada país, sólo se cubre entre el 50 y 60% del tratamiento, que es a demanda en el 85 a 95% de los casos. Sólo 5 a 15% reciben profilaxis, la mayoría secundaria. Pocos países tienen programa nacional o registros homogéneos. En llamado a la acción y conclusión para la región se recomienda: establecimiento de un registro latinoamericano unificado; estudios prospectivos de costo efectividad y evaluación de criterios en profilaxis secundaria; estudios comparativos de calidad de vida, individualización del tratamiento e implementación de la profilaxis en forma global en Latinoamérica.
Prophylactic treatment in the management of hemophilia has been a crucial factor in improving the prognosis and quality of life for people with hemophilia (PCH). However, it is not globally implemented. In Latin America it is difficult to assess the status of PCH and the its management does not conform to ideal standards. The GLAITH group discussed the problem in Latin America. A survey of its members and its findings were discussed at a meeting in Bogota in May 2013. Proportions of hemophilia A and B were 75-90% and 10-25% respectively. Severe hemophilia represents 26-55% of cases. A high percentage of PCH have hemophilic arthropathy. The general care and specific treatments of PCH vary by country, only 50-60% of the treatment is covered and in 85-95% of the cases are performed on an ondemand basis. Just 5-15% receives prophylaxis, most of them secondary. Few countries have a national program or homogeneous records. Finally the GLAITH group proceeded to develop a conclusion and call to action for the region where the following points are recommended: the establishment of a unified Latin American registry; prospective cost-effectiveness studies and evaluation criteria related to secondary prophylaxis; comparative studies of quality of life with and without prophylaxis in the region; promotion of individualization of treatment and, the increase of primary and secondary prophylaxis globally in Latin America.
Subject(s)
Humans , Quality of Life , Hemophilia B/therapy , Hemophilia A/therapy , Primary Prevention/methods , Prognosis , Severity of Illness Index , Secondary Prevention/methods , Latin AmericaABSTRACT
BACKGROUND: While primary prophylaxis is a well-established and recommended treatment for children with severe hemophilia, there has not been Korean own data. The aim of this study is to lay the foundation for the optimal and available management of hemophilia in Korea.METHODS: We reviewed the medical records of the patients treated with regular factor VIII concentrates infusion as long term continuous treatment at Seoul Clinic of Korea Hemophilia Foundation since August, 2004, retrospectively. We analyzed the efficacy of prophylaxis with the frequency of joint bleed and clinically meaningful bleeding episodes. We also assessed the cost of primary prophylaxis, secondary prophylaxis and on-demand therapy by the factor concentrates consumption.RESULTS: The data from 65 patients were available to evaluate. Of 65 patients, 22 were treated with primary prophylaxis, and 43 with secondary prophylaxis. In the primary and secondary prophylaxis groups, there were 1.9 joint bleeds and 3.3 other bleeding episodes, 7.2 and 9.0, respectively. The mean consumption of factor concentrates per person for primary prophylaxis, secondary prophylaxis and on-demand therapy groups were 2,658.8 IU/kg/year, 2,665.1 IU/kg/year, and 1,271.9 IU/kg/year respectively.CONCLUSION: Now prophylaxis is the recommended treatment for severe hemophilia patients, we should manage our hemophilia patients with standard treatment. And the optimizing treatment regimen for each individual will be needed to establish by studies of individual pharmacokinetics and bleeding tendency.
Subject(s)
Child , Humans , Factor VIII , Hemophilia A , Hemorrhage , Joints , Korea , Medical Records , Retrospective StudiesABSTRACT
BACKGROUND: While primary prophylaxis is a well-established and recommended treatment for children with severe hemophilia, there has not been Korean own data. The aim of this study is to lay the foundation for the optimal and available management of hemophilia in Korea. METHODS: We reviewed the medical records of the patients treated with regular factor VIII concentrates infusion as long term continuous treatment at Seoul Clinic of Korea Hemophilia Foundation since August, 2004, retrospectively. We analyzed the efficacy of prophylaxis with the frequency of joint bleed and clinically meaningful bleeding episodes. We also assessed the cost of primary prophylaxis, secondary prophylaxis and on-demand therapy by the factor concentrates consumption. RESULTS: The data from 65 patients were available to evaluate. Of 65 patients, 22 were treated with primary prophylaxis, and 43 with secondary prophylaxis. In the primary and secondary prophylaxis groups, there were 1.9 joint bleeds and 3.3 other bleeding episodes, 7.2 and 9.0, respectively. The mean consumption of factor concentrates per person for primary prophylaxis, secondary prophylaxis and on-demand therapy groups were 2,658.8 IU/kg/year, 2,665.1 IU/kg/year, and 1,271.9 IU/kg/year respectively. CONCLUSION: Now prophylaxis is the recommended treatment for severe hemophilia patients, we should manage our hemophilia patients with standard treatment. And the optimizing treatment regimen for each individual will be needed to establish by studies of individual pharmacokinetics and bleeding tendency.
Subject(s)
Child , Humans , Factor VIII , Hemophilia A , Hemorrhage , Joints , Korea , Medical Records , Retrospective StudiesABSTRACT
Children with chronic liver disease or portal vein thrombosis frequently develop portal hypertension. The main complication of the portal hypertension are the development of esophageal varices who are at risk of variceal bleeding, which is associated with significant morbidity and mortality. Guidelines for adults with portal hypertension recommend performing esophagogastroduodenoscopy to identify those with varices that may benefit from prophylactic therapy, however there are no consensus in the pediatric age. This revision includes the incidence, diagnostic and the evidence for primary prophylactic treatment of the esophageal varices in children.
Pacientes pediátricos con daño hepático crónico o con trombosis de la vena porta frecuentemente desarrollan hipertensión portal. La principal complicación de la hipertensión portal es el desarrollo de várices esofágicas que conlleva riesgo de hemorragia variceal, la cual se asocia a significativa morbimortalidad. Las guías clínicas para adultos recomiendan realizar, en pacientes cirróticos, una endoscopia como screening para el diagnóstico de várices esofágicas y su tratamiento profiláctico, sin embargo, en pacientes pediátricos no existe consenso. En esta revisión se actualiza la incidencia, diagnóstico y evidencia del tratamiento profiláctico primario de várices esofágicas en pediatría.
Subject(s)
Humans , Child , Antibiotic Prophylaxis , Gastrointestinal Hemorrhage/prevention & control , Esophageal and Gastric Varices/surgery , Esophageal and Gastric Varices/drug therapy , Adrenergic beta-Antagonists/therapeutic use , Chronic Disease , Endoscopy , Gastrointestinal Hemorrhage/etiology , Hypertension, Portal/complications , Hepatic Insufficiency/complications , Esophageal and Gastric Varices/diagnosis , Esophageal and Gastric Varices/etiologyABSTRACT
Esta investigación tuvo como objetivo comparar los resultados del propanolol combinado o no con ligadura endoscópica (LE) en la profilaxis primaria del sangrado variceal en pacientes que acudieron al Servicio de Gastroenterología del Hospital Universitario de Maracaibo, durante los meses de Enero a Octubre 2009. La investigación fue de tipo correlacional, prospectiva, longitudinal. La población estuvo conformada por 40 pacientes entre 18 y 75 años de edad con Cirrosis Hepática y varices esofágicas de tamaño mediano-grande con o sin signos rojos, sin antecedentes de hemorragia digestiva superior. Se seleccionaron al azar 2 grupos, el primero estuvo representado por 20 pacientes, quienes fueron sometidos a LE combinado con Propanolol y el segundo por 20 pacientes tratados sólo con Propanolol. Para la recolección de datos se diseñó un cuestionario basado en las variables, dimensiones e indicadores propuestos en la investigación. Se demostró que el propanolol como monoterapia es tan efectivo como combinado con LE en la profilaxis primaria del sangrado variceal.
This research was aim to relate the results propranolol combined or not with endoscopic ligation (LE) in the primary prophylaxis of variceal bleeding in the Gastroenterology Service, Hospital Universitario de Maracaibo, during the months of January to October 2009. A correlational, prospective, longitudinal type study. The population was 40 patients between 18 and 75 years old with liver cirrhosis and esophageal varices of large-medium size, with no history of upper gastrointestinal bleeding. They were two randomly selected samples, the first is represented by 20 patients who underwent LE combined with propranolol and the second 20 patients treated with propranolol alone. For data collection a questionnaire was designed based on the variables, dimensions and indicators proposed in the research. We demonstrated that monotherapy with propranolol is as effective as combined with LE in the primary prophylaxis of variceal bleeding.