Manejo de la trombocitopenia inmune primaria. Comparación de dos cohortes históricas / Management of primary immune thrombocytopenia. A comparison between two historical cohorts

Introducción: En los últimos años se han experimentado cambios en el manejo de los pacientes con trombocitopenia inmune primaria. En este estudio se revisan las características de los pacientes con trombocitopenia inmune primaria del Hospital Infantil Universitario Niño Jesús y su evolución. Además, analizamos los cambios en el abordaje de los pacientes diagnosticados antes y después de 2011, año en el que se publicó la guía de la Sociedad Española de Pediatría. Material y métodos: Se han revisado retrospectivamente los datos de pacientes con trombocitopenia inmune primaria en seguimiento en nuestro hospital desde el año 2000. El paquete estadístico utilizado para el análisis fue SPSS Statistics 22.0 (IBM Corp, Chicago, IL, EE.UU.). Resultados: Se han revisado 235 pacientes pediátricos con trombocitopenia inmune primaria, observando que algunas características al diagnóstico, como la edad menor de 5años y los antecedentes previos de infección, pueden influir en la probabilidad de recuperación. Con respecto al cambio de manejo de los pacientes, a partir de 2011 las dosis de esteroides recibidas durante el primer mes y el primer año se han reducido de forma significativa, así como el número de días del primer ingreso, pasando de 5 a 3días. Las esplenectomías también se han reducido significativamente. Conclusiones: Desde el año 2011 se han producido cambios en el abordaje de nuestros pacientes: reciben una menor dosis de esteroides, permanecen menos días ingresados y se ha reducido el número de esplenectomías sin aumentar los sangrados y sin disminuir la tasa de respuestas. Además, observamos que la edad menor de 5años y el antecedente de infección previa al diagnóstico están relacionados con una mayor tasa de recuperación. (AU)

Introduction: In recent years, there have been changes in the management of patients with primary immune thrombocytopenia. In this study, a review is presented of the characteristics and outcomes of children with primary immune thrombocytopenia in a children's hospital (Hospital Infantil Niño Jesús, Madrid, Spain). Moreover, an analysis is made of the changes in the care of these patients diagnosed before and after 2011, when new guidelines were published by the Spanish Society of Paediatric Haematology Oncology (SEHOP). Material and methods: Data from a cohort of primary immune thrombocytopenia patients followed up in this hospital have been retrospectively reviewed. The statistical package used for the analysis was SPSS Statistics 22.0 (IBM Corp, Chicago, IL, USA). Results: A review is presented on the clinical data from 235 paediatric patients diagnosed with primary immune thrombocytopenia. It was observed that some features at diagnosis, such as age younger than 5years and a previous history of infection, influenced the probability of cure. Regarding the changes in the management of patients since 2011, the steroid doses received during the first month and the first year, and the number of days corresponding to the patient's first admission have both significantly decreased. Splenectomies were also significantly reduced. Conclusions: Since 2011, there have been changes in the medical care of our primary immune thrombocytopenia patients: they receive lower doses of steroids, they stay fewer days in the hospital, and the number of splenectomies has decreased without increasing bleeding or worsening the clinical evolution. Furthermore, it was observed that age younger than 5years and a history of infection prior to diagnosis were related to higher chances of recovery. (AU)

Management of primary immune thrombocytopenia. A comparison between two historical cohorts.

INTRODUCTION: In recent years, there have been changes in the management of patients with primary immune thrombocytopenia. In this study, a review is presented of the characteristics and outcomes of children with primary immune thrombocytopenia in a children's hospital (Hospital Infantil Niño Jesús). Moreover, an analysis is made of the changes in the care of these patients diagnosed before and after 2011, when new guidelines were published by the Spanish Society of Paediatric Haematology Oncology (SEHOP). MATERIAL AND METHODS: Data from a cohort of primary immune thrombocytopenia patients followed up in this hospital have been retrospectively reviewed. The statistical package used for the analysis was SPSS Statistics 22.0 (IBM Corp., Chicago, IL, USA). RESULTS: A review is presented on the clinical data from 235 paediatric patients diagnosed with primary immune thrombocytopenia. It was observed that some features at diagnosis, such as age younger than five years and a previous history of infection, influenced the probability of cure. Regarding the changes in the management of patients since 2011, the steroid doses received during the first month and the first year, and the number of days corresponding to the patient's first admission have both significantly decreased. Splenectomies were also significantly reduced. CONCLUSIONS: Since 2011, there have been changes in the medical care of our primary immune thrombocytopenia patients: they receive lower doses of steroids, they stay fewer days in the hospital, and the number of splenectomies has decreased without increasing bleeding or worsening the clinical evolution. Furthermore, it was observed that age younger than 5 years and a history of infection prior to diagnosis were related to higher chances of recovery.

Manifestaciones orales en pacientes con Trombocitopenia inmune primaria. Reporte de caso / Oral manifestations in primary immune thrombocytopenia patients. Case report

Resumen Introducción: La trombocitopenia inmune primaria (PTI) es una enfermedad caracterizada por la destrucción acelerada de plaquetas o la producción inadecuada de estas. Se puede clasificar según su etiología en primaria y secundaria. Métodos: Se presenta el caso de un paciente sexo masculino de 30 años de edad, que acude al servicio de urgencia dental del Hospital Barros Luco, por lesiones hemorrágicas mucosa oral y pequeñas manchas rojas en manos, brazos y espalda, de veinticuatro horas de evolución, asintomáticas y sin causa atribuible. Al examen físico se corrobora la presencia de múltiples vesículas hemorrágicas en región bucal y petequias en las zonas descritas. Se realiza diagnóstico presuntivo de PTI que se confirma al obtener un recuento plaquetario (RP) de 2000 uL. Se deriva a medicina interna para evaluación y manejo. La literatura describe que para confirmar esta enfermedad se requiere una historia clínica exhaustiva, descartar una patología sistémica o infecciosa previa y pruebas de laboratorio que evidencien recuento normal de células sanguíneas con una trombocitopenia marcada. Conclusión: El odontólogo tiene un rol significativo en el manejo multidisciplinario en este tipo de cuadros donde el conocimiento de manifestaciones orales de enfermedades sistémicas es de vital importancia para el diagnóstico, derivación y posterior tratamiento de estas.

Abstract Introduction: Primary immune thrombocytopenia (ITP) is a disease characterized by accelerated destruction of platelets or inadequate production of platelets. It can be classified according to its etiology in primary and secondary. Methods: We present the case of a 30-year-old male patient, who comes to the dental emergency service of the Barros Luco Hospital, for oral mucosa hemorrhagic lesions and small red spots on the hands, arms and back, 24 hours of evolution, asymptomatic and without attributable cause. Physical examination corroborates the presence of multiple hemorrhagic vesicles in the oral region and petechiae in the areas described. A presumptive diagnosis of ITP is made and confirmed by obtaining a platelet count (RP) of 2000 uL. He is referred to internal medicine for evaluation and management. The literature describes that confirming this disease requires an exhaustive medical history, ruling out a previous infectious or systemic pathology, and laboratory tests that show normal blood cell counts with marked thrombocytopenia. Conclusions: The dentist has a significant role in multidisciplinary management in this type of condition, where knowledge of oral manifestations of systemic diseases is of vital importance for the diagnosis, referral and subsequent treatment of these. Oral manifestations in primary immune thrombocytopenia patients. Case report

[Primary immune thrombocytopenia: Experience of a specialised clinic]. / Trombocitopenia inmune primaria: experiencia de una consulta especializada.

INTRODUCTION: Although primary immune thrombocytopenia (ITP) is rare in childhood, it is the most frequent cause of thrombocytopenia. There have been attempts to establish risk factors to predict the progression of the disease in order to optimise its management, which has changed in recent years due to, among other reasons, specialised care. MATERIAL AND METHODS: A retrospective, observational and analytical study was conducted on patients diagnosed with ITP over a 3-year period in a Paediatric Haematology specialist clinic. RESULTS: From the epidemiological, clinical and analytical point of view, the characteristics of this group are similar to others. Most of the patients (23/31, 74.2%) had ITP for less than 12 months, with there being no serious complications related to the disease or the treatment received. It was established that risk factors were related to being slowly evolving (lower event-free survival (EFS)) with no statistical significance, female gender, age over 10 years, leukopenia absence of initial severe thrombocytopenia, and non-specialised care. The absence of a history of infection was significantly related to a lower EFS. CONCLUSIONS: The epidemiological and analytical risk factors for a slowly evolving ITP are the same that described in the literature. Patients treated before the beginning of specialised care also had a lower EFS. These data seem to support the current recommendation that rare diseases should be managed in specialised units.

[Protocol for the study and treatment of primary immune thrombocytopenia: PTI-2018]. / Protocolo de estudio y tratamiento de la trombocitopenia inmune primaria: PTI-2018.

Primary immune thrombocytopenia, formerly known as immune thrombocytopenic purpura, is a disease for which the clinical and therapeutic management has always been controversial. The ITP working group of the Spanish Society of Paediatric Haematology and Oncology has updated its guidelines for diagnosis and treatment of primary immune thrombocytopenia in children, based on current guidelines, bibliographic review, clinical assays, and member consensus. The main objective is to reduce clinical variability in diagnostic and therapeutic procedures, in order to obtain best clinical results with minimal adverse events and good quality of life.

Trombocitopenia inmune primaria e infección por citomegalovirus y Epstein Barr virus: autoinmunidad versus inmunosupresión / Immune thrombocytopenia primary and cytomegalovius, Epstein Barr virus infection: immunosuppression versus autoimmunity

La trombocitopenia inmune primaria está asociada con múltiples factores que pueden conducir a la pérdida de autotolerancia a los antígenos en la superficie de las plaquetas y los megacariocitos. Varios agentes infecciosos han sido implicados. Los virus herpes debido a la alta prevalencia en la población y el tropismo por las células linfoides se han relacionado con el desarrollo de esta entidad. Se realizó una revisión bibliográfica utilizando motores de búsqueda como Ebsco y Pubmed que permitió el acceso a artículos relacionados en revistas arbitradas. Se recolectó y organizó información sobre la implicación de la infección por citomegalovirus y EBV en el desarrollo de trombocitopenia inmune primaria. La mayoría de los artículos indican que en estos virus, la naturaleza no citolítica de la replicación favorece el tiempo de interacción y modulación en las células huésped que son frecuentemente linfocitos B, T y asesinas naturales. El diagnóstico oportuno y la terapia adecuada en estos pacientes contribuyen a la inmunomodulación de la autorreactividad y eliminación viral que, a la luz de los conocimientos actuales, es esencial para el tratamiento clínico integrado. Es necesario considerar el monitoreo del estado serológico y molecular de estos herpes virus, en pacientes en los que la historia natural de la enfermedad sugiere su asociación, especialmente en trombocitopenia inmunitaria primaria o secundaria; por el alto nivel de relación de los mecanismos de producción de la autoinmunidad, la dismielopoyesis y la linfoproliferación, con la patogénesis de la infección por estos virus(AU)

Primary immune thrombocytopenia is associated with multiple factors that may lead to loss of self-tolerance to the antigens on the surface of platelets and megakaryocytes. Several infectious agents have been implicated. Herpes viruses due to the high prevalence in the population and tropism by the lymphoid cells have been related to the development of this entity. A bibliographic review was made using search engines such as Ebsco and Pubmed that allowed access to related articles in magazines arbitrated. Information was collected and organized that involved the role of cytomegalovirus and EBV infection in the development of ITP. Most of the articles indicate that in these viruses, the non-cytolytic nature of replication favors the time of interaction and modulation on host cells that are frequently B, T and natural killer lymphocytes. Timely diagnosis and appropriate therapy in these patients contributes to the immunomodulation of self-reactivity and viral elimination, in the light of current knowledge, is essential for integrated clinical treatment. Consider monitoring the serological and molecular status of these herpes viruses in patients in whom the natural history of the disease suggests their association, especially in primary or secondary immune thrombocytopenia; by the high level of relation of the mechanisms of production of the autoimmunity, the dysmielopoyesis and the lymph proliferation with the pathogenesis of the infection by these viruses(AU)

Evolución de un cuadro de hemorragia intracraneal en lactante con trombocitopenia inmune primaria / Evolution of a case of intracraneal hemorrhage in infant with primary immune trombocytopenia

La trombocitopenia inmune primaria (TIP) es una enfermedad hematológica de causa inmunológica que presenta una plaquetopenia inferior a 100 000 plaquetas/mm3 y tiene una incidencia de 4 casos por cada 100 000 habitantes, siendo más prevalente entre los 2 a 6 años de edad. Esta entidad continúa siendo un desafío con respecto a la terapéutica y puede conllevar a complicaciones graves muy difíciles de resolver una vez establecidas.Se presenta el caso de un lactante menor de sexo masculino que curso con por un cuadro de palidez generalizada con aparición súbita de hematoma en mucosa oral, máculas equimóticas y hemorragias puntiformes en toda la superficie corporal. En los exámenes realizados se evidenciaron trombocitopenia y anemia grave, llegando al diagnóstico de trombocitopenia inmune primaria inicialmente manejada con transfusiones y posteriormente con corticoides. A pesar del manejo, el cuadro evolucionó con complicaciones de hemorragia intraparenquimatosa y declino neurológico.

Primary immune thrombocytopenia (PIT) is a hematological disease of immunological cause that has platelet count less than 100 000 platelets / mm3 and has an incidence of 4 cases per 100 000 inhabitants, being more prevalent between 2 and 6 years of age. This entity remains a challenge with regard to therapeutics and can lead to serious complications that are difficult to resolve once established. We report the case of a young male infant who has a generalized pallor with sudden onset of hematoma in the oral mucosa, equimotic macules and punctate hemorrhages throughout the body surface. In the examinations performed thrombocytopenia and severe anemia were evident, arriving at the diagnoses of idiopathic thrombocytopenic purpura initially managed with transfusions and later with corticoids. despite the treatment the case progress whith complications of intraparenchymal hemorrhage accompanied by neurological decline

Manifestaciones orales en pacientes con Trombocitopenia inmune primaria.Reporte de caso

Introducción: La trombocitopenia inmune primaria (PTI) es una enfermedad caracterizada por la destrucción acelerada de plaquetas o la producción inadecuada de estas. Se puede clasificar según su etiología en primaria y secundaria. Métodos: Se presenta el caso de un paciente sexo masculino de 30 años de edad, que acude al servicio de urgencia dental del Hospital Barros Luco, por lesiones hemorrágicas mucosa oral y pequeñas manchas rojas en manos, brazos y espalda, de veinticuatro horas de evolución, asintomáticas y sin causa atribuible. Al examen físico se corrobora la presencia de múltiples vesículas hemorrágicas en región bucal y petequias en las zonas descritas. Se realiza diagnóstico presuntivo de PTI que se confirma al obtener un recuento plaquetario (RP) de 2000 uL. Se deriva a medicina interna para evaluación y manejo. La literatura describe que para confirmar esta enfermedad se requiere una historia clínica exhaustiva, descartar una patología sistémica o infecciosa previa y pruebas de laboratorio que evidencien recuento normal de células sanguíneas con una trombocitopenia marcada. Conclusión: El odontólogo tiene un rol significativo en el manejo multidisciplinario en este tipo de cuadros donde el conocimiento de manifestaciones orales de enfermedades sistémicas es de vital importancia para el diagnóstico, derivación y posterior tratamiento de estas.

Introduction: Primary immune thrombocytopenia (ITP) is a disease characterized by accelerated destruction of platelets or inadequate production of platelets. It can be classified according to its etiology in primary and secondary. Methods: We present the case of a 30-year-old male patient, who comes to the dental emergency service of the Barros Luco Hospital, for oral mucosa hemorrhagic lesions and small red spots on the hands, arms and back, 24 hours of evolution, asymptomatic and without attributable cause. Physical examination corroborates the presence of multiple hemorrhagic vesicles in the oral region and petechiae in the areas described. A presumptive diagnosis of ITP is made and confirmed by obtaining a platelet count (RP) of 2000 uL. He is referred to internal medicine for evaluation and management. The literature describes that confirming this disease requires an exhaustive medical history, ruling out a previous infectious or systemic pathology, and laboratory tests that show normal blood cell counts with marked thrombocytopenia. Conclusions: The dentist has a significant role in multidisciplinary management in this type of condition, where knowledge of oral manifestations of systemic diseases is of vital importance for the diagnosis, referral and subsequent treatment of these. Oral manifestations in primary immune thrombocytopenia patients. Case report

Trombocitopenia inmune primaria refractaria: opciones terapéuticas / Refractory primary immune thrombocytopenia: treatment options

La trombocitopenia inmune primaria (PTI) es un trastorno autoinmune adquirido caracterizado por una disminución transitoria o persistente del recuento de plaquetas con riesgo incrementado de sangramiento. La forma crónica de la enfermedad afecta fundamentalmente a los adultos. Puesto que las remisiones espontáneas son muy poco frecuentes, los pacientes son tratados desde el inicio, usualmente con esteroides. Aproximadamente un tercio de los casos no responden a los regímenes esteroideos y el tratamiento de segunda línea es la esplenectomía, tratamiento con el que se logran las mayores tasas de curación. Sin embargo, entre el 10 y el 30 por ciento de los pacientes no responden a la exéresis quirúrgica del bazo, que constituyen el grupo de pacientes con PTI crónica refractarios al tratamiento. No existen evidencias sobre cuál esquema terapéutico es el más efectivo en estos casos, por lo que el tratamiento continúa siendo empírico. En este trabajo se revisan las diferentes opciones terapéuticas que pueden ser utilizadas en la PTI crónica refractaria, el mecanismo de acción de las drogas, las dosis y los efectos adversos más frecuentes(AU)

The primary immune thrombocytopenia (PIT) is an acquired autoimmune disorder characterized by transient or persistent decreased platelet count with increased risk of bleeding. The chronic form of this disease primarily affects adults. Since spontaneous remissions are rare, patients usually are treated with steroids from the start. Approximately one third of the cases does not respond to steroid regimens; the second-line treatment is splenectomy, achieving the highest cure rates. However, between 10 to 30 percent of patients do not respond to spleen surgical removal, which is the group of patients with refractory chronic PIT. There is no evidence on whether this therapeutic regimen is most effective in these cases, so the treatment remains empirical. In this paper, various treatment options that can be used in refractory chronic PIT, the action mechanism of drugs, doses and more frequent adverse effects were reviewed(AU)

Trombocitopenia inmune primaria refractaria: opciones terapéuticas / Refractory primary immune thrombocytopenia: treatment options

La trombocitopenia inmune primaria (PTI) es un trastorno autoinmune adquirido caracterizado por una disminución transitoria o persistente del recuento de plaquetas con riesgo incrementado de sangramiento. La forma crónica de la enfermedad afecta fundamentalmente a los adultos. Puesto que las remisiones espontáneas son muy poco frecuentes, los pacientes son tratados desde el inicio, usualmente con esteroides. Aproximadamente un tercio de los casos no responden a los regímenes esteroideos y el tratamiento de segunda línea es la esplenectomía, tratamiento con el que se logran las mayores tasas de curación. Sin embargo, entre el 10 y el 30 por ciento de los pacientes no responden a la exéresis quirúrgica del bazo, que constituyen el grupo de pacientes con PTI crónica refractarios al tratamiento. No existen evidencias sobre cuál esquema terapéutico es el más efectivo en estos casos, por lo que el tratamiento continúa siendo empírico. En este trabajo se revisan las diferentes opciones terapéuticas que pueden ser utilizadas en la PTI crónica refractaria, el mecanismo de acción de las drogas, las dosis y los efectos adversos más frecuentes

The primary immune thrombocytopenia (PIT) is an acquired autoimmune disorder characterized by transient or persistent decreased platelet count with increased risk of bleeding. The chronic form of this disease primarily affects adults. Since spontaneous remissions are rare, patients usually are treated with steroids from the start. Approximately one third of the cases does not respond to steroid regimens; the second-line treatment is splenectomy, achieving the highest cure rates. However, between 10 to 30 percent of patients do not respond to spleen surgical removal, which is the group of patients with refractory chronic PIT. There is no evidence on whether this therapeutic regimen is most effective in these cases, so the treatment remains empirical. In this paper, various treatment options that can be used in refractory chronic PIT, the action mechanism of drugs, doses and more frequent adverse effects were reviewed