It is not clear if platelet responses are sustained after thrombopoietin receptor agonist (ar-TPO) withdrawal in paediatric patients. A multicentre retrospective observational study was performed in children with chronic immune thrombopenia (cITP) to describe ar-TPO tapering and withdrawal in patients who had achieved a sustained complete response to ar-TPOs. Ten patients (eltrombopag n = 6, romiplostim n = 4) were included. Treatment withdrawal was performed after a mean tapering time of 7.6 months. Two patients relapsed (median follow-up time of 24 months). Slow tapering and withdrawal of ar-TPOs can be safely performed in cITP paediatric patients after achieving a sustained complete response.
Hematologic Agents , Purpura, Thrombocytopenic, Idiopathic , Receptors, Thrombopoietin , Benzoates/therapeutic use , Blood Platelets , Child , Hematologic Agents/therapeutic use , Humans , Hydrazines/therapeutic use , Platelet Count , Purpura, Thrombocytopenic, Idiopathic/drug therapy , Pyrazoles/therapeutic use , Receptors, Fc/therapeutic use , Receptors, Thrombopoietin/agonists , Recombinant Fusion Proteins/therapeutic use , Thrombopoietin/therapeutic use
La trombocitopenia inmune primaria, anteriormente conocida como púrpura trombocitopénica inmune, es una enfermedad cuyo manejo diagnóstico y terapéutico ha sido siempre controvertido. La Sociedad Española de Hematología y Oncología Pediátricas, a través del grupo de trabajo de la PTI, ha actualizado el documento con las recomendaciones protocolizadas para el diagnóstico y tratamiento de esta enfermedad, basándose en las guías clínicas disponibles actualmente, revisiones bibliográficas, ensayos clínicos y el consenso de sus miembros. El objetivo principal es disminuir la variabilidad clínica en los procedimientos diagnósticos y terapéuticos con el fin de obtener los mejores resultados clínicos, los mínimos efectos adversos y preservar la calidad de vida
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
Humans , Child , Purpura, Thrombocytopenic, Idiopathic/therapy , Quality of Life , Purpura, Thrombocytopenic, Idiopathic/diagnosis
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.
Purpura, Thrombocytopenic, Idiopathic/therapy , Quality of Life , Child , Humans , Purpura, Thrombocytopenic, Idiopathic/diagnosis
El documento de consenso sobre el diagnóstico, tratamiento y seguimiento de la trombocitopenia inmune primaria fue elaborado en 2010 por especialistas con reconocida experiencia en esta enfermedad bajo el auspicio de la Sociedad Española de Hematología y Hemoterapia y la Sociedad Española de Hematología y Oncología Pediátricas, con el fin de adaptar a España las recomendaciones de los documentos de consenso internacional recientemente publicados. La decisión de iniciar tratamiento se basa en las manifestaciones hemorrágicas y en la cifra de plaquetas (<20×109/L). El tratamiento de primera línea son los glucocorticoides, aunque durante un plazo limitado de 4-6 semanas, reservándose la adición de inmunoglobulinas intravenosas para pacientes con hemorragia grave. La esplenectomía es el tratamiento de segunda línea más eficaz. Para los pacientes refractarios a la esplenectomía y para aquellos con contraindicación o rechazo, los nuevos agentes trombopoyéticos son los fármacos de elección por su eficacia y excelente perfil de seguridad. El resto de las opciones terapéuticas presentan tasa de respuesta y duración muy variables, y carecen de estudios controlados que permitan establecer recomendaciones claras. El seguimiento debe individualizarse, aunque, como mínimo, en pacientes sin tratamiento activo, se recomienda un hemograma cada 3-6 meses, y programas de educación al paciente para que consulte en caso de hemorragia, cirugía o procedimiento invasor y gestación. En una considerable proporción de la población pediátrica la enfermedad tiene tendencia a la remisión espontánea. Los glucocorticoides a altas dosis en pauta corta y las inmunoglobulinas intravenosas son el tratamiento de elección. Los tratamientos de segunda línea y los posteriores deben controlarse en centros especializados
The consensus document on the diagnosis, treatment and monitoring of primary immune thrombocytopenia was developed in 2010 by specialists with recognized expertise in this disease under the auspices of the Spanish Society of Hematology and Hemotherapy and the Spanish Society of Pediatric Hematology and Oncology, with the aim to adapt to Spain the recommendations of the recently published international consensus documents. The decision to start treatment is based on bleeding manifestations and platelet count (<20×109/L). The first-line treatment is corticosteroids, albeit for a limited period of 4-6 weeks. The addition of intravenous immunoglobulin is reserved to patients with severe bleeding. Splenectomy is the most effective second-line treatment. For patients refractory to splenectomy and those with contraindications or patient refusal, the new thrombopoietic agents are the drugs of choice due to their efficacy and excellent safety profile. The other treatment options have highly variable response rates, and the absence of controlled studies does not allow to establish clear recommendations. Monitoring should be individualized. In patients without active treatment, blood counts are recommended every 3-6 months, and the patient should be instructed to consult in case of bleeding, surgery or invasive procedure and pregnancy. In most of the pediatric population, the disease tends to spontaneous remission. High-dose corticosteroids in short course and intravenous immunoglobulin are the treatment of choice. Second- and further-line treatments should be monitored in specialized centers (AU)
Humans , Thrombocytopenia/diagnosis , Thrombocythemia, Essential/diagnosis , Thrombocytopenia/therapy , Splenectomy , Glucocorticoids/therapeutic use , Immunoglobulins/administration & dosage , Thrombopoiesis
The consensus document on the diagnosis, treatment and monitoring of primary immune thrombocytopenia was developed in 2010 by specialists with recognized expertise in this disease under the auspices of the Spanish Society of Hematology and Hemotherapy and the Spanish Society of Pediatric Hematology and Oncology, with the aim to adapt to Spain the recommendations of the recently published international consensus documents. The decision to start treatment is based on bleeding manifestations and platelet count (<20×10(9)/L). The first-line treatment is corticosteroids, albeit for a limited period of 4-6 weeks. The addition of intravenous immunoglobulin is reserved to patients with severe bleeding. Splenectomy is the most effective second-line treatment. For patients refractory to splenectomy and those with contraindications or patient refusal, the new thrombopoietic agents are the drugs of choice due to their efficacy and excellent safety profile. The other treatment options have highly variable response rates, and the absence of controlled studies does not allow to establish clear recommendations. Monitoring should be individualized. In patients without active treatment, blood counts are recommended every 3-6 months, and the patient should be instructed to consult in case of bleeding, surgery or invasive procedure and pregnancy. In most of the pediatric population, the disease tends to spontaneous remission. High-dose corticosteroids in short course and intravenous immunoglobulin are the treatment of choice. Second- and further-line treatments should be monitored in specialized centers.
Purpura, Thrombocytopenic, Idiopathic , Aged , Benzoates/therapeutic use , Child , Female , Glucocorticoids/therapeutic use , Humans , Hydrazines/therapeutic use , Immunoglobulins, Intravenous/therapeutic use , Immunologic Factors/therapeutic use , Pregnancy , Pregnancy Complications, Hematologic/diagnosis , Pregnancy Complications, Hematologic/therapy , Purpura, Thrombocytopenic, Idiopathic/diagnosis , Purpura, Thrombocytopenic, Idiopathic/therapy , Pyrazoles/therapeutic use , Quality of Life , Receptors, Fc/therapeutic use , Receptors, Thrombopoietin/agonists , Recombinant Fusion Proteins/therapeutic use , Splenectomy , Thrombopoietin/therapeutic use
