Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia
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Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: cytokine essays, NGS, Fecal microbiome, Erythropoietin.
- Кому может быть актуально
- Состояния в реестре: Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, Chronic Idiopathic Neutropenia, Autoimmune Neutropenia. Базовые параметры: от 18 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Италия
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
Evaluation of the Diagnostic/Therapeutic Course of Patients With Autoimmune Cytopenias (Autoimmune Hemolytic Anemia AIHA, Immune Thrombocytopenia ITP, Chronic Idiopathic/Autoimmune Neutropenia CIN/AIN) and Identification of Predictive and Prognostic Markers.
Обзор
The goal of this observational study is to characterize the diagnostic and therapeutic management of autoimmune cytopenias including autoimmune hemolytic anemia, immune thrombocytopenia, and chronic idiopathic/autoimmune neutropenia. The main aims to answer are: * evaluation of traditional and novel diagnostic tools including immunohematology, cytokine essays, bone marrow studies, molecular findings, and fecal microbiome. * evaluation of type and sequence of the therapies administered, the response rates, and the adverse events. * evaluation of clinical and laboratory (immunologic, molecular, and morphologic) predictors of outcome. * evolution of autoimmune cytopenias into myelodysplastic syndromes. * a subgroup of patients with myelodysplastic syndromes will be included to evaluate the presence of immunologic events, autoimmune activation, and red cell metabolism. Participants will receive a clinical/laboratory diagnostic workup as per current clinical practice. Furthermore They will be sampled at baseline (peripheral blood and feces for microbiome) and followed up for at least 3 years to evaluate their clinical course, therapeutic management and outcome.
Подробное описание
This observational study will characterize the diagnostic and therapeutic management of autoimmune cytopenias including autoimmune hemolytic anemia, immune thrombocytopenia, and chronic idiopathic/autoimmune neutropenia to evaluate predictors of outcome. Additionally, a subgroup of patients with myelodysplastic syndromes (diagnosed according to current WHO 5th edition 2022) will be included to evaluate the presence of autoimmune activation, and red cell metabolism.
Вмешательства
- Биопрепарат cytokine essays
evaluation of immunomodulatory cytokines by ELISA kits on peripheral blood samples - Биопрепарат NGS
evaluation of somatic mutations commonly associated with myeloid neoplasm and immunodeficiencies by next generation sequencing on peripheral blood samples - Биопрепарат Fecal microbiome
evaluation of fecal microbiome on fecal samples - Препарат Erythropoietin
evaluation of recombinant erythropoietin use, safety and efficacy in patients with autoimmune hemolytic anemia according to clinical practice - Препарат Luspatercept
evaluation of cytokine levels, molecular profile and bone marrow microenvironment by single cell analysis in patients treated with luspatercept according to clinical practice - Препарат Thrombopoietin Receptor Agonist
evaluation of TPO-RA use, safety and efficacy in patients with ITP according to clinical practice - Препарат G-CSF
evaluation of G-CSF use, safety and efficacy in patients with CIN/AIN according to clinical practice
Первичные конечные точки
- sensitivity of autoantibody testing in autoimmune cytopenias [Срок оценки: 2021-2026]
- specificity of autoantibody testing in autoimmune cytopenias [Срок оценки: 2021-2026]
- sensitivity of bone marrow trephine in autoimmune cytopenias [Срок оценки: 2021-2026]
Вторичные конечные точки (6)
- overall response rate [Срок оценки: 2021-2026]
- Evaluation of somatic mutations [Срок оценки: 2021-2026]
- Evaluation of pyruvate kinase activity [Срок оценки: 2021-2026]
- Evaluation of microbiome [Срок оценки: 2021-2026]
- Single cell RNA expression [Срок оценки: 2021-2026]
- Evaluation of cytokine levels [Срок оценки: 2021-2026]
Критерии участия
Критерии включения
- Diagnosis of autoimmune cytopenias (AIHA/ITP/CIN/AIN)
- age >/= 18 years
- ability to sign informed consent
- availability to undergo 3 year follow up
- for the subgroup of patients with myelodysplastic syndrome: bone marrow evaluation showing >/= 10% dysplastic features of at least one lineage along with MDS defining cytopenia and/or MDS defining cytogenetics.
Критерии исключения
- any condition impeding the acquisition of the informed consent
- immune cytopenia diagnosis preceding >/= 6 months the enrolment
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Когортное
Центры проведения
Италия · 1 центр
- Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico — Milan
Публикации
- Fattizzo B, Pedone GL, Brambilla C, Pettine L, Zaninoni A, Passamonti F, Barcellini W. Recombinant erythropoietin in autoimmune hemolytic anemia with inadequate bone marrow response: a prospective analysis. Blood Adv. 2024 Mar 12;8(5):1322-1327. doi: 10.1182/bloodadvances.2023011798. PMID 38029356
- Versino F, Revelli N, Villa S, Pettine L, Zaninoni A, Prati D, Passamonti F, Barcellini W, Fattizzo B. Transfusions in autoimmune hemolytic anemias: Frequency and clinical significance of alloimmunization. J Intern Med. 2024 Mar;295(3):369-374. doi: 10.1111/joim.13753. Epub 2023 Nov 27. PMID 38013593
Идентификаторы
NCT: NCT05931718 · CYTOPAN