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Anales de Pediatria Aug 2019Primary immune thrombocytopenia, formerly known as immune thrombocytopenic purpura, is a disease for which the clinical and therapeutic management has always been...
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.
Topics: Child; Humans; Purpura, Thrombocytopenic, Idiopathic; Quality of Life
PubMed: 31178291
DOI: 10.1016/j.anpedi.2019.04.014 -
Blood Aug 2022Immune thrombocytopenia (ITP) is the most common acquired thrombocytopenia in children and is caused by immune-mediated decreased platelet production and increased...
Immune thrombocytopenia (ITP) is the most common acquired thrombocytopenia in children and is caused by immune-mediated decreased platelet production and increased platelet destruction. In the absence of a diagnostic test, ITP must be differentiated from other thrombocytopenic disorders, including inherited platelet disorders. In addition, a diagnosis of secondary ITP due to a primary immune deficiency with immune dysregulation may not be apparent at diagnosis but can alter management and should be considered in an expanding number of clinical scenarios. The diagnostic evaluation of children with thrombocytopenia will vary based on the clinical history and laboratory features. Access to genotyping has broadened the ability to specify the etiology of thrombocytopenia, whereas increasing access to immunophenotyping, functional immunologic and platelet assays, and biochemical markers has allowed for more in-depth evaluation of patients. With this greater availability of testing, diagnostic algorithms in patients with thrombocytopenia have become complex. In this article, we highlight the diagnostic evaluation of thrombocytopenia in children with a focus on ITP, including consideration of underlying genetic and immune disorders, and use hypothetical patient cases to describe disease manifestations and strategies for treatment of pediatric ITP.
Topics: Biomarkers; Blood Platelets; Child; Humans; Leukopenia; Neuroblastoma; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
PubMed: 34479363
DOI: 10.1182/blood.2020006480 -
British Journal of Haematology Oct 2023A subset of individuals with 'primary' or 'idiopathic' immune thrombocytopenia (ITP) who fail to respond to conventional first- and second-line agents or who lose... (Review)
Review
A subset of individuals with 'primary' or 'idiopathic' immune thrombocytopenia (ITP) who fail to respond to conventional first- and second-line agents or who lose responsiveness are considered to have 'refractory' disease (rITP), placing them at increased risk of bleeding and complications of intensive treatment. However, the criteria used to define the refractory state vary among studies, which complicates research and clinical investigation. Moreover, it is unclear whether rITP is simply 'more severe' ITP, or if there are specific pathogenic pathways that are more likely to result in refractory disease, and whether the presence or development of rITP can be established or anticipated based on these differences. This paper reviews potential biological features that may be associated with rITP, including genetic and epigenetic risk factors, dysregulation of T cells and cytokine networks, antibody affinity and specificity, activation of complement, impaired platelet production and alterations in platelet viability and clearance. These findings indicate the need for longitudinal studies using novel clinically available methodologies to identify and monitor pathogenic T cells, platelet antibodies and other clues to the development of refractory disease.
Topics: Humans; Purpura, Thrombocytopenic, Idiopathic; Antibodies; Antibody Affinity; Blood Platelets; Cytokines; Thrombocytopenia
PubMed: 37735546
DOI: 10.1111/bjh.19083 -
Journal of Thrombosis and Haemostasis :... Mar 2023The von Willebrand factor-directed nanobody caplacizumab has greatly changed the treatment of immune thrombotic thrombocytopenic purpura (iTTP) in recent years. Data...
BACKGROUND
The von Willebrand factor-directed nanobody caplacizumab has greatly changed the treatment of immune thrombotic thrombocytopenic purpura (iTTP) in recent years. Data from randomized controlled trials established efficacy and safety.
OBJECTIVES
This study aims to address open questions regarding patient selection, tailoring of therapy duration, obstacles in prescribing caplacizumab in iTTP, effect on adjunct treatment, and outcomes in the real-world setting.
METHODS
We report retrospective, observational cohorts of 113 iTTP episodes treated with caplacizumab and 119 historical control episodes treated without caplacizumab. We aggregated data from the caplacizumab phase II/III trials and real-world data from France, the United Kingdom, Germany, and Austria (846 episodes, 396 treated with caplacizumab, and 450 historical controls).
RESULTS
Caplacizumab was efficacious in iTTP, independent of the timing of therapy initiation, but curtailed the time of active iTTP only when used in the first-line therapy within 72 hours after diagnosis and until at least partial ADAMTS13-activity remission. Aggregated data from multiple study populations showed that caplacizumab use resulted in significant absolute risk reduction of 2.87% for iTTP-related mortality (number needed to treat 35) and a relative risk reduction of 59%.
CONCLUSION
Caplacizumab should be used in first line and until ADAMTS13-remission, lowers iTTP-related mortality and refractoriness, and decreases the number of daily plasma exchange and hospital stay. This trial is registered at www.
CLINICALTRIALS
gov as #NCT04985318.
Topics: Humans; Purpura, Thrombotic Thrombocytopenic; Retrospective Studies; Single-Domain Antibodies; Purpura, Thrombocytopenic, Idiopathic; Treatment Outcome; Thrombosis; ADAMTS13 Protein
PubMed: 36696206
DOI: 10.1016/j.jtha.2022.11.010 -
Postgraduate Medical Journal Feb 2022
Topics: Humans; Purpura Fulminans; Purpura
PubMed: 37066566
DOI: 10.1136/postgradmedj-2021-140510 -
British Journal of Haematology Apr 2024Immune thrombocytopenia (ITP) in pregnancy is challenging for both mother and fetus. Understanding the pathophysiology, treatments, and risks to the mother and fetus... (Review)
Review
Immune thrombocytopenia (ITP) in pregnancy is challenging for both mother and fetus. Understanding the pathophysiology, treatments, and risks to the mother and fetus leads to proper management resulting in successful pregnancy and delivery in almost all cases. ITP in a pregnant woman has many similarities to ITP not in pregnancy although gestational thrombocytopenia can be confused with ITP. However, recognizing differences is instrumental in avoiding bleeding complications and toxicities of treatment. This Nutshell review focuses on the natural history of ITP in pregnancy, its treatment, and dilemmas.
Topics: Pregnancy; Female; Humans; Purpura, Thrombocytopenic, Idiopathic; Platelet Count; Pregnancy Complications, Hematologic; Thrombocytopenia
PubMed: 38263610
DOI: 10.1111/bjh.19230 -
American Family Physician Mar 2024
Topics: Humans; Purpura; IgA Vasculitis; Skin Neoplasms
PubMed: 38574218
DOI: No ID Found -
Cutis Jan 2024
Topics: Humans; Factitious Disorders; Psychotic Disorders; Autoimmune Diseases; Purpura; Skin Diseases, Vascular
PubMed: 38478944
DOI: 10.12788/cutis.0957 -
Blood Dec 2022
Topics: Humans; Qi; Purpura, Thrombocytopenic, Idiopathic; Decitabine; Myeloid-Derived Suppressor Cells; Thrombocytopenia
PubMed: 36580341
DOI: 10.1182/blood.2022018373 -
Hematology. American Society of... Dec 2023Thrombocytopenia in ill children is common; accurately diagnosing the underlying etiology is challenging and essential for appropriate management. Triggers for... (Review)
Review
Thrombocytopenia in ill children is common; accurately diagnosing the underlying etiology is challenging and essential for appropriate management. Triggers for accelerated consumption of platelets are numerous; common downstream mechanisms of clearance include platelet trapping in microvascular thrombi, phagocytosis, and platelet activation. Thrombocytopenia with microangiopathic hemolytic anemia (MAHA) is frequently due to disseminated intravascular coagulation. Thrombotic microangiopathy (TMA) is a subgroup of MAHA. Specific TMA syndromes include thrombotic thrombocytopenic purpura, complement-mediated TMA (CM-TMA), and Shiga toxin-mediated hemolytic uremic syndrome. Isolated thrombocytopenia is characteristic of immune thrombocytopenia; however, concomitant cytopenias are frequent in critically ill patients, making the diagnosis difficult. Immune thrombocytopenia with large vessel thrombosis is a feature of heparin-induced thrombocytopenia and antiphospholipid antibody syndrome. In addition, thrombocytopenia is common with macrophage activation, which is characteristic of hemophagocytic lymphohistiocytosis. While thrombocytopenia in ill patients can be driven by hypoproliferative processes such as myelosuppression and/or bone marrow failure, this review will focus on consumptive thrombocytopenia due to immune and nonimmune causes.
Topics: Child; Humans; Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombotic Thrombocytopenic; Hemolytic-Uremic Syndrome; Thrombotic Microangiopathies; Anemia, Hemolytic; Thrombosis
PubMed: 38066886
DOI: 10.1182/hematology.2023000465