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British Journal of Haematology Oct 2023Immune thrombocytopenia (ITP) is an autoimmune haematological disorder characterized by immune-mediated thrombocytopenia and a variable risk of bleeding. Despite the... (Review)
Review
Immune thrombocytopenia (ITP) is an autoimmune haematological disorder characterized by immune-mediated thrombocytopenia and a variable risk of bleeding. Despite the availability of multiple treatment options, some patients are considered refractory since they do not achieve a platelet count response to multiple treatments and are at risk of bleeding. The term 'refractory' has been used to identify this patient group; however, with the advent of multiple lines of treatment, its meaning has become ambiguous. To address this issue, we reviewed previous definitions of refractory ITP, solicited the views of ITP experts and collected data from registries to inform a definition. Twenty ITP experts who attended the 7th Expert Meeting of the Intercontinental Cooperative ITP Study Group in September 2022 answered a web-based survey: 95% felt that there was a need for a new definition of refractory ITP for clinical and research purposes. The use of the term refractory, accompanied by a clear indication of the type and timing of failed treatments, was supported by 85% of respondents. Preliminary data on the frequency of refractory patients from the McMaster and Norwegian ITP Registries demonstrated that the proportion of adult ITP patients who had failed first-line therapy, rituximab, thrombopoietin receptor agonists, any immune suppressant medication and splenectomy ranged from 0.4% to 3.8%. We propose a definition of refractory ITP that could be evaluated in future studies.
Topics: Humans; Adult; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia; Platelet Count; Emotions; Immunosuppressive Agents
PubMed: 37642211
DOI: 10.1111/bjh.19075 -
Methods in Molecular Biology (Clifton,... 2023Accurate estimation of ADAMTS13 (a disintegrin-like and metalloprotease with thrombospondin type 1 motif, member 13) activity level is necessary for diagnosis and...
Accurate estimation of ADAMTS13 (a disintegrin-like and metalloprotease with thrombospondin type 1 motif, member 13) activity level is necessary for diagnosis and management of thrombotic microangiopathies (TMA). In particular, it permits distinction between thrombotic thrombocytopenic purpura (TTP) and other TMAs, prompting disorder appropriate treatment. Manual and automated quantitative assays of ADAMTS13 activity are commercially available, some providing results within less than an hour, but they require specialist equipment and personnel and tend to only be available in specialized diagnostic facilities. Technoscreen ADAMTS13 Activity is a rapid, commercially available, semiquantitative screening test employing flow-through technology and an ELISA activity assay principle. It is a simple to perform screening tool, not requiring specialist equipment or personnel. The colored end point is compared to a reference color chart containing four color intensity indicators corresponding to ADAMTS13 activity levels of 0, 0.1, 0.4, or 0.8 IU/mL. Reduced levels detected in the screening test should be confirmed by quantitative assay. The assay lends itself to use in nonspecialized laboratories, remote, and point-of-care settings.
Topics: Humans; ADAM Proteins; Purpura, Thrombotic Thrombocytopenic; Enzyme-Linked Immunosorbent Assay; Diagnosis, Differential; ADAMTS13 Protein
PubMed: 37204734
DOI: 10.1007/978-1-0716-3175-1_34 -
Haematologica Feb 2023
Topics: Humans; Purpura, Thrombotic Thrombocytopenic
PubMed: 35488359
DOI: 10.3324/haematol.2022.281095 -
Tidsskrift For Den Norske Laegeforening... Apr 2024
Topics: Humans; Purpura; Male
PubMed: 38651721
DOI: 10.4045/tidsskr.23.0604 -
The Journal of Pediatrics Feb 2020
Topics: Child; Cough; Diagnosis, Differential; Early Diagnosis; Exanthema; Female; Humans; Orbit; Purpura
PubMed: 31685229
DOI: 10.1016/j.jpeds.2019.09.080 -
Postgraduate Medical Journal Aug 2020
Topics: Anti-Bacterial Agents; Biopsy; Debridement; Disseminated Intravascular Coagulation; Early Diagnosis; Female; Gangrene; Humans; Middle Aged; Purpura Fulminans; Skin Ulcer; Time-to-Treatment
PubMed: 31801806
DOI: 10.1136/postgradmedj-2019-137247 -
The Journal of Pediatrics Dec 2019
Topics: Child, Preschool; Exercise; Humans; Male; Orbital Diseases; Purpura
PubMed: 31472928
DOI: 10.1016/j.jpeds.2019.07.046 -
Journal of Thrombosis and Haemostasis :... Aug 2021Targeted therapy of immune thrombotic thrombocytopenic purpura (iTTP) requires acurate and prompt diagnosis and differentiation from complement-mediated hemolytic uremic... (Review)
Review
Targeted therapy of immune thrombotic thrombocytopenic purpura (iTTP) requires acurate and prompt diagnosis and differentiation from complement-mediated hemolytic uremic syndrome and other causes of thrombotic microangiopathy. ADAMTS-13 (A Disintegrin And Metalloprotease with ThromboSpondin-1 Domain, member 13) evaluation (activity and inhibitors or anti-ADAMTS-13 IgG) is the key for diagnosis and further management of patients with suspected iTTP during acute episode and in clinical response or remission. Clinical trial results and real-world data have demonstrated the efficacy and safety of the triple therapy consisting of therapeutic plasma exchange, caplacizumab, and immunosuppressives (e.g., corticosteroids and rituximab) for acute iTTP. Such a therapeutic strategy has significantly accelerated the normalization of platelet counts, decreased the length of stays in the intensive care unit and the hospital, but most importantly reduced the mortality rate. The present review highlights some of the important advancements for the diagnosis and management of iTTP and proposes triple therapy as the standard of care for acute iTTP today.
Topics: ADAMTS13 Protein; Humans; Plasma Exchange; Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombotic Thrombocytopenic; Standard of Care; Thrombospondin 1
PubMed: 34060225
DOI: 10.1111/jth.15406 -
La Revue de Medecine Interne Jan 2021Secondary forms of immune thrombocytopenia (ITP) represent approximately 20% of all ITP cases in adulthood and this rate increases with age. Since some causes may...
Secondary forms of immune thrombocytopenia (ITP) represent approximately 20% of all ITP cases in adulthood and this rate increases with age. Since some causes may influence both the prognosis and outcome but also the management of ITP, a minimal workup must be performed at ITP diagnosis to look for an associated or underlying cause. Among adults, B-cell lymphomas and mainly chronic lymphocytic leukemia, systemic auto-immune diseases such as systemic lupus or primary immunodeficiencies mainly represented by common variable immunodeficiency are the most frequent causes of secondary ITP. Whereas first-line therapy used for secondary ITP is usually similar to the one commonly used in primary ITP and relies mostly on corticosteroids±intravenous immunoglobulin according to the severity of bleeding, second and third-line treatments must take into account the type and degree of activity of the underlying disease.
Topics: Adrenal Cortex Hormones; Adult; Common Variable Immunodeficiency; Humans; Immunoglobulins, Intravenous; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
PubMed: 33139079
DOI: 10.1016/j.revmed.2020.08.004 -
La Revue de Medecine Interne Apr 2022
Topics: Humans; Leg; Purpura
PubMed: 35307224
DOI: 10.1016/j.revmed.2022.03.001