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American Journal of Therapeutics
Topics: Humans; Piperacillin; Purpura, Thrombocytopenic, Idiopathic; Anti-Bacterial Agents; Thrombocytopenia
PubMed: 37713713
DOI: 10.1097/MJT.0000000000001521 -
British Journal of Haematology Oct 2023Rituximab and thrombopoietin receptor agonists (TPO-RAs) have profoundly changed the management of immune thrombocytopenia (ITP) over the last 20 years. Even if most... (Review)
Review
Rituximab and thrombopoietin receptor agonists (TPO-RAs) have profoundly changed the management of immune thrombocytopenia (ITP) over the last 20 years. Even if most current guidelines put splenectomy, rituximab and TPO-RAs on the same treatment level, most clinicians and patients clearly prefer to postpone splenectomy and to multiply the lines of medical treatment before considering surgery. The management of ITP refractory to rituximab and TPO-RAs is challenging. Splenectomy is currently performed much less frequently because of a better knowledge of its complications, particularly severe late infections and deep vein thrombosis, and the inability to reliably predict its effectiveness. Furthermore, there is a reluctance to propose splenectomy when other treatments have been ineffective, based on the not well-documented risk that splenectomy could not be effective in such a case. The objective of this update was to review the most recent published data on the long-term tolerability and side effects of splenectomy and the predictors of response and efficacy, especially for patients exposed to multiple medical lines. This update can help physicians and patients with failure of multiple lines of therapy make an informed decision on the indication for splenectomy with the help of up-to-date data.
Topics: Humans; Adult; Purpura, Thrombocytopenic, Idiopathic; Rituximab; Splenectomy; Emotions; Physicians; Thrombocytopenia
PubMed: 37735555
DOI: 10.1111/bjh.19077 -
Critical Reviews in Clinical Laboratory... Dec 2023Thrombotic thrombocytopenic purpura (TTP) is a rare and potentially fatal disease for which rapid diagnosis is crucial for patient outcomes. Deficient activity (< 10%)... (Review)
Review
Thrombotic thrombocytopenic purpura (TTP) is a rare and potentially fatal disease for which rapid diagnosis is crucial for patient outcomes. Deficient activity (< 10%) of the liver enzyme, ADAMTS13, is the pathophysiological hallmark of TTP, and measurement of the enzyme activity can establish the diagnosis of TTP with high accuracy. Thus, along with the clinical history, appropriate laboratory assessment of a suspected case of TTP is essential for diagnosis and treatment. Here, we present a review of the available laboratory tests that can assist clinicians in establishing the diagnosis of TTP, with special focus on ADAMTS13 assays, including the measurement of the antigen and activity, and detection of autoantibodies to ADAMTS13.
Topics: Humans; Purpura, Thrombotic Thrombocytopenic; ADAM Proteins; Autoantibodies
PubMed: 37452521
DOI: 10.1080/10408363.2023.2232039 -
Pediatric Nephrology (Berlin, Germany) Nov 2023IgA vasculitis is the most common vasculitis in children and is often complicated by acute nephritis (IgAVN). Risk of chronic kidney disease (CKD) among children with... (Observational Study)
Observational Study
BACKGROUND
IgA vasculitis is the most common vasculitis in children and is often complicated by acute nephritis (IgAVN). Risk of chronic kidney disease (CKD) among children with IgAVN remains unknown. This study aimed to describe the clinical management and kidney outcomes in a large cohort of children with IgAVN.
METHODS
This observational cohort study used the PEDSnet database to identify children diagnosed with IgAV between January 1, 2009, and February 29, 2020. Demographic and clinical characteristics were compared among children with and without kidney involvement. For children followed by nephrology, clinical course, and management patterns were described. Patients were divided into four categories based on treatment: observation, renin-angiotensin-aldosterone system (RAAS) blockade, corticosteroids, and other immunosuppression, and outcomes were compared among these groups.
RESULTS
A total of 6802 children had a diagnosis of IgAV, of whom 1139 (16.7%) were followed by nephrology for at least 2 visits over a median follow-up period of 1.7 years [0.4,4.2]. Conservative management was the most predominant practice pattern, consisting of observation in 57% and RAAS blockade in 6%. Steroid monotherapy was used in 29% and other immunosuppression regimens in 8%. Children receiving immunosuppression had higher rates of proteinuria and hypertension compared to those managed with observation (p < 0.001). At the end of follow-up, 2.6 and 0.5% developed CKD and kidney failure, respectively.
CONCLUSIONS
Kidney outcomes over a limited follow-up period were favorable in a large cohort of children with IgAV. Immunosuppressive medications were used in those with more severe presentations and may have contributed to improved outcomes. A higher resolution version of the Graphical abstract is available as Supplementary information.
Topics: Humans; Child; IgA Vasculitis; Immunoglobulin A; Nephritis; Renal Insufficiency, Chronic; Disease Progression
PubMed: 37316676
DOI: 10.1007/s00467-023-06023-8 -
British Journal of Haematology Oct 2023Immune thrombocytopenia (ITP) is characterized by a dysregulated immune response against platelets, affecting both their destruction and production. A role for an... (Review)
Review
Immune thrombocytopenia (ITP) is characterized by a dysregulated immune response against platelets, affecting both their destruction and production. A role for an abnormal T-cell compartment has been established in ITP pathogenesis and treatments that increase platelet counts in patients with ITP have shown improvements in T-cell profiles. On the other hand, patients who were refractory to treatment appear to retain the T-cell abnormalities as before. Myeloid-derived suppressive cells (MDSCs) are also emerging as key contributors to the immune pathology of ITP and response to treatment. In this review, we will discuss how various treatments affect the T-cell and MDSC compartments in ITP. The review will focus on studies that have examined the underlying mechanisms and/or genetic basis responsible for refractoriness to a given treatment and highlight remaining challenges in identifying factors and mechanisms to predict response to treatment.
Topics: Humans; Myeloid-Derived Suppressor Cells; Purpura, Thrombocytopenic, Idiopathic; T-Lymphocytes; Thrombocytopenia; Myeloid Cells
PubMed: 37735552
DOI: 10.1111/bjh.19079 -
British Journal of Haematology Oct 2023It is known that patients with immune thrombocytopenia (ITP) have fatigue and impairment of health-related quality of life (HRQoL). However, it is hypothesized that... (Review)
Review
It is known that patients with immune thrombocytopenia (ITP) have fatigue and impairment of health-related quality of life (HRQoL). However, it is hypothesized that patients with refractory ITP have additional burdens that should be considered. Specifically, fatigue is more pronounced in patients with refractory disease, there are additional side effects from second- and third-line treatments, additional anxiety about the long-term course of the disease, impairment in HRQoL resulting from heavy menstrual bleeding and concerns related to family planning. The burden of disease, therefore, should be carefully assessed and considered in these patients. However, researchers have utilized numerous tools for evaluating HRQoL and fatigue, making comparison of data across studies challenging. There is a need to standardize assessment using either disease-specific or generic instruments that can be easily implemented in routine clinical practice. Additionally, whether treatment of low platelet count and bleeding symptoms will have a positive influence on HRQoL remains to be seen and published evidence is conflicting. Nevertheless, improvement of HRQoL is a major treatment goal for both patients and physicians and should be especially considered when treating patients with refractory ITP.
Topics: Humans; Purpura, Thrombocytopenic, Idiopathic; Quality of Life; Thrombocytopenia; Anxiety; Fatigue
PubMed: 37735553
DOI: 10.1111/bjh.19068 -
Blood May 2024
Topics: Humans; ADAMTS13 Protein; Purpura, Thrombotic Thrombocytopenic; Female; Single-Domain Antibodies; Male; Adult; Middle Aged
PubMed: 38696192
DOI: 10.1182/blood.2024023862 -
Internal Medicine Journal Feb 2024
Topics: Humans; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
PubMed: 37975334
DOI: 10.1111/imj.16246 -
Blood Mar 2024
Topics: Humans; Purpura Fulminans; Complement System Proteins; Receptors, Complement
PubMed: 38483408
DOI: 10.1182/blood.2023023334 -
Immunological Medicine Dec 2023Immune thrombocytopenia (ITP) is a thrombocytopenic condition induced by autoimmune mechanisms and includes secondary ITP with underlying diseases such as connective... (Review)
Review
Immune thrombocytopenia (ITP) is a thrombocytopenic condition induced by autoimmune mechanisms and includes secondary ITP with underlying diseases such as connective tissue diseases (CTD). In recent years, it has been elucidated that the subsets of the ITP are associated with complement abnormalities but much remains unclear. To perform a literature review and identify the characteristics of complement abnormalities in ITP. PUBMED was used to collect the literature published up to June 2022 related to ITP and complement abnormalities. Primary and secondary ITP (CTD-related) were examined. Out of the collected articles, 17 were extracted. Eight articles were related to primary ITP (pITP) and 9 to CTD-related ITP. Analysis of the literature revealed that the ITP severity was inversely correlated with serum C3, C4 levels in both ITP subgroups. In pITP, a wide range of complement abnormalities was reported, including abnormalities of initial proteins, complement regulatory proteins, or the end products. In CTD-related ITP, reported complement abnormalities were limited to the initial proteins. Activation of the early complement system, mainly through activation of C3 and its precursor protein C4, was reported for both ITPs. On the other hand, more extensive complement activation has been reported in pITP.
Topics: Humans; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia; Complement System Proteins; Complement Activation; Hereditary Complement Deficiency Diseases
PubMed: 37237432
DOI: 10.1080/25785826.2023.2213976