Primary and secondary immune thrombocytopenia (ITP): Time for a rethink

González-López, Tomás José; Bárez, Abelardo; Bernardo-Gutiérrez, Angel; Bernat, Silvia; Martínez‐Carballeira, Daniel; Jarque-Ramos, I.; Soto, Inmaculada; Jiménez-Bárcenas, Reyes; Fernández‐Fuertes, Fernando

doi:10.1016/j.blre.2023.101112

Cited by 10 publications

(3 citation statements)

References 125 publications

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“…The same axis imbalance occurs during chronic thromboembolic pulmonary hypertension, as recently reported by Newnham and colleagues [15]. ADAMTS13 deficiency could be caused by acquired autoantibody-mediated functional inhibition (iTTP) [16] or by more than 200 inherited mutations within the ADAMTS13 locus, causing a congenital disease (cTTP) also known as Upshaw-Schulman syndrome [17,18]. A cTTP diagnosis must be confirmed by genetic analyses to identify ADAMTS13 mutations in homozygous or heterozygous states [19].…”

Section: Introductionmentioning

confidence: 76%

A Long-Term Follow-Up Study in Immune-Mediated Thrombotic Thrombocytopenic Purpura: What Are the Outcomes?

Bonifacio,

Roselli,

Schifone

et al. 2023

JCM

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Endothelium damage triggers the multimeric protein von Willebrand factor (VWF) release and subsequent binding to platelets, which are recruited at sites of vascular injury. A complex and fragile equilibrium between circulating levels of von Willebrand factor and its metalloprotease, ADAMTS13, is responsible for the hemostatic balance. However, the presence of autoantibodies targeting ADAMTS13 results in an increase in von Willebrand factor, mainly in its ultra-large multimers. The latter lead to platelet aggregation, the formation of thrombi and microangiopathic hemolytic anemia. This pathologic condition, known as immune-mediated thrombotic thrombocytopenic purpura (iTTP), occurs with high morbidity and a high rate of relapses. In this work, the long-term follow-up of 40 patients with iTTP is reported. We assessed ADAMTS13 activity, plasmatic VWF levels and the ADAMTS13/VWF ratio, comparing iTTP relapsing patients with remitting ones. A decrease in the ADAMTS13/VWF ratio, along with a reduced ADAMTS13 activity, could serve as predictive and sensitive biomarkers of incoming relapses.

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Section: Introductionmentioning

confidence: 76%

A Long-Term Follow-Up Study in Immune-Mediated Thrombotic Thrombocytopenic Purpura: What Are the Outcomes?

Bonifacio,

Roselli,

Schifone

et al. 2023

JCM

View full text Add to dashboard Cite

show abstract

“…The last decade has seen an exponential increase in therapeutic options for rare hematologic diseases. The latter encompass benign conditions, including congenital anemias, autoimmune cytopenias, bone marrow failure syndromes (i.e., aplastic anemia and paroxysmal nocturnal hemoglobinuria), and rare hemostatic disorders, as well as neoplastic ones (i.e., low-risk myelodysplastic syndromes and systemic mastocytosis) [1][2][3][4][5][6]. On the whole, these disorders often represent a diagnostic challenge given their rarity, but also due to their heterogeneous clinical phenotype.…”

mentioning

confidence: 99%

“…for many years, representing a true unmet clinical need. Novel treatment, including oral small molecules, intravenous or subcutaneous monoclonal antibodies, small interfering RNAs, gene therapy, and many others have the potential to change the natural history of these disorders [1][2][3][4][5][6]. In this Special Issue of Pharmaceuticals, experts in rare congenital and acquired hematologic disorders have addressed the above-mentioned topics, giving evidence-based and personal insights on this exciting evolving scenario.…”

mentioning

confidence: 99%