Recent progress in ITP treatment

Rodeghiero, Francesco

doi:10.1007/s12185-022-03527-1

Cited by 7 publications

(5 citation statements)

References 62 publications

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“…Avatrombopag is another TPO-RA registered for refractory ITP treatment [48]. It is a small molecule, similar to eltrombopag and is administered initially as one 20 mg pill daily [49].…”

Section: Discussionmentioning

confidence: 99%

Thrombopoietin receptor agonist and rituximab combination therapy in patients with refractory primary immune thrombocytopenia

Witkowski,

Ryżewska,

Robak

2024

Blood Coagulation &Amp; Fibrinolysis

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The aim of our study was to evaluate the efficacy of this therapy in patients with refractory primary immune thrombocytopenia. It is crucial to develop alternative treatment methods for this patient group in order to achieve better response. This combination therapy combines two different mechanisms of action, which is promising in terms of targeting pathophysiology of immune thrombocytopenia. We conducted a retrospective study, which included all patients who were diagnosed with refractory primary immune thrombocytopenia and received TPO-RA and rituximab at the General Hematology Department, Copernicus Memorial Hospital in Lodz, Poland. We assessed the response, time to response and treatment-free remission (TFR). After 1 month of treatment, the complete response (CR1, PLT >100 g/l) was achieved in 62.5% patients, and response (R1, PLT >30 g/l) was achieved in 62.5% patients. The median PLT was 175 × 109/l. Within 1 month of treatment, 87.5% of patients achieved TFR. Adequately, after 6 months, CR6 and R6 was 62.5 and 75%. The median PLT was 182 × 109/l. Treatment-free remission 6 months after completion was in 50% of patients. The study group achieved response to treatment, which suggests that combination of TPO-RA and rituximab is effective and relatively well tolerated. Prospective study on larger group of patients is needed to better evaluate the efficiency and safety of this treatment.

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“…Avatrombopag is another TPO-RA registered for refractory ITP treatment [48]. It is a small molecule, similar to eltrombopag and is administered initially as one 20 mg pill daily [49].…”

Section: Discussionmentioning

confidence: 99%

Thrombopoietin receptor agonist and rituximab combination therapy in patients with refractory primary immune thrombocytopenia

Witkowski,

Ryżewska,

Robak

2024

Blood Coagulation &Amp; Fibrinolysis

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“…ITP antibodies may also induce desialylation of megakaryocytes, impairing platelet production 73 . Experimental 74 and anecdotal clinical studies 75 support a salutary effect of neuraminidase inhibition on platelet counts. Desialylation of platelet glycoproteins has been reported in ITP, 76 and a few patients with rITP responded when oseltamivir was added to their therapy 76 .…”

Section: Impaired Platelet Production and Non‐immune Causes Of Accele...mentioning

confidence: 99%

Pathogenesis of refractory ITP: Overview

Cines

2023

Br J Haematol

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SummaryA 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.

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“…76 It was first approved in Japan by Pharmaceuticals and Medical Devices Agency (PMDA) for the treatment of thrombocytopenia associated with CLD in adult patients. 77 In 2018, the substance was also approved for this indication by FDA and EMA. In single patients, portal vein thrombosis has been reported as serious AE associated with lusutrombopag.…”

Section: Schlüsselwörtermentioning

confidence: 99%

Update on the Use of Thrombopoietin-Receptor Agonists in Pediatrics

Gebetsberger,

Streif,

Dame

2024

Hamostaseologie

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This review summarizes the rationale and current data on the use of thrombopoietin receptor agonists (TPO-RAs) for treating severe thrombocytopenia in infants, children, and adolescents. It focuses on substances that have been approved by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for pediatric patients. Romiplostim and eltrombopag are already established as second-line treatment for persistent or chronic immune thrombocytopenia (ITP). As in adults, TPO-RAs are currently also evaluated in severe aplastic anemia (SAA), chemotherapy-induced thrombocytopenia (CIT), myelodysplastic syndromes (MDS), and poor engraftment after hematopoietic stem cell transplantation in pediatric and adolescent patients. Moreover, studies on the implication of TPO-RA in treating rare inherited thrombocytopenias, such as Wiskott-Aldrich syndrome (WAS), congenital amegakaryocytic thrombocytopenia (CAMT), or MYH9-associated thrombocytopenia, deserve future attention. Current developments include testing of avatrombopag and lusutrombopag that are approved for the treatment of thrombocytopenia associated with chronic liver disease (CLD) in adult patients. In pediatric and adolescent medicine, we expect in the near future a broader use of TPO-RAs as first-line treatment in primary ITP, thereby considering immunomodulatory effects that increase the rate of sustained remission off-treatment, and a selective use in rare inherited thrombocytopenias based on current clinical trials.

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Recent progress in ITP treatment

Cited by 7 publications

References 62 publications

Thrombopoietin receptor agonist and rituximab combination therapy in patients with refractory primary immune thrombocytopenia

Thrombopoietin receptor agonist and rituximab combination therapy in patients with refractory primary immune thrombocytopenia

Pathogenesis of refractory ITP: Overview

Update on the Use of Thrombopoietin-Receptor Agonists in Pediatrics

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