Current therapeutic strategies and perspectives in refractory ITP: What have we learned recently?

Lv, Yao; Shi, Huiping; Liu, Hong; Zhou, Lu

doi:10.3389/fimmu.2022.953716

Cited by 8 publications

(6 citation statements)

References 123 publications

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“…The treatment response was divided into complete response (CR), response (R) and no response (NR) [19] . The overall response rate (OR) was the total of patients who have achieved CR and R. NR was de ned as platelet count < 30×10 9 /L, or less than a twofold increase in the baseline count, or bleeding events when the patient had received an appropriate dose of eltrombopag for 8 weeks [20] .…”

Section: E Cacy and Adverse Effects Analysismentioning

confidence: 99%

The long-term efficacy of eltrombopag in children with immune thrombocytopenia

Yang,

Sang,

Yang

et al. 2024

Preprint

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Immune thrombocytopenia (ITP) is the most common autoimmune disorder characterized by decreased platelet counts and impaired platelet production. Eltrombopag has been demonstrated to be safe and effective for children with ITP. It is reported eltrombopag can achieve a sustained response off treatment. However, data on its overall efficacy and safety profile are scarce in children. This study aimed to investigate the long-term efficacy of eltrombopag in children with ITP. Treatment overall response (OR), complete response (CR), response (R), durable response (DR), no response (NR), treatment free remission (TFR), and relapse rate, were assessed in 103 children with ITP during eltrombopag therapy. The OR rate, CR rate, R rate, DR rate, NR rate, TFR, and relapse rate were 67.0%, 55.3%, 11.7%, 56.3%, 33.0%, 60%, 36.2%, respectively. Importantly, we discovered that newly diagnosed ITP patients showed a higher DR rate, TFR rate and lower relapse rate compared to persistent and chronic ITP patients. Furthermore, the CR rate, DR rate, and TFR rate of 5 patients under six months were 100%. None of them suffered relapse. The most common adverse event (AEs) was hepatotoxicity (7.77%). Our study highlighted the critical role of eltrombopag as the second-line treatment in children with ITP who were intolerant to first-line therapy.

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Section: E Cacy and Adverse Effects Analysismentioning

confidence: 99%

The long-term efficacy of eltrombopag in children with immune thrombocytopenia

Yang,

Sang,

Yang

et al. 2024

Preprint

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“…In addition, very little is known about the immunopathophysiology of refractory ITP or whether it may be different or altered which leads to refractoriness. Currently, there is no consensus on the definition of refractory ITP, nor evidence‐based treatment guidelines for these patients and this has become one of the biggest challenges in ITP management 4,5 …”

Section: General Aspects Of Drug Refractoriness In Immune Thrombocyto...mentioning

confidence: 99%

“…Currently, there is no consensus on the definition of refractory ITP, nor evidence-based treatment guidelines for these patients and this has become one of the biggest challenges in ITP management. 4,5 One of the challenges in managing ITP is predicting which patients will respond to treatment and which will not, and several potential diagnostic approaches have been proposed for the prediction of therapeutic responses in ITP. 6 For example, Jolink et al 6 have proposed investigations into the…”

mentioning

confidence: 99%

The role of T cells and myeloid‐derived suppressor cells in refractory immune thrombocytopenia

Yazdanbakhsh,

Provan,

Semple

2023

Br J Haematol

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SummaryImmune 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.

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“…The treatment of patients, who developed refractory ITP is challenging. There is an ongoing discussion regarding various management approaches of refractory ITP, including implementation of combination regimens [16]. A durable response can be achieved in patients who are administered TPO-RA with immunosuppression [17].…”

Section: Introductionmentioning

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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Current therapeutic strategies and perspectives in refractory ITP: What have we learned recently?

Cited by 8 publications

References 123 publications

The long-term efficacy of eltrombopag in children with immune thrombocytopenia

The long-term efficacy of eltrombopag in children with immune thrombocytopenia

The role of T cells and myeloid‐derived suppressor cells in refractory immune thrombocytopenia

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

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