Investigational drugs for immune thrombocytopenia

Newland, Adrian C.

doi:10.1080/13543784.2022.2075340

Cited by 5 publications

(6 citation statements)

References 99 publications

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“…A phase III trial NCT05029635 is in progress to further assess the safety and efficacy of HMPL-523 in patients with refractory ITP. It can be used as an alternative agent to fostamatinib for SYK inhibition [ 27 , 28 ]. The drug is not yet approved by FDA and is not available in the market for ITP.…”

Section: Discussionmentioning

confidence: 99%

Safety and Efficacy of Tyrosine Kinase Inhibitors in Immune Thrombocytopenic Purpura: A Systematic Review of Clinical Trials

Ali¹,

Anwar²,

Aiman

et al. 2023

JoX

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Immune thrombocytopenic purpura (ITP) is an acquired antibody or cell-mediated platelet damage or decreased platelet production. Steroids, IV immunoglobulins (IVIG), and Rho-anti-D antibodies are the commonly used initial treatments for ITP. However, many ITP patients either do not respond or do not maintain a response to initial therapy. Splenectomy, rituximab, and thrombomimetics are the commonly used second-line treatment. More treatment options include tyrosine kinases inhibitors (TKI), including spleen tyrosine kinase (Syk) and Bruton’s tyrosine kinase (BTK) inhibitors. This review aims to assess the safety and efficacy of TKIs. Methods: Literature was searched on PubMed, Embase, WOS, and clinicaltrials.gov using keywords, “tyrosine kinase” and “idiopathic thrombocytopenic purpura”. PRISMA guidelines were followed. Results: In total, 4 clinical trials were included with 255 adult patients with relapsed/refractory ITP. In all, 101 (39.6%) patients were treated with fostamatinib, 60 (23%) patients with rilzabrutinib, and 34 (13%) with HMPL-523. Patients treated with fostamatinib achieved a stable response (SR) and overall response (OR) in 18/101 (17.8%) and 43/101 (42.5%) of the patients, respectively, while SR and OR were achieved in 1/49 (2%) and 7/49 (14%) of the patients, respectively, in the placebo group. Patients treated with HMPL-523 (300 mg dose expansion) achieved an SR and OR in 5/20 (25%) and 11/20 (55%) of the patients, respectively, while SR and OR were achieved in 1/11 (9%) of the patients treated with the placebo. Patients treated with rilzabrutinib achieved an SR in 17/60 (28%) patients. Dizziness (1%), hypertension (2%), diarrhea (1%), and neutropenia (1%) were serious adverse events in fostamatinib patients. Rilzabrutinib or HMPL-523 patients did not require a dose reduction due to drug-related adverse effects. Conclusions: Rilzabrutinib, fostamatinib, and HMPL-523 were safe and effective in the treatment of relapsed/refractory ITP.

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Section: Discussionmentioning

confidence: 99%

Safety and Efficacy of Tyrosine Kinase Inhibitors in Immune Thrombocytopenic Purpura: A Systematic Review of Clinical Trials

Ali¹,

Anwar²,

Aiman

et al. 2023

JoX

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“…However, factors that have been reported to be associated with a chronic course in ITP include an impaired bone marrow microenvironment, female gender, older age at presentation, gradual initiation of disease, frequent bleeding, immunological markers and higher platelets at baseline [14]. The pathogenic role of T-cells in ITP has also been investigated, including a potential focus on new therapies [15]. Intracranial haemorrhage is a rare complication of ITP in children, with 53% of the reported incidents occurring during the chronic phase.…”

Section: Discussionmentioning

confidence: 99%

Immune Thrombocytopenia in Children with Neurofibromatosis Type 1

2023

Arch Pediatr

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Introduction:Neurofibromatosis type 1 (NF1) is the most common neurocutaneous syndrome. Primary immune thrombocytopenia (ITP) is a haematological autoimmune disorder that is the most common cause of thrombocytopenia in children. Autoimmune diseases have been reported in association with NF1, either by coincidence or predisposition. The aim of the study was to present our experience with children with NF1 and ITP who were treated in an NF1 clinic and haematology unit of a tertiary paediatric referral hospital. Methods: The clinical database of the NF1 multidisciplinary clinic of Schneider Children's Medical Centre of Israel was reviewed for all patients age 0-18 years diagnosed and followed with NF1 and ITP during 2017-2022 We present the children and discuss our knowledge regarding the link between NF1 and ITP, the natural history of ITP in individuals with NF1, and possible causes of this association. Results: During the study, 290 children with NF1 and younger than 18 years were referred to our NF1 clinic 1% (3/290) of the patients were also diagnosed with ITP. Two of our three patients developed chronic ITP and required frequent therapy; one of the two had intracranial bleeding. Conclusion: Our study suggest that the incidence of ITP is increased in NF1with the possibility of persistent ITP and more severe bleeding complications The relation between NF1 and autoimmune phenomena still needs to be elucidated.

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“…In addition, several drugs that will soon provide us with new treatment tools are currently in the clinical trial phase with clearly encouraging results. Therefore, it is likely that ITP treatment in the coming years will be quite different from today and that our view will change in a short period [28]. Who knows?…”

Section: Clinical Management Of Itpmentioning

confidence: 99%

“…There are currently new drugs in clinical trials (efgartigimod, rilzabrutinib, sutimlimab and others) that should be approved for use in ITP in the next few years. As mentioned above, it is likely that data from these clinical trials will soon change our view of ITP treatment [28].…”

Section: New Classes Of Treatment Are In the Pipelinementioning

confidence: 99%

Current Concepts in the Diagnosis and Management of Adult Primary Immune Thrombocytopenia: Our Personal View

González-López

Newland

2023

Medicina

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Primary immune thrombocytopenia (ITP) is an acquired blood disorder that causes a reduction in circulating platelets with the potential for bleeding. The incidence of ITP is slightly higher in adults and affects more women than men until 60 years, when males are more affected. Despite advances in basic science, primary ITP remains a diagnosis of exclusion. The disease is heterogeneous in its clinical behavior and response to treatment. This reflects the complex underlying pathophysiology, which remains ill-understood. Platelet destruction plays a role in thrombocytopenia, but underproduction is also a major contributing factor. Active ITP is a proinflammatory autoimmune disease involving abnormalities within the T and B regulatory cell compartments, along with several other immunological abnormalities. Over the last several years, there has been a shift from using immunosuppressive therapies for ITP towards approved treatments, such as thrombopoietin receptor agonists. The recent COVID-19 pandemic has hastened this management shift, with thrombopoietin receptor agonists becoming the predominant second-line treatment. A greater understanding of the underlying mechanisms has led to the development of several targeted therapies, some of which have been approved, with others still undergoing clinical development. Here we outline our view of the disease, including our opinion about the major diagnostic and therapeutic challenges. We also discuss our management of adult ITP and our placement of the various available therapies.

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Investigational drugs for immune thrombocytopenia

Cited by 5 publications

References 99 publications

Safety and Efficacy of Tyrosine Kinase Inhibitors in Immune Thrombocytopenic Purpura: A Systematic Review of Clinical Trials

Safety and Efficacy of Tyrosine Kinase Inhibitors in Immune Thrombocytopenic Purpura: A Systematic Review of Clinical Trials

Immune Thrombocytopenia in Children with Neurofibromatosis Type 1

Current Concepts in the Diagnosis and Management of Adult Primary Immune Thrombocytopenia: Our Personal View

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