Ignite Creation Date:
2024-06-16 @ 11:51 AM
Last Modification Date:
2024-10-26 @ 3:32 PM
Study NCT ID:
NCT06458803
Status:
COMPLETED
Last Update Posted:
2024-06-17
First Post:
2024-06-04
Brief Title:
Efficacy and Safety of Eltrombopag for Refractory Thrombocytopenia Associated With Connective Tissue Disease
Sponsor:
Tongji Hospital
Organization:
Tongji Hospital
Study Overview
Official Title:
Efficacy and Safety of Eltrombopag for the Treatment of Refractory Thrombocytopenia Associated With Connective Tissue Disease
Status:
COMPLETED
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Immune thrombocytopenia ITP is a haemorrhagic disorder often associated with CTD Corticosteroids are the first-line treatment for CTD-associated thrombocytopenia but not all patients respond well Eltrombopag is an oral small molecule thrombopoietin receptor agonist It interacts with the transmembrane domain of the thrombopoietin receptor and stimulates platelet production This study is designed to evaluate the efficacy and safety of eltrombopag in patients with refractory CTD-ITP It is a single-centre retrospective observational study involving a cohort of 52 patients diagnosed with CTD-RITP who received eltrombopag between 2013 and 2023 Follow-up data will be systematically collected and analysed to evaluate the therapeutic efficacy and safety of the drug The study will provide valuable insight into the benefit of eltrombopag in CTD-RITP by reviewing baseline characteristics and performing subsequent clinical assessments to determine drug response and adverse events
Detailed Description:
Immune thrombocytopenia ITP is a hemorrhagic disorder typically attributed to the formation of autoantibodies against platelet antigens Patients experience decreased platelet count clinically presenting as purpura petechiae mucosal bleeding and increased menstrual bleeding ITP is often associated with connective tissue diseases CTD with reports indicating a concurrent presence of ITP in 10-15 of systemic lupus erythematosus SLE patients and 78 of primary Sjögrens syndrome SS patients
Currently corticosteroids are the first-line therapy for CTD-associated thrombocytopenia with second-line options including immunosuppressive agents intravenous immunoglobulin IVIG splenectomy and rituximab However not all patients respond favorably to these treatments Patients with CTD who are unresponsive or have a low response to conventional first- and second-line therapies with platelet counts below 30109L are considered to have CTD-related refractory ITP RITP There are currently no internationally unified diagnostic criteria for RITP For adult RITP diagnosis criteria proposed by George et al include being diagnosed with ITP under the premise that treatment with glucocorticoids andor splenectomy is ineffective age 18 years ③ duration of illness 6 months ④ absence of other conditions causing thrombocytopenia ⑤ platelet count 30109L
Eltrombopag is an oral small-molecule non-peptide thrombopoietin receptor agonist that interacts with the transmembrane domain of the thrombopoietin receptor stimulating platelet production and increasing platelet counts To evaluate the efficacy and safety of eltrombopag in CTD-RITP this study will conduct a single-center retrospective observational analysis of 52 patients with CTD-RITP who received eltrombopag treatment between 2013 and 2023 recording their follow-up information Patient characteristics at baseline will be analyzed and drug efficacy and safety will be assessed through follow-up examinations at different time points
During treatment patients may receive other ITP medications for maintenance therapy such as glucocorticoids azathioprine danazol cyclosporine A and mycophenolate mofetil excluding those concurrently using other TPO receptor agonists Patient follow-up examinations will be continuously recorded for at least six months with monthly records and patients followed until the last follow-up time if less than six months Univariate analysis descriptive analysis or non-parametric tests single and multiple logistic regression analysis and multiple correspondence analysis MCA will be used to analyze early clinical predictive factors for drug response Patient drug responsiveness will be judged based on laboratory test results and clinical symptoms ① Complete remission CR PLT 100109L with no bleeding tendency ② Partial remission PR PLT 50109L but 100109L with no bleeding tendency or platelet count at least twice that of pre-treatment ③ No remission NR Does not meet criteria ① or ② Mann-Kendall test will be used to analyze trends in PLT changes among patients during treatment and the rates of CR PR and NR at different time points 4 8 12 24 weeks will be calculated to determine drug onset time and remission degree The incidence of major adverse reactions to eltrombopag hepatotoxicity thrombosis will be statistically analyzed at 24 weeks to assess drug safety
This project aims to evaluate the efficacy and safety of eltrombopag in treating CTD-RITP patients providing evidence for formulating treatment plans for CTD-RITP patients It aims to guide physicians in considering the use of eltrombopag when selecting treatment methods understanding individual differences in patient response to eltrombopag efficacy and helping physicians develop more personalized treatment plans based on early clinical predictive factors to improve treatment targeting and effectiveness Furthermore it aims to observe the long-term effects of eltrombopag treatment and explore the optimal dosage course of treatment and best combination therapy with other drugs
Currently corticosteroids are the first-line therapy for CTD-associated thrombocytopenia with second-line options including immunosuppressive agents intravenous immunoglobulin IVIG splenectomy and rituximab However not all patients respond favorably to these treatments Patients with CTD who are unresponsive or have a low response to conventional first- and second-line therapies with platelet counts below 30109L are considered to have CTD-related refractory ITP RITP There are currently no internationally unified diagnostic criteria for RITP For adult RITP diagnosis criteria proposed by George et al include being diagnosed with ITP under the premise that treatment with glucocorticoids andor splenectomy is ineffective age 18 years ③ duration of illness 6 months ④ absence of other conditions causing thrombocytopenia ⑤ platelet count 30109L
Eltrombopag is an oral small-molecule non-peptide thrombopoietin receptor agonist that interacts with the transmembrane domain of the thrombopoietin receptor stimulating platelet production and increasing platelet counts To evaluate the efficacy and safety of eltrombopag in CTD-RITP this study will conduct a single-center retrospective observational analysis of 52 patients with CTD-RITP who received eltrombopag treatment between 2013 and 2023 recording their follow-up information Patient characteristics at baseline will be analyzed and drug efficacy and safety will be assessed through follow-up examinations at different time points
During treatment patients may receive other ITP medications for maintenance therapy such as glucocorticoids azathioprine danazol cyclosporine A and mycophenolate mofetil excluding those concurrently using other TPO receptor agonists Patient follow-up examinations will be continuously recorded for at least six months with monthly records and patients followed until the last follow-up time if less than six months Univariate analysis descriptive analysis or non-parametric tests single and multiple logistic regression analysis and multiple correspondence analysis MCA will be used to analyze early clinical predictive factors for drug response Patient drug responsiveness will be judged based on laboratory test results and clinical symptoms ① Complete remission CR PLT 100109L with no bleeding tendency ② Partial remission PR PLT 50109L but 100109L with no bleeding tendency or platelet count at least twice that of pre-treatment ③ No remission NR Does not meet criteria ① or ② Mann-Kendall test will be used to analyze trends in PLT changes among patients during treatment and the rates of CR PR and NR at different time points 4 8 12 24 weeks will be calculated to determine drug onset time and remission degree The incidence of major adverse reactions to eltrombopag hepatotoxicity thrombosis will be statistically analyzed at 24 weeks to assess drug safety
This project aims to evaluate the efficacy and safety of eltrombopag in treating CTD-RITP patients providing evidence for formulating treatment plans for CTD-RITP patients It aims to guide physicians in considering the use of eltrombopag when selecting treatment methods understanding individual differences in patient response to eltrombopag efficacy and helping physicians develop more personalized treatment plans based on early clinical predictive factors to improve treatment targeting and effectiveness Furthermore it aims to observe the long-term effects of eltrombopag treatment and explore the optimal dosage course of treatment and best combination therapy with other drugs
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None