Clinical trial NCT05311930: Efficacy and Safety of TPO Receptor Agonists in the Treatment of Elderly ITP Patients
Official title: Efficacy and Safety of TPO Receptor Agonists as First-line Drugs in the Treatment of Newly Diagnosed Elderly ITP Patients in China-an Open Label Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Primary Immune Thrombocytopenia
Keywords: hetrombopag
Primary Immune Thrombocytopenia
platelets
Lead sponsor: Wuhan Union Hospital, China
Sponsor class: OTHER
Collaborators: name: Jiangsu Hengrui Pharmaceutical Co., Ltd.
First posted: 2022-04-05
Enrollment: 69
Sex: ALL
Minimum age (years): 60
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This is a single-arm open-label trial. After enrollment, patients received an initial dose of 2.5 mg of hetropoda for 4 weeks, and blood routine monitoring was performed regularly during treatment.
Primary purpose: OTHER

Brief summary: Elderly ITP patients have many underlying diseases, hormone contraindications and many adverse reactions during the use of hormones. TPO-RAs are oral small-molecule non-peptide drugs. Retrospective studies have shown that they have good efficacy and high safety in elderly patients. Therefore, this study is a prospective trial to evaluate TPO-RAs as the first-choice drug for the treatment of elderly ITP patients with contraindications to hormones, aiming to improve the efficacy-risk ratio of elderly patients

Detailed description: Primary immune thrombocytopenia (ITP) is an immune disorder characterized by decreased production and increased destruction of platelets. In recent years, with the in-depth exploration of its pathogenesis and the continuous influx of new drugs, the status of second-line treatment drugs, mainly TPO receptor agonists (TPO-RAs), has been continuously improved, which has brought great importance to the treatment and management of ITP patients. However, for elderly ITP patients with severe underlying diseases, poor hormone tolerance, severe adverse reactions or hormone contraindications, whether TPO receptor agonists can be used as the first-choice drug and its efficacy and safety are still lacking relevant research, and for elderly ITP patients There is a lack of uniform guidelines for the treatment and management of patients. Limited retrospective studies have shown that TPO receptor agonists have good safety and efficacy, and are expected to become the recommended drugs for the treatment of elderly patients with ITP. However, whether TPO receptor agonists can be directly used as the first-choice drug for newly diagnosed elderly ITP patients who are not suitable for first-line treatment and its efficacy are uncertain.

This study will include newly diagnosed elderly ITP patients with hormonal contraindications or potential serious side effects of hormonal therapy, take the TPO-RA drug hetropoda as the first-choice treatment drug, and explore the effectiveness of hetrompopag in such patients and safety analysis. This study will provide new ideas and clinical basis for standardized and individualized treatment of elderly ITP patients, and provide practical experience for promoting the establishment of elderly ITP treatment guidelines.

Eligibility criteria: Inclusion Criteria:

* 1. The patient voluntarily signed the informed consent; 2. The patient is a newly diagnosed ITP patient, aged ≥60 years old; 3. Two consecutive PLTs \< 30×109/L, or two consecutive PLTs \< 30×109/L≤PLT\<50×109/L but with risk factors such as bleeding (such as previous bleeding history and/or anticoagulation/antiplatelet) Concomitant medication) or age \> 75 years; 4. Have not received first-line treatment such as hormones, IVIG, etc.; 5. There is any hormonal contraindication (with active peptic ulcer, recent gastrointestinal anastomosis, corneal ulcer, severe hypertension (high blood pressure ≥ grade 2), diabetes with poor blood sugar control, infection that cannot be controlled by antibiotics ( Bacterial and viral infections), heart failure and adrenal hyperfunction, severe mental illnesses such as epilepsy, severe osteoporosis, rheumatoid arthritis, tuberculosis, fractures, patients with combined antithrombotic and antiplatelet drugs, etc.); 6. The following clinical biochemical indicators must be within ±20% of the upper and lower limits of normal values: creatinine, alanine aminotransferase, aspartate aminotransferase, total bilirubin and alkaline phosphatase.

Exclusion Criteria:

* 1. Exclude immune diseases such as systemic lupus erythematosus, antiphospholipid syndrome, etc.; 2. Exclude drug-related thrombocytopenia; 3. Bone marrow-related examinations suggest the presence of other primary diseases of the blood system (such as MDS, AA, thrombotic thrombocytopenic purpura, etc.) or the presence of myelofibrosis MF≥2; 4. Participated in other clinical trials affecting platelet count and function 3 months before the trial; 5. Previously received first-line therapy such as hormones, IVIG; 6. Previous use of TPO-RAs or poor efficacy against known TPO drugs; 7. The patient has experienced severe arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism), or clinical symptoms suggest thrombophilia; 8. HIV, hepatitis B or C seropositive or a history of liver cirrhosis or portal hypertension; 9. Life-threatening bleeding (WHO bleeding score 4) or the patient is expected to require salvage treatment before the first dose; 10. Has a history of malignant tumor or is accompanied by malignant tumor; 11. The investigator believes that there are any other circumstances that may cause the subjects to fail to complete the study or bring obvious risks to the subjects.

Primary outcomes:
measure: Treatment response
timeFrame: 28 days
Secondary outcomes:
measure: Remission rate
timeFrame: 28 days
measure: Drug efficacy
timeFrame: 28 days
measure: Evaluation of effectiveness
timeFrame: 28 days
measure: Adverse events
timeFrame: 6 months from treatment
measure: Adverse events
timeFrame: 6 months from treatment
measure: Side effects of drugs
timeFrame: 1 month from treatment
Other outcomes:
Locations:
Study officials:
affiliation: Wuhan Union Hospital, China
name: Heng Mei, PhD
role: PRINCIPAL_INVESTIGATOR
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