Hematopoietic Stem Cell Transplantation, Thrombocytopenia
Conditions
Keywords
etamsylate, Hematopoietic Stem Cell Transplantation recipients, thrombocytopenia, bleeding events
Brief summary
This study employs a prospective, randomized, double-blind, placebo-controlled design. It aims to compare the efficacy of etamsylate versus placebo in preventing bleeding complications in patients with thrombocytopenia following hematopoietic stem cell transplantation.
Detailed description
This study employs a prospective, randomized, double-blind, placebo-controlled design. It aims to compare the efficacy of etamsylate versus placebo in preventing bleeding complications in patients with thrombocytopenia following hematopoietic stem cell transplantation. The primary endpoint is the incidence of death or bleeding events (WHO grade ≥2) within 30 days. Secondary endpoints focus on the safety profile of etamsylate versus placebo.
Interventions
DRUGetamsylate
etamsylate Injection, administer by intravenous infusion, diluted with 100ml of 0.9% Sodium Chloride Injection, twice daily.
DRUGPlacebo
0.9% Sodium Chloride Injection, 100 ml, twice daily, intravenous drip
Sponsors
First Affiliated Hospital of Zhejiang University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Age between 18 and 70 years (inclusive), regardless of gender; 2. Patients diagnosed with a hematological disease requiring hematopoietic stem cell transplantation; 3. Expected platelet count ≤ 10 x 10⁹/L persisting for 5 days or more; 4. Normal coagulation function; 5. Adequate organ function: Aspartate Aminotransferase (AST) and Alanine Aminotransferase (ALT) ≤ 3 times the Upper Limit of Normal (ULN), Total Bilirubin ≤ 2 x ULN; Serum Creatinine ≤ 2 x ULN; Creatinine Clearance ≥ 50 mL/min (calculated using the Cockcroft-Gault formula); Left Ventricular Ejection Fraction (LVEF) ≥ 50% as measured by Echocardiography (ECHO); 6. Voluntary provision of signed informed consent, with the ability to understand and comply with all study requirements.
Exclusion criteria
1. Diagnosis of acute promyelocytic leukemia confirmed according to WHO diagnostic criteria; 2. Pregnant or breastfeeding women, and women of childbearing potential unwilling to use effective contraception; 3. Presence of active bleeding or infection; 4. History of diagnosed primary immune thrombocytopenia or hemolytic uremic syndrome; 5. Patients with known hereditary or acquired hemorrhagic disorders; 6. Patients receiving anticoagulant or antiplatelet therapy; 7. Patients with severe cardiac insufficiency (left ventricular ejection fraction \[EF\] \< 60%); or severe arrhythmias: history of clinically significant QTc prolongation (male \> 450 ms; female \> 470 ms), ventricular tachycardia, atrial fibrillation, second-degree heart block; myocardial infarction within 1 year prior to enrollment; or symptomatic coronary artery disease requiring medication; patients with severe liver impairment (liver function indices \[ALT, TBIL\] \> 3 times the upper limit of normal \[ULN\]); 8. Patients with severe pulmonary insufficiency (obstructive and/or restrictive ventilatory defects); 9. Patients with severe renal insufficiency (renal function index \[Cr\] \> 2 times ULN); or 24-hour urinary creatinine clearance rate (Ccr) \< 50 ml/min; 10. Patients with mental disorders or other conditions preventing provision of informed consent and compliance with study treatment and procedural requirements; 11. Other reasons deemed by the investigator to make the patient ineligible for inclusion.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| WHO grade 2 or higher bleeding events or death. | 30 days | The incidence of WHO grade 2 or higher bleeding events or death within 30 days of medication administration. Etamsylate will be considered effective compared to placebo when the incidence in the experimental group is 14.2% lower than that in the placebo group. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The number of days without WHO grade 2 or higher bleeding events | 30 days | The number of days without WHO grade 2 or higher bleeding events within 30 days of medication administration. Etamsylate will be considered effective compared to placebo when the number of days without WHO grade 2 or higher bleeding events is 2 days more in the experimental group than in the placebo group. |
| The number of platelet transfusions | The number of platelet transfusions within 30 days of medication administration. Etamsylate will be considered effective compared to placebo when the number of platelet transfusions in the experimental group is 2.5 fewer than in the placebo group. | — |
Countries
China
Contacts
CONTACTYi Luo
CONTACTLuxin Yang
PRINCIPAL_INVESTIGATORYi Luo
The First Affiliated Hospital, College of Medicine, Zhejiang University Principal Investigator
Outcome results
None listed