Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Avatrombopag.
- Who it may be relevant to
- Registry conditions: Platelet Recovery After Umbilical Cord Blood Transplantation. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Efficacy and Safety of Avatrombopag for Platelet Recovery After Unrelated Cord Blood Transplantation in Patients With Bone Marrow Failure Disease: a Single-center, Single-arm, Phase II Trial
Overview
To evaluate the efficacy and safety of Avatrombopag for platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease
Detailed description
Persistent thrombocytopenia is a common complication after umbilical cord blood transplantation (UCBT), which necessitates platelet transfusion and leads to increased transplant-related mortality. This is a single-center, single-arm, phase II clinical trial aimed at evaluating the efficacy and safety of Avatrombopag for platelet recovery after UCBT in patients with bone marrow failure disease (BMFD). The study was conducted in patients diagnosed with BFMD and receiving UCBT. After the subjects signed and informed and qualified, they received Avatrombopag treatment from the first day to the 28th day after transplantation. In this study, the cumulative of platelet implantation rate at 28 days after transplantation was used as the main therapeutic index, and 40 patients were planned to be enrolled.
Interventions
- Drug Avatrombopag
Indications for drug reduction and withdrawal: excluding blood transfusion factors, the reduction will be 20mg/d when PLT≥50×10\^9/L; Drug administration will be stopped when PLT≥100×10\^9/L.
Primary outcome measures
- Time of platelet engraftment [Time frame: 28 days]
- The cumulative incidence of platelet engraftment at 28 days after transplantation [Time frame: 28 days]
Secondary outcome measures (7)
- The cumulative incidence of platelet recovery rate at 28 days after transplantation [Time frame: 28 days]
- Total platelet transfusion during 4 weeks of treatment [Time frame: 28 days]
- neutrophil reconstitution [Time frame: 28 days]
- erythroid reconstitution [Time frame: 28 days]
- Megakaryocyte levels in bone marrow smears 4 weeks after treatment [Time frame: 4 weeks]
- Time of platelet recovery [Time frame: 100 days]
- Time of platelet ≥100×10^9/L. [Time frame: 100 days]
Eligibility criteria
Inclusion criteria
- Age ≥12 years old, male or female;
- Patients diagnosed with bone marrow failure disease including aplastic anemia (AA), Fanconi anemia (FA), paroxysmal nocturnal hemoglobinuria (PNH) and undergoing UCBT;
- ECOG score ≤2;
- Voluntary participation in this clinical trial, patients fully understand the trial content and sign the informed consent；
Exclusion criteria
- Pregnant or lactating women；
- Known allergy to Avatrombopag;
- A history of severe thrombotic events or known risk factors for thrombosis or active thromboembolism requiring anticoagulation;
- A history of platelet dysfunction or bleeding prone disease or severe bleeding (requiring more than 2 units of red blood cell infusion or a hematocrit drop of ≥10%) within 7 days prior to screening;
- Chronic active hepatitis B and C;
- Repeat or multiple transplantation or multiple organ transplantation;
- HIV positive, EBV-DNA positive, CMV-DNA positive;
- Have severe infection or complicated with serious heart, liver, lung, kidney, neurological or metabolic diseases;
- A serious adverse event (Level 4 or 5) as defined by the Standard General Terminology for Adverse Events Version 4.0 occurred during the pre-treatment period;
- Participants participate in another clinical study with any exploratory drug or device within 30 days prior to baseline visit; Observational studies are allowed;
- Subjects with cognitive impairment or uncontrolled mental illness;
- Subjects and/or authorized family members to refuse treatment with Avatrombopag;
- Those who are judged by the researchers as not suitable for inclusion (such as accompanying medical history, which may affect the safety of the subjects or it is estimated that the treatment cannot be adhered to due to financial problems).
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
China · 1 center
- The First Affiliated Hospital of University of Science and Technology of China (Anhui Prov — Hefei
Identifiers
NCT: NCT05823376 · AVA&MDS-001