Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia

Institute of Hematology & Blood Diseases Hospital, China

Status and phase: Enrolling; Phase 2

Conditions: Sever Aplastic Anaemia; Elderly (People Aged 65 or More); Immunosuppressive Treatment

Treatments: Drug: Dose-attenuated IST and Hetrombopag

Study type: Interventional

Funder types: Other

Identifiers

NCT07010237

IIT2025013

Details and patient eligibility

About

This is a prospetive,Single-Center, Single-Arm, Phase IIB Clinical Study.This study aims to evaluate the efficacy and safety of dose-attenuated IST combined with Hetrombopag in elderly patients (≥65 years) with VSAA/SAA.

Full description

Design : Prospective, single-center, single-arm, open-label Phase IIB trial.

Intervention :

P-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag : 15 mg daily. Follow-Up : Weekly for 24 weeks, with bone marrow evaluation at 24 weeks.

Inclusion Criteria :

Confirmed diagnosis of VSAA/SAA. Age ≥65 years. Completion of all screening assessments. Ability to swallow oral medication. Signed informed consent (by patient or legal guardian if patient is incapacitated).

Exclusion Criteria :

Clonal cytogenetic abnormalities (excluding isolated -Y or +8). Prior treatment with ATG/high-dose cyclophosphamide. Prior cyclosporine/tacrolimus use >12 months. Prior TPO-RA therapy >3 months. Uncontrolled malignancies or conditions contraindicating ATG. Severe organ dysfunction (e.g., creatinine ≥177 μmol/L). Investigator judgment of unsuitability.

Enrollment: 65 estimated patients

Sex: All

Ages: 65+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clinical diagnosis of VSAA/SAA.
• Age ≥65 years.
• Completion of all screening assessments.
• Must be able to swallow tablets.
• Signed informed consent (by patient or legal guardian if patient is incapacitated).

Exclusion criteria

• Clonal cytogenetic abnormalities (excluding isolated -Y or +8).
• Prior treatment with ATG/high-dose cyclophosphamide.
• Prior cyclosporine/tacrolimus use >12 months.
• Prior TPO-RA therapy >3 months.
• Uncontrolled malignancies or conditions contraindicating ATG.
• Severe organ dysfunction (e.g., creatinine ≥177 μmol/L).
• Investigator judgment of unsuitability.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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65 participants in 1 patient group

A-IST

Experimental group

Description:

Porcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily.

Treatment:

Drug: Dose-attenuated IST and Hetrombopag