Lower-dose immunosuppression plus hetrombopag for severe aplastic anemia in people 65 and older

Dose-attenuated IST and Hetrombopag in the Treatment of Elderly (≥65 Years) Patients With Very Severe/Severe Aplastic Anemia: A Single-Center, Single-Arm, Phase IIB Clinical Study on Efficacy and Safety

This study tests whether a lower-dose immunosuppressive regimen combined with hetrombopag can improve blood counts and safety for people aged 65 and older with severe or very severe aplastic anemia.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 65 (estimated)
• Ages: 65 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: cyclophosphamide
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT07010237 on ClinicalTrials.gov

What this trial studies

This is a prospective, single-center, single-arm, open-label Phase IIB trial testing dose-attenuated immunosuppressive therapy (IST) combined with hetrombopag in patients aged 65 or older with very severe or severe aplastic anemia. Treatment consists of P-ATG 15 mg/kg IV daily for 5 days, cyclosporine 3 mg/kg/day adjusted to trough levels of 100–200 μg/L, and hetrombopag 15 mg daily. Participants are followed weekly for 24 weeks with a bone marrow evaluation at week 24 to document hematologic response and monitor safety. Key eligibility excludes clonal cytogenetic abnormalities (except isolated -Y or +8), prior ATG/high-dose cyclophosphamide, prolonged prior calcineurin inhibitor or TPO-RA use, uncontrolled malignancy, or severe organ dysfunction.

Who should consider this trial

Good fit: Ideal candidates are people aged 65 or older with confirmed very severe or severe aplastic anemia who can swallow oral medication and do not have disqualifying clonal cytogenetics, uncontrolled cancer, or severe organ dysfunction.

Not a fit: Patients with prior ATG or high-dose cyclophosphamide, prolonged prior cyclosporine/tacrolimus use, recent TPO-RA exposure longer than three months, active uncontrolled malignancy, severe organ failure, or disqualifying cytogenetic abnormalities are excluded and unlikely to benefit from enrollment.

Why it matters

Potential benefit: If successful, this approach could increase response rates and reduce transfusion needs while offering a lower-toxicity option for older patients who tolerate standard IST poorly.

How similar studies have performed: Prior studies combining TPO-receptor agonists (such as eltrombopag) with standard IST have improved responses in SAA, but hetrombopag plus dose-attenuated IST specifically in elderly patients is less well studied.

Eligibility criteria

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.

Where this trial is running

Tianjin, Tianjin Municipality

• Red Blood Cell Diseases Center — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Principal investigator: Jun Shi, PhD — Institute of Hematology & Blood Diseases Hospital, CAMS
• Study coordinator: Jianping Li, MD

Conditions Sever Aplastic Anaemia; Elderly; Immunosuppressive Treatment