eligibility_summary
Eligibility: Age ≥12 with treatment‑naïve severe/very severe aplastic anemia, ECOG ≤2, unable/unwilling for allogeneic HSCT, and consent/ability to comply. Exclude: >4 wks prior IST or TPO‑RA, allergy to ALG, cyclosporine, herombopag, or cyclophosphamide, uncontrolled infection (HBV/HCV allowed unless severe liver failure, hepatitis‑associated AA allowed), HIV/syphilis, severe organ failure/major comorbidities, solid tumor chemo/radiation ≤5y, pregnancy/lactation, other investigator concerns.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
Phase 2 single-arm study (China) of “hypo-CASH” as frontline therapy for severe/very severe aplastic anemia: anti-lymphocyte globulin (ALG), cyclosporine A (CsA), hetrombopag (TPO receptor agonist), plus reduced-dose cyclophosphamide. Drug types and mechanisms: ALG (polyclonal antibody) depletes/suppresses T lymphocytes, CsA (calcineurin inhibitor) blocks T-cell activation by inhibiting calcineurin–NFAT and IL-2 transcription, cyclophosphamide (alkylating agent) provides additional lymphotoxic immunosuppression via DNA crosslinking, hetrombopag (oral small-molecule TPO-RA) stimulates c-Mpl to promote megakaryocyte and hematopoietic stem/progenitor cell proliferation. Targets/pathways: autoreactive T cells, calcineurin–NFAT/IL-2 signaling, DNA in proliferating lymphocytes, and the TPO–c-Mpl pathway to restore marrow hematopoiesis.