eligibility_summary
Eligible: 40–60, refractory severe idiopathic aplastic anemia after ≥6 mo ATG+cyclosporine±eltrombopag or relapse, HSCT approved, geno-identical, 10/10, or haplo donor, no DSA (MFI<1500), ECOG≤2, no uncontrolled infection, adequate cardiac, hepatic (AST/ALT≤3N, bili≤2N), renal (CrCl≥50) function, insured, consent, contraception. Exclude: clonal evolution (chr7/complex), HIV/HTLV/HBV/HCV with cytolysis, cancer <5y, pregnancy/breastfeeding, live vaccines <2 mo, major cardiac/renal disease, drug contraindications/allergy, psychiatric/legal/state protection, other trials.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
manual_review_required
ai_summary
Phase II single-arm trial of an optimized allogeneic bone marrow HSCT for refractory/relapsed severe aplastic anemia (age 40–60), centered on post-transplant cyclophosphamide (PTCy). Interventions: conditioning with Thymoglobulin/ATG (polyclonal antibody, T‑cell depletion), fludarabine (purine analog antimetabolite, lymphodepletion), cyclophosphamide (alkylator, immunoablation), and low‑dose TBI (2 Gy). GVHD prophylaxis: PTCy (selective killing of proliferating alloreactive T cells), tacrolimus (calcineurin inhibitor, blocks IL‑2/NFAT signaling), mycophenolate mofetil (IMPDH inhibitor, limits lymphocyte proliferation). Supportive: G‑CSF (growth factor, CSF3R activation for neutrophil recovery) and rituximab (anti‑CD20 mAb, B‑cell depletion to prevent EBV‑PTLD). Targets/pathways: donor/host T cells and alloreactivity, calcineurin–NFAT–IL‑2 axis, de novo guanine synthesis, DNA crosslinking, marrow niche, CD20+ B cells, granulopoiesis.