eligibility_summary
Eligibility: Myelofibrosis (primary or secondary to PV/ET), no matched sibling or unrelated donor, but a haploidentical donor is available, signed consent. Exclusions: active infection, ECOG >2, expected survival <30 days, inability to cooperate, deemed unsuitable after discussion.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
manual_review_required
ai_summary
Intervention: Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) using Dac/TT/Bu/Flu/ATG conditioning and standard GVHD prophylaxis. Drug types and mechanisms: Decitabine (hypomethylating antimetabolite, DNMT inhibitor → DNA hypomethylation/cytotoxicity), Thiotepa (alkylating agent, DNA cross-linking), Busulfan (alkylating myeloablative agent, DNA cross-linking), Fludarabine (purine analogue antimetabolite, inhibits DNA synthesis, lymphodepleting), Anti-thymocyte globulin/ATG (polyclonal antibody, T-cell depletion). GVHD prophylaxis: Cyclosporine (calcineurin inhibitor, blocks IL-2/T-cell activation), Mycophenolate mofetil (IMPDH inhibitor, blocks lymphocyte proliferation), short-course Methotrexate (antimetabolite, DHFR inhibition, T-cell suppression). Targets: recipient hematopoietic stem/progenitor cells and T cells (myeloablation/immune reset), donor CD34+ HSC engraftment, T-cell activation pathways (calcineurin–IL-2), nucleotide synthesis (IMPDH, DHFR), DNA methylation.