This is a prospective, single-arm, open-label, exploratory clinical study designed to evaluate the safety and efficacy of Elranatamab for inhibitor eradication in adults with moderate-to-severe hemophilia A with inhibitors. A total of 15-20 patients aged ≥18 years will be enrolled. Elranatamab will be administered with a stepwise dose-escalation regimen of 12 mg on Day 1 and 32 mg on Day 4 during Week 1, followed by the target dose of 38 mg once weekly during Weeks 2-3. Patients will then enter a 4-week follow-up period. If the inhibitor titer decreases by \<20% from baseline at Week 3, two additional weekly doses of 38 mg may be administered before follow-up. Treatment response will be assessed according to changes in inhibitor titers following treatment.
Male or female patients with moderate-to-severe hemophilia A (factor VIII activity <2%).
Aged 18 to 65 years, inclusive.
Positive factor VIII inhibitor detected on at least two consecutive occasions (inhibitor titer >0.6 BU/mL).
Baseline factor VIII inhibitor titer >10 BU/mL at the time of enrollment.
Known hypersensitivity to Elranatamab or any of its excipients.
Presence of other autoimmune diseases or a need for immunosuppressive therapy for reasons unrelated to this study.
History of malignancy within 5 years prior to screening, with the exception of adequately treated carcinoma in situ of the cervix or non-metastatic cutaneous squamous cell carcinoma or basal cell carcinoma.
History of severe recurrent or chronic infections, or acute infection requiring systemic treatment with antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungal agents within 4 weeks before the first dose or during the screening period, or superficial skin infection requiring systemic therapy within 1 week before the first dose.
Single group
Elranatamab
DrugPatients receive Elranatamab at 12 mg on Day 1 and 32 mg on Day 4 during Week 1, followed by the target dose of 38 mg once weekly during Weeks 2-3. Patients will then enter a 4-week follow-up period. If the inhibitor titer decreases by \<20% from baseline at Week 3, two additional weekly doses of 38 mg may be administered before follow-up. If the inhibitor titer decreases by \>20% from baseline at Week 3, no further treatment will be given, and patients will proceed directly to follow-up.
Incidence of Treatment-Emergent Adverse Events (AES)
AES was assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0.
The proportion of patients who achieve successful inhibitor eradication
the number of patients with successful inhibitor eradication divided by the total number of patients in the study, expressed as a percentage.
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