This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) <30 × 10^9/L and/or hemoglobin (HGB) <90 g/L at enrollment. If the phase I results demonstrate an acceptable safety profile and allow determination of the maximum tolerated dose (MTD), the phase II part will be initiated directly to evaluate the efficacy of isatuximab.
Inclusion Criteria:
Exclusion Criteria:
zhanglele@ihcams.ac.cnâ€15811139278‬
The phase I part is the dose-escalation stage dose escalation will follow a standard 3+3 design. The phase II part is a single-arm study using Simon's two-stage design.
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