This prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA.
Age ≥ 12 years
Diagnosis of severe or very severe aplastic anemia
Patients are unconditionally or unacceptable undergoing allogeneic hematopoietic stem cell transplantation
ECOG performance status ≤2
Received > 4 weeks of TPO-RA drug before treatment
Received > 4 weeks of immunosuppressive therapy before treatment
History of thromboembolic disease
Intolerance to Romiplostim N01 or cyclosporine
Single group
standard IST combined with Romiplostim N01
DrugRomiplostim N01 is a class of TPO receptor agonists. Its TPO peptide can bind to TPO receptors on megakaryocyte precursors in the bone marrow and activate them, triggering a series of intracellular signalling pathways, including JAK2/STAT5, PI3K/Akt, MEK/ERK, and p38, which ultimately lead to gene transcription and increased megakaryocyte proliferation and differentiation.
Overall response rate
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
Overall response rate
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
Time to hematological response
Duration time was calculated from enrollment to achieve hematological response.
Incidence of adverse events
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
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Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
Qilu Pharmaceutical Co., Ltd.
Collaborator
