Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

A Prospective, Single Center, Single-arm Study on the Efficacy and Safety of Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

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.

Inclusion Criteria:

• 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
• Willing and able to comply with the requirements for this study and written informed consent.

Exclusion Criteria:

• 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
• Allergy to ALG
• Presence of uncontrolled active infection
• Presence of uncontrolled hypertension (≥140/90mmHg), diabetes mellitus (fasting blood glucose≥ 7.0 mmol/L or random blood glucose ≥ 11.1 mmol/L)
• Abnormal liver and kidney function: Aspartate Transaminase (AST) or Alanine Transaminase (ALT) > 3 ULN, creatinine ≥ 2.5 ULN
• History of chemoradiotherapy for malignant solid tumors
• History of other systemic serious illnesses
• Females who are pregnant/lactating or need pregnancy
• Patients considered to be ineligible for the study by the investigator for reasons other than the above