Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA

Launched by PEKING UNION MEDICAL COLLEGE HOSPITAL · May 16, 2024

Keywords

ClinConnect Summary

Conduct a comparative evaluation of the effectiveness and safety of Luspatercept combined with cyclosporine versus cyclosporine monotherapy in the treatment of newly diagnosed non-transfusion-dependent non-severe aplastic anemia (NSAA). Patients were randomized in a 1:1 ratio and assigned to one of two groups: Group A, Luspatercept combined with cyclosporine: received Luspatercept (1.0 mg/kg, subcutaneous injection every 3 weeks), cyclosporine (3-5mg/kg/day), adjusted based on hematological parameters, for at least 6 months to assess efficacy. Effective patients continued to receive cyclosp...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Age ≥ 18 years
• • 2. Hemoglobin level of 6-10 g/dL
• • 3. Definition of NSAA: Patients with AA diagnosis but not SAA or VSAA diagnosis (at least two of the following conditions can be diagnosed as AA: (i) Hemoglobin \< 100 g/L; (ii) Platelet count \< 50×10\^9/L; (iii) Neutrophil count \< 1.5×10\^9/L. SAA diagnosis criteria include less than 25% (or 25-50%, but residual hematopoietic cells \< 30%) of bone marrow cells, plus at least two of the following conditions: (i) Neutrophil count \< 0.5×10\^9/L; (ii) Platelet count \< 20×10\^9/L; (iii) Retroperitoneal lymph node count \< 20×10\^9/L. VSAA meets the criteria for SAA, but with neutrophil count \< 0.2×10\^9/L. (British guidelines, 2015))
• • 4. No active infection
• • 5. No other concurrent neoplasms (except in situ carcinoma)
• • 6. Baseline liver and renal function within 1.5 times of normal value
• • 7. No pregnancy or breastfeeding
• • 8. Agree to sign informed consent form
• • 9. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
• Exclusion Criteria:
• • 1. Congenital aplastic anemia
• • 2. Presence of chromosomal aberrations
• • 3. Cytogenetic evidence of clonal hematological myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML)
• • 4. PNH clone ≥50%
• • 5. Previous use of alemtuzumab, any ATG, or any dose of cyclosporine for immunosuppressive treatment
• • 6. Previous hematopoietic stem cell transplant (HSCT)
• • 7. Uncontrolled infection or bleeding under standard treatment
• • 8. Allergy to rituximab, cyclosporine, or excipients
• • 9. History of allergy to polyethylene glycol (PEG) 80
• • 10. Active infection or cirrhosis of the liver or portal hypertension due to HIV, HCV, or HBV
• • 11. Screening QTcF (Fridericia QT corrected formula) less than 450 milliseconds or less than 480 milliseconds of bundle branch block determined by three ECG averages, and assessed on-site; unstable angina; uncontrolled hypertension (\>180/100 mmHg); pulmonary hypertension
• • 12. Any malignant tumor within 5 years, except local basal cell carcinoma; previous thromboembolic event, history of myocardial infarction or stroke (including antiphospholipid syndrome); currently using anticoagulants
• • 13. Pregnant or lactating women
• • 14. Participated in another clinical trial within 3 months

Trial Officials