Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of TD-NSAA

Launched by PEKING UNION MEDICAL COLLEGE HOSPITAL · Jul 26, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness and safety of a treatment called rhTPO when used together with a medication called cyclosporine, compared to using cyclosporine alone. The focus is on patients who have a condition known as transfusion-dependent non-severe aplastic anemia (TD-NSAA), which means they need regular blood transfusions due to low blood cell counts. The goal is to see if adding rhTPO can help improve their condition and reduce the need for transfusions.

To be eligible for this study, participants must be at least 18 years old and have a confirmed diagnosis of TD-NSAA. They should have been dependent on blood transfusions for at least four months and meet certain health criteria related to their blood cell counts and overall organ function. Participants will need to sign an informed consent form, meaning they agree to take part in the study and understand what it involves. This trial is not yet recruiting participants, so if you or a loved one are interested, it’s best to keep an eye out for announcements regarding when recruitment will begin.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• 1. aged ≥ 18 years, male and female; 2. patients with a clear diagnosis of NSAA who are dependent on transfusion therapy:
• • 1. Meet the Camitta NSAA criteria;
• • 2. accompanied by at least one of the following abnormalities: (1) dependence on component blood transfusion therapy, at least one component blood transfusion every 8 weeks on average, and the duration of transfusion dependence ≥ 4 months, the indication of component blood transfusion: HGB ≤ 60g / L; (2) PLT ≤ 10 × 10 \^ 9 / L, or PLT ≤ 30 × 10 \^ 9 / L with a significant tendency to bleed; (3) neutrophils ≤ 0.5 × 10 \^ 9 / L.
• • 3. Excluding other haematological and non-haematological diseases that cause pancytopenia; 3. ECOG PS score 0-2, expected survival ≥ 3 months with follow-up; 4. functional levels of major organs must meet the following requirements: 1) Total bilirubin (TBIL) ≤ 1.5 times the upper limit of normal (ULN); 2) alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x ULN; 3) blood creatinine (Cr) ≤ 1.5 x ULN; 5. has not been treated with platelet receptor agonist (TPO-RA) analogues and other immunosuppressant analogues; 6. the subject is not suitable or willing to receive haematopoietic stem cell transplantation therapy; 7. no history of serious heart, lung, liver, kidney and other important organs and endocrine system diseases; 8. Voluntarily enroll in the study, sign the informed consent, have good compliance and willing to cooperate with the follow-up.
• Exclusion Criteria:
• • 1. have used other clinical investigational drugs within 4 weeks;
• • 2. a history of primary myelodysplastic syndromes (MDS), primary paroxysmal sleep haemoglobinuria (PNH) and leukaemia, as well as congenital bone marrow failure syndromes (IBMFS), such as Fanconi's anaemia (FA) and congenital dyskeratosis (DC)
• • 3. history of cirrhosis or history of portal hypertension;
• • 4. congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiring medication within 1 year prior to enrolment, or myocardial infarction or cerebral infarction within 3 months prior to enrolment;
• • 5. HIV infection;
• • 6. severe autoimmune disease or immunodeficiency disease;
• • 7. suffering from malignant tumour
• • 8. severe mental disorders;
• • 9. a known history of allergy to the drug components of this regimen;
• • 10. in the opinion of the investigator, it is not appropriate to participate in this trial, e.g., any other medical, social or psychological factors that may affect safety or compliance with the study procedures.
• • Compliance with study procedures.