CsA+ATG+AVA vs. CsA+AVA for the Treatment of Newly-diagnosed SAA in the Elderly

Peking Union Medical College Hospital86 enrolled

Overview

This is a multicenter, prospective, randonmized study. Our previous retrospective study showed that for SAA patients who were intolerant to ATG, CsA+ eltrombopag (EPAG) had similar efficacy to CsA+ATG+EPAG. Since the action mechanism of AVA and EPAG is not exactly the same, and the metabolic level of the elderly is not the same as that of younger patients, it is unknown whether there are predictive factors of efficacy in the treatment of AVA. We wondered whether CsA+AVA could achieve an efficacy similar to CsA+ATG+AVA in the Elderly. Meanwhile, to explore the predictive factors of efficacy, to find out a safe and effective treatment strategy for the Elderly.

Aplastic anemia (AA) can be divided into severe AA (SAA) and non-severe AA (NSAA), according to the severity of the disease. For SAA patients with o condition of hematopoietic stem cell transplantation, guideline recommended ciclosporin A (CsA) + anti-thymocyte immunoglobulin (ATG) + thrombopoietin receptor agonists (TPO-RA) as the first-line therapy. Our previous retrospective study showed that for SAA patients who were intolerant to ATG, CsA+ eltrombopag (EPAG) had similar efficacy to CsA+ATG+EPAG. However, the adverse effects such as hepatotoxicity and gasrointestinal disorder limit its application in elderly SAA.Compared with EPAG, AVA has no hepatotoxicity and less other adverse effects, but there was few studies for AA. Our previous study demonstrated that AVA was effective in 55% of refractory AA. Since the action mechanism of AVA and EPAG is not exactly the same, and the metabolic level of the elderly is not the same as that of younger patients, it is unknown whether there are predictive factors of efficacy in the treatment of AVA. Thus, we wondered whether CsA+AVA could achieve an efficacy similar to CsA+ATG+AVA in the Elderly.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Eligibility

Sex: ALLMin age: 60 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Patients with newly diagnosed severe aplastic anemia, aged greater than 60 years. 2. Patients met the diagnostic criteria of severe aplastic anemia (SAA). 3. Complete all screening assessments as outlined in the test protocol. 4. Without or with no more than 1 month treatment of ciclosporine, tacrolimus, glucocortocoid, or TPO-RAs. 5. Agree to sign the informed consent form. Exclusion Criteria: 1. Known diagnosis of congenital hematopoietic failure disorders (e.g. Fanconi anemia) and other causes of cytopenia. 2. Patients with uncontrolled bleeding and/or infection despite standard treatment. 3. Patients with previous history of hematopoietic stem cell transplantation. 4. Patients with previous history of thrombosis in 1 year. 5. Patients with concurrent malignancy. 6. Those who are considered unsuitable for enrollment by the investigator. 7. Abnormal renal function: creatinine \> 1.2 normal upper limit, albumin \< 0.9 normal lower limit, or CLcr \< 30 ml/min. 8. Abnormal liver function: transaminase \> 2.5 normal upper limit, or tota bilirubin \> 2.5 normal upper limit. 9. Patients with severe heart, liver or renal disease.

Outcomes

Primary Outcomes

Overall response rate

Proportion of patient who achieved partial response or complete response

Time frame: 6 month

Secondary Outcomes

Complete response rate

Proportion of patient who achieved complete response (HGB\>100g/L, PLT\>100×109/L, ANC\>1.0×109/L)

Time frame: 6 month

Relapse rate

Proportion of patient who relapse

Time frame: 12 month

Adverse event rate

Proportion of patient who adverse events

Time frame: 3, 6, 12 month

CsA+ATG+AVA vs. CsA+AVA for the Treatment of Newly-diagnosed SAA in the Elderly

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes