Beta thalassemia
Conditions
Interventions
Observation Group:Luspatercept 1.0mg/kg ,injection every 21 days
Sponsors
West China Hospital,Sichuan University
Eligibility
Sex/Gender
All
Age
18 Years to 90 Years
Inclusion criteria
Inclusion criteria: (1)aged 18 or above; (2) ß thalassemia or Hbe/ß thalassemia, allowing for deletion, mutation, or duplication of the a-globin gene; (3) transfusion volume =7.5 u in the first 24 weeks; (4) no blood transfusion was performed at least 8 weeks before enrollment; (5) baseline HB was less than or equal to 9 g/dL (based on at least two measurements within 4 weeks before enrollment, each measurement interval =1 week; HB values within 21 days after blood transfusion were excluded); (6) ECOG PS 0-1
Exclusion criteria
Exclusion criteria: (1) patients who were involved in an interventional clinical study with other drugs or medical devices; (2) pregnant or lactating women; (3) who were allergic to rotazip and/or injectable rotazip excipients; (4) severe hepatic insufficiency (ALT or AST =3 x ULN) ; (5) severe renal impairment (EGFR & LT; 30 ml/min/1.73 m 3 or ESRD) ; (6) heart disease, heart Failure of New York Heart Association (NYHA-RGraderade 3 or greater, or severe arrhythmias requiring treatment, or recent myocardial infarction within 6 months after randomization; (7) patients had uncontrolled hypertension; Controlled hypertension was considered grade =1 according to NCI CTCAE version 5.0; (8) patients with a history of thrombosis or stroke within 24 weeks before enrollment; (9) any other significant medical condition, laboratory abnormality or mental illness that the researcher considers unsuitable for inclusion;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hb;Proportion of patients with mean Hb increase of 1.0 g/d or more from baseline without blood transfusion; | — |
Secondary
| Measure | Time frame |
|---|---|
| Mean changes in Hb concentration from baseline in patients without blood transfusion ;Proportion of patients with mean Hb increase of 1.0 g/d or more without blood transfusion;Proportion of patients who maintain transfusion independence for 12 weeks and 16 weeks;Changes in SF from baseline; | — |
Countries
China
Contacts
Public ContactHe Chuan
West China Hospital,Sichuan University
Outcome results
None listed