Transfusion dependent thalassemia
Conditions
Interventions
1:Rotercept (0.6mg/kg) q3w s.c.+ Thalidomide 1.8mg/kg q12h p.o.
2:Rotercept (1.25mg/kg) q3w s.c.+ Thalidomide 1.8mg/kg q12h p.o.
3:Rotercept (1.25mg/kg) q3w s.c.+ Thalidomide 3.3mg/kg q12h p.o.
Sponsors
Sun Yat-sen Memorial Hospital, Sun Yat-sen University
Eligibility
Sex/Gender
All
Age
12 Years to 25 Years
Inclusion criteria
Inclusion criteria: (1) =12 years old, =25 years old; (2) A clear diagnosis of transfusion-dependent beta-thalassemia (which can be combined with static and mild alpha-thalassemia) with a blood transfusion cycle =35 days and a red blood cell infusion volume of 6-12u within the first 12 weeks of enrollment could be provided with a record of red blood cell infusion volume within the first 12 weeks of enrollment (3) Join the study voluntarily and sign the informed consent;
Exclusion criteria
Exclusion criteria: (1) Pregnant or lactating women (2) Allergic to Rotercept and/or Rotercept for injection (3) Severe hepatic insufficiency (liver enzyme (ALT or AST) =3xULN) (4) Severe renal impairment (eGFR < 30 ml/min/1.73m3 or patients with end-stage renal disease) (5) Heart disease, New York Heart Association (NYHA) grade 3 or higher heart failure, or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months. (6) The patient had uncontrolled hypertension, and according to NCI CTCAE version 5.0, controlled hypertension in this protocol was considered to be = grade 1. (7) Patients with a history of deep vein thrombosis or stroke during the first 24 weeks of enrollment (8) ESA, Rotercept, thalidomide, or hydroxyurea were used in the first 8 weeks of enrollment (9) Patients with Grade 3-4 adverse reactions that can not be alleviated after past use of Rotercept or thalidomide (10) any other significant medical condition, laboratory abnormality or mental illness that the investigator deems unsuitable for inclusion (11) History of hematopoietic stem cell transplantation, complete/partial splenectomy, splenic embolization.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| overall response rate;incidence of side effect; | — |
Secondary
| Measure | Time frame |
|---|---|
| The proportion of patients with 100% reduction in red cell (RBC) infusion burden from baseline at any 12 weeks during 24 weeks of treatment;Mean cumulative blood transfusion volume and number of transfusions from baseline at weeks 1-12 and 1-24;Proportion of =8 weeks and =12 weeks transfusion independent(TI); Change in mean serum ferritin (SF) levels from baseline; 27/5000 Changes in cardiac and hepatic iron concentrations from baseline at 24 weeks of treatment ;Changes in reticulocyte and fetal hemoglobin levels from baseline at 24 weeks of treatment; 35/5000 Proportion of patients with <50% reduction in red cell (RBC) infusion burden from baseline at 24 weeks of treatment; | — |
Countries
China
Contacts
Public ContactJianpei Fang
Sun Yat-sen Memorial Hospital, Sun Yat-sen University
Outcome results
None listed