Lower-risk Myelodysplastic Syndromes
Conditions
Brief summary
This study aims to compare the efficacy and safety of recombinant human thrombopoietin (rhTPO) + cyclosporine (CsA) and CsA monotherapy in the treatment of lower-risk myelodysplastic neoplasms with thrombocytopenia.
Interventions
DRUGrhTPO
rhTPO 300U/kg/d, subcutaneous injection, once a month for 14 consecutive days
CsA 3-5mg/kg/d
Sponsors
Peking Union Medical College Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age ≥ 18 years old; 2. Confirmed diagnosis of lower-risk MDS; 3. Platelet ≤ 30×109/L, or \< 50×109/L and accompanied by bleeding events; 4. Baseline liver and kidney functions \<2 ULN; 5. Signed the informed consent form; 6. ECOG 0-2;
Exclusion criteria
1. History of leukemia or stem cell transplantation, treatment-related MDS or malignant tumors; 2. With splenomegaly or myelofibrosis; 3. Infections or bleeding that cannot be controlled by standard treatment; 4. Any concurrent malignant tumors within the past 5 years, or basal cell carcinoma of the skin at the local site; 5. History of thromboembolic events, heart attack or stroke and current use of anticoagulants; 6. Previously received cyclosporine (CsA) or ATG within 6 months; 7. Previously used IL-11, rhTPO or TPO-RA; 8. Pregnant or lactating women; 9. Participated in other clinical trials within 3 months.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| HI-P | 1-month, 2-month, 3-month, 6-month | hematological response - platelet |
| ORR | 1-month, 2-month, 3-month, 6-month | ORR=CRR+PRR+HI |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of bleeding incidents and severity assessed by the World Health Organization Bleeding Scale | 3-month, 6-month | minimum value = 0, maximum value = 4; higher scores indicate worse outcome |
| Platelet transfusion independence | 3-month, 6-month | according to the number and interval of platelet transfusions |
| Time to CR/OR | through study period, an average of 6 months | Time from baseline to first response |
| Duration of HI-P | through study period, an average of 6-month | Time to patients achieving HI-P to losing response |
| Quality of life score measured by the Short Form-36 Health Survey | 3-month | minimum value = 0, maximum value = 100; higher scores indicate better quality of life |
| Clonal evolution | 12-month | The incidence of elevation of blast cells in bone marrow and peripheral blood within one year, and the incidence of progressing to higher-risk MDS or AML within one year |
| OS | 12-month | Overall survival (OS), defined as the time from randomization/treatment start to death from any cause. |
Contacts
CONTACTBing Han, Doctor
Outcome results
None listed