Trial Outcomes & Findings for Prospective Study of Patients With Thrombocytopenia Following HSCT (NCT NCT02487563)
NCT ID: NCT02487563
Last Updated: 2020-05-06
Results Overview
Platelet response refers to a sustained increase (stable or increasing level) of at least 30×10E9/L independent of transfusion for 3 days.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
97 participants
Primary outcome timeframe
Up to 4 weeks after the treatment
Results posted on
2020-05-06
Participant Flow
Two participants from different institutions submitted the applications at the same time when there was only one quota left. Both of them met the inclusion criteria and were enrolled.
Participant milestones
| Measure |
Experimental Group 1
Decitabine in combination with rhTPO.
Decitabine: Decitabine
rhTPO: rhTPO
|
Experimental Group 2
Decitabine
Decitabine: Decitabine
|
Control Group
Conventional treatment except decitabine.
Conventional Treatment: immunoglobulin, glucocorticoid etc
|
|---|---|---|---|
|
Overall Study
STARTED
|
32
|
33
|
32
|
|
Overall Study
COMPLETED
|
30
|
30
|
31
|
|
Overall Study
NOT COMPLETED
|
2
|
3
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Experimental Group 1
n=30 Participants
Decitabine 15 mg/m2 daily intravenously for consecutive 3 days (day 1 to day 3), combined with rhTPO 15000 U daily subcutaneously beginning on day 4 and continuing until the response was achieved or the time of initial evaluation.
|
Experimental Group 2
n=30 Participants
Decitabine 15 mg/m2 daily intravenously for consecutive 3 days (day 1 to day 3) alone.
|
Control Group
n=31 Participants
Conventional therapies with recommended options including rhTPO, eltrombopag, immunoglobulin, rituximab, interleukin-11 and glucocorticoids, alone or in combination.
|
Total
n=91 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
30 years
n=30 Participants
|
36 years
n=30 Participants
|
36 years
n=31 Participants
|
31 years
n=91 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=30 Participants
|
12 Participants
n=30 Participants
|
15 Participants
n=31 Participants
|
32 Participants
n=91 Participants
|
|
Sex: Female, Male
Male
|
25 Participants
n=30 Participants
|
18 Participants
n=30 Participants
|
16 Participants
n=31 Participants
|
59 Participants
n=91 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
China
|
30 Participants
n=30 Participants
|
30 Participants
n=30 Participants
|
31 Participants
n=31 Participants
|
91 Participants
n=91 Participants
|
|
Diagnosis
Acute lymphocytic leukemia
|
8 Participants
n=30 Participants
|
10 Participants
n=30 Participants
|
11 Participants
n=31 Participants
|
29 Participants
n=91 Participants
|
|
Diagnosis
Acute myeloid leukemia
|
15 Participants
n=30 Participants
|
14 Participants
n=30 Participants
|
12 Participants
n=31 Participants
|
41 Participants
n=91 Participants
|
|
Diagnosis
Chronic myeloid leukemia
|
0 Participants
n=30 Participants
|
2 Participants
n=30 Participants
|
1 Participants
n=31 Participants
|
3 Participants
n=91 Participants
|
|
Diagnosis
Myelodyplastic syndrome
|
5 Participants
n=30 Participants
|
4 Participants
n=30 Participants
|
4 Participants
n=31 Participants
|
13 Participants
n=91 Participants
|
|
Diagnosis
Non-Hodgkin lymphoma
|
2 Participants
n=30 Participants
|
0 Participants
n=30 Participants
|
3 Participants
n=31 Participants
|
5 Participants
n=91 Participants
|
|
Transplant Donor
Haploidentical donor
|
23 Participants
n=30 Participants
|
20 Participants
n=30 Participants
|
21 Participants
n=31 Participants
|
64 Participants
n=91 Participants
|
|
Transplant Donor
Matched related donor
|
5 Participants
n=30 Participants
|
8 Participants
n=30 Participants
|
6 Participants
n=31 Participants
|
19 Participants
n=91 Participants
|
|
Transplant Donor
Matched unrelated donor
|
2 Participants
n=30 Participants
|
2 Participants
n=30 Participants
|
4 Participants
n=31 Participants
|
8 Participants
n=91 Participants
|
|
ABO compatitibilty
ABO matched
|
15 Participants
n=30 Participants
|
13 Participants
n=30 Participants
|
18 Participants
n=31 Participants
|
46 Participants
n=91 Participants
|
|
ABO compatitibilty
ABO mismatched
|
15 Participants
n=30 Participants
|
17 Participants
n=30 Participants
|
13 Participants
n=31 Participants
|
45 Participants
n=91 Participants
|
|
Conditioning
Busulfan / cyclophosphamide
|
26 Participants
n=30 Participants
|
27 Participants
n=30 Participants
|
29 Participants
n=31 Participants
|
82 Participants
n=91 Participants
|
|
Conditioning
Others
|
4 Participants
n=30 Participants
|
3 Participants
n=30 Participants
|
2 Participants
n=31 Participants
|
9 Participants
n=91 Participants
|
|
Mononuclear cell infused
|
9.78 10E8/kg
n=30 Participants
|
10.50 10E8/kg
n=30 Participants
|
9.40 10E8/kg
n=31 Participants
|
10.04 10E8/kg
n=91 Participants
|
|
Neutrophils recovery
|
12 days
n=30 Participants
|
12 days
n=30 Participants
|
13 days
n=31 Participants
|
12 days
n=91 Participants
|
|
Enrollment time
|
103 days
n=30 Participants
|
105 days
n=30 Participants
|
79 days
n=31 Participants
|
99 days
n=91 Participants
|
|
Platelet counts at enrollment
|
14 10E9 platelets/L
n=30 Participants
|
12 10E9 platelets/L
n=30 Participants
|
17 10E9 platelets/L
n=31 Participants
|
15 10E9 platelets/L
n=91 Participants
|
|
Megakaryocyte counts at enrollment
|
2.9 cells/cm^2
n=30 Participants
|
3.2 cells/cm^2
n=30 Participants
|
3.1 cells/cm^2
n=31 Participants
|
3.0 cells/cm^2
n=91 Participants
|
PRIMARY outcome
Timeframe: Up to 4 weeks after the treatmentPlatelet response refers to a sustained increase (stable or increasing level) of at least 30×10E9/L independent of transfusion for 3 days.
Outcome measures
| Measure |
Experimental Group 1
n=30 Participants
Decitabine in combination with rhTPO.
Decitabine: Decitabine
rhTPO: rhTPO
|
Experimental Group 2
n=30 Participants
Decitabine
Decitabine: Decitabine
|
Control Group
n=31 Participants
Conventional treatment except decitabine.
Conventional Treatment: immunoglobulin, glucocorticoid etc
|
|---|---|---|---|
|
Number of Participants With Platelet Count Recovery
|
20 Participants
|
22 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: Up to 4 weeks after the treatmentThe total number of megakaryocytes as well as the platelet-shedding megakaryocytes of bone marrow smears (per cm2) was counted and cross-checked by blinded observers.
Outcome measures
| Measure |
Experimental Group 1
n=30 Participants
Decitabine in combination with rhTPO.
Decitabine: Decitabine
rhTPO: rhTPO
|
Experimental Group 2
n=30 Participants
Decitabine
Decitabine: Decitabine
|
Control Group
n=31 Participants
Conventional treatment except decitabine.
Conventional Treatment: immunoglobulin, glucocorticoid etc
|
|---|---|---|---|
|
Megakaryocyte Count
|
6.7 cells/cm^2
Interval 1.2 to 10.0
|
7.5 cells/cm^2
Interval 3.1 to 14.1
|
3.3 cells/cm^2
Interval 1.8 to 6.0
|
Adverse Events
Experimental Group 1
Serious events: 4 serious events
Other events: 8 other events
Deaths: 1 deaths
Experimental Group 2
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
Control Group
Serious events: 3 serious events
Other events: 7 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Experimental Group 1
n=30 participants at risk
Decitabine 15 mg/m2 daily intravenously for consecutive 3 days (day 1 to day 3), combined with rhTPO 15000 U daily subcutaneously beginning on day 4 and continuing until the response was achieved or the time of initial evaluation.
|
Experimental Group 2
n=30 participants at risk
Decitabine 15 mg/m2 daily intravenously for consecutive 3 days (day 1 to day 3) alone.
|
Control Group
n=31 participants at risk
Conventional therapies with recommended options including rhTPO, eltrombopag, immunoglobulin, rituximab, interleukin-11 and glucocorticoids, alone or in combination.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Bleeding events
|
3.3%
1/30 • Study period (4 weeks)
|
3.3%
1/30 • Study period (4 weeks)
|
0.00%
0/31 • Study period (4 weeks)
|
|
Infections and infestations
Infections
|
6.7%
2/30 • Study period (4 weeks)
|
0.00%
0/30 • Study period (4 weeks)
|
6.5%
2/31 • Study period (4 weeks)
|
|
Cardiac disorders
Shock
|
3.3%
1/30 • Study period (4 weeks)
|
0.00%
0/30 • Study period (4 weeks)
|
3.2%
1/31 • Study period (4 weeks)
|
Other adverse events
| Measure |
Experimental Group 1
n=30 participants at risk
Decitabine 15 mg/m2 daily intravenously for consecutive 3 days (day 1 to day 3), combined with rhTPO 15000 U daily subcutaneously beginning on day 4 and continuing until the response was achieved or the time of initial evaluation.
|
Experimental Group 2
n=30 participants at risk
Decitabine 15 mg/m2 daily intravenously for consecutive 3 days (day 1 to day 3) alone.
|
Control Group
n=31 participants at risk
Conventional therapies with recommended options including rhTPO, eltrombopag, immunoglobulin, rituximab, interleukin-11 and glucocorticoids, alone or in combination.
|
|---|---|---|---|
|
Hepatobiliary disorders
Hypohepatia
|
6.7%
2/30 • Study period (4 weeks)
|
3.3%
1/30 • Study period (4 weeks)
|
3.2%
1/31 • Study period (4 weeks)
|
|
Cardiac disorders
Chest distress
|
3.3%
1/30 • Study period (4 weeks)
|
3.3%
1/30 • Study period (4 weeks)
|
0.00%
0/31 • Study period (4 weeks)
|
|
Gastrointestinal disorders
Anorexia
|
3.3%
1/30 • Study period (4 weeks)
|
0.00%
0/30 • Study period (4 weeks)
|
0.00%
0/31 • Study period (4 weeks)
|
|
General disorders
Fatigue
|
6.7%
2/30 • Study period (4 weeks)
|
3.3%
1/30 • Study period (4 weeks)
|
3.2%
1/31 • Study period (4 weeks)
|
|
Nervous system disorders
Headache
|
3.3%
1/30 • Study period (4 weeks)
|
0.00%
0/30 • Study period (4 weeks)
|
0.00%
0/31 • Study period (4 weeks)
|
|
Gastrointestinal disorders
Nausea
|
3.3%
1/30 • Study period (4 weeks)
|
0.00%
0/30 • Study period (4 weeks)
|
0.00%
0/31 • Study period (4 weeks)
|
|
Infections and infestations
Fever
|
3.3%
1/30 • Study period (4 weeks)
|
6.7%
2/30 • Study period (4 weeks)
|
3.2%
1/31 • Study period (4 weeks)
|
|
Eye disorders
Blurred vision
|
0.00%
0/30 • Study period (4 weeks)
|
3.3%
1/30 • Study period (4 weeks)
|
0.00%
0/31 • Study period (4 weeks)
|
|
Metabolism and nutrition disorders
Hypeluricemia
|
0.00%
0/30 • Study period (4 weeks)
|
3.3%
1/30 • Study period (4 weeks)
|
0.00%
0/31 • Study period (4 weeks)
|
|
Skin and subcutaneous tissue disorders
Erythra
|
0.00%
0/30 • Study period (4 weeks)
|
3.3%
1/30 • Study period (4 weeks)
|
3.2%
1/31 • Study period (4 weeks)
|
|
Blood and lymphatic system disorders
Bleeding events
|
10.0%
3/30 • Study period (4 weeks)
|
6.7%
2/30 • Study period (4 weeks)
|
9.7%
3/31 • Study period (4 weeks)
|
|
Infections and infestations
Infection
|
0.00%
0/30 • Study period (4 weeks)
|
0.00%
0/30 • Study period (4 weeks)
|
3.2%
1/31 • Study period (4 weeks)
|
Additional Information
Yaqiong Tang
Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University
Phone: +86 18896588075
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place