Trial Outcomes & Findings for Antifugal Effect of Recombinant Human Granulocyte-macrophage Stimulating Factor (rhGM-CSF) in Patients Post Allo-HSCT (NCT NCT01232504)
NCT ID: NCT01232504
Last Updated: 2014-11-04
Results Overview
The incidence of proven and probable Invasive fungal diseases (IFD) within 100 days post transplantation
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
206 participants
Primary outcome timeframe
100 day post transplant
Results posted on
2014-11-04
Participant Flow
Participant milestones
| Measure |
rhGM-CSF Group
subcutaneous recombinant human granulocyte-macrophage stimulating factor (rhGM-CSF) 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF+ rhGM-CSF Group
a combination of 2-3μg/kg/d recombinant human granulocyte-macrophage stimulating factor (rhGM-CSF) and 2-3μg/kg/d recombinant human granulocyte stimulating factor (rhG-CSF) each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
subcutaneous recombinant human granulocyte stimulating factor (rhGM-CSF) 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Overall Study
STARTED
|
68
|
69
|
69
|
|
Overall Study
COMPLETED
|
59
|
67
|
61
|
|
Overall Study
NOT COMPLETED
|
9
|
2
|
8
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Antifugal Effect of Recombinant Human Granulocyte-macrophage Stimulating Factor (rhGM-CSF) in Patients Post Allo-HSCT
Baseline characteristics by cohort
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF+ rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
Total
n=206 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
30.68 years
n=5 Participants
|
32.55 years
n=7 Participants
|
32.04 years
n=5 Participants
|
31.75 years
n=4 Participants
|
|
Age, Categorical
<=18 years
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
22 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
60 Participants
n=5 Participants
|
64 Participants
n=7 Participants
|
60 Participants
n=5 Participants
|
184 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
32 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
74 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
51 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
44 Participants
n=5 Participants
|
132 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
68 Participants
n=5 Participants
|
69 Participants
n=7 Participants
|
69 Participants
n=5 Participants
|
206 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
China
|
68 participants
n=5 Participants
|
69 participants
n=7 Participants
|
69 participants
n=5 Participants
|
206 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 100 day post transplantPopulation: The number of intention-to-treat patients is 206. The incidence of invasive fungal infection analysis within 100 day is based on intention-to-treat (ITT)patients .
The incidence of proven and probable Invasive fungal diseases (IFD) within 100 days post transplantation
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Incidences of Invasive Fungal Diseases (IFD)
|
11.76 percentage of partipants
|
10.14 percentage of partipants
|
23.19 percentage of partipants
|
SECONDARY outcome
Timeframe: 100 days post transplantPopulation: The number of intention-to-treat patients is 206. The hematological engraftment analysis within 100 day is based on intention-to-treat (ITT)patients .
The median time of neutrophil and platelet recovery .
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Hematological Engraftment
Median time for neutrophil recovery to 0.5×10(9)/L
|
13 days
Interval 9.0 to 25.0
|
11 days
Interval 5.0 to 28.0
|
11 days
Interval 6.0 to 28.0
|
|
Hematological Engraftment
Median time for platelet recovery to 50 × 10(9)/L
|
18 days
Interval 10.0 to 200.0
|
19 days
Interval 10.0 to 200.0
|
25 days
Interval 12.0 to 200.0
|
SECONDARY outcome
Timeframe: 100 days post transplantPopulation: The number of intention-to-treat patients is 206. The transplant related mortality analysis within 100 day is based on intention-to-treat (ITT) patients .
Transplant related mortality within 100 days after Allogeneic Stem Cell Transplantation (Allo-HSCT).
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Transplant Related Mortality
|
8.8 percentage of participants
Interval 3.3 to 18.2
|
8.7 percentage of participants
Interval 3.3 to 17.9
|
21.7 percentage of participants
Interval 12.7 to 33.3
|
SECONDARY outcome
Timeframe: 100 days post transplantPopulation: The number of intention-to-treat patients is 206. The incidence of aGVHD analysis is based on intention-to-treat (ITT) patients .
Incidence of Ⅱ- Ⅳacute graft versus host disease (aGVHD) within 100 days after allogeneic stem cell transplantation (Allo-HSCT).The severity of acute GVHD in the three main target organs (skin, liver, gastrointestinal tract) was assigned stage 1 to 4 based on accepted criteria (Consensus Conference on Acute GVHD Grading).
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Incidence of Ⅱ- Ⅳ Acute Graft Versus Host Disease (aGVHD)
|
30.88 percentage of partipants
Interval 20.2 to 43.3
|
24.64 percentage of partipants
Interval 15.1 to 36.5
|
21.74 percentage of partipants
Interval 12.7 to 33.3
|
SECONDARY outcome
Timeframe: 3-1099 daysPopulation: The number of intention-to-treat patients is 206. The IFD related mortality analysis is based on intention-to-treat (ITT) patients .
IFD-related mortalities after a median follow-up of 600 days.
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
IFD Related Mortality
|
1.47 percentage of participants
Interval 0.04 to 7.92
|
1.45 percentage of participants
Interval 0.04 to 7.81
|
11.59 percentage of participants
Interval 5.14 to 21.57
|
SECONDARY outcome
Timeframe: 3～1099 daysPopulation: The number of intention-to-treat patients is 206. The IFD related mortality analysis is based on intention-to-treat (ITT) patients .
Relapse related mortality after a median follow-up of 600 days.
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Relapse Related Mortality
|
5.8 percentage of participants
|
8.7 percentage of participants
|
4.35 percentage of participants
|
SECONDARY outcome
Timeframe: 3-1099 daysPopulation: The number of intention-to-treat patients is 206. The IFD related mortality analysis is based on intention-to-treat (ITT) patients .
Graft versus host disease (aGVHD) related mortality after a median follow-up of 600 days .
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Graft Versus Host Disease (aGVHD) Related Mortality
|
8.82 percentage of participants
|
4.35 percentage of participants
|
7.25 percentage of participants
|
SECONDARY outcome
Timeframe: 3-1099 daysPopulation: The number of intention-to-treat patients is 206. The IFD related mortality analysis is based on intention-to-treat (ITT) patients .
Hemorrhage related mortality after a median follow-up of 600 days
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Hemorrhage Related Mortality
|
5.88 percentage of participants
|
1.45 percentage of participants
|
1.45 percentage of participants
|
SECONDARY outcome
Timeframe: 3～1099 days)Population: The number of intention-to-treat patients is 206. The IFD related mortality analysis is based on intention-to-treat (ITT) patients .
Infection related mortality after a median follow-up of 600 days.
Outcome measures
| Measure |
rhGM-CSF Group
n=68 Participants
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 Participants
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 Participants
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Infection Related Mortality
|
1.47 percentage of participants
|
5.8 percentage of participants
|
14.50 percentage of participants
|
Adverse Events
rhGM-CSF Group
Serious events: 0 serious events
Other events: 32 other events
Deaths: 0 deaths
rhG-CSF + rhGM-CSF Group
Serious events: 0 serious events
Other events: 31 other events
Deaths: 0 deaths
rhG-CSF Group
Serious events: 0 serious events
Other events: 28 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
rhGM-CSF Group
n=68 participants at risk
subcutaneous rhGM-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF + rhGM-CSF Group
n=69 participants at risk
a combination of 2-3μg/kg/d rhGM-CSF and 2-3μg/kg/d rhG-CSF each after transplantation, started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
rhG-CSF Group
n=69 participants at risk
subcutaneous rhG-CSF 5-7μg/kg/d , started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days).
|
|---|---|---|---|
|
Infections and infestations
fever
|
47.1%
32/68 • 28 days post transplant
|
44.9%
31/69 • 28 days post transplant
|
40.6%
28/69 • 28 days post transplant
|
Additional Information
Chun Wang
Department of Hematology, Shanghai Jiao Tong University Affiliated First People's Hospital, China
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place