Trial Outcomes & Findings for A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous Plerixafor (NCT NCT01696461)
NCT ID: NCT01696461
Last Updated: 2023-09-14
Results Overview
Donor mobilization following plerixafor was considered successful if ≥ 2.0x106 CD34+ cells/kg recipient weight was collected in no more than two leukapheresis collections.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
127 participants
Primary outcome timeframe
donation
Results posted on
2023-09-14
Participant Flow
Eligible donor-recipient pairs were recruited at 12 US hematopoietic cell transplantation (HCT) transplant centers between July 2013 and December 2014. Recipients who met eligibility criteria provided written informed consent prior to any study procedure being performed. In order for the recipient to proceed in the study, intended donors were required to consent as well.
Participant milestones
| Measure |
Related Donors Receiving Plerixafor
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Donor eligibility was determined according to institutional standards Donors met the following criteria:
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 106/kg.
|
Recipients, Reduced Intensity
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total).
|
Recipients Myeloablative Regimen
Myeloablative (one of four general regimens):
Busulfan (\> 9 mg/kg po or iv total) with fludarabine Busulfan (\> 9 mg/kg po or iv total) with cyclophosphamide Total body irradiation (\> 1000 cGy) plus etoposide Total body irradiation (\> 500 cGy) plus cyclophosphamide
|
|---|---|---|---|
|
Overall Study
STARTED
|
64
|
33
|
30
|
|
Overall Study
COMPLETED
|
64
|
33
|
30
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous Plerixafor
Baseline characteristics by cohort
| Measure |
Related Donors Receiving Plerixafor
n=64 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Myeloablative Conditioning Regimen
n=30 Participants
Myeloablative (one of four general regimens):
Busulfan (\> 9 mg/kg po or iv total) with fludarabine Busulfan (\> 9 mg/kg po or iv total) with cyclophosphamide Total body irradiation (\> 1000 cGy) plus etoposide Total body irradiation (\> 500 cGy) plus cyclophosphamide
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
Total
n=127 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
1 Participants
n=483 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
61 Participants
n=93 Participants
|
30 Participants
n=4 Participants
|
30 Participants
n=27 Participants
|
121 Participants
n=483 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
5 Participants
n=483 Participants
|
|
Sex: Female, Male
Female
|
23 Participants
n=93 Participants
|
14 Participants
n=4 Participants
|
24 Participants
n=27 Participants
|
61 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
41 Participants
n=93 Participants
|
16 Participants
n=4 Participants
|
9 Participants
n=27 Participants
|
66 Participants
n=483 Participants
|
|
Region of Enrollment
United States
|
64 participants
n=93 Participants
|
30 participants
n=4 Participants
|
33 participants
n=27 Participants
|
127 participants
n=483 Participants
|
PRIMARY outcome
Timeframe: donationPopulation: Recipients were not mobilized with plerixafor.
Donor mobilization following plerixafor was considered successful if ≥ 2.0x106 CD34+ cells/kg recipient weight was collected in no more than two leukapheresis collections.
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=64 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Percentage of Donors Whose Cells Were Successfully Mobilized and Collected With a Sufficient CD34+ Cell Dose Using Plerixafor as the Mobilizing Agent, Using an Intention-to-treat Analysis.
|
63 Participants
|
—
|
SECONDARY outcome
Timeframe: baseline, Day 1, Day 2, Day 3Population: Recipients were not analyzed for this outcome measure
Incidence and severity of acute toxicities before and during apheresis experienced by donors receiving plerixafor. Acute toxicities are graded according to the NCI Common Terminology Criteria for Adverse Events, version 4.0. This outcome measure is descriptive. Grade of maximum toxicity across all time points is reported. Higher grades denote worse outcomes. 0 = None, 1 = Mild, 2 = Moderate, 3 = severe.
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=64 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Incidence and Severity of Acute Toxicities
Max Modified toxicity criteria (MTC) = 0
|
19 Participants
|
—
|
|
Incidence and Severity of Acute Toxicities
Max Modified toxicity criteria (MTC) = 1
|
34 Participants
|
—
|
|
Incidence and Severity of Acute Toxicities
Max Modified toxicity criteria (MTC) = 2
|
10 Participants
|
—
|
|
Incidence and Severity of Acute Toxicities
Max Modified toxicity criteria (MTC) = 3
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: 30 minutes, 60 minutes, 120 minutes, 240 minutes, 1 month, 6 months, 12 months post donation for each subjectPopulation: Recipients did not receive plerixafor and were not examined for this outcome.
Adverse effects experienced by donors receiving plerixafor up to one year post donation. Number of participants with a maximum MTC \>0 reported at each individual time point. Adverse effects are graded according to the NCI Common Terminology Criteria for Adverse Events, version 4.0. This outcome measure is descriptive. Participants can experience adverse effects at more than one time point evaluated. Higher grades denote worse outcomes. 0 = None, 1 = Mild, 2 = Moderate, 3 = severe.
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=64 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Adverse Effects
30 minutes after administration of plerixafor
|
23 Participants
|
—
|
|
Adverse Effects
60 minutes after administration of plerixafor
|
28 Participants
|
—
|
|
Adverse Effects
120 minutes after administration of plerixafor
|
27 Participants
|
—
|
|
Adverse Effects
240 minutes after administration of plerixafor
|
24 Participants
|
—
|
|
Adverse Effects
One month after administration of plerixafor
|
22 Participants
|
—
|
|
Adverse Effects
Six months after administration of plerixafor
|
7 Participants
|
—
|
|
Adverse Effects
Twelve months after administration of plerixafor
|
7 Participants
|
—
|
SECONDARY outcome
Timeframe: Day +1 through neutrophil recovery or Day 21 (whichever is first)Population: Patients were classified to one of the two conditioning regimens - myeloablative (MAC) or reduced intensity conditioning (RIC). A total of 30 MAC and 33 RIC patients were analyzed for this outcome. Neutrophil engraftment was defined as time from day 0 of HCT to the first of 3 consecutive measurements of absolute neutrophil count ≥0.5 × 109/L. Platelet engraftment was defined as the number of days from day 0 of HCT to the first of 3 consecutive values of platelet count ≥20 × 109/L.
Incidence of and kinetics of neutrophil and platelet recovery by day 100 in recipients after transplantation of hematopoietic cells mobilized with plerixafor.
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Incidence of and Kinetics of Neutrophil and Platelet Recovery After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Neutrophil engraftment
|
100 Percentage probability of engraftment
Interval 100.0 to 100.0
|
100 Percentage probability of engraftment
Interval 100.0 to 100.0
|
|
Incidence of and Kinetics of Neutrophil and Platelet Recovery After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Platelet engraftment
|
100 Percentage probability of engraftment
Interval 100.0 to 100.0
|
97 Percentage probability of engraftment
Interval 97.0 to 100.0
|
SECONDARY outcome
Timeframe: Chimerism was evaluated at serial timepoints post HCT in patients in both RIC and MAC strata. Chimerism was assessed at Day +28, +100, +180, and +365Population: Chimerism is not evaluated in donor arm.
T-cell (CD3+) and myeloid (CD33+) chimerism in recipients after transplantation of hematopoietic cells mobilized with plerixafor. This outcome measure is descriptive.
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
T-cell chimerism (Day 100)
|
97 percentage of donor cell chimerism
Interval 76.0 to 100.0
|
84 percentage of donor cell chimerism
Interval 64.0 to 100.0
|
|
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Myeloid chimerism (Day 180)
|
100 percentage of donor cell chimerism
Interval 89.0 to 100.0
|
100 percentage of donor cell chimerism
Interval 5.0 to 100.0
|
|
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
T-cell chimerism (Day 180)
|
100 percentage of donor cell chimerism
Interval 91.0 to 100.0
|
95 percentage of donor cell chimerism
Interval 5.0 to 100.0
|
|
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
T-cell chimerism (Day 365)
|
100 percentage of donor cell chimerism
Interval 100.0 to 100.0
|
100 percentage of donor cell chimerism
Interval 0.0 to 100.0
|
|
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Myeloid chimerism (Day 28)
|
100 percentage of donor cell chimerism
Interval 98.0 to 100.0
|
100 percentage of donor cell chimerism
Interval 91.0 to 100.0
|
|
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
T-cell chimerism (Day 28)
|
92 percentage of donor cell chimerism
Interval 59.0 to 100.0
|
80 percentage of donor cell chimerism
Interval 50.0 to 100.0
|
|
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Myeloid chimerism (Day 100)
|
99 percentage of donor cell chimerism
Interval 91.0 to 100.0
|
100 percentage of donor cell chimerism
Interval 17.0 to 100.0
|
|
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Myeloid chimerism (Day 365)
|
100 percentage of donor cell chimerism
Interval 100.0 to 100.0
|
100 percentage of donor cell chimerism
Interval 0.0 to 100.0
|
SECONDARY outcome
Timeframe: Day 28Population: Donor population did not receive a transplant
Incidence of primary graft failure in recipients after transplantation of hematopoietic cells mobilized with plerixafor. This outcome measure is descriptive.
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Primary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 100Population: Donor arm did not receive a transplant. This group was not analyzed for graft-vs-host disease
Incidence of acute graft-versus-host disease (GVHD) in recipients after transplantation of hematopoietic cells mobilized with plerixafor
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Incidence of Acute Graft-versus-host Disease (GVHD)
Grade 2-4 aGVHD by day 100
|
53 Percentage of participants
Interval 35.0 to 71.0
|
18 Percentage of participants
Interval 7.0 to 33.0
|
|
Incidence of Acute Graft-versus-host Disease (GVHD)
Grade 3-4 aGVHD by day 100
|
17 Percentage of participants
Interval 6.0 to 32.0
|
3 Percentage of participants
Interval 0.0 to 12.0
|
SECONDARY outcome
Timeframe: Day 28, 100, 180, 365Population: Donor population did not receive a transplant. Immune reconstitution was not measured in the donor population.
Rate and quality of immune reconstitution as evidenced by peripheral blood immunophenotype after transplantation of hematopoietic cells mobilized with plerixafor.
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Immune Reconstitution
CD3+ (Day 28)
|
589 Cells*10^3/mm^3
Interval 48.0 to 2250.0
|
478 Cells*10^3/mm^3
Interval 49.0 to 1485.0
|
|
Immune Reconstitution
CD4+ (Day 28)
|
363 Cells*10^3/mm^3
Interval 23.0 to 1364.0
|
214 Cells*10^3/mm^3
Interval 28.0 to 676.0
|
|
Immune Reconstitution
CD8+ (Day 28)
|
197 Cells*10^3/mm^3
Interval 19.0 to 837.0
|
127 Cells*10^3/mm^3
Interval 8.0 to 918.0
|
|
Immune Reconstitution
CD3+ (Day100)
|
647 Cells*10^3/mm^3
Interval 61.0 to 1671.0
|
529 Cells*10^3/mm^3
Interval 40.0 to 1387.0
|
|
Immune Reconstitution
CD4+ (Day 180)
|
352 Cells*10^3/mm^3
Interval 16.0 to 851.0
|
257 Cells*10^3/mm^3
Interval 18.0 to 769.0
|
|
Immune Reconstitution
CD8+ (Day 180)
|
272 Cells*10^3/mm^3
Interval 26.0 to 1080.0
|
219 Cells*10^3/mm^3
Interval 37.0 to 862.0
|
|
Immune Reconstitution
CD3+ (Day 365)
|
794 Cells*10^3/mm^3
Interval 62.0 to 1780.0
|
1094 Cells*10^3/mm^3
Interval 207.0 to 2448.0
|
|
Immune Reconstitution
CD4+ (Day 365)
|
495 Cells*10^3/mm^3
Interval 27.0 to 932.0
|
457 Cells*10^3/mm^3
Interval 92.0 to 775.0
|
|
Immune Reconstitution
CD8+ (Day 365)
|
264 Cells*10^3/mm^3
Interval 25.0 to 963.0
|
460 Cells*10^3/mm^3
Interval 106.0 to 1608.0
|
|
Immune Reconstitution
CD4+ (Day 100)
|
339 Cells*10^3/mm^3
Interval 20.0 to 714.0
|
265 Cells*10^3/mm^3
Interval 25.0 to 650.0
|
|
Immune Reconstitution
CD8+ (Day 100)
|
242 Cells*10^3/mm^3
Interval 12.0 to 1069.0
|
162 Cells*10^3/mm^3
Interval 13.0 to 998.0
|
|
Immune Reconstitution
CD3+ (Day 180)
|
638 Cells*10^3/mm^3
Interval 58.0 to 1964.0
|
699 Cells*10^3/mm^3
Interval 69.0 to 1517.0
|
SECONDARY outcome
Timeframe: day 365Population: Donor population did not receive a transplant. Only those with prior infection were eligible to be analyzed for this outcome measure
Percentage of recipients with prior CMV infection whose CMV was reactivated after transplantation with cell mobilized with plerixafor
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=15 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=26 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Incidence of Cytomegalovirus (CMV) Reactivation After Transplantation With Cells Mobilized With Plerixafor.
|
3 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: Day 180, 365Population: Related donors did not receive a transplant and were not examined for this outcome.
Incidence of treatment-related mortality and disease relapse/progression in recipients after transplantation of hematopoietic cells mobilized with plerixafor
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Treatment-related Mortality and Disease Relapse/Progression
Treatment-related mortality (180 days)
|
7 Percentage probability of outcome
Interval 1.0 to 18.0
|
3 Percentage probability of outcome
Interval 0.0 to 12.0
|
|
Treatment-related Mortality and Disease Relapse/Progression
Treatment-related mortality (365 days)
|
17 Percentage probability of outcome
Interval 6.0 to 32.0
|
6 Percentage probability of outcome
Interval 1.0 to 17.0
|
|
Treatment-related Mortality and Disease Relapse/Progression
Relapse/progression of disease (180 days)
|
30 Percentage probability of outcome
Interval 15.0 to 47.0
|
28 Percentage probability of outcome
Interval 14.0 to 45.0
|
|
Treatment-related Mortality and Disease Relapse/Progression
Relapse/progression of disease (365 days)
|
30 Percentage probability of outcome
Interval 15.0 to 48.0
|
30 Percentage probability of outcome
Interval 16.0 to 47.0
|
SECONDARY outcome
Timeframe: Day 180, 365Population: Donor population did not receive a transplantation and therefore was not analyzed for this outcome
Probability of progression-free and overall survival after transplantation of hematopoietic cells mobilized with plerixafor
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Progression-free and Overall Survival
Progression free survival (180 days)
|
63 Percentage probability of outcome
Interval 46.0 to 79.0
|
69 Percentage probability of outcome
Interval 52.0 to 83.0
|
|
Progression-free and Overall Survival
Progression free survival (365 days)
|
53 Percentage probability of outcome
Interval 36.0 to 71.0
|
64 Percentage probability of outcome
Interval 47.0 to 79.0
|
|
Progression-free and Overall Survival
Overall survival (180 days)
|
87 Percentage probability of outcome
Interval 72.0 to 96.0
|
79 Percentage probability of outcome
Interval 63.0 to 91.0
|
|
Progression-free and Overall Survival
Overall survival (365 days)
|
63 Percentage probability of outcome
Interval 46.0 to 79.0
|
70 Percentage probability of outcome
Interval 53.0 to 84.0
|
SECONDARY outcome
Timeframe: donationCellular composition of allografts mobilized with plerixafor (stem/progenitor cells, T/B/ (Natural killer) NK-cells)
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=64 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Cellular Composition of Allografts
CD3+
|
5.96 cells*10^8/kg
Interval 2.72 to 13.0
|
—
|
|
Cellular Composition of Allografts
CD3+CD4+
|
3.57 cells*10^8/kg
Interval 2.0 to 9.4
|
—
|
|
Cellular Composition of Allografts
CD3+CD8+
|
2.18 cells*10^8/kg
Interval 0.48 to 5.0
|
—
|
|
Cellular Composition of Allografts
CD19+
|
1.47 cells*10^8/kg
Interval 0.23 to 7.43
|
—
|
|
Cellular Composition of Allografts
CD56+
|
0.38 cells*10^8/kg
Interval 0.0 to 1.1
|
—
|
SECONDARY outcome
Timeframe: Day 365Population: Donor arm did not receive a transplant. This group was not analyzed for graft-versus-host disease.
Incidence of chronic graft-versus-host disease (GVHD) in recipients after transplantation of hematopoietic cells mobilized with plerixafor
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Incidence of Chronic Graft-versus-host Disease (GVHD)
|
52 Percentage of participants
Interval 33.0 to 70.0
|
39 Percentage of participants
Interval 23.0 to 57.0
|
SECONDARY outcome
Timeframe: Day 365Population: Donor population did not receive a transplant.
Incidence of secondary graft failure in recipients after transplantation of hematopoietic cells mobilized with plerixafor. This outcome measure is descriptive.
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=30 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
n=33 Participants
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
Secondary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: donationCD34+ cell count of allografts mobilized with plerixafor
Outcome measures
| Measure |
Related Donors Receiving Plerixafor
n=64 Participants
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Eligible donors determined according to institutional standards
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 10E6/kg.
|
Recipients, Reduced Intensity Conditioning Regimen
Reduced Intensity (one of three general regimens):
Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
|
|---|---|---|
|
CD34+ Cell Count of Allografts
|
4.68 cells*10^6/kg
Interval 0.9 to 9.6
|
—
|
Adverse Events
Related Donors Receiving Plerixafor
Serious events: 1 serious events
Other events: 44 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Related Donors Receiving Plerixafor
n=64 participants at risk
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Donor eligibility was determined according to institutional standards Donors met the following criteria:
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 106/kg.
|
|---|---|
|
Vascular disorders
Vasovagal reaction
|
1.6%
1/64 • Number of events 1
Recipients were not followed for adverse events.
|
Other adverse events
| Measure |
Related Donors Receiving Plerixafor
n=64 participants at risk
Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent.
Donor eligibility was determined according to institutional standards Donors met the following criteria:
* 18-65 years of age
* 6/6 HLA-matched sibling
* Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
* Serum creatinine \<1.5 x institution ULN or estimated creatinine clearance (CLCR) \>50 mL/min
Treatment Description:
* Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later.
* Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 106/kg.
|
|---|---|
|
General disorders
Periorbital edema
|
1.6%
1/64
Recipients were not followed for adverse events.
|
|
General disorders
Urticaria
|
4.7%
3/64
Recipients were not followed for adverse events.
|
|
General disorders
Headache
|
14.1%
9/64
Recipients were not followed for adverse events.
|
|
General disorders
Paresthesia
|
26.6%
17/64
Recipients were not followed for adverse events.
|
|
General disorders
Dizziness
|
17.2%
11/64
Recipients were not followed for adverse events.
|
|
General disorders
Nausea
|
15.6%
10/64
Recipients were not followed for adverse events.
|
|
General disorders
Vomiting
|
4.7%
3/64
Recipients were not followed for adverse events.
|
|
General disorders
Bloating
|
23.4%
15/64
Recipients were not followed for adverse events.
|
|
General disorders
Diarrhea
|
23.4%
15/64
Recipients were not followed for adverse events.
|
|
General disorders
Flatulence
|
29.7%
19/64
Recipients were not followed for adverse events.
|
|
General disorders
Abdominal pain
|
20.3%
13/64
Recipients were not followed for adverse events.
|
|
General disorders
Arthalgia
|
1.6%
1/64
Recipients were not followed for adverse events.
|
|
Surgical and medical procedures
Injection site reaction
|
18.8%
12/64
Recipients were not followed for adverse events.
|
Additional Information
Dr. Steven Devine
National Marrow Donor Program/BeTheMatch
Phone: 7634068239
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place