Trial Outcomes & Findings for Evaluation of the Spectra Optia® Mononuclear Cell Collection Procedure (NCT NCT01292486)

Results Overview

Neutrophil recovery is defined as the day on which the peripheral blood absolute neutrophil count exceeds 500/μL (ANC500)for the first of three consecutive measurements obtained on different days following transplant of peripheral blood stem cells in patients treated with myeloablative therapy for their underlying disease. As this was a test of non-inferiority, the null hypothesis to be tested (H0) was that the difference between the observed day to neutrophil recovery and the historical median day of neutrophil recovery was greater than two days. At two of the enrolling sites, Duke and Emory Universities, the median day to ANC500 was 12, while at the other two sites, Indiana University and the University of Utah, it was 11 days. Consequently, in the equation below, site specific-historic medians were compared to the observed days to achieve ANC500. H0: D \> \|2\|, where D = Observed median day of neutrophil recovery - Site specific historic median day of neutrophil recovery.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

32 participants

Primary outcome timeframe

up to 28 days following transplant

Results posted on

2013-05-01

Participant Flow

Patients requiring a first peripheral blood stem cell collection and transplant for multiple myeloma were recruited at four clinical centers from March through October, 2011.

This was a single arm study, which evaluated Multiple Myeloma patients who received autologous stem-cell transplants collected using the Spectra Optia Apheresis System, following myeloablative therapy. The study was limited to subjects who were expected to demonstrate normal neutrophil recovery.

Participant milestones

Participant milestones
Measure	All Per Protocol Patients This was a single arm study, which evaluated Multiple Myeloma patients who received autologous stem-cell transplants collected using the Spectra Optia Apheresis System, following myeloablative therapy. The study was limited to subjects who were expected to demonstrate normal neutrophil recovery.
Overall Study STARTED	32
Overall Study COMPLETED	26
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	All Per Protocol Patients This was a single arm study, which evaluated Multiple Myeloma patients who received autologous stem-cell transplants collected using the Spectra Optia Apheresis System, following myeloablative therapy. The study was limited to subjects who were expected to demonstrate normal neutrophil recovery.
Overall Study Protocol Violation	2
Overall Study Did not meet screening criteria	2
Overall Study Adverse Event	1
Overall Study Physician Decision	1

Baseline Characteristics

Evaluation of the Spectra Optia® Mononuclear Cell Collection Procedure

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patients With Multiple Myeloma n=32 Participants Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=5 Participants
Age, Categorical >=65 years	12 Participants n=5 Participants
Age Continuous	63 years n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	23 Participants n=5 Participants
Region of Enrollment United States	32 participants n=5 Participants

PRIMARY outcome

Timeframe: up to 28 days following transplant

Population: Twenty-six patients were evaluable per protocol.

Neutrophil recovery is defined as the day on which the peripheral blood absolute neutrophil count exceeds 500/μL (ANC500)for the first of three consecutive measurements obtained on different days following transplant of peripheral blood stem cells in patients treated with myeloablative therapy for their underlying disease. As this was a test of non-inferiority, the null hypothesis to be tested (H0) was that the difference between the observed day to neutrophil recovery and the historical median day of neutrophil recovery was greater than two days. At two of the enrolling sites, Duke and Emory Universities, the median day to ANC500 was 12, while at the other two sites, Indiana University and the University of Utah, it was 11 days. Consequently, in the equation below, site specific-historic medians were compared to the observed days to achieve ANC500. H0: D \> \|2\|, where D = Observed median day of neutrophil recovery - Site specific historic median day of neutrophil recovery.

Outcome measures

Outcome measures
Measure	Patients With Multiple Myeloma n=26 Participants Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
Days Until Neutrophil Recovery Following Peripheral Blood Stem Cell Transplant Minus the Historical Median Day Until Recovery.	0 Days Interval -1.0 to 2.0

SECONDARY outcome

Timeframe: up to 28 days following transplant

Population: All per protocol patients for whom platelet recovery data was available.

The time to platelet recovery is defined as the day following stem-cell transplant (Day 0) on which the platelet count exceeds 20,000/μL, for the first of three consecutive measurements obtained on different days, without platelet transfusion support within the preceding 7 days.

Outcome measures

Outcome measures
Measure	Patients With Multiple Myeloma n=19 Participants Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
Days Until Platelet Recovery	20 days Interval 11.0 to 28.0

SECONDARY outcome

Timeframe: up to 7 days

Population: Data to calculate CD34+ cell collection efficiency was available for 39 collections on 24 of 26 per protocol patients.

Collection efficiency (CE) is defined as the percentage of any given cellular subset, processed by the system, that is collected from the subject. CD34+ is a cell surface marker found on pluripotent hematopoeitic stem cells.

Outcome measures

Outcome measures
Measure	Patients With Multiple Myeloma n=39 MNC collections Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
CD34+ Cell Collection Efficiency.	70 % of processed CD34+ cells collected Interval 39.0 to 92.0

SECONDARY outcome

Timeframe: up to 7 days

Population: Data was available to calculate MNC collection efficiency on 35 collections performed on 22 of the 26 per protocol patients.

Collection efficiency (CE) is defined as the percentage of any given cellular subset, processed by the system, that is collected from the subject. Determination of collection efficiency depends on an estimate of the average concentration of target cells in the patient's blood. Because these cells are continuously being removed during the collection, and are undergoing variable replacement from the bone marrow, this estimate will not be completely accurate. Underestimation of the concentration of target cells processed can lead to collection efficiencies of greater than 100%.

Outcome measures

Outcome measures
Measure	Patients With Multiple Myeloma n=35 MNC collections Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
Mononuclear Cel (MNC) Collection Efficiency	60 % of processed MNCs that were collected Interval 35.0 to 115.0

SECONDARY outcome

Timeframe: up to 7 days

Population: Data to calculate platelet collection efficiency was available for 38 MNC collections on 24 of the 26 per protocol patients.

Platelet contamination of the cell product was measured as the platelet collection efficiency, that is, as the percent of platelets processed that were collected.

Outcome measures

Outcome measures
Measure	Patients With Multiple Myeloma n=38 MNC collections Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
Platelet Collection Efficiency	19 % of processed platelets collected Interval 13.0 to 38.0

SECONDARY outcome

Timeframe: 7 days

Population: Data was available from 38 MNC collections from 25 of the 26 per protocol patients.

The hematocrit of the collected product was used to quantitate Red Blood Cell (RBC) contamination.

Outcome measures

Outcome measures
Measure	Patients With Multiple Myeloma n=38 MNC collections Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
Hematocrit of MNC Product	1.8 hematocrit % Interval 1.1 to 4.5

SECONDARY outcome

Timeframe: 7 days

Population: Data to calculate the percent of product WBCs that were segmented granulocytes or bands was available for 38 MNC collections on 25 of the 26 per protocol patients.

Granulocyte contamination of the MNC product was quantitated as the percent of total product White Blood Cells (WBC) that were segmented granulocytes or bands.

Outcome measures

Outcome measures
Measure	Patients With Multiple Myeloma n=25 Participants Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
Granulocyte % of MNC Product	29.0 percentage of product WBCs Interval 4.0 to 72.0

Adverse Events

Patients With Multiple Myeloma

Serious events: 2 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Patients With Multiple Myeloma n=30 participants at risk Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
Infections and infestations Fever and hypotension	3.3% 1/30 • Number of events 1 • During stem cell collection (3-6 hours) and following reinfusion of collected stem cell product until neutrophil recovery (1-28 days). Two participants were removed from the study before they underwent a collection on the Spectra Optia Apheresis System and were not at risk.
Infections and infestations Pneumonia/Sepsis	3.3% 1/30 • Number of events 1 • During stem cell collection (3-6 hours) and following reinfusion of collected stem cell product until neutrophil recovery (1-28 days). Two participants were removed from the study before they underwent a collection on the Spectra Optia Apheresis System and were not at risk.

Other adverse events

Other adverse events
Measure	Patients With Multiple Myeloma n=30 participants at risk Multiple myeloma patients requiring myeloablative therapy and a first autologous hematopoetic stem cell transplant.
General disorders Citrate Reaction	16.7% 5/30 • Number of events 5 • During stem cell collection (3-6 hours) and following reinfusion of collected stem cell product until neutrophil recovery (1-28 days). Two participants were removed from the study before they underwent a collection on the Spectra Optia Apheresis System and were not at risk.
Infections and infestations Fever	6.7% 2/30 • Number of events 2 • During stem cell collection (3-6 hours) and following reinfusion of collected stem cell product until neutrophil recovery (1-28 days). Two participants were removed from the study before they underwent a collection on the Spectra Optia Apheresis System and were not at risk.

Additional Information

Jerome R Bill, M.D.

Terumo BCT

Phone: 303-231-4729

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60