Trial Outcomes & Findings for Trial of Two Stem Cell Doses To Reduce Transplant Induced Symptom Burden (NCT NCT00651937)

Results Overview

MD Anderson Symptom Inventory (MDASI) Scale regularly administered during the first year following transplantation. This instrument is brief, easily understood, and provides a measure of the intensities of cancer-related symptoms. Participants rate the intensity of physical, affective, and cognitive symptoms on 0 to 10 numeric scales from "not present" (score of 0) to "as bad as you can imagine" (score of 10). Participants also rate amount of interference with daily activities caused by symptoms on 0 to 10 numeric scales from "did not interfere" (Score of 0) to interfered completely (score of 10).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

80 participants

Primary outcome timeframe

The first 7 days post-transplant.

Results posted on

2020-01-14

Participant Flow

Recruitment Period: March 13, 2008 to June 5, 2013. All recruitment done at The University of Texas MD Anderson Cancer Center.

Participant milestones

Participant milestones
Measure	Standard Dose Stem Cell Group Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive either a standard (4-6×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.	High Dose Stem Cell Group Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive high dose (10-15×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.
Overall Study STARTED	39	41
Overall Study COMPLETED	35	40
Overall Study NOT COMPLETED	4	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Standard Dose Stem Cell Group Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive either a standard (4-6×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.	High Dose Stem Cell Group Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive high dose (10-15×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.
Overall Study Withdrawal by Subject	4	1

Baseline Characteristics

Trial of Two Stem Cell Doses To Reduce Transplant Induced Symptom Burden

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Standard Dose Stem Cell Group n=39 Participants Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive either a standard (4-6×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.	High Dose Stem Cell Group n=41 Participants Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive high dose (10-15×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.	Total n=80 Participants Total of all reporting groups
Age, Continuous	64 years n=5 Participants	65 years n=7 Participants	65 years n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants	12 Participants n=7 Participants	25 Participants n=5 Participants
Sex: Female, Male Male	26 Participants n=5 Participants	29 Participants n=7 Participants	55 Participants n=5 Participants
Region of Enrollment United States	39 participants n=5 Participants	41 participants n=7 Participants	80 participants n=5 Participants

PRIMARY outcome

Timeframe: The first 7 days post-transplant.

Population: Intention to treat analysis, all treated participants. One participant in High Dose group did not complete adequate number of MDASI's for analysis.

MD Anderson Symptom Inventory (MDASI) Scale regularly administered during the first year following transplantation. This instrument is brief, easily understood, and provides a measure of the intensities of cancer-related symptoms. Participants rate the intensity of physical, affective, and cognitive symptoms on 0 to 10 numeric scales from "not present" (score of 0) to "as bad as you can imagine" (score of 10). Participants also rate amount of interference with daily activities caused by symptoms on 0 to 10 numeric scales from "did not interfere" (Score of 0) to interfered completely (score of 10).

Outcome measures

Outcome measures
Measure	Standard Dose Stem Cell Group n=39 Participants Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive either a standard (4-6×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.	High Dose Stem Cell Group n=41 Participants Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive high dose (10-15×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.
Mean Symptom Severity Burden as Measured by MDASI Scores	4.27 score on a scale Standard Deviation 2.372	4.26 score on a scale Standard Deviation 2.264

PRIMARY outcome

Timeframe: 28 day course of ASCT

Population: Intention to treat analysis, all treated participants. One participant in High Dose group did not complete adequate number of MDSAIs for analysis.

MD Anderson Symptom Inventory (MDASI) Scale regularly administered during the first year following transplantation. This instrument is brief, easily understood, and provides a measure of the intensities of cancer-related symptoms. Participants rate the intensity of physical, affective, and cognitive on 0 to 10 numeric scales from "not present" (score of 0) to "as bad as you can imagine" (score of 10). Participants also rate amount of interference with daily activities caused by symptoms on 0 to 10 numeric scales from "did not interfere" (Score of 0) to interfered completely (score of 10)

Outcome measures

Outcome measures
Measure	Standard Dose Stem Cell Group n=35 Participants Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive either a standard (4-6×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.	High Dose Stem Cell Group n=40 Participants Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive high dose (10-15×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.
Mean Symptom Severity Burden as Measured by MDASI Scores	228.4 score on a scale Standard Deviation 191.0	223.0 score on a scale Standard Deviation 94.0

Adverse Events

Standard Dose Stem Cell Group

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

High Dose Stem Cell Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Standard Dose Stem Cell Group n=35 participants at risk Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive either a standard (4-6×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.	High Dose Stem Cell Group n=40 participants at risk Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive high dose (10-15×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.
Immune system disorders Graft Failure	2.9% 1/35 • Number of events 1 • Adverse event collection through the last day of active treatment day 0 (stem cell infusion) up to 30 days as outlined in the treatment plan.	0.00% 0/40 • Adverse event collection through the last day of active treatment day 0 (stem cell infusion) up to 30 days as outlined in the treatment plan.

Other adverse events

Other adverse events
Measure	Standard Dose Stem Cell Group n=35 participants at risk Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive either a standard (4-6×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.	High Dose Stem Cell Group n=40 participants at risk Patients undergoing ASCT for MM who were ≥60 years old or had AL amyloidosis were randomized to receive high dose (10-15×106 cells/kg) of CD34+ cells after melphalan 200 mg/m2.
Blood and lymphatic system disorders Graft Failure	2.9% 1/35 • Number of events 1 • Adverse event collection through the last day of active treatment day 0 (stem cell infusion) up to 30 days as outlined in the treatment plan.	0.00% 0/40 • Adverse event collection through the last day of active treatment day 0 (stem cell infusion) up to 30 days as outlined in the treatment plan.

Additional Information

Dr. Richard E Champlin,MD/Chair, Stem Cell Transplantation

The University of Texas (UT) MD Anderson Cancer Center

Phone: 713- 203-8632

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place