Trial Outcomes & Findings for Blood Stem Cell Transplant With Low Dose Chemotherapy for Relapsed Follicular Non-Hodgkin's Lymphoma (BMT CTN 0701) (NCT NCT00912223)

Last Updated: 2022-12-09

Results Overview

Patients are considered a failure for this endpoint if they die, or if they relapse/progress or receive anti-lymphoma therapy not including planned post-transplant radiation.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

65 participants

Primary outcome timeframe

Year 2

Results posted on

2022-12-09

Participant Flow

Participants were enrolled between April 2009 and November 2012 from 21 different transplant centers.

Participant milestones

Participant milestones
Measure	Hematopoietic Stem Cell Transplant Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Overall Study STARTED	65
Overall Study COMPLETED	62
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Hematopoietic Stem Cell Transplant Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Overall Study Withdrawal by Subject	1
Overall Study Not transplanted	1
Overall Study Transformed disease	1

Baseline Characteristics

Blood Stem Cell Transplant With Low Dose Chemotherapy for Relapsed Follicular Non-Hodgkin's Lymphoma (BMT CTN 0701)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Age, Continuous	55 years n=5 Participants
Sex: Female, Male Female	23 Participants n=5 Participants
Sex: Female, Male Male	39 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	58 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Karnofsky Performance Score 100	29 participants n=5 Participants
Karnofsky Performance Score 90	28 participants n=5 Participants
Karnofsky Performance Score 80	5 participants n=5 Participants
Comorbidity Index Score 0	31 participants n=5 Participants
Comorbidity Index Score 1-2	16 participants n=5 Participants
Comorbidity Index Score >3	15 participants n=5 Participants
Disease Status CR2	32 participants n=5 Participants
Disease Status PR1	6 participants n=5 Participants
Disease Status ≥ PR2	16 participants n=5 Participants
Disease Status Stable Disease	2 participants n=5 Participants
Disease Status Unknown	6 participants n=5 Participants
Number of Prior Chemotherapy Regimens 2	14 participants n=5 Participants
Number of Prior Chemotherapy Regimens 3	16 participants n=5 Participants
Number of Prior Chemotherapy Regimens 4	12 participants n=5 Participants
Number of Prior Chemotherapy Regimens ≥ 5	20 participants n=5 Participants
Donor Type (HLA match) Matched Related Donor (6/6 allele)	33 participants n=5 Participants
Donor Type (HLA match) Matched Unrelated Donor (8/8 allele)	29 participants n=5 Participants
Recipient Cytomegalovirus Status Positive	35 participants n=5 Participants
Recipient Cytomegalovirus Status Negative	27 participants n=5 Participants

PRIMARY outcome

Timeframe: Year 2

Patients are considered a failure for this endpoint if they die, or if they relapse/progress or receive anti-lymphoma therapy not including planned post-transplant radiation.

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Progression-Free Survival (PFS)	73 percentage of participants Interval 60.0 to 82.0

SECONDARY outcome

Timeframe: Day 30

Primary graft failure is defined as a donor peripheral blood T cell chimerism \< 5% at Day +30 post-transplant. Secondary Graft Failure is defined as documented engraftment followed by loss of graft as defined by donor peripheral blood T cell chimerism \< 5%.

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Graft Failure Primary Graft Failure	0 participants
Graft Failure Secondary Graft Failure	0 participants

SECONDARY outcome

Timeframe: Days 30 and 100

Donor engraftment is defined as \> 5% donor peripheral blood T cell chimerism by Day +30 post-transplant in the setting of Absolute Neutrophil Count (ANC) recovery (ANC \>500/mm\^3 for 3 consecutive days).

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Donor Cell Engraftment Day 30	94 percentage of donor T-cell chimerism Interval 25.0 to 100.0
Donor Cell Engraftment Day 100	96 percentage of donor T-cell chimerism Interval 25.0 to 100.0

SECONDARY outcome

Timeframe: Day 60

Neutrophil Recovery is defined as ANC \> 500/mm\^3 for 3 consecutive days.

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Time to Neutrophil Recovery	13 days Interval 8.0 to 48.0

SECONDARY outcome

Timeframe: Day 100

The event is the incidence of grades II-IV acute GVHD from day of transplant, where grade IV is worst. The first day of acute GVHD onset at a certain grade will be used to calculate a cumulative incidence curve for that acute GVHD grade. GVHD should be monitored in accordance with BMT CTN manual of procedures guidelines. Acute GVHD grading was based on the consensus conference criteria (Przepiorka, et. al., 1994) and the Center for International Blood and Marrow Transplant Research (CIBMTR) grading criteria.

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Acute Graft-versus-Host Disease (GVHD)	27 percentage of participants Interval 16.0 to 39.0

SECONDARY outcome

Timeframe: Year 2

The event is the incidence and severity of chronic GVHD from day of transplant, a cumulative incidence curve will be computed along with a 95% confidence interval at two years post-transplant. Death prior to occurrence of chronic GVHD will be considered as a competing risk.

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Chronic GVHD	61 percentage of participants Interval 48.0 to 74.0

SECONDARY outcome

Timeframe: Years 2 and 3

The event is death from any cause.

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Overall Survival 2 years	84 percentage of participants Interval 72.0 to 91.0
Overall Survival 3 years	82 percentage of participants Interval 70.0 to 90.0

SECONDARY outcome

Timeframe: Year 3

The event is death occurring in patients in continuous complete remission. The TRM distribution will be estimated by the Kaplan-Meier curve.

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Treatment-related Mortality (TRM)	16 percentage of participants Interval 8.0 to 27.0

SECONDARY outcome

Timeframe: Year 2

Population: no data collected

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Year 2

Population: No data collected

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Year 1

Quantitative immunoglobulins (IgG)

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Immunologic Reconstitution	431 mg/dL Interval 79.0 to 1430.0

SECONDARY outcome

Timeframe: Year 2

Number of participants that experiences at least one grade 3 - 5 toxicity during the first two years, where grade 5 is worst. Toxicity grades are based on the NCI CTCAE Version 3.0.

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=62 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Incidence of Toxicities	50 participants

SECONDARY outcome

Timeframe: Baseline, Days 28 and 365

Population: Serum RTX concentrations were collected at baseline (n=57), day +28 (n=56), and day +365 (n=44) for a compliance rate of 92%, 90%, and 80% at the assigned time points.

RTX concentration levels within participants

Outcome measures

Outcome measures
Measure	Hematopoietic Stem Cell Transplant n=57 Participants Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Serum Rituximab (RTX) Levels Baseline	42100 ng/mL Interval 500.0 to 70200.0
Serum Rituximab (RTX) Levels Day 28	341000 ng/mL Interval 500.0 to 670000.0
Serum Rituximab (RTX) Levels Day 365	920 ng/mL Interval 500.0 to 122000.0

Adverse Events

Hematopoietic Stem Cell Transplant

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Hematopoietic Stem Cell Transplant n=62 participants at risk Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.
Infections and infestations Appendicitis	1.6% 1/62 • Number of events 1 • 3 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Vascular disorders Deep vein thrombosis	1.6% 1/62 • Number of events 1 • 3 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.

Other adverse events

Adverse event data not reported

Additional Information

Adam Mendizabal, PhD

The Emmes Corporation

Phone: 301-251-1161

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place