Trial Outcomes & Findings for Combination Chemotherapy, Bone Marrow Transplant, and Post Transplant Cyclophosphamide for Hematologic Cancer (NCT NCT00134017)

Results Overview

Percentage of participants who developed grades II-IV and grades III-IV acute GVHD. Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade I (skin involvement stages 1+ to 2+, with no liver or GI involvement), Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

142 participants

Primary outcome timeframe

Day 100

Results posted on

2018-08-31

Participant Flow

Participant milestones

Participant milestones
Measure	Bone Marrow Transplant Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Overall Study STARTED	142
Overall Study COMPLETED	142
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Combination Chemotherapy, Bone Marrow Transplant, and Post Transplant Cyclophosphamide for Hematologic Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bone Marrow Transplant n=142 Participants Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Age, Categorical <=18 years	16 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	123 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age, Continuous	47 years n=5 Participants
Sex: Female, Male Female	66 Participants n=5 Participants
Sex: Female, Male Male	76 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 100

Population: The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. GVHD data was collected on 15 additional participants for a total of 132. GVHD data was not collected on the remaining 10 participants.

Percentage of participants who developed grades II-IV and grades III-IV acute GVHD. Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade I (skin involvement stages 1+ to 2+, with no liver or GI involvement), Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+).

Outcome measures

Outcome measures
Measure	Bone Marrow Transplant n=132 Participants Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Percentage of Participants Who Develop Acute Graft-versus-host Disease (GVHD) Grades II-IV acute GVHD	49 Participants
Percentage of Participants Who Develop Acute Graft-versus-host Disease (GVHD) Grades III-IV acute GVHD	10 Participants

SECONDARY outcome

Timeframe: Up to one year

Population: The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. Engraftment data was not collected on the remaining 25 participants. Recipients of related-donor (n=78) and unrelated-donor (n=39) transplants were reported separately.

Median number of days to neutrophil and platelet engraftment.

Outcome measures

Outcome measures
Measure	Bone Marrow Transplant n=117 Participants Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Days to Engraftment Neutrophil engraftment, related donors	23 days Interval 2.0 to 100.0
Days to Engraftment Neutrophil engraftment, unrelated donors	25 days Interval 2.0 to 100.0
Days to Engraftment Platelet engraftment, related donors	31 days Interval 5.0 to 354.0
Days to Engraftment Platelet engraftment, unrelated donors	35 days Interval 5.0 to 354.0

SECONDARY outcome

Timeframe: Day 30, Day 60

Population: The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. Chimerism data was not collected on the remaining 25 participants. One participant died prior to Day 30 and was not analyzed for this outcome.

Number of patients who achieved 100% donor chimerism.

Outcome measures

Outcome measures
Measure	Bone Marrow Transplant n=116 Participants Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Chimerism Day 30	99 Participants
Chimerism Day 60	101 Participants

SECONDARY outcome

Timeframe: Day 100, 2 years

Population: The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. NRM data was not collected on the remaining 25 participants.

Percentage of participants who died for BMT-related reasons.

Outcome measures

Outcome measures
Measure	Bone Marrow Transplant n=117 Participants Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Non-relapse Mortality Day 100	6 percentage of participants Interval 2.0 to 13.0
Non-relapse Mortality 2 years	15 percentage of participants Interval 8.0 to 24.0

SECONDARY outcome

Timeframe: 2 years

Population: The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. Relapse data was not collected on the remaining 25 participants.

Percentage of participants who developed relapse or progressive disease.

Outcome measures

Outcome measures
Measure	Bone Marrow Transplant n=117 Participants Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Relapse	44 percentage of participants Interval 34.0 to 53.0

Adverse Events

Bone Marrow Transplant

Serious events: 20 serious events

Other events: 0 other events

Deaths: 66 deaths

Serious adverse events

Serious adverse events
Measure	Bone Marrow Transplant n=142 participants at risk Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis
Infections and infestations Aspergillus pneumonia	0.70% 1/142 • Number of events 1 • Up to one year Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization. Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.
Immune system disorders Graft failure	4.2% 6/142 • Number of events 6 • Up to one year Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization. Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.
Immune system disorders Graft versus host disease	0.70% 1/142 • Number of events 1 • Up to one year Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization. Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.
Immune system disorders Multiorgan failure	0.70% 1/142 • Number of events 1 • Up to one year Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization. Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.
Respiratory, thoracic and mediastinal disorders Pulmonary failure	4.9% 7/142 • Number of events 7 • Up to one year Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization. Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.
Respiratory, thoracic and mediastinal disorders Pulmonary hemorrhage	0.70% 1/142 • Number of events 1 • Up to one year Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization. Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.
Infections and infestations Septic shock	0.70% 1/142 • Number of events 1 • Up to one year Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization. Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.
Hepatobiliary disorders Veno-occlusive disease	2.8% 4/142 • Number of events 4 • Up to one year Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization. Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.

Other adverse events

Adverse event data not reported

Additional Information

Leo Luznik, MD

Johns Hopkins University

Phone: 4105027732

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place