Trial Outcomes & Findings for Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators' Expression (NCT NCT03480360)
NCT ID: NCT03480360
Last Updated: 2024-07-23
Results Overview
Define 100-day survival of subjects
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE3
Target enrollment
21 participants
Primary outcome timeframe
100 days post date of peripheral blood transplant
Results posted on
2024-07-23
Participant Flow
21 individuals signed consent. 1 individual was deemed ineligible during screening. Therefore 20 individuals were evaluated for the primary endpoint.
Participant milestones
| Measure |
Johns Hopkins' Conditioning Regimen
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
18
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Johns Hopkins' Conditioning Regimen
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Overall Study
Death
|
2
|
Baseline Characteristics
Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators' Expression
Baseline characteristics by cohort
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
10 Participants
n=5 Participants
|
|
Age, Continuous
|
59.55 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=5 Participants
|
|
Number of Evaluable Participants
|
20 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 100 days post date of peripheral blood transplantDefine 100-day survival of subjects
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Survived to 100-Days Post-transplant
|
18 Participants
|
PRIMARY outcome
Timeframe: One year post date of peripheral blood transplantDefine one year survival of subjects
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Survived to One Year Post-Transplant.
|
16 Participants
|
PRIMARY outcome
Timeframe: Post-peripheral blood transplantDefine number of subjects who experience a successful engraftment: Defined as absolute neutrophil count \> 500/mm3 and platelets \> 20,000/mcl for three consecutive days (count first day as engraftment)
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Experienced a Successful Engraftment
|
18 Participants
|
PRIMARY outcome
Timeframe: 100 days post-peripheral blood transplantDefine response to treatment at 100 days post-peripheral blood transplant. The Standard International Criteria for responses for each disease will be used, based on CIBMTR (Center for International Blood and Marrow Transplant Research) criteria.
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Achieved a Response to Treatment at 100 Days
|
16 Participants
|
PRIMARY outcome
Timeframe: One year post-peripheral blood transplantDefine response to treatment at one year post-peripheral blood transplant. The Standard International Criteria for responses for each disease will be used, based on CIBMTR (Center for International Blood and Marrow Transplant Research) criteria.
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Achieved a Response to Treatment at One Year
|
13 Participants
|
PRIMARY outcome
Timeframe: Post-peripheral blood transplantDefine subjects who experienced toxicities associated with this treatment regimen
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Experienced Toxicities Associated With This Treatment Regimen
|
16 Participants
|
PRIMARY outcome
Timeframe: Post-peripheral blood transplantDefine subjects who had incidence of acute GVHD
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Had Incidence of Acute GVHD
|
3 Participants
|
PRIMARY outcome
Timeframe: Post-peripheral blood transplantDefine subjects who had incidence of chronic GVHD
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Had Incidence of Chronic GVHD
|
9 Participants
|
PRIMARY outcome
Timeframe: Days 30, 60, and 90 post-peripheral blood transplantDefine subjects who experience donor-recipient chimerism following transplant at days 30, 60 and 90. All patients were assessed for donor-recipient chimerism at days 30, 60, and 90, but only one patient experienced chimerism. Day 90 for this patient is reported.
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Experienced Donor-Recipient Chimerism Following Transplant at Days 30, 60, and 90.
|
1 Participants
|
PRIMARY outcome
Timeframe: 100 days post-peripheral blood transplantDefine subjects who experienced treatment-related mortality within the first 100 days post-peripheral blood transplant
Outcome measures
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 Participants
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Number of Participants Who Experienced Treatment-Related Mortality Within the First 100 Days
|
2 Participants
|
SECONDARY outcome
Timeframe: Days 30, 60, and 90 post-transplantPopulation: Blood samples were collected, however laboratory analysis was not performed due to lack of funding. There are no plans to potentially analyze the samples in the future, therefore these samples were not, and will not be, analyzed.
To characterize the incidence of immune checkpoint regulators (V-domain Ig Suppressor of T-cell Activation, cytotoxic T-lymphocyte-associated protein 4 \[CTLA\], Programmed cell death protein 1 \[PD-1\]) during early immune recovery following an allogeneic stem cell transplant.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Post-transplant through study completion or death, assessed up to 3 years post-transplantPopulation: Blood samples were collected, however laboratory analysis was not performed due to lack of funding. There are no plans to potentially analyze the samples in the future, therefore these samples were not, and will not be, analyzed.
In those patients experiencing GVHD, the study team will define the checkpoint regulator expression on MDSCs
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Post-transplant through study completion or death, assessed up to 3 years post-transplantPopulation: Blood samples were collected, however laboratory analysis was not performed due to lack of funding. There are no plans to potentially analyze the samples in the future, therefore these samples were not, and will not be, analyzed.
In those patients experiencing GVHD, the study team will define the peripheral blood mononuclear cells and myeloid subsets.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Post-transplant through study completion or death, assessed up to 3 years post-transplantPopulation: Blood samples were collected, however laboratory analysis was not performed due to lack of funding. There are no plans to potentially analyze the samples in the future, therefore these samples were not, and will not be, analyzed.
In those patients experiencing GVHD, the study team will define the myeloid subsets.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Post-transplant through study completion or death, assessed up to 3 years post-transplantPopulation: Blood samples were collected, however laboratory analysis was not performed due to lack of funding. There are no plans to potentially analyze the samples in the future, therefore these samples were not, and will not be, analyzed.
In those patients experiencing GVHD, the study team will define the MDSCs frequency.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Days 30, 60, and 90 post-transplantPopulation: Blood samples were collected, however laboratory analysis was not performed due to lack of funding. There are no plans to potentially analyze the samples in the future, therefore these samples were not, and will not be, analyzed.
To characterize the prevalence of immune checkpoint regulators (VISTA, CTLA-4, PD-1) during early immune recovery following an allogeneic stem cell transplant.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Days 30, 60, and 90 post-transplantPopulation: Blood samples were collected, however laboratory analysis was not performed due to lack of funding. There are no plans to potentially analyze the samples in the future, therefore these samples were not, and will not be, analyzed.
Flow cytometry will be used to characterize the function of immune checkpoint regulators (VISTA, CTLA-4, PD-1) during early immune recovery following an allogeneic stem cell transplant.
Outcome measures
Outcome data not reported
Adverse Events
Johns Hopkins' Conditioning Regimen
Serious events: 3 serious events
Other events: 16 other events
Deaths: 5 deaths
Serious adverse events
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 participants at risk
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
General disorders
Edema Limbs
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Gastrointestinal disorders
GI GVHD
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Hepatobiliary disorders
Liver GVHD
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Skin and subcutaneous tissue disorders
Skin GVHD
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Gastrointestinal disorders
Diarrhea
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Investigations
Platelet count decreased
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Nervous system disorders
Encephalopathy
|
5.0%
1/20 • Number of events 2 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
Other adverse events
| Measure |
Johns Hopkins' Conditioning Regimen
n=20 participants at risk
Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
Cyclophosphamide: 14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
Fludarabine: 30 mg/m2 daily for 5 days
Total Body Irradiation: 200 centigray (cGy) for one day (day -1)
Tacrolimus: 1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellcept: dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csf: 5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
Peripheral Blood Transplant: cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
|
|---|---|
|
Blood and lymphatic system disorders
Anorexia
|
75.0%
15/20 • Number of events 18 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
General disorders
Edema
|
20.0%
4/20 • Number of events 4 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
General disorders
Rigors
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
General disorders
Fever
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Cardiac disorders
Dysrhythmia
|
15.0%
3/20 • Number of events 4 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Gastrointestinal disorders
Diarrhea
|
25.0%
5/20 • Number of events 5 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Infections and infestations
C. Diff
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Renal and urinary disorders
Hematuria
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Immune system disorders
Cytokine release syndrome
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Nervous system disorders
Encephalopathy
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Psychiatric disorders
Delirium
|
10.0%
2/20 • Number of events 3 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Nervous system disorders
Dysesthesia
|
10.0%
2/20 • Number of events 2 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Gastrointestinal disorders
Mucositis oral
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
General disorders
Central Line
|
5.0%
1/20 • Number of events 1 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
|
Gastrointestinal disorders
Nausea
|
5.0%
1/20 • Number of events 3 • Monitoring of all acute toxicities (grade 3 or higher) occurred until the time of discharge (post-transplant). After discharge, until day 100, only serious toxicities (grade 4 or higher) were recorded. All-Cause Mortality was monitored from the time of transplant to three years post-transplant.
|
Additional Information
Kenneth Meehan, MD
Dartmouth-Hitchcock Medical Center
Phone: (603) 650-4628
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place