Trial Outcomes & Findings for Immunosuppression Withdrawal for Stable Pediatric Liver Transplant Recipients (NCT NCT01638559)
NCT ID: NCT01638559
Last Updated: 2019-10-07
Results Overview
Number of participants that are operationally tolerant, defined as those who successfully withdraw from immunosuppression and maintain normal allograft status as assessed by liver biopsy and liver tests 12 months after complete immunosuppression withdrawal.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
161 participants
Primary outcome timeframe
12 Months after complete immunosuppression withdrawal
Results posted on
2019-10-07
Participant Flow
161 participants were enrolled (11 sites in the US,1 site in Canada) between August 2012 and April 2014. N=88 of the enrolled participants were eligible to initiate immunosuppression withdrawal (ISW) and the remaining N=73 participants were terminated (e.g., ineligible to proceed with ISW) based on biopsy findings or other pre-specified criteria.
Informed consent was obtained from eligible individuals who then underwent a study-mandated biopsy to determine if they were eligible to initiate immunosuppression withdrawal, based on pre-specified histologic and other criteria.
Participant milestones
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
|---|---|
|
Overall Study
STARTED
|
88
|
|
Overall Study
COMPLETED
|
85
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Subject declined to travel for biopsy
|
1
|
Baseline Characteristics
7 participants were on Cyclosporine, and 81 were on Tacrolimus for Immunosuppression, for a total of 88 analyzed participants.
Baseline characteristics by cohort
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=88 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal.
|
|---|---|
|
Age, Categorical
<=18 years
|
88 Participants
n=88 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=88 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=88 Participants
|
|
Age, Continuous
|
10.4 years
STANDARD_DEVIATION 3.4 • n=88 Participants
|
|
Sex: Female, Male
Female
|
49 Participants
n=88 Participants
|
|
Sex: Female, Male
Male
|
39 Participants
n=88 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
12 Participants
n=88 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
74 Participants
n=88 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=88 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=88 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=88 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=88 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=88 Participants
|
|
Race (NIH/OMB)
White
|
76 Participants
n=88 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=88 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=88 Participants
|
|
Region of Enrollment
Canada
|
6 participants
n=88 Participants
|
|
Region of Enrollment
United States
|
82 participants
n=88 Participants
|
|
Entry Calcineurin Inhibitor (CNI) Daily Dose
Cyclosporine
|
65.7 mg
STANDARD_DEVIATION 22.99 • n=7 Participants • 7 participants were on Cyclosporine, and 81 were on Tacrolimus for Immunosuppression, for a total of 88 analyzed participants.
|
|
Entry Calcineurin Inhibitor (CNI) Daily Dose
Tacrolimus
|
2.1 mg
STANDARD_DEVIATION 1.33 • n=81 Participants • 7 participants were on Cyclosporine, and 81 were on Tacrolimus for Immunosuppression, for a total of 88 analyzed participants.
|
|
Screening Biopsy Ishak Stage
0
|
19 Participants
n=88 Participants
|
|
Screening Biopsy Ishak Stage
1
|
57 Participants
n=88 Participants
|
|
Screening Biopsy Ishak Stage
2
|
12 Participants
n=88 Participants
|
|
Screening Child Health Related Quality of Life Scores on a Scale
All Participants - Total Generic Score
|
80.7 Quality of Life Scores on a Scale
STANDARD_DEVIATION 13.69 • n=88 Participants • Number of Participants Analyzed added for each row.
|
|
Screening Child Health Related Quality of Life Scores on a Scale
All Participants - Total Fatigue Score
|
74.9 Quality of Life Scores on a Scale
STANDARD_DEVIATION 17.75 • n=88 Participants • Number of Participants Analyzed added for each row.
|
|
Screening Child Health Related Quality of Life Scores on a Scale
All Participants - Total Transplant Score
|
85.6 Quality of Life Scores on a Scale
STANDARD_DEVIATION 9.54 • n=88 Participants • Number of Participants Analyzed added for each row.
|
|
Screening Child Health Related Quality of Life Scores on a Scale
Tolerant Participants - Total Generic Score
|
80.7 Quality of Life Scores on a Scale
STANDARD_DEVIATION 14.59 • n=33 Participants • Number of Participants Analyzed added for each row.
|
|
Screening Child Health Related Quality of Life Scores on a Scale
Tolerant Participants - Total Fatigue Score
|
75.6 Quality of Life Scores on a Scale
STANDARD_DEVIATION 15.82 • n=33 Participants • Number of Participants Analyzed added for each row.
|
|
Screening Child Health Related Quality of Life Scores on a Scale
Tolerant Participants - Total Transplant Score
|
86.0 Quality of Life Scores on a Scale
STANDARD_DEVIATION 10.16 • n=33 Participants • Number of Participants Analyzed added for each row.
|
|
Screening Child Health Related Quality of Life Scores on a Scale
Non-Tolerant Participants - Total Generic Score
|
80.6 Quality of Life Scores on a Scale
STANDARD_DEVIATION 13.26 • n=55 Participants • Number of Participants Analyzed added for each row.
|
|
Screening Child Health Related Quality of Life Scores on a Scale
Non-Tolerant Participants - Total Fatigue Score
|
74.5 Quality of Life Scores on a Scale
STANDARD_DEVIATION 18.94 • n=55 Participants • Number of Participants Analyzed added for each row.
|
|
Screening Child Health Related Quality of Life Scores on a Scale
Non-Tolerant Participants - Total Transplant Score
|
85.4 Quality of Life Scores on a Scale
STANDARD_DEVIATION 9.27 • n=55 Participants • Number of Participants Analyzed added for each row.
|
PRIMARY outcome
Timeframe: 12 Months after complete immunosuppression withdrawalPopulation: Intent-to-Treat
Number of participants that are operationally tolerant, defined as those who successfully withdraw from immunosuppression and maintain normal allograft status as assessed by liver biopsy and liver tests 12 months after complete immunosuppression withdrawal.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=88 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Number of Operationally Tolerant Participants
|
33 Participants
|
—
|
SECONDARY outcome
Timeframe: Time from immunosuppression withdrawal through a minimum of 36 months and a maximum of 48 months of follow-upPopulation: Intent-to-Treat
This composite endpoint is comprised of clinical complications related to immunosuppression withdrawal and is defined as the occurrence of any of the following: death or graft loss, histologic evidence of refractory acute rejection or biopsy confirmed chronic rejection (CR).
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=88 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Number of Participants With Clinical Complications Usually Attributed to Immunosuppression
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Time from immunosuppression withdrawal through a minimum of 36 months and maximum of 48 months of follow-upPopulation: Participants that either restarted immunosuppression or increased their dose of immunosuppression
The median time (in days) from start of withdrawal from immunosuppression drugs to increasing or re-starting immunosuppression.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=50 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Time to Increased Immunosuppression or Re-Initiation of Immunosuppression
|
204 days
Interval 167.0 to 246.0
|
—
|
SECONDARY outcome
Timeframe: Time from immunosuppression withdrawal through a minimum of 36 months and maximum of 48 months of follow-upPopulation: Intent-to-Treat
The median time (in weeks) from biopsy proven rejection to resolution of rejection defined as both liver function tests Alanine Aminotransferase (ALT) and Gamma-Glutamyl Transferase (GGT) returning to ≤ 1.5 the baseline values.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=35 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Time to Resolution of Rejection
|
13 Weeks
Interval 8.4 to 16.6
|
—
|
SECONDARY outcome
Timeframe: Time from immunosuppression withdrawal through a minimum of 36 months and maximum of 48 months of follow-upPopulation: Intent-to-Treat
Number of biopsies that were diagnosed as histologic acute rejection in participants who initiated immunosuppression withdrawal by severity of rejection episode. Rejection severity (mild, moderate, severe) is based on the Banff global assessment grade according to the central pathology reading of the liver biopsy. Mild severity criteria: rejection infiltrate in a minority of triads that is generally mild and confined within the portal spaces. Moderate rejection criteria: rejection infiltrate expanding most or all of the triads. Severe rejection criteria: rejection infiltrate expanding most or all of the triads with spillover into periportal areas and moderate to severe perivenular inflammation that extends into the hepatic parenchyma and is associated with perivenular hepatocyte necrosis. BPAR: biopsy-proven acute rejection.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=88 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Number and Severity of Biopsies Read as Histologic Acute Rejection
Mild
|
43 Biopsies Diagnosed as BPAR
|
—
|
|
Number and Severity of Biopsies Read as Histologic Acute Rejection
Moderate
|
7 Biopsies Diagnosed as BPAR
|
—
|
|
Number and Severity of Biopsies Read as Histologic Acute Rejection
Severe
|
0 Biopsies Diagnosed as BPAR
|
—
|
SECONDARY outcome
Timeframe: Time from immunosuppression withdrawal through a minimum of 36 months and maximum of 48 months of follow-upPopulation: Participants who experienced rejection (biopsy-proven or clinical)
The clinical severity of acute rejection was descriptively analyzed using hierarchical categories, as follows: * Dose increase: Increase in IS dose and/or frequency but to a level less than the regimen at study entry, prior to initiating ISW * Reinstitution: Returning to the regimen at study entry, prior to ISW * Intensification: Increased IS dose compared with the dose at study entry, prior to ISW * Conversion: Change to different IS drug * Addition: Initiation of a second IS drug; * Corticosteroids: Administration of any intravenous or oral corticosteroids * Antibody (Ab) treatment: Administration of any rabbit thymoglobulin; usually with corticosteroids
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=88 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Clinical Severity of Acute Rejection
Dose increase
|
0.068 Proportion
Interval 0.025 to 0.143
|
—
|
|
Clinical Severity of Acute Rejection
Reinstitution
|
0.261 Proportion
Interval 0.173 to 0.366
|
—
|
|
Clinical Severity of Acute Rejection
Intensification
|
0.227 Proportion
Interval 0.145 to 0.329
|
—
|
|
Clinical Severity of Acute Rejection
Conversion
|
0.011 Proportion
Interval 0.0 to 0.062
|
—
|
|
Clinical Severity of Acute Rejection
Addition
|
0.023 Proportion
Interval 0.0 to 0.08
|
—
|
|
Clinical Severity of Acute Rejection
Corticosteroids
|
0.364 Proportion
Interval 0.264 to 0.473
|
—
|
|
Clinical Severity of Acute Rejection
Ab treatment
|
0 Proportion
Interval 0.0 to 0.041
|
—
|
SECONDARY outcome
Timeframe: Time from start of immunosuppression withdrawal through discontinuation of withdrawal, a maximum of 52 weeksPopulation: Participants who failed immunosuppression withdrawal.
Reasons participants discontinued immunosuppression withdrawal, such as Biopsy Proven Acute Rejection, Chronic Rejection, Clinical Rejection, Death, Pregnancy, etc.). Only the root cause for discontinuation for each subject is presented in these results if multiple events led to discontinuation of immunosuppression withdrawal.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=33 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Reason for Discontinuation of Withdrawal
Biopsy Proven Acute Rejection
|
30 Participants
|
—
|
|
Reason for Discontinuation of Withdrawal
Clinical Rejection
|
3 Participants
|
—
|
SECONDARY outcome
Timeframe: Time from screening biopsy to end of study (month 48) biopsyPopulation: Intent-to-Treat -Of the original 88 participants, 3 participants did not finish the study and 1 participant did not complete the final liver biopsy.
The impact of ISW on allograft fibrosis using the Ishak scoring system to measure the change in fibrosis from the screening liver biopsy to the end-of-study (month-48) liver biopsy. In the Ishak histologic scoring system, the higher the score/stage, the more fibrosis: Scores range from 0 to 6, with 6 representing the most fibrosis: 0=No fibrosis; 1=Fibrous expansion of some portal areas, with or without short fibrous septa; 2=Fibrous expansion of most portal areas, with or without short fibrous septa; 3=Fibrous expansion of most portal areas, with occasional portal to portal bridging; 4=Fibrous expansion of portal areas with marked bridging (portal to portal) as well as portal to central; 5=Marked bridging (portal to portal and/or portal to central) with occasional nodules (incomplete cirrhosis); and 6=Cirrhosis, probable or definite. Decrease in score from screening (baseline) indicates improvement
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=84 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Impact of Immunosuppression Withdrawal (ISW) on Allograft Histology
Ishak Score Change of -1
|
18 Participants
|
—
|
|
Impact of Immunosuppression Withdrawal (ISW) on Allograft Histology
Ishak Score Change of 0
|
43 Participants
|
—
|
|
Impact of Immunosuppression Withdrawal (ISW) on Allograft Histology
Ishak Score Change of 1
|
19 Participants
|
—
|
|
Impact of Immunosuppression Withdrawal (ISW) on Allograft Histology
Ishak Score Change of 2
|
3 Participants
|
—
|
|
Impact of Immunosuppression Withdrawal (ISW) on Allograft Histology
Ishak Score Change of 3
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Time from immunosuppression withdrawal through a minimum of 36 months and a maximum of 48 months of follow-upPopulation: Participants that Completed Withdrawal and were Operationally Tolerant
Median participant duration of operational tolerance. Duration of operational tolerance is defined as the number of days that participants are not taking immunosuppression medications.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=33 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Duration of Operational Tolerance
|
1209.5 days
Interval 1205.0 to 1214.0
|
—
|
SECONDARY outcome
Timeframe: Time from starting immunosuppression withdrawal until immunosuppression withdrawal failure, maximum 52 weeksPopulation: Intent-to-Treat who failed immunosuppression withdrawal
The mean percent of immunosuppression (IS) dose reduction from baseline to the time of immunosuppression withdrawal failure. Immunosuppression withdrawal failure is defined as any incidence of increasing immunosuppression medications instead of completing withdrawal.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=33 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Change in Immunosuppression Medication (Calcineurin Inhibitor) Dose From Start of Immunosuppression Withdrawal to the Time of Immunosuppression Withdrawal Failure
|
-76.1 percentage of dose
Interval -80.59 to -71.63
|
—
|
SECONDARY outcome
Timeframe: Time from immunosuppression withdrawal through a minimum of 36 months and maximum of 48 months of follow-upPopulation: Intent-to-Treat participants who were not operationally tolerant and who remained on the same medication throughout the study. Three subjects that were not operationally tolerant converted to alternate immunosuppression medications.
Change of immunosuppression (IS) dose from baseline to end of study for all participants not deemed tolerant by the trial definition either due to discontinuing IS withdrawal or completing withdrawal but not meeting the criteria for tolerance on the primary endpoint biopsy assessment.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=52 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
Participants who were not operationally tolerant.
|
|---|---|---|
|
Change in Immunosuppression Medication Dose From Study Initiation of Withdrawal to the End of the Study
|
0.4 percentage of dose
Interval -21.12 to 21.98
|
—
|
SECONDARY outcome
Timeframe: Time from immunosuppression withdrawal through a minimum of 36 months and maximum of 48 months of follow-upPopulation: Intent-to-Treat
Health related quality of life was measured by the PedsQL 4.0 Generic Core scale, the Multidimensional Fatigue scale, and the PedsQL 3.0 Transplant module. Change was calculated as the difference between the questionnaire completed at the initiation of withdrawal and at month 36 for the total generic score, the total fatigue score, and total transplant score. This change was calculated separately for tolerant and non-tolerant subjects. Each score ranges from 0-100, with a higher score indicating a better quality of life.
Outcome measures
| Measure |
Participants That Initiated Immunosuppression Withdrawal (ISW)
n=33 Participants
Pediatric liver transplant recipients with stable liver tests (ALT and GGT), no evidence of rejection in the preceding 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal..
|
Participants Who Were Not Operationally Tolerant
n=55 Participants
Participants who were not operationally tolerant.
|
|---|---|---|
|
Change in Child Health Related Quality of Life Scores Between Tolerant and Non-tolerant Subjects
Total Generic Score
|
3.4 Quality of Life Scores on a Scale
Interval -0.8 to 7.7
|
1.2 Quality of Life Scores on a Scale
Interval -2.1 to 4.5
|
|
Change in Child Health Related Quality of Life Scores Between Tolerant and Non-tolerant Subjects
Total Fatigue Score
|
5.0 Quality of Life Scores on a Scale
Interval 1.2 to 8.8
|
2.0 Quality of Life Scores on a Scale
Interval -3.9 to 7.8
|
|
Change in Child Health Related Quality of Life Scores Between Tolerant and Non-tolerant Subjects
Total Transplant Score
|
5.7 Quality of Life Scores on a Scale
Interval 1.7 to 9.8
|
0.9 Quality of Life Scores on a Scale
Interval -2.5 to 4.2
|
Adverse Events
All Enrolled Participants
Serious events: 23 serious events
Other events: 87 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Enrolled Participants
n=161 participants at risk
Pediatric liver transplant recipients with stable liver function tests, no evidence of rejection in the past 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
0.62%
1/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Gastrointestinal disorders
Intestinal obstruction
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Hepatobiliary disorders
Bile duct stenosis
|
1.2%
2/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Hepatobiliary disorders
Cholangitis
|
1.2%
2/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Immune system disorders
Transplant rejection
|
1.9%
3/161 • Number of events 4 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Cellulitis
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Gastroenteritis
|
1.2%
2/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Gastroenteritis viral
|
1.2%
2/161 • Number of events 5 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Pharyngitis
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Pneumonia
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Post procedural cellulitis
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Varicella
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Viral infection
|
1.2%
2/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Injury, poisoning and procedural complications
Post procedural bile leak
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
1.2%
2/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Injury, poisoning and procedural complications
Ulna fracture
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Investigations
Liver function test abnormal
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Nervous system disorders
Grand mal convulsion
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Psychiatric disorders
Aggression
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Psychiatric disorders
Depression
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Psychiatric disorders
Major depression
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Psychiatric disorders
Oppositional defiant disorder
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Psychiatric disorders
Suicidal ideation
|
1.2%
2/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Reproductive system and breast disorders
Menorrhagia
|
1.2%
2/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
1.2%
2/161 • Number of events 2 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.62%
1/161 • Number of events 1 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
Other adverse events
| Measure |
All Enrolled Participants
n=161 participants at risk
Pediatric liver transplant recipients with stable liver function tests, no evidence of rejection in the past 2 years, and at least 4 years post-transplant, and a qualifying liver biopsy at screening underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
10.6%
17/161 • Number of events 25 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Gastrointestinal disorders
Diarrhoea
|
9.9%
16/161 • Number of events 21 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Gastrointestinal disorders
Vomiting
|
5.6%
9/161 • Number of events 12 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
General disorders
Influenza like illness
|
9.9%
16/161 • Number of events 36 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
General disorders
Pyrexia
|
8.7%
14/161 • Number of events 19 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Immune system disorders
Hypersensitivity
|
6.2%
10/161 • Number of events 11 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Immune system disorders
Transplant rejection
|
23.0%
37/161 • Number of events 43 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Ear infection
|
8.1%
13/161 • Number of events 26 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Gastroenteritis
|
8.7%
14/161 • Number of events 17 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Gastroenteritis viral
|
13.0%
21/161 • Number of events 28 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Influenza
|
8.7%
14/161 • Number of events 16 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Nasopharyngitis
|
21.7%
35/161 • Number of events 140 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Pharyngitis streptococcal
|
9.9%
16/161 • Number of events 20 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Sinusitis
|
7.5%
12/161 • Number of events 24 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Upper respiratory tract infection
|
13.0%
21/161 • Number of events 36 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Infections and infestations
Viral infection
|
11.8%
19/161 • Number of events 39 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Investigations
Liver function test abnormal
|
14.9%
24/161 • Number of events 39 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Nervous system disorders
Headache
|
10.6%
17/161 • Number of events 37 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.3%
15/161 • Number of events 21 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.2%
10/161 • Number of events 10 • Time of enrollment through end of study participation (e.g., up to 48 months).
Safety tables include N=161 participants,inclusive of those that were not eligible (N=73) and eligible (N=88) to proceed with immunosuppression withdrawal (ISW): * Adverse events (AEs) were collected from the time of enrollment (i.e., assessment for eligibility to initiate immunosuppression withdrawal \[ISW\]) through the end of study participation; * Prior to initiating ISW, only AEs temporally related to study procedures were collected; and, * All AEs were collected ≥initiation of ISW.
|
Additional Information
Director, Clinical Research Operations Program
DAIT/NIAID
Phone: 301-594-7669
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place