Trial Outcomes & Findings for Pentoxifylline Therapy in Biliary Atresia (NCT NCT01774487)
NCT ID: NCT01774487
Last Updated: 2024-01-11
Results Overview
The investigators will track the serum conjugated bilirubin (CB) levels over the course of therapy in patients receiving 90 days of PTX (this laboratory test is drawn as part of routine care). Normal CB is 0.0-0.3 mg/dL, with a higher number of patients meeting this indicating a better outcome.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
17 participants
Primary outcome timeframe
12 weeks after starting therapy
Results posted on
2024-01-11
Participant Flow
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria (those who received the Kasai portoenterostomy are in Group 1 and those diagnosed too late for the Kasai portoenterostomy are in Group 2)
Participant milestones
| Measure |
Pentoxifylline - Group 1 (Treatment Starts 3-5 Days After Kasai Portoenterostomy)
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
Pentoxifylline - Group 2 (Patient Diagnosed Too Late for Kasai Portoenterostomy)
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
5
|
|
Overall Study
COMPLETED
|
8
|
1
|
|
Overall Study
NOT COMPLETED
|
4
|
4
|
Reasons for withdrawal
| Measure |
Pentoxifylline - Group 1 (Treatment Starts 3-5 Days After Kasai Portoenterostomy)
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
Pentoxifylline - Group 2 (Patient Diagnosed Too Late for Kasai Portoenterostomy)
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
|---|---|---|
|
Overall Study
Did not tolerate taste
|
4
|
4
|
Baseline Characteristics
Pentoxifylline Therapy in Biliary Atresia
Baseline characteristics by cohort
| Measure |
Pentoxifylline Group 1
n=12 Participants
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
Pentoxifylline Group 2
n=5 Participants
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
12 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
49 days
STANDARD_DEVIATION 26 • n=5 Participants
|
120 days
STANDARD_DEVIATION 30 • n=7 Participants
|
69 days
STANDARD_DEVIATION 42 • n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 weeks after starting therapyThe investigators will track the serum conjugated bilirubin (CB) levels over the course of therapy in patients receiving 90 days of PTX (this laboratory test is drawn as part of routine care). Normal CB is 0.0-0.3 mg/dL, with a higher number of patients meeting this indicating a better outcome.
Outcome measures
| Measure |
Pentoxifylline - Group 2
n=1 Participants
Infants not receiving the KP will be given PTX for 12 weeks, and platelet levels at 2 years of age will be calculated and reported as average and standard deviation.
|
Pentoxifylline - Group 1
n=8 Participants
Infants receiving the Kasai portoenterostomy (KP) will be given PTX for 12 weeks, and the number whose CB levels return to normal (0.0-0.3 mg/dL) will be calculated. Historically, \~60% of such infants have normal CB levels after 12 weeks. The study will determine whether PTX improves this percentage.
|
|---|---|---|
|
Number of Participants With Normal Serum Conjugated Bilirubin Levels 12 Weeks After Starting PTX (Pentoxifylline) Therapy
|
0 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: 12 weeks after starting therapyThe investigators will track the weight of patients over the course of therapy in patients receiving 90 days of PTX (this is recorded as part of routine clinical care). The weight will then be compared to standards to calculate a z-score. Normal weight Z-score is greater than or equal to 0, with a higher number of patients meeting this indicating a better outcome.
Outcome measures
| Measure |
Pentoxifylline - Group 2
n=1 Participants
Infants not receiving the KP will be given PTX for 12 weeks, and platelet levels at 2 years of age will be calculated and reported as average and standard deviation.
|
Pentoxifylline - Group 1
n=8 Participants
Infants receiving the Kasai portoenterostomy (KP) will be given PTX for 12 weeks, and the number whose CB levels return to normal (0.0-0.3 mg/dL) will be calculated. Historically, \~60% of such infants have normal CB levels after 12 weeks. The study will determine whether PTX improves this percentage.
|
|---|---|---|
|
Number of Participants Achieving Zero or Positive Weight Z-scores 12 Weeks After Starting PTX Therapy
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 2 years of agePopulation: No patient in Group 2 survived with their native liver to 2 years of age.
The investigators will record the ALT levels at age two years, in patients who had previously been treated with PTX therapy and still have their native liver. Range of normal values: 14-45 U/L, with a higher level indicating a worse outcome.
Outcome measures
| Measure |
Pentoxifylline - Group 2
Infants not receiving the KP will be given PTX for 12 weeks, and platelet levels at 2 years of age will be calculated and reported as average and standard deviation.
|
Pentoxifylline - Group 1
n=6 Participants
Infants receiving the Kasai portoenterostomy (KP) will be given PTX for 12 weeks, and the number whose CB levels return to normal (0.0-0.3 mg/dL) will be calculated. Historically, \~60% of such infants have normal CB levels after 12 weeks. The study will determine whether PTX improves this percentage.
|
|---|---|---|
|
Alanine Amino Transferase (ALT) Levels at 2 Years of Life
|
—
|
160 U/L
Standard Deviation 122
|
SECONDARY outcome
Timeframe: 2 years of agePopulation: No patient in Group 2 survived with their native liver to 2 years of age.
The investigators will measure spleen size by ultrasound at 2 years of age, in patients who had received PTX therapy earlier and still have their native liver. "Normal" spleen size range (10th-90th percentile) at this age is 6.4-8.6 cm, with a value exceeding this range indicating a worse outcome.
Outcome measures
| Measure |
Pentoxifylline - Group 2
Infants not receiving the KP will be given PTX for 12 weeks, and platelet levels at 2 years of age will be calculated and reported as average and standard deviation.
|
Pentoxifylline - Group 1
n=4 Participants
Infants receiving the Kasai portoenterostomy (KP) will be given PTX for 12 weeks, and the number whose CB levels return to normal (0.0-0.3 mg/dL) will be calculated. Historically, \~60% of such infants have normal CB levels after 12 weeks. The study will determine whether PTX improves this percentage.
|
|---|---|---|
|
Spleen Size at 2 Years of Age
|
—
|
10.0 cm
Standard Deviation 1.8
|
SECONDARY outcome
Timeframe: Baseline and up to two years after therapy finishesThe investigators will track time to liver transplant. The shorter time to liver transplant indicates a worse outcome.
Outcome measures
| Measure |
Pentoxifylline - Group 2
n=1 Participants
Infants not receiving the KP will be given PTX for 12 weeks, and platelet levels at 2 years of age will be calculated and reported as average and standard deviation.
|
Pentoxifylline - Group 1
n=2 Participants
Infants receiving the Kasai portoenterostomy (KP) will be given PTX for 12 weeks, and the number whose CB levels return to normal (0.0-0.3 mg/dL) will be calculated. Historically, \~60% of such infants have normal CB levels after 12 weeks. The study will determine whether PTX improves this percentage.
|
|---|---|---|
|
Time to Liver Transplant
|
273 days
Standard Deviation NA
The is only 1 patient in Group 2
|
317 days
Standard Deviation 95
|
SECONDARY outcome
Timeframe: 2 years of agePopulation: No patient in Group 2 survived with their native liver to 2 years of age.
The investigators will record platelet levels at age two years, in patients who had previously been treated with PTX therapy and still have their native liver. Scale 189-403\*10\^3 Platelets/μL, with a lower level indicating a worse outcome.
Outcome measures
| Measure |
Pentoxifylline - Group 2
Infants not receiving the KP will be given PTX for 12 weeks, and platelet levels at 2 years of age will be calculated and reported as average and standard deviation.
|
Pentoxifylline - Group 1
n=6 Participants
Infants receiving the Kasai portoenterostomy (KP) will be given PTX for 12 weeks, and the number whose CB levels return to normal (0.0-0.3 mg/dL) will be calculated. Historically, \~60% of such infants have normal CB levels after 12 weeks. The study will determine whether PTX improves this percentage.
|
|---|---|---|
|
Platelet Levels at 2 Years of Life
|
—
|
208 10^3 Platelets/μL
Standard Deviation 102
|
Adverse Events
Pentoxifylline - Group 1 (Treatment Starts 3-5 Days After Kasai Portoenterostomy)
Serious events: 6 serious events
Other events: 4 other events
Deaths: 0 deaths
Pentoxifylline - Group 2 (Patient Diagnosed Too Late for Kasai Portoenterostomy)
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pentoxifylline - Group 1 (Treatment Starts 3-5 Days After Kasai Portoenterostomy)
n=8 participants at risk
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
Pentoxifylline - Group 2 (Patient Diagnosed Too Late for Kasai Portoenterostomy)
n=1 participants at risk
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
|---|---|---|
|
Gastrointestinal disorders
Failure to thrive
|
37.5%
3/8 • Number of events 5 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
100.0%
1/1 • Number of events 5 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
|
Hepatobiliary disorders
Cholangitis
|
25.0%
2/8 • Number of events 3 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
0.00%
0/1 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
|
Hepatobiliary disorders
Ascites
|
0.00%
0/8 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
100.0%
1/1 • Number of events 2 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
|
Hepatobiliary disorders
Bile leak
|
12.5%
1/8 • Number of events 1 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
0.00%
0/1 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
Other adverse events
| Measure |
Pentoxifylline - Group 1 (Treatment Starts 3-5 Days After Kasai Portoenterostomy)
n=8 participants at risk
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
Pentoxifylline - Group 2 (Patient Diagnosed Too Late for Kasai Portoenterostomy)
n=1 participants at risk
All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days.
The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.
Pentoxifylline: 20 mg/kg/day divided in 3 doses, given orally for 90 days
|
|---|---|---|
|
Hepatobiliary disorders
ALT >2.5X baseline
|
12.5%
1/8 • Number of events 1 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
0.00%
0/1 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
|
Hepatobiliary disorders
Bc >2.5X baseline
|
0.00%
0/8 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
0.00%
0/1 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
|
Blood and lymphatic system disorders
Platelets <150,000/ul
|
37.5%
3/8 • Number of events 3 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
100.0%
1/1 • Number of events 1 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
2/8 • Number of events 2 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
100.0%
1/1 • Number of events 5 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
|
Product Issues
Stopped medication before 12 week period
|
25.0%
2/8 • Number of events 2 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
|
0.00%
0/1 • Adverse event (AE) data was collected for 12 weeks after the Kasai portoenterostomy (duration of time when patient received medication during the study). Note: Some secondary outcomes were collected at age 2 years, but AEs (included all-cause mortality) were collected for 12 weeks after Kasai portoenterostomy to capture those AEs that could possibly be related to treatment.
1. AEs related to the progression of biliary atresia (BA) include (elevated liver function tests, thrombocytopenia, ascites, cholangitis, failure to thrive, bile leak) 2. AEs related to PTX, as previously reported, include: vomiting/spitting out medications, abdominal discomfort, cough, anemia with new heart murmur, rhinorrhea, diaper rash, irritability, and temporary hearing loss. Serious AEs were any AEs requiring hospitalization.
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Additional Information
Sanjiv Harpavat
Baylor College of Medicine and Texas Children's Hospital
Phone: 832-824-2099
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place