Trial Outcomes & Findings for Cisplatin-Based Chemotherapy and/or Surgery in Treating Young Patients With Adrenocortical Tumor (NCT NCT00304070)

Last Updated: 2024-02-28

Results Overview

The model used for comparison will be an exponential model with a constant failure rate of 0.053 (stratum I), 0.347 (stratum II), 0.602 (stratum III and IV) per year for the first two years and 0 after that. The one-sample one-sided log-rank test comparing the observed data with the hypothesized model (Woolson, 1981) of size 0.05 will be used to assess whether the data are consistent with the target models. Since this test has independent increments, the method of Lan and DeMets will be used to derive the p-values for testing procedure.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

78 participants

Primary outcome timeframe

Up to five years after enrollment

Results posted on

2024-02-28

Participant Flow

Participant milestones

Participant milestones
Measure	Stratum 1 Stage I Disease (surgery, observation)	Stratum 2 Stage II Disease (surgery, observation)	Stratum 3 Stage III OR IV Disease (chemotherapy)
Overall Study STARTED	24	15	39
Overall Study COMPLETED	21	8	27
Overall Study NOT COMPLETED	3	7	12

Reasons for withdrawal

Reasons for withdrawal
Measure	Stratum 1 Stage I Disease (surgery, observation)	Stratum 2 Stage II Disease (surgery, observation)	Stratum 3 Stage III OR IV Disease (chemotherapy)
Overall Study Death	0	0	3
Overall Study Lost to Follow-up	0	0	1
Overall Study Physician Decision	0	0	3
Overall Study Withdrawal by Subject	0	0	1
Overall Study Progressive Disease	3	7	4

Baseline Characteristics

Cisplatin-Based Chemotherapy and/or Surgery in Treating Young Patients With Adrenocortical Tumor

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Stratum 1 n=24 Participants Stage I Disease (surgery, observation)	Stratum 2 n=15 Participants Stage II Disease (surgery, observation)	Stratum 3 n=39 Participants Stage III OR IV Disease (chemotherapy)	Total n=78 Participants Total of all reporting groups
Age, Continuous	2 years n=5 Participants	3 years n=7 Participants	7 years n=5 Participants	5 years n=4 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	10 Participants n=7 Participants	27 Participants n=5 Participants	51 Participants n=4 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	5 Participants n=7 Participants	12 Participants n=5 Participants	27 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	12 Participants n=5 Participants	7 Participants n=7 Participants	16 Participants n=5 Participants	35 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants	8 Participants n=7 Participants	20 Participants n=5 Participants	37 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	6 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	4 Participants n=4 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	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants	12 Participants n=7 Participants	28 Participants n=5 Participants	55 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	6 Participants n=5 Participants	0 Participants n=7 Participants	7 Participants n=5 Participants	13 Participants n=4 Participants
Region of Enrollment Canada	0 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants	5 participants n=4 Participants
Region of Enrollment United States	12 participants n=5 Participants	7 participants n=7 Participants	24 participants n=5 Participants	43 participants n=4 Participants
Region of Enrollment Brazil	12 participants n=5 Participants	6 participants n=7 Participants	12 participants n=5 Participants	30 participants n=4 Participants

PRIMARY outcome

Timeframe: Up to five years after enrollment

Outcome measures

Outcome measures
Measure	Stratum 1 n=24 Participants Stage I Disease (surgery, observation)	Stratum 2 n=15 Participants Stage II Disease (surgery, observation)	Stratum 3 n=39 Participants Stage III OR IV Disease (chemotherapy)
Five Year Event-free Survival (EFS)	0.86 Estimated probability five year EFS Interval 0.62 to 0.95	0.53 Estimated probability five year EFS Interval 0.26 to 0.74	0.51 Estimated probability five year EFS Interval 0.33 to 0.66

SECONDARY outcome

Timeframe: Up to 182 Days After Enrollment

The proportion of patients assigned to receive chemotherapy that experience CTC Version 4 grade 3 or higher anemia at any time during protocol therapy

Outcome measures

Outcome measures
Measure	Stratum 1 n=39 Participants Stage I Disease (surgery, observation)	Stratum 2 Stage II Disease (surgery, observation)	Stratum 3 Stage III OR IV Disease (chemotherapy)
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Allergic Reaction	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Abdominal Infection	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Abdominal Pain	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Acidosis	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Activated Partial Thromboplastin Time Prolonged	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Adrenal Insufficiency	5 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Alanine Aminotransferase Increased	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Anemia	22 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Anorexia	7 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Aspartate Aminotransferase Increased	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Blood Bilirubin Increased	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Cardiac Disorders - Other, Specify	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Catheter Related Infection	3 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Colitis	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Confusion	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Dehydration	3 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Depressed Level of Consciousness	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Diarrhea	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Dyspnea	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Enterocolitis Infectious	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Esophagitis	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Febrile Neutropenia	16 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Fever	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Gastrointestinal Disorders - Other, S	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Generalized Muscle Weakness	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of GGT Increased	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hearing Impaired	6 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Heart Failure	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hyperglycemia	3 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hyperkalemia	3 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hypertension	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hypocalcemia	3 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hypoglycemia	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hypokalemia	9 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hypomagnesemia	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hyponatremia	7 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hypophosphatemia	4 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hypotension	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Hypoxia	3 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Infections and Infestations - Other,	7 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of INR Increased	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Left Ventricular Systolic Dysfunction	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Lung Infection	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Lymphocyte Count Decreased	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Toxicity Associated with Mitotane	4 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Mucositis Oral	6 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Nausea	5 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Neutrophil Count Decreased	20 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Obstruction Gastric	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Pain	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Peripheral Motor Neuropathy	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Peripheral Sensory Neuropathy	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Pharyngitis	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Platelet Count Decreased	20 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Pneumonitis	3 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Premature Menopause	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Rash Maculo-papular	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Sepsis	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Skin Infection	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Sore Throat	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Upper Respiratory Infection	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Urinary Tract Infection	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Vascular Access Complication	2 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Ventricular Arrhythmia	1 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Vomiting	5 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of White Blood Cell Decreased	16 participants	—	—
Toxicity Associated With Chemotherapy Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 Incidence of Wound Infection	1 participants	—	—

SECONDARY outcome

Timeframe: Up to 1 month after surgery

Population: Sixty-nine eligible patients received surgery of the primary tumor site or RPLND. Complication rates were considered over the entire population regardless of Arm/Group assignment. One patient had grade 3 abdominal pain attributed to surgery.

Any patient who dies because of surgery or has a grade 3 or 4 toxicity possibly, probably or likely related to surgery will be considered as having experienced a surgical complication. The complication rate is estimated as the proportion of evaluable patients that have a complication.

Outcome measures

Outcome measures
Measure	Stratum 1 n=69 Participants Stage I Disease (surgery, observation)	Stratum 2 Stage II Disease (surgery, observation)	Stratum 3 Stage III OR IV Disease (chemotherapy)
Complications Associated With Radical Adrenalectomy and RLND	1 participants	—	—

SECONDARY outcome

Timeframe: At study enrollment

Population: Seventy-five eligible patients had tumor imaging done at the time of study enrollment and evaluated for the presence of lymph node involvement

The number eligible patients who have lymph node involvement by imaging at study enrollment.

Outcome measures

Outcome measures
Measure	Stratum 1 n=75 Participants Stage I Disease (surgery, observation)	Stratum 2 Stage II Disease (surgery, observation)	Stratum 3 Stage III OR IV Disease (chemotherapy)
Frequency of Lymph Node Involvement by Imaging.	71 Participants	—	—

SECONDARY outcome

Timeframe: At study enrollment

Population: The proportion of patients in each subpopulation are compared.This test is dependent on the number of patients from whom blood can be obtained as well as the frequency of the relevant mutation in each group (Number of patients from Brazil: 23. Number of patients not from Brazil: 31)

The proportion of patients in each subpopulation are compared.This test is dependent on the number of patients from whom blood can be obtained as well as the frequency of the relevant mutation in each group.

Outcome measures

Outcome measures
Measure	Stratum 1 n=54 Participants Stage I Disease (surgery, observation)	Stratum 2 Stage II Disease (surgery, observation)	Stratum 3 Stage III OR IV Disease (chemotherapy)
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. C229R mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. C229R mutation in Patients not from Brazil	2 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. E180K mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. E180K mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. G245C mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. G245C mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. I254T mutation in p53 in Patients from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. I254T mutation in Patients not from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. L265Q mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. L265Q mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. P47S mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. P47S mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. Q52fs mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. Q52fs mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R158L mutation in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R158L mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. G245S mutation in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. G245S mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R213P mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R213P mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R248L mutation in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R248L mutation in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R282W mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R282W mutation in p53 in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R283H mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R283H mutation in p53 in Patients not from Brazil	31 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R337H mutation in p53 in Patients from Brazil	20 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R337H mutation in p53 in Patients not from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R342X mutation in p53 in Patients from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. R342X mutation in p53 in Patients not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. T125T c375G>A muation in p53 in Pts from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. T125T c375G>A mutation in p53 in pts not from Braz	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. T125T splice in DBD in pts from Brazil	0 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. T125T splice in DBD in pts not from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. wild type p53 in Patients from Brazil	1 participants	—	—
Incidence and Type of Germline TP53 Mutations in Non-Brazilian Children and Children From Southern Brazil by Deoxyribonucleic Acid (DNA) Sequencing and Affymetrix Gene Chip Analysis. wild type p53 in Patients not from Brazil	16 participants	—	—

SECONDARY outcome

Timeframe: Patients who had surgery at time of enrollment.

Population: Fifty-eight eligible patients had material examined for the presence of (beta)-catenin mutations.

The number of eligible patients who have A43 del33bp mutation of (beta)-catenin.

Outcome measures

Outcome measures
Measure	Stratum 1 n=58 Participants Stage I Disease (surgery, observation)	Stratum 2 Stage II Disease (surgery, observation)	Stratum 3 Stage III OR IV Disease (chemotherapy)
Molecular Alterations and Embryonal Markers in Children With ACT - A43 del33bp Mutation of (Beta)-Catenin. A43 del33bp mutation of (beta)-catenin	1 Participants	—	—
Molecular Alterations and Embryonal Markers in Children With ACT - A43 del33bp Mutation of (Beta)-Catenin. children with ACT - wild type (beta)-catenin	51 Participants	—	—

SECONDARY outcome

Timeframe: Up to one year or while on protocol therapy, whichever is less

The number of eligible patients who have surgical resection of the primary tumor and have tumor spillage at the time of resection.

Outcome measures

Outcome measures
Measure	Stratum 1 n=69 Participants Stage I Disease (surgery, observation)	Stratum 2 Stage II Disease (surgery, observation)	Stratum 3 Stage III OR IV Disease (chemotherapy)
Frequency of Tumor Spillage at the Time of Tumor Resection	15 Participants	—	—

Adverse Events

Stratum 3

Serious events: 1 serious events

Other events: 38 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Stratum 3 n=39 participants at risk Stage III OR IV Disease (chemotherapy)
Respiratory, thoracic and mediastinal disorders Pneumonitis	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.

Other adverse events

Other adverse events
Measure	Stratum 3 n=39 participants at risk Stage III OR IV Disease (chemotherapy)
Infections and infestations Abdominal infection	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Abdominal pain	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Acidosis	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations Activated partial thromboplastin time prolonged	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Endocrine disorders Adrenal insufficiency	12.8% 5/39 • Number of events 5 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Psychiatric disorders Agitation	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations Alanine aminotransferase increased	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Immune system disorders Allergic reaction	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Skin and subcutaneous tissue disorders Alopecia	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Blood and lymphatic system disorders Anemia	61.5% 24/39 • Number of events 24 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Anorexia	17.9% 7/39 • Number of events 7 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Psychiatric disorders Anxiety	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations Aspartate aminotransferase increased	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Musculoskeletal and connective tissue disorders Back pain	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations Blood bilirubin increased	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Cardiac disorders Cardiac disorders - Other, specify	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Catheter related infection	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Renal and urinary disorders Chronic kidney disease	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Colitis	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Psychiatric disorders Confusion	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Dehydration	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Nervous system disorders Depressed level of consciousness	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Diarrhea	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Nervous system disorders Dysarthria	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Respiratory, thoracic and mediastinal disorders Dyspnea	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Enterocolitis infectious	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Esophagitis	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
General disorders Fatigue	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Blood and lymphatic system disorders Febrile neutropenia	41.0% 16/39 • Number of events 16 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
General disorders Fever	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Eye disorders Flashing lights	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Gastrointestinal disorders - Other, specify	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations GGT increased	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Musculoskeletal and connective tissue disorders Growth suppression	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Psychiatric disorders Hallucinations	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Ear and labyrinth disorders Hearing impaired	23.1% 9/39 • Number of events 9 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Cardiac disorders Heart failure	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Hyperglycemia	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Hyperkalemia	10.3% 4/39 • Number of events 4 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Vascular disorders Hypertension	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Hypocalcemia	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Hypoglycemia	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Hypokalemia	23.1% 9/39 • Number of events 9 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Hypomagnesemia	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Hyponatremia	17.9% 7/39 • Number of events 7 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Metabolism and nutrition disorders Hypophosphatemia	12.8% 5/39 • Number of events 5 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Vascular disorders Hypotension	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Respiratory, thoracic and mediastinal disorders Hypoxia	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Infections and infestations - Other, specify	17.9% 7/39 • Number of events 7 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations INR increased	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
General disorders Irritability	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Cardiac disorders Left ventricular systolic dysfunction	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Lung infection	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations Lymphocyte count decreased	7.7% 3/39 • Number of events 3 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
General disorders Malaise	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Mucositis oral	17.9% 7/39 • Number of events 7 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Nausea	15.4% 6/39 • Number of events 6 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations Neutrophil count decreased	51.3% 20/39 • Number of events 20 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Obstruction gastric	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
General disorders Pain	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Nervous system disorders Peripheral motor neuropathy	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Nervous system disorders Peripheral sensory neuropathy	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Pharyngitis	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations Platelet count decreased	53.8% 21/39 • Number of events 21 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Respiratory, thoracic and mediastinal disorders Pneumonitis	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Endocrine disorders Precocious puberty	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Reproductive system and breast disorders Premature menopause	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Skin and subcutaneous tissue disorders Rash maculo-papular	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Eye disorders Retinal vascular disorder	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Sepsis	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Skin infection	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Respiratory, thoracic and mediastinal disorders Sore throat	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Upper respiratory infection	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Urinary tract infection	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Injury, poisoning and procedural complications Vascular access complication	5.1% 2/39 • Number of events 2 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Cardiac disorders Ventricular arrhythmia	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Gastrointestinal disorders Vomiting	15.4% 6/39 • Number of events 6 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations Weight loss	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Investigations White blood cell decreased	41.0% 16/39 • Number of events 16 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.
Infections and infestations Wound infection	2.6% 1/39 • Number of events 1 Serious and other adverse events were collected only for Stratum 3 patients who received systemic intervention. Stratum 1 and Stratum 2 patients did not receive chemotherapy as part of protocol treatment.

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: 626-447-0064

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Must obtain prior Sponsor approval.
Publication restrictions are in place

Restriction type: OTHER