Pediatrics

This partially randomized phase II/III trial studies how well, in combination with surgery, cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or hepatocellular carcinoma. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy may kill more tumor cells than one type of chemotherapy alone.

This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.

This phase II trial studies how well naive T-cell depletion works in preventing chronic graft-versus-host disease in children and young adults with blood cancers undergoing donor stem cell transplant. Sometimes the transplanted white blood cells from a donor attack the body's normal tissues (called graft versus host disease). Removing a particular type of T cell (naive T cells) from the donor cells before the transplant may stop this from happening.

This phase II Pediatric MATCH screening and multi-sub-trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one line of standard systemic therapy and/or for which no standard treatment exists that has been shown to prolong survival. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with genetic changes or abnormalities (mutations) may benefit more from treatment which targets their tumor's particular genetic mutation, and may help doctors plan better treatment for patients with solid tumors or non-Hodgkin lymphomas.

Cystic Fibrosis Related Diabetes has been identified by the CF community as one of the top ten priorities for CF research. In CF clinical decline due to dysglycemia begins early, prior to diagnosis of diabetes and increases mortality from pulmonary disease. There is presently no way to determine who, of those with dysglycemia, will experience clinical compromise. However, the CF Center in Milan has found that measurable age- and sex-dependent variables on oral glucose tolerance testing (OGTT) predict β-cell failure-the primary driver of decline in CF. the investigators propose a multi-center trial to develop nomograms of age and sex dependent reference values for OGTT-derived measures including glucose, insulin, c-peptide, and the resultant OGTT-derived estimates of β-cell function, β cell sensitivity to glucose, and oral glucose insulin sensitivity (OGIS) and to determine correlation of these with clinical status (FEV-1, BMI z score, number of pulmonary exacerbations over the past 12 months). In a subset of the cohort the investigators will perform additional studies to determine possible mechanisms driving abnormal β cell function, including the role of lean body mass (as measured by DXA), impact of incretin (GLP-1, GIP) and islet hormones (glucagon, pancreatic polypeptide) on β cell function and the relationship of reactive hypoglycemia and catecholamine responses to β cell function, as well as the relationship of β cell sensitivity to glucose as determined by our model to abnormalities in blood glucose found in a period of free living after the study (determined by continuous glucose monitoring measures (Peak glucose, time spent \>200 mg/dl, standard deviation). the investigators will also develop a biobank of stored samples to allow expansion to the full cohort if warranted and to enable future studies of dysglycemia and diabetes in CF. the investigator's eventual goal is utilization of the nomograms to determine the minimum number of measures to accurately predict risk for clinical decline from dysglycemia in CF.

This phase III trial studies iobenguane I-131 or lorlatinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not harm normal cells. Lorlatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving iobenguane I-131 or lorlatinib and standard therapy may work better compared to lorlatinib and standard therapy alone in treating younger patients with neuroblastoma or ganglioneuroblastoma.

This randomized phase III trial studies how well combination chemotherapy (vincristine sulfate, dactinomycin, cyclophosphamide alternated with vincristine sulfate and irinotecan hydrochloride or vinorelbine) works compared to combination chemotherapy plus temsirolimus in treating patients with rhabdomyosarcoma (cancer that forms in the soft tissues, such as muscle), and has an intermediate chance of coming back after treatment (intermediate risk). Drugs used work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Combination chemotherapy and temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether chemotherapy plus temsirolimus is more effective than chemotherapy alone in treating patients with intermediate-risk rhabdomyosarcoma.

The goal of this study is to determine the effects of stimulant medication on disruptive behavior, function, preference and choice; however, it is primarily methodological and will add to current research by establishing an effective evaluation of the impact of stimulant medication on these behaviors. Three behavior assessments for children and adolescents diagnosed with AD/HD who exhibit disruptive behavior will be conducted:

1. Preference assessments will be conducted to determine whether preference for social and nonsocial items and activities differs under medication and non-medication conditions.
2. Functional analyses will be conducted to determine whether stimulant medication has an effect on the frequency and function or purpose of disruptive behavior.
3. Choice assessments will be conducted to evaluate the impact of stimulant medication on impulse control/delay discounting.

This study will be conducted in three phases. For each of the 5 to 10 participants there will be 8 total visits. The first 4 visits will entail a preference assessment, followed by a functional analysis. On visits 1 and 3, the participant will be asked to take his/her stimulant medication as is typically done; however, on visits 2 and 4, the participant will be asked to refrain from taking the medication.

For visits 5-8, participants will continue to participate in preference assessments, but will also be presented with a choice arrangement with work and play. In the choice arrangement, participants will be given four work cards and four play cards that they can organize in any order. Work cards will be associated with a brief academic task and play cards will be associated with a brief play period using high-preferred toys/activities. On visits 5 and 7 the participant will be asked to take his or her stimulant medication as usual, while on visits 6 and 8 the participant will be asked to refrain from taking his or her medication.

This phase II trial studies how well inotuzumab ozogamicin works in treating younger patients with B-lymphoblastic lymphoma or CD22 positive B acute lymphoblastic leukemia that has come back (relapsed) or does not respond to treatment (refractory). Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a toxic agent called ozogamicin. Inotuzumab attaches to CD22 positive cancer cells in a targeted way and delivers ozogamicin to kill them.