Nutritional and Metabolic Diseases

All Conditions

Neoplasms

Rare Diseases

Malnutrition

Neuroblastoma

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neuroepithelioma

Children undergoing treatment for Neuroblastoma at ITACI in Sao Paolo in Brazil who are under the age of 18 years.

Elena Ladas, Phd, RD

Karina Viani, RD

Vicente Odone, MD, PhD

This study proposes to investigate the effect of treatment of neuroblastoma on nutritional status, assessed by body mass index (BMI) z score, and body composition evaluated by mid-upper arm circumference (MUAC), from diagnosis through 1-year post end of treatment. The study also aims to investigate the nutritional status and its role in toxicities, infection, survival rates, disease relapse, cost of care and readmission rates, as well as health-related quality of life. The study will take place in Sao Paolo in Brazil, where an estimated sample of 50 children with neuroblastoma will be recruited for the period of 2 years.

In Brazil, cancer represents the leading cause of death by disease in children and adolescents from 1 to 19 years old. Neuroblastomas correspond to nearly 10% of all cancer diagnoses among children younger than 15 years of age, with an incidence of 1 case per 100,000 children of this age group. About 500 new cases of neuroblastoma (NB) are diagnosed annually in Brazil. The complexity and intensity of treatment result in several treatment-related toxicities (TRT) that make it challenging to maintain nutritional status in these nutritionally at-risk patients. In Brazil, up to 26% of undernutrition at diagnosis has been found in patients with solid tumors. In neuroblastoma, high prevalence rates of malnutrition have been reported elsewhere with up to 80% of children at diagnosis and 20-50% during treatment classified as undernourished. It's important to underscore that nutritional status is a modifiable risk factor, therefore it needs more attention and close monitoring. It is important to better comprehend the effect of treatment for neuroblastoma on nutritional status and body composition, for which prospective longitudinal studies are required. Therefore, in this prospective cohort study, the investigators will evaluate the fluctuations in nutritional status over the course of treatment in children with neuroblastoma at the study center in São Paulo, Brazil. The results of this study will drive the development of evidence-based guidelines for the nutritional care of children with neuroblastoma and set research priorities for subsequent investigations.

Blood collection to bank plasma and serum will occur at diagnosis, after completion of immune therapy and at 12 month post-treatment.

For micronutrient analysis, 3 to 5 mL of the patient's venous blood will be collected without anticoagulant to study To obtain serum and plasma, to bank for future micronutrient analysis, blood samples (10 mL) will be collected by venipuncture.

Nutritional Status in Neuroblastoma in Sao Paolo, Brazil

The Role of Nutritional Status in the Treatment of Neuroblastoma

The database is secured in Brazil research site at ITACI. De-identified data is shared with Columbia University Medical Center via REDCap database system - managed by Columbia University

Effect of neuroblastoma treatment in nutritional status: Nutritional status will be measured by BMI Z score

Effect of neuroblastoma treatment in body composition: Body composition will be measured by mid-upper arm circumference (MUAC)

Effect of neuroblastoma treatment in body composition: Body composition will be measured by BIA

Investigate association of nutritional status and the occurrence of grade 3/4 toxicities using NCI Common Toxicity Criteria (CTC)

Study the association of nutritional status and infection classified from moderate to severe neutropenia (ANC \< 500)

Survival is defined by both overall survival (time from enrollment to death from any cause) and event-free survival (time from enrollment to the first episode of relapse, disease progression or death).

Investigate the association of nutritional status and disease relapse

Investigate the association of nutritional status and the cost of care defined by the sum of the cost of duration of hospital stay and cost of readmission rates. We will evaluate the values individually and collectively.

Health-related quality of life will be measured by the Health Utilities Preschool(HuPS) survey for 2 -5 years old or by Health Utilities Index (HUI) for 5 years and older (assessment is not considered for children under 2 years), during and after treatment, and its association with nutritional status and body composition. These are complementary instruments for use in the contiguous age groups. They are scored in the same way.

Investigate the association of nutritional status and diet quality, measured by 24h dietary recalls

Instituto de Tratamento do Câncer Infantil

Columbia University

Blood and Lymph Conditions

Immune System Diseases

Lymphoma

Leukemia

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Lymphoma, Follicular

Lymphoma, Large B-Cell, Diffuse

Lymphoma, B-Cell

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Hematologic Diseases

Lymphoma, Non-Hodgkin

Lymphosarcoma

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Follicular Lymphoma

B-cell Lymphoma

Diffuse Large B-Cell Lymphoma

Acute Graft Versus Host Disease

Antineoplastic Agents

All Drugs and Chemicals

Tisagenlecleucel

Antineoplastic Agents, Immunological

Children/young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia who received tisagenlecleucel infusion

Adult patients with relapsed/refractory Diffuse Large B-cell Lymphoma who received tisagenlecleucel infusion

Patients of any gender aged 18 year or older, with relapsed/refractory Follicular Lymphoma who received tisagenlecleucel infusion.

Prospective observational study. There is no treatment allocation. Patients prescribed with tisagenlecleucel in the commercial setting or out-of-specification (OOS) are eligible to enroll into this study

tisagenlecleucel

Novartis Pharmaceuticals

This will be a multicenter, national, non-interventional, prospective cohort study

Eligible participants will be pediatric (\<18 years) and adult patients (aged 18 years or older) with B-cell malignancies who have received tisagenlecleucel through the commercial setting or out-of-specification (OOS) use in Brazil. We will collect data prospectively and complement missing information with retrospective data collection, when necessary. It is anticipated that approximately 200 patients will be enrolled in the cohort over 5 years divided among the study indications.

Since this is a non-interventional study, no administration of study drug or application of questionnaires will be mandated by this protocol. The study will consist of a "Pre-infusion" and a "Post infusion follow-up period" for up to 15 years post tisagenlecleucel infusion. All patients will be followed until death or last scheduled visit, whichever comes first.

For the study, "pre-infusion" and "follow-up post infusion" phases are defined as:

* "Pre-infusion" will consist of the patient's information from the time of diagnosis untiljust prior to infusion with tisagenlecleucel.
* "Follow-up Post infusion" information will comprise any information from the infusionof tisagenlecleucel onwards.

Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies

Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies: a 15-year Prospective Registry Study on Three Cohorts.

The overall response rate will be defined as the total proportion of participants exhibiting either the best overall response (BOR) of complete or partial responses and the proportion of patient with BOR of CR/PR or CR/CRi for ALL patients will be reported along with its 95% CI.

For ALL participants, the BOR will be defined as a CR or a CRi in accordance with National Comprehensive Cancer Network (NCCN) guidelines and previous guidelines (Appelbaum et al 2007)(Cheson et al 2003).

For lymphomas, the BOR will be defined as a CR or PR in accordance with the Cheson response criteria (Cheson et al 2007) and the Lugano classification (Cheson et al 2016).

The percentage of B-cell ALL patients who achieve a Best Overall Response (BOR) of CR or CRi with a Minimal residual disease (MRD) negative bone marrow will be provided with 95% CI.

Duration of overall response (DOR) applies only to patients whose best overall disease response was either:

* CR or PR for patients with lymphomas, or
* CR or a CRi for patients with ALL.

DOR will be defined as the time from the date of first documented disease response (Complete Response (CR) or PR for patients with lymphomas, and Complete Remission (CR) or CRi for patients with ALL), whichever occurs first, to the date of first documented progression or first documented relapse according to indication, or to the date of death due to the underlying disease.

In case a patient does not have progression/relapse or death due to underlying disease (defined as the event for this outcome) prior to data cut-off, DOR will be censored at the date of the last assessment on or prior to the earliest censoring event.

RFS is measured by the time from date of first documented disease response as CR or CRi to relapse or death due to any cause in ALL patients.

In case a patient does not have relapse or death due to any cause prior to data cutoff, RFS will be censored at the date of the last adequate assessment on or prior to the earliest censoring event.

EFS is the time from date of first tisagenlecleucel infusion to treatment failure, relapse or death from any cause, whichever occurred first, for B-cell ALL patients.

PFS is defined as the time from the date of first infusion to the date of event defined as the first documented progression of lymphoma or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of the last adequate assessment.

In case a patient does not have progression or death prior to data cutoff, PFS will be censored at the date of the last adequate assessment on or prior to the earliest censoring event.

Overall survival is the time from date of first tisagenlecleucel infusion to the date of death due to any reason, In case a patient is alive at the date of last contact on or before data cutoff, OS is censored at the date of last contact.

Dates of hematological recovery (i.e., dates of Absolute Neutrophil Count (ANC) and platelet recovery) will be collected.

ANC recovery is defined as an ANC of ≥ 0.5 × 109/L (500/mm\^3) for 3 consecutive laboratory values obtained on different days. Date of ANC recovery is the date of the first of 3 consecutive laboratory values where the ANC is ≥ 0.5 × 109/L (CIBMTR).

The first date of the 3 consecutive laboratory values obtained on different days where the platelet count was ≥ 20 × 109/L should be recorded. It should be ensured that no platelet transfusions were administered for 7 days immediately preceding this date (CIBMTR).

The type and frequency of SAEs and AE of special interest (including secondary malignancies) will be collected

Incidence and severity of Cytokine release syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) among Human T-cell Lymphotropic Virus (HTLV) 1 and 2 positive versus HTLV 1 and 2 negative patients.

For CRS AE the protocol will follow the American Society of Transplant and Cellular Therapy (ASTCT) CRS Consensus Grading.

For ICANS AE the protocol will follow ASTCT consensus as well. Which establishes the Immune effector Cell-associated Encephalopathy (ICE Score) for adults/ adolescents and Cornell Assessment of Pediatric Delirium (CAPD) for pediatric patients under 12 years

Number of patients with confirmed secondary malignancies diagnosis will be collected

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Nutritional Status in Neuroblastoma in Sao Paolo, Brazil	NCT03517280	Neuroblastoma		- 18 Years	Columbia University
Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies	NCT05541341	Diffuse Large B... Acute Lymphobla... Follicular Lymp...	tisagenlecleuce...	- 100 Years	Novartis
Nutritional Status in Neuroblastoma in Sao Paolo, Brazil	NCT03517280	Neuroblastoma		- 18 Years	Columbia University

Spots Global Cancer Trial Database for brazil

Every month we try and update this database with for brazil cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for brazil

Every month we try and update this database with for brazil cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for brazil cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.