Neuroblastoma (NB)

This is a Phase II clinical trial investigating the effectiveness and safety of a four-drug combination-Chidamide, Dinutuximab Beta, Irinotecan, and Temozolomide-for children with relapsed or refractory neuroblastoma. The primary goal is to evaluate how well this regimen works to control the cancer, while the secondary goal is to closely monitor its safety and side effects in these young patients.

Neuroblastoma (NB) is a malignant neoplasm of the sympathetic nervous system, occurring in 1 in 8,000 live births, accounting for 6-10% of all childhood malignant neoplasms and responsible for 12-15% of mortality -, making it the most common and life-threatening extracranial tumor in childhood. Patients with stage 4 high-risk NB is the subgroup with the poorest prognosis. Within this group, two subgroups with an extremely unfavorable disease course are distinguished: patients with a poor response to the induction phase of therapy (refractory disease) and patients with relapsed or progressive disease. Nowadays, 10-15% of patients show a poor end-induction response, whereas achieving a good end-induction response associated with better long-term survival. Improvement of the response to induction therapy may contribute to better treatment outcomes in newly diagnosed high-risk NB patients and can be achieved by intensification of the induction phase to decrease the number of patients with refractory disease. Also intensification of the second-line therapy may contribute to better responses in patients with relapsed and progressive disease. Protocol aimed to overcome heterogeneous tumor drug resistance through the synergistic interaction of cytostatic and immunobiological agents in combination with NK cell therapy. This approach combines cytotoxic agents with anti-GD2 monoclonal antibodies (mAb) to enhance antitumor activity. Cultured, ex vivo-activated autologous NK cells are infused to compensate for effector cell depletion during therapy and to augment antibody-dependent cellular cytotoxicity (ADCC), potentially improving clinical outcomes. This comprehensive approach opens novel prospects for enhancing treatment efficacy in patients with refractory and relapsed high-risk NB. The expected outcomes of this protocol include a significant increase in therapeutic efficacy indicators - objective response rate (ORR), overall survival (OS), progression-free survival (PFS) and relapse-free survival (RFS), as well as in patient quality of life.

The goal of this prospective observational study is to evaluate the diagnostic performance of ¹²³I-MIBG SPECT/CT and SSTR PET in Chinese children with suspected or confirmed neuroblastoma (NB). The main questions it aims to answer are: * What are the sensitivity and specificity of each modality-alone and combined-for initial staging and for detecting relapse or metastasis? * Can SSTR-targeted PET reliably identify MIBG-negative NB lesions and help select candidates for peptide-receptor radionuclide therapy (PRRT)? Pediatric patients (≤18 years) undergoing routine evaluation for NB will receive both imaging studies; results will be correlated with histopathology, clinical course, and 24-month follow-up.

The goal of this clinical trial is to assess safety and efficacy of systemic injection of allogenic NK cells in patients with refractory/recurrent high-risk neuroblastoma. Is the injection of allogenic nk cells safe in patients with R/R high-risk neuroblastoma? Is the injection of allogenic nk cells effective in patients with R/R high-risk neuroblastoma? We will compare the NK cell administration group with a control group that receives conventional treatment to determine whether the intervention is safe and effective

The goal of this observational study is to validate the EQ-5D-Y-3L and EQ-5D-Y-5L in measuring health-related quality of life (HRQoL) for paediatric patients with different health conditions in China. The main research questions focus on comparing the psychometric performance of EQ-5D-Y-3L and EQ-5D-Y-5L, evaluating the differences between self-complete (SC), interviewer-administered (IA), and proxy-administered (Proxy) versions, and assessing their validity against other HRQoL measures like PedsQL. The study will recruit 360 inpatient-caregiver dyads from three disease groups, i.e., pneumonia, paediatric central nervous system (CNS) solid tumours, and immune thrombocytopenic purpura (ITP), using cross-sectional and longitudinal surveys. Baseline assessments will be conducted at hospital admission, with follow-up at hospital discharge (within 14 days). A qualitative component will explore acceptability and interpretation of EQ-5D-Y. The findings will contribute to the methodological development of EQ-5D-Y, with potential implications for HRQoL measurement and economic evaluations in paediatric healthcare.

Title: Safety and efficacy of CAR-T cell therapy for relapsed/refractory neuroblastoma and desmoplastic small round cell tumors: a single-arm, open-label trial. The CART used in this study will be provided by Shanghai YaKe Biotechnology Ltd. Aims: 1. To evaluate the safety and efficacy of GD2/B7H3 CAR-T therapy for relapsed/refractory neuroblastoma, and observe its pharmacokinetic/pharmacodynamic characteristics and the survival of CAR-T cells in relapsed/refractory neuroblastoma patients. 2. To evaluate the safety and efficacy of GD2/B7H3 CAR-T therapy for relapsed/refractory desmoplastic small round cell tumor, and observe its pharmacokinetic/pharmacodynamic characteristics and the survival of CAR-T cells in desmoplastic small round cell tumor patients. Patients: Relapsed/refractory neuroblastoma; Relapsed/refractory desmoplastic small round cell tumor. CAR-T therapy: Lymphodepletion treatment will be performed within 14 days prior to CAR-T cell infusion: intravenous chemotherapy based on fludarabine 25mg/m² and cyclophosphamide 500mg/m² for 1 to 3 days. CAR-T cells will then be infused intravenously, with a dosage of 1.00 to 10.00 × 10⁶/kg of CAR-positive T cells. Research period: CAR-T cell infusion will be followed up for one year, or until adverse events resolve, progression occurs, or the patient transitions to other treatments. Outcome measures: Incidence of adverse events related to CAR-T therapy, as well as their intensity and duration; Pharmacokinetic/pharmacodynamic characteristics of CAR-T in patients and the survival of CAR-T cells. Overall response rate (ORR) after CAR-T cell infusion, including complete response (CR) and partial response (PR); Overall survival (OS), progression-free survival (PFS), event-free survival (EFS), time to progression (TTP), and duration of response (DOR) after CAR-T cell infusion;