A grade PMID 42321916
View analysis →Finding therapies hidden in 37,265 pediatric cancer papers.
Neurocompute scores pediatric oncology literature, surfaces overlooked therapeutic signals, and turns fragmented childhood cancer research into a living discovery terminal.
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All ranked pediatric cancer papers
This single-center phase 1 trial reports that repeated intracerebroventricular B7-H3 CAR T-cell infusions were feasible and tolerable in children and young adults with diverse recurrent/refractory CNS tumors or non-pontine diffuse midline glioma, with two partial responses among 26 treated patients.
The trial provides human evidence that repeated locoregional delivery of B7-H3 CAR T cells is feasible and tolerable; it supports, but does not establish, the hypothesis that targeting B7-H3 within the CNS could produce clinically meaningful antitumor activity in selected pediatric CNS tumors.
This phase II trial enrolled 60 patients with high-risk neuroblastoma in second or later complete remission and reported 2- and 5-year progression-free survival rates of 55% and 50% after five cycles of naxitamab plus stepped-up GM-CSF, while acknowledging potential confounding from subsequent investigational therapies.
The trial provides clinical evidence that naxitamab plus stepped-up GM-CSF can be administered as post-relapse remission consolidation with encouraging long-term progression-free survival; it remains an inference, rather than a controlled conclusion, that this regimen itself prolongs remission because no comparator is reported and many patients received post-protocol vaccine therapy.
This narrative review synthesizes mechanisms of anthracycline cardiotoxicity and evidence for pharmacologic cardioprotection, identifying dexrazoxane as the only approved preventive agent with adult and pediatric support while describing several less-established strategies.
The reviewed evidence supports dexrazoxane and suggests modest cardiac-function preservation with selected neurohormonal agents and statins; it is plausible, but not established by this review, that early risk-guided use of these or emerging metabolic therapies could reduce anthracycline-related cardiac injury in pediatric oncology patients.
In this multicentre phase 1 expansion study, 25 of 44 children, adolescents, and young adults with relapsed or refractory AML treated at the recommended phase 2 dose of venetoclax plus cytarabine-based therapy achieved complete response with or without haematological recovery after one cycle, with frequent severe toxicities and two grade 5 events.
The clinical results support the hypothesis that adding venetoclax to high-dose cytarabine-based regimens can induce remissions, including measurable-residual-disease-negative responses, in some young patients with relapsed or refractory AML; whether venetoclax improves survival or outcomes over chemotherapy alone remains an inference requiring randomized testing.
This systematic review and pooled analysis of seven studies reports that, among 87 pediatric patients receiving azacitidine or decitabine before HSCT, MDS—particularly decitabine-treated MDS—showed deeper responses than JMML, with complete remission in MDS associated with better post-HSCT survival than progressive disease.
The pooled evidence supports disease-specific response patterns to pre-HSCT hypomethylating therapy; it suggests—but does not establish—that decitabine may be an effective bridging option for pediatric MDS, whereas JMML may require strategies addressing RAS/MAPK-driven biology rather than reliance on hypomethylating therapy alone.
This review summarizes available pediatric ALL efficacy and toxicity evidence for targeted and immune-based therapies, including blinatumomab, inotuzumab ozogamicin, and tisagenlecleucel, and advocates evaluating their earlier integration into frontline treatment.
The record indicates that targeted therapies have clinical roles in relapsed or refractory pediatric ALL; the authors infer that incorporating selected agents into frontline therapy for high-risk patients could improve responses and remission duration while reducing reliance on toxic chemotherapy, but this prospective benefit is not established by the supplied record.
This multicenter prospective trial enrolled 109 assessable pediatric patients with newly diagnosed classical Hodgkin lymphoma and reported that response-adapted chemotherapy produced complete remission in 94 patients, limited radiotherapy use to 19.3%, and yielded 5-year EFS of 88.5% and OS of 100% at 58 months median follow-up.
The reported clinical evidence supports the feasibility of withholding radiotherapy from children and adolescents who achieve complete remission after risk- and response-adapted chemotherapy; it remains an inference, not established by a randomized comparison, that this approach preserves disease control while reducing late radiotherapy-related toxicity.
This systematic review of eight early-phase trials reports that 63 treated pediatric and young adult patients with recurrent or refractory primary CNS tumors received CAR-T cells targeting several antigens, with feasible delivery, reversible immune toxicities, infrequent objective responses, frequent disease stabilization, and evidence of CNS trafficking and immune activation.
The reviewed clinical evidence supports that CNS-directed CAR-T cells can reach or activate within the CNS and may stabilize disease in some heavily pretreated patients; it remains an inference requiring prospective confirmation that optimizing antigen targets, administration routes, dosing, and toxicity management will produce durable survival benefit, particularly in diffuse midline glioma.
In a Danish multicentre open-label randomized trial involving 88 febrile episodes in 70 children with cancer and high-risk febrile neutropenia without microbiologically documented infection, stopping intravenous antibiotics after 48 hours of defervescence and clinical stability reduced antibiotic exposure compared with continuing until neutrophil recovery, with similar observed serious adverse-event rates and no deaths.
The trial provides direct evidence that clinically stable, afebrile children meeting the study's eligibility criteria can have empirical antibiotics discontinued earlier to reduce antibiotic exposure; it is reasonable to infer potential stewardship and toxicity benefits, but these were not directly demonstrated, and safety for rare outcomes remains uncertain because the trial was not powered for them.
In a single-centre phase 2 trial, 106 patients aged 16–60 years with relapsed or refractory acute leukaemia received organ-sparing total marrow and lymphoid irradiation plus high-dose cyclophosphamide and etoposide before allogeneic HCT, with an estimated 2-year progression-free survival of 34% and frequent grade 3–4 toxicities.
The trial provides evidence that 2000 cGy total marrow and lymphoid irradiation can be delivered with high-dose cyclophosphamide and etoposide before allogeneic HCT in this selected population; it supports—but does not prove—the hypothesis that targeting marrow and lymphoid tissues while limiting vital-organ irradiation could preserve antileukaemic conditioning intensity with acceptable organ toxicity and clinically useful disease control.
This workshop report prioritizes therapeutic strategies for high-risk rhabdomyosarcoma, including FGFR4-directed therapies, fusion-protein and transcriptional-regulator degraders, genotype-specific ROR2 targeting, B7-H3 antibody-drug conjugates, and rational MEK-based combinations.
The record supports these approaches as expert-selected research priorities rather than validated treatments; it hypothesizes that targeting subtype-specific drivers and surface antigens, or combining pathway inhibitors with chemotherapy, could improve efficacy or reduce toxicity in high-risk rhabdomyosarcoma.
In a single-center retrospective cohort of 30 children with relapsed or refractory high-risk neuroblastoma who received more than five cycles of dinutuximab beta with chemotherapy, GM-CSF, and isotretinoin, response rates increased during extended treatment, including best responses after cycle 5, alongside substantial grade ≥3 toxicities.
The study provides preliminary clinical evidence that selected patients able to continue dinutuximab beta–based chemoimmunotherapy beyond five cycles may achieve additional or maintained responses; whether extending treatment itself improves survival or outweighs cumulative toxicity remains an inference requiring a controlled prospective comparison.