A grade PMID 42321916
View analysis →Finding therapies hidden in 38,927 pediatric cancer papers.
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A grade PMID 42690647
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All ranked pediatric cancer papers
In a Wt1+/R394W mouse model of WT1 glomerulopathy, ultrasound-guided renal artery delivery of integrin αvβ3-targeted angiopoietin-1 mRNA nanocomplexes localized expression to glomeruli and reduced albuminuria, podocyte loss, endothelial injury, and glomerulosclerosis.
The record provides preclinical evidence that restoring reduced glomerular angiopoietin-1 through kidney-targeted mRNA delivery can slow WT1-associated glomerular injury; it remains an inference that this approach will be safe, technically feasible, and disease-modifying in children.
This narrative review synthesizes molecular classifications, treatment evidence, and emerging risk-adapted strategies for infant embryonal CNS tumors, highlighting durable survival with radiation-sparing regimens in favorable-risk SHH-activated medulloblastoma but persistently poor outcomes in several other subgroups.
The reviewed evidence supports molecular profiling as a tool for diagnosis and risk stratification and indicates that selected infants—particularly those with favorable-risk SHH-activated medulloblastoma—may benefit from regimens that delay or avoid craniospinal irradiation; it remains an inference requiring prospective validation that subgroup-specific targeted therapies, immunotherapy, liquid biopsy, and other emerging approaches will improve survival or neurodevelopmental outcomes across these rare tumors.
This Cochrane review of six pediatric retinoblastoma studies found moderate-certainty evidence from one RCT that intra-arterial chemotherapy probably improves globe salvage versus intravenous chemotherapy without a demonstrated overall-survival difference, while evidence for adding intravenous chemotherapy to intra-arterial therapy was largely low or very low certainty.
Evidence supports the hypothesis that first-line intra-arterial chemotherapy may improve eye preservation compared with intravenous chemotherapy in children with retinoblastoma; inferring a preferred regimen or added value from combined intravenous and intra-arterial treatment remains premature because survival, toxicity, recurrence, metastasis, and long-term outcomes are uncertain or inadequately reported.
In a prospective phase II study of transplant-eligible patients aged 18–60 years with intermediate-risk AML, mutation clearance after induction was used to direct HiDAC consolidation, and the 33 clearance-positive patients had longer relapse-free survival than historical HiDAC-treated controls, although the comparison did not meet the prespecified significance threshold.
The study provides preliminary evidence that broad leukemia-associated mutation clearance may identify patients who can achieve favorable relapse-free survival with HiDAC consolidation; it remains an inference, requiring randomized confirmation, that this strategy can safely select patients for chemotherapy rather than allogeneic transplantation.
This retrospective Italian case series reports improved chronic-wound healing without treatment-related adverse events in two children and one young adult with recessive dystrophic epidermolysis bullosa receiving weekly topical beremagene geperpavec alongside multidisciplinary supportive care.
The reported evidence associates topical B-VEC treatment with meaningful wound improvement in three patients; it is reasonable—but not established by this uncontrolled series—to hypothesize that HSV1-mediated delivery of full-length COL7A1 can promote durable wound closure and reduce wound burden when integrated with comprehensive supportive care.
This meta-analysis of eight retrospective single-center cohorts comprising 1,100 children with intracranial ependymoma reports pooled five-year survival and local-control outcomes after proton therapy, modest reported late-toxicity rates, and an association between subtotal resection and inferior local control and progression-free survival.
The supplied evidence supports proton therapy as a clinically used radiotherapy approach with favorable pooled outcomes, while the proposal that modern intensity-modulated proton dose escalation could improve control after subtotal resection remains an untested hypothesis requiring prospective comparative evaluation.
This three-patient case report, including one 8-year-old child, describes responses after modified DC–CIK cells loaded with tumour stem cell membrane microparticles were given for heavily pretreated relapsed or refractory T/NK-cell lymphoid malignancies, with two complete remissions, one partial response, and no treatment-related adverse events reported.
The reported responses support the preliminary hypothesis that tumour stem cell membrane microparticle loading may enhance DC–CIK targeting of refractory T/NK-cell malignancies; however, comparative studies are required to determine whether the cellular product caused the responses, improves durability, or is acceptably safe.
In 259 newly diagnosed AML patients drawn from two clinical trials and one retrospective study, seven-day venetoclax plus one of three dose-adjusted intensive chemotherapy regimens produced a 90.3% composite complete remission rate, 92.2% flow-cytometric MRD negativity, and estimated 24-month overall survival of 72.9%.
The reported clinical outcomes support seven-day venetoclax plus dose-adjusted intensive chemotherapy as a potentially active induction strategy; it may preserve efficacy while reducing myelosuppression relative to longer venetoclax schedules, but that comparative safety advantage is an inference because no longer-duration control group or detailed toxicity comparison is reported.
In an assessor-blinded randomized trial of 128 children aged 6–12 years receiving their first chemotherapy, immersive virtual reality was associated with reduced anxiety and fewer reported episodes of anticipatory nausea, chemotherapy-induced nausea and vomiting, and chemotherapy-induced vomiting compared with control.
The trial provides evidence that immersive virtual reality can improve short-term chemotherapy-related symptom control in children; it may act as a scalable nonpharmacologic adjunct through distraction or reduced anticipatory distress, but that mechanism was not directly tested in the supplied record.
This meta- and network meta-analysis of 16 trials involving 1,602 participants with Hodgkin lymphoma reports pooled response rates and suggests differential response rankings for camrelizumab, dual-checkpoint inhibition, and checkpoint inhibitors combined with conventional therapy.
The reported comparative response signals support the hypothesis that selected PD-1/PD-L1 inhibitor combinations may improve response rates in Hodgkin lymphoma; however, this is an inference from aggregate and network comparisons, not proof of superiority, safety, survival benefit, or pediatric-specific efficacy.
In 100 children with Crohn's disease who achieved remission after exclusive enteral nutrition induction, cyclic exclusive enteral nutrition reduced 12-month relapse compared with daily partial enteral nutrition (49% versus 76%) in an open-label, endpoint-blinded randomized trial.
The trial provides evidence that intermittent cycles of exclusive enteral nutrition can maintain drug-free remission more effectively than low-dose daily partial enteral nutrition in selected pediatric Crohn's disease responders; any relevance to pediatric oncology or treatment-associated gastrointestinal disease would be speculative because no patients with cancer were studied.
This review summarizes ALK-directed vaccines, CAR-T and other cellular therapies, and antibody-based approaches for pediatric ALK-positive tumors, citing endogenous ALK-specific immune responses and preclinical efficacy while emphasizing that clinical safety and effectiveness remain unproven.
Evidence in the supplied abstract indicates that ALK is a tumor driver and oncoantigen, with spontaneous ALK-specific immune responses in patients and efficacy of ALK-directed immunotherapies in preclinical models; it is therefore inferred—not clinically established—that these approaches could complement ALK inhibitors, address resistance, and improve long-term disease control.