Alberto S. Pappo
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View article: Belzutifan for HIF2A-Related Pheochromocytoma and Paraganglioma: A Retrospective Study of Real-World Data
Belzutifan for HIF2A-Related Pheochromocytoma and Paraganglioma: A Retrospective Study of Real-World Data Open
Belzutifan is a highly effective and well-tolerated therapeutic option for EPAS1(HIF2A)-related PPGL, producing substantial biochemical and radiographic responses, improved blood pressure control, and resolution of erythrocytosis. These fi…
View article: Supplemental Table 2 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 2 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 2. Genes included on multigene panel tests.
View article: Supplemental Table 3 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 3 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 3. Total cohort of 78 Patients with thyroid carcinoma, including those who did and did not undergo genetic testing.
View article: Supplemental Table 4 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 4 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 4. Patient Cancer and Treatment History.
View article: Supplemental Table 5 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 5 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 5. Thyroid carcinoma treatment and outcomes for patients with germline CPG PV.
View article: Supplemental Table 1 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 1 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 1. Germline genetic testing completed.
View article: ESFT13: A Phase II Study Evaluating the Addition of Window and Maintenance Therapy to a Standard Chemotherapy Backbone for the Treatment of High-Risk Ewing Sarcoma
ESFT13: A Phase II Study Evaluating the Addition of Window and Maintenance Therapy to a Standard Chemotherapy Backbone for the Treatment of High-Risk Ewing Sarcoma Open
Study Aim: Patients with high-risk Ewing sarcoma (ES) have dismal outcomes despite aggressive multimodal therapy. This phase II, single-institution study evaluated the response rate to two up-front cycles of irinotecan, temozolomide, and t…
View article: Supplemental Table 3 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 3 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 3. Total cohort of 78 Patients with thyroid carcinoma, including those who did and did not undergo genetic testing.
View article: Supplemental Table 2 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 2 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 2. Genes included on multigene panel tests.
View article: Supplemental Table 1 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 1 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 1. Germline genetic testing completed.
View article: Supplemental Table 5 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 5 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 5. Thyroid carcinoma treatment and outcomes for patients with germline CPG PV.
View article: Supplemental Table 4 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Supplemental Table 4 from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Supplemental Table 4. Patient Cancer and Treatment History.
View article: Data from Contribution of Germline Predisposition to Pediatric Thyroid Cancer
Data from Contribution of Germline Predisposition to Pediatric Thyroid Cancer Open
Purpose:Pediatric thyroid carcinoma is a rare cancer for which germline genetic factors remain incompletely understood. To gain further insight, we examined the prevalence and spectrum of pathogenic variants (PV) in cancer-predisposing gen…
View article: Comparing Whole-Body Diffusion-weighted MRI to Conventional Imaging: Staging Pediatric Bone and Soft-Tissue Sarcomas
Comparing Whole-Body Diffusion-weighted MRI to Conventional Imaging: Staging Pediatric Bone and Soft-Tissue Sarcomas Open
Whole-body diffusion-weighted MRI demonstrated agreement with conventional imaging and higher lesion sensitivity but lower specificity than PET/CT in staging pediatric bone and soft-tissue sarcomas.
View article: Acute cardiotoxicity in pediatric and adolescent patients with solid tumors treated with tyrosine kinase inhibitors
Acute cardiotoxicity in pediatric and adolescent patients with solid tumors treated with tyrosine kinase inhibitors Open
Background Multitargeted tyrosine kinase inhibitors (MTKIs) have increasingly been used for the treatment of pediatric and young adult patients with solid tumors. Whereas cardiac toxicity is documented in adults, data remain limited in ped…
View article: Clinicopathologic Features, Management, and Outcomes Of Pediatric <i>CRTC1::TRIM11</i> and <i>MED15::ATF1</i> Tumors With Spitzoid Morphology: A Case Series
Clinicopathologic Features, Management, and Outcomes Of Pediatric <i>CRTC1::TRIM11</i> and <i>MED15::ATF1</i> Tumors With Spitzoid Morphology: A Case Series Open
Limited information is available on the clinicopathologic features and management of pediatric CRTC1::TRIM11 (CTRTs) and MED15::ATF1 ‐rearranged tumors (MARTs). In this series of pediatric CTRTs ( n = 5) and MARTs ( n = 2), all tumors exhi…
View article: <i>EWSR1::CREM</i> Fusion in a Pediatric Patient With Testicular Leydig Cell Tumor
<i>EWSR1::CREM</i> Fusion in a Pediatric Patient With Testicular Leydig Cell Tumor Open
Sex cord‐stromal tumors are rare in pediatric patients. Leydig cell tumors are a rare subset of sex cord‐stromal tumors characterized by unique molecular alterations, including TERT fusions and mutations of CTNNB1 , FOXO4 , TP53 , NBN , MT…
View article: Elective Discontinuation of Larotrectinib in Pediatric Patients With TRK Fusion Sarcomas and Related Mesenchymal Tumors
Elective Discontinuation of Larotrectinib in Pediatric Patients With TRK Fusion Sarcomas and Related Mesenchymal Tumors Open
Larotrectinib is a highly selective tropomyosin receptor kinase (TRK) inhibitor with efficacy in children with TRK fusion tumors. We evaluated patient outcomes after elective discontinuation of larotrectinib in the absence of disease progr…
View article: Paediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration
Paediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration Open
Fewer than 10 % of children with diffuse midline glioma (DMG) survive 2 years from diagnosis. Radiation therapy remains the cornerstone of treatment and there are no medicinal products with regulatory approval. Although the biology of DMG …
View article: Head and neck paraganglioma in Pacak-Zhuang syndrome
Head and neck paraganglioma in Pacak-Zhuang syndrome Open
Background Head and neck paragangliomas (HNPGLs) are typically slow-growing, hormonally inactive tumors of parasympathetic paraganglia. Inactivation of prolyl-hydroxylase domain-containing 2 protein causing indirect gain-of-function of hyp…
View article: 136P Efficacy and safety of larotrectinib (laro) as first-line treatment for paediatric (paed) patients (pts) with TRK fusion cancer
136P Efficacy and safety of larotrectinib (laro) as first-line treatment for paediatric (paed) patients (pts) with TRK fusion cancer Open
Laro is the first-in-class, highly selective, central nervous system (CNS)-active TRK inhibitor approved for tumour-agnostic use in pts with TRK fusion cancer based on a robust and durable overall response rate (ORR) in both adult and paed…
View article: Impact of ACCELERATE Paediatric Strategy Forums: a review of the value of multi-stakeholder meetings in oncology drug development
Impact of ACCELERATE Paediatric Strategy Forums: a review of the value of multi-stakeholder meetings in oncology drug development Open
In a landscape of an increasing number of products and histology and age agnostic trials for rare patient cancer, prioritization of products is required. Paediatric Strategy Forums, organized by ACCELERATE and the European Medicines Agency…
View article: Immune-related adverse events from immune checkpoint inhibitors in pediatric patients: A literature review and case series
Immune-related adverse events from immune checkpoint inhibitors in pediatric patients: A literature review and case series Open
Background: Immune checkpoint inhibitors (ICIs) are used to treat many adult cancers and are now being used to treat select pediatric cancers. Many toxicities resulting from ICIs, also known as immune-related adverse events (IRAEs), have b…
View article: Adolescent and Young Adult (AYA) Oncology, Version 2.2024, NCCN Clinical Practice Guidelines in Oncology
Adolescent and Young Adult (AYA) Oncology, Version 2.2024, NCCN Clinical Practice Guidelines in Oncology Open
This selection from the NCCN Guidelines for Adolescent and Young Adult (AYA) Oncology focuses on considerations for the comprehensive care of AYA patients with cancer. Compared with older adults with cancer, AYA patients have unique needs …