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Research

Idiosyncratic nature of voriconazole photosensitivity in children undergoing cancer therapy

In adults, the unpredictability of voriconazole pharmacokinetics, particularly in those patients receiving chemotherapy, is well recognised. A paucity of...

Research

Germ-line and somatic DICER1 mutations in pineoblastoma

This study suggests that germ-line DICER1 mutations make a clinically significant contribution to PinB, establishing DICER1 as an important susceptibility...

Research

Hepatic sinusoidal obstruction syndrome during chemotherapy for childhood medulloblastoma: Report of a case and review of the literature

We report a patient with high-risk anaplastic medulloblastoma who developed severe HSOS during her second cycle of maintenance chemotherapy.

Research

Comparative drug screening in NUT midline carcinoma

The NUT midline carcinoma (NMC) is a rare but fatal cancer for which systematic testing of therapy options has never been performed.

Research

IDH mutant high-grade gliomas

Gliomas are the most common type of malignant primary central nervous system (CNS) tumors, resulting in significant morbidity and mortality in children and adolescent and young adult (AYA) patients. The discovery of mutations in isocitrate dehydrogenase (IDH) genes has dramatically changed the classification and understanding of gliomas.  IDH mutant gliomas have distinct clinical, pathological, and molecular features including a favorable prognosis and response to therapy compared to their wildtype counterparts.

Research

Metabolic and Psychological Impact of a Pragmatic Exercise Intervention Program in Adolescent and Young Adult Survivors of Pediatric Cancer-Related Cerebral Insult

Adolescent and young adult survivors of childhood brain tumors and cranial irradiation should be screened for metabolic and psychological well-being

Research

Clinical, Radiologic, Pathologic, and Molecular Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma

We report clinical, radiologic, and molecular factors that correlate with survival in children and young adults with diffuse intrinsic pontine glioma

Research

"Pre-emptive strike"-the case for early treatment of hepatic sinusoidal obstruction syndrome with defibrotide

We describe the case of a child with a right 11th rib primitive neuroectodermal tumor who developed HSOS following focal radiotherapy and actinomycin-D treatment

Research

Activation of ERBB4 in Glioblastoma Can Contribute to Increased Tumorigenicity and Influence Therapeutic Response

Despite low ERBB4 mRNA in glioblastoma, the functional effects of increased ERBB4 activation identify ERBB4 as a potential prognostic and therapeutic target

Research

Irreversible growth plate fusions in children with medulloblastoma treated with a targeted hedgehog pathway inhibitor

We report on 3 children treated with vismodegib who developed widespread growth plate fusions that persist long after cessation of therapy.