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Now showing items 71-80 of 124
100,000 Genomes Pilot on Rare-Disease Diagnosis in Health Care - Preliminary Report.
(MASSACHUSETTS MEDICAL SOC, 2021-11-11)
BACKGROUND: The U.K. 100,000 Genomes Project is in the process of investigating the role of genome sequencing in patients with undiagnosed rare diseases after usual care and the alignment of this research with health care ...
Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: phase 1 trial results.
(NATURE PORTFOLIO, 2023-05-01)
Neuroblastomas harbor ALK aberrations clinically resistant to crizotinib yet sensitive pre-clinically to the third-generation ALK inhibitor lorlatinib. We conducted a first-in-child study evaluating lorlatinib with and ...
BRIM-P: A phase I, open-label, multicenter, dose-escalation study of vemurafenib in pediatric patients with surgically incurable, BRAF mutation-positive melanoma.
(WILEY, 2018-05-01)
BACKGROUND: Vemurafenib, a selective inhibitor of BRAF kinase, is approved for the treatment of adult stage IIIc/IV BRAF V600 mutation-positive melanoma. We conducted a phase I, open-label, dose-escalation study in pediatric ...
Outcome after treatment for sebaceous carcinoma: A multicenter study.
(WILEY, 2022-03-01)
BACKGROUND: Sebaceous carcinoma (SC) is a rare malignant tumour whereby, comprehensive long-term data are scarce. This study aimed to assess the outcome of patients treated with resection for SC. METHODS: Patients treated ...
Molecular testing of rhabdomyosarcoma in clinical trials to improve risk stratification and outcome: A consensus view from European paediatric Soft tissue sarcoma Study Group, Children's Oncology Group and Cooperative Weichteilsarkom-Studiengruppe.
(ELSEVIER SCI LTD, 2022-09-01)
Rhabdomyosarcomas (RMSs) are the most common soft tissue sarcomas in children/adolescents less than 18 years of age with an annual incidence of 1-2/million. Inter/intra-tumour heterogeneity raise challenges in clinical, ...
A randomized phase III study of 72 h infusional versus bolus bleomycin in BEP (bleomycin, etoposide and cisplatin) chemotherapy to treat IGCCCG good prognosis metastatic germ cell tumours (TE-3).
(OXFORD UNIV PRESS, 2017-06-01)
BACKGROUND: Bleomycin is an integral part of combination chemotherapy in germ cell tumours. Pulmonary toxicity often necessitates drug cessation and death occurs in 1%-2% of patients. A continuous infusion of bleomycin ...
Mutations in Epigenetic Regulation Genes Are a Major Cause of Overgrowth with Intellectual Disability.
(CELL PRESS, 2017-05-04)
To explore the genetic architecture of human overgrowth syndromes and human growth control, we performed experimental and bioinformatic analyses of 710 individuals with overgrowth (height and/or head circumference ≥+2 SD) ...
Prevalence and impact of severe fatigue in adolescent and young adult cancer patients in comparison with population-based controls.
(SPRINGER, 2017-09-01)
PURPOSE: The current study determined the prevalence of severe fatigue in adolescent and young adult (AYA) cancer patients (aged 18-35 years at diagnosis) consulting a multidisciplinary AYA team in comparison with gender- ...
Adolescent and young adult (AYA) lymphoma survivors report lower health-related quality of life compared to a normative population: results from the PROFILES registry.
(TAYLOR & FRANCIS LTD, 2017-02-01)
BACKGROUND: Trying to simultaneously achieve developmental milestones and cope with a life-threatening disease may place adolescents and young adults (AYAs) at risk for impaired health-related quality of life (HRQoL) later ...
Normal Tissue Complication Probability (NTCP) Modelling of Severe Acute Mucositis using a Novel Oral Mucosal Surface Organ at Risk.
(ELSEVIER SCIENCE LONDON, 2017-04-01)
AIMS: A normal tissue complication probability (NTCP) model of severe acute mucositis would be highly useful to guide clinical decision making and inform radiotherapy planning. We aimed to improve upon our previous model ...