Pediatric blood & cancer
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Pediatric blood & cancer · Mar 2013
Phase I trial and pharmacokinetic study of sorafenib in children with neurofibromatosis type I and plexiform neurofibromas.
Sorafenib targets multiple pathways thought to be crucial in growth of plexiform neurofibroma (PN) in children with neurofibromatosis type 1 (NF1). Sorafenib has been tolerated with manageable toxicities in adults and children with refractory cancer. We conducted a separate study in this population. Monitoring long-term toxicities such as effects on growth and obtaining additional pharmacokinetic data were of importance due to the young age and long duration of therapy seen in previous phase I trials in children with NF1. ⋯ Children with NF1 and PN did not tolerate sorafenib at doses substantially lower than the MTD in children and adults with malignant solid tumors. Future trials with targeted agents for children with NF1 may require a more conservative starting dose and separate definitions of dose limiting toxicities (DLT) than children with cancer.
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Pediatric blood & cancer · Mar 2013
Clinical TrialEfficacy and safety of transdermal buprenorphine in the management of children with cancer-related pain.
The current study investigated the efficacy, safety, tolerability, and compliance of a transdermal buprenorphine delivery system for the management of chronic cancer pain in the pediatric population. ⋯ Transdermal buprenorphine was found to represent an efficient, safe and well tolerated approach to the management of children's chronic cancer pain.
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Pediatric blood & cancer · Mar 2013
PROMIS pediatric measures in pediatric oncology: valid and clinically feasible indicators of patient-reported outcomes.
Establishing the ability of children and adolescents with cancer to complete the NIH-sponsored PROMIS pediatric measures electronically and the preliminary validity estimates of the measures (both full item banks and short forms) in pediatric oncology will contribute to our knowledge of the impact of cancer treatment on these young patients. ⋯ Children and adolescents in treatment for cancer or in survivorship and ranging from 8 to 17 years of age can complete multiple PROMIS pediatric measures using a computer interface during an outpatient clinic visit or inpatient admission. Findings establish known-group validity for PROMIS pediatric measures in pediatric oncology.