Long lasting anti-hemophilia factor safe in kids
Recombinant factor VIII fusion protein safely extends time between infusions
2015-04-24
(Press-News.org) Children with hemophilia A require three to four infusions each week to prevent bleeding episodes, chronic pain and joint damage. The effect on quality of life can be significant, due to time and discomfort associated with infusions. For these reasons, under dosing is common, leaving children at increased risk for bleeding episodes and even death.
This extended half-life factor VIII enables patients to receive one or two infusions each week without an increased risk of bleeding. The first report on the safety and efficacy of this therapy in children under 12 years old - led by Guy Young, MD, director of the Hemostasis and Thrombosis Center of Children's Hospital Los Angeles - has been published in the Journal of Thrombosis and Haemostasis.
People with hemophilia are treated with replacement therapy and infused with the clotting factor deficient in their blood. Treatment is prophylactic; individuals receive infusions in order to keep the factor at a protective blood level that will result in clotting if the person is injured. The currently used, standard factor VIII persists in the blood for a relatively short amount of time, making it necessary to infuse it frequently - often every other day.
The new, extended therapy combines recombinant factor VIII with a fusion protein that allows the molecule to remain in the circulation longer - translating into a need for less frequent treatment. Young and his colleagues found that twice-weekly infusions of this novel therapy were well tolerated and resulted in a low incidence of bleeding events in children with severe hemophilia A. They also reported that no patients developed antibodies against the factor, also known as "inhibitors".
"Children's Hospital Los Angeles is home to one of the largest hemophilia treatment centers in the world," said Young. "Through clinical trials, we are able to offer our patients the very latest treatments, often before they are available anywhere else. In this trial, we demonstrated the safety and efficacy of a medication that will allow children with severe hemophilia A to be treated with a safe and effective medicine but with a reduced burden of the treatment." Young is also an associate professor of Pediatrics at the Keck School of Medicine of the University of Southern California.
INFORMATION:
Additional contributors include J. Mahlangu, University of the Witwatersrand, Johannesburg; R. Kulkarni, Michigan State University, East Lansing; B. Nolan, Our Lady's Children's Hospital, Dublin; R. Liesner, Great Ormond Street Hospital for Children, London; J. Pasi, Barts and the London Comprehensive Care Centre, London; C. Barnes, Royal Children's Hospital, Melbourne; S. Neelakantan, G. Gambino, L.M. Cristiano, G.F. Pierce, and G. Allen, Biogen, Cambridge, MA. Funding was provided by Biogen.
About Children's Hospital Los Angeles
Children's Hospital Los Angeles has been named the best children's hospital on the West Coast and among the top five in the nation for clinical excellence with its selection to the prestigious U.S. News & World Report Honor Roll. Children's Hospital is home to The Saban Research Institute, one of the largest and most productive pediatric research facilities in the United States.
For more information, visit CHLA.org. Follow us on our blog http://researchlablog.org/.
Media contact: Ellin Kavanagh, ekavanagh@chla.usc.edu, 323-361-8505 or 323-361-1812
ELSE PRESS RELEASES FROM THIS DATE:
2015-04-24
Ahmad M. Khalil, PhD, knew the odds were against him -- as in thousands upon thousands to one.
Yet he and his team never wavered from their quest to identify the parts of the body responsible for revving up one of the most aggressive forms of breast cancer, HER2+. This month in Breast Cancer Research and Treatment, Khalil and his colleagues at Case Western Reserve University proved the power of persistence; from a pool of more than 30,000 possibilities, they found 38 genes and molecules that most likely trigger HER2+ cancer cells to spread.
By narrowing what was once ...
2015-04-24
TORONTO, April 24, 2015-- Preliminary results of a study of patients with prostate cancer show that MR tractography may be a reliable quantitative imaging biomarker to assess prostate cancer treatment response to androgen deprivation and radiation therapy, according to a team of researchers at Brigham and Women's Hospital and Massachusetts General Hospital in Boston. Quantitative evaluation shows higher tract densities after androgen deprivation and radiation therapy, reflecting gland shrinkage and subsequent fibrosis.
Twenty-two patients with elevated prostate-specific ...
2015-04-24
Stem cells naturally cling to feeder cells as they grow in petri dishes. Scientists have thought for years that this attachment occurs because feeder cells serve as a support system, providing stems cells with essential nutrients.
But a new study that successfully grew stem cells with dead, or fixed, feeder cells suggests otherwise.
The discovery, described in the Journal of Materials Chemistry B, challenges the theory that feeder cells provide nutrients to growing stem cells. It also means that the relationship between the two cells is superficial, according to ...
2015-04-24
Aid workers who provide shelter following natural disasters, such as hurricanes or earthquakes, should consider long-term archaeological information about how locals constructed their homes in the past, and what they do when they repair and rebuild. Archaeologists and international humanitarian organizations are both involved in recovery, with the former doing this for the past, and the latter for the present. So says Alice Samson of the University of Cambridge in the UK, leader of an archaeological overview of building practices used in the Caribbean 1,400 to 450 years ...
2015-04-24
New research published today in the journal Nature Communications represents a potentially fundamental shift in our understanding of how nerve cells in the brain generate the energy needed to function. The study shows neurons are more independent than previously believed and this research has implications for a range of neurological disorders.
"These findings suggest that we need to rethink the way we look at brain metabolism," said Maiken Nedergaard, M.D., D.M.Sc., co-director of the University of Rochester Center for Translational Neuromedicine and lead author of ...
2015-04-24
(April 15th, 2015) Coeliac disease is one of the most common life-long conditions in Europe, yet many people remain undiagnosed and lengthy diagnostic delays may be putting lives at risk. Today, doctors are being urged to consider testing for Coeliac disease in anyone showing signs and symptoms of the condition and to consider screening everyone in high-risk groups.
A paper published in this month's special Coeliac disease (CD) issue of the UEG Journal assessed the viability of screening for CD in the general population and concluded that screening of first-degree relatives ...
2015-04-24
April 24, 2015 - A simple method of testing "twilight vision" gives reliable results in identifying people who have decreased visual acuity under low light conditions, according to a study in the May issue of Optometry and Vision Science, official journal of the American Academy of Optometry. The journal is published by Wolters Kluwer.
Using filters to test at a light level 100 times lower than for daylight visual acuity testing, vision care professionals can obtain "reliable and repeatable" measurements of twilight vision, report Jason S. Ng, OD, PhD, and colleagues ...
2015-04-24
MAYWOOD, Il. - The recent Great Recession was accompanied by a significant and sustained increase in major depression in U.S. adults, according to a Loyola study published in the Journal of Clinical Psychiatry.
Prevalence of major depression increased from 2.33 percent during the years 2005-2006 to 3.49 percent in 2009-2010 to 3.79 percent in 2011-2012, according to the study by Loyola University Chicago Stritch School of Medicine researchers.
Prevalence of less-severe depression increased from 4.1 percent in 2005-2006 to 4.79 percent in 2009-2010, but then declined ...
2015-04-24
Leesburg, VA, April 24, 2015--Digital breast tomosynthesis (DBT) increases the rate of cancer detection in women with dense breast tissue by as much as 67%, according to new research from the Einstein Medical Center in Philadelphia.
"There are a lot of data showing that screening with DBT increases cancer detection, but much less is known about the effect of density and lesion type on detection rates," said coauthor Caroline Ling. "We found a striking increase in detection among women with dense breasts called back for mass and asymmetry relative to nondense breasts."
The ...
2015-04-24
Functional analysis of a cell, which is the fundamental unit of life, is important for gaining new insights into medical and pharmaceutical fields. For efficiently studying cell functions, it is essential to reconstruct cellular microenvironments by parallel manipulation of single cells. Various cell manipulation techniques including fluidic, optical, and electrical techniques have been developed.
However, all these techniques lack flexibility with respect to changes in the cellular types, number, and places. In addition, the manipulations, which have been conducted in ...
LAST 30 PRESS RELEASES:
[Press-News.org] Long lasting anti-hemophilia factor safe in kids
Recombinant factor VIII fusion protein safely extends time between infusions
