Children’s Hospital of Pittsburgh of UPMC Experts Receive Research Awards From Patient-Centered Outcomes Research Institute

PITTSBURGH, PA - June 17, 2013 - Two experts at Children's Hospital of Pittsburgh of UPMC have been selected for research awards by the Patient-Centered Outcomes Research Institute (PCORI), one for $2 million to develop an online tool for the management of sickle cell disease, and one for $1 million to improve detection and intervention in cases of child abuse through the use of electronic medical records.

Lakshmanan Krishnamurti, M.D., and Rachel P. Berger, M.D., M.P.H., are leading two of 51 PCORI projects totaling $88.6 million over three years that PCORI has approved to conduct patient-centered comparative clinical effectiveness research (CER) projects.

Dr. Krishnamurti, clinical director of hematology in the Division of Hematology/Oncology at Children's Hospital, is the lead investigator on a research project to create an online tool for people with sickle cell disease (SCD). SCD is an inherited disorder that affects 90,000 individuals in the United States, largely of minority origin, according to the American Journal of Hematology. SCD is associated with chronic multisystem manifestations as well as substantial morbidity, premature mortality, individual suffering, health care costs and loss of productivity.

"Sickle cell disease is a serious disease with multiple treatment options such as a drug called hydroxyurea which modifies the course of the disease, monthly blood transfusions that decrease the proportion of abnormal red blood cells in the circulation, and bone marrow transplantation that replaces the patient's marrow with that of marrow from a matched healthy donor and essentially eliminates the production of abnormal red blood cells," Dr. Krishnamurti said.

"These treatments can prevent complications and improve the lives of patients, but they can be associated with significant side effects. It can be difficult for families to make decisions on the treatment that is best for them, their disease severity and their preferences. This tool will help patients and families make informed decisions about the treatments that are best for their individual situation and in keeping with their values and preferences."

Dr. Berger, member of the Child Protection Team in the Child Advocacy Center at Children's Hospital, will lead a research project using Children's Hospital's electronic medical record to help improve outcomes for children of abuse and decrease disparities in screening for child physical abuse.

"We are honored to have been awarded this grant from PCORI," said Dr. Berger. "Child abuse truly is an under-recognized public health epidemic and is one of the leading causes of death and disability of infants and toddlers. The key to decreasing the morbidity and mortality is early detection and intervention."

"The goal of our study is to determine whether we can improve and standardize the way in which physicians and other health care professionals screen for physical abuse in infants and toddlers. By standardizing screening, we hope to decrease or even eliminate the racial disparities that currently exist in which infants and toddlers receive the recommended screening," she added.

"These projects reflect PCORI's commitment to support patient-centered comparative effectiveness research, a new approach to health research that emphasizes the inclusion of patients and caregivers at all stages of the study process," said Joe Selby, M.D., M.P.H., executive director, PCORI. "The research will provide patients and those who care for them, better information about the health care decisions they face."

All projects were selected through a highly competitive review process in which scientists, patients, caregivers and other stakeholders helped evaluate more than 400 applications for funding. Proposals were evaluated on the basis of scientific merit, how well they engage patients and other stakeholders, their methodological rigor, and how well they fit within PCORI's national research priorities.

The awards are part of PCORI's second cycle of primary research funding. This new round of funding follows PCORI's initial approval of $40.7 million in support for 25 projects under the institute's national research priorities. All awards in this most recent round of funding were approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.

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About Children's Hospital of Pittsburgh of UPMC
Renowned for its outstanding clinical services, research programs and medical education, Children's Hospital of Pittsburgh of UPMC has helped establish the standards of excellence in pediatric care. From ambulatory care to transplantation and cardiac care, talented and committed pediatric experts care for infants, children and adolescents who make more than 1 million visits to Children's and its many neighborhood locations each year. Children's also has been named consistently to several elite lists of pediatric health care facilities, including ranking 7th among children's hospitals and schools of medicine (FY 2011) in funding provided by the National Institutes of Health, and is one of 10 pediatric hospitals in the United States named to U.S. News & World Report's Honor Roll of America's "Best Children's Hospitals" for 2013–2014.

About PCORI
The Patient-Centered Outcomes Research Institute (PCORI) is an independent, non-profit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed health care decisions. PCORI is committed to continuously seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.

Andrea Kunicky, 412-692-6254, andrea.kunicky@chp.edu

Marc Lukasiak, 412-692-7919, marc.lukasiak@chp.edu