Focus on Improving Clinical Outcomes for Pediatric Hematologic Disorders

Known as an "orphan" disease due to the low incidence of occurrence, pediatric acquired aplastic anemia affects approximately two to four children out of every million each year.

Researchers from the Angie Fowler Adolescent & Young Adult Cancer Institute at University Hospitals Rainbow Babies & Children's Hospital (UH Rainbow) will present data focused on improving clinical outcomes for pediatric hematologic disorders at the 58th Annual Meeting of the American Society of Hematology (ASH).

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‘Data focused on improving clinical outcomes for pediatric hematologic disorders was presented at the 58th Annual Meeting of the American Society of Hematology (ASH).’

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The meeting, held from December 3 to 6, 2016, in San Diego, California, gathers a global community of more than 20,000 hematologists to share education and research on the most pressing topics in hematology.

Ashish Gupta, a pediatric hematology fellow at UH Rainbow, will share results from one of the largest quality controlled retrospective studies of children with acquired aplastic anemia. The data makes a compelling case for the pediatric hematology community to revisit the current treatment algorithm for this rare disease.

The small population makes robust outcomes research difficult to come by. Dr. Gupta and colleagues collected 10 years' worth of data about more than 5,000 children with acquired aplastic anemia from the quality-controlled Pediatric Health Information Systems (PHIS) database to analyze the effectiveness of the current treatment algorithm and compare it to the effectiveness of newer therapeutic approaches. The PHIS database includes data from 45 U.S. children's hospitals.

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"Today, if a child with aplastic anemia has a matched related donor, we recommend proceeding with bone marrow transplant," said Dr. Gupta. "If no such optimum donor exists, as is true for almost 80% of children, immunosuppressive therapy (IST) is the standard of care, despite high rates of disease recurrence following treatment conclusion. As the outcomes of matched unrelated donor transplant options have improved, we wanted to know if patients with aplastic anemia were also experiencing better long term outcomes compared to immunosuppressive therapy, which is known to have high failure rates. Incidence of complications with bone marrow transplant also increases with delay in transplant."

Through extensive data analysis, the research team found that outcomes were comparable between matched related bone marrow transplant and matched unrelated donor transplants. Interestingly, common post-transplant complications such as graft versus host disease or graft failure were actually lower for patients who underwent a transplant from a matched unrelated donor.

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"Children particularly tolerate transplants better than adults," added Dr. Gupta. "It is reasonable to recommend matched unrelated donor transplants in place of IST if a suitable donor is available."

Dr. Gupta's presentation appears as part of the ASH Poster Session (Abstract 2395) entitled Outcomes Research-Non-Malignant Conditions. Another UH Rainbow presenter, Irina Pateva, pediatric hematology/oncology at UH Rainbow and Assistant Professor of Pediatrics at Case Western Reserve University School of Medicine, will present findings in red blood cell transfusions for critically ill children as part of the Basic Science and Clinical Practice in Blood Transfusion Poster Session. Additionally, UH Seidman Cancer Center physicians will present data on five additional clinical research topics in adult hematology and oncology.

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"ASH is one of the premier meetings for all hematologists," said Jignesh Dalal, Director, Pediatric Bone Marrow Transplant at UH Rainbow and Professor of Pediatrics at Case Western Reserve University School of Medicine. "The pediatric hematology team at UH Rainbow is world-class and focused on improving outcomes for all children. Selection to present at this meeting reflects this fact and will greatly enhance the body of knowledge we use to care for children around the world."

Source-Eurekalert