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