Researchers from the Angie Fowler Adolescent & Young Adult Cancer Institute at University Hospitals Rainbow Babies & Children's Hospital (UH Rainbow) presented data focused on improving clinical outcomes for paediatric hematologic disorders 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 haematologists to share education and research on the most pressing topics in haematology.

Ashish Gupta, MBBS, MPH, a paediatric haematology fellow at UH Rainbow, will share results from one of the largest quality controlled retrospective studies of children with acquired aplastic anaemia.

The data makes a compelling case for the paediatric haematology community to revisit the current treatment algorithm for this rare disease.

Known as an "orphan" disease due to the low incidence of occurrence, paediatric acquired aplastic anaemia affects approximately 2 to 4 children out of every million each year.

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 anaemia from the quality-controlled Paediatric Health Information Systems (PHIS) database to analyse 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.

"Today, if a child with aplastic anaemia 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 percent 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 anaemia 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.

"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 appeared as part of the ASH Poster Session (Abstract 2395) entitled Outcomes Research-Non-Malignant Conditions.

Another UH Rainbow presenter, Irina Pateva, MD, paediatric haematology/oncology at UH Rainbow and Assistant Professor of Paediatrics 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 haematology and oncology.

"ASH is one of the premier meetings for all haematologists," said Jignesh Dalal, MD, Director, Paediatric Bone Marrow Transplant at UH Rainbow and Professor of Paediatrics at Case Western Reserve University School of Medicine. "The paediatric haematology 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: University Hospitals Cleveland Medical Center