When the pediatric hematology saints went marching into #ASH22, oh how we all wanted to be in that number. From new breakthroughs in our understanding of infant iron deficiency anemia to novel agents in the treatment of childhood acute leukemia, the 64th ASH Annual Meeting and Exposition in New Orleans, Louisiana, was brimming — like a trumpeter down on Basin Street — with awe-inspiring talent and wonder.
Oh, and did that sun begin to shine!
Our pediatric ASH parade kicked off with a look into the neurocognitive complications and associated epigenetic changes of survivors of pediatric Hodgkin lymphoma. We know that long-term survivors of childhood cancer are at an increased risk of early cognitive degeneration, but now we have biological evidence – epigenetic proof, to be exact – of accelerated cognitive aging in these patients. Utilizing genome-wide DNA methylation analysis, Dr. AnnaLynn Williams and her team showed that long-term survivors of Hodgkin lymphoma experienced statistically significant epigenetic age acceleration, manifesting clinically as deficits in memory, visual and motor processing, and executive functioning. I can’t help but wonder: are we looking at the next big tool to assess cognitive function in pediatric cancer survivors? Time will tell…
Oh, a new world is revealed!
The march continued down the Mighty Mississippi with groundbreaking updates in targeted therapy for pediatric leukemia. Dr. Sarah Tasian explored the unique pathophysiology of Philadelphia chromosome-like acute lymphoblastic leukemia (Ph-like ALL), a high-risk leukemia subtype that, despite its title, does not arise from the classic t(9;22) BCR::ABL1 fusion protein. Patients with Ph-like ALL express activating mutations in tyrosine kinase signaling pathways, and numerous clinical trials evaluating the therapeutic efficacy of tyrosine kinase inhibitors in pediatric Ph-like ALL show great promise. Dr. Katherine Tarlock similarly delved into the exciting new targeted approaches for pediatric acute myelogenous leukemia (AML), such as of FMS-like tyrosine kinase 3, menin, IDH, and BCL-2 inhibitors. Dr Tarlock also touched upon several AML blast-specific targets that may be ideal for chimeric antigen receptorT-cell immunotherapy, including mesothelin and folate receptor 1 (FOLR1). Dr. Rishi Sury Kotecha rounded out the educational program with novel insights into the treatment of infant ALL, a notoriously aggressive cancer with generally dismal prognosis. However, targeted therapies, especially against MLL fusion proteins, may offer new hope in our battle against infant ALL.
It didn’t end there, no! Topics in classical hematology glistened like Café du Monde’s delicious, powdery beignets at #ASH22. Dr. George Goshua (shown left), presented his team’s efforts to create a disease simulation of alloimmunized patients with sickle cell disease (SCD). Their simulation predicted that a U.S.-wide alloantibody exchange for SCD would not only reduce the cumulative lifetime risk of delayed hemolytic transfusion reaction-specific mortality, but also save upwards of $1.5 billion health care dollars. Though their findings pertain solely to SCD, a world of opportunity exists for alloantibody exchange systems in other hematologic conditions, and the savings — both to cost and life — are potentially enormous. Grace Power, BSc, (shown right) of Dalhousie University further highlighted a significantly increased prevalence of iron deficiency in formula fed infants when compared to breastfed infants. Ms. Power and her team suggest that this discrepancy is likely secondary, in part, to the absence of lactoferrin in formula, which impairs iron absorption. Their results suggest that current international guidelines regarding iron supplementation in infancy may be strikingly inadequate, and a new risk-stratified approach to iron supplementation may be more effective.
No doubt, there is a lot to sing about in the world of pediatric hematology today – and, oh, what a wonderful world it is! The legendary Louis Armstrong would be proud. So, looking ahead to next year, when the pediatric hematology saints go marching into #ASH23 – po’ boys and beignets a-plenty – ask yourself this: Do you want to be in that number?
Dr. Jonathan Hermel and Dr. David Hermel indicated no relevant conflicts of interest.