Pediatric Hematology-Oncology Specialists Report Significant Research Findings at Major Annual Conference

Investigators from Hackensack University Medical Center, including Hackensack Meridian Children’s Health and Hackensack Meridian John Theurer Cancer Center (part of the National Cancer Institute-designated Lombardi Comprehensive Cancer Center at Georgetown University), presented findings from their investigations assessing new diagnostic and treatment approaches for noncancerous blood disorders and blood cancers in children and adolescents at the 65th Annual Meeting of the American Society of Hematology. 

"While we have made great strides in curing many children with blood cancers, others continue to have a high rate of relapse," explained Burton Appel, MD, MBA, associate professor of Pediatrics, Hackensack Meridian School of Medicine and associate director, Children’s Cancer Institute, Joseph M. Sanzari Children’s Hospital. "Our researchers are helping to advance the field by assessing new approaches to stem cell transplantation, gene therapy for sickle cell disease, and other methods to improve outcomes in young patients with malignant and nonmalignant blood diseases.”

Pediatric hematology-oncology studies presented by Joseph M. Sanzari physicians include:

PET Has Value for Guiding Hodgkin Lymphoma Treatment. (Abstract 1699, Burton Appel, MD) Positron emission tomography (PET) plays an important role in staging and assessing treatment response in classic Hodgkin lymphoma, but little has been published about its use in patients with nodular lymphocyte-predominant Hodgkin lymphoma (nLPHL). As part of a Children’s Oncology Group study, researchers showed that PET response following three cycles of chemotherapy was highly predictive of outcome in pediatric patients with low-risk nLPHL, and that intermediate-risk patients with negative PET following chemotherapy achieved excellent outcomes.

Lovo-Cel Gene Therapy Is Safe and Effective for Sickle Cell Disease. (Abstract 1051 and Oral Presentation, Stacey Rifkin-Zenenberg, DO) This study reports 5 years of follow-up data from two of the largest clinical trials of gene therapy to date for sickle cell disease in adults as well as patients ages 12-17. There was complete resolution of vaso-occlusive events (VOEs) in 91% of patients and complete resolution of severe VOEs in 97% of patients within 6-18 months of a single lovo-cel infusion. Pain intensity, pain interference, and fatigue also improved over time. Ongoing long-term follow-up is continuing.

HLA-DRB1 HED Predicts Improved Survival After a Donated Stem Cell Transplant. (Abstract 2232, Christine Camacho-Bydume, MD) Despite advances in disease monitoring and maintenance therapies, the risk of relapse after allogeneic (donated) stem cell transplantation in patients with blood cancers remains a major concern. High HLA evolutionary divergence (HED) promotes a diverse repertoire of antigens, which may heighten tumor surveillance and enhance the ant-cancer effect of the graft. This study showed that high HLA-DRB1 HED led to better survival and a lower risk of relapse in patients with hematologic malignancies after allogeneic hematopoietic stem cell transplantation. HLA-DRB1 may play a more integral role in tumor surveillance, which may have important implications for donor selection, particularly HLA-mismatched settings.

Pediatric Patients with Immune Thrombocytopenia (ITP) May Be Weaned from Medication. (Abstract 3960, Melanie Degliuomini, MD) Thrombopoietin receptor agonists (TPO-RAs) can be discontinued in adults, but there has been little in the literature about waning children with ITP from these drugs. This study showed that 18% of pediatric patients with ITP could be successfully weaned with a low rate of ITP exacerbation. It was easier to wean patients from TPO-RAs quickly if treatment was initiated within 3 months of diagnosis. TPO-RA treatment duration was also shorter for patients who started treatment within 12 months of diagnosis.

Lab Study Demonstrates Role of Cytotoxic CD8 T Cells. (Abstract 2056, Johannes Zakrzewski, MD) The Artificial Immune Modulation (AIM) platform mimics natural dendritic cell function. In this lab-based study, researchers used the AIM platform to show that cytotoxic CD8 T lymphocytes influence the effectiveness of T-cell engager therapies.