HEMATOLOGY, ONCOLOGY AND STEM CELL TRANSPLANT
RESEARCH HIGHLIGHTS
Riley Children’s is engaged in leading-edge pediatric oncology and hematology clinical trials through membership in the following organizations: ■ Children’s Oncology Group ■ Early Phase Network ■ Therapeutic Advances in Childhood Leukemia ■ Pacific Pediatric Neuro-Oncology Consortium ■ Neurofibromatosis Clinical Trials Consortium ■ Consortium of Investigators of Vascular Anomalies ■ North American Consortium for Histiocytosis ■ Sickle Cell Transplant Advocacy and Research Alliance ■ Pediatric Transplantation and Cellular Therapy Consortium ■ Bone Marrow Transplant Clinical Trials Network ■ North American Pediatric Aplastic Anemia Consortium ■ Globin Regional Data and Discovery Network (Sickle cell registry)
Expert oncologists and hematologists at Riley Children’s Health combine leading- edge medicine with holistic, personalized care to treat children with cancer and blood disorders. With access to the latest clinical trials and therapeutic options, our skilled specialists treat the full spectrum of pediatric cancers, including aggressive disease and cancers that are no longer responsive to conventional therapy.
DIVISION CHIEF Brian D. Weiss, MD Professor of Clinical Pediatrics, IU School of Medicine > Connect with Dr. Weiss on Doximity.
PROGRAM DISTINCTIONS
Specialized programs ■ Precision Genomics Program ■ Neurofibromatosis Program ■ Stem Cell/Immunotherapy Program
■ Ranked among the leading pediatric cancer programs in the nation by U.S. News & World Report ■ Home to the only pediatric stem cell transplant and CAR-T cell therapy programs in Indiana ■ Part of the only National Cancer Institute- designated Comprehensive Cancer Center in Indiana ■ Largest comprehensive sickle cell program in Indiana: – Only National Institutes of Health (NIH)- funded telemedicine program in the nation for pediatric sickle cell disease – One of 40 institutions in the National Alliance of Sickle Cell Disease Centers – Only Lifespan Sickle Cell Disease Center of Excellence in the state ■ Early access to clinical trials and newly developed cancer treatments and research through the Children’s Oncology Group Phase I and Pilot Consortium ■ One of the few children’s hospitals in the nation with a multidisciplinary treatment program for neurofibromatosis ■ One of the few programs regionally to offer an experimental testicular tissue cryopreservation protocol
38 board certified physicians
See full team member list >
■ Comprehensive Bleeding Program and Women and Girls Blood Disorder Clinic of Excellence ■ Thrombosis and Thrombophilia Program ■ Sickle Cell Disease Program (includes a multidisciplinary program for high-risk patients) ■ Fertility Preservation Program ■ Survivorship Program ■ Developmental Therapeutics Program ■ Adolescent and Young Adult Oncology Program Our program is affiliated with the Brown Center for Immunotherapy at IU School of Medicine (offering access to investigator-initiated clinical trials, including a CD4 CAR-T trial) and the Indiana University Gene Therapy Testing Laboratory—the second-largest clinical grade viral vector lab in the nation. Our program is accredited or certified by: ■ Foundation for the Accreditation of Cellular Therapy ■ Association for the Advancement of Blood & Biotherapies ■ American Society for Histocompatibility & Immunogenetics Advanced therapies and procedures ■ CAR-T trials for leukemia (PLAT Phase I and Phase II trials) ■ Gene therapy for beta thalassemia and sickle cell disease (Bluebird Bio and Vertex) ■ NK cell infusions following stem cell transplant
NOTABLE PUBLICATIONS Clinical Cancer Research: September 2023 Combined CDK4/6 and ERK1/2 Inhibition Enhances Antitumor Activity in NF1-Associated Plexiform Neurofibroma Journal of Clinical Oncology: December 2023 Prospective External Validation of the Esbenshade Vanderbilt Models Accurately Predicts Bloodstream Infection Risk in Febrile Non-Neutropenic Children With Cancer The Lancet, Child and Adolescent
> View active hematology and oncology clinical trials at Riley Children’s.
Research study topics ■ New detection techniques and treatments for sarcoma in patients with neurofibromatosis type 1 ■ New therapies for bleeding disorders ■ Novel combination therapies to target replication stress in models of osteosarcoma ■ Determining how newly discovered T cell-specific long non-coding RNA genes may improve outcomes of acute graft-versus-host disease and other T cell-mediated diseases
■ Only NIH-funded telemedicine study in the nation for pediatric sickle cell disease ■ 4 research grants awarded in 2023 ■ 12 open hematology studies focused on sickle cell disease, bone marrow failure, immune thrombocytopenia and thrombosis
Health: November 2023 The Evolving Treatment Landscape for Children with Sickle Cell Disease
133 research publications in 2023
Stem Cell Transplant and Immunotherapy Volume: 2023 28 Autologous stem cell transplants 22 Allogeneic matched (related or unrelated) donor transplants 11 Cellular therapy treatments, including infusions of CAR-T, viral specific T cells, mesenchymal stem cells or NK cells
Riley Children’s achieved a 100%, 100-day survival rate for allogeneic transplants in 2023.
Five-year survival rates
80% overall
93% for leukemia patients
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Annual Report 2023 – 2024
317.944.2143
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