Treating Little Heroes

May 12, 2023

Cancer is never on the agenda for one’s childhood. When illness strikes, everyone wants to see children get well as quickly, safely, and permanently as possible. The Cancer Center at Children’s Hospital of Philadelphia (CHOP) is a pediatric cancer treatment facility staffed by internationally recognized pediatric oncologists. The CHOP physicians, who have expertise in the rarest and most common pediatric cancers, work together to diagnose and treat patients using an evidence-based approach backed by the latest research advancements. It is not a surprise that the US News and World Report rated CHOP as the best children’s cancer hospital for 2022/2023, as each child, at every stage of treatment, has a personalized care plan and is getting the best possible recommendations, and some of the most significant breakthroughs in the treatment of pediatric cancers have happened there.

Cancer is not the only life debilitating illness. The Division of Hematology at CHOP provides a multidisciplinary team and services for children and adolescents with all hematologic disorders (other than malignancies). Such conditions and life-threatening diseases require top of the line care, and the OMI was thrilled to have world-renowned physicians at Schloss Arenberg this week, from May 7-13, 2023, for the Salzburg CHOP Seminar in Pediatric Hematology and Oncology.

Dr. Alix Seif, Associate Professor at the Center for Childhood Cancer Research, Children’s Hospital of Philadelphia, and Dr. Michele P. Lambert, Associate Professor in the Department of Pediatrics at the Perelman School of Medicine of the University of Pennsylvania and Attending Physician in the Division of Hematology at Children’s Hospital of Philadelphia, served as the course directors for the seminar. Dr. Leo Kager, Professor of Pediatrics at the Medical University of Vienna and Head of the Outpatient Department of Hematology and Oncology at Saint Anna Children’s Hospital, co-led the course. Dr. Kavita Desai, Dr. Jason L. Freedman, and Dr. Milen Minkov completed the group of faculty members.

The faculty presented an incredible series of lectures for the fellows, including topics such as Brain Tumors, Acute Lymphoblastic Leukemia, Novel Therapies for Hemoglobinopathies, Hemophagocytic Lymphohistiocytosis, Immune Dysregulation, Supportive and Palliative Care, and more!
  • 27 fellows
  • 18 countries
  • 11th course in Pediatric Hematology and Oncology

Content Focal Points: Hematology

Our understanding of the pathophysiologic processes underlying classical hematologic diseases is expanding resulting in the development of novel, targeted therapies for diseases across the spectrum from bleeding disorders and thrombosis to sickle cell disease and thalassemia to the autoimmune cytopenias and thrombotic thrombocytopenic purpura. Understanding the role of immune dysregulation in all aspects of hematology, and leveraging tools developed for oncology, rheumatology and immunology has allowed for targeted treatment with decreased toxicities. Continuing to develop care networks will expand access to care and allow development of novel treatments and ensure access to curative therapies for expanded patient populations.

Content Focal Points: Oncology

We heard about the updated European staging system for rhabdomyosarcoma and novel genomic discoveries in osteosarcoma that may lead to new targeted therapies for this challenging diagnosis. Targeted therapies and precision medicine approaches were presented in a dedicated talk and peppered throughout the sessions, including in her talk on current brain tumor management where she showed dramatic benefits of selumetinib for neurofibromatosis. An outstanding session on rational approaches to state-of-the-art supportive care practices for children undergoing cancer treatment or stem cell transplantation was followed by a review of current practice-changing clinical trial data and emerging immunotherapy approaches for acute leukemias.

In addition to top-of the line lecture content, the fellows had two afternoons to present their case presentations, in which they shared real scenarios they have seen in their personal work or based on patients they are currently treating. This opportunity is invaluable, as the faculty can ask questions, provide advice and feedback, and all learn more about scenarios from around the world via intense discussion. nine cases were chosen as excellent case presentations by the faculty, which will be featured on the OMI’s e-learning platform.

After a great week of learning, networking, and discussion, the fellows marked the end of the seminar week with a celebratory three-course graduation dinner and ceremony. The week may have come to an end, but the personal connections and knowledge gained this week will surely continue with the fellows!

Learning Objectives for Participants:

  1. Review common oncologic presentations, diagnoses, treatments, and advances.
  2. Describe recent diagnostic and therapeutic advances in histiocytic and other immune dysregulation syndromes.
  3. Highlight innovations in use of small molecule inhibitors, targeted immunotherapies, and cellular therapies in stem cell transplantation, solid and liquid tumors.
  4. Enumerate common cancer predisposition and inherited marrow failure syndromes.
  5. Recognize optimal approaches to supportive and palliative care for pediatric cancer patients.
  6. Evaluate novel treatment modalities for non-malignant hematologic disorders.