Curative Therapies for Bone Marrow Failure

Aug 18, 2021 from 8 a.m. - 9 a.m. (ET)

Bone marrow failure in children arises either from immune dysregulation or genetic predisposition. The advent of readily available germline and somatic genetic platforms now allow for an accurate diagnosis in the majority of children. Dr. Olsen will discuss recent advances in the diagnostics and classification of bone marrow failure disorders. He will also discuss recent data regarding outcomes for patients with specific disorders, and how these data have led to recent changes in indications for HSCT. Finally, he will discuss recent clinical trial data exploring methods to make alternative donor transplant for bone marrow failure safer and more efficacious.

Learning objectives

  1. Review an update on diagnostic approaches for inherited and acquired Bone Marrow Failure (BMF) syndromes.
  2. Discuss treatment algorithms in Bone Marrow Failure, with an emphasis on HSCT indications.
  3. Describe novel approaches to HSCT for BMF syndromes aimed at reduced toxicity and improving success.




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ACCME Accreditation Statement: Children’s Hospital of Philadelphia is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

AMA Credit Designation Statement: Children's Hospital of Philadelphia designates this live activity for a maximum of 1.00 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Disclosure Statement: Timothy Olson, MD (Speaker), Alix Seif, MD (Moderator), Katie Lockwood, MD, MEd, (CHOP Talks Course Director), and Monica S. Williams (CME Planner) and others as appropriate have no relevant financial relationship with a commercial interest to disclose.


Shelly Norbury


Related specialties: hematology, oncology, general pediatrics