Cord Blood Transplant for Non-Malignant Disorders

The Cord Blood Transplant for Non-Malignant Disorders webinar, presented by the ASTCT Cord Blood SIG, was aired on Wednesday, May 20 at 12 p.m. CT/1 p.m. ET. The session consisted of two 25-minute presentations by Dr. Caridad Martinez and Dr. Joanne Kurtzberg, followed by a 10-minute Q&A session.

Session Description:

This webinar provide an overview of the role of cord blood transplantation in the treatment of non-malignant disorders, highlighting its unique advantages as a stem cell source. The first presentation focuses on the use of cord blood transplantation for Severe Combined Immunodeficiency (SCID), including indications, timing, and clinical outcomes. The second presentation addresses cord blood transplantation for Krabbe disease, with emphasis on early intervention, disease modification, and long-term patient outcomes. Together, these talks offer practical insights into patient selection, transplant strategies, and emerging considerations in this evolving field.

Presenters:

Caridad Martinez, MD

Texas Children's Hospital, Baylor College of Medicine; Houston, TX

Joanne Kurtzberg, MD

Duke University School of Medicine; Durham, North Carolina

Moderator:

Alan Bidgoli, DO

Children’s Healthcare of Atlanta, Emory University School of Medicine; Atlanta, GA

Session Learning Objectives:

Upon completion of the activity, participants should be able to:

• Describe the principles and indications of cord blood transplantation in non-malignant disorders, including its advantages compared to other stem cell sources.
• Explain the role of cord blood transplantation in the management of Severe Combined Immunodeficiency (SCID), including patient selection, timing of transplant, and outcomes.
• Summarize the pathophysiology and clinical progression of Krabbe disease and outline how cord blood transplantation modifies disease course when performed early.
• Compare transplant approaches, conditioning regimens, and clinical outcomes between cord blood transplantation for SCID and Krabbe disease to evaluate factors influencing treatment success.