Approximately 1 year after the US FDA approved CAR T-cell therapy for children with acute lymphoblastic leukemia (ALL), researchers at The University of Texas MD Anderson Cancer Center (TX, USA) and the Pediatric Acute Lung Injury and Sepsis Investigators Network (PALISI) have published treatment guidelines for managing the treatment in Nature Reviews Clinical Oncology.
“CAR T-cell therapy has been associated with remarkable response rates for children and young adults with ALL, yet this innovative form of cellular immunotherapy has resulted in unique and severe toxicities which can lead to rapid cardiorespiratory and/or neurological deterioration,” explained first author Kris Mahadeo (University of Texas MD Anderson Cancer Center). “This novel therapy requires the medical vigilance of a diverse multi-disciplinary team and associated clinical infrastructure to ensure optimal patient outcomes.”
In 2017, MD Anderson published guidelines on the management of adult patients receiving CAR T-cell therapy. However, early signs and symptoms of toxicity in children brought attention to pediatric-specific monitoring including escalation of care based on parent and caregiver concerns.
Some examples of the recommendations include:
Monitoring for cytokine release syndrome (CRS) using pediatric normal ranges for organ function.
Promptly addressing parent and/or caregiver concerns as early signs or symptoms of CRS can be subtle and best recognized by those who know the child best.
MD Anderson’s CAR T-cell-therapy-associated Toxicity (CARTOX) program collaborated with PALISI and its Hematopoietic Stem Cell Transplantation (HSCT) sub-group in creating the comprehensive guidelines for treating children with cancer receiving CAR T-cell therapy.
“CARTOX, which oversees care for MD Anderson CAR T-cell therapy patients, is the first stand-alone immune effector cellular therapy program to earn accreditation from the Foundation for the Accreditation of Cellular Therapy (FACT),” explained senior author Elizabeth Shpall (University of Texas MD Anderson Cancer Center). “The program provides oversight for more than 20 active immune effector cell research protocols and two approved standard of care therapies at MD Anderson, and it is clear these new guidelines will serve as an important new model for care of CAR T-cell patients.”