Project Term
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Project Summary
While many immunotherapy studies in children with leukemia focus on establishing early responses, this project studies what life is like, and what health and social challenges are experienced, in those who survive their leukemia long-term. Using data from over 700 patients across many hospitals, we aim to identify factors that predict long-term remission, side effects, and quality of life. We will also collect patient-reported experiences to better understand survivorship and improve care.
Lay Abstract
CAR T-cell therapy is a form of immunotherapy that has transformed outcomes for children and young adults with B-cell acute lymphoblastic leukemia (B-ALL), offering the possibility of long-term remission for patients with otherwise limited treatment options. However, while many patients achieve remission, we still do not fully understand why some remain cancer-free while others relapse, or why some develop long-term side effects after treatment.
This project brings together a network of approximately 50 hospitals across North America through the Pediatric Real-World CAR Consortium to better understand what happens to patients after CAR T-cell therapy, and what health and socioeconomical challenges survivors face. We have already collected detailed clinical data from more than 600 children and young adults treated with CAR T cells, creating one of the largest datasets of its kind studying long-term outcomes in children with leukemia treated with CAR T cells. This includes information about patients, their disease, treatments, outcomes, and complications during treatment and over time.
Using this dataset, we will study patterns of survival, relapse, and long-term side effects. We aim to identify key factors, such as disease burden at treatment, prior therapies, and markers of immune function, that help predict whether a patient will achieve a lasting cure versus a brief non-durable remission. By applying advanced statistical methods and machine learning, we plan to develop a risk score that can help doctors estimate an individual patient’s likelihood of long-term success after CAR T-cell therapy. This will help guide treatment through anticipating if patients are likely to have a durable cure and can be spared additional toxic therapies, or if CAR T cells as a single therapy are likely to only achieve a brief response and subsequent therapies are warranted to achieve long-term cure.
In addition to clinical outcomes, this project focuses on the patient experience. We are collecting patient-reported outcomes through a survey called WeCARe (We’re Evaluating CAR Effects), which captures information not typically found in medical records. This includes quality of life, cognitive and physical functioning, and challenges such as access to care, financial strain, and other social factors that may affect recovery.
By integrating clinical data with patient-reported experiences, we aim to provide a more complete picture of survivorship after CAR T-cell therapy. Ultimately, this work will help guide follow-up care, identify patients who may benefit from additional treatments, and inform future therapies designed to improve both survival and quality of life for children with leukemia.
Program
