Using CD19 chimeric antigen receptor‐T cell therapy in a 4‐month‐old patient with infantile acute lymphoblastic leukemia

Prolonged remission using CD19 chimeric antigen receptor-T cell therapy followed by haploidentical transplantation in a 5-month-old patient with infantile acute lymphoblastic leukemia

“…[1] The approach has been limited due to an infant's smaller size and blood volume; concerns include cellular collection safety, the timing of autologous T-cell harvest during a patient's treatment course, the optimal infusion dose, and the management of post-infusion toxicities. [10,[17][18][19] There are inherent difficulties in obtaining sufficient autologous T-cells in infants to generate a suitable CAR-T product. [17][18][19][20][21] Whether CAR-T therapy alone is satisfactory to achieve durable long-term remissions remains under investigation for all recipients with little information in the setting of aggressive infantile ALL.…”

“…[10,[17][18][19] There are inherent difficulties in obtaining sufficient autologous T-cells in infants to generate a suitable CAR-T product. [17][18][19][20][21] Whether CAR-T therapy alone is satisfactory to achieve durable long-term remissions remains under investigation for all recipients with little information in the setting of aggressive infantile ALL. Failure following CAR-T therapy is frequently secondary to antigen escape.…”

“…[14,22] Multiple reports demonstrate superior outcomes and sustained remissions in pediatric and adult patients with r/r ALL receiving CAR-T as a bridge to allogeneic HSCT; [10,14,15] however, there is a dearth of data supporting the use of CAR-T therapy in infants. [18] Here we present our experience in a high-risk infant with r/r KMT2A-r ALL who successfully received the CD19 CAR-T cell therapy, tisagenlecleucel, in tandem with haploidentical donor HSCT. This is the youngest infant to survive and achieve prolonged remission following CAR-T therapy and subsequent HSCT.…”

“…This is the youngest infant to survive and achieve prolonged remission following CAR-T therapy and subsequent HSCT. [18] CASE DESCRIPTION Our patient is an African American female diagnosed at 6-weeks of age who presented for evaluation of persistent vomiting, lethargy and an unusual skin rash after several weeks of concern for failure to thrive. Initial white blood cell (WBC) count was 834x10 9 /L with 98% circulating lymphoblasts.…”

Corrigendum

“…In the article by David C. Shyr et al., 1 the authors wish to add some text in the acknowledgement section:…”