Selective T cell‐depleted haploidentical hematopoietic stem cell transplantation for relapsed/refractory neuroblastoma

• Published in: Pediatric transplantation 2018-09, Vol.22 (6), p.e13240-n/a
• Main Authors: Liu, Anthony P. Y., Lee, Pamela P. W., Kwok, Janette S. Y., Leung, Rock Y. Y., Chiang, Alan K. S., Ha, Shau‐Yin, Cheuk, Daniel K. L., Chan, Godfrey C. F.
• Format: Article
• Language: English
• Subjects: CD19 antigen; CD3 antigen; CD45RA antigen; Chimerism; Engraftment; Fludarabine; Graft rejection; Graft-versus-host reaction; haploidentical; hematopoietic stem cell transplantation; Hematopoietic stem cells; Immunotherapy; Leukocytes (neutrophilic); Lymphocytes T; Natural killer cells; Neuroblastoma; Patients; Peripheral blood; relapse; Remission; Sepsis; Stem cell transplantation; Stem cells; T cell receptors
• ISSN: 1397-3142; 1399-3046
• DOI: 10.1111/petr.13240

Relapsed/refractory NB carries a bleak outcome, warranting novel treatment options. HaploHSCT induces a graft‐versus‐NB effect via natural killer cell alloreactivity. Review of patients with relapsed/refractory NB who underwent haploHSCT with ex vivo T‐cell depletion in our unit from 2013 through 2018. Ten patients were identified (male=5; median age at haploHSCT=6.45 y, range: 3.49‐11.02 y). Indications were relapsed in 7 and refractoriness in 3; disease status at haploHSCT was CR in 2, PR in 6, and PD in 2. All patients received peripheral blood stem cell grafts after ex vivo T‐cell depletion (CD3/CD19‐depletion=1; TCR‐αβ/CD19‐depletion=4; CD3/CD45RA‐depletion=4; and TCR‐αβ/CD45RA‐depletion=1). Conditioning regimens were fludarabine‐based. Neutrophils engrafted on median D + 10 (range: D + 9 to +13), and platelets engrafted (≥20 × 109/L) on median D + 8 (range: D + 5 to D + 14). Early T‐ and NK‐cell recovery were evident. Of the 10 patients, acute rejection developed in 1 (who died of PD despite rescue HSCT), and 1 died of sepsis before engraftment; 8 experienced full donor‐chimerism post‐HSCT. Among the 8, 6 experienced CR, 1 died of PD, and 1 died of pulmonary hypertensive crisis before evaluation. At publication, 4 were in remission (2.8, 7.4, 28.5, and 58.9 months). No significant GvHD occurred. HaploHSCT with selective ex vivo T‐cell depletion may be a safe and useful salvage strategy for relapsed/refractory NB.