Abstract
This retrospective analysis aimed to address the efficacy of total nodal irradiation (TNI)-based reconditioning regimens in pediatric patients with graft failure/rejection after allogeneic hematopoietic cell transplantation. Thirty-three pediatric patients with malignant (n=25) and nonmalignant diseases (n=8) were treated with a TNI-based reconditioning regimen. All patients received a 7-Gy single dose combined with anti-T lymphocyte antibody OKT3 (n=16), anti-thymocyte globulin (n=24), fludarabine (n=31), and/or thiotepa (n=28), followed by an infusion of peripheral blood stem cells (n=31) or bone marrow transplant (n=2). Twenty-eight of 33 patients had haploidentical family donors. After a median of 11days, engraftment was seen in 32 of 33 children. Two children died 34days after retransplantation because of either disease relapse or treatment-related multiple organ failure. Severe acute toxicity was reported in only 1 child (systemic inflammatory response syndrome-like reaction; recovery after cortisone treatment). The average follow-up was 60.2months (range, 1.1-162.5months). Event-free and overall survival rates at 2/5years follow-up were 62.0%/58.6% and 65.1%/61.7%, respectively. Despite sustained engraftment, 12 patients died from disease relapse (n=3), Moschkowitz syndrome (n=1), or multiple organ failure (n=8). Follow-up data were available for 18 of 21 survivors, with a median follow-up of 92.8months (range, 3.6-162.5months). Hypothyroidism was present in 78.6% of patients, and sex/growth hormonal insufficiencies were reported for 37.5%. Mean forced expiratory volume in 1second after TNI was 84%; mean vital capacity was 79%. Severe growth failure (<3rd percentile) occurred in 28.6% (height) and 35.7% (weight) of patients. No secondary malignancies were reported. In the high-risk group of patients with graft failure/rejection after allogeneic hematopoietic cell transplantation, the TNI-based reconditioning regimen seems to allow sustained engraftment combined with a favorable toxicity profile, leading to long-term event-free and overall survival. Late toxicity after a median follow-up of over 7.5years includes growth failure, manageable hormonal deficiencies, and a low risk of decrease of lung function.
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More From: International Journal of Radiation Oncology, Biology, Physics
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