Late effects in high‐risk neuroblastoma survivors treated with high‐dose chemotherapy and stem cell rescue

• Published in: Pediatric blood & cancer Vol. 66; no. 1; pp. e27421 - n/a
• Main Authors: Elzembely, Mahmoud M., Dahlberg, Ann E., Pinto, Navin, Leger, Kasey J., Chow, Eric J., Park, Julie R., Carpenter, Paul A., Baker, K. Scott
• Format: Journal Article
• Language: English
• Published: United States 01.01.2019
• Subjects: high‐dose chemotherapy; late effects; neuroblastoma; neuroblastoma; late effects; high-dose chemotherapy
• ISSN: 1545-5009; 1545-5017
• DOI: 10.1002/pbc.27421

Background Current treatment strategies have improved the outcome of high‐risk neuroblastoma (HRNB) at the cost of increasing acute and late effects of treatment. Although high‐dose chemotherapy with stem cell rescue (HDC‐SCR) has replaced total body irradiation (TBI) based HRNB therapy, late effects of therapy remain a significant concern. Objectives To describe late effects prevalence, severity, and risks after HDC‐SCR. Methods Retrospective chart review of relapse‐free HRNB survivors ≥1 year after single HDC‐SCR between 2000 and 2015 at Fred Hutchinson Cancer Research Center. Results Sixty‐one survivors (30 males) were eligible. Median age (years) at SCR was 3.5 years (range 0.7‐27 years) and median posttransplant follow‐up was 5.4 years (1.2‐16.3 years) . Fifty‐three (86.9%) survivors developed late effects that increased over time (P < 0.001) and varied in severity from grade 1 (35) to grade 5 (1). These were unrelated to gender or age. High‐frequency hearing loss seen in 82% of survivors was the most common abnormality present and 43% of those required hearing aids. Seventeen (27.9%) survivors developed dental late effects and these were most common in children <2 years of age at transplant (P = 0.008). Other toxicities included endocrine (18%), orthopedic (14.8 %), renal (3.9%), melanotic nevi (8.2%), neuropsychological impairments (8.2%), subsequent malignancies (4.9%), pulmonary (4.9%), cardiac (4.9%), and focal nodular liver hyperplasia (3.3%). At 9 years posttransplant, the median height and weight Z‐scores were significantly lower than Z‐scores at the time of HDC‐SCR (–0.01/–1.08, P < 0.001; –0.14/–0.78, P = 0.005). Conclusion Avoidance of TBI does not mitigate the need to provide diligent, ongoing surveillance for late effects.