Progression‐free survival of two cases of high‐risk neuroblastoma with refractory/relapsed disease following surgery alone

Outcome for the vast majority of high‐risk neuroblastoma patients with refractory or relapsed disease is dismal. We report two high‐risk patients who remain progression‐free for more than 113 and 18 months following the diagnosis of refractory/relapsed disease who were treated with surgery alone. Complete resolution of a refractory thoracic mass and relapsed liver nodules was observed in one patient. The refractory/relapsed disease in the second patient has remained stable. In both cases, the tumor showed histologic evidence of neuroblastoma maturation. These cases demonstrate that refractory/relapsed neuroblastoma is clinically heterogeneous and highlight the need for better biomarkers to optimize patient care. Pediatr Blood Cancer 2013; 60: 512–514. © 2012 Wiley Periodicals, Inc.     

Bronchiectasis following treatment for high‐risk neuroblastoma: A case series

High‐risk (HR) neuroblastoma remains a very challenging disease to treat and long‐term cure is only possible with intensive, multimodal treatment including chemotherapy, high‐dose therapy, radiotherapy, surgery, and immunotherapy. As a result, treatment‐related morbidity and late effects are common in survivors. This report outlines a case series of six patients who developed a chronic productive cough following treatment for HR neuroblastoma. High‐resolution computed tomography scanning confirmed the diagnosis of bronchiectasis. Two of the patients who have undergone immunological testing demonstrate hypogammaglobulinaemia and impaired vaccine response. Persistent cough in patients treated for neuroblastoma warrants investigation and consideration of immunological referral.     

Value of surgical resection in children with high‐risk neuroblastoma

Background

The value of gross total resection (GTR) for children with high‐risk neuroblastoma (NB) is controversial. We hypothesized that patients undergoing GTR would demonstrate improved overall survival (OS) compared those having <GTR.

Methods

Using a single institutional database, we reviewed the medical records of all children with high‐risk NB undergoing hematopoietic stem cell transplantation (HSCT) as part of multimodality therapy from 1990 to 2012. Children had received surgical care at multiple institutions (n = 14) prior to HSCT and were divided into two groups based on extent of surgical resection: GTR (no visible or palpable disease at end of operation) and <GTR (no surgery, biopsy only, or subtotal resection). Kaplan–Meier curves and Cox hazards models evaluated differences in overall survival (OS).

Results

One hundred four children underwent HSCT, and 87 (83.6%) had adequate data for analysis. Thirty eight percent had GTR while 62% had <GTR prior to HSCT. There was no significant difference in OS in patients undergoing GTR compared to <GTR (Log rank test: P = 0.49). Post‐hoc analysis demonstrated a survival advantage for patients undergoing >90% resection compared to <90% resection (P = 0.008). Multivariable Cox models confirmed these findings with improved survival in children undergoing >90% vs. <90% resection but no difference in GTR vs. <GTR.

Conclusion

Gross total resection prior to HSCT in high‐risk NB patients is not associated with improved OS compared to <GTR; however, these results suggest that >90% resection is associated with improved OS compared to less than 90% resection. Pediatr Blood Cancer 2015;62:1529–1535. © 2015 Wiley Periodicals, Inc.     

Survival of high‐risk pediatric neuroblastoma patients in a developing country

Little information is available about survival of high‐risk pediatric neuroblastoma patients in developing countries. We aimed to assess survival among high‐risk pediatric neuroblastoma patients in La Plata, Argentina. Individuals eligible for our cohort were aged <20 yr when diagnosed with high‐risk neuroblastoma and received cancer‐directed therapy including stem cell transplantation at Hospital de Niños Sor Maria Ludovica between February 1999 and February 2015. We estimated overall survival probabilities using an extended Kaplan–Meier approach. Our study population comprised 39 high‐risk neuroblastoma patients, of whom 39% were aged >4 yr at diagnosis, 54% were male, and 62% had adrenal neuroblastoma. We observed 18 deaths, and the median survival time of our study population was 1.7 yr. The five‐yr overall survival probability was 24% (95% CL: 10%, 41%). In contrast, five‐yr survival of high‐risk neuroblastoma patients ranges between 23% and 76% in developed countries. Survival among high‐risk neuroblastoma patients is generally poor regardless of geographic location, but our results illustrate dramatically worse survival for patients in a developing country. We speculate that the observed survival differences could be attenuated or eliminated with improvements in treatment and supportive care, but addressing these issues will require creative solutions because of resource limitations.     

Multimodality treatment for recurrent neuroblastoma in the central nervous system

Survival for patients with recurrent central nervous system (CNS) neuroblastoma remains poor. A single-institutional study demonstrated the potential of multimodality therapy, including compartmental intrathecal radioimmunotherapy (cRIT) with ¹³¹I-3F8 or ¹³¹I-8H9 to increase the survival of neuroblastoma patients with CNS relapse. However, not all patients are able to receive this therapy. We report three patients with CNS neuroblastoma who remain disease-free 3–9 years after receiving multimodality treatment without cRIT. Additional studies to identify patients most likely to benefit from cRIT are warranted.