Is Detection of Relapse by Surveillance Imaging Associated With Longer Survival in Patients With Rhabdomyosarcoma?

BACKGROUND: We investigated whether surveillance imaging had an impact on post-relapse survival in patients with rhabdomyosarcoma (RMS). We hypothesized that relapse detected by imaging (group IM) would be associated with longer survival compared with relapse detected with a clinical sign or symptom (group SS). MATERIALS AND METHODS: We performed an observational multi-institutional study in 127 patients with relapsed RMS comparing overall survival (OS) after relapse using Kaplan-Meier and Cox proportional hazards analyses. RESULTS: Relapse was detected in 60 (47%) group IM and 67 (53%) SS patients. Median follow-up in survivors was 4 years (range 1.0 to 16.7 y). Four-year OS rates were similar between group IM (28%, 95% confidence interval [CI]: 14%-40%) and SS (21%, 95% CI: 11%-31%) ( P =0.14). In multivariable analyses accounting for institution, age at diagnosis, time to relapse, risk group at diagnosis, and primary site, not receiving chemotherapy (hazard ratio [HR]: 6.8, 95% CI: 2.8-16.6), radiation (HR: 3, 95% CI: 1.7-5.3), or surgery (HR: 2.8, 95% CI: 1.6-4.8) after relapse were independently associated with poor OS. CONCLUSION: These results on whether surveillance imaging provides survival benefit in patients with relapsed RMS are inconclusive. Larger studies are needed to justify current surveillance recommendations. Chemotherapy, radiotherapy and surgery to treat recurrence prolong OS.

Application of lung volume reduction surgery for a child with filamin A (FLNA) mutations.

Diffuse lung disease in early childhood due to mutations in the filamin A gene has been recently reported. Clinical outcomes vary among individuals indicating variability in phenotype but a substantial proportion of reported cases in early life have ended up in death or lung transplantation. We recently encountered a school-aged child in whom the diagnosis of a filamin A mutation was delayed and the natural history of emphysematous lung disease was altered by serial lung volume reduction surgeries. She eventually underwent a bilateral lung transplant and we report the natural history of her disease and treatments applied herein.

Protocol incorporating airway CT decreases negative bronchoscopy rates for suspected foreign bodies in pediatric patients.

BACKGROUND: Foreign body aspiration (FBA) is the 4th leading cause of death in children between the ages 1-5. Although direct laryngoscopy and bronchoscopy (DL&B) is the reference standard for diagnosis of pediatric airway foreign bodies, there is a high negative bronchoscopy rate, exposing patients to unnecessary operative and anesthetic risks and costs. METHODS: A clinical care protocol entailing the selective use of low-dose non-contrast airway computed tomography (CT) for children with an intermediate risk for FBA on the basis of clinical exam and chest radiography was implemented to decrease the negative DL&B rate. A retrospective review was conducted to compare negative bronchoscopy rates before and after implementation of the new protocol and the diagnostic performance characteristics of airway CT for airway foreign bodies were analyzed. RESULTS: After implementation of the airway FB clinical care protocol entailing selective airway CT, the overall negative bronchoscopy rate decreased from an institutional historical rate of 37% (54/145) to 17% (10/56) (p = .06). The overall sensitivity, specificity, and positive and negative predictive value of airway CT for FB was 91%, 100%, 100%, and 97% respectively. CONCLUSIONS: Low-dose non-contrast airway CT is highly sensitive and specific for airway foreign bodies, and its selective use in a clinical care protocol for children with suspected foreign body aspiration could greatly reduce the negative bronchoscopy rate, thereby decreasing operative risks and costs.

A phase 2 study of bortezomib in combination with ifosfamide/vinorelbine in paediatric patients and young adults with refractory/recurrent Hodgkin lymphoma: a Children's Oncology Group study.

A Children's Oncology Group clinical trial aimed to determine if bortezomib (B) increased the efficacy of ifosfamide and vinorelbine (IV) in paediatric Hodgkin lymphoma (HL). This study enrolled 26 relapsed HL patients (<30 years) treated with two to four cycles of IVB. The primary endpoint was anatomic complete response (CR) after two cycles. Secondary endpoints included overall response (OR: CR + partial response) at study completion compared to historical controls [72%; 95% confidence interval (CI): 59-83%]. Although few patients achieved the primary objective, OR with IVB improved to 83% (95% CI: 61-95%; p = 0.32). Although not statistically different, results suggest IVB may be a promising combination.

Acquired von Willebrand syndrome and Wilms tumor: not always benign.

Current literature suggests that acquired von Willebrand syndrome associated with Wilms tumor (AVWS-WT) occurs infrequently and usually has little clinical significance. Treatment strategies are thus poorly defined. We describe two patients with AVWS-WT and profuse bleeding who required intensive multimodal therapy, including aggressive blood component and factor replacement and plasmapheresis. They achieved adequate surgical hemostasis only after the renal vessels were ligated, with resolution of the coagulopathy upon tumor removal. Our experience suggests that AVWS-WT is not always benign. A careful bleeding history should always be obtained in patients with suspected renal tumors for consideration of pre-operative screening for AVWS.