The Efficacy of Alternate Systemic Intravenous Chemotherapy and Intra-arterial Chemotherapy Approach for Eye Globe Salvage in Retinoblastoma / Journal of the Korean Cancer Association, 대한암학회지

Purpose@#The advances in the treatment of retinoblastoma have enabled salvaging the globe in advanced stages with intra-arterial chemotherapy (IAC). We developed a strategy of alternate application of systemic intravenous chemotherapy (IVC) and IAC (referred to as alternate systemic IVC and IAC; ASIAC) to reduce central nervous metastases during IAC and examined its efficacy and safety in eye globe salvage in this study. @*Materials and Methods@#Between January 2010 and February 2021, 43 eyes of 40 patients received ASIAC treatment for retinoblastoma at the Yonsei Cancer Center, Yonsei University Health System. Their medical records were reviewed retrospectively to evaluate the eye salvage rate (ESR), defined from diagnosis to enucleation. High-risk retinoblastoma was defined as group D or E by the International Classification of Retinoblastoma. @*Results@#The study enrolled 38 and five cases of high-risk and low-risk retinoblastoma, respectively. In total, 178 IAC and 410 IVC courses were administered, with a median of 4 (interquartile range [IQR], 3.0 to 5.0) IAC and 9 (IQR, 6.0 to 11) IVC courses per eye, respectively. The 5-year ESR was 60.4%±8.7% for the whole cohort, 100% for low-risk retinoblastoma, and 53.6%±9.8% for high-risk retinoblastoma. Among those diagnosed since 2015, the 5-year ESR for high-risk retinoblastoma was 63.5%±14.0%. Fifteen eyes underwent enucleation; no viable tumor was found in three enucleated eyes. There were no deaths in this cohort. @*Conclusion@#Primary IAC-IVC (i.e., ASIAC) for patients with retinoblastoma was tolerable and effective in salvaging the eye and maintaining survival.

Primary Extragonadal Germ Cell Tumors in Klinefelter Syndrome: 10-Years of Experience from a Single Institute / 임상소아혈액종양

Background@#Approximately 8% of male patients presenting with primary mediastinal germ cell tumors (GCTs) have Klinefelter syndrome (KS), while patients diagnosed with retroperitoneal GCTs also exhibit a range of chromosomal abnormalities. The exact mechanism underlying the development of GCTs in Klinefelter syndrome is unknown, but KS frequently goes underdiagnosed as a result of its varied symptoms and a low general awareness of this condition. Thus, the Children’s Oncology Group recommends screening of Klinefelter syndrome in pediatric and adolescent male subjects who present with GCTs. @*Methods@#We retrospectively reviewed the medical records of extragonadal germ cell tumor patients treated at Severance hospital, department of pediatrics or division of pediatric hematology-oncology over the last ten years. @*Results@#A total of 95 patients with extragonadal germ cell tumors were included in this study. Karyotyping was done in eight patients out of 95 patients, three patients with KS and one patient with Down syndrome. Twelve of extragonadal GCT patients presented at mediastinum, with most common histology of mature teratoma, and three patients presented with chromosomal abnormalities, two with KS and one with Down syndrome. A total of nine patients were diagnosed with retroperitoneal GCTs and only one had KS. @*Conclusion@#We described the characteristics of 95 cases of extragonadal GCTs. Although the mechanism of extragonadal GCTs in KS is not clear, karyotyping in pediatric and adolescent extragonadal GCT patients could be helpful in figuring out chromosomal abnormalities including KS and their roles in GCT pathophysiology, which can contribute to improve one’s health.

Usefulness of the RESP, PRESERVE, and ECMOnet scores for extracorporeal membrane oxygenation in children with acute respiratory distress syndrome

PURPOSE: With increasing use of extracorporeal membrane oxygenation (ECMO) for acute respiratory distress syndrome (ARDS) in children, development of standardized strategies for survival prediction has become crucial; however, this has not been accomplished yet. We evaluated the adult scoring systems for survival prediction used for their applicability in pediatric ARDS and validated them. METHODS: A total of 11 children with ARDS receiving ECMO from 2013 to 2014 were evaluated with adult scoring systems, including the Respiratory Extracorporeal-membrane-oxygenation Survival Prediction (RESP), the PRedicting dEath for SEvere ARDS on VV-ECMO (PRESERVE), and the ECMOnet scores. We compared the scores on these scales and the clinical characteristics between survivors and nonsurvivors. RESULTS: Eight of the 11 children died (72.7%). The PRESERVE score (survivors vs. nonsurvivors: 2 vs. 5.25, P=0.048), and the ECMOnet score (4.1 vs. 5.63, P=0.048) were lower in survivors than in nonsurvivors. They correctly predicted mortality prediction. There was no significant difference in the RESP score between survivors and non-survivors (−4.33 vs. −2.62, P=0.63). The parameters that showed significant differences in this study were peak inspiratory pressure, platelet, and delta neutrophil index. All children who were under immunocompromised conditions, such as those with tumors, or underwent hematopoietic stem cell transplantation died. The immunocompromised status should be considered an important factor for survival prediction in children with ARDS. CONCLUSION: This is the first pilot study to apply the survival prediction scoring system to pediatric ARDS with ECMO. It is necessary to establish and modify the survival prediction score system for pediatric ARDS with ECMO.

Protein-Losing Enteropathy as a Complication of the Ketogenic Diet

The ketogenic diet is an effective treatment for the patients with intractable epilepsy, however, the diet therapy can sometimes be discontinued by complications. Protein–losing enteropathy is a rarely reported serious complication of the ketogenic diet. We present a 16-month-old Down syndrome baby with protein-losing enteropathy during the ketogenic diet as a treatment for West syndrome. He suffered from diarrhea, general edema and hypoalbuminemia which were not controlled by conservative care for over 1 month. Esophagogastroduodenoscopy and stool alpha-1 antitrypsin indicated protein-losing enteropathy. Related symptoms were relieved after cessation of the ketogenic diet. Unexplained hypoalbuminemia combined with edema and diarrhea during ketogenic suggests the possibility of protein-losing enteropathy, and proper evaluation is recommended in order to expeditiously detect it and to act accordingly.