Dinutuximab Beta Maintenance Therapy in Patients with High-Risk Neuroblastoma in First-Line and Refractory/Relapsed Settings-Real-World Data.

Dinutuximab beta is approved for the maintenance treatment of patients with high-risk neuroblastoma (HR-NB), including patients with relapsed/refractory (R/R) disease. However, the data on its use in real-world clinical practice is limited. We retrospectively reviewed the clinical records of 54 patients with HR-NB who received maintenance therapy with dinutuximab beta in first-line (37 patients) or R/R settings (17 patients) at three centers in Poland. Of the 37 patients who received first-line treatment, twenty-eight had a complete response, two had a partial response, three had progressive disease, and four relapsed at the end of treatment. The median overall survival (OS) was 24.37 months, and the three-year progression-free survival (PFS) and OS were 0.63 and 0.80, respectively. Of the 17 patients in the R/R group, 11 had a complete response, two had a partial response, one had stable disease, and three had progressive disease or relapsed at the end of treatment. The median OS was 33.1 months and the three-year PFS and OS were 0.75 and 0.86, respectively. Treatment was generally well tolerated, including in patients with co-morbidities and those who had experienced toxicities with previous therapies. These findings demonstrate that the use of dinutuximab beta is feasible and beneficial as a first-line or R/R treatment in routine clinical practice in Poland.

Isolated central nervous system relapses in patients with high-risk neuroblastoma -clinical presentation and prognosis: experience of the Polish Paediatric Solid Tumours Study Group.

Although isolated central nervous system (CNS) relapses are rare, they may become a serious clinical problem in intensively treated patients with high-risk neuroblastoma (NBL). The aim of this study is the presentation and assessment of the incidence and clinical course of isolated CNS relapses. Retrospective analysis involved 848 NBL patients treated from 2001 to 2019 at 8 centres of the Polish Paediatric Solid Tumours Study Group (PPSTSG). Group characteristics at diagnosis, treatment and patterns of relapse were analysed. Observation was completed in December 2020. We analysed 286 high risk patients, including 16 infants. Isolated CNS relapse, defined as the presence of a tumour in brain parenchyma or leptomeningeal involvement, was found in 13 patients (4.5%; 8.4% of all relapses), all of whom were stage 4 at diagnosis. Isolated CNS relapses seem to be more common in young patients with stage 4 MYCN amplified NBL, and in this group they may occur early during first line therapy. The only or the first symptom may be bleeding into the CNS, especially in younger children, even without a clear relapse picture on imaging, or the relapse may be clinically asymptomatic and found during routine screening. Although the incidence of isolated CNS relapses is not statistically significantly higher in patients after immunotherapy, their occurrence should be carefully monitored, especially in intensively treated infants, with potential disruption of the brain-blood barrier.

[Conservative treatment in patients with unilateral retinoblastoma]. / Próba leczenia zachowawczego u pacjentów z jednooczna postacia retinoblastoma.

UNLABELLED: THE AIM of our study was to evaluate results of conservative treatment of patients with unilateral retinoblastoma. MATERIAL AND METHODS: Twenty one patients, 11 boys and 10 girls aged 2 months to 4, 5 years (median age 1 year) were studied. Local disease advancement according to Reese-Elsworth was defined in all patients. Neoadjuvant chemotherapy consisting of Vincristine, Etoposide and Carboplatin was administered. After every 2 courses tumour response was evaluated. Sixteen patients were treated with chemotherapy alone. Local treatment including brachytherapy, thermochemotherapy and cryotherapy was implemented and the choice of the method depended on the tumour's localization, size and response to chemotherapy. Statistical analysis using demographic data and survival curves were performed. RESULTS: On completion of treatment all patients achieved tumour regression. Eleven patients are progression free with a follow-up from 10 months to 6 years 4 months (median--2 yrs 5m). In 10 patients relapse was observed. A total of seven enucleations were performed in the examined group. In histopathological examination viable tumour cells were present in all removed eye balls. Distant metastases were not observed in any of these patients. All patients are alive with a follow-up from 10 months to 9 yrs 6 months (median--4 yrs 7 months) from diagnosis. Disease free survival and ocular survival is 44% and 54% respectively. CONCLUSION: Neoadjuvant chemotherapy in unilateral retinoblastoma allows to avoid enucleation in some patients.

[Tumours in newborns and infants up to three months of life. One institution experience]. / Guzy u noworodków i malych niemowlat. Doswiadczenia wlasne.

INTRODUCTION: Newborns and infants up to three months of life are a specific group of population in paediatric oncology due to immaturity of tissues and organs and rarity of neoplastic diseases in this group of patients (pts). There are no strict therapeutic procedures established for these children. THE AIM of our study was to examine distribution of tumours in newborns and infants up to 3 months of age treated in our institution and to present our own experience in the treatment of these patients. MATERIAL AND METHODS: Medical records of 71 pts (37 boys and 34 girls) treated from 1996 to 2004 were reviewed. Distribution of tumour types in newborns and babies from 1 to 3 months of age was analyzed separately. Due to similar growth pattern, response to treatment and it's side effects in newborns and small infants, treatment results were evaluated for the whole group. RESULTS: there were 50 newborns. The most common diagnosis in this group was germ cell tumours (GCT) which constituted 60% of all tumours, amongst them 52% were mature teratomas (MT). The second most common was neuroblastoma (NBL) 22%. There were also 3 cases of soft tissue sarcomas (STS), 2 central nervous system tumours (CNS), 2 retinoblastoma (RB), 2 hepatoblastoma (HB). In the group of 21 babies aged 1-3 months NBL was the commonest (37%) followed by RB, CNS tumours (14% of each) HB and MT (10% of each) and Wilms tumour (WT) and immature teratoma (IT) each 5%. Surgery alone was performed in 48 pts. It concerned pts with MT--28, IT--3 pts, yolk sac tumour (YST)--1 pt and malignant tumours (stage I and II): 8-NBL, 2-CNS tumours, 2 STS, 3-HB, 1-WT. Forty two pts from this group are alive. Six pts died: 2 from surgical complications, 1 from sepsis, 1 of congenital heart defect and 1 from unknown reason at the age of 18 months. It was a patient with severe infantile cerebral palsy. One pt died of disease--relapse of yolk sac tumour, 2 years 4 months after surgery of MT. Eleven pts underwent combined treatment of chemotherapy and surgery: 5 with stage III and IV NBL, 6 with other tumours. One pt with STS at the age of 1 yr 6 months was the tumour bed irradiated after surgery for microscopic tumour residual. Four pts are alive, 4 with NBL and 5 with other tumours. Two pts died from disease: one with NBL and one with IT. Chemotherapy alone was administered to 7 pts in whom local advancement of disease enabled surgery and to pts with RBL. Three out of 7 pts are alive all with RBL. Four pts died: 3 from disease, 1 from infectious complications. Four pts with NBL (2 stage IV and 2 stage IVS) were treated with irradiation to the liver only. Two pts (st. IV) died and 2 (st. IVS) pts are alive. One pt relapsed at age of 2 yrs 3 mths, probably at the primary site which was not visualized at primary diagnosis. One pt, critically ill, died before any treatment. Fifty six out of 70 pts (80%) are alive with a follow up from 1 year to 9 yrs 11 months (median- 4 yrs 4 months). Fourteen pts died (20%), 8 from disease and 6 of other reasons. CONCLUSIONS: 1. GCT and neuroblastoma are the most common tumours in newborns and infants up to 3 months of age. 2. Newborns and small infants with advanced neoplastic disease, similarly to older children can be cured with chemotherapy. 3. Individual approach is warranted in newborns and small infants and treatment should be carried out in specialized centres. 4. All patients who completed treatment of any tumour type should be followed up by a pediatric oncologist.

[Cancer among adolescents 15-19 years of age. Own experience]. / Nowotwory u mlodziezy w wieku 15-19 lat. Badania wlasne.

BACKGROUND: Adolescents aged 15-19 years are variably included in analyses of childhood cancer. They should be considered separately because tumours that occur in adolescents differ from those in younger children. AIM: To describe the distribution of tumour types and treatment results in this group of patients treated in the Department of Pediatric Oncology at The Children's Memorial Health Institute. METHODS: Retrospective analysis of medical records of patients aged 15-19 years treated in our institution was performed. It included demographic data, tumour types and treatment results. RESULTS: Between 1998-2004, 207 pts, 110 boys and 97 girls aged 15-19 yrs (median--16.5 yrs) were treated. Distribution of tumours was as follows: CNS tumours--74 pts (35.7%), HD -18 pts (8.7%), NHL--13 pts (6.3%), bone tumours--31 pts (15%), STS--23 pts (11.1%), gonadal tumours--12 pts (5.8%), carcinomas--16 pts (7.7%), hepatomas--5 pts (2.4%), neuroblastoma--3pts (1.5%) and other 12 pts (5.8%). Out of 207 pts 130 are alive (62.8%). Seventy seven (37.2%) pts died--64 (83.1%) from disease, 9 (11.6%) from chemotherapy complications, 4 due to other reasons. 111 pts completed treatment and are disease free for 11 months to 7 yrs (median 3 yrs 11 mos) from diagnosis. Nineteen patients are still treated. Treatment results are as follows: CNS tumours--58.1%, HD - 88.8%, NHL--69.2%, bone tumours--51.6%, STS--65.2%, gonadal tumours--83.3%, carcinomas-- 56.25%. CONCLUSIONS: Spectrum of malignancies that occur in adolescents 15-19 years of age differs from younger children. Unlike younger patients epithelial carcinomas of adults are observed in this age group. Outcome of treatment is inferior to younger patients. Adolescents should be offered optimal treatment. This specific group should be studied in many aspects.