Therapy results in pediatric Hodgkin lymphoma - does less mean better? Experience from a single children's oncology center.

Therapy results in pediatric Hodgkin lymphoma reflect remarkable progress in pediatric oncology. In the last decade, relevant development of new therapeutic options for children with refractory or relapsed disease has been made. In this study, we retrospectively analyzed therapy results and risk factors in children treated in a single oncology center according to five therapeutic protocols. Data from 114 children treated by a single institution between 1997 and 2022 were analyzed. Classic Hodgkin lymphoma therapy results were divided into four therapeutic periods: 1997-2009, 2009-2014, 2014-2019, and 2019-2022. For nodular lymphocyte-predominant Hodgkin lymphoma, data from one therapeutic protocol was analyzed. For the entire group, the 5-year probability of overall survival was 93.5%. There were no statistically significant differences between therapeutic periods. The occurrence of B symptoms at diagnosis and incidence of relapse were risk factors for death (p = 0.018 and p < 0.001). Relapse occurred in 5 cases. The 5-year probability of relapse-free survival for the entire group was 95.2%, without significant differences between groups. Patients treated between 1997 and 2009 had over a sixfold higher risk for events, defined as primary progression, relapse, death, or incidence of secondary malignancies (OR = 6.25, p = 0.086). The 5-year probability of event-free survival for all patients was 91.3%. Five patients died, and the most common cause of death was relapse. Modern therapeutic protocols in pediatric Hodgkin lymphoma are marked by excellent outcomes. Patients with disease relapses have a notably high risk of death, and the development of new therapeutic options for this group remains one of the main goals of current trials.

Coordinated medical care for children with neurofibromatosis type 1 and related RASopathies in Poland.

Coordinated medical care offered in Poland for patients suffering from neurofibromatosis type 1 and related RASopathies combines complex multispecialty consultation with permanent supervision and the patient's oriented longitudinal care. Neurofibromatosis type 1 is one of the most common single gene disorders in the global population, observed in 1 out of 2500-3000 live births. It is a primary neoplasia disease with 100% penetration of the gene mutation but remarkable age-dependent onset of different disease signs and symptoms, outstanding clinical heterogeneity between patients even in one family and lack of genotype-phenotype correlation, a high rate of spontaneous mutation exceeding 50%, and multiple comorbidities among which increased risk of malignancy is the most important. Medical practice proved that not only patient-oriented complex but also coordinated care provided in centers of competence is indispensable for patients and the families and provides a sense of medical security to them in conjunction with public health costs rationalization.

Lymphocyte Subpopulations in Patients With Neurofibromatosis Type 1-associated Optic Pathway Gliomas and Plexiform Neurofibromas.

BACKGROUND/AIM: Neurofibromatosis type 1 (NF1) is characterized by the occurrence of multisystem tumors, among which the most characteristic are optic pathway gliomas (OPGs) and plexiform neurofibromas (PNFs). With the development of new anticancer drugs targeting the immune system, it is important to examine the immunological status of patients with NF1. Furthermore, the immune system has been suggested as a probable modulator of NF1-associated phenotypes. The objective of this study was the analysis of lymphocyte subset populations with respect to the presence of PNFs and OPGs. PATIENTS AND METHODS: Fifty-three patients with NF1 diagnosed with OPG/PNF were analyzed for lymphocyte subpopulations. RESULTS: Significantly lower levels of B-cells, T-cells and natural killer (NK) cells were observed in the group of patients with PNFs compared to those with OPG. CONCLUSION: Our observation may indicate a correlation between weakened functioning of the immune system and the formation of PNFs.

Demographical Profile and Spectrum of Multiple Malignancies in Children and Adults with Neurocutaneous Disorders.

BACKGROUND/AIM: Neurocutaneous disorders, also referred as phacomatoses, are congenital disorders manifesting at different ages with central nervous system and cutaneous abnormalities. Analysis of the demographic and clinical profile of patients with phacomatoses in the context of the incidence and spectrum of malignancy. MATERIALS AND METHODS: This is a retrospective analysis of 20 years of data in a single-center study in Poland. RESULTS: Phacomatoses were diagnosed in 45.6% (913/2,003) of referred patients, including 61.4% children. The distribution of phacomatoses included: neurofibromatosis type 1 (NF1) in 92.4%, tuberous sclerosis complex (TSC) 3.9%, neurofibromatosis type 2 (NF2) 2.0%, Klippel-Trenaunay syndrome 0.5%, Von Hippel-Lindau syndrome 0.5%, and other sporadic diseases 0.7%. Non-phacomatosis patients were diagnosed mainly for cafe-au-lait-macules (42.8%). The frequency of malignancy was 9.4% (86/913), including 9.1% in patients with NF1; 27.8% in NF2; and 8.3% in TSC. Multiple malignancies were diagnosed in 0.7% and 7% of all phacomatosis and malignancy-diagnosed patients, respectively. CONCLUSION: The risk of malignancy in patients with phacomatoses was 21.3-fold higher than in the general population. The risk of secondary malignancy was 7%.

[Neurofibromatosis type 1--own experiences]. / Nerwiakowlókniakowatosc typu 1. W obserwacjach wlasnych.

PURPOSE: Neurofibromatosis type 1 is one of the most common inherited syndromes. The aim of this study was to evaluate eye symptoms on this disease. MATERIAL AND METHODS: 52 patients with neurofibromatosis type 1 were observed (28 males and 24 females), age between 3 and 49 years old (mean 21). The patients were divided into five groups depending on the age: 0-10 years old, 11-20 years old, 21-30 years old, 31-40 years old and older than 40 years. Frequency of the eye symptoms was estimated in each group. RESULTS: The eye sings were observed in 69.2%. Frequency of the eye symptoms were higher in the older groups. After 21th years of age ophthalmological signs were observed in all patients. The most common were café-au-lait spots on the lids, Lisch nodules on the iris, changes in CNS, especially gliomas of the optic pathway, nodular neurofibromas in the orbit region. CONCLUSIONS: Eye signs of the disease may be noticed in the most patients with NF 1. After 21th years of age ophthalmological symptoms are observed in all patients. The frequency of typical well known for NF 1 signs were different and characteristic for each age group.

[Pathogenesis and the most frequent symptoms of neurofibromatosis type 1]. / Patogeneza i najczestsze objawy nerwiakowlókniakowatosci typu 1.

PURPOSE: This study was to evaluate pathogenesis, symptoms, clinical course and possible treatment of neurofibromatosis type 1. Neurofibromatosis type 1, von Recklinghausen's disease, is one of the phacomatoses. It belongs to the most frequent inherited diseases in human population. This disease is autosomal dominant, but new spontaneous mutation are also common. The symptoms are caused by disorders in the melanocytes and gliocytes. In ophthalmological examination Lisch nodules, café-au lait spots, neurofibromas of the lids, optic pathway gliomas and deformation of the orbit bones, can be observed. The symptoms are different, depend upon the age and demonstrate charateristic evolution through the lifetime.