Pediatric Neurosurgery最新文献

Background: Paediatric low-grade gliomas (pLGGs) are the most common primary brain tumour in children. Though considered benign, slow-growing lesions with excellent overall survival, their long-term morbidity can be significant, both from the tumour and secondary to treatment. Vast progress has been made in recent years to better understand the molecular biology underlying pLGGs, with promising implications for new targeted therapeutic strategies.

Summary: A multi-layered classification system of biologic subgroups, integrating distinct molecular and histological features has evolved to further our clinical understanding of these heterogeneous tumours. Though surgery and chemotherapy are the mainstays of treatment for pLGGs, many tumours are not amenable to surgery and/or progress after conventional chemotherapy. Therapies targeting common genetic aberrations in the RAS-mitogen-activated protein kinase (RAS/MAPK) pathway have been the focus of many recent studies and offer new therapeutic possibilities. Here, we summarise the updated molecular classification of pLGGs and provide a review of current treatment strategies, novel agents, and open trials.

Key messages: (1) There is a need for treatment strategies in pLGG that provide lasting tumour control and better quality of survival through minimising toxicity and protecting against neurological, cognitive, and endocrine deficits. (2) The latest World Health Organisation classification of pLGG incorporates a growing wealth of molecular genetic information by grouping tumours into more biologically and molecularly defined entities that may enable better risk stratification of patients, and consideration for targeted therapies in the future. (3) Novel agents and molecular-targeted therapies offer new therapeutic possibilities in pLGG and have been the subject of many recent and currently open clinical studies. (4) Adequate molecular characterisation of pLGG is therefore imperative in today's clinical trials, and treatment responses should not only be evaluated radiologically but also using neurological, visual, and quality of life outcomes to truly understand treatment benefits.

Introduction: Children with spinal muscular atrophy (SMA) and progressive neuromuscular scoliosis often require early growth-friendly spinal implant (GFSI) treatment for deformity correction with implant fixation either through pedicle screws or bilateral to the spine using ribto pelvis fixation. It has been proposed that the latter fixation may change the collapsing parasol deformity via changes in the rib-vertebral angle (RVA) with a positive effect on thoracic and lung volume. The purpose of this study was to analyze the effect of paraspinal GFSI with bilateral rib-to-pelvis fixation on the parasol deformity, RVA, thoracic, and lung volumes.

Methods: SMA children with (n = 19) and without (n = 18) GFSI treatment were included. Last follow-up was before definite spinal fusion at puberty. Scoliosis and kyphosis angles, parasol deformity, and index, as well as convex and concave RVA, were measured on radiographs, whereas computed tomography images were used to reconstruct thoracic and lung volumes.

Results: In all SMA children (n = 37; with or without GFSI), convex RVA was smaller than concave values at all times. GFSI did not crucially influence the RVA over the 4.6-year follow-up period. Comparing age- and disease-matched adolescents with and without prior GFSI, no effect of GFSI treatment could be detected on either RVA, thoracic, or lung volumes. Parasol deformity progressed over time despite GFSI.

Conclusion: Despite different expectations, implantation of GFSI with bilateral rib-to-pelvis fixation did not positively influence parasol deformity, RVA and/or thoracic, and lung volumes in SMA children with spinal deformity directly and over time.

Introduction: The aim of this cohort study was to assess the outcome of single-level selective dorsal rhizotomy (SDR) in children and young adults with spastic cerebral palsy (CP) treated at our institution, focusing on patient-reported outcome measures (PROMs) and quality of life (QoL) of patients and their caregivers.

Methods: We included consecutive patients undergoing SDR from 2018 to 2020 at our institution. Subjective outcome was measured through PROMs, while functional outcome was measured through baseline characteristics, operative outcome, as well as short- and long-term follow-up. Furthermore, the effect of age at the time of surgery on patient/caregiver satisfaction was analyzed.

Results: Seven patients (3 female, 43%) with a median age at surgery of 11.9 years (IQR 8.7-15.5) were included. All patients had a Gross Motor Function Classification (GMFCS) score of at least IV before surgery. Five surgeries were palliative and two non-palliative. Based on PROMs, SDR showed very good QoL and health-related outcome measures for both palliative and non-palliative patients. Patient/caregiver satisfaction was higher for the early subgroup (age ≤11) than the late subgroup (age >11). Functional outcome showed reduced spasticity in both groups. Blood transfusions were never needed, while no cerebrospinal fluid leak, infection, or permanent morbidity was seen.

Conclusion: Based on PROMs, SDR leads to high satisfaction and improved QoL, especially if done at an early age. Further studies with larger cohorts are necessary to underline and confirm our observations.