Programmable valve breakage in shunt systems of children with posthemorrhagic hydrocephalus after minor head trauma-a case series.

PURPOSE: We report five rare cases of programmable valve breakage (Codman Hakim-Medos valve) in shunt systems of children with posthemorrhagic hydrocephalus. Only four similar studies have been published in the current literature. METHODS: Between 2013 and 2018, five children with posthemorrhagic hydrocephalus were admitted to the pediatric department. All patients had a history of slight blows to the head in a minor trauma and follow up MRI scans. After initial clinical examination, cranial computed tomography (CT) and X-ray were conducted. RESULTS: In all cases, pumping the reservoir resulted in very slow refilling. The cranial CT in one patient showed slit ventricles confirming the suspicion of overdrainage, the other cases a slight enhancement of the hydrocephalus. In lateral X-rays of the skull in comparison to the first X-ray control of the shunt valve, the pressure control chamber could be seen dislocated in the inferior part of the reservoir in all cases. Surgery revealed that the shunt valve was broken. The pressure control chamber had dropped to the bottom of the reservoir. After implantation of a new shunt valve, the symptoms resolved completely in all five children. Overall this complication occurred in 4.3% (5 of 85 implanted Codman Hakim-Medos valve) of all children necessitating ventriculoperitoneal shunt implantation between January 2013 and December 2018. CONCLUSION: The well-accepted Codman Hakim-Medos programmable valve is part of a tube-system, which is designed to offer the possibility of a reliable and precise treatment of hydrocephalus. Various mechanical and non-mechanical complications of shunt systems have been reported. Valve breakage is a very rare condition, often missed, and must be kept in mind when trauma and prior MRI scan are reported.

Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany.

BACKGROUND: Spinal muscular atrophy (SMA) is a neuromuscular disorder characterized by muscle weakness and muscle atrophy. Nusinersen acts as a splicing modifier and has recently been approved for intrathecal treatment of SMA. OBJECTIVE: Prior to approval, nusinersen was provided to patients with SMA type 1 in Germany within an Expanded Access Program (EAP). In contrast to previous clinical trials, children of different age groups and different stages of the disease were treated with nusinersen. METHODS: We conducted a prospective, longitudinal data collection of patients treated with nusinersen within the EAP in seven neuromuscular centers in Germany. Standardized assessments including CHOP-INTEND and HINE-2 motor milestones were performed at baseline and 60 and 180 days after start of treatment. RESULTS: Data from 61 SMA type 1 patients (mean age 21.08 months, range 1-93) were available for analysis. After six months of treatment, 47 children (77.0%) improved by ≥4 points in CHOP INTEND score. Mean change in CHOP INTEND score was 9.0±8.0 points. Nineteen patients (31.1%) improved by ≥2 points in HINE-2 motor milestones. Regression analysis revealed age at onset of treatment as major determinant of change in CHOP INTEND from baseline. CONCLUSION: When analyzing a broad spectrum of SMA type 1 patients, many children showed an improvement of motor function after six months of treatment with nusinersen, which is generally not expected within the natural course of the disease. Long-term observation and follow-up of patients with later onset types of SMA are crucial to understand the clinical impact of treatment with nusinersen.