Establishment of Hong Kong Neuromuscular Disorder Patient Registry

Introduction: Neuromuscular disorders (NMDs) are a group of diseases affecting the peripheral nervous system (1). Many NMDs cause disability or even premature death (2). We aim to design and establish a robust NMD patient registry in Hong Kong. Methods: By modelling international NMD patient registries, we designed patient-professional reported questionnaires to collect the demographic, clinical c haracteristics, genetic details, family history, investigation findings and specific treatment of NMD patients. Patients were recruited through Hong Kong West Cluster (DKCH, QMH) and Kowloon Central Cluster (HKCH). We also developed self-registration online platform. p<0.05 was considered statistically significant. Findings: Since June 2019, 125 NMD patients have been enrolled in the registry with 12 participants registered online. The registry recruited 13 types of NMDs, including spinal muscular atrophy (SMA) (n=31), Duchenne muscular dystrophy (DMD) (n=19) and congenital myopathy (n=18). The age range was 7 months to 63 years old. 65.6% of those enrolled were children (<18 years old). 63.2% were male. 64.8% of the patients had genetic diagnosis. The registry has contributed to two studies. The first one is a prospective study of clinical efficiency of Nusinersen in SMA patients (n=22). 14/16 SMA patients showed improvement in at least one of motor performance (CHOP intend/RULM/HINE/HFMSE) and health-related quality of life after 1st year of treatment. The second study is the reactogenicity and immunogenicity study of the COVID-19 vaccine in DMD patients (n=4). Data will be available in October. Conclusion: Hong Kong Patient registry has contributed to ongoing and new research study to optimise medical care.

Combating the coronavirus disease 2019 pandemic in the HONG KONG hospital authority paediatric infectious disease centre

Since the coronavirus disease 2019 (COVID-19) outbreak in late December 2019, and escalation of the public hospital infectious disease outbreak response level to the highest "emergency" level three days after two COVID-19 cases were diagnosed on 22 January 2020, all public hospitals implemented a series of isolation policies, rescheduled clinical services and mobilised resources to support frontline clinical staff. This article reviewed these multi-level policies adopted in the Department of Paediatrics and Adolescent Medicine, Princess Margaret Hospital, which have ensured that the healthcare system was not overwhelmed and frontline staff was not over-stressed and infected.