Barcelona Clinic Liver Cancer and Hong Kong Liver Cancer staging systems for prediction of survival among Hepatocellular Carcinoma patients.

INTRODUCTION: We aimed to compare the Barcelona Clinic Liver Cancer (BCLC) and Hong Kong Liver Cancer (HKLC) staging systems. MATERIALS AND METHODS: This is a retrospective study on patients with newly diagnosed hepatocellular carcinoma (HCC) at the University Malaya Medical Centre between 2011 and 2014. Survival times were analysed using the Kaplan- Meier procedure and comparison between groups was done using the log rank test. RESULTS: The data of 190 patients was analysed. Chronic hepatitis B was the most common aetiology for HCC (43.7%), but a large proportion was cryptogenic or non-alcoholic steatohepatitis-related (41.6%). Only 11.1% were diagnosed early (BCLC Stage 0-A) while majority were diagnosed at an intermediate stage (BCLC Stage B, 53.7%). The median survival rate was significantly different between the different groups when either of the staging systems was used (p<0.05 for all comparisons). However, the two staging systems lacked agreement (weighted kappa 0.519, 95%CI: 0.449, 0.589) with significant difference in median survival rates between BCLC Stage A and HKLC Stage 2, and between BCLC Stage C and HKLC Stage 4. CONCLUSION: Both staging systems were able to stratify patients according to survival, but they only had moderate agreement with significant differences observed in two groups of the staging systems.

Biological disease-modifying antirheumatic drugs in juvenile idiopathic arthritis of polyarticular course, enthesitis-related arthritis, and psoriatic arthritis: a consensus statement.

OBJECTIVES: Juvenile idiopathic arthritis (JIA) is the most common type of inflammatory arthritis in children. Treatment options have been expanded since the introduction of biologics, which are highly effective. The existing local JIA treatment guideline was published more than a decade ago, when use of biologics was not as common. In this article, we review the latest evidence on using biologics in three JIA subtypes: JIA of polyarticular course (pcJIA), enthesitis-related arthritis (ERA), and psoriatic arthritis (PsA). Based on the latest information, an update on eligibility, response assessment, termination, and safety information for using biologics in these patients was performed. CONSENSUS PROCESS: The JIA Work Group, which consisted of nine paediatricians experienced in managing JIA, was convened in 2016. Publications before July 2017 were screened. Eligible articles were clinical trials, extension studies, systemic reviews, and recommendations from international societies and regulatory agencies about the use of biologics in pcJIA, ERA, and PsA. Evidence extraction, appraisal, and drafting of propositions were performed by two reviewers. Extracted evidence and drafted propositions were presented and discussed at the first two meetings. Overwhelming consensus was obtained at the final meeting in May 2018. Seven practice consensus statements were formulated. Regular review should be performed to keep the practice evidence-based and up-to-date.

Risk factors associated with 1-year mortality among patients with HIV-associated tuberculosis in areas with intermediate tuberculosis burden and low HIV prevalence.

INTRODUCTION: Data are limited regarding risk factors for mortality among patients with human immunodeficiency virus (HIV)-associated tuberculosis (TB) in areas with low HIV prevalence and intermediate TB burden, such as the Western Pacific region. This study aimed to assess such risk factors in Hong Kong, which has an intermediate TB burden and low HIV prevalence. METHODS: We conducted a retrospective cohort analysis of adult patients reported to the Hong Kong TB-HIV Registry between 2006 and 2015. Baseline characteristics were compared with Kaplan-Meier estimates. Cox proportional hazards regression modelling was used to identify factors associated with mortality. RESULTS: Of 299 patients studied, 21 (7.0%) died within 12 months of anti-TB treatment (median [interquartile range], 7.5 [3.8-10] months). The median age of death was 54 (interquartile range, 40.5-75.0) years. The cause of death was TB in five and unrelated to TB in the remaining 16. Cox proportional hazards regression showed that older age (adjusted hazard ratio=4.5; 95% confidence interval [CI]=1.4-14.9), history of drug addiction (4.6; 95% CI=1.6-13.0), and low baseline CD4 cell count of <50/µL (2.9; 95% CI=1.1-7.7) were independent risk factors for death within 12 months. CONCLUSION: This study complements previous studies by providing information regarding risk factors associated with mortality among patients with HIV-associated TB in areas with intermediate TB burden and low HIV prevalence. The results from our study may guide targeted measures to improve survival in other areas with intermediate TB burden and low HIV prevalence, such as the Western Pacific region.

Hepatitis B virus infection among children of hepatitis B surface antigen positive mothers in a Malaysian hospital.

INTRODUCTION: There have been no published data on the transmission of hepatitis B virus (HBV) infection among children of hepatitis B surface antigen (HBsAg) positive mothers in Malaysia. METHODS: This is a cross-sectional study of all the children of HBsAg-positive mothers who delivered at the University of Malaya Medical Centre between 1993 and 2000. RESULTS: A total of 60 HBsAg-positive mothers and their 154 children participated in the study. HBsAg was detected in four children (2.6%) while IgG antibody to the hepatitis B core antigen (anti-HBc IgG) was detected in seventeen children (11.0%). The mother's age at childbirth was significantly lower in the children with detectable HBsAg (22.5±6.1 years vs. 29.7±4.5 years, p=0.043) and anti-HBc IgG (26.6±6.1 years vs. 30.0±4.3 years, p=0.004). Children born in the 1980s were significantly more likely to have detectable HBsAg (18.8% vs. 0.7%, p=0.004) and anti-HBc IgG (37.5% vs. 8.0%, p=0.000) compared with those born later. All children with detectable HBsAg were born via spontaneous vaginal delivery, and hepatitis B immunoglobulin was either not given or the administration status was unknown. The majority of mothers with chronic HBV infection (70.4%) were not under any regular follow-up for their chronic HBV infection and the main reason was the lack of awareness of the need to do so (47.4%). CONCLUSION: Transmission of HBV infection among children of HBsAg-positive mothers in Malaysia is low. However, attention needs to be given to the high rate of HBsAgpositive mothers who are not on any regular follow-up.

Missed opportunities for earlier HIV-testing in patients with HIV infection referred to a tertiary hospital, a cross-sectional study.

INTRODUCTION: In Malaysia, the prevalence of missed opportunities for HIV-testing is unknown. Missed opportunities have been linked to late diagnosis of HIV and poorer outcome for patients. We describe missed opportunities for earlier HIV-testing in newly-HIV-diagnosed patients. METHODS: Cross sectional study. Adult patients diagnosed with HIV infection and had at least one medical encounter in a primary healthcare setting during three years prior to diagnosis were included. We collected data on sociodemographic characteristics, patient characteristics at diagnosis, HIV-related conditions and whether they were subjected to risk assessment and offered HIV testing during the three years prior to HIV diagnosis. RESULTS: 65 newly HIV-diagnosed patients (male: 92.3%; Malays: 52.4%; single: 66.7%; heterosexual: 41%; homosexual 24.6%; CD4 <350 at diagnosis: 63%). 93.8% were unaware of their HIV status at diagnosis. Up to 56.9% had presented with HIV-related conditions at a primary healthcare facility during the three years prior to diagnosis. Slightly more than half were had risk assessment done and only 33.8% were offered HIV-testing. CONCLUSIONS: Missed opportunities for HIV-testing was unacceptably high with insufficient risk assessment and offering of HIV-testing. Risk assessment must be promoted and primary care physicians must be trained to recognize HIV-related conditions that will prompt them to offer HIVtesting.

Enteropathy-associated T-cell lymphoma: An extremely rare cause of chronic diarrhoea.

Chronic diarrhoea in tropical countries may be due to a myriad of causes from infective to non-infective. This case report illustrates the challenges faced in the investigation of a middle-age Chinese gentleman who presented with chronic diarrhoea and weight loss. The diagnosis of type II enteropathy-associated T-cell lymphoma (EATL) was finally made. The diagnosis of EATL was least suspected as the condition is almost unheard of in this part of the world. The epidemiology, presentation, diagnosis, management and prognosis of this rare condition are discussed.

Accelerating prevention of mother-to-child transmission of HIV: ten-year experience of universal antenatal HIV testing programme in a low HIV prevalence setting in Hong Kong.

Hong Kong has a low prevalence of HIV infection at less than 0.01%. Universal Antenatal HIV Testing Programme (UATP) was launched in all public antenatal units in September 2001. In 2008, voluntary rapid HIV testing was introduced in all public labour units to fill up the gap for pregnant women without HIV testing during the antenatal period. This study evaluated the performance of UATP and rapid HIV testing with indicators. From September 2001 to December 2011, process and outcome indicators for monitoring and evaluation were collected from the service providers in the form of monthly return of workload statistics and case-based statistics of each identified HIV-positive pregnancy via reporting forms. A total of 479,160 antenatal HIV tests and 2,675 rapid tests were performed in the study period. The acceptance rate for UATP and rapid HIV testing was 98% and 80.4% respectively. With the implementation of rapid HIV testing in January 2008, the proportion of pregnant women with HIV status discerned before delivery increased from 84.9% in 2006 to over 99.5% since 2008. The HIV prevalence in UATP and rapid HIV testing was 0.02% and 0.1% respectively. Fifty-three (68%) out of 78 HIV-infected pregnant women identified from the programme have delivered locally. Forty-three (81%) of them delivered by caesarean section and 50 (94%) of them were given antiretrovirals for intervention. Only three children born before the implementation of rapid HIV testing were HIV-infected. In conclusion, UATP and its rapid HIV testing component have been highly accepted and effective in the prevention of perinatal HIV transmission in Hong Kong.

Confronting Inequalities and Bridging the Divide: A Retrospective Study Assessment of Country-Level COVID-19 Vaccine Equality with a Cox Regression Model.

COVID-19 vaccination is vital in reducing illness, hospitalization, and mortality in the face of this global pandemic. However, COVID-19 vaccination rates worldwide remain below WHO public health targets, and persistent structural inequities reduce vaccine uptake likelihood among populations of low socioeconomic status. We conducted a cross-sectional study based on publicly available data from the Our World in Data project. We included all 124 countries with available open epidemic data and a population of more than 5 million. We used a Cox Regression Model, with population, population density, median age, human development index, GDP per capita, gender inequality index, healthcare access and quality index, hospital beds per thousand people, completion rate of primary education, infection cases of COVID-19 by the end of 2022, and death rate due to COVID-19 by the end of 2022 as predictors for model hazard rates of completion of 50% population vaccination. According to our study, countries with higher populations, higher population density, higher human development index, lower gender inequality index, and lower hospital beds per 1000 people had a higher hazard rate, which means they were more likely to achieve 50% population vaccination faster. By utilizing the time to achieve vaccination rate goals as our primary endpoint, we evaluated inequity from a dual perspective, considering both the differences in vaccination rates and the duration required to attain them. Consequently, this study employed survival analysis approaches to gain a comprehensive understanding of vaccine drivers and population-level trends nationally and inform all communities from a statistical perspective to prepare for health emergencies. Development-level standing modified the effects of equal access to COVID-19 vaccination on cumulative cases and mortality, for which countries of low or medium human development tended to fare worse in outcomes than high human development countries. As COVID-19 vaccination efforts evolve, healthcare professionals, scholars, and policymakers need to identify the structural impediments to equitable vaccination awareness, access, and uptake so that future vaccination campaigns are not impeded by these barriers to immunization. Recognizing the complex nature of this significant barrier, it is evident that no single statistical analysis method can comprehensively address all intricacies.

Transcriptome profiling of UPF3B/NMD-deficient lymphoblastoid cells from patients with various forms of intellectual disability.

The nonsense-mediated mRNA decay (NMD) pathway was originally discovered by virtue of its ability to rapidly degrade aberrant mRNAs with premature termination codons. More recently, it was shown that NMD also directly regulates subsets of normal transcripts, suggesting that NMD has roles in normal biological processes. Indeed, several NMD factors have been shown to regulate neurological events (for example, neurogenesis and synaptic plasticity) in numerous vertebrate species. In man, mutations in the NMD factor gene UPF3B, which disrupts a branch of the NMD pathway, cause various forms of intellectual disability (ID). Using Epstein Barr virus-immortalized B cells, also known as lymphoblastoid cell lines (LCLs), from ID patients that have loss-of-function mutations in UPF3B, we investigated the genome-wide consequences of compromised NMD and the role of NMD in neuronal development and function. We found that ~5% of the human transcriptome is impacted in UPF3B patients. The UPF3B paralog, UPF3A, is stabilized in all UPF3B patients, and partially compensates for the loss of UPF3B function. Interestingly, UPF3A protein, but not mRNA, was stabilised in a quantitative manner that inversely correlated with the severity of patients' phenotype. This suggested that the ability to stabilize the UPF3A protein is a crucial modifier of the neurological symptoms due to loss of UPF3B. We also identified ARHGAP24, which encodes a GTPase-activating protein, as a canonical target of NMD, and we provide evidence that deregulation of this gene inhibits axon and dendrite outgrowth and branching. Our results demonstrate that the UPF3B-dependent NMD pathway is a major regulator of the transcriptome and that its targets have important roles in neuronal cells.

Legislations combating counterfeit drugs in Hong Kong.

OBJECTIVE: To understand legislation combating counterfeit drugs in Hong Kong. DESIGN: This study consisted of two parts. In part I, counterfeit drugs­related ordinances and court cases were reviewed. In part II, indepth interviews of the stakeholders were described. SETTING: Hong Kong. PARTICIPANTS: All Hong Kong ordinances were screened manually to identify those combating counterfeit drugs. Court cases were searched for each of the identified cases. Then, the relevant judgement justifications were analysed to identify sentencing issues. Indepth interviews with the stakeholders were conducted to understand their perceptions about such legislation. RESULTS: Trade Marks Ordinance, Patents Ordinance, Trade Descriptions Ordinance, and Pharmacy and Poisons Ordinance were current legislative items combating counterfeit drugs. Sentencing criteria depended on: intention to deceive, quantity of seized drugs, presence of expected therapeutic effect or toxic ingredients, previous criminal records, cooperativeness with Customs officers, honest confessions, pleas of guilty, types of drugs, and precautionary measures to prevent sale of counterfeit drugs. Stakeholders' perceptions were explored with respect to legislation regarding the scale and significance of the counterfeit drug problem, penalties and deterrents, drug-specific legislation and authority, and inspections and enforcement. CONCLUSIONS: To plug the loopholes, a specific law with heavy penalties should be adopted. This could be supplemented by non-legal measures like education of judges, lawyers, and the public; publishing the names of offending pharmacies; and emphasising the role of pharmacists to the public.

Tuberculin sensitivity testing and treatment of latent tuberculosis remains effective for tuberculosis control in human immunodeficiency virus-infected patients in Hong Kong.

OBJECTIVE: To evaluate whether a policy to treat latent tuberculosis identified by annual tuberculin sensitivity testing is effective for tuberculosis control in human immunodeficiency virus-infected patients in Hong Kong. DESIGN: Historical cohort study. SETTING: Integrated Treatment Centre, Department of Health, Hong Kong. PATIENTS: Patients infected with human immunodeficiency virus without a history of tuberculosis were offered annual tuberculin sensitivity testing, coupled with treatment of latent tuberculosis if they tested positive. All such patients were followed for new tuberculosis. RESULTS: In all, 1154 patients on antiretroviral therapy, contributing to 5587 patient-years of observation, were analysed; 1032 patients (89%) received annual tuberculin sensitivity testing. Their baseline characteristics, including CD4 counts and other risk factors for tuberculosis, did not differ significantly from those who declined testing. The overall incidence rate of tuberculosis was 0.59 case per 100 patient-years. It was lower in those who received annual tuberculin sensitivity testing than those who did not (0.41 vs 3.85 per 100 patient-years; P<0.0001). Only a low baseline CD4 count and a history of tuberculin sensitivity testing were shown to be significant indicators of incident tuberculosis using multivariate analysis. The hazard ratio was 0.36 (95% confidence interval, 0.16-0.85; P=0.02) for those with a baseline CD4 count of 100/mm3 or above, and 0.26 (95% confidence interval, 0.08-0.77; P=0.016) for those who received annual tuberculin sensitivity testing. The incidence of tuberculosis was highest within 90 days of antiretroviral therapy initiation. CONCLUSION: The established policy continues to be effective. The high risk of tuberculosis during the early period of antiretroviral therapy supports early use of tuberculin sensitivity testing. Alternatively, the strategy of universal isoniazid preventive therapy at antiretroviral therapy initiation could be studied for those with very low baseline CD4 counts.

Mortality and health services utilisation among older people with advanced cognitive impairment living in residential care homes.

OBJECTIVES: To study the demography, clinical characteristics, service utilisation, mortality, and predictors of mortality in older residential care home residents with advanced cognitive impairment. DESIGN: Cohort longitudinal study. SETTING: Residential care homes for the elderly in Hong Kong West. PARTICIPANTS: Residents of such homes aged 65 years or more with advanced cognitive impairment. RESULTS: In all, 312 such residential care home residents (71 men and 241 women) were studied. Their mean age was 88 (standard deviation, 8) years and their mean Barthel Index 20 score was 1.5 (standard deviation, 2.0). In all, 164 (53%) were receiving enteral feeding. Nearly all of them had urinary and bowel incontinence. Apart from Community Geriatric Assessment Team clinics, 119 (38%) of the residents attended other clinics outside their residential care homes. In all, 107 (34%) died within 1 year; those who died within 1 year used significantly more emergency and hospital services (P<0.001), and utilised more services from community care nurses for wound care (P=0.001), enteral feeding tube care (P=0.018), and urinary catheter care (P<0.001). Independent risk factors for 1-year mortality were active pressure sores (P=0.0037), enteral feeding (P=0.008), having a urinary catheter (P=0.0036), and suffering from chronic obstructive pulmonary disease (P=0.011). A history of pneumococcal vaccination was protective with respect to 1-year mortality (P=0.004). CONCLUSION: Residents of residential care homes for the elderly with advanced cognitive impairment were frail, exhibited multiple co-morbidities and high mortality. They were frequent users of out-patient, emergency, and in-patient services. The development of end-of-life care services in residential care homes for the elderly is an important need for this group of elderly.

Treatment outcomes after early initiation of antiretroviral therapy for human immunodeficiency virus-associated tuberculosis.

OBJECTIVE: To evaluate the optimal timing for initiating antiretroviral therapy in patients with human immunodeficiency virus (HIV)-associated tuberculosis in Hong Kong. DESIGN: Historical cohort. SETTING. Tuberculosis and Chest Service and Special Preventive Programme, Public Health Service Branch, Centre for Health Protection, Department of Health, Hong Kong. PATIENTS: Consecutive patients with HIV-associated tuberculosis in a territory-wide TB-HIV registry encountered from 1996 to 2009. RESULTS: Of the 260 antiretroviral therapy-naïve patients with HIV-associated tuberculosis, 32 (12%) had antiretroviral therapy initiated within 2 months after starting anti-tuberculosis treatment (early antiretroviral therapy). Early antiretroviral therapy was associated with a more favourable outcome (cure or treatment completion without relapse) at 24 months (91% vs 67%; P=0.007) than those with antiretroviral therapy started later or not initiated, and remained an independent predictor of a favourable outcome after adjustment for potential confounders. Adverse effects from anti-tuberculosis drugs tended to occur more frequently in patients with early antiretroviral therapy (13/32 or 41%) compared with the remainder (59/228 or 26%; P=0.08). A significantly higher proportion of patients in the former group experienced immune reconstitution inflammatory syndrome than in the latter group (7/32 or 22% vs 9/228 or 4%; P<0.001). There was no death attributable to immune reconstitution inflammatory syndrome. CONCLUSIONS: Early initiation of antiretroviral therapy is associated with more favourable tuberculosis treatment outcomes in patients with HIV-associated tuberculosis with a low CD4 count (<200/µL). Drug co-toxicity and immune reconstitution inflammatory syndrome that may be increased by earlier initiation of antiretroviral therapy does not undermine tuberculosis treatment outcomes to a significant extent.

Towards data-driven tele-medicine intelligence: community-based mental healthcare paradigm shift for smart aging amid COVID-19 pandemic.

Purpose: Telemedicine are experiencing an unprecedented boom globally since the beginning of the COVID-19 pandemic. As the most vulnerable groups amid COVID-19, the digital delivery of healthcare poses great challenges to the elderly population, caregiver, health service providers, and health policy makers. To bridge the service delivery gaps between the telemedicine demand side and supply side, explore evidence-based approach for integrated care, address challenges for aging policy, and build foundation for the development of data-driven and community-based telemedicine, our R&D team applied translational research to design and develop telemedicine "SMART" for enhancing elderly mental health wellbeing amid COVID-19. Our aim is to investigate the preparedness mechanisms of mental health disease including response, intervention, and connection these three healthcare delivery pipelines with the collection, consolidation, and synergy of heath parameters and social determinants, using data analytics approach to achieve Evidence-Based Medicine (EBM). Methods: A mix of quantitative and qualitative research design for scientifically rigorous consultation and analysis was conducted from Jan 2020 to June 2021 in Hong Kong. An exploratory and descriptive qualitative design was used in this study. The data were collected through focus group discussions conducted from elderly and their caregivers living in 10 main districts of Hong Kong. Our research pilot tested "SMART" targeting for elderly with mental health improvement needs. Baseline questionnaire with 110 tele-medicine product users includes questions on demographic information, self-rated mental health digital adoption. The follow-up five focus group discussions with 57 users (elderly and their caregivers) further explore the social determinants of telemedicine transformation and help propose the integrated telemedicine paradigm shift framework establishment, development, and enhancement. Results: Grounded on the baseline needs assessment and feedbacks collected, it is evident that multi-dimensional health information from the four various streams (community, clinic, home, remote) and customized digital health solutions are playing a key role in addressing elderly mental health digital service needs and bridging digital divide. The designed tele-medicine product lines up health service provider (supplier side) and elderly specific needs (demand side) with our three-level design, enables elderly and their families to follow and control their own health management and connect with the service provider, community of practice (CoP), and health policy makers. Conclusion: It's beneficial to involve elderly and gerontechnology stakeholders as part of Community-Based Participatory Research (CBPR) before and throughout the developing and delivery phases an integrated and age-friendly digital intervention. The challenges in applying and disseminating telemedicine reflected by the elderly and caregivers can be used as important input for further development and indicators for the sustainable and integrated elderly primary care framework. Supplementary Information: The online version contains supplementary material available at 10.1007/s13755-022-00198-4.

Antibacterial activity of ZnO nanoparticles with a modified surface under ambient illumination.

In various practical applications, nanomaterials typically have functionalized surfaces. Yet, the studies of toxicity and antibacterial activity of functionalized nanoparticles are scarce. We investigated the effect of surface modifications on antibacterial activity of ZnO under ambient illumination, and we found that nanoparticles coated with different surface modifying reagents could exhibit higher or lower toxicity compared to bare ZnO, depending on the surface modifying reagent used. Different surface modifying reagent molecules resulted in differences in the release of Zn(2+) ions and the production of reactive oxygen species (ROS). However, the antibacterial activity did not correlate with the ROS levels or the Zn(2+) ion release. One of the surface-modified ZnO samples exhibited significantly lower Zn(2+) ion release while at the same time exhibiting improved antibacterial activity. In all cases, damage of the cell wall membranes and/or changes in the membrane permeability have been observed, together with the changes in ATR-FTIR spectra indicating differences in protein conformation. Mechanisms of antibacterial activity are discussed.

Spindle cell/sclerosing rhabdomyosarcoma with DCTN1::ALK fusion: broadening the molecular spectrum with potential therapeutic implications.

Spindle cell/sclerosing rbabdomyosarcoma (RMS) is a recently characterized variant of RMS with several distinct molecular subtypes. We describe an example occurring in the tongue of a 10-year-old boy with a novel DCTN1::ALK fusion. The tumor exhibited infiltrative growth and was comprised of fascicles and focally whorls of spindle cells with eosinophilic cytoplasm, in a collagenous or myxoid stroma. Moderate cytologic atypia, mitotic activity (2/10 HPFs), and perineural invasion were identified. The tumor cells expressed actin, desmin, MyoD1, myogenin, and ALK. An in-frame fusion between DCTN1 exon 26 and ALK exon 20 was detected by RNA sequencing, which was confirmed by split reads and supported by FISH studies. The tumor showed an indolent behavior with local recurrence 3 years after excision. This study broadens the molecular spectrum of spindle cell/sclerosing RMS and this molecular aberration may represent a potential therapeutic target for unresectable or disseminated disease.

Enzyme replacement therapy for mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): experience in Hong Kong.

Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) is a very rare inherited lysosomal storage disease. We evaluated the efficacy and safety of weekly infusions of recombinant human arylsulfatase B as enzyme replacement therapy for two patients in whom this condition was advanced. The primary outcome variables were the distance walked in a 6-minute walk test, forced vital capacity, and ejection fraction. The secondary outcome variables were the number of stairs climbed in a 3-minute stair climbing test, joint mobility, urinary glycosaminoglycan excretion, auto-continuous positive airway pressure study and liver size. After 24 weeks of treatment, patient A walked 40 m (36%) and patient B walked 66 m (58%) more in the walk test than at baseline. After 48 weeks, in patient A the corresponding improvements were 142 m (129%) in the walk test and 33 stairs (60%) in the 3-minute stair climbing test, and in patient B the respective improvements were 198 m (174%) and 77 stairs (140%). There was a significant decline in urinary glycosaminoglycan excretion and improvement in range of motion of joints in both patients. The auto-continuous positive airway pressure study revealed improvements in patient A, while other efficacy variables remained static. There were no drug-related adverse events or allergic reactions reported during and after the infusions of recombinant human arylsulfatase B. Recombinant human arylsulfatase B significantly improves endurance and reduces urinary glycosaminoglycan excretion. The drug is generally safe and well tolerated.