Background: There is a substantial expansion in the number of biologics available for the treatment of JIA and childhood rheumatic disease over the past decades. However, there is no report on their use and safety profile in the local paediatric population. Method of study: This is a multi-centred retrospective cohort study, recruiting patients followed up in three Paediatric departments in Hong Kong. Subjects who were followed up for childhood-onset rheumatic diseases and was treated with any of the biologic DMARs on or before the 31st December 2022, were reviewed. Results: A total of 96 patients who received biologic DMARs in the studied period were reviewed. Sixty patients were diagnosed to have JIA, cSLE (10), uveitis (8), refractory KD (7), JDM (2), COVID-MISC (3), and others (6). Concerning the JIA group, male: female is 1.4:1. 38% of the patients had polyarticular JIA and 50% had ERA who failed conventional treatment with various cDMARS. Concerning the nature of biologics, 85% of JIA patients used different types of TNF-blockers, including etanercept (38.3%), adalimumab (40%), golimumab (5%) and infliximab (3.3%). Six JIA patients used tocilizumab and one used abatacept as second-line treatment after fail response to TNF-blockers. One JIA patient was on tofacitinib to avoid subcutaneous injection. For other biologics, infliximab for refractory KD (8), rituximab for cSLE and JDM (7 and 1), belimumab in cSLE (2) and anakinra in COVID-MISC (4). One JIA-ERA patient developed lymphoma six months after stopping adalimumab and recovered after chemotherapy. One cSLE patient received rituximab and developed EBV-related CNS lymphoma with repeated infections that required hospitalization. Most patients tolerated the biologic DMARs well without complications. There was no tuberculosis infection or activation. Two cSLE patients developed herpes zoster after rituximab infusion and required hospital treatment. Two patients on adalimumab developed recurrent folliculitis and one had to stop treatment. One patient developed bacteria endophthalmitis while on adalimumab. Four patients on adalimumab developed injection site reactions and one patient need to change to another biologic. One patient on adalimumab developed positive lupus serology without clinical features. Conclusion: A broad spectrum of biologics has been used in children with various rheumatic diseases without major complications. Although rare, 2 patients developed lymphoma during the study period. Although there is no causal relationship identified with the use of biologics, continuous monitoring of the long-term safety of these drugs in the paediatric cohort is recommended.
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.
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.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.
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