Christiaan Scott is Professor of Paediatric Rheumatology the University of Ottawa and the Children’s Hospital of Eastern Ontario. He is the former head of pediatric rheumatology at the University of Cape Town and the Red Cross War Memorial Children’s Hospital and the former director of the University of Cape Town’s Clinical Research Centre. His research and education work focuses on the care of patients with rare and rheumatic diseases in less resourced communities. Dr Scott has a special interest in education and access to care in musculoskeletal conditions, especially in Fibrodysplasia Ossificans Progressiva (FOP). He is the Chairperson of the Global Task Force for Paediatric Musculoskeletal Health and Medical Director of Tin Soldiers Global, a rare disease identification program. He is a member and former Chair of the International Clinical Council for FOP. He serves on the editorial board of Paediatric Rheumatology Online Journal and Best Practice and Research Clinical Rheumatology.
Research Projects
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Development of CARRA/PReS-endorsed consensus Core and Expanded Datasets in childhood-onset systemic lupus erythematosus for international registry-based research
25/01/2025
Children and adolescents with childhood-onset systemic lupus erythematosus (cSLE) generally experience more severe disease manifestations when compared with people with adult-onset SLE (aSLE) [[1], [2], [3]]. Patients with cSLE experience more active and aggressive disease, greater medication burden and more frequent involvement of internal organs, such as renal, haematological and neuropsychiatric manifestations [[4], [5], [6], [7]]. Children with cSLE have a higher standardised mortality rate when compared with people who develop lupus in adulthood (aSLE) [[8], [9]]. Several cSLE registries exist worldwide, each intended to enhance our understanding of the epidemiology, phenotypes and prognosis of cSLE while also providing the opportunity to capture real-world data on medication response rates and toxicities [[2], [10], [11]]. Due to the rarity of cSLE, however, the study of specific disease manifestations remains underpowered using existing individual registries. Treatment paradigms for cSLE are currently based predominantly on adult lupus trial data, supplemented with data from relatively small paediatric clinical trials and observational studies [[11], [12]]. It remains unclear in which instances children with cSLE have similar treatment responses to individuals with aSLE, and many cSLE manifestations, such as neuropsychiatric disease, are still understudied. Furthermore, there is regional variability in the manifestations of cSLE that likely influences diagnosis, surveillance, treatment response and prognosis of the disease [13]. These challenges highlight the need for international clinical research that focuses specifically on cSLE [[2], [14], [15]]. Importantly, registry data from large cohorts serve as a powerful tool for unifying data collection for this rare and clinically heterogeneous disease to establish prognosis and determine optimal treatment and long-term safety.
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Palovarotene in fibrodysplasia ossificans progressiva: review and perspective
23/01/2025
Palovarotene is a retinoic acid receptor gamma (RAR-γ) agonist that was originally designed for treatment of chronic obstructive pulmonary disease (COPD) by Roche Pharmaceuticals [Citation1]. Although it did not demonstrate a significant benefit in individuals with advanced COPD, preclinical studies in FOP mouse models suggested that drug repurposing could potentially be useful for the treatment of fibrodysplasia ossificans progressiva (FOP) [Citation2–4]. Phase-2 and -3 clinical trials on palovarotene, as well as a parallel natural history study (NHS), were the first worldwide clinical studies to be conducted and completed in patients with FOP [Citation5–7]. After this promising start the regulatory review process was complicated by several hurdles, but as of this writing, palovarotene is approved for use in preventing new heterotopic bone formation in FOP in the U.S.A., Canada, and Australia, but not in Europe. In this article, we summarize the history of palovarotene, the preclinical and clinical studies, the regulatory processes, and clinical recommendations from experts in FOP clinical care.
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Current treatment in macrophage activation syndrome worldwide: a systematic literature review to inform the METAPHOR project
26/07/2024
MAS treatment remains largely empirical and based on expert consensus. Although promising data are emerging, results from large cohorts and standardized trials are still required for most medications used to treat MAS. Multinational data on systemic juvenile idiopathic arthritis (sJIA)-associated MAS highlighted several disparities in its management in relation to geographic location of the treating centre and subspecialty of the caring physicians [10]. Recently, the first international recommendations for the early-stage management of HLH/MAS have been published [11]. Despite their milestone relevance, these guidelines focus on the initial management of the spectrum of haemophagocytic syndromes and do not specifically address the treatment of MAS. Furthermore, there is a particular lack of evidence on the therapeutic approach to MAS associated with rheumatological conditions other than sJIA. It is thus conceivable that a wide heterogeneity in the management of MAS exists, due to differences in treatment strategies, access to medications and involvement of different specialists.