Identifying and categorizing compounds that reduce corneal transforming growth factor beta induced protein levels: a scoping review

Sciriha, Gabriella Guo; Sultana, Janet; Borg, Joseph

doi:10.1080/17512433.2022.2142560

Cited by 3 publications

(1 citation statement)

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“…Drug repurposing has been used in relatively common diseases such as Alzheimer’s disease, where finding viable therapeutic agents is very challenging, but it has also been used in rare diseases, such as corneal dystrophy. 22 The role of repurposing emerges from the present study as the majority of drugs which have been included in trials were off-label drugs. The main limitation for GPA, as well as many other rare diseases, is that the pathophysiology is not fully understood, making it difficult to find therapeutic targets for drugs to reach.…”

Section: Discussionmentioning

confidence: 87%

The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform

Sultana,

Camilleri,

Crisafulli

et al. 2023

Therapeutic Advances in Rare Disease

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To date, there is no published overview of the drug pipeline in granulomatosis with polyangiitis (GPA), a rare disease. The aim of this study was to identify clinical trials from two study repositories. A review of clinical trials was conducted using publicly available data. Clinicaltrials.gov and International Clinical Trials Registry Platform were searched from inception until 25 September 2022. Only GPA-specific studies were included; these were described in detail. A total of 137 studies were identified in the trial repositories, of which 108 (79%) studies were found to concern GPA. Of these 108 studies, 67 enrolled GPA patients to investigate pharmacotherapy in this disease (62%). Most studies included all severity types ( n = 51; 76%); the scope of almost half of the studies was remission induction ( n = 33; 49%). The drug class which was by the most widely investigated in trials was the non-corticosteroid immunosuppressant drug class (46; 68.7%), monoclonal antibodies (32; 47.8%), and corticosteroids (31; 46.3%). There is a need for more GPA trials to generate evidence on effectiveness in terms of severity-specificity and maintenance of remission.

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Section: Discussionmentioning

confidence: 87%