The use of Ambroxol for the treatment of Gaucher disease: A systematic review

Abstract Gaucher disease (GD) is a heterogeneous condition requiring tailored treatment approaches. The aim of this systematic review was to synthesise and evaluate current evidence pertaining to the use of Ambroxol for the treatment of GD. Published and unpublished literature databases, conference proceedings and the reference lists of included studies were searched until 23 November 2023. A narrative synthesis was performed. Database search and risk of bias assessment were performed independently by two reviewers. Twenty‐one studies (182 patients) were included. The evidence was low in quality. Variable responses to Ambroxol were observed. Response rates were 36% and 55% in two studies reporting on type 1 GD. One study found a 22% response rate in type 2 GD, whereas another study found 29% of patients with type 3 GD reported neurological improvements. No severe adverse events were reported in the literature, with mild and reversible side effects reported. Varying response rates are to be expected (29%–100%) when treating neurological manifestations. Varying degrees of symptomatic improvement for the treatment of GD were noted in the literature. Multidisciplinary team input and clinical judgement are advised to provide personalized treatment of this complex and multi‐faceted condition

Predisposing factors for Hoffa’s fat pad syndrome: a systematic review

Abstract Background Hoffa’s fat pad syndrome has been defined as impingement of Hoffa’s fat pad, leading to oedema and fibrosis. The primary aim of this systematic review was to identify morphological differences in Hoffa’s fat pad between patients with and without Hoffa’s fat pad syndrome, evaluating them as risk factors predisposing to its development. The secondary aim was to summarize and evaluate current evidence pertaining to the management of Hoffa’s fat pad syndrome. Materials and Methods The protocol for this review was prospectively registered (PROSPERO registration: CRD42022357036). Electronic databases, currently registered studies, conference proceedings and the reference lists of included studies were searched. All studies evaluating differences in Hoffa’s fat pad anatomy under imaging between patients with and without Hoffa’s fat pad syndrome were included, as well as those exploring epidemiological factors predisposing to its development (ethnicity, employment status, sex, age and BMI), and studies reporting on the effect of treatment on Hoffa’s fat pad morphology. Results A total of 3871 records were screened. Twenty one articles satisfied the inclusion criteria, evaluating 3603 knees of 3518 patients. Patella alta, increased tibial tubercle-tibial groove distance, and increased trochlear angle were found to predispose the development of Hoffa’s fat pad syndrome. Trochlear inclination, sulcus angle, patient age and BMI were not associated with this condition. The link between Hoffa’s fat pad syndrome and ethnicity, employment, patellar alignment, Hoffa’s fat pad composition, physical activity and other pathological processes cannot be established due to lack of evidence. No studies reporting on treatment for Hoffa’s fat pad syndrome were identified. Though weight loss and gene therapy may provide symptomatic relief, further research is required to corroborate these claims. Conclusion Current evidence suggests that high patellar height, TT-TG distance, and trochlear angle predispose the development of Hoffa’s fat pad syndrome. In addition, trochlear inclination, sulcus angle, patient age and BMI do not seem to be associated with this condition. Further research should explore the link between Hoffa’s fat pad syndrome and sport as well as other conditions pertaining to the knee. In addition, further study evaluating treatment approaches for Hoffa’s fat pad syndrome is required

Does medial patellofemoral ligament reconstruction result in femoral tunnel enlargement? A systematic review

Abstract Background Medial patellofemoral ligament (MPFL) reconstruction is a common surgical procedure for treating patellar instability. The primary aim of this systematic review was to determine whether MPFL reconstruction (MPFLR) leads to femoral tunnel enlargement (FTE). The secondary aims were to explore the clinical effects and risk factors of FTE. Electronic databases (MEDLINE, Global Health, Embase), currently registered studies, conference proceedings and the reference lists of included studies were searched independently by three reviewers. There were no constraints based on language or publication status. Study quality assessment was conducted. 3824 records were screened in the initial search. Seven studies satisfied the inclusion criteria, evaluating 380 knees in 365 patients. Rates of FTE following MPFLR ranged from 38.7 to 77.1%. Five low quality studies reported FTE did not lead to detrimental clinical outcomes as assessed with the Tegner, Kujala, IKDC, and Lysholm scores. There is conflicting evidence regarding change in femoral tunnel width over time. Three studies (of which two had a high risk of bias) reported age, BMI, presence of trochlear dysplasia and tibial tubercle-tibial groove distance did not differ between patients with and without FTE, suggesting these are not risk factors for FTE. Conclusion FTE is a common postoperative event following MPFLR. It does not predispose poor clinical outcomes. Current evidence lacks the ability to identify its risk factors. The reliability of any conclusions drawn is hindered by the low level of evidence of the studies included in this review. Larger prospective studies with long-term follow up are required to reliably ascertain the clinical effects of FTE