OBJECTIVEIn moderate-to-severe Graves' orbitopathy (GO), rituximab is recommended as second-line therapy in patients non-responsive to intravenous glucocorticoids. We aimed to evaluate rituximab as early second-line therapy, as data is scarce and contradictory.METHODSIn this non-randomized, controlled, interventional study, patients with GO started on intravenous glucocorticoids. After 4 weeks, patients with <2 points improvement in clinical activity score (CAS) were switched to rituximab [Non-Responders Rituximab (NR-RTX) group] and were compared to the remaining patients who continued with intravenous glucocorticoids for 12 weeks [Responders-Glucocorticoid (R-GC) group]. A retrospective group of non-responsive patients who were provided regular care with intravenous glucocorticoids for 12 weeks was used as control [Non-Responders-Regular Care (NR-RC) group]. Background data and CAS were recorded for all groups at 0, 4, 12, 18, and 68 weeks. Quality of life (QoL) and safety data were collected from the NR-RTX and R-GC groups.RESULTSThe NR-RTX group (n=12) was similar to the others at baseline except for a 1-point lower median CAS compared to the NR-RC group (n=12) (p=0.03), and for having twice as many men compared to the R-GC group (n=13) (p=0.03). At 4 weeks, a linear mixed model indicated that the R-GC group had a 1.21-point (95%CI: -2.40 to -0.02) lower value for CAS compared to the NR-RTX group. CAS for all groups converged over time. Similar models for QoL revealed no treatment or time effects.CONCLUSIONSwitch to RTX early in the treatment course did not result in better CAS or QoL, compared to continuous intravenous glucocorticoids.

2024-09-01·European Journal of Surgical Oncology

Clinical trials involving rare gynaecological cancers

Article

作者: Harasani, Klejda ; Čelebić, Aleksandar ; Tripac, Irina ; Calleja-Agius, Jean

Clinical trials that investigate therapies for rare gynaecological cancers (RGC) are essential to provide evidence-based data towards new effective and safe treatments, however, they present unique challenges. The main objective of this narrative review is to summarize completed phase III clinical trials investigating therapies for RGC and to discuss the outcomes of these trials. PRISMA guidelines were used to report the steps of the review. We searched the WHO's International Clinical Trials Registry Platform, clinicaltrials.gov and PubMed/Medline for publications reporting results from clinical trials on RGC including at least one medication. From 31 identified phase III clinical trials, 13 were still ongoing, four were terminated and just eight (25.8 %) had posted results and/or publications. The latter completed trials were mostly multi-center and located in at least two continents, participants were mainly adults, and the recruitment period varied from about 2.5 to 10.5 years. Allocation was randomized with parallel assignment in 7 out of 8 trials, while only one trial had double masking. The most common primary outcome measure was progression-free survival (PFS), followed by overall survival (OS). Patient-reported outcomes were secondary outcome measures in four completed trials, assessing quality of life by various questionnaires. Most of the trials did not meet their primary endpoints. By highlighting the scarcity of clinical trials on RGC, our findings further emphasize the need for designing, conducting and sustaining phase III clinical trials to investigate innovative therapies for these conditions and report meaningful outcome measures.

100 项与 RGC 相关的药物交易

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