Teprotumumab for patients with active thyroid eye disease: a pooled data analysis, subgroup analyses, and off-treatment follow-up results from two randomised, double-masked, placebo-controlled, multicentre trials

Quality of Life in Patients with Chronic Thyroid Eye Disease in the United States

“…QOL remained heavily impaired, as indicated by overall, vision-related, and appearance-related GO-QOL scores that averaged approximately 60 points. Interestingly, these scores were nearly identical to those observed in the teprotumumab clinical trial population of recent-onset (≤ 9 months), active, moderate-to-severe TED patients [ 13 ]. Our findings are in agreement with patient experiences [ 19 ] and prior studies outside of the US, which have shown a marked impact of TED on patient-reported QOL [ 20 – 24 ].…”

“…Further, QOL was previously evaluated in two placebo-controlled trials in 171 randomized moderate-to-severe active TED patients with recent (≤ 9 months) symptom onset. Baseline QOL was measured in these patients with a TED-specific instrument (Graves’ Ophthalmopathy Quality of Life questionnaire [GO-QOL]) and demonstrated a low mean QOL score (61 out of 100) [ 13 ]. However, the long-term QOL impact of chronic TED on US patients has not been well reported.…”

Response to comment by Smith on the 2021 EUGOGO guidelines

“…The authors of this response are in line with Dr Smith that the antiinsulin-like growth factor 1 receptor inhibiting monoclonal antibody, teprotumumab is a very promising drug for the management of active and moderate-tosevere GO (8), and this is repeatedly and clearly stated in the text part, the table of recommendations, and in the legend of box three (2). However, guidelines are 'made for everybody', offering evidence-based recommendations, which should be possible to apply and follow worldwide.…”

“…reported hearing loss, cognitive decline, and amyloid encephalopathy, etc.) (8,9) are pending and warranted.…”

Orbital Signaling in Graves’ Orbitopathy

“…Further studies have highlighted the importance of down stream factors of IGF1-PI3K signalling and revealed that FOXOs, may mediate both TSHR and IGF1R signalling pathways in GO (64). The notion is further supported by recent successful trial of teprotumumabmonoclonal antibody which blocks IGF1R -in reducing proptosis in patients with GO (65,66). Whilst effective medical treatment for GO is welcome, some concerns have been raised about these trials including the lack of orbital imaging and the fact that despite QOL scores being improved in the teprotumumab group, all patients scores remained low (67).…”