Improved patient-reported outcomes (PROs) are increasingly becoming a key treatment objective in acromegaly. Validated PROs were used to assess disease and treatment burden in the MPOWERED phase 3 trial in acromegaly, which also assessed safety and efficacy of oral octreotide capsules (OOC; MYCAPSSA®) compared to injectable SRLs (iSRLs).
Eligible patients had acromegaly diagnosis, biochemical control of acromegaly (insulin-like growth factor I <1.3 × upper limit of normal; mean integrated growth hormone, <2.5 ng/ml) and ≥6 months iSRL treatment (octreotide or lanreotide). Eligible patients entered a 26-week Run-in phase to determine the effective OOC dose; responders at week 24 then entered a 36-week randomized controlled treatment (RCT) phase receiving OOC or iSRLs in a 3:2 ratio. The Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ) is a recently validated tool that includes 27 items in 6 domain scores for PROs in acromegaly.1 Acro-TSQ data were collected at baseline (reflecting outcomes on iSRLs), end of Run-in (reflecting outcomes on OOC), and end of RCT (OOC or iSRLs).
Of 146 enrolled patients, 92 entered RCT (OOC, N = 55; iSRLs, N = 37). Acro-TSQ scores at the end of Run-in (26 weeks OOC treatment) were compared to baseline (iSRLs). In the 92 patients randomized, 3 of 5 Acro-TSQ domains (emotional reaction, treatment convenience, and treatment satisfaction) showed significant improvement at end of Run-in compared to baseline. Injection site interference was not assessed as no injection site reactions were observed with OOC. Other domains showed a nonstatistically significant pattern of improvement at end of Run-in when compared to baseline. Patients randomized to iSRLs in the RCT after receiving OOC in the Run-in (N = 37) reported more anxiety (RCT end, 53%; Run-in end, 29%) and frustration (RCT end, 45%; Run-in end, 34%) with iSRLs compared to OOC. Overall treatment satisfaction was higher while receiving OOC (Run-in end, 92%; after receiving iSRLs in RCT, 75%). Breakthrough symptoms were reported more frequently with iSRLs (31%) than OOC (15%) at the end of RCT.
Higher patient satisfaction, convenience and emotional well-being, and improved symptom control based on the newly validated Acro-TSQ PRO reporting tool were observed with OOC compared to iSRLs in patients enrolled in the MPOWERED trial.
1Fleseriu M, et al. Pituitary. 2020 Aug;23(4):347358.
22 May 2021 - 26 May 2021