Improvement in symptoms and health-related quality of life in acromegaly patients: a systematic review and meta-analysis.

BACKGROUND: While biochemical response is often used as primary study outcome, improvement in symptoms and health-related quality of life (HRQoL) is the relevant goal for patients to consider treatment successful. We performed a systematic review and meta-analysis to assess the effect of treatment on symptoms and HRQoL in acromegaly. METHODS: Seven electronic databases were searched for longitudinal studies assessing patient-reported symptoms or HRQoL in acromegaly. Meta-analyses were performed to assess differences during treatment for the Acromegaly Quality of Life Questionnaire (AcroQoL) and Patient-Assessed Acromegaly Symptom Questionnaire (PASQ), and standardized mean difference (SMD) for individual symptoms (interpretation: 0.2 small, 0.5 moderate, and 0.8 large effect). Treatment naive and previously treated patients were assessed separately. RESULTS: Forty-six studies with 3301 patients were included; 24 contributed to quantitative analyses. Thirty-six studies used medication as main treatment, one transsphenoidal adenomectomy, and nine various treatments. Both symptoms and HRQoL improved: AcroQoL increased: 2.9 (95% CI: 0.5, 5.3) points, PASQ decreased: -2.3 (95% CI: -1.3, -3.3) points, and individual symptom scores decreased: paresthesia -0.9 (95% CI: -0.6, -1.2), hyperhidrosis -0.4 (95% CI: -0.1, -0.6), fatigue -0.3 (95% CI: -0.1, -0.6), arthralgia -0.3 (95% CI: -0.1, -0.5), headache -0.3 (95% CI: 0.0, -0.6), and soft tissue swelling -0.2 (95% CI: 0.0, -0.4). CONCLUSION: Symptoms and HRQoL improved during acromegaly treatment. Consensus is needed on which symptoms should be included in a potential Core Outcome Set, taking into account symptom frequency, severity, and sensitivity to change, which can be used in clinical practice and as outcome in trials.