Abstract
BackgroundWhereas biochemical response is often used as a 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.MethodsSeven 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.ResultsForty-six studies with 3301 patients were included; 24 contributed to quantitative analyses. Thirty-six studies used medication as main treatment, 1 transsphenoidal adenomectomy, and 9 various treatments. Symptoms and HRQoL both improved: AcroQoL increased 2.9 points (95% CI, 0.5 to 5.3 points), PASQ decreased –2.3 points (95% CI, –1.3 to –3.3 points), and individual symptom scores decreased for paresthesia –0.9 (95% CI, –0.6 to –1.2), hyperhidrosis –0.4 (95% CI, –0.1 to –0.6), fatigue –0.3 (95% CI, –0.1 to –0.6), arthralgia –0.3 (95% CI, –0.1 to –0.5), headache –0.3 (95% CI, 0.0 to –0.6), and soft-tissue swelling –0.2 (95% CI, 0.0 to –0.4).ConclusionSymptoms 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.
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More From: The Journal of Clinical Endocrinology & Metabolism
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