How to Define Success in Prolactinoma Treatment-A Systematic Review and Theoretical Framework.

Area of Science:

• Endocrinology
• Clinical Medicine
• Medical Research Methodology

Background:

• Lack of consensus on outcome sets for prolactinoma treatment evaluation hinders comparative analysis.
• Existing literature reports diverse and often subjective parameters for assessing treatment success.

Purpose of the Study:

• To evaluate reported outcome parameters in prolactinoma treatment literature.
• To propose objective, clinically relevant outcome sets for evaluating treatment success.

Main Methods:

• Systematic review of 137 articles up to February 2, 2024.
• Extraction of biochemical, radiological, clinician-reported, and patient-reported outcomes (PROs).
• Development of objective definitions for disease remission, control, and recurrence.

Main Results:

• Significant heterogeneity in reported prolactin (23 parameters) and radiological (73 parameters) measures.
• Absence of PROs in all reviewed studies; clinician-reported findings in 70 articles.
• Numerous definitions for remission (27), control (3), and recurrence (20) were identified.
• Proposal of distinct biochemical and clinical definitions for remission/recurrence, incorporating symptomology, gonadal function, and radiology.

Conclusions:

• Heterogeneity in outcome reporting impedes comparison of prolactinoma treatment outcomes.
• Proposed objective definitions and a comprehensive outcome set facilitate standardized evaluation.
• Implementation of these parameters will enable better comparison across treatments and centers, advancing prolactinoma care.