Update of the Brazilian Clinical Practice Guideline for primary immune thrombocytopenia: methodological process and main changes for diagnosis and treatment in the Brazilian Unified Health System (SUS)

BACKGROUND: Primary immune thrombocytopenia requires diagnostic exclusion and individualized management. Recent changes in the therapeutic landscape and health technology assessment recommendations of Brazil prompted an update of the national Clinical Practice Guidelines for immune thrombocytopenia in the Unified Health System (SUS). OBJECTIVES: This study aims to describe the methodological process used to update the guidelines for primary immune thrombocytopenia, summarize the primary conceptual, diagnostic, and therapeutic updates, and emphasize the integration of health technology assessment into treatment pathways within the Brazilian Unified Health System. METHODS: The update followed the technical guide of the Brazilian Ministry of Health for guideline development. Recommendations were appraised by the National Committee for Health Technology Incorporation (Conitec) and drafted by a multidisciplinary panel including health technology assessment researchers, clinical experts and policymakers. Systematic reviews, economic evaluations, budget impact analyses, stakeholder input and public consultation drafted the final recommendations. RESULTS: The updated Clinical Practice Guidelines changed the title and conceptual framing to reflect contemporary disease nomenclature and immune‑mediated pathophysiology, reinforced a diagnostic approach based on clinical evaluation and exclusion of alternative causes of thrombocytopenia, and reorganized classification criteria. Rituximab and romiplostim were incorporated for selected adult and pediatric populations with refractory, chronic or corticosteroid‑dependent disease, whereas dapsone was not incorporated. Treatment sequencing was clarified, positioning immunosuppressive therapies before thrombopoietin receptor agonists and specifying age‑related indications. A public consultation refined the wording and interpretation of the therapeutic sequencing, particularly for pediatric care. CONCLUSION: The updated Brazilian Clinical Practice Guidelines for primary immune thrombocytopenia modernizes disease framing, expands treatment options and improves organization of therapeutic decision‑making in the Unified Health System (SUS), illustrating how health technology assessment outputs can be integrated into evidence‑informed national clinical protocols.