Clinical recommendations for diagnosis and management of Mendelian susceptibility to mycobacterial disease in resource-limited settings

资源匮乏地区对孟德尔遗传性分枝杆菌病进行诊断和治疗的临床建议

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Abstract

BACKGROUND: Mendelian susceptibility to mycobacterial disease (MSMD) is a rare inborn error of immunity caused by defects in IL-12 or IFN-γ signaling pathways, essential for intracellular pathogen control. While classically linked to nontuberculous mycobacteria, MSMD also predisposes to bacterial, fungal, and viral infections. OBJECTIVE: We sought to provide a practical framework for diagnosing and managing MSMD in African settings. Recommendations integrate clinical evaluation, baseline immunologic testing, and molecular diagnostics. METHODS: A review of clinical presentation, diagnostic strategies, and therapeutic approaches was conducted, emphasizing applicability in resource-limited environments. RESULTS: MSMD presents across a wide clinical spectrum that includes severe disseminated early-onset infections and localized later-onset disease. While classically characterized by increased susceptibility to nontuberculous mycobacteria, MSMD also predisposes to bacterial, fungal, and viral infections. Standard immunologic screening often yields unremarkable findings, necessitating a tiered diagnostic approach that includes molecular testing for confirmation. Long-term antimicrobial therapy remains the cornerstone of management, with prophylaxis warranted in recurrent or severe cases. Early identification significantly reduces morbidity and mortality. Molecular diagnostic methods facilitate genotype-guided management, enabling precision medicine approaches in this context. CONCLUSION: Timely recognition of MSMD, particularly in tuberculosis-endemic settings, is critical to prevent severe disease progression. A structured diagnostic algorithm, combined with molecular testing and individualized treatment strategies, enhances outcomes. MSMD underscores the significance of genotype-driven management.

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