Case reports reveal significant tumor shrinkage and rare diseases management with lorlatinib, suggesting careful monitoring of neurotoxicity.
Pseudosarcomatous myofibroblastic proliferation (PMP) is a rare anaplastic lymphoma kinase, ALK fusion-associated tumor-like lesion that requires differentiation from inflammatory myofibroblastic tumor (IMT). This article reports two cases of bladder ALK fusion-positive PMP: a 16-year-old male achieved partial remission (PR) postoperatively with lorlatinib but experienced memory decline and mood disturbances, which resolved after dose reduction; a 34-year-old female showed significant tumor shrinkage (PR) without severe adverse reactions. The study confirms the marked efficacy of ALK inhibitors in ALK-positive PMP, but highlights the need to monitor neurotoxicity in adolescent patients. Through analysis of the FN1(Fibronectin 1)-ALK fusion mechanism and literature review, this study emphasizes the importance of pathological differentiation, individualized treatment, and dynamic cognitive monitoring, providing insights for precision therapy in rare diseases.
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Sun et al. (2025) studied this question.
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