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Background: The landscape of first-line treatment for acute myeloid leukemia (AML) patients ineligible for intensive chemotherapy has changed remarkably after venetoclax approval. Accumulating real-world data further apprises us with more knowledgeable use. To assess the efficacy and safety challenges in the real-life setting of the combination of hypomethylated agent (HMA) and venetoclax, we conducted a multi-center retrospective study. Methods: Forty adult AML patients treated with the combination of HMA and venetoclax as a first-line treatment after full approval (2020) were included. To confirm VIALE-A results, this group was compared to a historical cohort of 17 chemotherapy-ineligible AML patients treated with HMA monotherapy before 2020. Results: The combination of HMA-venetoclax achieved a composite complete response rate of 86.8% (p < 0.001), median overall survival, and event-free survival of 33.8 and 19.7 months, respectively, in a median follow-up of 17.8 months (pos < 0.001, HR = 0.276, CI: 0.132–0.575, pEFS = 0.004, HR = 0.367, CI: 0.174–0.773). High rates of neutropenia (90%) and consequent infection rates (57.5%) were noted. Only 55% of our patients received antifungal prophylaxis, as its use remains controversial, and invasive fungal infections were presented in 7.5%. Conclusions: Evidently, venetoclax-HMA yields high response rates and profound survival benefits in real life and has changed our approach to alternative chemotherapy options. [ABSTRACT FROM AUTHOR]

Multiple myeloma is a plasma cell dyscrasia with an age-standardized incidence of 3 - 4 per 100,000 in the Caucasian population. It is the second most common hematological malignancy after non-Hodgkin lymphoma, representing 1% of all cancers. Herein, we present a case report of multiple myeloma with ocular involvement as a sign of recurrence. A 62-year-old woman, with a known history of lambda light chain multiple myeloma, presented with reduced visual acuity in both eyes while on maintenance chemotherapy. The patient also had mild unsteadiness and fatigue. Fundus examination revealed bilateral optic disc swelling and hemorrhages of the posterior pole. Magnetic resonance imaging disclosed no abnormalities. Although no biopsy of the optic nerve was possible, intracranial pressure was elevated and cerebrospinal fluid was riddled with neoplastic cells, affirming the diagnosis. After 2 months of chemotherapy, visual function and the appearance of the posterior pole returned to normal. In cases of multiple myeloma, mechanisms, such as hyperviscosity syndrome, microvascular impairment and optic nerve and meningeal infiltration on a cellular level may have played a pivotal role in the ocular involvement, which can be the first sign of recurrence., Competing Interests: The authors declare that they do not have any conflict of interest., (Copyright 2024, Pantelidou et al.)