Exploring the effectiveness of cytarabine in people living with HIV with progressive multifocal leukoencephalopathy: a retrospective cohort study in Chongqing, China.

Background: The incidence of progressive multifocal leukoencephalopathy (PML) in people living with HIV (PLWH) is approximately 2 %-4 % globally, and there is currently no effective therapeutic strategy. Cytarabine has been reported to increase the survival probability of PLWH with PML; however, its effectiveness remains controversial. The present study aimed to explore whether cytarabine improves the outcomes of PLWH with PML.

Methods: We retrospectively collected data from PLWH who were admitted to the hospital and were diagnosed with PML from January 1, 2019, to October 31, 2023, in Chongqing, China, and then stratified the patients into two groups according to whether they did or did not receive cytarabine treatment. The clinical outcomes and mortality rates were assessed.

Results: A total of 41 patients were included in our study. The median age at the time of hospital admission was 44 years [interquartile range (IQR), 36-51]. The patient cohort had a median CD4+ T-cell count of 68.0 (38.5-109.5) cells/mL and a median HIV viral load of 5.05 (2.53, 5.76) log10 copies/ml. 15 patients received intravenous cytarabine and antiretroviral therapy (ART), and 26 patients did not receive cytarabine (24 patients received ART, and 2 patients did not receive ART). In the cytarabine group, 7 patients (46.7 %) died, 4 patients (26.7 %) improved, 2 patients (13.3 %) stabilized, and 2 patients (13.3 %) worsened. In the noncytarabine group, 15 (57.7 %) patients died, 7 patients (26.9 %) improved, 3 patients (11.5 %) stabilized, and 1 patient (3.9 %) worsened; the clinical outcomes did not significantly differ between the two groups (P > 0.05). The overall mortality rate and 30-day, 90-day, 180-day and one-year mortality rates were lower in the cytarabine group than in the noncytarabine group, but the differences were not significant (P > 0.05).

Conclusions: Our results suggest that treatment with cytarabine seems to fail to improve the survival rates of PLWH with PML. Our study is small and cannot definitively rule out cytarabine activity against PML. Future studies in a larger cohort and longer observations are needed.