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Promise and Challenges of Checkpoint Inhibitor Therapy for Progressive Multifocal Leukoencephalopathy in HIV
Sydney Corey1, Bryan R Smith2, Irene C M Cortese3
1Experimental Immunotherapeutics Unit, National Institute of Neurological Disorders and Stroke, National Institutes of Health, 10 Center Drive, 5C103, Bethesda, MD, 20892-1684, USA.
Purpose Of Review:
Progressive multifocal leukoencephalopathy (PML) is a severe opportunistic infection that remains an important cause of morbidity and mortality in people living with HIV (PLWH). Immune checkpoint molecules are negative regulators of the immune response that have been targeted as a strategy to bolster anti-viral immunity in PML, with varied outcomes reported. While initiation and optimization of antiretroviral therapy remains the standard of care in HIV-related PML, the specific opportunities and risks for checkpoint blockade in these cases should be explored.
Recent Findings:
As of April 15, 2022, only 5 of the 53 total published cases of PML treated with checkpoint blockade had underlying HIV infection; four of these had a favorable outcome. The risk of promoting immune reconstitution inflammatory syndrome is a major concern and underscores the importance of patient selection and monitoring. Checkpoint blockade warrants further exploration as a potentially promising option for treatment escalation in HIV-related PML.
Insights
Checkpoint blockade shows promise for treating progressive multifocal leukoencephalopathy (PML) in people living with HIV (PLWH). Further research is needed to explore its potential and risks in HIV-related PML cases.
Area of Science:
- Immunology
- Infectious Diseases
- Neurology
Background:
- Progressive multifocal leukoencephalopathy (PML) is a serious opportunistic infection impacting people living with HIV (PLWH).
- Immune checkpoint molecules regulate immune responses and are explored for enhancing anti-viral immunity in PML.
- Antiretroviral therapy is the standard care for HIV-related PML, but novel treatments are needed.
Purpose of the Study:
- To review the role and outcomes of immune checkpoint blockade in managing HIV-related PML.
- To explore the opportunities and risks associated with using checkpoint blockade in PLWH with PML.
- To assess the potential of checkpoint blockade as an escalation therapy for HIV-associated PML.
Main Methods:
- Systematic review of published literature on PML treated with checkpoint blockade, focusing on cases with underlying HIV infection.
- Analysis of patient outcomes, including favorable responses and adverse events like immune reconstitution inflammatory syndrome (IRIS).
- Evaluation of patient selection criteria and monitoring strategies for checkpoint blockade in HIV-related PML.
Main Results:
- Out of 53 published cases of PML treated with checkpoint blockade, 5 involved patients with HIV.
- Four of the five HIV-positive patients experienced favorable outcomes with checkpoint blockade treatment.
- Immune reconstitution inflammatory syndrome (IRIS) is a significant risk that requires careful patient monitoring.
Conclusions:
- Checkpoint blockade demonstrates potential as a treatment escalation strategy for HIV-related PML.
- Careful patient selection and vigilant monitoring are crucial to mitigate risks such as IRIS.
- Further investigation into checkpoint blockade is warranted to optimize its use in managing PML in PLWH.
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