Assessing Overall Survival and Treatment Impact in Progressive Multifocal Leukoencephalopathy
Author(s)
Katharina Buesch, MSc1, Iain A. Kaan, MBA, DrPH2;
1KJM Büsch Consulting GmbH, Zug, Switzerland, 2Aeolian Logic Pte. Ltd, Singapore, Singapore
1KJM Büsch Consulting GmbH, Zug, Switzerland, 2Aeolian Logic Pte. Ltd, Singapore, Singapore
OBJECTIVES: There is no specific treatment against JC polyomavirus (JCV) and progressive multifocal leukoencephalopathy (PML) is usually fatal. The objective of the analysis was to characterize overall survival and the impact of treatment for PML.
METHODS: Two independent researchers extracted patient-level data from case studies identified via a systematic literature review, focusing on individuals diagnosed with PML and the impact of treatment. Information such as patient demographics, survival status, duration from symptom onset to diagnosis, and overall survival post-diagnosis was collected. Kaplan-Meier methods were used to evaluate survival outcomes for treated vs untreated individuals. Outcomes were classified as improved/stable disease (no worsening or improvement), worsened disease (deterioration with survival beyond 3 months post-diagnosis), and ineffective treatment (worsening symptoms and death within 3 months), based on descriptions provided in the original case study reports.
RESULTS: A total of 323 individuals were identified, of which 219 (68%) were treated for their PML at diagnosis (40% female, 58% male, 2% not reported; mean (SD) age at PML diagnosis 52.1 years (15.7)). Median time from symptom onset to diagnosis was similar for treated and untreated individuals (1.44 months vs 1.56 months, respectively). However, median time from diagnosis to death differed significantly: 27.00 months for treated individuals compared to 7.92 months for those not treated. Outcomes were reported for 203 individuals (93% of the treated cohort): 114 (56%) showed improved or stable disease, 40 (20%) experienced worsened disease, and 49 (24%) had ineffective treatment.
CONCLUSIONS: Treated individuals with PML demonstrated longer overall survival compared to those not treated, though outcomes varied, highlighting the need for further research. Prioritising timely intervention is critical to improving outcomes for individuals affected by PML.
METHODS: Two independent researchers extracted patient-level data from case studies identified via a systematic literature review, focusing on individuals diagnosed with PML and the impact of treatment. Information such as patient demographics, survival status, duration from symptom onset to diagnosis, and overall survival post-diagnosis was collected. Kaplan-Meier methods were used to evaluate survival outcomes for treated vs untreated individuals. Outcomes were classified as improved/stable disease (no worsening or improvement), worsened disease (deterioration with survival beyond 3 months post-diagnosis), and ineffective treatment (worsening symptoms and death within 3 months), based on descriptions provided in the original case study reports.
RESULTS: A total of 323 individuals were identified, of which 219 (68%) were treated for their PML at diagnosis (40% female, 58% male, 2% not reported; mean (SD) age at PML diagnosis 52.1 years (15.7)). Median time from symptom onset to diagnosis was similar for treated and untreated individuals (1.44 months vs 1.56 months, respectively). However, median time from diagnosis to death differed significantly: 27.00 months for treated individuals compared to 7.92 months for those not treated. Outcomes were reported for 203 individuals (93% of the treated cohort): 114 (56%) showed improved or stable disease, 40 (20%) experienced worsened disease, and 49 (24%) had ineffective treatment.
CONCLUSIONS: Treated individuals with PML demonstrated longer overall survival compared to those not treated, though outcomes varied, highlighting the need for further research. Prioritising timely intervention is critical to improving outcomes for individuals affected by PML.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO181
Topic
Clinical Outcomes
Topic Subcategory
Clinician Reported Outcomes, Relating Intermediate to Long-term Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases