Association between molecular monitoring and long-term outcomes in chronic myelogenous leukemia patients treated with first line imatinib.

OBJECTIVE: Molecular monitoring using quantitative polymerase chain reaction (qPCR) of BCR-ABL mRNA transcripts using the international scale (IS) is recommended by the National Comprehensive Cancer Network and the European LeukemiaNet for patients with chronic myelogenous leukemia in chronic phase (CML-CP). This study assessed the impact of the frequency of qPCR testing on progression-free survival (PFS). RESEARCH DESIGN AND METHODS: This retrospective chart review of 402 CML-CP patients on first line imatinib therapy, performed by 38 community-based US physicians, analyzed the impact of the frequency of molecular monitoring on the risk of progression and PFS. MAIN OUTCOME MEASURES: Time to progression and progression-free survival. RESULTS: Over the 3 year study, 13.2% of patients did not have any qPCR monitoring and 46.3% had 3-4 qPCR tests per year; 5.7% of CML-CP patients progressed to accelerated/blast phase or died. Compared to patients with no qPCR monitoring, those with 3-4 qPCR tests per year had a lower risk of progression (HR = 0.085; p = 0.001) and longer PFS (HR = 0.088; p = 0.001) after adjusting for potential confounders, as did those patients with 1-2 qPCR tests per year (both p < 0.02). Results were consistent after adjusting for Sokal score when available. CONCLUSION: This is the first study to document the clinical impact of frequent molecular monitoring, and the findings underscore the importance of regular molecular monitoring in delivering quality care for CML. These findings could be subject to unobserved confounders.

A longitudinal analysis of costs associated with change in disease activity in systemic lupus erythematosus.

OBJECTIVES: To estimate the economic consequences of changes in disease activity on healthcare resource utilization (HRU) and costs. METHODS: A retrospective longitudinal study of systemic lupus erythematosus (SLE) patients receiving care in a regional integrated health delivery system in the US from 01/2004 through 03/2011 was conducted using electronic health records, medical chart reviews, and claims. Eligible patients were ≥18 years old, with ≥1 rheumatologist-confirmed SLE diagnosis and ≥1 eligible rheumatology encounter. Patients were continuously enrolled ≥90 days before and ≥30 days after the encounters. Charts were manually reviewed to estimate SLEDAI scores. Average unit costs of each medical procedure, facility use, and prescription were estimated from a payer perspective (2011 USD) using a managed care claims database. HRU and costs were calculated for the 30-day period surrounding every SLEDAI score date (10 days before and 19 after). Relationships between HRU/costs and SLEDAI scores were estimated using mixed-effect models. RESULTS: Overall, 178 SLE patients were included; mean age was 50.6 years, 91% were female, and 95.5% Caucasian. Patients had a total of 1343 encounters with SLEDAI scores over an average period of 1035 days. Reductions of SLEDAI scores were associated with reductions in HRU and costs. SLEDAI score reductions of 4-points were associated with reductions of 10% HRU and 14% costs over a 30-day period; reductions of 8-points had associated reductions of 19% HRU and 26% costs; and reductions of 10-points had associated reductions of 23% HRU and 31% costs. Annualized, changes in SLEDAI scores are associated with changes of $2485 (SLEDAI score change: 10-6), $4624 (10-2), and $5579 (10-0), respectively. CONCLUSION: Reductions in disease activity were associated with substantial reductions of HRU and costs. LIMITATIONS: Only short-term effects of disease activity change were investigated, disregarding other potential benefits of low disease activity on long-term organ damage prevention or comorbidities.