Background Chronic Graft-Versus-Host Disease (cGVHD) is a complication of hematopoietic cell transplantation (HCT). While the clinical outcomes of cGVHD are well documented, few studies have assessed its treatment practices in the real-world. The objectives of this study are to quantify the prevalence of cGVHD, to examine provider prescribing patterns, and to evaluate the healthcare cost and resource utilization (HCRU) in a real-world US cGVHD population. Methods This study analyzed de-identified claims from the Medicare FFS 5% sample for beneficiaries enrolled from 2013-2016 and Pharmetrics commercial 2013-2018 databases to identify cGVHD in allogenic HCT patients. cGVHD was identified based on ICD-9/10 diagnosis codes for cGVHD or unspecified GVHD with a first diagnosis >180 days post HCT, or subsequent unspecified GVHD diagnosis >12 months post index diagnosis. Chronic GVHD prevalence was estimated by calculating age-adjusted prevalence rates within the Medicare and Pharmetrics sample populations and applying rates to the total US patient subpopulations as determined by CMS and Census data. Prevalence estimates were based on the last complete year of both Medicare FFS and Pharmetrics data (2016). Longitudinal and Line of Therapy (LOT) analyses were based on data from 2013-2018. A new LOT was defined as starting with the addition of systemic therapy to a patient's cGVHD regimen, regardless of prior lines of therapy or prior treatment. Treatments that stopped and restarted within 60 days were considered continuous treatment. Healthcare costs were calculated by adding the inpatient, outpatient, and pharmacy insurer and beneficiary paid amounts for the commercially insured population. Total HCRU was assessed using the number of inpatient and outpatient visits following the initial cGVHD diagnosis. Results In 2016, the projected prevalence of cGVHD in the US based on the Medicare FFS and Pharmetrics commercial databases was 14,017 individual patients. Within 3 years post allogeneic HCT, 42% of patients developed cGVHD; 66% of cGVHD patients had a prior diagnosis of acute GVHD. The majority of cGVHD patients received at least one systemic therapy; 71% and 47% of cGVHD patients progressed to a second and third LOT, respectively (Table 1). Of patients that received a second and third LOT, the average time from diagnosis to the second and third LOT was approximately 7 months and 10 months, respectively. Over 80% of cGVHD patients received systemic corticosteroid therapy for the treatment of cGVHD within 12 months post diagnosis, and 41% of cGVHD patients were receiving a corticosteroid within the 30 days prior to diagnosis. Within the 12 months post cGVHD diagnosis, most patients received a corticosteroid or a corticosteroid combination as a first LOT (57%), which decreased slightly as patients progressed to second and third line of therapy (49% and 48%, respectively). A total of 25 unique therapeutic agents and over 150 combinations were used in second and third LOT. While newer agents, such as ibrutinib and ruxolitinib, are continuing to increase in utilization among cGVHD patients, these therapies are only used among 1% (ibrutinib) and 1-3% (ruxolitinib) of patients through their first three lines of therapy in the patients captured in Pharmetrics commercial database through June 2018. In the 12 months post diagnosis, cGVHD patients had an average of 21.0 GVHD-related inpatient and outpatient visits (2.8 inpatient and 18.2 outpatient visits). In 2016 the average total annual cost per commercially insured cGVHD patient was $291,357. Conclusion A significant proportion of allogenic HCT patients continue to develop cGVHD, and despite advances in the understanding of cGVHD, corticosteroids remain the mainstay of therapy. However, most cGVHD patients are not adequately managed with first line corticosteroids, and many patients are cycling through several therapies, likely in part due to lack of efficacy and toxicity associated with currently available treatments. Real-world utilization of systemic therapies is highly variable, particularly for patients who progress beyond the first LOT, which highlights the need for evidence-based treatment approaches. cGVHD is a highly burdensome complication of allogenic HCT, and safer, more effective treatments are needed as many patients are not currently well managed on available therapies. Disclosures Bachier: Viracyte: Consultancy; Kadmon Corporation, LLC: Consultancy; Sanofi: Speakers Bureau. Aggarwal:Kadmon Corporation, LLC: Employment, Equity Ownership. Hennegan:Kadmon Corporation, LLC: Consultancy. Milgroom:Kadmon Corporation, LLC: Consultancy. Francis:Kadmon Corporation, LLC: Consultancy. Rotta:Jazz: Speakers Bureau; Kadmon Corporation, LLC: Consultancy.
BackgroundEssential tremor (ET) is among the most common movement disorders in adults. While ET is diagnosed and primarily characterized by the presence of tremor, it also can impact cognition, sleep, mood, and motor functioning more broadly. The manifestations of ET can have various consequences, including difficulty with activities of daily living (ADL), embarrassment, and overall decline in health-related quality of life, which have not been fully explored in prior studies.ObjectiveWe performed a systematic literature review to comprehensively characterize the burden experienced by patients with ET from the clinical and humanistic perspectives, focusing on outcomes beyond tremor.MethodsThis systematic literature review followed Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Searches in PubMed, Embase, and Cochrane Library identified original, observational studies of the clinical and humanistic burden in adult patients with ET published in English between 2010 and 2020. Studies assessing epidemiology, treatment patterns, or disease management were excluded. Search results were screened according to pre-determined eligibility criteria. Data from included studies were collected, independently verified, and qualitatively synthesized.ResultsFollowing the screening of 2,303 records and 145 full-text articles, 39 studies were identified. There was significant heterogeneity in study designs, statistical approaches, and patient cohorts across the included studies. Patients with ET in these studies exhibited more severe disabilities and reduced independence compared to healthy individuals, and they often struggled to perform ADL and relied on caregivers for physical and emotional support. Patients also experienced various issues with movement and balance, increased risk of falls, depression, anxiety, poor sleep quality, and psychosocial consequences including embarrassment, apathy, and enfeeblement.ConclusionA systematic literature review of non-tremor manifestations and/or consequences of ET identified far-reaching negative impacts on patients' ability to function independently and revealed accompanying psychosocial effects, including social fear and embarrassment. The reduced function and psychosocial deficits observed in patients with ET result in significant clinical and humanistic burdens, decreasing quality of life. Future studies should evaluate this condition beyond the tremor itself to provide an improved understanding of the multi-dimensional burden of the disease, thereby highlighting the need to diagnose and appropriately manage patients with ET.
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