scholarly journals Estimation of the Economic Burden Associated with Major Surgery Due to Von Willebrand Disease Based on Claims Data from the USA

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4692-4692
Author(s):  
Abiola Oladapo ◽  
Yanyu Wu ◽  
Mei Lu ◽  
Sepehr Farahbakhshian ◽  
Bruce Ewenstein
Keyword(s):  
Health Care ◽  
Economic Burden ◽  
Surgical Procedure ◽  
Von Willebrand Disease ◽  
Major Surgery ◽  
Employment Equity ◽  
Health Care Database ◽  
Equity Ownership ◽  
Von Willebrand ◽  
Anxiety Depression

Background: von Willebrand disease (VWD), a rare, inherited bleeding disorder, is associated with impaired hemostasis resulting from a quantitative or qualitative deficit in von Willebrand factor. Limited information exists on the economic burden associated with major surgeries in this patient population, and real-world data may help determine the impact of these procedures. Aims: To estimate the incremental economic burden associated with major surgeries in patients with VWD compared with patients without VWD who had similar types of surgery. Methods: Accessing data from the Truven US health care database (January 2008 to December 2018), we analyzed data from patients with VWD (based on ≥2 diagnoses from different hospital admissions/physician visits, excluding laboratory and radiology orders) and patients without VWD who had undergone a major surgical procedure. The surgical procedure was defined as a medical claim associated with a major therapeutic operating room procedure (International Classification of Diseases, 9th/10th revision codes) or a major procedure (Current Procedural Terminology code). For patients with VWD, the surgical procedure had to have occurred on or after their first VWD diagnosis. Patients without VWD who had undergone major surgeries were selected from a 1% random sample of the Truven database. Patients from both groups (ie, VWD and non-VWD) were included in the study if they had continuous health care plan enrollment ≥12 months prior to (baseline period) and ≥12 months following (study period) their first major surgery, no diagnosis of acquired coagulation factor deficiency, and not undergone surgery used to reduce bleeding associated with VWD (ie, uterine ablation, nasal ablation, or hysterectomy). Patients with VWD were matched (1:1) with patients without VWD using propensity score matching. Health care resource utilization (HCRU: inpatient [IP] admission, emergency room [ER] visits, and outpatient [OP] visits) and associated costs (pharmacy or medical; adjusted to 2018 US dollars [USD] using the medical component of the Consumer Price Index) were measured over the 12-month study period. Adjusted analyses controlling for age, sex, region, health plan, index year, Charlson Comorbidity Index (CCI), comorbidity profile (anemia, anxiety, depression, fatigue, and obesity), and baseline HCRU were conducted using generalized linear regression models. Results: After propensity score matching, 2972 patients with VWD and 2972 patients without VWD who had ≥1 major surgery were selected for analysis (mean [SD] age, 40.53 [20.56] and 40.94 [20.33] years, respectively; female, 73.3% and 73.6%, respectively). Mean (SD) CCI was 0.66 (1.26) and 0.64 (1.30), respectively; and anemia, anxiety, depression, fatigue, and obesity were present in a similar proportion of each group (7−18% of patients with or without VWD). The most common major surgeries were musculoskeletal or digestive in patients with or without VWD (39.6% and 25.0% vs 37.1% and 23.4%, respectively). Patients with VWD were significantly (P<0.0001) more likely to have an IP admission (odds ratio [OR], 1.71; 95% confidence interval [CI], 1.52−1.92) or ER visit (OR, 1.41; 95% CI, 1.25−1.59) than patients without VWD. They also had significantly (P<0.0001) more frequent IP admissions (incidence rate ratio [IRR], 1.47; 95% CI, 1.35−1.60), ER visits (IRR, 1.44; 95% CI, 1.31−1.59), and OP visits (IRR, 1.16; 95% CI, 1.11−1.21) than patients without VWD. Patients with VWD incurred significantly (P<0.0001) higher total health care costs than patients without VWD ($50,733.89 USD vs $30,154.84 USD). The majority of costs were medical: $41,943.22 USD in patients with VWD and $26,233.83 USD in patients without VWD. Conclusions: In this large retrospective analysis of a US commercial health care database, patients with VWD incurred significantly higher HCRU and associated costs following major surgeries compared with patients without VWD who had similar surgeries. Disclosures Oladapo: Baxalta US Inc., a Takeda company: Employment, Equity Ownership. Wu:Shire US Inc., a Takeda company: Employment, Other: a Takeda stockowner. Lu:Baxalta US Inc., a Takeda company: Employment, Equity Ownership. Farahbakhshian:Shire US Inc., a Takeda company: Employment, Equity Ownership. Ewenstein:Baxalta US Inc., a Takeda company: Employment, Equity Ownership, Other: a Takeda stock owner.

scholarly journals Prevalence and Burden of Major Bleeding Events in Patients with Von Willebrand Disease Based on Claims Data from the USA

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2222-2222
Author(s):  
Mei Lu ◽  
Abiola Oladapo ◽  
Yanyu Wu ◽  
Sepehr Farahbakhshian ◽  
Bruce Ewenstein
Keyword(s):  
Major Bleeding ◽  
Economic Burden ◽  
Von Willebrand Disease ◽  
Red Blood Cell Transfusion ◽  
Bleeding Events ◽  
Real World Data ◽  
Employment Equity ◽  
Equity Ownership ◽  
Major Bleeding Events ◽  
Von Willebrand

Abstract Background: Von Willebrand disease (VWD) is the most common inherited bleeding disorder (clinically symptomatic prevalence rate ~1:10,000). Patients with VWD have impaired hemostasis due to a quantitative or qualitative deficit in von Willebrand Factor (VWF) that alters platelet adhesion and collagen binding and/or decreases FVIII concentrations. Most patients with VWD present with mild-to-moderate mucosal bleeding (epistaxis, menorrhagia), although life-threatening bleeding may also occur, especially in patients with VWD type 3 and some forms of VWD type 2. While bleeds associated with VWD have been well described in the literature, information on the burden associated with major bleeding events (MBE) is limited. Real-world data can be used to help understand the clinical and economic impact of these events. Aims: To estimate the prevalence, healthcare resource utilization (HCRU), and costs associated with MBE among patients with VWD. Methods: Patients with VWD with ≥1 year of continuous enrollment since the eligibility start date were identified from Truven databases (01/2008-12/2016). Patients with MBEs were identified using a medical claim associated with an ICD-9/10 CM diagnosis code for intracranial, GI, or eye bleed; or menorrhagia, epistaxis, and joint bleeds that required red blood cell transfusion in an inpatient (IP) setting or within 7 days of diagnosis in an outpatient (OP) setting. Prevalence was calculated as the proportion of eligible patients with ≥1 MBE during the observation period (from start to the end of continuous eligibility). To evaluate economic burden, patients with ≥1 MBE on or after the first diagnosis of VWD were compared with patients with no MBEs. HCRU and cost in the 12-month continuous enrollment period following the first MBE were compared with those from a similar 12-month period for patients without MBEs. Regression models were used, controlling for demographics, health plan, index year, Charlson Comorbidity Index (CCI), comorbidities, thrombotic events, and HCRU during the 12-month continuously enrolled baseline period. For patients with MBEs, the proportion of patients with comorbidities was compared between the 12-month baseline and study periods using McNemar test. Results: 19,785 VWD patients were identified (mean age 34 years, 75% female) During a median observation of 4 years, 15.1% of patients experienced ≥1 MBE (mean rate: 0.11 ± 0.64 MBE/year). GI bleeding was the most prevalent MBE, occurring in 13.4% of all patients. Although not common, the prevalence of intracranial bleeds (1.1%) was slightly higher in males than females (1.7% vs 0.9%). In the sample to evaluate economic burden, 773 patients with ≥1 MBE (age 44.5 ± 20.1 years) and 4285 patients without MBEs (age 34.2 ± 19.5 years) were selected. Patients with MBEs were significantly (p<0.01) more likely to have an IP admission (OR, 4.1; 95% CI, 3.4-5.0), ER visit (1.8; 1.5-2.1), or OP visit (4.9; 1.8-13.4); they also had significantly longer IP stays (IRR, 3.9; 95% CI, 3.1-4.9) and more frequent IP admissions (3.2; 2.8-3.8), ER visits (2.0; 1.8-2.3), and OP visits (1.3; 1.2-1.3), compared to those without MBEs. Patients with MBEs incurred significantly (p<0.01) higher total healthcare costs (adjusted mean difference, $20,890; 95% CI, $15,524-$29,254) than those without MBEs. Among the 773 patients with MBEs, approximately 1 in 4 patients had a MBE (26.8%) diagnosed in the IP setting. The overall annual mean (± SD) IP length of stay (LOS) was 7.4 ± 19.4 days, with intracranial bleeds associated with the longest mean IP LOS (14.3 ± 19.4 days). The readmission rate was 3.1% for any MBE, and 2.5% for the same type of MBE as the initial bleed. The proportions of patients with anemia (24.2% vs 15.9%; p<0.01) and anxiety (18.6% vs 14.2%; p<0.01) were significantly higher after the MBE than before. Conclusions: In this large retrospective analysis of data from a US commercial healthcare plan, ~15% of patients with VWD experienced MBEs, mostly GI bleeds. While our estimation of some MBEs may be conservative, this is the first study to use a large dataset with sound statistical methods to evaluate the burden associated with MBEs in this population. MBEs were associated with additional comorbidities and high HCRU and costs (driven by inpatient costs), so optimal therapy is essential to prevent MBEs in patients with VWD. Disclosures Lu: Shire: Employment, Equity Ownership. Oladapo:Shire: Employment, Equity Ownership. Wu:Shire: Employment. Farahbakhshian:Shire: Employment, Equity Ownership. Ewenstein:Shire: Employment, Equity Ownership.

The Economic Burden of Short-Term Adverse Events Associated with the CHOP Chemotherapy Regimen in Patients with Lymphoproliferative Disorders in the United States: A Comprehensive Literature Review

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5826-5826
Author(s):  
Crystal Watson ◽  
Arie Barlev ◽  
Jodie Worrall ◽  
Steve Duff ◽  
Rachel Beckerman
Keyword(s):  
Adverse Events ◽  
Resource Utilization ◽  
Economic Burden ◽  
Healthcare Resource ◽  
The United States ◽  
Healthcare Resource Utilization ◽  
Short Term ◽  
Employment Equity ◽  
Comprehensive Literature Review ◽  
Equity Ownership

Objectives: CHOP (vincristine, cyclophosphamide, prednisone, doxorubicin) is a treatment option for post-transplant lymphoproliferative disorder (PTLD) following solid organ transplant, an aggressive and potentially fatal disease. The most common and impactful CHOP-related adverse events (AEs) are febrile neutropenia (FN), chemotherapy-induced (CI) peripheral neuropathy (PN), infection, CI-anemia (A), and CI-nausea and vomiting (NV). These CHOP-related AEs have a large humanistic burdensignificant impact to quality of life (QoL) of patients, especially shortly after treatment. The evidence for a positive QoL benefit associated with some AE treatments (e.g., erythropoietin stimulating agents [ESA], granulocyte colony stimulating factors) is inconsistent and many patients likely remain with QoL deficits even after treatment. The impact of these short-term CHOP-related AEs is likely to be accompanied by an increase in healthcare resource utilization and costs. The objective of this study was to explore the economic burden of short-term CHOP-associated AEs in PTLD patients. Since PTLD is a rare disease with limited available data, we expanded our search to include all patients with lymphoproliferative disorders (LPD). Methods: Short-term (within several months after treatment) AEs associated with CHOP with an incidence of >4% in patients with LPDs were determined and sourced from the published literature and cancer websites. A comprehensive literature search was conducted using PubMed and EMBASE to identify economic burden studies published from 2010 to 2018 of the AEs associated with CHOP and its components in the United States (US). Studies incorporating rituximab alongside CHOP (CHOP + R) were also included as this is a valid treatment option for PTLD patients. Economic burden was defined as the management costs and resource utilization associated with treating CHOP-emergent adverse events. The conduct of this comprehensive literature review was guided by the PRISMA protocol wherein the research question (using the PICOS format), search strategy, target short-term AEs, and inclusion and exclusion criteria were pre-specified in detail. Results: Overall, 3,946 non-duplicate citations were screened, 39 studies were included for abstraction and no studies included patients with PTLD. Studies were methodologically heterogenous, with approximately half (56%) based on some form of retrospective analysis or prospective observational study. FN was the AE most commonly encountered, followed by CIA, infection, CI-nausea and vomiting, and CIPN. FN was an important driver of hospitalization (proportion of FN patients with hospitalization was up to 83.2%) and extended length of stay (LOS) was substantial for several AEs (LOS range in days: infection, 8.4-23.6; FN, 7.9-19.7). Mean LOS was longer in FN patients with multiple hospitalizations as well as in FN patients with comorbidities. Rates of transfusion in CI-anemia patients varied dramatically, from 10.8% to 47.4%. Transfusion rates were attenuated by ESA use in LPD patients, although a significant proportion of anemic cancer patients receiving ESAs still required transfusions. Total management costs were highly variable, ranging from nominal for events such as CIPN to over $197,000 in hospitalization costs per infection discharge per patients complicated with clostridium difficile. One recent study showed the inpatient costs attributable to FN were $33,006 per patient per episode. Studies identified CINV as a top reason for unplanned service use, but no studies were identified assessing its economic impact in LPD patients. Outpatient care costs for each AE varied but tended to have a low to moderate economic impact. The costs attributable to several AEs (FN, infection) were highest in the first cycle of chemotherapy. Conclusions: Several common short-term AEs due to CHOP in the LPD population were associated with substantial healthcare resource utilization and costs that were primarily driven by increased hospitalization and length of inpatient stays. Costs for FN and infections associated with CHOP ranged from $33,000 to over $197,000, demonstrating the high economic burden to the US healthcare system. No PTLD-specific studies were found, highlighting the absence of published data addressing the economic burden associated with chemotherapy in PTLD patients and the need for effective and tolerable therapies. Disclosures Watson: Atara Biotherapeutics: Employment, Equity Ownership. Barlev:Atara Biotherapeutics: Employment, Equity Ownership. Worrall:Atara Biotherapeutics, Inc: Consultancy. Duff:Atara Biotherapeutics, Inc: Consultancy. Beckerman:Atara Biotherapeutics, Inc: Consultancy.

The Economic Burden of Pleural Effusions (PE) In Patients with Chronic Myeloid Leukemia (CML).

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3841-3841
Author(s):  
Eric Qiong Wu ◽  
Annie Guerin ◽  
Vamsi Bollu ◽  
Denise Williams ◽  
Amy Guo ◽  
...  
Keyword(s):  
Resource Utilization ◽  
Medical Cost ◽  
Economic Burden ◽  
Regression Models ◽  
Index Date ◽  
Healthcare Resource ◽  
Healthcare Resource Utilization ◽  
Total Medical Cost ◽  
Employment Equity ◽  
Equity Ownership

Abstract Abstract 3841 Background: Ph+ CML patients may develop PE, as an adverse event of some tyrosine kinase inhibitors (TKI) drug therapy. PE is characterized by an excessive accumulation of fluid in the fluid-filled space that surrounds the lungs. PE requires medical care, may compromise the course of CML treatment, and have economic consequence beyond the costs of treating PE. Aim: To compare healthcare resource utilization and costs between CML patients treated with a TKI who developed PE and their matched PE-free controls. Methods: MarketScan and Ingenix Impact databases (2001-2009) were combined to identify adult CML patients (ICD-9CM code 205.1×) who received ≥1 prescription of imatinib, dasatinib, or nilotinib before the index date and had continuous enrollment ≥6 months prior to and after the index date. The index date was defined as 30 days before the first PE diagnosis (ICD-9CM code 511.9×) for patients with PE and was randomly selected among all the eligible calendar dates (i.e., following a prescription for a TKI and a diagnosis for CML) for the PE-free controls. Patients were followed for 6 months after the index date. PE and PE-free patients were matched on a 1:1 ratio using propensity score matching. PE-related (i.e., medical claims with a PE diagnosis) resource utilization (inpatient [IP], outpatient [OP], emergency room [ER] and other medical visits) and costs were estimated for PE patients. To estimate the overall incremental impact of PE, all-cause and CML-related (i.e., medical services associated with a diagnosis code of 205.1×) resource utilization and costs were compared between PE and PE-free controls. All costs were reported in 2009 US dollars. Incidence rate ratios (IRR) for healthcare resource utilization were estimated by Poisson regression models. Incremental costs were estimated using generalized linear models or two-part models. Multivariate regression models controlled for age, gender, treatment duration with tyrosine kinase inhibitor, other chemotherapy, bone marrow or stem cell transplant, CML complexity, Charlson comorbidity index, adverse events, and comorbidities. Results: The study included 179 matched pairs. On average, patients were 63.4 and 63.8 years old with 41% and 49% of the population being female for PE-free and PE patients, respectively. During the study period, PE patients were estimated to have an average of 0.62 PE-related IP admissions, 8.43 IP days, 0.06 ER admissions, and 1.76 OP visits. Compared to PE-free patients, PE patients had more than 7 times as many IP days (IRR=7.23; p<.01), almost 3 times as many IP admissions (IRR=2.96; p<0.01), almost twice as many OP visits (IRR=1.98; p<.01) and ER visits (IRR=1.77; p<.01). Especially, PE patients had almost 10 times as many CML-related IP days (IRR=9.91; p<.01), more than 3 times as many CML-related IP admissions (IRR=3.95; p<0.01), twice as many CML-related OP visits (IRR=2.16; p<.01), and almost 6 times as many CML-related ER visits (IRR=5.60; p<.01). On average, PE-related medical costs were estimated at $11,015 per patient, where 84.2% was accounted for by IP costs. Total costs for all-cause related medical services were estimated at $37,566 for PE patients and $14,841 for PE-free patients. After adjusting for confounding factors, the incremental total medical cost of PE patients was $22,299 (p<.01), mostly due to the incremental OP cost ($12,931; p<.01) and IP cost ($8,737; p<.01). Similarly, PE patients incurred higher CML-related medical costs compared to PE-free patients, with a $15,859 (p<.01) incremental cost. Conclusion: Compared to PE-free patients, PE patients have a substantial economic burden with higher PE-related costs, CML-related costs, and total medical cost. Disclosures: Wu: Analysis Group, Inc.: Employment. Guerin:Analysis Group, Inc.: Employment. Bollu:Novartis: Employment, Equity Ownership. Williams:Novartis: Employment, Equity Ownership. Guo:Novartis Pharmaceuticals Corporation: Employment, Equity Ownership. Ponce de Leon Barido:Analysis Group, Inc.: Employment. Yu:Analysis Group, Inc.: Employment.

Clinical and Economic Burden During Hospitalizations Among Cancer Patients with Febrile Neutropenia: Evidence From U.S. Hospitals, 2007–2010

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 239-239
Author(s):  
Brian Dulisse ◽  
Xiaoyan Li ◽  
Julie A. Gayle ◽  
Richard L. Barron ◽  
Frank R. Ernst ◽  
...  
Keyword(s):  
Cancer Patients ◽  
Hodgkin Lymphoma ◽  
Economic Burden ◽  
Case Fatality ◽  
Tumor Type ◽  
Index Hospitalization ◽  
Employment Equity ◽  
Equity Ownership ◽  
Infection Types ◽  
Tumor Types

Abstract Abstract 239 Background: Febrile neutropenia (FN) is a serious complication of myelosuppressive chemotherapy that often requires hospitalization. Published burden-of-illness estimates for FN-related hospitalizations were either based on clinical practice more than a decade ago (Caggiano et al Cancer 2005, Kuderer et al Cancer 2006) or derived from small samples (Schilling et al Exp Ther Med 2011). Methods: A retrospective cohort study was conducted to provide updated estimates using 2007–2010 hospital discharge data from a database maintained by Premier and containing service records of over 400 geographically diverse hospitals. It is one of the largest hospital databases in the U.S. The study population included adult patients with 1 of 6 tumor types (breast, lung, colorectal, ovarian cancers; non-Hodgkin lymphoma [NHL]; and Hodgkin lymphoma), discharge diagnoses of neutropenia (ICD-9 code 288.0x) with fever or infection, and receipt of intravenous antibiotics. The average hospitalization cost, case fatality rate, and average length of stay (LOS) associated with each patient's first FN-related hospitalization (index hospitalization) were computed with associated 95% confidence intervals (CIs) for all tumor types combined and stratified by tumor type. Detailed costs and resource utilization components within index hospitalizations were also examined and tallied. Tumor-type-specific multivariate linear regressions (for costs and LOS) and logistic regressions (for mortality) were conducted to assess the effect of infection types and comorbidities on study outcomes, adjusting for other patient and hospital characteristics. FN-related 30-day readmission rates after index hospitalizations were also estimated. All cost measures reflected actual direct costs to hospitals and were adjusted to 2010 dollars. Results: Hospitalization with FN was identified in 16,273 cancer patients. The mean (SD) age was 63 (14) years; 49% were aged ≥65 years; and 60% were female. Hospitalization costs and clinical outcomes of index hospitalizations varied by tumor type and by discharge status (Table). For all tumor types combined, 19% of patients were treated in an intensive care unit (ICU) setting during index hospitalizations, with average LOS of 5.2 days spent in ICU. The estimated models identified certain infection types and comorbidities as potential risk factors for inpatient mortality and predictors of higher economic burden. Of note, breast cancer patients with diagnosed septicemia/bacteremia (N=656) had average costs that were $5,664 (95% CI: $4,233–$7,095) higher than those with other infections (N=2,623), average LOS that was 1.7 days (95% CI: 1.0–2.3) longer, and a higher case fatality rate (risk ratio [as approximated by odds ratio]: 4.12, 95% CI: 2.6–6.5), after adjusting for other observed potential confounders. Higher average costs were also observed in NHL patients with diagnosed renal disease (N=1,263) than in those without renal disease (N=4,174) (adjusted difference: $10,408, 95% CI: $8,391–$12,425). The FN-related 30-day readmission rate after index hospitalization was 5.9% for all tumor types combined. The rate was 9.9% for NHL and 8.6% for Hodgkin lymphoma, higher than that in patients with other tumor types (2.3%–4.1%). Conclusions: FN-related hospitalizations among cancer patients are expensive, resource-intensive, and associated with considerable mortality risk. Substantial differences in the clinical and economic burden of FN exist depending on tumor types, infection types, and comorbidities. Disclosures: Dulisse: Premier healthcare alliance: Employment. Li:Amgen Inc.: Employment, Equity Ownership. Gayle:Premier healthcare alliance: Employment. Barron:Amgen Inc.: Employment, Equity Ownership. Ernst:Premier healthcare alliance, which contracted with Amgen to conduct this study.: Employment. Rothman:Dr. Rothman is an employee of RTI Health Solutions, an independent non-profit research organization that does work for government agencies and pharmaceutical companies.: Employment. Legg:Amgen Inc.: Employment, Equity Ownership. Kaye:RTI Health Solutions (a business unit of RTI International): Employment.

Safety and Effectiveness of Desmopressin for the Management of Delivery and Major Surgery in Patients with Mild-Moderate Von Willebrand Disease: Final Analysis of the Prodeswil Study

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 759-759
Author(s):  
Augusto Bramante Federici ◽  
Giancarlo Castaman ◽  
Alfonso Iorio ◽  
Emily Oliovecchio ◽  
Prodeswil Investigators
Keyword(s):  
Side Effects ◽  
Clinical Efficacy ◽  
Oral Surgery ◽  
Biological Response ◽  
Von Willebrand Disease ◽  
Major Surgery ◽  
Efficacy And Safety ◽  
Inclusion Criteria ◽  
Bleeding Episodes ◽  
Von Willebrand

Abstract Introduction. Despite the fact that desmopressin (DDAVP) is considered the treatment of choice in the majority of patients with inherited von Willebrand disease (VWD), there are still open questions about the efficacy and safety of the use of DDAVP to manage recurrent bleeding episodes, delivery and major surgery. In fact, to avoid tachyphylaxis and possible side effects with repeated DDAVP injections, VWF concentrates are usually preferred to manage delivery and major surgery also in VWD patients proven to be DDAVP-responsive. No prospective data have been reported so far to correlate biological response with DDAVP clinical efficacy in VWD and guide clinical practice. Design, aims and methods. ProDesWilis an investigator-driven Pro spective study on D esmopressin e fficacy and s afety of patients with inherited von Wil lebrand disease assessed at enrolment for DDAVP biological response during a test-infusion. Inclusion criteria: VWD diagnosis without any age restriction according to bleeding score >4, VWF activity levels <45U/dL, and at least another affected member in the family. DDAVP Biological Response: VWD were classified as complete, partial or no-responders as previously reported (Blood 2008;111:3531). Treatment regimens: DDAVP given intravenously or subcutaneously (0.3 ug/Kg) or by nasal spray (4 ug/Kg) according to preparations available in different countries with or without standard doses of anti-fibrinolytic agents (Epsilon-amino-caproic acid, EACA or tranexamic acid, TA). In case of major surgery, DDAVP was used together with TA using a 3dayOn-4dayOff-3dayOn schedule. DDAVP efficacy-safety evaluated with criteria currently used for VWF concentrates, with poor efficacy defined when VWF concentrates were needed. Patients were prospectively followed up for 24 months by ProDesWil Investigators who reported detailed information about DDAVP therapy used for bleeds, deliveries, oral and minor/major surgeries Results. 268 patients were enrolled in this 24-month prospective study. DDAVP-biological response: 225/268 (85%) patients met inclusion criteria as VWD1(n=184), VWD1C (n=14), VWD2A(n=15), VWD2M(n=12). The 14 VWD1C with accelerated clearance carried C1130F and R1205H mutations. DDAVP biological response was complete, partial and absent in 89%, 10% and 1% of all VWD. DDAVP clinical efficacy and safety: during the 24-month follow-up 84/225 (37.3%) received DDAVP for bleeding episodes (n=104), oral surgeries (n=33), deliveries (n=12), other minor/major surgeries (n=25). Total DDAVP injections were 652 with median, range/episode during bleeds (2,1-12), oral surgeries (1,1-10), deliveries (3,1-13), minor/major surgeries (3.6,1-16). Clinical efficacy was excellent/good in bleeds (93.3%), oral surgery (100%), deliveries (91.7%), minor/major surgeries (92.3%). All VWD treated for oral surgery with (75%) and without (25%) EACA/TA had excelled/good outcomes. Efficacy was rated excellent/good in 96/104 bleeds, with 8 episodes rated poor during menorrhagia (n=4) in 2 VWD1 and 2 VWD2A, during nose (n=2) and gastrointestinal (n=2) bleeds in 3 VWD2A and 1 VWD1C. 11/12 deliveries had excellent/good outcome with poor response in only one VWD1C who required VWF concentrates after caesarean section. All the 11/25 cases with minor surgeries had excellent outcomes. Among the 14/25 major surgeries [abdominal (n=5), hysterectomy (n=3), tonsillectomy (n=3), orthopaedic and others (n=3)] 2 episodes (partial resection of kidney in VWD2A and tonsillectomy in VWD1) were rated poor. Side effects (16 cases) were mainly minor (flushing, headache, tachycardia) with water retention reported only in 2 patients who received >12 doses of DDAVP for delivery or major surgery. Conclusions: DDAVP is an effective, safe and cheap treatment for managing patients with mild-moderate VWD who showed complete biological response to this drug. Therefore, DDAVP must be always recommended as first line therapy in responsive VWD not only in bleeds and oral surgery but also in deliveries and major surgeries. On the other hands, DDAVP should be used with caution in VWD2A and VWD1 with partial response. The additional use of EACA/TA should be considered especially when DDAVP is required for more than 4 days. Disclosures No relevant conflicts of interest to declare.

Antithrombotic prophylaxis in patients with von Willebrand disease undergoing major surgery: when is it necessary?

2008 ◽  
Vol 28 (2) ◽  
pp. 215-219 ◽  
Author(s):  
Massimo Franchini ◽  
Giovanni Targher ◽  
Martina Montagnana ◽  
Giuseppe Lippi
Keyword(s):  
Von Willebrand Disease ◽  
Major Surgery ◽  
Antithrombotic Prophylaxis ◽  
Von Willebrand

Health Care Utilization and Associated Costs in Elderly Persons with Non-CML Myeloproliferative Neoplasms: Real-World Evidence From a United States Medicare Population

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4273-4273 ◽  
Author(s):  
Sudeep Karve ◽  
Gregory L Price ◽  
Keith L Davis ◽  
Gerhardt M Pohl ◽  
Richard A Walgren
Keyword(s):  
Health Care ◽  
Health Care Utilization ◽  
Index Date ◽  
Myeloproliferative Neoplasms ◽  
Care Utilization ◽  
Cell Transplant ◽  
Elderly Persons ◽  
Employment Equity ◽  
Equity Ownership

Abstract Abstract 4273 Background: Non-CML myeloproliferative neoplasms (MPNs), which include essential thrombocythemia (ET), polycythemia vera (PV), myelofibrosis (MF) and MPN not otherwise specified (MPN-NOS), are characterized by activation of JAK2 signaling and abnormal blood cell production. Median survival ranges from months to years for MF and up to a decade or more for PV and ET. Some symptomatic treatment options exist, but with the exception of hematopoietic stem cell transplant, none are curative. Although MPN incidence is highest in persons aged ≥65 years, little is known about overall health care utilization and costs in elderly persons with these diseases. MPNs are more prevalent in the elderly and therefore Medicare enrollees are a highly relevant source for US-based resource utilization and cost data for these diseases. Objective: To compare all-cause health care utilization and costs from four subtypes of elderly MPN patients (ET, PV, MF and MPN-NOS) with matched non-MPN/non-cancer controls. Methods: Retrospective data were taken from the Survey, Epidemiology, and End Results (SEER)-Medicare linked database in the US, which combines clinical information from the SEER cancer registry (MPN reporting has been required since 2001) with medical and pharmacy claims for Medicare enrollees. Patients with a new MPN diagnosis between Jan 1, 2001 and Dec 31, 2007 were selected and evaluated for all-cause health care utilization and costs from Jan 1, 2008 (index date) through Dec 31, 2008 (follow-up end date). Patients were classified by MPN subtype based on the most recent diagnosis information (ICD-O-3 from the SEER registry or ICD-9-CM from Medicare claims) before the index date. Patients who died before follow-up end, had HMO or discontinuous Medicare enrollment during the follow-up year, had enrollment based on end stage renal disease, or a diagnosis of a non-MPN malignancy before follow-up end were excluded from the study. Separate non-MPN/non-cancer control groups were selected for each MPN subtype and matched (5:1) on birth year, gender, ethnicity, geography, and reason for Medicare eligibility. Per patient health care utilization and costs during the follow-up year were aggregated and stratified by care setting. Costs were adjusted to 2010 US$ and represent amounts reimbursed by Medicare to providers. Costs were compared between MPN cases and controls using univariate t-tests. Results: A total of 1,355 MPN patients (n = 445 ET, 684 PV, 81 MF, 145 MPN-NOS) were identified for study inclusion and assigned matching controls. For ET, PV, MF and MPN-NOS cases, respectively, mean [SD] age at index was 75.5 [9.7], 70.8 [11.3], 70.8 [10.4] and 74.1 [8.9] years and % female was 69.0, 43.9, 54.3, and 55.2. Mean [SD] years between first MPN diagnosis and study index date was 3.1 [2.0], 3.4[1.9], 2.7 [2.0], and 3.1 [2.1] for ET, PV, MF and MPN-NOS cases, respectively. A significantly (p<0.05) higher proportion of MPN cases, regardless of subtype, had ≥1 hospitalization during follow-up vs. controls (ET vs. control: 22% vs. 16%, PV vs. control: 27% vs. 15%, MF vs. control: 31% vs. 12%, MPN-NOS vs. control: 36% vs. 17%). Mean [SD] total days of hospital care were similarly higher in MPN cases (ET vs. control: 2.7 [12.8] vs. 1.6 [6.6], PV vs. control: 2.6 [7.0] vs. 1.7 [9.5], MF vs. control: 2.5 [6.2] vs. 1.2 [5.9], MPN-NOS vs. control: 4.0 [10.0] vs. 2.1 [13.7]), although the PV vs. control difference was not statistically significant. The ER visit rate during follow-up was also significantly (p<0.05) higher in MPN cases (ET vs. control: 34% vs. 24%, PV vs. control: 38% vs. 25%, MF vs. control: 46% vs. 21%, MPN-NOS vs. control: 44% vs. 29%). All-cause costs for MPN cases vs. matched controls are presented in the figure. Mean total costs per patient, driven equally by inpatient and outpatient services, were significantly (p<0.001) higher in MPN cases (ET vs. control: $11,259 vs. $8,897, PV vs. control: $13,337 vs. $8,530, MF vs. control: $20,917 vs. $7,367, MPN-NOS vs. control: $20,174 vs. $9,800). Conclusions: Total health care costs during a given year for elderly patients with MPNs are 1.3 to 3 times higher (depending on subtype) than those of matched controls. These findings may help inform future cost effectiveness evaluations of novel MPN treatments, as well as decision making in the provision of optimal MPN care within a Medicare system in which resources are finite and must be allocated ethically and efficiently. Disclosures: Karve: RTI Health Solutions: Consultancy, Research Funding. Price:Eli Lilly and Company: Employment, Equity Ownership. Davis:Eli Lilly, Merck, GlaxoSmithKline, Bristol-Myers Squibb, Pfizer, Eisai, Sanof-Aventis, Gilead Sciences, MedImmune: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Pohl:Eli Lilly and Company: Employment, Equity Ownership. Walgren:Eli Lilly and Company: Employment, Equity Ownership.

Differences In Treatment Patterns and Costs Among Diffuse Large B-Cell Lymphoma Patients Treated In The Clinic Vs. The Hospital Outpatient Setting

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 1751-1751 ◽  
Author(s):  
Carolina Reyes ◽  
Stacey Dacosta Byfield ◽  
Laura K. Becker ◽  
Art Small
Keyword(s):  
Health Care ◽  
B Cell ◽  
Cell Lymphoma ◽  
B Cell Lymphoma ◽  
Treatment Patterns ◽  
Employment Equity ◽  
Large B Cell Lymphoma ◽  
Equity Ownership ◽  
Large B Cell

Abstract Background Diffuse large B-cell lymphoma (DLBCL) is the most common subtype of Non-Hodgkin's Lymphoma (NHL) accounting for approximately 30% of newly diagnosed casesi. DLBCL is an aggressive form of NHL and without treatment, median survival estimates are <1 year.ii Rituximab in combination with CHOP (cyclophosphamide, doxorubicin, vincristine and prednisone) is recommended first-line therapy for DLBCL patients and has been shown to improve overall survival compared with CHOP alone (previous standard therapy).iii In addition, published evidence suggests that receipt of granulocyte-colony stimulating factor (G-CSF) may improve outcomes among patients who initiate CHOP-based therapy.iv It is unclear whether differences in treatment and outcomes exist among cancer patients by site where care is delivered. This study examines differences in treatment patterns, health care resource use and costs among DLBCL patients receiving rituximab (R) or R+ chemotherapy in the office/clinic (OC) setting vs. the hospital outpatient (HOSP) setting. Methods This retrospective study used medical and pharmacy claims (1/2007 - 7/2012) from a national US commercial health plan to identify patients at least18 years old with ≥2claims for R. Patients were required to have evidence of DLBCL (≥1 claim with ICD-9-CM 200.78 or ≥2 claims with unique diagnosis codes from ICD-9-CM 200.70 to 200.77) and be enrolled in the health plan for ≥6 months before and after the index date (date of the first R claim). The follow-up period, that is, the episode of care (EOC), was the date of the first R infusion through 30 days after the last infusion prior to a gap in R administration of at least 7 months; those with less than 6 months of follow-up due to death were included. Patients with multiple cancers or receipt of R at both the OC and HOSP setting during the EOC were excluded. Differences in number of infusions, receipt G-CSF, healthcare utilization and per-patient per-month (PPPM) health care costs by cohort were examined. Results A total of 491 patients were identified, 65% OC (n=320) and 35% HOSP (n=171): by insurance type, 140 Medicare Advantage patients, 39% HOSP and 351 commercially insured patients, 33% HOSP. From 2007 to 2011/2012, the percentage of patients in HOSP increased from 32% to 43%. Descriptive results are shown in the Table. The cohorts had similar mean age, baseline Charlson comorbidity index scores and similar EOC lengths. However, compared to the OC cohort, the HOSP cohort had fewer infusions during the EOC and fewer infusions per month. In addition, fewer HOSP patients had evidence of combination therapy and receipt of any G-CSF during the EOC. HOSP patients also had significantly higher rates of emergency room visits, but not hospitalizations compared to OC patients. Total PPPM costs during the EOC as well as average costs of anti-cancer systemic therapy drugs plus administration costs incurred on days of rituximab infusions were significantly higher among the HOSP cohort compared to the OC cohort. Conclusions Increasing proportions of DLBCL patients receive infusions in the HOSP setting. HOSP patients had fewer infusions per month and incurred greater costs on the day of infusion compared to the OC cohort. There were fewer patients in HOSP with evidence of G-CSF during the EOC compared to OC patients. Overall, total PPPM costs were higher among the HOSP cohort compared to the OC cohort. Future research is warranted to assess the impact of these differences on clinical outcomes by site of care. [i] Armitage et al. JCO 1998;16(8):2780-95 [ii] Mey et al. Swiss Med Wkly 2012;140:w13511 [iii] NCCN Guidelines Version 1.2013 Diffuse Large B-cell Lymphoma [iv] Donnelly, et al, Leuk Lymphoma. 2000;39(1-2):67-75 Disclosures: Reyes: Genentech, inc: Employment, Equity Ownership. Dacosta Byfield:Genentech, Inc: Genentech contracted with OptumInsight to conducting the work described in the abstract. Stacey is employed at Optum but did not receive funds directly from Genentech and employment is not contingent on work with Genentech., Genentech contracted with OptumInsight to conducting the work described in the abstract. Stacey is employed at Optum but did not receive funds directly from Genentech and employment is not contingent on work with Genentech. Other; OptumInsight: Employment. Becker:Genentech, Inc: Genentech contracted with OptumInsight to conducting the work described in the abstract. Laura is employed at Optum but did not receive funds directly from Genentech and employment is not contingent on work with Genentech., Genentech contracted with OptumInsight to conducting the work described in the abstract. Laura is employed at Optum but did not receive funds directly from Genentech and employment is not contingent on work with Genentech. Other; OptumInsight: Employment. Small:Genentech, Inc: Employment, Equity Ownership.

Efficacy and safety of a new generation von Willebrand factor/factor VIII concentrate (Wilate®) in the management of perioperative haemostasis in von Willebrand disease patients undergoing surgery

2011 ◽  
Vol 105 (06) ◽  
pp. 1072-1079 ◽  
Author(s):  
Mario von Depka-Prondzinski ◽  
Jerzy Windyga ◽  
Keyword(s):  
Factor Viii ◽  
Von Willebrand Factor ◽  
Perioperative Management ◽  
Von Willebrand Disease ◽  
Major Surgery ◽  
Open Label ◽  
New Generation ◽  
Type 3 ◽  
Von Willebrand ◽  
Willebrand Factor

SummaryThe aim of this study was to assess the efficacy of Wilate®, a new generation, plasma-derived, high-purity, double virus-inactivated von Willebrand factor (VWF) and factor VIII (FVIII) concentrate (ratio close to physiological 1:1) in the perioperative management of haemostasis in von Willebrand disease (VWD). Data for VWD patients who received Wilate® for perioperative management were obtained from four European, prospective, open-label, non-controlled, non-randomised, multicentre phase II or III clinical trials. A total of 57 surgical procedures were performed (major: n = 27; minor n = 30) in 32 patients. The majority of patients (n = 19, 59.4%) had type 3 VWD, 9 (28.1%) had type 2 VWD and four (12.5%) had type 1 VWD. During major surgery, median daily FVIII dose and mean number of infusions were 25 IU•kg-1 FVIII (VWF:RCô23 IU•kg-1) and 11.0, respectively. Corresponding values for minor surgery were 35 IU•kg-1 (VWF:RCo ~32 IU•kg-1) and 1.5. The efficacy of Wilate® was rated by the investigator as excellent or good in 51 of 53 (96%) procedures. Tolerability was rated as very good or good in 100% of major surgeries (27 of 27) and minor surgeries (29 of 29). Wilate® is an effective and well-tolerated VWF/FVIII replacement therapy in the perioperative management of haemostasis in patients with VWD. It can be administered at a similar FVIII dose, but at a lower VWF dose, as compared to older generation products. Clinical benefits were shown in a population with a high proportion of type 3 VWD patients.

Sign in / Sign up

Export Citation Format

Share Document