Development of a novel shared decision making aid for primary immunodeficiency diseases.

Aim: To describe development of a shared decision making (SDM) aid in treating primary immunodeficiency diseases (PID) with immunoglobulin replacement therapy (IGRT). Materials & methods: Expert engagement and qualitative formative research informed development. IGRT administration features were prioritized using object-case best-worst scaling (BWS) methodology. The aid was assessed by US adults self-reporting PID and revised following interviews/mock treatment-choice discussions with immunologists. Results: Patients participating in interviews (n = 19) and mock treatment-choice discussions (n = 5) deemed the aid useful/accessible and supported the utility of BWS, with content and BWS exercises refined following participant feedback. Conclusion: Formative research led to an improved SDM aid/BWS exercise, and illustrated how the aid may improve treatment decision making. The aid may help less-experienced patients and facilitate efficient SDM.

Shared decision making and developing a decision aid Shared decision making happens when patients and doctors work together to choose treatment options based on a patient's concerns, preferences, goals and values, as well as medical information. The aim of this project was to develop a decision aid to help patients with primary immunodeficiency diseases (PID), in which part of the body's immune system is missing or doesn't function correctly. This will allow patients to better understand and communicate with the healthcare team on their preferences about immunoglobulin treatments, which fight infection by boosting antibody (protein) levels in the blood. The authors talked to experts and reviewed existing information to decide what treatment features the aid should consider. Patients with PID then tested the aid, and changes were made based on their feedback. Doctors specializing in treating PID also provided their feedback. The final aid was judged to be helpful and easy to use by the participants. With further research, this aid could be used to help inexperienced patients better understand what immunoglobulin treatment features are most important to them, and support shared decision-making between patients and their doctors.

Von Willebrand Disease Epidemiology, Burden of Illness and Management: A Systematic Review.

Introduction: Although hereditary von Willebrand disease (VWD) is the most common bleeding disorder, its epidemiology is not well understood. A systematic review (PROSPERO CRD42020197674/CRD42021244374) on the epidemiology/burden of illness of VWD was conducted to better understand patients' unmet needs. Methods: Observational studies (published January 1, 2010 to April 14, 2021) were identified in MEDLINE and Embase databases, using free-text keywords and thesaurus terms for VWD and outcomes of interest. Pragmatic web-based searches of the gray literature, including conference abstracts, were performed, and reference lists of retained publications were manually searched for additional sources. Case reports and clinical trials (phase 1-3) were excluded. Outcomes of interest were incidence, prevalence, mortality, patient characteristics, burden of illness, and therapeutic management/treatments currently used for VWD. Results: Of the 3095 identified sources, 168 were included in this systematic review. Reported VWD prevalence (22 sources) ranged from 108.9 to 2200 per 100,000 in population-based studies and from 0.3 to 16.5 per 100,000 in referral-based studies. Reported times between first symptom onset and diagnosis (two sources; mean 669 days; median 3 years) highlighted gaps in timely VWD diagnosis. Bleeding events reported in 72-94% of the patients with VWD (all types; 27 sources) were mostly mucocutaneous including epistaxis, menorrhagia, and oral/gum bleeding. Poorer health-related quality of life (three sources) and greater health care resource utilization (three sources) were reported for patients with VWD than in general populations. Conclusion: Available data suggest that patients with VWD experience high disease burden in terms of bleeding, poor quality of life, and health care resource utilization.

Impact of von Willebrand Disease on Women's Health Outcomes: A Matched Cohort Database Study.

Objective: To understand the impact of von Willebrand disease (VWD) on women's health, a retrospective cohort study was conducted using UK Clinical Practice Research Datalink (CPRD) GOLD database and Hospital Episode Statistics (HES) Admitted Patient Care data from 1988 to 2016. Materials and Methods: Hysterectomy and heavy menstrual bleeding (HMB) events were identified by recorded disease/clinical codes and compared in women with and without VWD (matched 1:10 by birth and CPRD record start years [±2 years], and general practice attended). Incidence rates and incidence rate ratios (IRR) were calculated; risks were estimated using the Kaplan-Meier method. Results: HMB was recorded after cohort entry in 388 of 1,335 women (29.1%) with VWD and 1,524 of 12,463 women (12.2%) without VWD. The cumulative incidence of HMB was higher in women with versus without VWD across all ages (p < 0.0001), and irrespective of prior HMB status (p < 0.001). Women with VWD were more likely to have HMB compared with women without VWD; IRR adjusted for age and prior HMB status was 2.74 (95% confidence interval [CI]: 2.44-3.07). Hysterectomy was recorded in 88 of 1,374 women (6.4%) with VWD and 320 of 12,791 women (2.5%) without VWD. The cumulative incidence of hysterectomy was higher for women with versus without VWD (p < 0.0001), and highest among women aged ≥30 years at cohort entry. Women with VWD aged 30 - 39 years were more likely to undergo hysterectomy than women without VWD; IRR adjusted for prior HMB was 3.58 (95% CI: 2.36 - 5.44). Conclusions: These findings highlight the substantial impact of VWD on women's health.

Recent advances in therapeutic options for rare hemostatic disorders: selected poster extracts of recent research in hemophilia A, congenital hemophilia with inhibitors, von Willebrand disease, and thrombotic thrombocytopenic purpura presented at the 29th congress of the International Society on Thrombosis and Haemostasis (ISTH 2021, Jul 17-21; virtual congress).

Hemophilia, von Willebrand disease (VWD), and thrombotic thrombocytopenic purpura (TTP) are rare diseases affecting normal hemostasis. Although they differ in their pathogenesis and clinical manifestation, if left undiagnosed and untreated, all these conditions can result in severe long-term consequences and can be potentially life-threatening. This article summarizes a poster series funded by Takeda and presented virtually at the 29th annual congress of the International Society on Thrombosis and Haemostasis (ISTH) in 2021: Data from real-world evidence highlight the importance of joint health and personalized prophylaxis to prevent bleeding for patients with hemophilia, the need to further raise disease awareness in support of timely diagnosis and access to treatment in general practice settings for patients with VWD, and describe the clinical burden for patients with TTP and the importance to advance treatment options for these patients.

Infusion parameters of 20% subcutaneous immunoglobulin for primary immunodeficiency diseases among patient support program participants.

BACKGROUND: The safety and efficacy of subcutaneous immune globulin 20% (human) solution (Cuvitru; Ig20Gly) for primary immunodeficiency disease (PID) have been demonstrated in 2 pivotal trials. OBJECTIVE: To describe patient characteristics and infusion parameters of patients with PID initiating Ig20Gly outside of a clinical trial setting. METHODS: This retrospective, observational study analyzed records of patients participating in the HelloCuvitru program, a patient support program in the United States providing Ig20Gly free of charge for the first 4 infusions to patients aged 2 years or older who had PID and no previous experience of Ig20Gly. Data were collected retrospectively from patient records and during nurse visits. RESULTS: A total of 817 patients (88% of 931 enrolled) completed 4 infusions. At the fourth Ig20Gly infusion, the median (interquartile range) dose was 0.55 (0.46-0.69) g/kg/mo, infusion rate per site was 40 (30.0-50.0) mL, and infusion rate per site was 47 (42.5-53.3) mL/h/site. By the fourth infusion, most patients (58%) received Ig20Gly at 2 infusion sites every 7 (30%) or 14 (25%) days. Median prescribed Ig20Gly dose per month was similar across age groups; median infusion volume per site increased with age. Most patients younger than 18 years received infusions every 14 days; patients aged 18 years or older were more likely to receive infusions weekly. Infusion parameters were similar regardless of whether patients received previous immunoglobulin subcutaneously or intravenously. CONCLUSION: In this large, real-world population of patients with PID, most Ig20Gly infusions were administered for less than 1 hour and required fewer than 2 infusion sites, consistent with the pivotal trials. Infusion rate per site was similar regardless of age, previous immunoglobulin treatment, or infusion frequency.

A systematic literature review of the effects of immunoglobulin replacement therapy on the burden of secondary immunodeficiency diseases associated with hematological malignancies and stem cell transplants.

INTRODUCTION: Secondary immunodeficiency diseases (SID) caused by hematological malignancies (HMs), stem cell transplant (SCT), and associated therapies are mainly characterized by the presence of hypogammaglobulinemia or antibody production deficits. AREAS COVERED: The authors summarized the scientific literature on disease burden of SIDs in patients who had HMs or SCT. Systematic searches were conducted to identify English-language articles from 1994-2020, reporting on clinical, humanistic, and economic burdens of SID due to HMs or SCT. Definitions of SID and serum immunoglobulin G thresholds varied across 24 eligible studies. In most (n = 16) studies, patients received immunoglobulin replacement therapy (IGRT). Several studies found IGRT was associated with significant reductions in rates of infection and antimicrobial use. However, 1 study found no statistically significant difference in antibiotic use with IGRT. Only 3 studies reported on quality of life, and no economic studies were identified. EXPERT OPINION: Overall, the findings show several beneficial effects of IGRT on clinical outcomes and quality of life; however, disparate definitions, infrequent reporting of statistical significance, and scarcity of clinical trial data after the 1990s present areas for further investigation. This paucity indicates an unmet need of current evidence to assess the benefits of IGRT in SID.

Time trends in utilization of G-CSF prophylaxis and risk of febrile neutropenia in a Medicare population receiving adjuvant chemotherapy for early-stage breast cancer.

PURPOSE: The purpose of this study is to assess temporal trends in the use of granulocyte colony-stimulating factor (G-CSF) prophylaxis and risk of febrile neutropenia (FN) among older women receiving adjuvant chemotherapy for early-stage breast cancer. METHODS: Women aged ≥ 66 years with diagnosis of early-stage breast cancer who initiated selected adjuvant chemotherapy regimens were identified using the SEER-Medicare data from 2002 to 2012. Adjusted, calendar-year-specific proportions were estimated for use of G-CSF primary prophylaxis (PP) and secondary prophylaxis and FN risk in the first and the second/subsequent cycles during the first course of chemotherapy, using logistic regression models. calendar-year-specific mean probabilities were estimated with covariates set to modal values. RESULTS: Among 11,107 eligible patients (mean age 71.7 years), 74% received G-CSF in the first course of chemotherapy. Of all patients, 5819 (52%) received G-CSF PP, and among those not receiving G-CSF PP, only 5% received G-CSF secondary prophylaxis. The adjusted proportion using G-CSF PP increased from 6% in 2002 to 71% in 2012. During the same period, the adjusted risk of FN in the first cycle increased from 2% to 3%; the adjusted risk increased from 1.5% to 2.9% among those receiving G-CSF PP and from 2.3% to 3.5% among those not receiving G-CSF PP. CONCLUSION: The use of G-CSF PP increased substantially during the study period. Although channeling of higher-risk patients to treatment with G-CSF PP is expected, the adjusted risk of FN among patients treated with G-CSF PP tended to be lower than among those not receiving G-CSF PP.

Hemodialysis patients' preferences for the management of anemia.

BACKGROUND: Patient engagement in end-stage renal disease (ESRD) is expected to result in a more patient-centered approach to care that aligns with patients' values, preferences, and goals for treatment. Nevertheless, no previous studies of which we are aware have evaluated patients' benefit-risk preferences for the management of anemia associated with ESRD. The primary objective of this study was to quantify the tradeoffs patients are willing to make between cardiovascular risks associated with some anemia medicines and red blood cell (RBC) transfusions. A secondary objective was to quantify the importance of avoiding transfusion-related risks. METHODS: A survey instrument was developed from the clinical literature, clinician input, patient-education resources, and a patient focus group. The survey instrument was qualitatively pretested before its administration to a broader sample of patients. The National Kidney Foundation invited individuals in the United States to participate in the survey. In a discrete-choice experiment (DCE), respondents chose between two hypothetical anemia medications in a series of questions. Each medication was defined by symptom relief, frequency of transfusions, cardiovascular risk, mode of administration, and out-of-pocket cost. The survey also included a best-worst scaling (BWS) exercise to quantify the importance of avoiding attributes of blood transfusions. Results from the DCE were used to estimate relative importance and marginal willingness to pay. Results from the BWS were converted to relative importance weights. RESULTS: A total of 200 individuals completed the survey. Patients were willing to accept a 6% medication-related risk of heart attack to avoid having two RBC transfusions per month. Symptom relief and mode of administration were of moderate importance. The most important transfusion-related risk to avoid was transfusion-related lung injury. CONCLUSIONS: Patients with ESRD and anemia have measurable treatment preferences and are willing to accept risks associated with anemia medications to avoid transfusions.

Clinical factors influencing the decision to order red blood cell transfusions for a sample of US dialysis patients
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AIM: To characterize the clinical context for the decision to order red blood cell (RBC) transfusions in dialysis patients. MATERIALS AND METHODS: Retrospective review of medical records from three integrated health systems serving chronic dialysis patients. Subjects were randomly selected from all patients who received at least one transfusion between January 2009 and December 2013. Data abstracted included transfusion setting, prescribing clinician type, patient demographics and hemoglobin (Hb) concentration prior to transfusion, and cataloguing and prioritizing of clinical factors for their contribution to the decision to transfuse. Data from one system were stratified between transfusions before and after the 2011 dialysis payment reform and anemia drug label changes. RESULTS: Charts for 590 patients were reviewed. The primary reason for transfusion was low Hb (51%), medical conditions (22%), symptoms of anemia (18%), surgery-related (6%), and undetermined (3%). In 93% of cases, multiple factors were cited as contributors to the transfusion decision. Mean Hb prior to transfusion was 7.2 g/dL in patients where low Hb was the primary reason for transfusion (range: 4.0 - 9.9 g/dL). CONCLUSIONS: The decision to transfuse dialysis patients is influenced by multiple patient factors and medical conditions, of which low Hb is the main contributor to this decision about half of the time.
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Granulocyte colony-stimulating factors in the prevention of febrile neutropenia: review of cost-effectiveness models.

INTRODUCTION: We reviewed the evolution of the methods used in cost-effectiveness analyses of granulocyte colony-stimulating factors (G-CSFs) in the primary and secondary prevention of febrile neutropenia (FN) in patients receiving myelosuppressive cancer chemotherapy. Areas covered: FN is a side effect of myelosuppressive chemotherapy associated with significant morbidity, mortality, and costs. The risk of FN may depend on the drugs used within a chemotherapy regimen, and an FN event may cause chemotherapy dose reductions or delays in subsequent cycles. Expert commentary: More recent pharmacoeconomic models have reflected these clinical observations by modeling sequential chemotherapy regimens to account for FN risk on a per-cycle basis, and by accounting for chemotherapy dose reductions and consequent survival losses.

Burden of Chemotherapy-Induced Febrile Neutropenia Hospitalizations in US Clinical Practice, by Use and Patterns of Prophylaxis with Colony-Stimulating Factor.

INTRODUCTION: Evidence suggests that many cancer chemotherapy patients who are candidates for colony-stimulating factor (CSF) prophylaxis do not receive it or receive it inconsistent with guidelines, and that such patients have a higher risk of febrile neutropenia hospitalization (FNH). Little is known about the number and consequences of FNH by use/patterns of CSF prophylaxis in US clinical practice. METHODS: A retrospective cohort design and private healthcare claims data were employed. Study population comprised adults who received a chemotherapy course with a high-risk regimen, or an intermediate-risk regimen (if ≥1 FN risk factor present), for non-metastatic breast cancer or non-Hodgkin's lymphoma (NHL); each chemotherapy cycle within the course and each FNH episode within the cycles were identified. Consequences included mortality, inpatient days, and costs (US$2013) during FNH. Use (yes/no) and patterns (agent, administration day/duration) of CSF prophylaxis were evaluated within cycles in which FNH episodes occurred. RESULTS: Among all FNH episodes (n=6,355; 109 episodes per 1,000 patients), 41.3% (95% CI: 40.1-42.5) occurred among patients who did not receive CSF prophylaxis in that cycle, and 8.8% (8.1-9.5) occurred among those who received CSF prophylaxis on the same day as chemotherapy. Among FNH episodes occurring in patients who received daily CSF agents (2% of CSF use), 56.1% (44.1-68.0) received prophylaxis <7 days during the cycle. Results for FNH consequences were comparable. CONCLUSIONS: In this retrospective evaluation, one-half of FNH episodes, outcomes, and costs among cancer chemotherapy patients who were candidates for CSF prophylaxis occurred in those who either did not receive it or received it inconsistent with guidelines.

Effects of the prospective payment system on anemia management in maintenance dialysis patients: implications for cost and site of care.

BACKGROUND: The 2011 expanded Prospective Payment System (PPS) and contemporaneous Food and Drug Administration label revision for erythropoiesis-stimulating agents (ESAs) were associated with changes in ESA use and mean hemoglobin levels among patients receiving maintenance dialysis. We aimed to investigate whether these changes coincided with increased red blood cell transfusions or changes to Medicare-incurred costs or sites of anemia management care in the period immediately before and after the introduction of the PPS, 2009-2011. METHODS: From US Medicare end-stage renal disease (ESRD) data (Parts A and B claims), maintenance hemodialysis patients from facilities that initially enrolled 100 % into the ESRD PPS were identified. Dialysis and anemia-related costs per-patient-per-month (PPPM) were calculated at the facility level, and transfusion rates were calculated overall and by site of care (outpatient, inpatient, emergency department, observation stay). RESULTS: More than 4100 facilities were included. Transfusions in both the inpatient and outpatient environments increased. In the inpatient environment, PPPM use increased by 11-17 % per facility in each quarter of 2011 compared with 2009; in the outpatient environment, PPPM use increased overall by 5.0 %. Site of care for transfusions appeared to have shifted. Transfusions occurring in emergency departments or during observation stays increased 13.9 % and 26.4 %, respectively, over 2 years. CONCLUSIONS: Inpatient- and emergency-department-administered transfusions increased, providing some evidence for a partial shift in the cost and site of care for anemia management from dialysis facilities to hospitals. Further exploration into the economic implications of this increase is necessary.

"Same-Day" administration of pegfilgrastim following myelosuppressive chemotherapy: clinical practice and provider rationale.

PURPOSE: To describe patient- and practice-related factors that physicians report affect their clinical decision to administer prophylactic pegfilgrastim to patients <24 h after completion of a myelosuppressive chemotherapy cycle (i.e., "same-day" pegfilgrastim). METHODS: Oncologists, hematologists, and hematologist-oncologists enrolled in a US national physician panel were invited to participate in a cross-sectional, web-based survey to assess physicians' reasons for prescribing "same-day" pegfilgrastim. Physicians were screened as eligible if they reported prescribing "same-day" pegfilgrastim within the previous 6 months. The survey assessed physician perspectives and physician-perceived patient/caregiver preferences. RESULTS: Of 17,478 invited physicians, 386 answered the screening questions; 151 (39.1 %) were eligible, agreed to participate, and completed the survey. Physicians estimated that overall 41.3 % of their patients treated with myelosuppressive chemotherapy received pegfilgrastim and that 31.6 % treated with pegfilgrastim received it on a "same-day" schedule. Approximately 36 % of physicians relied primarily on their clinical judgment when deciding to administer "same-day" pegfilgrastim. The clinical consideration reported most commonly by physicians as moderately or very important when deciding to administer "same-day" pegfilgrastim was previous febrile neutropenia (77.6 %). The most important patient-related consideration in the decision to administer "same-day" pegfilgrastim was patient/caregiver travel distance, and the most important practice-related consideration was the burden to the physician's practice of "next-day" administration (vs. same-day), reported by 84.7 % and 65.1 % of physicians as moderately or very important, respectively. CONCLUSIONS: While clinical judgment, patients' risk factors, and practice burden were principal influences favoring "same-day" pegfilgrastim administration, physician-perceived patient preferences and logistical barriers also have important roles in this decision.

Annual patient and caregiver burden of oncology clinic visits for granulocyte-colony stimulating factor therapy in the US.

OBJECTIVE: Prophylactic treatment with granulocyte-colony stimulating factors (G-CSFs) is indicated for chemotherapy patients with a significant risk of febrile neutropenia. This study estimates the annual economic burden on patients and caregivers of clinic visits for prophylactic G-CSF injections in the US. METHODS: Annual clinic visits for prophylactic G-CSF injections (all cancers) were estimated from national cancer incidence, chemotherapy treatment and G-CSF utilization data, and G-CSF sales and pricing information. Patient travel times, plus time spent in the clinic, were estimated from patient survey responses collected during a large prospective cohort study (the Prospective Study of the Relationship between Chemotherapy Dose Intensity and Mortality in Early-Stage (I-III) Breast Cancer Patients). Economic models were created to estimate travel costs, patient co-pays and the economic value of time spent by patients and caregivers in G-CSF clinic visits. RESULTS: Estimated total clinic visits for prophylactic G-CSF injections in the US were 1.713 million for 2015. Mean (SD) travel time per visit was 62 (50) min; mean (SD) time in the clinic was 41 (68) min. Total annual time for travel to and from the clinic, plus time at the clinic, is estimated at 4.9 million hours, with patient and caregiver time valued at $91.8 million ($228 per patient). The estimated cumulative annual travel distance for G-CSF visits is 60.2 million miles, with a total transportation cost of $28.9 million ($72 per patient). Estimated patient co-pays were $61.1 million, ∼$36 per visit, $152 per patient. The total yearly economic impact on patients and caregivers is $182 million, ∼$450 per patient. LIMITATIONS: Data to support model parameters were limited. Study estimates are sensitive to the assumptions used. CONCLUSIONS: The burden of clinic visits for G-CSF therapy is a significant addition to the total economic burden borne by cancer patients and their families.

Risk of chemotherapy-induced febrile neutropenia with early discontinuation of pegfilgrastim prophylaxis in US clinical practice.

PURPOSE: Accumulating evidence suggests that not all cancer chemotherapy patients who receive first-cycle pegfilgrastim prophylaxis continue to receive it in subsequent cycles and that these patients may be subsequently at higher risk of febrile neutropenia (FN). Additional evidence from US clinical practice is warranted. METHODS: Data from two US private healthcare claims repositories were employed. The source population comprised adults who received "intermediate-risk" or "high-risk" chemotherapy regimens for solid cancers or non-Hodgkin's lymphoma and first-cycle pegfilgrastim prophylaxis. From the source population, all patients who did not receive second-cycle pegfilgrastim prophylaxis ("comparison patients") were matched (1:1) to those who received it ("pegfilgrastim patients") based on cancer, regimen, and propensity score. Odds ratios (OR) for FN-broad and narrow definitions-during the second chemotherapy cycle were estimated for comparison patients versus pegfilgrastim patients using generalized estimating equations. RESULTS: A total of 2245 comparison patients (5.3 % of source population) were matched to pegfilgrastim patients; cohorts were well-balanced on baseline characteristics. Second-cycle FN incidence proportions for comparison and pegfilgrastim patients were 3.8 versus 2.2 % based on broad definition and 2.6 versus 0.8 % based on narrow definition; corresponding OR were 1.7 (95 % CI 1.2-2.5, p = 0.002) and 3.5 (95 % CI 2.0-6.0, p < 0.001). Results were similar within cancer/regimen-subgroups and were robust when using alternative methods for confounding adjustment. CONCLUSIONS: In this retrospective evaluation of cancer chemotherapy patients who received first-cycle pegfilgrastim prophylaxis in US clinical practice, a clinically relevant minority did not receive second-cycle prophylaxis. Second-cycle FN odds among this subset were significantly higher than they were among those who continued prophylaxis.

Risk of chemotherapy-induced febrile neutropenia in cancer patients receiving pegfilgrastim prophylaxis: does timing of administration matter?

PURPOSE: Contrary to the approved indication for pegfilgrastim prophylaxis, some patients receive it on the same day as the last administration of chemotherapy in clinical practice, which could adversely impact risk of febrile neutropenia (FN). An evaluation of the timing of pegfilgrastim prophylaxis and FN risk was undertaken. METHODS: A retrospective cohort design and data from two US private health care claims repositories were employed. Study population comprised adults who received intermediate/high-risk chemotherapy regimens for solid tumors or non-Hodgkin's lymphoma (NHL) and received pegfilgrastim prophylaxis in ≥1 cycle; all cycles with pegfilgrastim were pooled for analyses. Odds ratios (OR) for FN during the cycle were estimated for patients who received pegfilgrastim on the same day (day 1) as the last administration of chemotherapy versus days 2-4 from chemotherapy completion. RESULTS: The study population included 45,592 patients who received pegfilgrastim in 179,152 cycles (n = 37,095 in cycle 1); in 12 % of cycles, patients received pegfilgrastim on the same day as chemotherapy. Odds of FN were higher for patients receiving pegfilgrastim prophylaxis on the same day as chemotherapy versus days 2-4 from chemotherapy in cycle 1 (OR = 1.6, 95 % CI = 1.3-1.9, p < 0.001) and all cycles (OR = 1.5, 95 % CI = 1.3-1.6, p < 0.001). CONCLUSIONS: In this large-scale evaluation of adults who received intermediate/high-risk regimens for solid tumors or NHL in US clinical practice, FN incidence was found to be significantly higher among those who received pegfilgrastim prophylaxis on the same day as chemotherapy completion versus days 2-4 from chemotherapy completion, underscoring the importance of adhering to the indicated administration schedule.

Trends in prevalence of patient case-mix adjusters used in the Medicare dialysis payment system.

OBJECTIVES: The Medicare End-Stage Renal Disease Prospective Payment System (PPS) used data from 2006-08 to set weights for each case-mix adjuster that is part of the bundled payment formula. The details of the population case-mix were not made public, and little is known about consistency of case-mix over time. This study estimated the prevalence of case-mix adjusters during 2006-2008 and analyzed changes in case-mix prevalence from 2000-2008. METHODS: Cross-sectional cohort study using United States Renal Data System data for Medicare dialysis patients. Three 3-year cohorts (2000-02, 2003-05, 2006-08) were analyzed for changes over time in case-mix prevalence. RESULTS: Double-digit trends were observed in many case-mix categories between 2000-02 and 2006-08. Large declines were observed in prevalence of patients with low BMI, pericarditis, new to dialysis, and ages 18-44. Large increases were observed in chronic co-morbidities, pneumonia and age cohort 80+. CONCLUSIONS: Substantial changes in case-mix adjuster prevalence suggest the PPS payment formula should be regularly updated.

Prevalence of gastrointestinal stromal tumour (GIST) in the United Kingdom at different therapeutic lines: an epidemiologic model.

BACKGROUND: The prevalence of patients with gastrointestinal stromal tumourgst (GIST) who fail currently available treatments imatinib and sunitinib (third-line treatment-eligible GIST) is unknown, but is expected to be below an ultra-orphan disease threshold of 2/100,000 population used in England and Wales. Our study was designed to estimate the prevalence and absolute number of UK patients with unresectable/metastatic GIST at first-, second- and eventually third-line treatment. METHODS: Our open population model estimates the probability that the prevalence of UK third-line treatment-eligible GIST patients will remain under the ultra-orphan disease threshold. Model parameters for incidence, proportion of unresectable/metastatic disease and survival estimates for GIST patients were obtained from a targeted literature review and a UK cancer register. The robustness of the results was checked through differing scenarios taking extreme values of the input parameters. RESULTS: The base-case scenario estimated a prevalence of third-line treatment-eligible GIST of 1/100,000 and a prevalence count of 598 with a 99.9% likelihood of being below the ultra-orphan disease threshold. The extreme scenarios, one-way and probabilistic sensitivity analyses and threshold analysis confirmed the robustness of these results. CONCLUSIONS: The prevalence of third-line treatment-eligible GIST is very low and highly likely below the ultra-orphan disease threshold.