ALBERT

Description: Menorrhagia is a common clinical problem among reproductive age women and annually 5% of reproductive age women seek medical attention, usually with gynecologists and other primary care physicians, for this symptom. Since underlying bleeding disorders are common in women presenting with menorrhagia, and referral for comprehensive hemostatic testing of substantial numbers of women with otherwise unexplained menorrhagia is problematic from the public health and cost perspective, a short, easy-to-administer screening tool comprised of 8 questions for identifying women with menorrhagia for hemostatic evaluation was previously developed (Am J Ob Gyn2008;198:e1–163e8). In the present study, the validity of the screening tool was evaluated in a multi-site, prospectively recruited cohort of women with menorrhagia. 232 women with menorrhagia age 18 and older with a pictorial blood assessment chart (PBAC) score 〉 100 were recruited from 5 US centers as potential subjects for a prospective cross-over study for evaluation of intranasal DDAVP versus tranexamic acid. All subjects underwent comprehensive laboratory testing for bleeding disorders, including VWF, platelet aggregation/ATP release, and factor assays. Study participants were administered a questionnaire which included the 8 screening tool questions in 4 categories, including history of duration and severity of menorrhagia, anemia treatment, excessive bleeding with hemostatic challenges, and family diagnosis of bleeding disorder. A screening tool was considered positive if there was a positive response for any of the questions in the four categories. Sensitivity of the screening tool with 95% confidence interval was calculated for bleeding disorders and also separately for low VWF (ristocetin cofactor 〈 50%), and platelet function defects.217 women with menorrhagia including 78% white and 16% black women with complete data were evaluated. In this population, a positive screening tool had a sensitivity of 89% (95% CI, 83–93) for bleeding disorders, 89% for platelet function defects (95% CI, 82–94), and 73% for low VWF (95% CI, 39–94). The sensitivity for bleeding disorders was 87% (95% CI, 79–92) among white women and 94% (95% CI, 79–99) among black women. Adding a PBAC score 〉 185 increased the sensitivity of the screening tool for bleeding disorders to 95% (95% CI, 90–98). Using a multi-site US population of adult women with menorrhagia, this study confirms the benefit of a short screening tool to assist primary care physicians in the selection of women with menorrhagia to refer for comprehensive hemostatic testing and evaluation. population of adult women with menorrhagia, this study confirms the benefit of a short screening tool to assist primary care physicians in the selection of women with menorrhagia to refer for comprehensive hemostatic testing and evaluation.

Description: Introduction: Menorrhagia is a common health problem affecting ∼5-10% of women. The optimal management of menorrhagia among women with abnormal laboratory hemostasis is uncertain. The goal of this study was to determine the effect of intranasal desmopressin (IN-DDAVP) vs. oral tranexamic acid (TA) on menstrual blood loss (MBL) and quality of life (QOL). Methods: In a cross-over study design, 117 consenting women 18–50 years of age with menorrhagia (defined as subjective report of heavy bleeding and a pictorial blood assessment chart [PBAC] score 〉 100) with a negative gynecological evaluation and abnormal laboratory hemostasis (62% platelet dysfunction, 15% von Willebrand disease, 23% other coagulation defect or combined) were randomly assigned to initial IN-DDAVP or TA therapy each for two menstrual cycles, followed by cross-over to the second study drug for an additional two cycles. MBL by PBAC was measured at baseline and after each menstrual cycle while QOL was assessed by four validated instruments: Health Related Quality of Life (HRQOL), SF-36®, Center for Epidemiologic Studies Depression Scale (CES-D) and the Ruta menorrhagia questionnaire. Results: There was a statistically significant decrease in the PBAC for both treatments. On average, the estimated decrease in the PBAC from baseline for IN-DDAVP was −66.0 (CI = (−89.5, −42.6)) and for TA, −107.8 (CI = (−131.5, −84.1)). The decrease in the PBAC score was larger for TA with a difference of 41.8 (p-value = 0.0002, CI = (20.4, 63.2)) between the two treatments. In the multivariable analysis, the test for treatment-cycle number interaction was not significant, suggesting no carry-over effect. The test for a treatment type effect was significant (p

Description: Background: Continuous prophylaxis, or the routine replacement of deficient clotting factor, is the standard of care therapy for individuals with severe hemophilia A and joint bleeding. In the United States (US), the majority of eligible patients are prescribed continuous prophylaxis, including approximately 75% of children 〈 20 years of age. However, up to 30% of severe hemophilia A patients develop neutralizing alloantibodies that render factor replacement ineffective requiring inhibitor eradication through induction of immune tolerance (ITI) therapy. Tolerized patients demonstrate successful elimination of inhibitors and normal response to FVIII concentrates. Non-tolerized patients can also be treated on prophylaxis using bypassing agents. Aim: This analysis used national surveillance data to compare joint and other outcomes in persons with severe hemophilia A on continuous prophylaxis in three groups: no history of an inhibitor, likely tolerized inhibitor and active (non-tolerized) inhibitor. Methods: The Community Counts bleeding disorders surveillance project is funded by the Centers for Disease Control and Prevention (CDC) through a cooperative agreement awarded to the American Thrombosis and Hemostasis Network (ATHN) in partnership with the US Hemophilia Treatment Center Network (USHTCN). The Registry for Bleeding Disorders Surveillance component collects detailed medical information on patients with bleeding disorders who receive treatment within the USHTCN. Likely tolerized inhibitors were defined as having a history of a previous inhibitor and currently being treated with FVIII concentrate; active inhibitors were defined as having a history of a previous inhibitor and being treated with bypassing agents. Using the Registry component, the following data elements, stratified by age and inhibitor status, were extracted from the initial visit form: demographics, treatment regimen, mobility, cumulative joint bleeding, invasive joint procedures, pain and opioid use. Results: Data on 1,300 persons with severe hemophilia A were analyzed including: 739 with a negative inhibitor history; 410 likely tolerized inhibitor patients; and 151 with an active inhibitor. The 410 patients represented 73% of participants with a positive inhibitor history, similar to the North American Inhibitor Registry (DiMichele D, Haemophilia 2009;15:320-8). Participants with likely tolerized inhibitors were treated using continuous prophylaxis in similar proportion to participants with no history of inhibitor (83% vs 78%, respectively), and more often than participants with active inhibitor (83% vs 65%); however outcomes of participants on continuous prophylaxis were similar in all three groups (Table 1). Patients 〈 20 years of age had less disability, and fewer joint bleeds, invasive procedures, pain and opioid use compared with adult participants. Conclusions: Improved outcomes on preventive strategies may be best determined prospectively in younger patients due to the lesser degree of established morbidity. Persons with severe hemophilia A on continuous prophylaxis with likely tolerized inhibitors (using FVIII) or with active inhibitors (using bypassing agents) demonstrate outcomes similar to those without inhibitors. However, more age- and treatment regimen- stratified analysis is needed for more precise comparisons. Future findings may contribute to the perspective that prophylaxis should be the standard of care for all hemophilia patients regardless of inhibitor status. Disclosures Manco-Johnson: Bayer: Honoraria, Research Funding; Baxalta: Honoraria; NovoNordisk: Honoraria; BiogenIdec: Honoraria; CSL Behring: Honoraria. Kulkarni:Baxter: Membership on an entity's Board of Directors or advisory committees, Research Funding; Bayer: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novo Nordisk: Membership on an entity's Board of Directors or advisory committees, Research Funding; BPL: Membership on an entity's Board of Directors or advisory committees; Kedrion: Membership on an entity's Board of Directors or advisory committees; Pfizer: Membership on an entity's Board of Directors or advisory committees; Biogen: Research Funding, Speakers Bureau. Buckner:Novo Nordisk: Consultancy; Genentech: Consultancy; Baxalta: Consultancy.

Description: Introduction: An estimated 30% of persons with hemophilia (PWH) in the US do not receive care at federally-funded hemophilia treatment centers (HTCs). These estimates suggest that perhaps 6,000 PWH and an unknown number of persons with other genetic bleeding disorders receive care elsewhere (non-HTC PWBD). Data on non-HTC PWBD are not collected on a regular basis nor maintained in a central database. Consequently, little is known about their characteristics, treatment, or care. CDC partnered with a national non-profit, consumer-focused organization, Hemophilia Federation of America (HFA), on the CHOICE Project to collect information on non-HTC PWBD to better estimate disease burden and inform planning, decision making, and programming. The objective of this analysis is to describe the treatment, sources, and patterns of care for non-HTC PWBD. Methods: From 04/2013-07/2015, HFA recruited US PWBD 〉/=18 years old (adults) and caregivers of children with bleeding disorders for CHOICE through email, social media, print, and word-of-mouth to take a ~20 minute survey in English or Spanish, online or on paper. Non-HTC PWBD were solicited specifically but others were not excluded from participation. Participants' status as non-HTC PWBD was determined using an algorithm based on responses to specific survey questions. For this analysis, survey elements included demographics, diagnosis, treatment regimen and treatment products used, and health services utilization (usual source of care, frequency of care, barriers to regular care, delay of care, hospital emergency room (ER) utilization, and hospitalizations within the past 12 months). Results: One hundred seventy-seven (22%) participants from 805 eligible surveys were identified as non-HTC PWBD: 52.5% male; 72.7% adult; mean age 33 years (±19). One hundred fifty-six (88.1%) reported a single bleeding disorder diagnosis; 21 reported multiple diagnoses. See Tables 1 and 2: The most commonly used treatments for hemophilia A or B were recombinant clotting factor concentrate (factor) (75.9%), topical or non-plasma products (e.g., desmopressin) (19.5%), and plasma-derived factor (11.5%). Non-plasma or topical products (50.0%), plasma-derived factor VIII (47.1%), and hormonal contraceptives (26.5%) were used to treat von Willebrand disease. Over 77% of those using intravenous product infused at home; 45.3% self-infused. Of those using factor, 47.5% used it to prevent bleeds with 38.2% practicing continuous prophylaxis. Approximately 32% did not visit a healthcare provider regularly for their bleeding disorder; top reasons included no need for regular care (51.9%) and expense (22.2%). One hundred seven (61.5%) respondents had one (38.1%) or more (61.9%) healthcare providers from whom they received care for their bleeding disorder. Nearly 88% (94) visited a hematologist; when a hematologist was not visited, the family practitioner was the next most common provider used (6.6%). The most common places for care were the doctor's office (49.4%), ER (16.1%), and hospital outpatient department (12.1%). In the last 12 months, 33.3% visited an ER and 18.6% were admitted to a hospital due to their bleeding disorder; 32.9% delayed or did without needed care due mostly to expense/lack of insurance (54.4%), lack of healthcare providers (21.7%), or inconvenience (17.4%). Children were more likely than adults to: have a usual healthcare provider (75.0% vs. 56.8%, p=.03); visit a healthcare provider regularly for care of their bleeding disorder (97.9% vs. 57.0%, p