M. Koerper

Sites of initial bleeding episodes, mode of delivery and age of diagnosis in babies with haemophilia diagnosed before the age of 2 years: a report from The Centers for Disease Control and Prevention’s (CDC) Universal Data Collection (UDC) project

Summary.u2002 Lack of detailed natural history and outcomes data for neonates and toddlers with haemophilia hampers the provision of optimal management of the disorder. We report an analysis of prospective data collected from 580 neonates and toddlers aged 0–2 years with haemophilia enrolled in the Universal Data Collection (UDC) surveillance project of the Centers for Disease Control and Prevention (CDC). This study focuses on a cohort of babies with haemophilia whose diagnosis was established before the age of two. The mode of delivery, type and severity of haemophilia, onset and timing of haemorrhages, site(s) of bleeding, provision of prophylaxis with coagulation factor replacement therapy, and the role played by the federally funded Haemophilia Treatment Centers (HTC) in the management of these infants with haemophilia were evaluated. Seventy‐five per cent of haemophilic infants were diagnosed early, in the first month of life, especially those with a family history or whose mothers were known carriers; infants of maternal carriers were more likely to be delivered by C‐section. Involvement of an HTC prior to delivery resulted in avoidance of the use of assisted deliveries with vacuum and forceps. Bleeding from the circumcision site was the most common haemorrhagic complication, followed by intra‐ and extra‐cranial haemorrhages and bleeding from heel stick blood sampling. Eight per cent of the infants were administered factor concentrate within 24 h of birth; more than half were treated to prevent bleeding. This study highlights the significant rate and the sites of initial bleeding unique to very young children with haemophilia and underscores the need for research to identify optimal evidence‐based recommendations for their management.

Unresolved issues in diagnosis and management of inherited bleeding disorders in the perinatal period: A White Paper of the Perinatal Task Force of the Medical and Scientific Advisory Council of the National Hemophilia Foundation, USA

Summary.u2002 Haemophilia and inherited bleeding disorders in newborns and their carrier mothers pose unique challenges. The pattern of bleeding and the causes and risk factors for bleeding are decidedly different than an older child or an adult with haemophilia/inherited bleeding disorder. This document outlines the needs for further research and education, summarizes the state of the art background information and provides guidance regarding research, education and access to care issues in this population.

Haemophilia Utilization Group Study - Part Va (HUGS Va): design, methods and baseline data

Summary.u2002 To describe the study design, procedures and baseline characteristics of the Haemophilia Utilization Group Study – Part Va (HUGS Va), a US multi‐center observational study evaluating the cost of care and burden of illness in persons with factor VIII deficiency. Patients with factor VIII level ≤30%, age 2–64u2003years, receiving treatment at one of six federally supported haemophilia treatment centres (HTCs) were enrolled in the study. Participants completed an initial interview including questions on socio‐demographical characteristics, health insurance status, co‐morbidities, access to care, haemophilia treatment regimen, factor utilization, self‐reported joint pain and motion limitation and health‐related quality of life. A periodic follow‐up survey collected data regarding time lost from usual activities, disability days, health care utilization and outcomes of care. HTC clinicians documented participants’ baseline clinical characteristics and pharmacy dispensing records for 2u2003years. Between July 2005 and July 2007, 329 participants were enrolled. Average age was 9.7u2003years for children and 33.5u2003years for adults; two‐thirds had severe haemophilia. The distributions of age, marital status, education level and barriers to haemophilia care were relatively consistent across haemophilic severity categories. Differences were found in participants’ employment status, insurance status and income. Overall, children with haemophilia had quality of life scores comparable to healthy counterparts. Adults had significantly lower physical functioning than the general US population. As one of the largest economic studies of haemophilia care, HUGS Va will provide detailed information regarding the burden of illness and health care utilization in the US haemophilia A population.

Physical activity and health outcomes in persons with haemophilia B

Regular participation in physical activity helps to prevent damage and maintain joint health in persons with haemophilia. This study describes self‐reported physical activity participation among a sample of people with haemophilia B in the US and measures its association with health‐related quality of life (HRQoL). Data on 135 participants aged 5–64 years were abstracted from Hemophilia Utilization Group Study Part Vb. The International Physical Activity Questionnaire assessed physical activity among participants aged 15–64 years, and the Childrens Physical Activity Questionnaire abstracted from the Canadian Community Health Survey was used for participants aged 5–14 years. SF‐12 was used to measure HRQoL and the EuroQol (EQ‐5D‐3L) was used to measure health status for participants older than 18 years of age. PedsQL was used to measure HRQoL in children aged 5–18 years. Sixty‐two percent of participants in the 15–64 year‐old age cohort reported a high level of physical activity, 29% reported moderate activity and 9% reported low activity. For children aged 5–14 years, 79% reported participating in physical activity for at least 4 days over a typical week. Based on the 2008 Physical Activity Guidelines for Americans, 79% of adults achieved the recommended physical activity level. Multivariable regression models indicated that adults who engaged in a high level of physical activity reported EQ‐5D Visual Analogue Scale (VAS) scores that were 11.7 (P = 0.0726) points greater than those who engaged in moderate/low activity, indicating better health outcomes. Among children, no statistically significant differences in health outcomes were found between high and moderate or low activity groups.

Factor IX inhibitors and anaphylaxis in haemophilia B

I. WARRIER, B. M. EWENSTEIN, M. A. KOERPERY3 A. SHAPIR0 , N . KEY, D. DIMICHELEY6 R. T. MILLER, J. PASI,* G. E. RIVARD,9 S. S. SOMMER, F. BERGMANN and J. M. LUSHER Department of Haematology and Oncology, Childrens Hospital of Michigan and Wayne State University, 3901 Beaubien Blvd, Detroit MI 48201-2196, USA, Hatward Medical School, and Brigham and Womens Hospital, 75 Francis Street, Boston MA 02115, USA, UCSF Medical Centre, 1466 Fourth Avenue, Sun Francisco, C A 94143, USA, Riley Hospital, 702 Barnhill Drive, Indianapolis, IN 46202, USA, University of Minnesota Hospital and Clinic. Box 713,420 Delaware Street, S.E., Minneapolis, MN 55455,USA, 6New York Hospital, Cornell Medical Centre, 525 E. 68th Street, New York, NY 10021, USA, 7L0s Angeles Childrens Hospital, 4650 Sunset Blvd, Los Angeles, CA 90027, USA, Royal Free Hospital, Pond Street, London NW3 20G, UK. Hospital Ste.Justine, 31 75 C!re-Sainte-Catherine, Montreal, Quebec, Canada, Mayo CliniclFoundation, Rochester, MN 55905, USA and Department of Pediatrics, Hannover Medical School, Hanover, Germany

More on: Are randomized clinical trials the only truth? Not always

See also Aledort L, Ljung R, Blanchette V on behalf of the International Prophylaxis Study Group (IPSG). Are randomized clinical trials the only truth? Not always. J ThrombHaemost 2006; 4: 503–4;Mannucci PM. Need for randomized trials in hemophilia. J ThrombHaemost 2006; 4: 501–2; Iorio A, Stobart K, Bolton-Maggs P. Can evidence harm? Certainly not hemophilia treatment and community. J Thromb Haemost 2006; 4: 505–6.

MASAC Consensus Conference: impediments to conducting clinical research in persons with haemophilia, von Willebrand's disease and rare bleeding disorders

A consensus conference conducted by the Medical and Scientific Advisory Council of the National Hemophilia Foundation was held in New Orleans, LA, on November 11, 2010, to discuss the impediments to conducting clinical research in persons with haemophilia, von Willebrands disease and rare bleeding disorders. The conference combined presentations providing academic, non‐profit and industry perspectives with periods of open discussion. The objective of this conference was to identify the many challenges involved in facilitating U.S. Food and Drug Administration approval of innovative products for these patient populations.

PSY3 HEALTH-CARE UTILIZATION AND COST IN PERSONS WITH FACTOR VIII DEFICIENCY: RESULTS OF THE HUGS VA STUDY

effectiveness of HLA-B*5801 genotyping compared to no testing. The incidence of SJS/TEN was estimated based on case reports from Health Product Vigilance Center of Thailand in year 2009. The prevalence of HLA-B*5801 was obtained from Thai population reported in dbMHC database, while the association of gene and SJS/TEN was based on a meta-analysis. Cost of SJS/TEN management and case-fatality rate were derived from National Inpatient Governmental Hospital Database in year 2007. We used PG5801 DNA detection kit as a genotyping tool with 100% sensitivity and specifi city. We varied genotyping costs and selected values that would make the costeffectiveness values being 100,000 or 300,000 THB/life-year gained. One-way sensitivity analysis was undertaken to identify infl uential parameters. RESULTS: The estimated life-years (LY) were 21.9999 and 21.9964 for testing and no testing groups, respectively. Setting the genotyping cost as 393 and 1085 THB resulted in a potentially cost-effectiveness range of 100,000 and 300,000 THB/LY, respectively. The most infl uential parameters were the cost of genotyping and SJS/TEN management. CONCLUSIONS: Pharmacogenetic testing for HLA-B*5801 appears to be potentially cost-effective if the testing cost falls in the range of 393 and 1085 THB. It was important to note that this analysis has not taken into account sequelaes associated with SJS/TEN and has not performed based on the societal perspective yet. Policymakers should consider our fi ndings for guiding health policy during decision-making process.

PSY32 HEALTH CARE UTILIZATION AND FACTOR COST IN HEMOPHILIA

LBP treatment rates. Wilcoxon signed-rank tests were used to compare study period direct (medical and drug) costs from third-party payer perspective. RESULTS: During the 6-month study period, duloxetine-treated patients vs. controls had signifi cantly lower rates of other pharmacological therapy (34.2% vs. 43.4% narcotic opioids, p 0.004; 30.4% vs. 43.2% NSAIDs, p 0.001; 17.4% vs. 26.2% muscle relaxants, p 0.001; 10.6% vs. 20.4% corticosteroids, p 0.001) and non-invasive therapy (16.4% vs. 35.6% chiropractic therapy, p 0.001; 13.0% vs. 34.2% physical therapy, p 0.001). Duloxetine-treated patients were also signifi cantly less likely to have a back surgery during the study period compared with controls (0.4% vs. 2.0%, respectively; p 0.021). Average 6-month direct costs were not signifi cantly different between duloxetine-treated patients and controls (

Anaphylactic response to factor IX replacement therapy in haemophilia B patients: complete gene deletions confer the highest risk

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