Hematology

Earnshaw SR, Graham CN, McDade CL, Spears JB, Kessler CM. Factor VIII alloantibody inhibitors: cost analysis of immune tolerance induction vs. prophylaxis and on-demand with bypass treatment. Haemophilia. 2015 May;21(3):310-9. doi: 10.1111/hae.12621

Summary:  Development of inhibitors (alloantibodies to exogenous factor VIII) is the most significant treatment complication in patients with haemophilia A. The only proven way to eradicate inhibitors is through immune tolerance induction (ITI), while bypassing agents are typically employed to treat or prevent bleeds in patients with high titre inhibitors. Costs of these approaches have not been well studied.

Davis KL, Cote I, Kaye JA, Gao H, Perez Ronco J, Seifeldin R, Mendelson E. Real-world assessment of clinical outcomes in lower-risk myelofibrosis patients receiving treatment with ruxolitinib. Poster presented at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition; December 6, 2014. San Francisco, CA. [abstract] Blood. 2014 Dec 6; 124(21):1857. doi: 10.1182/blood.V124.21.1857.1857

Introduction: Published trial data shows that ruxolitinib improves both splenomegaly-related and nonsplenomegaly-related constitutional symptoms in patients with intermediate-2 and high risk myelofibrosis (MF). However, few trial-based assessments of ruxolitinib in lower-risk MF patients have been conducted and no studies to date have made such assessments in a real-world population.

Xu H, Kaye JA, Saltus CW, Crawford J, Gasa E, Goodnough LT. Blood utilization and hemoglobin levels in cancer patients after label and coverage changes for erythropoiesis-stimulating agents. Expert Rev Hematol. 2014 Oct;7(5):617-33. doi: 10.1586/17474086.2014.943730

A comprehensive literature search was performed to examine the influence of changes in erythropoietin-stimulating agent (ESA) label and reimbursement policies on utilization of red blood cell transfusions and patient hemoglobin levels in US cancer patients receiving chemotherapy or anemia management. Studies conducted in ESA-treated patients showed an increase in transfusion rates when comparing the post-intervention period with pre-intervention period (range of relative change: 15-125%).

Rosseli D, Rueda J, Wolowacz SE, Brockbank JA, Abeysinghe SS. Cost-effectiveness of dabigatran compared with warfarin, apixaban, rivaroxaban and low molecular weight heparins for the treatment and secondary prevention of venous thromboembolism in Colombia. Poster presented at the 2014 ISPOR 17th Annual European Congress; November 2014. Amsterdam, The Netherlands.

Abstract not available at this time

DiBenedetti DB, Coles TM, Sharma T, Pericleous L, Kulkarni R. Assessing patients' and caregivers' perspectives on stability of factor VIII products for haemophilia A: a web-based study in the United States and Canada. Haemophilia. 2014 Jul;20(4):e296-303. doi: 10.1111/hae.12459

Haemophilia A is a rare inherited bleeding disorder characterized by an inability of the blood to clot normally. Patients can experience spontaneous or trauma-induced joint and soft tissue bleeding and must keep coagulation factor VIII (FVIII) accessible at all times; thus, FVIII product storage and stability are critical. Our primary objective was to assess haemophilia A patients' and caregivers' experiences and preferences with FVIII product storage and stability. A secondary objective was to evaluate the use of the social media site Facebook in recruitment.

Virkus RA, Løkkegaard E, Lidegaard O, Langhoff-Roos J, Nielsen AK, Rothman KJ, Bergholt T. Risk factors for venous thromboembolism in 1.3 million pregnancies: a nationwide prospective cohort. PLoS One. 2014 May 2;9(5):e96495. doi: 10.1371/journal.pone.0096495. eCollection 2014

Objective: To quantify risk factors for venous thromboembolism during pregnancy and the puerperal period. Design In a nationwide prospective cohort study we followed pregnant and puerperal women in Denmark from 1995 to 2009 for venous thromboembolism. Information on risk factors and confounders was retrieved from national registries. The diagnosis of venous thromboembolism was confirmed through medical charts. We calculated adjusted incidence rates per 10,000 women years and used Poisson regression to estimate effects during pregnancy and the puerperal period.

Arana A, Wait S, Santamas M, Ward A, Huybrechts K, Craig T. Thalassemia major: geographical variations in prevalence, survival and treatment patterns. Poster presented at the 16th International Conference on Pharmacoepidemiology; August 2000. [abstract] Pharmacoepidemiol Drug Saf. 9(S1):S96.

Abstract not available at this time

Arana A, Cid Ruzafa J, Rueda de Castro AM, Warner BA, O'Sullivan D. Evaluation of the clozaril® patient monitoring service (CPMS) in the prevention of clozaril/leponex-induced white-blood-celltoxicity. Presented at the 18th International Conference on Pharmacoepidemiology; August 2002.

Abstract not available at this time

Arboretti R, Tognoni G, Alberti D, Arana A. Pharmacosurveillance and quality of care of thalassaemic patients. A large scale epidemiological survey. Eur J Clin Pharmacol. 2001;56(12):915-22.


Caro JJ, Ward A, Craig T, Huybrecht K, Arana A, Wait S, Eleftheriou A. Impact of thalassemia major on patients and their families. Acta Haematol. 2002;107(3):150-7.

OBJECTIVE: To describe the burden of thalassemia major and its treatment, in terms of prevalence of iron-overload-related complications, direct and indirect costs, and the patient's physical and social well-being. METHODS:From October 1999 to May 2000 a survey of patients with thalassemia major was conducted in ten countries: Cyprus, Egypt, Greece, Hong Kong, India, Iran, Italy, Jordan, Taiwan, and the United States. RESULTS:1,888 questionnaires (65%) were returned.

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