Pathogen Safety of Long-Term Treatments for Bleeding Disorders: (Un)Predictable Risks and Evolving Threats

Giovanni Di Minno; Mariana Canaro; James W. Ironside; David Navarro; Carlo F. Perno; Andreas Tiede; Lutz Guertler

doi:10.1055/s-0033-1354420

Seminars in Thrombosis and Hemostasis, Inhaltsverzeichnis

Semin Thromb Hemost 2013; 39(07): 779-793
DOI: 10.1055/s-0033-1354420

Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Pathogen Safety of Long-Term Treatments for Bleeding Disorders: (Un)Predictable Risks and Evolving Threats

Giovanni Di Minno

¹Department of Clinical Medicine and Surgery, Regional Reference Centre for Coagulation Disorders, Napoli, Italy

,

Mariana Canaro

²Department of Hemostasis and Thrombosis, Son Espases Universitary Hospital, Palma de Mallorca, Spain

,

James W. Ironside

³National Creutzfeldt-Jakob Disease Surveillance Unit, School of Molecular and Clinical Medicine, University of Edinburgh, Western General Hospital, Edinburgh, United Kingdom

,

David Navarro

⁴Department of Microbiology, Microbiology Service, Hospital Clínico Universitario, School of Medicine, University of Valencia, Valencia, Spain

,

Carlo F. Perno

⁵Department of Experimental Medicine and Surgery, University of Rome Tor Vergata, Rome, Italy

,

Andreas Tiede

⁶Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany

,

Lutz Guertler

⁷Max von Pettenkofer Institute for Hygiene and Medical Microbiology, University of München, Munich, Germany

› Institutsangaben

Abstract

Substantial improvements in the safety of blood and plasma products for the management of bleeding disorders have been achieved in recent decades. This has led some clinicians to believe that the infectious threat is over and that inhibitor formation is the foremost complication of hemophilia therapy. On the contrary, elimination of all microbes from blood is difficult, potentially impossible, and there are always threats from emerging pathogens. The risk of infection transmission is also increasing due to greater exposure to products, increasing prophylaxis and high-dose regimens for immune tolerance, and longevity of hemophilia patients. Current products can be considered “reasonably safe,” but pathogen testing is not all-inclusive, and manufacturing and purification techniques are often not standardized. Although safer nonplasma-derived products are widely used, they are not available for all bleeding disorders, and so there is an ongoing need for plasma-derived products. This review will discuss the evolving risk from emerging pathogens in the context of the issues described. Reducing the risk from emerging infections requires global collaboration to devise ways to monitor and continue to improve blood safety.

Keywords

bleeding disorders - blood safety - emerging infectious agent(s)

Volltext

Referenzen

References
1 Srivastava A, Brewer AK, Mauser-Bunschoten EP , et al; Treatment Guidelines Working Group on Behalf of The World Federation of Hemophilia. Guidelines for the management of hemophilia. Haemophilia 2013; 19 (1) e1-e47
2 Miller CH, Benson J, Ellingsen D , et al; Hemophilia Inhibitor Research Study Investigators. F8 and F9 mutations in US haemophilia patients: correlation with history of inhibitor and race/ethnicity. Haemophilia 2012; 18 (3) 375-382
3 Schwarz J, Astermark J, Menius ED , et al; Hemophilia Inhibitor Genetics Study Combined Cohort. F8 haplotype and inhibitor risk: results from the Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort. Haemophilia 2013; 19 (1) 113-118
4 Lippi G, Favaloro EJ, Montagnana M, Manzato F, Guidi GC, Franchini M. Inherited and acquired factor V deficiency. Blood Coagul Fibrinolysis 2011; 22 (3) 160-166
5 Kessler CM. New perspectives in hemophilia treatment. Hematology (Am Soc Hematol Educ Program) 2005; 429-435
6 Brooker M. Registry of Clotting Factor Concentrates. 9th ed. Montreal, Quebec, Canada: World Federation of Hemophilia (WFH); ; 2012. http://www1.wfh.org/publications/files/pdf-1227.pdf . Accessed May 1, 2013
7 Franchini M, Mannucci PM. Past, present and future of hemophilia: a narrative review. Orphanet J Rare Dis 2012; 7 (1) 24
8 Tagliaferri A, Rivolta GF, Iorio A , et al; Italian Association of Hemophilia Centers. Mortality and causes of death in Italian persons with haemophilia, 1990-2007. Haemophilia 2010; 16 (3) 437-446
9 Chorba TL, Holman RC, Clarke MJ, Evatt BL. Effects of HIV infection on age and cause of death for persons with hemophilia A in the United States. Am J Hematol 2001; 66 (4) 229-240
10 Plug I, Van Der Bom JG, Peters M , et al. Mortality and causes of death in patients with hemophilia, 1992-2001: a prospective cohort study. J Thromb Haemost 2006; 4 (3) 510-516
11 Darby SC, Kan SW, Spooner RJ , et al; UK Haemophilia Centre Doctors' Organisation. The impact of HIV on mortality rates in the complete UK haemophilia population. AIDS 2004; 18 (3) 525-533
12 Fransen van de Putte DE, Fischer K, Pulles AE , et al. Non-fatal cardiovascular disease, malignancies, and other co-morbidity in adult haemophilia patients. Thromb Res 2012; 130 (2) 157-162
13 Yee TT, Lee CA. Transfusion-transmitted infection in hemophilia in developing countries. Semin Thromb Hemost 2005; 31 (5) 527-537
14 Branch AD, Van Natta ML, Vachon ML, Dieterich DT, Meinert CL, Jabs DA ; Studies of the Ocular Complications of AIDS Research Group. Mortality in hepatitis C virus-infected patients with a diagnosis of AIDS in the era of combination antiretroviral therapy. Clin Infect Dis 2012; 55 (1) 137-144
15 Franchini M, Tagliaferri A, Mengoli C, Cruciani M. Cumulative inhibitor incidence in previously untreated patients with severe hemophilia A treated with plasma-derived versus recombinant factor VIII concentrates: a critical systematic review. Crit Rev Oncol Hematol 2012; 81 (1) 82-93
16 Goedert JJ, Chen BE, Preiss L, Aledort LM, Rosenberg PS. Reconstruction of the hepatitis C virus epidemic in the US hemophilia population, 1940-1990. Am J Epidemiol 2007; 165 (12) 1443-1453
17 Svicher V, Cento V, Bernassola M , et al. Novel HBsAg markers tightly correlate with occult HBV infection and strongly affect HBsAg detection. Antiviral Res 2012; 93 (1) 86-93
18 Powell JS. Recombinant factor VIII in the management of hemophilia A: current use and future promise. Ther Clin Risk Manag 2009; 5 (2) 391-402
19 Sharma S, Sharma P, Tyler LN. Transfusion of blood and blood products: indications and complications. Am Fam Physician 2011; 83 (6) 719-724
20 Wagner S. Transfusion-related bacterial sepsis. Curr Opin Hematol 1997; 4 (6) 464-469
21 Pittman M. A study of bacteria implicated in transfusion reactions and of bacteria isolated from blood products. J Lab Clin Med 1953; 42 (2) 273-288
22 Goel R, Krishnamurti L. Mortality, health care utilization and associated diagnoses in hospitalized patients with haemophilia in the United States: first reported nationwide estimates. Haemophilia 2012; 18 (5) 688-692
23 Rothschild C, D'Oiron R, Borel-Derlon A , et al. Use of Haemate(®) P as immune tolerance induction in patients with severe haemophilia A who failed previous induction attempts: a multicentre observational study. Haemophilia 2013; 19 (2) 281-286
24 DiMichele D. Inhibitor development in haemophilia B: an orphan disease in need of attention. Br J Haematol 2007; 138 (3) 305-315
25 Ghosh K, Shetty S. Immune response to FVIII in hemophilia A: an overview of risk factors. Clin Rev Allergy Immunol 2009; 37 (2) 58-66
26 Makris M, Hay CR, Gringeri A, D'Oiron R. How I treat inhibitors in haemophilia. Haemophilia 2012; 18 (Suppl. 04) 48-53
27 Iorio A, Halimeh S, Holzhauer S , et al. Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost 2010; 8 (6) 1256-1265
28 Mannucci PM, Mancuso ME, Santagostino E. How we choose factor VIII to treat hemophilia. Blood 2012; 119 (18) 4108-4114
29 Ghio M, Contini P, Ottonello L , et al. Effect of clotting factors concentrates on lymphocyte and neutrophil function in vitro. Thromb Haemost 2003; 89 (2) 365-373
30 Franchini M. Plasma-derived versus recombinant Factor VIII concentrates for the treatment of haemophilia A: recombinant is better. Blood Transfus 2010; 8 (4) 292-296
31 Goodnough LT, Shander A, Brecher ME. Transfusion medicine: looking to the future. Lancet 2003; 361 (9352) 161-169
32 Ceccherini-Nelli L, Filipponi F, Mosca F, Campa M. The risk of contracting an infectious disease from blood transfusion. Transplant Proc 2004; 36 (3) 680-682
33 Glynn SA, Busch MP, Dodd RY , et al. Emerging infectious agents and the nation's blood supply: responding to potential threats in the 21st century. Transfusion 2013; 53 (2) 438-454
34 Trimble SR, Parker CS, Grant AM, Soucie JM, Reyes N. Assessing emerging infectious threats to blood safety for the blood disorders community. Am J Prev Med 2010; 38 (4, Suppl): S468-S474
35 Webby R, Hoffmann E, Webster R. Molecular constraints to interspecies transmission of viral pathogens. Nat Med 2004; 10 (12, Suppl): S77-S81
36 Lim JK, Murphy PM. Chemokine control of West Nile virus infection. Exp Cell Res 2011; 317 (5) 569-574
37 Sahlberg AS, Granfors K, Penttinen MA. HLA-B27 and host-pathogen interaction. Adv Exp Med Biol 2009; 649: 235-244
38 Kaur P, Basu S. Transfusion-transmitted infections: existing and emerging pathogens. J Postgrad Med 2005; 51 (2) 146-151
39 Wolfe ND, Dunavan CP, Diamond J. Origins of major human infectious diseases. Nature 2007; 447 (7142) 279-283
40 Zappa A, Amendola A, Romanò L, Zanetti A. Emerging and re-emerging viruses in the era of globalisation. Blood Transfus 2009; 7 (3) 167-171
41 Woolhouse ME, Haydon DT, Antia R. Emerging pathogens: the epidemiology and evolution of species jumps. Trends Ecol Evol 2005; 20 (5) 238-244
42 Chamberland ME. Emerging infectious agents: do they pose a risk to the safety of transfused blood and blood products?. Clin Infect Dis 2002; 34 (6) 797-805
43 Grillberger L, Kreil TR, Nasr S, Reiter M. Emerging trends in plasma-free manufacturing of recombinant protein therapeutics expressed in mammalian cells. Biotechnol J 2009; 4 (2) 186-201
44 Dodd RY, Leiby DA. Emerging infectious threats to the blood supply. Annu Rev Med 2004; 55: 191-207
45 Stramer SL, Hollinger FB, Katz LM , et al. Emerging infectious disease agents and their potential threat to transfusion safety. Transfusion 2009; 49 (Suppl. 02) 1S-29S
46 Smolinski S, Hamburg MA, Lederberg J. Microbial threats to health: emergence, detection, and response. Washington, DC: National Academies Press; 2003
47 Hughes RA, Swan AV, van Doorn PA. Intravenous immunoglobulin for Guillain-Barré syndrome. Cochrane Database Syst Rev 2012; 7: CD002063
48 Ludlam CA, Powderly WG, Bozzette S , et al. Clinical perspectives of emerging pathogens in bleeding disorders. Lancet 2006; 367 (9506) 252-261
49 Grazzini G, Liumbruno GM, Pupella S , et al. West Nile virus in Italy: a further threat to blood safety, a further challenge to the blood system. Blood Transfus 2008; 6 (4) 235-237
50 Ludlam CA, Turner ML. Managing the risk of transmission of variant Creutzfeldt Jakob disease by blood products. Br J Haematol 2006; 132 (1) 13-24
51 Liumbruno GM, Calteri D, Petropulacos K , et al. The Chikungunya epidemic in Italy and its repercussion on the blood system. Blood Transfus 2008; 6 (4) 199-210
52 Sheldon J, Soriano V. Hepatitis B virus escape mutants induced by antiviral therapy. J Antimicrob Chemother 2008; 61 (4) 766-768
53 Stramer SL, Zou S, Notari EP , et al. Blood donation screening for hepatitis B virus markers in the era of nucleic acid testing: are all tests of value?. Transfusion 2012; 52 (2) 440-446
54 Sharp CP, Lail A, Donfield S, Gomperts ED, Simmonds P. Virologic and clinical features of primary infection with human parvovirus 4 in subjects with hemophilia: frequent transmission by virally inactivated clotting factor concentrates. Transfusion 2012; 52 (7) 1482-1489
55 Soucie JM, De Staercke C, Monahan PE , et al; the US Hemophilia Treatment Center Network. Evidence for the transmission of parvovirus B19 in patients with bleeding disorders treated with plasma-derived factor concentrates in the era of nucleic acid test screening. Transfusion 2013; 53 (6) 1217-1225
56 Brown P. Creutzfeldt-Jakob disease: reflections on the risk from blood product therapy. Haemophilia 2007; 13 (Suppl. 05) 33-40
57 Hewitt PE, Llewelyn CA, Mackenzie J, Will RG. Creutzfeldt-Jakob disease and blood transfusion: results of the UK Transfusion Medicine Epidemiolgoical Review Study. Vox Sang 2006; 91 (3) 221-230
58 Wroe SJ, Pal S, Siddique D , et al. Clinical presentation and pre-mortem diagnosis of variant Creutzfeldt-Jakob disease associated with blood transfusion: a case report. Lancet 2006; 368 (9552) 2061-2067
59 Peden A, McCardle L, Head MW , et al. Variant CJD infection in the spleen of a neurologically asymptomatic UK adult patient with haemophilia. Haemophilia 2010; 16 (2) 296-304
60 Allain JP, Stramer SL, Carneiro-Proietti AB , et al. Transfusion-transmitted infectious diseases. Biologicals 2009; 37 (2) 71-77
61 Stein J, Besley J, Brook C , et al. Risk-based decision-making for blood safety: preliminary report of a consensus conference. Vox Sang 2011; 101 (4) 277-281
62 Bolton-Maggs PH. Optimal haemophilia care versus the reality. Br J Haematol 2006; 132 (6) 671-682
63 Evatt BL, Austin H, Leon G, Ruiz-Sáez A, De Bosch N. Haemophilia therapy: assessing the cumulative risk of HIV exposure by cryoprecipitate. Haemophilia 1999; 5 (5) 295-300
64 Spannagl M, Joch Ch, Heindl B. [Safety of plasma-derived factor concentrates: an example of pharmacovigilance with fibrinogen and factor XIII-concentrate]. Hamostaseologie 2006; 26 (3) (Suppl. 01) S36-S40
65 Lusher JM, Scharrer I. Evolution of recombinant factor VIII safety: KOGENATE and Kogenate FS/Bayer. Int J Hematol 2009; 90 (4) 446-454
66 Mackay GA, Knight RS, Ironside JW. The molecular epidemiology of variant CJD. Int J Mol Epidemiol Genet 2011; 2 (3) 217-227
67 Bishop MT, Pennington C, Heath CA, Will RG, Knight RS. PRNP variation in UK sporadic and variant Creutzfeldt Jakob disease highlights genetic risk factors and a novel non-synonymous polymorphism. BMC Med Genet 2009; 10: 146
68 Mead S, Poulter M, Uphill J , et al. Genetic risk factors for variant Creutzfeldt-Jakob disease: a genome-wide association study. Lancet Neurol 2009; 8 (1) 57-66
69 Bishop MT, Kovacs GG, Sanchez-Juan P, Knight RS. Cathepsin D SNP associated with increased risk of variant Creutzfeldt-Jakob disease. BMC Med Genet 2008; 9: 31
70 Franchini M, Tagliaferri A, Mannucci PM. The management of hemophilia in elderly patients. Clin Interv Aging 2007; 2 (3) 361-368
71 Mannucci PM, Schutgens RE, Santagostino E, Mauser-Bunschoten EP. How I treat age-related morbidities in elderly persons with hemophilia. Blood 2009; 114 (26) 5256-5263
72 Knight R, Stanley S, Wong M, Dolan G. Hemophilia therapy and blood-borne pathogen risk. Semin Thromb Hemost 2006; 32 (Suppl. 02) 3-9
73 Tapper ML. Emerging viral diseases and infectious disease risks. Haemophilia 2006; 12 (Suppl. 01) 3-7 , discussion 26–28
74 Grard G, Fair JN, Lee D , et al. A novel rhabdovirus associated with acute hemorrhagic fever in central Africa. PLoS Pathog 2012; 8 (9) e1002924
75 Health Protection Agency. Summary results of the second national survey of abnormal prion protein prevalence in archived appendix specimens. Health Protection Report 2012. http://www.hpa.org.uk/hpr/archives/2012/news3212.htm#bnrmlprn. Published August 2012. Accessed 5 June 2013.
76 Katz LM. Transfusion safety in the 21st century: how tightly should the blood community close the window(s)?. J Infect Dis 2008; 198 (9) 1258-1261