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REVIEW

Current Management of the Hemophilic Child: A Demanding Interlocutor. Quality of Life and Adequate Cost-Efficacy Analysis

, , , , &
Pages 687-702 | Received 18 Apr 2014, Accepted 31 May 2014, Published online: 09 Jul 2014

REFERENCES

  • Franchini M, Mannucci PM. Past, present and future of haemophilia: a narrative review. Orphanet J Rare Dis. 2012;7:24.
  • Franchini M, Lippi G. Recombinant factor VIII concentrates. Semin Thromb Hemost. 2010;36:493–497.
  • Mannucci PM. Back to the future: a recent history of haemophilia treatment. Haemophilia. 2008;14:10–18.
  • Franchini M, Coppola A, Molinari AC, Forum on: the role of recombinant factor VIII in children with severe haemophilia A. Haemophilia. 2009;15:578–586.
  • Coppola A, Tagliaferri A, Di Capua M, Franchini M. Prophylaxis in children with haemophilia: evidence-based achievements, old and new challenges. Semin Thromb Hemost. 2012;38:79–94.
  • Giordano P, Franchini M, Lassandro G, Issues in pediatric haemophilia care. Ital J Pediatr. 2013;39:24.
  • Arcieri R, Molinari AC, Farace S, Present and future challenges in the treatment of haemophilia: the patient's perspective. Blood Transfus. 2013;4:s82–85.
  • Tagliaferri A, Rivolta GF, Iorio A, Mortality and causes of death in Italian persons with haemophilia, 1990–2007. Haemophilia. 2010;16:437–446.
  • Peyvandi F, Garagiola I, Seregni S. Future of factor replacement treatment. J Thromb Haeamost. 2013;11:84–98.
  • Chuah MK, Nair N, van den Driessche T. Recent progress in gene therapy for hemophilia. Hum Gene Ther. 2012;23:557–565.
  • van Dijk K, Fischer K, van der Bom JG, Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed. Haemophilia. 2005;11:438–443.
  • Gringeri A, Lundin B, Mackensen SV, A randomized clinical trial of prophylaxis in children with haemophilia A (the ESPRIT Study). J Thromb Haemost. 2011;9:700–710.
  • Rosendaal G, Lafeber FP. Pathogenesis of haemophilic arthropathy. Haemophilia. 2006;12:117–121.
  • Aledort L, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophilics. The Orthopaedic Outcome Study Group. J Intern Med. 1994;236:391–399.
  • Fischer K, van der Bom JG, Mauser-Bunschoten EP, The effects of postponing prophylactic treatment on long-term outcome in patients with severe haemophilia. Blood. 2002;99:2337–2341.
  • Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992;232:25–32.
  • Srivastava A, Brewer AK, Mauser-Bunschoten EP, Treatment guidelines working group on behalf of the world federation of hemophilia. Guidelines for the management of haemophilia. Haemophilia Haemophilia. 2013;19:e1–47.
  • Donadel-Claeyssens S. Current co-ordinated activities of the PEDNET (European Paediatric Network for Haemophilia Management). Haemophilia. 2006;12:124–127.
  • Berntorp E, Boulyjenkov V, Brettler D, Modern treatment of haemophilia. Bull World Health Organ. 1995;73:691–701.
  • Manco-Johnson MJ, Abshire TC, Shapiro AD, Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357:535–544.
  • Colvin BT, Astermark J, Fischer K, European principles of haemophilia care. Haemophilia. 2008;14:361–374.
  • Richards M, Williams M, Chalmers E, A United Kingdom Haemophilia Centre Doctors’ Organization guideline approved by the British Committee for Standards in Haematology: guideline on the use of prophylactic factor VIII concentrate in children and adults with severe haemophilia A. Br J Haematol. 2010;149:498–507.
  • Iorio A, Marchesini E, Marcucci M, Clotting factor concentrates given to prevent bleeding and bleeding-related complications in people with hemophilia A or B. Cochrane Database Syst Rev. 2011;9:CD003429.
  • Fischer K, Astermark J, van der Bom JG, Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen. Haemophilia. 2002;8:753–760.
  • van den Berg HM, Fischer K, van der Bom JG. Comparing outcomes of different treatment regimens for severe haemophilia. Haemophilia. 2003;9:27–31.
  • Wu R, Luke KH, Poon MC, Low dose secondary prophylaxis reduces joint bleeding in severe and moderate haemophilic children: a pilot study in China. Haemophilia. 2011;17:70–74.
  • Celkan T, Özdemir N. Reduced early prophylaxis of children with haemophilia in a developing country, Turkey. Haemophilia. 2011;17:e840–e841.
  • Geraghty S, Dunkley T, Harrington C, Practice patterns in haemophilia A therapy—global progress towards optimal care. Haemophilia. 2006;12:75–81.
  • Petrini P. Identifying and overcoming barriers to prophylaxis in the management of haemophilia. Haemophilia. 2007;13:16–22.
  • Valentino LA, Ewenstein B, Navickis RJ, Wilkes MM. Central venous devices in haemophilia. Haemophilia. 2004;10:134–146.
  • Ljung R. The risk associated with indwelling catheters in children with haemophilia. Br J Haematol. 2007;138:580–586.
  • Mancuso ME, Mannucci PM, Sartori A, Feasibility of prophylaxis and immune tolerance induction regimens in haemophilic children using fully implantable central venous catheters. Br J Haematol. 2008;141:689–695.
  • Yeoh ZH, Furmedge J, Ekert J, Central venous access device-related infections in patients with haemophilia. J Paediatr Child Health. 2013;49:242–245.
  • Coppola A, Franchini M, Makris M, Thrombotic adverse events to coagulation factor concentrates for treatment of patients with haemophilia and von Willebrand disease: a systematic review of prospective studies. Haemophilia Haemophilia. 2012;18:e173–e187.
  • Mancuso M, Berardinelli L, Beretta C, Improved treatment feasibility in children with hemophilia using arteriovenous fistulae: the results after seven years of follow-up. Haematologica. 2009;94:687–692.
  • Bui S, Babre F, Hauchecorne S, Intravenous peripherally-inserted central catheters for antibiotic therapy in children with cystic fibrosis. J Cyst Fibros. 2009;8:326–331.
  • Santoro C, Baldacci E, Mercanti C, Mazzucconi MG. Tailored vs. standard dose prophylaxis in children with haemophilia A. Semin Thromb Hemost. 2013;39:711–722.
  • Petrini P. What factors should influence the dosage and interval of prophylactic treatment in patients with severe haemophilia A and B? Haemophilia. 2001;7:99–102.
  • Feldman BM, Pai M, Rivard GE, Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost. 2006;4:1228–1236.
  • Xang MX, Blanchette VS, Pullenayegum E, Age at first joint bleed and bleeding severity in boys with severe hemophilia A: Canadian Hemophilia Primary Prophylaxis Study Group. J Thromb Haemost. 2011;9:1067–1069.
  • Carcao M, Chambost H, Ljung R. Devising a best approach to prophylaxis in boys with severe haemophilia: evaluation of current treatment strategies. Haemophilia. 2010;16:4–9.
  • Hilliard P, Zourikian N, Blanchette V, Musculoskeletal health of subjects with hemophilia A treated with tailored prophylaxis: Canadian Hemophilia Primary Prophylaxis (CHPS) Study. J Thromb Haemost. 2013;11:460–466.
  • Collins PW. Personalized prophylaxis. Haemophilia. 2012;18:131–135.
  • Collins PW, Blanchette VS, Fischer K, Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb. Haemost. 2009;7:413–420.
  • Björkman S, Folkesson A, Jönsson S. Pharmacokinetics and dose requirements of factor VIII over the age range 3–74 years: a population analysis based on 50 patients with long-term prophylactic treatment for haemophilia A. Eur J Clin Pharmacol. 2009;65:989–998.
  • Collins PW, Björkman S, Fischer K, Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemost. 2010;8:269–275.
  • Carlsson M, Berntorp E, Björkman S, Lindvall K. Improved cost-effectiveness by pharmacokinetic dosing of factor VIII in prophylactic treatment of hemophilia A. Haemophilia. 1997;3:96–101.
  • Valentino LA, Mamonov V, Hellmann A, Prophylaxis Study Group. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost. 2012;10:359–367.
  • Lindvall K, Astermark J, Björkman S, Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy. Haemophilia. 2012;18:855–859.
  • Biss TT, Chan AK, Blanchette VS, The use of prophylaxis in 2663 children and adults with haemophilia: results of the 2006 Canadian national haemophilia prophylaxis survey. Haemophilia. 2008;14:923–930.
  • Santagostino E, Fasulo MR. Hemophilia A and hemophilia B: different types of diseases? Semin Thromb Hemost. 2013;39:697–701.
  • Tarantino MD, Ye X, Bergstrom F, The impact of the economic downturn and health care reform on treatment decisions for haemophilia A: patient, caregiver and health care provider perspectives. Haemophilia. 2013;19:51–58.
  • Gringeri A, von Mackensen S, Auerswald G, Health status and health-related quality of life of children with haemophilia from six West European countries. Haemophilia. 2004;10:26–33.
  • Gringeri A, von Mackensen S. Quality of life in haemophilia. Haemophilia. 2008;14:19–25.
  • Risebrough N, Oh P, Blanchette V, Cost-utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on-demand therapy in young children with severe haemophilia A. Haemophilia. 2008;14:743–752
  • Miners AH. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia. 2009;15:881–887.
  • Colombo GL, Di Matteo S, Mancuso ME, Santagostino E. Cost-utility analysis of prophylaxis versus treatment on demand in severe hemophilia A. ClinicoEconomics and Outcome Res. 2011;3:55–61.
  • Young NL, Bradley CS, Wakefield CD, How well does the Canadian Haemophilia Outcomes-Kids’ Life Assessment Tool (CHO-KLAT) measure the quality of life of boys with haemophilia? Pediatr Blood Cancer. 2006;47:305–311.
  • Coppola A, Cerbone AM, Mancuso G, Confronting the psychological burden of haemophilia. Haemophilia. 2011;17:21–27.
  • De Moerloose P, Urbancik W, van den Berg HM, Richards M. A survey of adherence to haemophilia therapy in six European countries: results and recommendations. Haemophilia. 2008;14:931–938.
  • Hacker MR, Geraghty S, Manco-Johnson M. Haemophilia. 2001;7:392–396.
  • Shapiro AD, Donfield SM, Lynn HS, Defining the impact of haemophilia: the Academic Achievement in Children with Hemophilia Study. Pediatrics. 2001;108:E105.
  • Ross C, Goldenberg NA, Hund D, Manco-Johnson MJ. Athletic participation in severe hemophilia: bleeding and joint outcomes in children on prophylaxis. Pediatrics. 2009;124:1267–1272.
  • Gregory M, Boddington P, Dimond R, Family communication about haemophilia. Haemophilia. 2007;13:189–198.
  • Canclini M, Saviolo-Negrin N, Zanon E, Psychological aspects and coping in haemophilic patients: a case-control study. Haemophilia. 2003;9:619–624.
  • Franchini M, Mannucci PM. Inhibithors of propagation of coagulation (factors VIII, IX and XI): a review of current therapeutic practice. Br J Clin Pharmacol. 2011;72:553–562.
  • Franchini M, Coppola A, Tagliaferri A, Lippi G. FEIBA versus NovoSeven in Hemophilia Patients with Inhibitors. Semin Thromb Hemost 2013;39:772–778.
  • Leissinger C, Gringeri A, Antmen B, Anti-inhibitor coagulant complex prophylaxis in haemophilia with inhibitors. N Engl J Med. 2011;365:1684–1692.
  • Konkle BA, Ebbesen LS, Erhardtsen E, Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost. 2007;5:1904–1913.
  • Gringeri A, Mantovani LG, Scalone L, Mannucci PM. Cost of care and quality of life for patients with haemophilia complicated by inhibitors: the COCIS Study Group. Blood. 2003;102:2358–2363.
  • Scalone L, Mantovani LG, Mannucci PM, Gringeri A. Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors. Haemophilia. 2006;12:154–162.
  • Coppola A, Santoro C, Franchini M, Understanding inhibitor development in haemophilia A: towards clinical prediction and prevention strategies. Haemophilia. 2010;16:13–19.
  • Astermark J. Inhibitor development: patient-determined risk factors. Haemophilia. 2010;16:66–70.
  • Gouw SC, van den Berg HM, Oldenburg J, F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis. Blood. 2012;119:2922–2934.
  • Astermark J, Donfield SM, Gomperts ED, The polygenic nature of inhibitors in haemophilia A: results from the Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort. Blood. 2013;121:1446–1454.
  • Matzinger P. The danger model: a renewed sense of self. Science. 2002;8:76–82.
  • Gouw SC, van der Bom JG, van den Berg HM. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood. 2007;109:4648–4654.
  • Maclean PS, Richards M, Williams M, Treatment related factors and inhibitor development in children with severe haemophilia A. Haemophilia. 2011; 17:282–287.
  • Gouw SC, van den Berg HM, Fischer K, Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013;121:4046–4055.
  • Coppola A, Tagliaferri A, Franchini M. Searching for the role of primary prophylaxis in preventing inhibitor development in hemophilia A. J Thromb Haemost. 2012;10:1954–1960.
  • Santagostino E, Mancuso ME, Rocino A, Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study. Br J Haematol. 2005;130:422–427.
  • Auerswald G, Bidlingmaier C, Kurnik K. Early prophylaxis/FVIII tolerization regimen that avoids immunological danger signals is still effective in minimizing FVIII inhibitor developments in previously untreated patients–long-term follow-up and continuing experience. Haemophilia. 2012;18:e18–e20.
  • Goudemand J, Rothschild C, Demiguel V, Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe haemophilia A. Blood. 2006;107:46–51.
  • Mancuso ME, Mannucci PM, Rocino A, Source and purity of factor VIII products as risk factors for inhibitor development in patients with hemophilia A. J Thromb Haemost. 2012;10:781–790.
  • Wight J, Paisley S The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia. 2003;9:418–435.
  • Iorio A, Halimeh S, Holzhauer S, 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;8:1256–1265.
  • Gouw SC, van der Bom JG, Ljung R, Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013;368:231–239.
  • Franchini M, Coppola C, Rocino A, Systematic review of the role of FVIII concentrates in inhibitor development in previously untreated patients with severe hemophilia A: a 2013 update. Semin Thromb Hemost. 2013;39:752–766.
  • Mannucci PM, Gringeri A, Peyvandi F, Santagostino E. Factor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasma-product exposed toddlers). Haemophilia. 2007;13:65–68.
  • Franchini M, Coppola A, Rocino A, Perceived challenges and attitudes to regimen and product selection from Italian haemophilia treaters. The 2013 AICE survey. Haemophilia. 2014;20:128–135.
  • Collins PW, Chalmers E, Hart DP, Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition). UK Haemophilia Centre Doctors Organization. Br J Haematol 2013; 160:153–170.
  • Astermark J, Morado M, Rocino A, Current European practice in immune tolerance induction therapy in patients with haemophilia and inhibitors. Haemophilia. 2006;12:363–371.
  • DiMichele D, Hoots WK, Pipe SW, International workshop on immune tolerance induction: consensus recommendations. Haemophilia. 2007;13:1–22.
  • Coppola A, Di Minno MN, Santagostino E. Optimizing management of immune tolerance induction in patients with severe haemophilia A and inhibitors: towards evidence-based approaches. Br J Haematol. 2010;150:515–528.
  • Hay CR, DiMichele DM; International Immune Tolerance Study. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood. 2012;119:1335–1344.
  • Coppola A, Margaglione M, Santagostino E, Factor VIII gene (F8) mutations as predictors of outcome in immune tolerance induction of hemophilia A patients with high-responding inhibitors. J Thromb Haemost. 2009;7:1809–1815.
  • Auerswald G, von Depka Prondzinski M, Ehlken B, Treatment patterns and cost-of-illness of severe haemophilia in patients with inhibitors in Germany. Haemophilia. 2004;10:499–508.
  • Colowick AB, Bohn RL, Avorn J, Ewenstein BM. Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper. Blood. 2000;96:1698–1702.
  • Di Minno MND, Di Minno G, Di Capua M, Cost of care of haemophilia with inhibitors. Haemophilia. 2010;16:e190–e201.
  • Kaufman RJ, Powell JS. Molecular approaches for improved clotting factors for haemophilia. Blood. 2013;122:3568–3574.
  • High KA. The gene therapy journey for hemophilia: are we there yet? Hematology. Am. Soc. Hematol. Educ. Program. 2012; 2012:375–381.
  • Nathwani AC, Tuddenham EG, Rangarajan S, Adenovirus-associated virus vector-mediated gene transfer in haemophilia B. N Engl J Med. 2011;365:2357–2365.
  • Mount JD, Herzog RW, Tillson DM, Sustained phenotypic correction of haemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood.2002;99:2670–2676.
  • McIntosh J, Lenting PJ, Rosales C, Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood. 2013;121:3335–3344.

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