Safety and efficacy of secondary prophylactic in Patients with severe hemophilia type A and B: A Systematic review and meta-analysis study
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Abstract
Context: Patients with hemophilia recieves coagulation factor replacement for lifetime. In iran, on-demand treatment method is used as standard. Clinical studies have shown significant improvements in clinical and economic outcomes as a result of the use of prophylaxis compared with other therapies. The aim of this study was to evaluate safety and efficacy of prophylaxis in patients with severe hemophilia type A and B.
Evidence Acquisition: This is a systematic review and meta-analysis in order to evaluate the safety and efficacy of prophylaxis treatment in patients with severe hemophilia. To this response, all clinical trials, cohorts, and case-control studies which have been investigated, published 1970 to sep 2017 and the results have been analyzed in STATA.
Results: 1439 studies were found in primary search and 17 of them had inclusion criteria. The mean annual bleeding rate in prophylays treatment was 2.8 times per person/year. This study also showed that in prophylaxis, the average incidence of adverse effects was 0.13 cases, and the severe adverse effects was 0.06 cases per person/year.
Conclusion: The analysis of the studies entered in this evaluation showed that the adverse effects were significantly lower in patients treated with prophylaxis than in patients treated with on-demand treatment. This difference was observed in severe adverse effect but it was not statistically significant and this shows that prophilaxis is safer than on-demand method. The lower annual bleeding rate in prophylaxis compared with the on-demand treatment method is also a sign of the effectiveness of prophilaxis.
1. Salinas-Escudero G, Galindo-Suarez RM, Rely K, Carrillo-Vega MF, Mucino-Ortega E. Cost-effectiveness analysis of prophylaxis vs "on demand" approach in the management in children with hemophilia A in Mexico (Provisional abstract). Boletin Medico del Hospital Infantil de Mexico [Internet]. 2013; 70(4):290-8.
2. Pipe S. Antihemophilic factor (recombinant) plasma/albumin-free method for the management and prevention of bleeding episodes in patients with hemophilia A. Biologics: targets & therapy. 2009; 3:117.
3. MA GC, CHANG SP, CHEN M, KUO SJ, CHANG CS, SHEN MC. The spectrum of the factor 8 (F8) defects in Taiwanese patients with haemophilia A. Haemophilia. 2008;14(4):787-95.
4. Qavidel A. results of the census plan for hemophilic patients in Iran. 2016[Persian].
5. Chen S-L. Economic costs of hemophilia and the impact of prophylactic treatment on patient management. Am J Manag Care. 2016;22(5):126-33.
6. Khosravi A, Aghamohamadi S, E. k. Mortality Profile in the Islamic Republic of Iran 2015(20 leading cause of death by sex and age group). Ministry of Health and Medical Education. 2015:1-17 [Persian].
7. Rodriguez‐Merchan E. Aspects of current management: orthopaedic surgery in haemophilia. Haemophilia. 2012;18(1):8-16.
8. Khanali Mojen L, Abed Saeedi Z, Eshghi P, Farahani H, Abdollah Gorgi F, Habibpanah B, et al. Comparison of quality of life between hemophilic children receiving prophylaxis and on demand therapy in Tehran. QUARTERLY FACULTY OF NURSING OF MIDWIFERY QUARTERLY. 2012;22(77):39-45 [Persian].
9. Coppola A, Cerbone A, Mancuso G, Mansueto M, Mazzini C, Zanon E. Confronting the psychological burden of haemophilia. Haemophilia. 2011;17(1):21-7.
10. Gringeri A, Von Mackensen S, Auerswald G, Bullinger M, Garrido RP, Kellermann E, et al. Health status and health‐related quality of life of children with haemophilia from six West European countries. Haemophilia. 2004;10(1):26-33.
11. Miners A. Revisiting the cost‐effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia. 2009;15(4):881-7.
12. Risebrough N, Oh P, Blanchette V, Curtin J, Hitzler J, Feldman B. Cost‐utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on‐demand therapy in young children with severe haemophilia A. Haemophilia. 2008;14(4):743-52.
13. Liesner R, Khair K, Hann I. The impact of prophylactic treatment on children with severe haemophilia. British Journal of Haematology. 1996;92(4):973-8.
14. Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. New England Journal of Medicine. 2007;357(6):535-44.
15. Smith PS, Teutsch SM, Shaffer PA, Rolka H, Evatt B. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. The Journal of pediatrics. 1996;129(3):424-31.
16. Szucs T, Öffner A, Schramm W. Socioeconomic impact of haemophilia care: results of a pilot study. Haemophilia. 1996;2(4):211-7.
17. Van Den Berg H, Fischer K, Van Der Bom J. Comparing outcomes of different treatment regimens for severe haemophilia. Haemophilia. 2003;9(1):27-31.
18. Miners A, Sabin C, Tolley K, Lee C. Primary prophylaxis for individuals with severe haemophilia: how many hospital visits could treatment prevent? Journal of internal medicine. 2000;247(4):493-9.
19. Miners A, Sabin C, Tolley K, Jenkinson C, Kind P, Lee C. Assessing health-related quality-of-life in individuals with haemophilia. Haemophilia: the official journal of the World Federation of Hemophilia. 1999;5(6):378-85.
20. Unim B, Veneziano MA, Boccia A, Ricciardi W, La Torre G. Haemophilia a: Pharmacoeconomic review of prophylaxis treatment versus on-demand. Scientific World Journal. 2015.
21. Khoriaty R, Taher A, Inati A, Lee C. A comparison between prophylaxis and on demand treatment for severe haemophilia. Clinical and laboratory haematology. 2005 Oct;27(5):320-3.
22. Fischer K, van der Bom JG, Molho P, Negrier C, Mauser-Bunschoten EP, Roosendaal G, et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia. 2002 Nov;8(6):745-52.
23. Collins P, Faradji A, Morfini M, Enriquez M, Schwartz L. Efficacy and safety of secondary prophylactic vs. on‐demand sucrose‐formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13‐month crossover study. Journal of Thrombosis and Haemostasis. 2010;8(1):83-9.
24. Fischer K, Van Den Berg M. Prophylaxis for severe haemophilia: Clinical and economical issues. Haemophilia. 2003;9(4):376-81.
25. Gringeri A, Lundin B, Mackensen S, Mantovani L, Mannucci P. A randomized clinical trial on prophylaxis Vs. episodic treatment in children with haemophilia A: the ESPRIT study [abstract]. Haemophilia. 2010; 16(4):29.
26. Valentino LA, Mamonov V, Hellmann A, Quon DV, Chybicka A, Schroth P, et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. Journal of thrombosis and haemostasis : JTH. 2012 Mar;10(3):359-67.
27. Manco-Johnson MJ, Kempton CL, Reding MT, Lissitchkov T, Goranov S, Gercheva L, et al. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). Journal of thrombosis and haemostasis : JTH. 2013; 11(6):1119-27.
28. Kavakli K, Yang R, Rusen L, Beckmann H, Tseneklidou-Stoeter D, Maas Enriquez M. Prophylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: Results from a randomized trial (LEOPOLD II). Journal of thrombosis and haemostasis : JTH. 2015; 13(3):360-9.
29. Antunes SV, Tangada S, Stasyshyn O, Mamonov V, Phillips J, Guzman-Becerra N, et al. Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors. Haemophilia [Internet]. 2013; 20(1):65-72.
30. Zhao Y, Xiao J, Yang R, Wu R, Hu Y, Beckmann H, et al. Efficacy of standard prophylaxis versus on-demand treatment with bayer's sucrose-formulated recombinant FVIII (rFVIII-FS) in Chinese children with severe hemophilia A. Pediatric Hematology and Oncology. 2017:1-11.
31. Bolton-Maggs PH, Pasi KJ. Haemophilias a and b. The lancet. 2003;361(9371):1801-9.
32. Panicker J, Warrier I, Thomas R, Lusher J. The overall effectiveness of prophylaxis in severe haemophilia. Haemophilia. 2003;9(3):272-8.
2. Pipe S. Antihemophilic factor (recombinant) plasma/albumin-free method for the management and prevention of bleeding episodes in patients with hemophilia A. Biologics: targets & therapy. 2009; 3:117.
3. MA GC, CHANG SP, CHEN M, KUO SJ, CHANG CS, SHEN MC. The spectrum of the factor 8 (F8) defects in Taiwanese patients with haemophilia A. Haemophilia. 2008;14(4):787-95.
4. Qavidel A. results of the census plan for hemophilic patients in Iran. 2016[Persian].
5. Chen S-L. Economic costs of hemophilia and the impact of prophylactic treatment on patient management. Am J Manag Care. 2016;22(5):126-33.
6. Khosravi A, Aghamohamadi S, E. k. Mortality Profile in the Islamic Republic of Iran 2015(20 leading cause of death by sex and age group). Ministry of Health and Medical Education. 2015:1-17 [Persian].
7. Rodriguez‐Merchan E. Aspects of current management: orthopaedic surgery in haemophilia. Haemophilia. 2012;18(1):8-16.
8. Khanali Mojen L, Abed Saeedi Z, Eshghi P, Farahani H, Abdollah Gorgi F, Habibpanah B, et al. Comparison of quality of life between hemophilic children receiving prophylaxis and on demand therapy in Tehran. QUARTERLY FACULTY OF NURSING OF MIDWIFERY QUARTERLY. 2012;22(77):39-45 [Persian].
9. Coppola A, Cerbone A, Mancuso G, Mansueto M, Mazzini C, Zanon E. Confronting the psychological burden of haemophilia. Haemophilia. 2011;17(1):21-7.
10. Gringeri A, Von Mackensen S, Auerswald G, Bullinger M, Garrido RP, Kellermann E, et al. Health status and health‐related quality of life of children with haemophilia from six West European countries. Haemophilia. 2004;10(1):26-33.
11. Miners A. Revisiting the cost‐effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia. 2009;15(4):881-7.
12. Risebrough N, Oh P, Blanchette V, Curtin J, Hitzler J, Feldman B. Cost‐utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on‐demand therapy in young children with severe haemophilia A. Haemophilia. 2008;14(4):743-52.
13. Liesner R, Khair K, Hann I. The impact of prophylactic treatment on children with severe haemophilia. British Journal of Haematology. 1996;92(4):973-8.
14. Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. New England Journal of Medicine. 2007;357(6):535-44.
15. Smith PS, Teutsch SM, Shaffer PA, Rolka H, Evatt B. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. The Journal of pediatrics. 1996;129(3):424-31.
16. Szucs T, Öffner A, Schramm W. Socioeconomic impact of haemophilia care: results of a pilot study. Haemophilia. 1996;2(4):211-7.
17. Van Den Berg H, Fischer K, Van Der Bom J. Comparing outcomes of different treatment regimens for severe haemophilia. Haemophilia. 2003;9(1):27-31.
18. Miners A, Sabin C, Tolley K, Lee C. Primary prophylaxis for individuals with severe haemophilia: how many hospital visits could treatment prevent? Journal of internal medicine. 2000;247(4):493-9.
19. Miners A, Sabin C, Tolley K, Jenkinson C, Kind P, Lee C. Assessing health-related quality-of-life in individuals with haemophilia. Haemophilia: the official journal of the World Federation of Hemophilia. 1999;5(6):378-85.
20. Unim B, Veneziano MA, Boccia A, Ricciardi W, La Torre G. Haemophilia a: Pharmacoeconomic review of prophylaxis treatment versus on-demand. Scientific World Journal. 2015.
21. Khoriaty R, Taher A, Inati A, Lee C. A comparison between prophylaxis and on demand treatment for severe haemophilia. Clinical and laboratory haematology. 2005 Oct;27(5):320-3.
22. Fischer K, van der Bom JG, Molho P, Negrier C, Mauser-Bunschoten EP, Roosendaal G, et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia. 2002 Nov;8(6):745-52.
23. Collins P, Faradji A, Morfini M, Enriquez M, Schwartz L. Efficacy and safety of secondary prophylactic vs. on‐demand sucrose‐formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13‐month crossover study. Journal of Thrombosis and Haemostasis. 2010;8(1):83-9.
24. Fischer K, Van Den Berg M. Prophylaxis for severe haemophilia: Clinical and economical issues. Haemophilia. 2003;9(4):376-81.
25. Gringeri A, Lundin B, Mackensen S, Mantovani L, Mannucci P. A randomized clinical trial on prophylaxis Vs. episodic treatment in children with haemophilia A: the ESPRIT study [abstract]. Haemophilia. 2010; 16(4):29.
26. Valentino LA, Mamonov V, Hellmann A, Quon DV, Chybicka A, Schroth P, et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. Journal of thrombosis and haemostasis : JTH. 2012 Mar;10(3):359-67.
27. Manco-Johnson MJ, Kempton CL, Reding MT, Lissitchkov T, Goranov S, Gercheva L, et al. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). Journal of thrombosis and haemostasis : JTH. 2013; 11(6):1119-27.
28. Kavakli K, Yang R, Rusen L, Beckmann H, Tseneklidou-Stoeter D, Maas Enriquez M. Prophylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: Results from a randomized trial (LEOPOLD II). Journal of thrombosis and haemostasis : JTH. 2015; 13(3):360-9.
29. Antunes SV, Tangada S, Stasyshyn O, Mamonov V, Phillips J, Guzman-Becerra N, et al. Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors. Haemophilia [Internet]. 2013; 20(1):65-72.
30. Zhao Y, Xiao J, Yang R, Wu R, Hu Y, Beckmann H, et al. Efficacy of standard prophylaxis versus on-demand treatment with bayer's sucrose-formulated recombinant FVIII (rFVIII-FS) in Chinese children with severe hemophilia A. Pediatric Hematology and Oncology. 2017:1-11.
31. Bolton-Maggs PH, Pasi KJ. Haemophilias a and b. The lancet. 2003;361(9371):1801-9.
32. Panicker J, Warrier I, Thomas R, Lusher J. The overall effectiveness of prophylaxis in severe haemophilia. Haemophilia. 2003;9(3):272-8.
| Files | ||
| Issue | Vol 3, No 1 (2019) | |
| Section | Articles | |
| DOI | https://doi.org/10.18502/htaa.v3i1.5636 | |
| Keywords | ||
| prophilaxis treatment severe haemophilia safety efficacy systematic review. | ||
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How to Cite
1.
Goudarzi R, Seraji S, Amiresmaili M, Gharibnavaz H. Safety and efficacy of secondary prophylactic in Patients with severe hemophilia type A and B: A Systematic review and meta-analysis study. Health Tech Ass Act. 2020;3(1).
