Evaluation of factor VIII prophylaxis of plasmatic origin in patients with severe hemophilia A
DOI:
https://doi.org/10.29193/RMU.38.4.1Keywords:
HEMOPHILIA A, FACTOR VIII, FUNCTIONAL INDEPENDENCE, PROPHYLAXISAbstract
Introduction: severe hemophilia A (SAH) is an hereditary hemorrhagic disease, caused by a factor VIII (FVIII) deficiency of less than 1%. It presents with joint bleeding mainly, which causes a hemophilic arthropathy, which affects its functional independence. The use of tertiary prophylaxis with FVIII offers benefits in adults by decreasing the annual bleeding rate (ABR) and improving functional independence.
Objective: to determine the percentage of patients who manage to maintain an FVIII level greater than 1%, to know if there is an improvement in functional independence, as well as a decrease in the ABR with the prophylactic regimen used.
Methods: observational, analytical study. Patients with SAH who were controlled at the Hospital de Clínicas “Dr. Manuel Quintela”, during the year 2020, in prophylaxis with FVIII for 12 months were included. Three samples separated in time for FVIII dosing were obtained and the ABR and functional independence were evaluated in each patient.
Results: 8 patients were analyzed, all presented FVIII higher than 1% at one hour and 24 hours after the administration of FVIII. The bleeding episodes were reduced 4.76 times with the use of Prophylaxis (p = 0.019). Functional independence showed that 5/8 patients improved at least 1 point in the Score.
Conclusions: tertiary prophylaxis in these patients was beneficial in reducing SAD and improving their functional capacity.
References
1) Josephson N. The haemophilias and their clinical management. Hematology Am Soc Hematol Educ Program 2013; 2013:261-7. doi: 10.1182/asheducation-2013.1.261.
2) Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, et al. Guidelines for the management of hemophilia. Haemophilia 2013; 19(1):e1-e47. doi: 10.1111/j.1365-2516.2012.02909.x.
3) Lorio A, Stonebraker JS, Chambost H, Makris M, Coffin D, Herr C, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med 2019; 171(8):540-6. doi: 10.7326/M19-1208.
4) Ferreira AA, Bustamante-Teixeira MT, Leite IC, Correa CS, Rodrigues DeO, da Cruz DT. Clinical and functional evaluation of the joint status of hemophiliac adults at a Brazilian blood center. Rev Bras Hematol Hemoter 2013; 35(1):23-8. doi: 10.5581/1516-8484.20130010.
5) Goddard NJ, Mann H. Diagnosis of haemophilic synovitis. Haemophilia 2007; 13(Suppl 3):14-9. doi: 10.1111/j.1365-2516.2007.01535.x.
6) Gurcay E, Eksioglu E, Ezer U, Tuncay R, Cakci A. Functional disability in children with hemophilic arthropathy. Rheumatol Int 2006; 26(11):1031-5. doi: 10.1007/s00296-006-0115-6.
7) Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Intern Med 1994; 236(4):391-9. doi: 10.1111/j.1365-2796.1994.tb00815.x.
8) Gilbert MS. Musculoskeletal complications of haemophilia: the joint. Haemophilia 2000; 6(Suppl 1):34-7. doi: 10.1046/j.1365-2516.2000.00044.x.
9) Retamales Castelletto E. Recomendaciones para la etapa pre-analítica, analítica y post-analítica en las prestaciones de coagulación. Santiago: Instituto Salud Pública de Chile, 2014. Abril, 2014.
10) Marlar RA, Cook J, Johnston M, Kitchen S, Machin SJ, Shafer D, et al. H47-A2 One-stage prothrombin time (PT) test and activated parcial thrombopastin time (APTT) test: approved guideline-Second Edition. Wayne, PA: Clinical and Laboratory Standards Institute, 2008.
11) Díaz A, Almagro D, Brito A. Sensibilidad del tiempo parcial de tromboplastina activado a la deficiencia de factores VIII y IX y a la heparina. Rev Cubana Hematol Inmunol Hemoter 2001; 17(1):41-8.
12) Dumoulin EN, Fiers L, Devreese KM. Investigation of sensitivity for coagulation factor deficiency in APTT and PT: how to perform it? Clin Chem Lab Med 2016; 54(5):e169-72. doi: 10.1515/cclm-2015-0670.
13) Lawrie AS, Kitchen S, Efthymiou M, Mackie IJ, Machi SJ. Determination of APTT factor sensitivity--the misguiding guideline. Int J Lab Hematol 2013; 35(6):652-7. doi: 10.1111/ijlh.12109.
14) Bowyer A, Kitchen S, Makris M. The responsiveness of different APTT reagents to mild factor VIII, IX and XI deficiencies. Int J Lab Med 2011; 33(2):154-8. doi: 10.1111/j.1751-553X.2010.01261.x.
15) Ljung R, Aronis-Vournas S, Kurnik-Auberger K, van den Berg M, Chambost H, et al. Treatment of children with haemophilia in Europe: a survey of 20 centers in 16 countries. Haemophilia 2000; 6:619-24. doi: 10.1046/j.1365-2516.2000.00427.x.
16) Moreno MM, Cuesta-Barriuso R. A history of prophylaxis in haemophilia. Blood Coagul Fibrinolysis 2019; 30(2):55-7. doi: 10.1097/MBC.0000000000000783.
17) Zanon E, Tagliaferri A, Pasca S, Ettorre CP, Notarangelo LD, Biasioli C, et al. Physical activity improved by adherence to prophylaxis in an Italian population of children, adolescents and adults with severe haemophilia A: the SHAPE Study. Blood Transfus 2020; 18(2):152-8. doi: 10.2450/2019.0040-19.
18) Gouider E. Show me the evidence: effectiveness of low-dose prophylaxis. Haemophilia 2020; 26 (Suppl 3):9-10. doi: org/10.1111/hae.13892.
19) Carcao M, Goudemand J. Los inhibidores en la hemofilia: información básica. 5a ed. Montreal: Federación Mundial de Hemofilia, 2018.
20) Poonnoose PM, Manigandan C, Thomas R, Shyamkumar NK, Kavitha ML, Bhattacharji S, et al. Functional Independence Score in Haemophilia: a new performance-based instrument to measure disability. Hemofilia 2005; 11(6):598-602. doi: 10.1111/j.1365-2516.2005.01142.x.
21) Marlar RA, Cook J, Johnston M, Kitchen S, Machin SJ, Shafer D, et al. One-stage Prothrombin Time (PT) test and Activated Parcial Thrombopastin Time (APTT) Test; Approved guideline-second edition. (Document H47-A2). Wayne: Clinical and Laboratory Standards Institute, 2008.
22) Coagulación. Pruebas globales y determinación de factores. Capítulo 6:249-333. En: Scazziota A. Fundamentos para el manejo práctico en el laboratorio de hemostasia. Buenos Aires: Grupo CAHT, 2013.
23) Favaloro JE, Geoffrey K, Soma M, Giuseppe L. How to optimize activated parcial thromboplastin time (APTT) testing: solutions to establishing and verifying normal reference intervals and assessing APTT reagents for sensitivity to heparin, lupus anticoagulant, and clothing factors. Semin Thromb Hemost. 2019; 45(1):22-35. doi: org/10.1055/s-0038-1677018.
24) Linskens EA, Devreese KMJ. Pre-analytical stability of coagulation parameters in plasma stored at room temperature. Int J Lab Hem 2018; 40(3):292-303. doi: 10.1111/ijlh.12784.
25) Siemens Healthcare. Incerto BCS System. Disponible en: https://www.siemens-healthineers.com/hemostasis/systems/bcs-xp-system [Consulta: 24 setiembre 2021].
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. J Thromb Haemost 2012; 10(3):359-67. doi: 10.1111/j.1538-7836.2011.04611.x.
27) Collins P, Faradji A, Morfini M, Enriquez MM, Schwartz LS. 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. J Throm Haemost 2010; 8(1):83-9. doi: 10.1111/j.1538-7836.2009.03650.x.
28) 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). Thromb Haemost 2013; 11(6):1119-27. doi: 10.1111/jth.12202.
29) Hua B, Lian X, Lee A, Poon MC, Zhao Y. Low-dose tertiary prophylactic therapy reduces total number of bleeds and improves the ability to perform activities of daily living in adults with severe haemophilia A: a single-centre experience from Beijing. Blood Coagul Fibrinolysis 2016; 27(2):136-40. doi:10.1097/MBC.0000000000000389.
30) Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood 2015; 125(13):2038-44. doi: 10.1182/blood-2015-01-528414.
2) Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, et al. Guidelines for the management of hemophilia. Haemophilia 2013; 19(1):e1-e47. doi: 10.1111/j.1365-2516.2012.02909.x.
3) Lorio A, Stonebraker JS, Chambost H, Makris M, Coffin D, Herr C, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med 2019; 171(8):540-6. doi: 10.7326/M19-1208.
4) Ferreira AA, Bustamante-Teixeira MT, Leite IC, Correa CS, Rodrigues DeO, da Cruz DT. Clinical and functional evaluation of the joint status of hemophiliac adults at a Brazilian blood center. Rev Bras Hematol Hemoter 2013; 35(1):23-8. doi: 10.5581/1516-8484.20130010.
5) Goddard NJ, Mann H. Diagnosis of haemophilic synovitis. Haemophilia 2007; 13(Suppl 3):14-9. doi: 10.1111/j.1365-2516.2007.01535.x.
6) Gurcay E, Eksioglu E, Ezer U, Tuncay R, Cakci A. Functional disability in children with hemophilic arthropathy. Rheumatol Int 2006; 26(11):1031-5. doi: 10.1007/s00296-006-0115-6.
7) Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Intern Med 1994; 236(4):391-9. doi: 10.1111/j.1365-2796.1994.tb00815.x.
8) Gilbert MS. Musculoskeletal complications of haemophilia: the joint. Haemophilia 2000; 6(Suppl 1):34-7. doi: 10.1046/j.1365-2516.2000.00044.x.
9) Retamales Castelletto E. Recomendaciones para la etapa pre-analítica, analítica y post-analítica en las prestaciones de coagulación. Santiago: Instituto Salud Pública de Chile, 2014. Abril, 2014.
10) Marlar RA, Cook J, Johnston M, Kitchen S, Machin SJ, Shafer D, et al. H47-A2 One-stage prothrombin time (PT) test and activated parcial thrombopastin time (APTT) test: approved guideline-Second Edition. Wayne, PA: Clinical and Laboratory Standards Institute, 2008.
11) Díaz A, Almagro D, Brito A. Sensibilidad del tiempo parcial de tromboplastina activado a la deficiencia de factores VIII y IX y a la heparina. Rev Cubana Hematol Inmunol Hemoter 2001; 17(1):41-8.
12) Dumoulin EN, Fiers L, Devreese KM. Investigation of sensitivity for coagulation factor deficiency in APTT and PT: how to perform it? Clin Chem Lab Med 2016; 54(5):e169-72. doi: 10.1515/cclm-2015-0670.
13) Lawrie AS, Kitchen S, Efthymiou M, Mackie IJ, Machi SJ. Determination of APTT factor sensitivity--the misguiding guideline. Int J Lab Hematol 2013; 35(6):652-7. doi: 10.1111/ijlh.12109.
14) Bowyer A, Kitchen S, Makris M. The responsiveness of different APTT reagents to mild factor VIII, IX and XI deficiencies. Int J Lab Med 2011; 33(2):154-8. doi: 10.1111/j.1751-553X.2010.01261.x.
15) Ljung R, Aronis-Vournas S, Kurnik-Auberger K, van den Berg M, Chambost H, et al. Treatment of children with haemophilia in Europe: a survey of 20 centers in 16 countries. Haemophilia 2000; 6:619-24. doi: 10.1046/j.1365-2516.2000.00427.x.
16) Moreno MM, Cuesta-Barriuso R. A history of prophylaxis in haemophilia. Blood Coagul Fibrinolysis 2019; 30(2):55-7. doi: 10.1097/MBC.0000000000000783.
17) Zanon E, Tagliaferri A, Pasca S, Ettorre CP, Notarangelo LD, Biasioli C, et al. Physical activity improved by adherence to prophylaxis in an Italian population of children, adolescents and adults with severe haemophilia A: the SHAPE Study. Blood Transfus 2020; 18(2):152-8. doi: 10.2450/2019.0040-19.
18) Gouider E. Show me the evidence: effectiveness of low-dose prophylaxis. Haemophilia 2020; 26 (Suppl 3):9-10. doi: org/10.1111/hae.13892.
19) Carcao M, Goudemand J. Los inhibidores en la hemofilia: información básica. 5a ed. Montreal: Federación Mundial de Hemofilia, 2018.
20) Poonnoose PM, Manigandan C, Thomas R, Shyamkumar NK, Kavitha ML, Bhattacharji S, et al. Functional Independence Score in Haemophilia: a new performance-based instrument to measure disability. Hemofilia 2005; 11(6):598-602. doi: 10.1111/j.1365-2516.2005.01142.x.
21) Marlar RA, Cook J, Johnston M, Kitchen S, Machin SJ, Shafer D, et al. One-stage Prothrombin Time (PT) test and Activated Parcial Thrombopastin Time (APTT) Test; Approved guideline-second edition. (Document H47-A2). Wayne: Clinical and Laboratory Standards Institute, 2008.
22) Coagulación. Pruebas globales y determinación de factores. Capítulo 6:249-333. En: Scazziota A. Fundamentos para el manejo práctico en el laboratorio de hemostasia. Buenos Aires: Grupo CAHT, 2013.
23) Favaloro JE, Geoffrey K, Soma M, Giuseppe L. How to optimize activated parcial thromboplastin time (APTT) testing: solutions to establishing and verifying normal reference intervals and assessing APTT reagents for sensitivity to heparin, lupus anticoagulant, and clothing factors. Semin Thromb Hemost. 2019; 45(1):22-35. doi: org/10.1055/s-0038-1677018.
24) Linskens EA, Devreese KMJ. Pre-analytical stability of coagulation parameters in plasma stored at room temperature. Int J Lab Hem 2018; 40(3):292-303. doi: 10.1111/ijlh.12784.
25) Siemens Healthcare. Incerto BCS System. Disponible en: https://www.siemens-healthineers.com/hemostasis/systems/bcs-xp-system [Consulta: 24 setiembre 2021].
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. J Thromb Haemost 2012; 10(3):359-67. doi: 10.1111/j.1538-7836.2011.04611.x.
27) Collins P, Faradji A, Morfini M, Enriquez MM, Schwartz LS. 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. J Throm Haemost 2010; 8(1):83-9. doi: 10.1111/j.1538-7836.2009.03650.x.
28) 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). Thromb Haemost 2013; 11(6):1119-27. doi: 10.1111/jth.12202.
29) Hua B, Lian X, Lee A, Poon MC, Zhao Y. Low-dose tertiary prophylactic therapy reduces total number of bleeds and improves the ability to perform activities of daily living in adults with severe haemophilia A: a single-centre experience from Beijing. Blood Coagul Fibrinolysis 2016; 27(2):136-40. doi:10.1097/MBC.0000000000000389.
30) Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood 2015; 125(13):2038-44. doi: 10.1182/blood-2015-01-528414.
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Published
2022-12-12
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1.
Sambolino F, Medina V, Bentos J, Torres R, Berro M, Canessa C, et al. Evaluation of factor VIII prophylaxis of plasmatic origin in patients with severe hemophilia A. Rev. Méd. Urug. [Internet]. 2022 Dec. 12 [cited 2024 Nov. 21];38(4):e38402. Available from: https://revista.rmu.org.uy/index.php/rmu/article/view/978
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