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__NOTOC__
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==Overview==
==Overview==
 
Clotting factor replacement is the mainstay of hemophilia treatment. Plasma-derived factor concentrates and recombinant factor concentrates are the two types used in the replacement therapy. Other products used as therapy include desmopressin acetate, epsilon amino caproic acid, and cryoprecipitate. Gene therapy has the potential to change the course of hemophilia therapy and care.
==Medical Therapy==
==Medical Therapy==
===Clotting Factor Replacement===
===Clotting Factor Replacement===
Line 10: Line 10:
*The two types of clotting factor concentrates used for replacement are:
*The two types of clotting factor concentrates used for replacement are:
'''1. Plasma-derived factor concentrates'''
'''1. Plasma-derived factor concentrates'''
*
*It is the primary replacement therapy in patients with hemophilia A in the developing countries.<ref name="pmid30107983">{{cite journal |vauthors=Kevane B, O'Connell N |title=The current and future role of plasma-derived clotting factor concentrate in the treatment of haemophilia A |journal=Transfus. Apher. Sci. |volume=57 |issue=4 |pages=502–506 |date=August 2018 |pmid=30107983 |doi=10.1016/j.transci.2018.07.012 |url=}}</ref><ref name="StonebrakerBrooker2010">{{cite journal|last1=Stonebraker|first1=J. S.|last2=Brooker|first2=M.|last3=Amand|first3=R. E.|last4=Farrugia|first4=A.|last5=Srivastava|first5=A.|title=A study of reported factor VIII use around the world|journal=Haemophilia|volume=16|issue=1|year=2010|pages=33–46|issn=13518216|doi=10.1111/j.1365-2516.2009.02131.x}}</ref>
*Has a lower risk of factor VIII alloantibody formation compared to the recombinant products.<ref name="pmid30107983">{{cite journal |vauthors=Kevane B, O'Connell N |title=The current and future role of plasma-derived clotting factor concentrate in the treatment of haemophilia A |journal=Transfus. Apher. Sci. |volume=57 |issue=4 |pages=502–506 |date=August 2018 |pmid=30107983 |doi=10.1016/j.transci.2018.07.012 |url=}}</ref><ref name="PeyvandiMannucci2016">{{cite journal|last1=Peyvandi|first1=Flora|last2=Mannucci|first2=Pier M.|last3=Garagiola|first3=Isabella|last4=El-Beshlawy|first4=Amal|last5=Elalfy|first5=Mohsen|last6=Ramanan|first6=Vijay|last7=Eshghi|first7=Peyman|last8=Hanagavadi|first8=Suresh|last9=Varadarajan|first9=Ramabadran|last10=Karimi|first10=Mehran|last11=Manglani|first11=Mamta V.|last12=Ross|first12=Cecil|last13=Young|first13=Guy|last14=Seth|first14=Tulika|last15=Apte|first15=Shashikant|last16=Nayak|first16=Dinesh M.|last17=Santagostino|first17=Elena|last18=Mancuso|first18=Maria Elisa|last19=Sandoval Gonzalez|first19=Adriana C.|last20=Mahlangu|first20=Johnny N.|last21=Bonanad Boix|first21=Santiago|last22=Cerqueira|first22=Monica|last23=Ewing|first23=Nadia P.|last24=Male|first24=Christoph|last25=Owaidah|first25=Tarek|last26=Soto Arellano|first26=Veronica|last27=Kobrinsky|first27=Nathan L.|last28=Majumdar|first28=Suvankar|last29=Perez Garrido|first29=Rosario|last30=Sachdeva|first30=Anupam|last31=Simpson|first31=Mindy|last32=Thomas|first32=Mathew|last33=Zanon|first33=Ezio|last34=Antmen|first34=Bulent|last35=Kavakli|first35=Kaan|last36=Manco-Johnson|first36=Marilyn J.|last37=Martinez|first37=Monica|last38=Marzouka|first38=Esperanza|last39=Mazzucconi|first39=Maria G.|last40=Neme|first40=Daniela|last41=Palomo Bravo|first41=Angeles|last42=Paredes Aguilera|first42=Rogelio|last43=Prezotti|first43=Alessandra|last44=Schmitt|first44=Klaus|last45=Wicklund|first45=Brian M.|last46=Zulfikar|first46=Bulent|last47=Rosendaal|first47=Frits R.|title=A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A|journal=New England Journal of Medicine|volume=374|issue=21|year=2016|pages=2054–2064|issn=0028-4793|doi=10.1056/NEJMoa1516437}}</ref><ref name="MakrisKessler2017">{{cite journal|last1=Makris|first1=M.|last2=Kessler|first2=C. M.|title=SIPPET trial: the answers|journal=Haemophilia|volume=23|issue=3|year=2017|pages=344–345|issn=13518216|doi=10.1111/hae.13239}}</ref><ref name="FallonLavin2018">{{cite journal|last1=Fallon|first1=P. G.|last2=Lavin|first2=M.|last3=O'Donnell|first3=J. S.|title=SIPPET: insights into factor VIII immunogenicity|journal=Journal of Thrombosis and Haemostasis|volume=16|issue=1|year=2018|pages=36–38|issn=15387933|doi=10.1111/jth.13886}}</ref>
'''2. Recombinant factor concentrates'''
*These concentrates are genetically engineered with the use of DNA technology.<ref>https://www.cdc.gov/ncbddd/hemophilia/treatment.html</ref>
*The absence of plasma and albumin has paved the way for negative transmission of bloodborne viruses.<ref>https://www.cdc.gov/ncbddd/hemophilia/treatment.html</ref>
===Complications Clotting Factor Replacement===
*Development of inhibitors (antibodies directed against the clotting factor concentrates)<ref>https://www.nhlbi.nih.gov/health-topics/hemophilia#Treatment</ref>
*Transmission of bloodborne pathogens<ref>https://www.nhlbi.nih.gov/health-topics/hemophilia#Treatment</ref>
===Other Products===
*The other products used as a part of medical therapy of hemophilia include:
'''1. Antifibrinolytics'''
*Tranexamic acid and epsilon amino caproic acid can be used in the medical therapy of hemophilia<ref name="pmid28338025">{{cite journal |vauthors=Watterson C, Beacher N |title=Preventing perioperative bleeding in patients with inherited bleeding disorders |journal=Evid Based Dent |volume=18 |issue=1 |pages=28–29 |date=March 2017 |pmid=28338025 |doi=10.1038/sj.ebd.6401226 |url=}}</ref><ref name="pmid28338025">{{cite journal |vauthors=Watterson C, Beacher N |title=Preventing perioperative bleeding in patients with inherited bleeding disorders |journal=Evid Based Dent |volume=18 |issue=1 |pages=28–29 |date=March 2017 |pmid=28338025 |doi=10.1038/sj.ebd.6401226 |url=}}</ref>
*Especially beneficial in preventing oral bleeding<ref name="pmid26704192">{{cite journal |vauthors=van Galen KP, Engelen ET, Mauser-Bunschoten EP, van Es RJ, Schutgens RE |title=Antifibrinolytic therapy for preventing oral bleeding in patients with haemophilia or Von Willebrand disease undergoing minor oral surgery or dental extractions |journal=Cochrane Database Syst Rev |volume= |issue=12 |pages=CD011385 |date=December 2015 |pmid=26704192 |doi=10.1002/14651858.CD011385.pub2 |url=}}</ref><ref name="pmid28338025">{{cite journal |vauthors=Watterson C, Beacher N |title=Preventing perioperative bleeding in patients with inherited bleeding disorders |journal=Evid Based Dent |volume=18 |issue=1 |pages=28–29 |date=March 2017 |pmid=28338025 |doi=10.1038/sj.ebd.6401226 |url=}}</ref><ref name="pmid28338025">{{cite journal |vauthors=Watterson C, Beacher N |title=Preventing perioperative bleeding in patients with inherited bleeding disorders |journal=Evid Based Dent |volume=18 |issue=1 |pages=28–29 |date=March 2017 |pmid=28338025 |doi=10.1038/sj.ebd.6401226 |url=}}</ref>
'''2. Desmopressin acetate'''
*Desmopressin acetate can be used to prevent or treat bleeding episodes in patients with hemophilia<ref name="pmid23815950">{{cite journal |vauthors=Franchini M, Mannucci PM |title=Hemophilia A in the third millennium |journal=Blood Rev. |volume=27 |issue=4 |pages=179–84 |date=July 2013 |pmid=23815950 |doi=10.1016/j.blre.2013.06.002 |url=}}</ref><ref name="pmid30004154">{{cite journal |vauthors=Hews-Girard J, Rydz N, Lee A, Goodyear MD, Poon MC |title=Desmopressin in non-severe haemophilia A: Test-response and clinical outcomes in a single Canadian centre review |journal=Haemophilia |volume=24 |issue=5 |pages=720–725 |date=September 2018 |pmid=30004154 |doi=10.1111/hae.13586 |url=}}</ref>
'''3. Cryoprecipitate'''
*Effective for mild to moderate bleeding<ref name="pmid24745720">{{cite journal |vauthors=Lenk H |title=Treatment of haemophilia patients in East Germany prior to and after reunification in 1990 |journal=Thromb. Res. |volume=134 Suppl 1 |issue= |pages=S57–60 |date=November 2014 |pmid=24745720 |doi=10.1016/j.thromres.2013.10.018 |url=}}</ref><ref name="pmid30392819">{{cite journal |vauthors=Jain S, Acharya SS |title=Management of rare coagulation disorders in 2018 |journal=Transfus. Apher. Sci. |volume=57 |issue=6 |pages=705–712 |date=December 2018 |pmid=30392819 |doi=10.1016/j.transci.2018.10.009 |url=}}</ref>
*Can have preventive as well as therapeutic action<ref name="pmid24745720">{{cite journal |vauthors=Lenk H |title=Treatment of haemophilia patients in East Germany prior to and after reunification in 1990 |journal=Thromb. Res. |volume=134 Suppl 1 |issue= |pages=S57–60 |date=November 2014 |pmid=24745720 |doi=10.1016/j.thromres.2013.10.018 |url=}}</ref><ref name="pmid24972790">{{cite journal |vauthors=Nascimento B, Goodnough LT, Levy JH |title=Cryoprecipitate therapy |journal=Br J Anaesth |volume=113 |issue=6 |pages=922–34 |date=December 2014 |pmid=24972790 |pmc=4627369 |doi=10.1093/bja/aeu158 |url=}}</ref><ref name="pmid30392819">{{cite journal |vauthors=Jain S, Acharya SS |title=Management of rare coagulation disorders in 2018 |journal=Transfus. Apher. Sci. |volume=57 |issue=6 |pages=705–712 |date=December 2018 |pmid=30392819 |doi=10.1016/j.transci.2018.10.009 |url=}}</ref>
===Gene Therapy===
*Gene therapy is the transfer of a functional gene to replace the hemophilic defective gene.<ref name="pmid28895852">{{cite journal |vauthors=Nathwani AC, Davidoff AM, Tuddenham EGD |title=Gene Therapy for Hemophilia |journal=Hematol. Oncol. Clin. North Am. |volume=31 |issue=5 |pages=853–868 |date=October 2017 |pmid=28895852 |doi=10.1016/j.hoc.2017.06.011 |url=}}</ref><ref name="pmid29222308">{{cite journal |vauthors=George LA |title=Hemophilia gene therapy comes of age |journal=Hematology Am Soc Hematol Educ Program |volume=2017 |issue=1 |pages=587–594 |date=December 2017 |pmid=29222308 |pmc=6142599 |doi=10.1182/asheducation-2017.1.587 |url=}}</ref>
*This therapy induces continuous endogenous expression of factor VIII or IX.<ref name="pmid28895852">{{cite journal |vauthors=Nathwani AC, Davidoff AM, Tuddenham EGD |title=Gene Therapy for Hemophilia |journal=Hematol. Oncol. Clin. North Am. |volume=31 |issue=5 |pages=853–868 |date=October 2017 |pmid=28895852 |doi=10.1016/j.hoc.2017.06.011 |url=}}</ref><ref name="pmid29222308">{{cite journal |vauthors=George LA |title=Hemophilia gene therapy comes of age |journal=Hematology Am Soc Hematol Educ Program |volume=2017 |issue=1 |pages=587–594 |date=December 2017 |pmid=29222308 |pmc=6142599 |doi=10.1182/asheducation-2017.1.587 |url=}}</ref>




Revision as of 01:43, 28 January 2019

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Associate Editor(s)-in-Chief: Sabawoon Mirwais, M.B.B.S, M.D.[2]

Overview

Clotting factor replacement is the mainstay of hemophilia treatment. Plasma-derived factor concentrates and recombinant factor concentrates are the two types used in the replacement therapy. Other products used as therapy include desmopressin acetate, epsilon amino caproic acid, and cryoprecipitate. Gene therapy has the potential to change the course of hemophilia therapy and care.

Medical Therapy

Clotting Factor Replacement

  • Clotting factor replacement is the mainstay of hemophilia treatment
  • The two types of clotting factor concentrates used for replacement are:

1. Plasma-derived factor concentrates

  • It is the primary replacement therapy in patients with hemophilia A in the developing countries.[1][2]
  • Has a lower risk of factor VIII alloantibody formation compared to the recombinant products.[1][3][4][5]

2. Recombinant factor concentrates

  • These concentrates are genetically engineered with the use of DNA technology.[6]
  • The absence of plasma and albumin has paved the way for negative transmission of bloodborne viruses.[7]

Complications Clotting Factor Replacement

  • Development of inhibitors (antibodies directed against the clotting factor concentrates)[8]
  • Transmission of bloodborne pathogens[9]

Other Products

  • The other products used as a part of medical therapy of hemophilia include:

1. Antifibrinolytics

  • Tranexamic acid and epsilon amino caproic acid can be used in the medical therapy of hemophilia[10][10]
  • Especially beneficial in preventing oral bleeding[11][10][10]

2. Desmopressin acetate

  • Desmopressin acetate can be used to prevent or treat bleeding episodes in patients with hemophilia[12][13]

3. Cryoprecipitate

  • Effective for mild to moderate bleeding[14][15]
  • Can have preventive as well as therapeutic action[14][16][15]

Gene Therapy

  • Gene therapy is the transfer of a functional gene to replace the hemophilic defective gene.[17][18]
  • This therapy induces continuous endogenous expression of factor VIII or IX.[17][18]


References

  1. ↑ 1.0 1.1 Kevane B, O'Connell N (August 2018). "The current and future role of plasma-derived clotting factor concentrate in the treatment of haemophilia A". Transfus. Apher. Sci. 57 (4): 502–506. doi:10.1016/j.transci.2018.07.012. PMID 30107983.
  2. ↑ Stonebraker, J. S.; Brooker, M.; Amand, R. E.; Farrugia, A.; Srivastava, A. (2010). "A study of reported factor VIII use around the world". Haemophilia. 16 (1): 33–46. doi:10.1111/j.1365-2516.2009.02131.x. ISSN 1351-8216.
  3. ↑ Peyvandi, Flora; Mannucci, Pier M.; Garagiola, Isabella; El-Beshlawy, Amal; Elalfy, Mohsen; Ramanan, Vijay; Eshghi, Peyman; Hanagavadi, Suresh; Varadarajan, Ramabadran; Karimi, Mehran; Manglani, Mamta V.; Ross, Cecil; Young, Guy; Seth, Tulika; Apte, Shashikant; Nayak, Dinesh M.; Santagostino, Elena; Mancuso, Maria Elisa; Sandoval Gonzalez, Adriana C.; Mahlangu, Johnny N.; Bonanad Boix, Santiago; Cerqueira, Monica; Ewing, Nadia P.; Male, Christoph; Owaidah, Tarek; Soto Arellano, Veronica; Kobrinsky, Nathan L.; Majumdar, Suvankar; Perez Garrido, Rosario; Sachdeva, Anupam; Simpson, Mindy; Thomas, Mathew; Zanon, Ezio; Antmen, Bulent; Kavakli, Kaan; Manco-Johnson, Marilyn J.; Martinez, Monica; Marzouka, Esperanza; Mazzucconi, Maria G.; Neme, Daniela; Palomo Bravo, Angeles; Paredes Aguilera, Rogelio; Prezotti, Alessandra; Schmitt, Klaus; Wicklund, Brian M.; Zulfikar, Bulent; Rosendaal, Frits R. (2016). "A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A". New England Journal of Medicine. 374 (21): 2054–2064. doi:10.1056/NEJMoa1516437. ISSN 0028-4793.
  4. ↑ Makris, M.; Kessler, C. M. (2017). "SIPPET trial: the answers". Haemophilia. 23 (3): 344–345. doi:10.1111/hae.13239. ISSN 1351-8216.
  5. ↑ Fallon, P. G.; Lavin, M.; O'Donnell, J. S. (2018). "SIPPET: insights into factor VIII immunogenicity". Journal of Thrombosis and Haemostasis. 16 (1): 36–38. doi:10.1111/jth.13886. ISSN 1538-7933.
  6. ↑ https://www.cdc.gov/ncbddd/hemophilia/treatment.html
  7. ↑ https://www.cdc.gov/ncbddd/hemophilia/treatment.html
  8. ↑ https://www.nhlbi.nih.gov/health-topics/hemophilia#Treatment
  9. ↑ https://www.nhlbi.nih.gov/health-topics/hemophilia#Treatment
  10. ↑ 10.0 10.1 10.2 10.3 Watterson C, Beacher N (March 2017). "Preventing perioperative bleeding in patients with inherited bleeding disorders". Evid Based Dent. 18 (1): 28–29. doi:10.1038/sj.ebd.6401226. PMID 28338025.
  11. ↑ van Galen KP, Engelen ET, Mauser-Bunschoten EP, van Es RJ, Schutgens RE (December 2015). "Antifibrinolytic therapy for preventing oral bleeding in patients with haemophilia or Von Willebrand disease undergoing minor oral surgery or dental extractions". Cochrane Database Syst Rev (12): CD011385. doi:10.1002/14651858.CD011385.pub2. PMID 26704192.
  12. ↑ Franchini M, Mannucci PM (July 2013). "Hemophilia A in the third millennium". Blood Rev. 27 (4): 179–84. doi:10.1016/j.blre.2013.06.002. PMID 23815950.
  13. ↑ Hews-Girard J, Rydz N, Lee A, Goodyear MD, Poon MC (September 2018). "Desmopressin in non-severe haemophilia A: Test-response and clinical outcomes in a single Canadian centre review". Haemophilia. 24 (5): 720–725. doi:10.1111/hae.13586. PMID 30004154.
  14. ↑ 14.0 14.1 Lenk H (November 2014). "Treatment of haemophilia patients in East Germany prior to and after reunification in 1990". Thromb. Res. 134 Suppl 1: S57–60. doi:10.1016/j.thromres.2013.10.018. PMID 24745720.
  15. ↑ 15.0 15.1 Jain S, Acharya SS (December 2018). "Management of rare coagulation disorders in 2018". Transfus. Apher. Sci. 57 (6): 705–712. doi:10.1016/j.transci.2018.10.009. PMID 30392819.
  16. ↑ Nascimento B, Goodnough LT, Levy JH (December 2014). "Cryoprecipitate therapy". Br J Anaesth. 113 (6): 922–34. doi:10.1093/bja/aeu158. PMC 4627369. PMID 24972790.
  17. ↑ 17.0 17.1 Nathwani AC, Davidoff AM, Tuddenham E (October 2017). "Gene Therapy for Hemophilia". Hematol. Oncol. Clin. North Am. 31 (5): 853–868. doi:10.1016/j.hoc.2017.06.011. PMID 28895852. Vancouver style error: initials (help)
  18. ↑ 18.0 18.1 George LA (December 2017). "Hemophilia gene therapy comes of age". Hematology Am Soc Hematol Educ Program. 2017 (1): 587–594. doi:10.1182/asheducation-2017.1.587. PMC 6142599. PMID 29222308.

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