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==Overview==
==Overview==
 
Clotting factor replacement is the mainstay of hemophilia treatment. [[Blood plasma|Plasma]]-derived factor concentrates and [[Recombinant DNA|recombinant]] factor concentrates are the two types used in the replacement [[therapy]]. Other products used as [[therapy]] include [[Desmopressin acetate (patient information)|desmopressin acetate]], [[Antifibrinolytic|antifibrinolytics]], and [[cryoprecipitate]]. [[Gene therapy]] has the potential to change the course of hemophilia [[therapy]] and [[Medical care|care]].
==Medical Therapy==
==Medical Therapy==
===Clotting Factor Replacement===
===Clotting Factor Replacement===
*Clotting factor replacement is the mainstay of hemophilia treatment
*[[Clotting]] factor replacement is the mainstay of hemophilia treatment
*The two types of clotting factor concentrates used for replacement are:
*The two types of [[clotting]] factor concentrates used as replacement are:
'''1. Plasma-derived factor concentrates'''
'''1. Plasma-derived factor concentrates'''
*
*It is the primary replacement [[therapy]] in [[Patient|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]] [[Antibodies|alloantibody]] formation compared to the [[Recombinant DNA|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 [[Genetic engineering|genetically engineered]] with the use of [[DNA]] technology.<ref>https://www.cdc.gov/ncbddd/hemophilia/treatment.html</ref>
*The absence of [[Blood plasma|plasma]] and [[albumin]] has paved the way for negative [[Transmission (medicine)|transmission]] of [[Blood-borne disease|bloodborne]] [[Virus|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 (medicine)|Transmission]] of [[Blood-borne disease|bloodborne]] [[Pathogen|pathogens]]<ref>https://www.nhlbi.nih.gov/health-topics/hemophilia#Treatment</ref>
===Other Products===
*The other products used as a part of [[Medicine|medical]] [[therapy]] of hemophilia include:
'''1. Antifibrinolytics'''
*[[Tranexamic acid]] and epsilon amino caproic acid can be used in the [[Medicine|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 [[Prevention (medical)|preventing]] [[Mouth|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 (patient information)|Desmopressin acetate]] can be used to [[Prevention (medical)|prevent]] or treat [[bleeding]] episodes in [[Patient|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 a [[Prevention (medical)|preventive]] as well as [[Therapy|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>
===Immune Tolerance Induction===
*[[Immune tolerance]] can be [[Induction (biology)|induced]] (by daily [[Injection (medicine)|injection]] of large amounts of [[factor VIII]] concentrate) to eradicate [[factor VIII]] inhibitors.<ref name="pmid30545924">{{cite journal |vauthors=Delignat S, Russick J, Gangadharan B, Rayes J, Ing M, Voorberg J, Kaveri SV, Lacroix-Desmazes S |title=Prevention of the anti-factor VIII memory B-cell response by inhibition of the Bruton's tyrosine kinase in experimental hemophilia A |journal=Haematologica |volume= |issue= |pages= |date=December 2018 |pmid=30545924 |doi=10.3324/haematol.2018.200279 |url=}}</ref>
===Gene Therapy===
*[[Gene therapy]] is the transfer of a functional [[gene]] to replace the [[Hemophilia|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 [[Endogeny|endogenous]] expression of [[factor VIII]] or [[Factor IX|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>


==References==
==References==

Latest revision as of 19:11, 31 May 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, antifibrinolytics, 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 as replacement are:

1. Plasma-derived factor concentrates

2. Recombinant factor concentrates

Complications Clotting Factor Replacement

Other Products

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

1. Antifibrinolytics

2. Desmopressin acetate

3. Cryoprecipitate

Immune Tolerance Induction

Gene Therapy

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. ↑ Delignat S, Russick J, Gangadharan B, Rayes J, Ing M, Voorberg J, Kaveri SV, Lacroix-Desmazes S (December 2018). "Prevention of the anti-factor VIII memory B-cell response by inhibition of the Bruton's tyrosine kinase in experimental hemophilia A". Haematologica. doi:10.3324/haematol.2018.200279. PMID 30545924.
  18. ↑ 18.0 18.1 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)
  19. ↑ 19.0 19.1 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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