Hemophilia A medical therapy: Difference between revisions

Jump to navigation Jump to search
No edit summary
No edit summary
Line 1: Line 1:
__NOTOC__
__NOTOC__
{{Hemophilia A}}
{{Hemophilia A}}
{{CMG}} {{FNY}}
{{CMG}}{{AE}}{{FNY}}


==Overview==
==Overview==
There is no definitive treatment for [[Hemophilia A]], the mainstay of therapy is supportive and preventative care. It is recommended that patients diagnosed with hemophilia be referred to hemophilia treatment centers (HTC), which provide coordinated care between physicians (usually hematologists), nurses, social workers and other staff who specialize in [[bleeding]] disorders. <ref> Konkle BA, Josephson NC, Nakaya Fletcher S. Hemophilia A. 2000 Sep 21 [Updated 2014 Jun 5]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2016. Available from: http://www-ncbi-nlm-nih-gov.laneproxy.stanford.edu/books/NBK1404/ </ref>
There is no definitive treatment for [[Hemophilia A]], the mainstay of therapy is supportive and preventative care. It is recommended that patients diagnosed with hemophilia be referred to hemophilia treatment centers (HTC), which provide coordinated care between physicians (usually hematologists), nurses, social workers and other staff who specialize in [[bleeding]] disorders.<ref> Konkle BA, Josephson NC, Nakaya Fletcher S. Hemophilia A. 2000 Sep 21 [Updated 2014 Jun 5]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2016. Available from: http://www-ncbi-nlm-nih-gov.laneproxy.stanford.edu/books/NBK1404/ </ref>


==Medical Therapy==
==Medical Therapy==
Most haemophilia patients require regular supplementation with [[intravenous]] [[recombinant]] [[factor VIII]], also known as replacement therapy. <ref name="pmid22551339">{{cite journal| author=Franchini M, Mannucci PM| title=Past, present and future of hemophilia: a narrative review. | journal=Orphanet J Rare Dis | year= 2012 | volume= 7 | issue=  | pages= 24 | pmid=22551339 | doi=10.1186/1750-1172-7-24 | pmc=3502605 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=22551339  }} </ref> This is highly individually determined. Apart from "routine" supplementation, extra factor concentrate is given around surgical procedures and after trauma, as well as emergently during any bleeding episode. Recombinant factor VIII concentrates do not contain any plasma or [[albumin]], and therefore do not carry the risk of transmitting bloodborne viruses. <ref> Treatment of Hemophilia – World Federation of Hemophilia. Available at http://www.wfh.org/en/page.aspx?pid=642. Accessed on Sept 20, 2016 </ref>
Most hemophilia patients require regular supplementation with [[intravenous]] [[recombinant]] [[factor VIII]], also known as replacement therapy.<ref name="pmid22551339">{{cite journal| author=Franchini M, Mannucci PM| title=Past, present and future of hemophilia: a narrative review. | journal=Orphanet J Rare Dis | year= 2012 | volume= 7 | issue=  | pages= 24 | pmid=22551339 | doi=10.1186/1750-1172-7-24 | pmc=3502605 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=22551339  }} </ref> This is highly individually determined. Apart from "routine" supplementation, extra factor concentrate is given around surgical procedures and after trauma, as well as emergently during any bleeding episode. Recombinant factor VIII concentrates do not contain any plasma or [[albumin]], and therefore do not carry the risk of transmitting bloodborne viruses.<ref> Treatment of Hemophilia – World Federation of Hemophilia. Available at http://www.wfh.org/en/page.aspx?pid=642. Accessed on Sept 20, 2016 </ref>


Other therapeutic options include [[cryoprecipitate]], [[fresh frozen plazma]] ([[FFP]]), [[desmopressin]] ([[DDAVP]]), and anti-fibrinoltytic agents.
Other therapeutic options include [[cryoprecipitate]], [[fresh frozen plazma]] ([[FFP]]), [[desmopressin]] ([[DDAVP]]), and anti-fibrinoltytic agents.
Line 21: Line 21:
*If a patient develops an inhibitor to factor VIII, physicians may either attempt to infuse larger doses of recombinant clotting factor, or try different clotting factor sources.  
*If a patient develops an inhibitor to factor VIII, physicians may either attempt to infuse larger doses of recombinant clotting factor, or try different clotting factor sources.  
*These antibodies may self-resolve with time. No definitive therapy exists otherwise for patients who develop inhibitors to factor VIII.
*These antibodies may self-resolve with time. No definitive therapy exists otherwise for patients who develop inhibitors to factor VIII.
*Recently, Immune Tolerance Induction (ITI) therapy is being researched as a means of helping patients with haemophilia and factor inhibitors. ITI involves overcoming the immune reaction and desensitizing the body to the foreign factor concentrate infusion. ITI is expensive, time-intensive, and requires the oversight of multiple medical professionals, and is best coordinated at a hemophilia treatment center (HTC). <ref> Inhibitors | Hemophilia | NCBDDD | CDC. Available at http://www.cdc.gov/ncbddd/hemophilia/inhibitors.html. Accessed on Sept 20, 2016 </ref>
*Recently, Immune Tolerance Induction (ITI) therapy is being researched as a means of helping patients with hemophilia and factor inhibitors. ITI involves overcoming the immune reaction and desensitizing the body to the foreign factor concentrate infusion. ITI is expensive, time-intensive, and requires the oversight of multiple medical professionals, and is best coordinated at a hemophilia treatment center (HTC).<ref> Inhibitors | Hemophilia | NCBDDD | CDC. Available at http://www.cdc.gov/ncbddd/hemophilia/inhibitors.html. Accessed on Sept 20, 2016 </ref>


==References==  
==References==  

Revision as of 05:59, 16 April 2017

Hemophilia A Microchapters

Home

Patient Information

Overview

Historical Perspective

Classification

Pathophysiology

Causes

Differentiating Hemophilia A from other Diseases

Epidemiology and Demographics

Risk Factors

Screening

Natural History, Complications and Prognosis

Diagnosis

Diagnostic Study of Choice

History and Symptoms

Physical Examination

Laboratory Findings

Electrocardiogram

X Ray

CT

MRI

Echocardiography or Ultrasound

Other Imaging Findings

Other Diagnostic Studies

Treatment

Medical Therapy

Surgery

Primary Prevention

Secondary Prevention

Cost-Effectiveness of Therapy

Future or Investigational Therapies

Case Studies

Case #1

Hemophilia A medical therapy On the Web

Most recent articles

Most cited articles

Review articles

CME Programs

Powerpoint slides

Images

American Roentgen Ray Society Images of Hemophilia A medical therapy

All Images
X-rays
Echo & Ultrasound
CT Images
MRI

Ongoing Trials at Clinical Trials.gov

US National Guidelines Clearinghouse

NICE Guidance

FDA on Hemophilia A medical therapy

CDC on Hemophilia A medical therapy

Hemophilia A medical therapy in the news

Blogs on Hemophilia A medical therapy

Directions to Hospitals Treating Hemophilia A

Risk calculators and risk factors for Hemophilia A medical therapy

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

Overview

There is no definitive treatment for Hemophilia A, the mainstay of therapy is supportive and preventative care. It is recommended that patients diagnosed with hemophilia be referred to hemophilia treatment centers (HTC), which provide coordinated care between physicians (usually hematologists), nurses, social workers and other staff who specialize in bleeding disorders.[1]

Medical Therapy

Most hemophilia patients require regular supplementation with intravenous recombinant factor VIII, also known as replacement therapy.[2] This is highly individually determined. Apart from "routine" supplementation, extra factor concentrate is given around surgical procedures and after trauma, as well as emergently during any bleeding episode. Recombinant factor VIII concentrates do not contain any plasma or albumin, and therefore do not carry the risk of transmitting bloodborne viruses.[3]

Other therapeutic options include cryoprecipitate, fresh frozen plazma (FFP), desmopressin (DDAVP), and anti-fibrinoltytic agents.

  • Cryoprecipitate contains moderately high concentrations of factor VIII and may be given for joint and muscle bleeds. However, as it is derived from blood it carries risk of viral contamination
  • FFP (fresh frozen plasma) contains clotting factors VIII and IX at significantly less concentrations than cryoprecipitate. As such, it is not routinely recommended as first-line therapy for hemophiliacs with bleeding episodes, given that the high amount of volume required to correct the coagulation deficit may result in circulatory volume overload.
  • DDAVP, or desmopressin, is a synthetic hormone which stimulates the release of factor VIII. It may be given to patients with mild, or sometimes moderate, hemophilia to treat minor bleeding
  • Anti-fibrinolytic medications, most commonly epsilon aminocarproic acid, may be given orally or intravenously to prevent clots from being dissolved. It is often given orally prior to dental work to reduce risk of bleeding.
  • Patients with hemophilia may also require transfusion of packed red blood cells if excessive bleeding results in anemia.

A particular therapeutic conundrum is the development of "inhibitor" antibodies against factor VIII due to frequent infusions.

  • 20-30% of patients with severe hemophilia A develop antibodies to clotting factor VIII, called inhibitors.
  • Inhibitors prevent replacement therapy from being effective as they target the recombinant factor VIII and prevent it from working.
  • If a patient develops an inhibitor to factor VIII, physicians may either attempt to infuse larger doses of recombinant clotting factor, or try different clotting factor sources.
  • These antibodies may self-resolve with time. No definitive therapy exists otherwise for patients who develop inhibitors to factor VIII.
  • Recently, Immune Tolerance Induction (ITI) therapy is being researched as a means of helping patients with hemophilia and factor inhibitors. ITI involves overcoming the immune reaction and desensitizing the body to the foreign factor concentrate infusion. ITI is expensive, time-intensive, and requires the oversight of multiple medical professionals, and is best coordinated at a hemophilia treatment center (HTC).[4]

References

  1. Konkle BA, Josephson NC, Nakaya Fletcher S. Hemophilia A. 2000 Sep 21 [Updated 2014 Jun 5]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2016. Available from: http://www-ncbi-nlm-nih-gov.laneproxy.stanford.edu/books/NBK1404/
  2. Franchini M, Mannucci PM (2012). "Past, present and future of hemophilia: a narrative review". Orphanet J Rare Dis. 7: 24. doi:10.1186/1750-1172-7-24. PMC 3502605. PMID 22551339.
  3. Treatment of Hemophilia – World Federation of Hemophilia. Available at http://www.wfh.org/en/page.aspx?pid=642. Accessed on Sept 20, 2016
  4. Inhibitors | Hemophilia | NCBDDD | CDC. Available at http://www.cdc.gov/ncbddd/hemophilia/inhibitors.html. Accessed on Sept 20, 2016

Template:WH Template:WS