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203. Centers for Disease Control and Prevention. Summary report of UDC national activity. Treatment/clinical characteristics (hemophilia). www2a.cdc.gov/ncbddd/htcweb/UDC_Report/UDC_Report.asp (Accessed 3 December 2011)

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Author(s)

Mindy L. Simpson, MD

Department of Pediatrics, Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, Illinois

Disclosures

Disclosure: Mindy L. Simpson, MD, has disclosed no relevant financial relationships.

Leonard A. Valentino, MD

Department of Pediatrics, Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, Illinois

Disclosures

Disclosure: Leonard A. Valentino, MD, has disclosed no relevant financial relationships.

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Elisa Manzotti

Publisher, Future Science Group, London, United Kingdom

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Disclosure: Elisa Manzotti has disclosed no relevant financial relationships.

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Laurie Barclay, MD

Freelance writer and reviewer, Medscape, LLC

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Disclosure: Laurie Barclay, MD, has disclosed no relevant financial relationships.

CME Reviewer(s)

Nafeez Zawahir, MD

CME Clinical Director, Medscape, LLC

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Disclosure: Nafeez Zawahir, MD, has disclosed no relevant financial relationships.

CME

Management of Joint Bleeding in Hemophilia

Authors: Mindy L. Simpson, MD; Leonard A. Valentino, MD
CME Released: 9/20/2012
THIS ACTIVITY HAS EXPIRED FOR CREDIT
Valid for credit through: 9/20/2013, 11:59 PM EST

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Target Audience and Goal Statement

This activity is intended for hematologists, radiologists, pediatricians, and other clinicians caring for patients with joint bleeding due to hemophilia.

The goal of this activity is to review the clinical presentation and management of hemarthrosis in patients with hemophilia.

Upon completion of this activity, participants will be able to:

Describe the clinical characteristics of joint bleeding in hemophilia
Describe prophylaxis to prevent joint bleeding in hemophilia
Describe management of acute hemarthrosis in patients with hemophilia

Disclosures

Author(s)

Mindy L. Simpson, MD

Department of Pediatrics, Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, Illinois

Disclosures

Disclosure: Mindy L. Simpson, MD, has disclosed no relevant financial relationships.

Leonard A. Valentino, MD

Department of Pediatrics, Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, Illinois

Disclosures

Disclosure: Leonard A. Valentino, MD, has disclosed no relevant financial relationships.

Editor

Elisa Manzotti

Publisher, Future Science Group, London, United Kingdom

Disclosures

Disclosure: Elisa Manzotti has disclosed no relevant financial relationships.

CME Author(s)

Laurie Barclay, MD

Freelance writer and reviewer, Medscape, LLC

Disclosures

Disclosure: Laurie Barclay, MD, has disclosed no relevant financial relationships.

CME Reviewer(s)

Nafeez Zawahir, MD

CME Clinical Director, Medscape, LLC

Disclosures

Disclosure: Nafeez Zawahir, MD, has disclosed no relevant financial relationships.

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Five-year View

Over the next 5 years, one or more drugs with a prolonged biological activity and/or novel mechanism of action will be approved for use in the USA.^[93,95–97] The advantages of these drugs in preventing joint and other bleeding are likely to be substantial,^[98] but their efficacy must be established through rigorous clinical trial programs. In addition to these new therapies, the next half century is likely to provide individuals treating hemophilia with an enhanced understanding of the benefits and risks of adjuvant therapies and better tools for measuring outcomes. Sensitive, noninvasive modalities for detecting subclinical joint bleeding are desperately needed now, and this need will become more urgent following the introduction of sustained-activity products, as patients may have prolonged periods with FVIII or FIX levels in the mild or moderate range. Such strategies are under investigation, and some may reach the clinic within 5 years.

Despite this optimistic perspective, one must keep in mind that only about 25% of the estimated 400,000 people with hemophilia worldwide receive adequate treatment. The World Federation of Hemophilia is attempting to close the gap in care for the remaining 75% by promoting proper diagnosis, management and care together with the provision of safe, effective treatment products. However, in many countries, the high cost of clotting factor concentrates for replacement therapy make this goal impossible, and the provision of prophylaxis, recommended as optimal care, unrealistic. Advocacy efforts for adequate product supplies are ongoing through the World Federation of Hemophilia and its national patient organizations. Cost-efficacy studies to identify the minimum effective dose to prevent bleeding and other initiatives are necessary to expand access to prophylaxis and to better understand how the conservation of precious products can be achieved in parts of the world where resources are limited. Novel approaches to care, such as once-weekly prophylactic dosing, will require multicenter, international prospective studies that compare efficacy with standard therapy.

Palliative approaches to blood-induced joint disease, such as joint fusion and joint replacement therapy, will continue to play an important role in the overall care of patients with hemophilia for the foreseeable future. Nonetheless, new strategies that can be undertaken before the development of end stage arthropathy are needed to improve HRQoL. To that end, it is imperative that targeted biological therapies directed at the earliest molecular and biochemical changes invoked by intra-articular blood be identified. Clinical trials of biological agents that use antibodies to block TNF-a (e.g., infliximab, adalimumab, golimumab and certolizumab) are approved for use in rheumatoid arthritis and other chronic inflammatory diseases.^[99,100] The efficacy and safety of this class of agents in controlling the inflammatory response of synovial tissues to blood in hemophilia patients must also be studied. Synovial membrane vascularization is a prominent feature of blood-induced joint damage, and antiangiogenesis drugs (e.g., bevacizumab) may be helpful in this setting, as they are in some forms of cancer.^[101] However, a cautionary note is warranted regarding the use of biological agents in hemophilia, since the patients most likely to benefit are young children who have experienced a first hemarthrosis. Pediatric trials of these drugs can only be considered after their efficacy and safety have been clearly demonstrated in preclinical models and dose-finding studies in adults.

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Key Issues

CME Information

References

CME

Management of Joint Bleeding in Hemophilia

Target Audience and Goal Statement

Disclosures

Author(s)

Mindy L. Simpson, MD

Leonard A. Valentino, MD

Editor

Elisa Manzotti

CME Author(s)

Laurie Barclay, MD

CME Reviewer(s)

Nafeez Zawahir, MD

Accreditation Statements

For Physicians

Instructions for Participation and Credit

Management of Joint Bleeding in Hemophilia: Five-year View

Five-year View

Table of Contents

References

Faculty and Disclosures

Author(s)

Mindy L. Simpson, MD

Leonard A. Valentino, MD

Editor

Elisa Manzotti

CME Author(s)

Laurie Barclay, MD

CME Reviewer(s)

Nafeez Zawahir, MD

CME

Management of Joint Bleeding in Hemophilia

Target Audience and Goal Statement

Disclosures

Author(s)

Mindy L. Simpson, MD

Leonard A. Valentino, MD

Editor

Elisa Manzotti

CME Author(s)

Laurie Barclay, MD

CME Reviewer(s)

Nafeez Zawahir, MD

Accreditation Statements

For Physicians

Instructions for Participation and Credit

Management of Joint Bleeding in Hemophilia: Five-year View

Five-year View

Table of Contents