Objective Status

  • 2 Baseline only
  • 2 Developmental
  • 0 Research

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About the Workgroup

Approach and Rationale

Blood disorders result when blood cannot clot or flow through the body properly. The type and severity of blood disorders often affect the amount of complications. Access to care, prompt diagnosis, proper treatment, and routine monitoring can give patients the best possible outcomes.

Core objectives selected by the BDBS Workgroup aim to decrease the severity of symptoms for patients with inherited blood disorders. Sickle cell disease is an inherited blood disorder that causes the body to produce sickle-shaped red blood cells that restrict the delivery of oxygen to body tissues. Hemophilia is a genetic disorder in which blood does not clot properly. Extended bleeding into joints and muscles is common in hemophilia and can lead to loss in range of motion, strength, and limb function. The core objectives can be achieved by increasing the number of people with sickle cell disease who receive disease-modifying therapies, and by decreasing the number of people with hemophilia who experience a joint bleed.

Developmental objectives supported by the workgroup include increasing the proportion of people who donate blood and increasing the proportion of people with von Willebrand disease seen in specialty centers. Von Willebrand disease is another rare inherited blood disorder that causes bleeding due to a failure in platelet function. The workgroup will focus on monitoring and securing data sources for the coming decade.

Understanding Blood Disorders and Blood Safety

Both hemophilia and sickle cell can result in frequent hospitalizations and outpatient visits, lower productivity at work, missed days of work and school, and a lower quality of life.

There are approximately 20,000 people in the United States with hemophilia.1 Repeated bleeding in joints can cause joint disease and disability, which is the most common complication of hemophilia. For patients with severe hemophilia, taking prescribed treatment regularly before a bleed occurs can lower the frequency of joint bleeds and prevent joint damage.2

Sickle cell disease affects over 100,000 Americans, predominately in black and Latino populations. Hydroxyurea has been an effective disease-modifying therapy used to treat sickle cell for decades.3,4,5,6,7,8,9  Given that national estimates on sickle cell population are lacking in the literature, the national prevalence of hydroxyurea use is also unknown. Including Medicare data in Healthy People 2030 will allow for the tracking and monitoring of hydroxyurea use to improve quality care for sickle cell anemia.

Citations

1.

Centers for Disease Control and Prevention. (2019). Data & Statistics on Hemophilia. Retrieved from https://www.cdc.gov/ncbddd/hemophilia/data.html

2.

Manco-Johnson. M.J., et al. (2007). Prophylaxis Versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia. New England Journal of Medicine, 357(6), 535-544. DOI: 10.1056/NEJMoa067659

3.

Stettler, N., McKiernan, C.M., Melin, C.Q., Adejoro, O.O., & Walczak, N.B. (2015). Proportion of Adults With Sickle Cell Anemia and Pain Crises Receiving Hydroxyurea. JAMA, 313(16), 1671-1672. DOI: 10.1001/jama.2015.3075

4.

Oyeku, S.O., et al. (2013). Parental and Other Factors Associated with Hydroxyurea Use for Pediatric Sickle Cell Disease. Pediatric Blood & Cancer, 60(4), 653–8. DOI: 10.1002/pbc.24381

5.

Haywood, C. Jr, et al. (2011). Examining the Characteristics and Beliefs of Hydroxyurea Users and Nonusers Among Adults with Sickle Cell Disease. American Journal of Hematology, 86(1), 85-87. DOI: 10.1002/ajh.21883

6.

Brawley, O.W., et al. (2008). National Institutes of Health Consensus Development Conference Statement: Hydroxyurea Treatment for Sickle Cell Disease. Annals of Internal Medicine, 148(12), 932-938. DOI: 10.7326/0003-4819-148-12-200806170-00220

7.

Green, N.S. & Barral, S. (2014). Emerging Science of Hydroxyurea Therapy for Pediatric Sickle Cell Disease. Pediatric Research, 75(1–2), 196-204. DOI: 10.1038/pr.2013.227

8.

U.S. Department of Health and Human Services. (2014). Evidence-based Management of Sickle Cell Disease [PDF file]. Retrieved from https://www.nhlbi.nih.gov/sites/default/files/media/docs/sickle-cell-disease-report%20020816_0.pdf

9.

Nevitt, S.J., Jones, A.P., & Howard, J. (2017). Hydroxyurea (Hydroxycarbamide) for Sickle Cell Disease. Cochrane Database of Systematic Reviews, 20(4). DOI: 10.1002/14651858.CD002202.pub2