Briefly describe a specific question, or set of related questions, about a health care test or treatment that this program should consider.

For "adult" patients with sickle cell disease what are the comparative related health complications which lead to morbidity? As a result, are there preventative measures that could be taken?

Does your question include a comparison of different health care approaches? (If no, your topic will still be considered.)

yes

If yes, explain the specific technologies, devices, drugs, or interventions you would like to see compared:

Preventive care versus no care.

What patients or group(s) of patients does your question apply to? (Please include specific details such as age range, gender, coexisting diagnoses, and indications for therapy.)

Adult patients with sickle cell disease (across ethnic groups) which have begun to experience related complications such as; cardio vascular diseases, urological diseases, pulmonary disease or skeletal diseases.

Are there subgroups of patients that your question might apply to? (For example, an ethnic group, stage or severity of a disease.)

Age 18 - 30 and 30 - 60 and 60 +

Describe the health-related benefits you are interested in. (For example, improvements in patient symptoms or problems from treatment or diagnosis.)

I am interested in discovering if early intervention in sickle cell disease complication preventions will result in prolonged patient health & reduced health care cost.

Describe any health-related risks, side effects, or harms that you are concerned about.

I am concerned about pre-mature disability and death of persons with sickle cell disease.

Appropriateness for EHC Program

Does your question include a health care drug, intervention, device, or technology available (or likely to be available) in the U.S.?

no

Which priority area(s) and population(s) does this topic apply to? (check all that apply)

EHC Priority Conditions (updated in 2008)

• Arthritis and nontraumatic joint disorders
• Cardiovascular disease, including stroke and hypertension
• Depression and other mental health disorders
• Functional limitations and disability
• Pulmonary disease/asthma

AHRQ Priority Populations

• Low income groups
• Minority groups
• Women
• Elderly
• Individuals with special health care needs, including individuals with disabilities or who need chronic care or end-of-life health care

Federal Health Care Program

• Medicaid
• Medicare

Importance

Describe why this topic is important.

I am an advocate for people "living" with sickle cell disease. What our community is concerned about is the adult population being left behind when it comes to preventative healtcare. The early death rate is too high and is related to complications of sickle cell disease.

What specifically motivated you to ask this question? (For example, you are developing a clinical guideline, working with a policy with large uncertainty about the appropriate approach, costly intervention, new research you have read, items in the media you may have seen, a clinical practice dilemma you know of, etc.)

People with sickle cell disease are living longer. The long-term health complications associated with sickle cell disease have not been qualitatively analyzed to warrant preventative care. (heart failure, bone loss, kidney damage, etc.) Thus, an aging population affects healthcare cost.

Does your question represent uncertainty for clinicians and/or policy-makers? (For example, variations in clinical care, controversy in what constitutes appropriate clinical care, or a policy decision.)

yes

If yes, please explain:

Care and analysis of children with sickle cell disease has been addressed. Agencies like HHS have made an effort to address/fund early childhood sickle cell screening and care. The result is children with sickle cell disease living longer. However, as they become adults their issues/care/treatment/complication analysis have been unaddressed.

Potential Impact

How will an answer to your research question be used or help inform decisions for you or your group?

We will pass this information on to our legislative and medical professional partners. The results could impact health care cost and thereby cause changes in treatment protocols.

Describe the timeframe in which an answer to your question is needed.

How about by 21 Jan 14?

Describe any health disparities, inequities, or impact on vulnerable populations your question applies to.

The population that suffers with sickle cell disease falls into all three categories of health dispartiy, inequity and vulnerable population. The number of people who suffer with sickle cell diease outnumber cystic fibrosis 3 to 1, however; it is underfunded by the same margin. The population usually falls into the "minority" population status, however, is migrating into mixed populations because of lack of education and awareness.

Nominator Information

Other Information About You: (optional)

Please choose a description that best describes your role or perspective: (you may select more than one category if appropriate)

We will pass this information on to our legislative and medical professional partners. The results could impact health care cost and thereby cause changes in treatment protocols.

Are you making a suggestion as an individual or on behalf of an organization?

Organization

Please tell us how you heard about the Effective Health Care Program

Research