Sickle cell disease is a complex genetic disorder with multi-system manifestations that requires specialized comprehensive care to achieve an optimal outcome. Comprehensive medical care includes ongoing patient and family education, periodic comprehensive evaluations and other disease-specific health maintenance services, timely and appropriate treatment of acute illness, and genetic counseling.

In addition to medical treatment, the management of sickle cell disease requires sensitivity to important psychosocial implications of the disease and services to address them. Barriers to appropriate health care include inadequate insurance coverage, transportation, and/or access to health care providers with expertise in the management of sickle cell disease. Important stresses that often affect a family's ability to cope with sickle cell disease include the economic and educational consequences of time lost from work and school and the impact of chronic illness on normal family functioning, including adjustment issues for non-affected siblings. Families live daily with the knowledge that unpredictable acute illnesses will interrupt daily life, and there are often feelings of powerlessness, frustration, and even anger. A general lack of community awareness about sickle cell disease and fear of stigmatization may limit the support available from extended family, friends, and the community at large. Prior experience with health care providers who lack knowledge, sensitivity, and compassion may contribute to delays in seeking appropriate health care and may engender adversarial relationships between families and providers. Failure to appreciate ethnic and cultural differences between providers and patients and families may also contribute to misunderstanding and lack of trust. Thus it is imperative that providers take time to listen to the concerns of patients and families, that they be sensitive to psychosocial as well as medical needs, and that they assist families in accessing available resources as needed.

Six core outcomes for Children with Special Health Care Needs* under the federal Healthy People 2010 Objectives provide guiding principles for the care of children with sickle cell disease.

1. All children with sickle cell disease will receive regular and ongoing comprehensive care within a medical home.

Optimal care requires the active involvement of professionals in pediatrics and hematology, nursing, social work, psychology, genetics, education, and counseling. The services they provide need to be coordinated through an appropriate medical home. For many patients, the medical home will be a multi-disciplinary sickle cell clinic that coordinates all aspects of comprehensive care in collaboration with the child's primary care physician or that provides specialty and primary care in one setting. In other cases the medical home may be provided by a knowledgeable primary care provider from whom the patient receives day-to-day care, with periodic referrals to sickle cell specialists for comprehensive evaluations and for the management and treatment of severe, life-threatening complications. The location of the medical home and extent to which care is provided by the primary care provider versus the multi-disciplinary sickle cell clinic will vary among patients and communities and will depend in part on the expertise of the primary care provider, access to a multi-disciplinary sickle cell clinic, family preference, and the frequency and severity of disease manifestations. Good communication among the family, primary care providers, and subspecialists is essential to provide coordinated care and to establish and maintain trust.

2. All families of children with sickle cell disease should have adequate private and/or public insurance to pay for the services they need.

Almost every child in the U.S. with sickle cell disease is eligible for health care coverage by commercial insurance, Medicaid, Medicare, SSI, or the Children's Health Insurance Plan (CHIP). It is imperative that providers assist families and patients to obtain and maintain adequate insurance coverage. For patients insured by managed care plans, ongoing access to providers with expertise in sickle cell disease may require advocacy by primary care providers and anticipation of payer requirements for prior authorization for specialty services.

3 All children with sickle cell disease will be screened early and continuously for special health care needs.

Individuals with sickle cell disease require ongoing screening for a variety of disease-related problems. All patients with sickle cell disease should have regularly scheduled comprehensive medical evaluations to review previous disease manifestations, document important baseline physical findings and laboratory values, monitor growth and development, and screen for signs of chronic organ damage. Comprehensive evaluations also provide an ideal setting for providing age-appropriate family and patient education and for evaluating and addressing psychosocial issues.

4. Services for children with sickle cell disease and their families should be organized in ways that families can use them easily.

Important health and other services may be available but difficult to access because of problems with transportation or parking or a lack of insurance coverage or prior authorization from managed care plans. Access to multidisciplinary comprehensive evaluations can be facilitated by the provision of outreach clinics in communities distant from tertiary care centers. Because acute illness can prove rapidly life-threatening, it is imperative that every child with sickle cell disease have a plan for around-the-clock access to a medical facility where knowledge and perspective about sickle cell disease is available, where evaluation and treatment can be promptly delivered, and where providers have access to baseline information about the patient. Other important services include social services, neurocognitive evaluations, and educational and vocational planning and counseling - all of which require communication and coordination among providers, educators, patients, and families. In many communities, patient and family support groups and other valuable supportive, educational, and counseling services are organized and provided by community-based groups, such as local chapters of the Sickle Cell Disease Association of America.

5. Families of children with sickle cell disease will participate in health care decision-making at all levels and will be satisfied with the services they receive.

Parents are ultimately responsible for decisions about their child. In order for parents to participate in decisions regarding their child's health care, they must receive accurate and ongoing education about the disease and about a variety of treatment options. Education should be provided in an open, non-judgmental, and mutually respectful environment. Providers should recognize that personal and cultural beliefs about illness and existing stresses and support systems may greatly impact the family's ability to cope with sickle cell disease and may appropriately influence their decisions. Patients and families should be encouraged to provide feedback about the care they receive and suggestions to improve it.

6. All youth with sickle cell disease will receive the services necessary to make appropriate transitions to all aspects of adult life, including adult health care, work, and independence.

The families of children with sickle cell disease should be encouraged to set appropriate goals for their children and to develop realistic strategies to achieve those goals. School personnel must be educated about sickle cell disease and encouraged to accommodate repeated and often unpredictable absences. During middle childhood and adolescence, education about sickle cell disease is increasingly directed towards the patient, as well as the family, with the expectation that adolescents will be knowledgeable about their disease and its management. Counseling about higher education and vocational choices should be realistic but avoid underestimating the patient's potential. The transition from pediatric to adult health care providers and institutions can be traumatic and requires prior discussion, preparation, and planning. The current shortage of health care providers with interest and expertise in the treatment of adults with sickle cell disease is a major problem that must be addressed.

* A National Agenda for Children with Special Health Care Needs. Measuring Success for Healthy People 2010: A working document. Maternal and Child Health Bureau, HRSA, September, 1999.

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