Published online May 1, 2008
PEDIATRICS
Vol. 121
No. 5
May 2008, pp.
e1426-e1429
(doi:10.1542/peds.2007-1944)
Challenges Associated With Prolonged Survival of Patients With Thalassemia: Transitioning From Childhood to Adulthood
Khaled Musallam, BSca,
Maria D. Cappellini, MDb and
Ali Taher, MDc
a Faculty of Medicine
c Department of Internal Medicine, Hematology-Oncology Division, American University of Beirut Medical Center, Beirut, Lebanon
b Department of Internal Medicine, University of Milan, Milan, Italy
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ABSTRACT
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Thalassemia is a chronic condition that presents a range of clinical and psychosocial challenges. Although recent advances in the treatment of thalassemia can prolong patient life spans, problems may arise when patients are transferred from pediatric health care settings to adult health care settings. These issues and our recommendations for handling them are discussed.
Key Words: health care transition • psychosocial • thalassemia
Over the past few decades, medicine has seen the clinical evolution of thalassemia major, an inherited disorder of blood hemoglobin. When thalassemia was first described, the prognosis for patients was poor because the condition was incompatible with life. However, as research into this debilitating disease progressed, it was discovered that continuous blood transfusions enabled patients to survive for up to 1 decade, until complications associated with iron toxicity from the ongoing transfusions led to death. Subsequent breakthroughs in iron chelation therapy, with deferoxamine and deferiprone, meant that patients with thalassemia could have a normal life span, provided they received regular transfusions and iron chelation therapy. However, because patients face the burden of a chronic illness, psychosocial issues can be a significant impediment to compliance with ongoing therapy. One of the primary challenges these patients encounter is adjusting to the transition from a pediatric health care system to an adult health care system.
Because of the nature of the poor prognosis, in the early days of treatment thalassemia was regarded as a condition of the young and was treated by pediatric teams. However, improvements in therapy and the availability of effective iron chelation regimens mean that patients can be expected to survive beyond adolescence, and adults now constitute 44% of the thalassemic population in the United States.1 Whereas in the past most children with special health care needs died before reaching adulthood, now >90% of the 
500000 children who transition into adulthood each year in the United States survive.2,3 This report discusses the clinical and psychosocial effects of thalassemia as patients move from the pediatric age group to adulthood and suggests possible ways to ease this important transition.
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CLINICAL PERSPECTIVE
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Thalassemia is a significant issue for medical centers throughout the world, because they must not only accommodate the constant demand for blood transfusions but also support the need for self-administered iron chelation therapy. Unfortunately, many centers remain focused primarily on the care of pediatric patients. If adult patients need to receive their treatment at such a center, then the staff members are faced with a number of challenges. For example, equipment at these thalassemia centers is designed primarily for pediatric patients. Also, nurses and maintenance staff members generally are accustomed to dealing with children and may not be familiar with adult-specific issues.1 For example, adult patients with thalassemia experience a spectrum of clinical complications not seen in pediatric patients, such as osteoporosis, cardiac disease, organ failure, and pain, which require the attention of specialists not commonly found in the pediatric setting.1 If adult patients with thalassemia choose to move from thalassemia centers to adult health care facilities, such as standard hospitals, then they face another problem because of the lack of thalassemia specialists proficient in managing their condition. The ideal solution would be to establish efficient communication between the medical committees at the 2 institutions. High-quality relationships and constant communication between health care providers seem to have a significant positive effect on the management of any chronic illness, especially when the illness requires such transitions.4
Compliance with treatment is the most important factor in ensuring the desired outcome for patients with thalassemia. However, psychosocial issues may negatively influence compliance, an effect that may differ significantly between children and adults. Although iron chelation therapy has been available for many years, the burdensome regimen of subcutaneous deferoxamine infusions can have a significant impact and affect compliance with treatment. The recent emergence of oral iron chelation therapy (with deferiprone) has helped lessen the burden of deferoxamine infusions and thus is improving compliance.5 If we, as treating physicians, educate our patients about the ongoing research aimed toward improving thalassemia care, we may give them the incentive to abide by current treatment plans and to look forward to future advances in treatment modalities.
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PSYCHOSOCIAL PERSPECTIVE
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Factors in Maturation
Regardless of gender, culture, religion, and socioeconomic status, the process of physical aging is accompanied by a psychological maturation that is reflected in an individual's interpretation of self, existence, and interpersonal relationships. Any experience spanning an individual's lifetime, such as living with a chronic disease, will inevitably affect his or her psychological development. Both the natural course of the disease and any interventions should always be taken into consideration in this regard. To do this effectively, one must identify the impact the disease and treatment may have on all facets of the patient's life. As far as patients with thalassemia are concerned, the following areas differ substantially in terms of the impact on pediatric and adult patient populations and therefore require special attention from health care providers and adult thalassemia care facilities.
Maturity and Education
Children with thalassemia usually are not aware of the gravity of their disease but, as they mature, they become more aware of the nature of thalassemia, either by their own accord or through their health care provider. This realization may create a level of denial and cause distress, but we cannot deny children's rights to knowledge regarding their own physical state. To overcome this, parents and physicians must be aware of an age-appropriate approach to disease education. They should consider the level of the patients' education and their psychological maturity before gradually discussing the reality of thalassemia as a disease. An age-appropriate approach might be to start with cartoon illustrations for children and eventually provide detailed scientific information supplemented by articles for young adults. The patients may then accept the seriousness of their disease and, it is hoped, be fully compliant with treatment.
The use of an adult psychologist may be helpful in providing a link between patients, school officials, the family, and the physician. It must kept in mind that, throughout the early course of the disease and its management, absenteeism from school and a lack of energy when performing academic duties may affect the education of the patient. Because of these issues, patients with thalassemia may find their future compromised. Psychologists may assist in alleviating these burdens and improve the chances of patients succeeding throughout their academic paths and future careers.
Independence
Aiming for an independent lifestyle, both physically and emotionally, usually begins at adolescence. Psychological separation and growth of self throughout adolescence are essential for the normal process of maturation of an individual. It is at this time of life that an individual begins to question the meanings of birth, existence, and death and the nature of causality. For patients with thalassemia, 2 main problems may arise. First, the adolescent patient has already been affected by a chronic illness. Overprotective, negligent, or hostile parents, uncompassionate peers and society, the burden of disease complications, blood transfusions, and iron chelation pumps all may contribute to a negative view of the world. Second, these views may be accompanied by feelings of fear, shame, and denial. All of these can be lessened through appropriate attention and guidance; this is why the roles of the parents and psychologist are fundamental early in childhood, to provide a "normal" atmosphere as the child develops, by giving support and direction. Without this, the development of the adult may be affected.
When adolescents with thalassemia embark on a new journey to become independent from their parents, they are faced not only with the burden of a psyche affected by chronic illness but also with additional serious responsibilities, compared with their healthy peers. The management of their condition requires much attention and planning, which previously might have been managed by their parents. For this reason, parents and physicians should involve patients with thalassemia in their own treatment plans, to create eventually self-sufficient adolescents. The patients should be taught to be responsible for their own regular appointments with thalassemia clinical and psychosocial specialists, blood transfusions and iron chelation sessions, and finances. It is the latter that may present the biggest problem, because it is undeniable that thalassemia treatment is costly. With time, adults with thalassemia will need to bear the weight of other financial responsibilities and the regular costs of treatment, which may worsen the situation. As discussed, adults with thalassemia may need to switch to an adult health care system where treatment is much more expensive than that provided at the thalassemia centers. Adolescent patients should be made aware of these future problems and ideally should be provided with some assistance (either governmental or through medical insurance companies).
Work
According to the study by Laurice,1 adult patients with thalassemia spend an average of 271 hours per month on care (including blood transfusions, deferoxamine therapy, doctor appointments, taking medications, medically related telephone calls, and travel time). Consequently, the chosen career path for a patient with thalassemia should be selected with consideration of the financial cost, the patient's physical capabilities, and the time-consuming nature of this disease. Once again, the role of the psychologist in the choice of a career path is important. Communication between health care providers and employers would be beneficial for creating a better working environment without interfering with the patient's positive attitude toward normality.
Interpersonal Relationships
The way in which patients with a chronic illness interact with others is often determined by their childhood and development. As children, patients with thalassemia might have felt the need to hide their disease from their peers out of shame of being different or not accepted by society (some children or adults may be so misinformed as to think the disease is infectious). The course of the disease and management also might have prevented the children from having enough time or energy to engage in play with their peers. Conversely, the patients might have felt comfortable enough to share their disease status with their friends and, in turn, developed much more healthy relationships. Psychologists should be aware of the fact that encouraging healthy peer relationships for young patients with thalassemia is important not only for improving compliance (because the patient then may not attempt to evade treatment to feel more "normal") but also for developing their future ability to engage in healthy relationships as adults and for easing much of the psychological trauma of thalassemia. Motivating self-acceptance and confidence in relationships may build a solid foundation for the patients. It is also worth noting that patients with thalassemia may be more likely to engage in drug abuse, smoking, and alcohol drinking as a means to fit into society or to escape their condition.
Marriage
Before recent advancements in thalassemia treatment, the concept of marriage and family was inconceivable to patients with thalassemia. As treatment has improved and patient survival has lengthened, living long enough to marry and to have children is now a real possibility. However, this may be affected by the natural course of the disease and inherent complications, because some abnormalities in sexual differentiation may occur. Because this can severely affect the physical and emotional relationships of adults with thalassemia with members of the opposite gender, it requires extensive counseling from an adult psychologist, especially as affected patients enter new romantic relationships. Moreover, alternative approaches to pregnancy should be discussed with the patient if natural conception is not possible.
Psychiatric Disorders
In the biopsychosocial model describing the pathogenesis of psychiatric conditions, the development of psychiatric disorders is related to the impact of the following sequence of events: genetic predisposition, early life events, and current stressful life events.6 It can be seen clearly that thalassemia could affect the latter 2 factors. This means that the chances of patients with thalassemia developing serious psychiatric conditions or mood disorders, beyond the anxiety, guilt, shame, or depression that may accompany any normal stressful situation, are increased. Therefore, careful monitoring and any necessary referrals to a psychiatrist by the specialized adult psychologist are recommended.
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CONCLUSIONS
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In light of the clinical and psychosocial difficulties in shifting from a pediatric health care system to an adult health care system we have discussed here, the following modifications to thalassemia management could be beneficial. First, modification of the health care system to provide necessary medical attention for adult patients with thalassemia could be achieved by employing more thalassemia specialists and adult psychologists at health care facilities or by improving thalassemia pediatric centers to include facilities equipped for proper handling of adult medical and psychosocial issues. Well-organized communication between the 2 systems is also suggested. A patient-centered, flexible, responsive, comprehensive, well-coordinated transition should be the primary target.6 In fact, many attempts are made each day to develop policies aimed at achieving the desired outcome for thalassemia treatment transitions. One example is the consensus statement on health care transitions for young adults with special health care needs, whose goals are to "ensure that physicians who provide primary or subspecialty care to young people with special health care needs (1) understand the rationale for transition from child-oriented to adult-oriented health care; (2) have the knowledge and skills to facilitate that process; and (3) know if, how, and when transfer of care is indicated."7
Second, a practical approach to ensure adequate psychological management is to consider scheduling regular appointments (every 3 or 6 months) with an adult psychologist who is informed by the thalassemia specialist about the specific needs of the patient. Funding boards should include the expenses of such visits in the assigned aid for patients. The psychologist's main role is to help patients and parents discuss and express their feelings about the illness, to help patients accept the illness and take care of themselves, to facilitate a normal lifestyle, and to support full control of the various issues mentioned in this report.
Third, competent primary care providers should be open-minded about the psychological aspects of having and treating an inherited disease and should be available to accompany and to support patients throughout their development. The organizational structure of the health care facility should be well prepared for the latter. If experienced specialists always rotate to different wards, then they will not be able to monitor the developmental well-being of patients. Proper scheduling is necessary to ensure that patients are treated by the same specialist each time.
Fourth, more research should focus on the needs of patients with thalassemia who are transferring from pediatric to adult care. Determining the extent to which adolescents are receiving health care transition services and understanding the main factors influencing the adequacy of such services are important gaps in current research.4 Controlled studies that involve patients and their families may provide convincing data, encouraging authorities to become involved in funding the process of service modification, recruiting the appropriate personnel, and providing adequate medical insurance. Qualitative and quantitative studies should be the main focus of health care societies in searching for ideal health care for thalassemia8,9 (Table 1).
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FOOTNOTES
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Accepted Nov 12, 2007.
Address correspondence to Ali Taher, MD, Department of Internal Medicine, American University of Beirut Medical Center, PO Box 11-0236, Riad El Solh 1107 2020, Beirut, Lebanon. E-mail: ataher{at}aub.edu.lb
The authors have indicated they have no financial relationships relevant to this article to disclose.
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PEDIATRICS (ISSN 1098-4275). ©2008 by the American Academy of Pediatrics
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ABSTRACT
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