Challenges Associated With Prolonged Survival of Patients With Thalassemia

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DOI: 10.1542/peds.2007-19442008;121;e1426Pediatrics

Khaled Musallam, Maria D. Cappellini and Ali Taher

Transitioning From Childhood to AdulthoodChallenges Associated With Prolonged Survival of Patients With Thalassemia:

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SPECIAL ARTICLE

Challenges Associated With Prolonged Survival of

Patients With Thalassemia: Transitioning FromChildhood to Adulthood

KhaledMusallam, BSca, MariaD. Cappellini, MDb, Ali Taher,MDc

aFaculty of Medicine and cDepartment of Internal Medicine, Hematology-Oncology Division, American University of Beirut Medical Center, Beirut, Lebanon; bDepartment

of Internal Medicine, University of Milan, Milan, Italy

The authors have indicated they have no financial relationships relevant to this article to disclose.

ABSTRACT

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 recommen-

dations for handling them are discussed.

OVER THE PAST few decades, medicine has seen the clinical evolution of thalas-semia 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, withdeferoxamine 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 effectiveiron 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 500 000 children who transition intoadulthood 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.

CLINICALPERSPECTIVE

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 centersto 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 signifi-

cantly 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

www.pediatrics.org/cgi/doi/10.1542/

peds.2007-1944

doi:10.1542/peds.2007-1944

Key Words

health care transition, psychosocial,

thalassemia

Accepted for publication 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: [email protected]

PEDIATRICS (ISSNNumbers:Print, 0031-4005;

Online, 1098-4275). Copyright 2008by the

AmericanAcademy of Pediatrics

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with treatment. The recent emergence of oral iron che-

lation 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 im-

proving thalassemia care, we may give them the incen-

tive to abide by current treatment plans and to look

forward to future advances in treatment modalities.

PSYCHOSOCIAL PERSPECTIVE

Factors in Maturation

Regardless of gender, culture, religion, and socioeco-

nomic status, the process of physical aging is accompa-

nied by a psychological maturation that is reflected in an

individuals interpretation of self, existence, and inter-

personal relationships. Any experience spanning an in-

dividuals lifetime, such as living with a chronic disease,

will inevitably affect his or her psychological develop-

ment. Both the natural course of the disease and any

interventions should always be taken into considerationin this regard. To do this effectively, one must identify

the impact the disease and treatment may have on all

facets of the patients life. As far as patients with thalas-

semia are concerned, the following areas differ substan-

tially in terms of the impact on pediatric and adult pa-

tient 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 dis-

tress, but we cannot deny childrens rights to knowledge

regarding their own physical state. To overcome this,

parents and physicians must be aware of an age-appro-

priate approach to disease education. They should con-

sider the level of the patients education and their psy-

chological 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 infor-

mation supplemented by articles for young adults. The

patients may then accept the seriousness of their diseaseand, 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 man-

agement, absenteeism from school and a lack of energy

when performing academic duties may affect the educa-

tion of the patient. Because of these issues, patients with

thalassemia may find their future compromised. Psy-

chologists 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 andemotionally, usually begins at adolescence. Psychologi-

cal separation and growth of self throughout adoles-

cence 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, anddeath and the nature of causality. For patients with

thalassemia, 2 main problems may arise. First, the ado-lescent patient has already been affected by a chronic

illness. Overprotective, negligent, or hostile parents, un-

compassionate peers and society, the burden of diseasecomplications, 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 re-

sponsibilities, compared with their healthy peers. Themanagement of their condition requires much attention

and planning, which previously might have been man-

aged by their parents. For this reason, parents and phy-

sicians should involve patients with thalassemia in their

own treatment plans, to create eventually self-sufficientadolescents. The patients should be taught to be respon-

sible for their own regular appointments with thalasse-

mia clinical and psychosocial specialists, blood transfu-sions and iron chelation sessions, and finances. It is the

latter that may present the biggest problem, because it isundeniable 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 toan 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 monthon care (including blood transfusions, deferoxamine

therapy, doctor appointments, taking medications, med-

ically related telephone calls, and travel time). Conse-

quently, the chosen career path for a patient with thalas-

semia should be selected with consideration of thefinancial cost, the patients 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

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and employers would be beneficial for creating a better

working environment without interfering with the pa-

tients positive attitude toward normality.

Interpersonal Relationships

The way in which patients with a chronic illness interactwith 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 misinformedas to think the disease is infectious). The course of the

disease and management also might have prevented thechildren 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 sta-

tus with their friends and, in turn, developed muchmore healthy relationships. Psychologists should be

aware of the fact that encouraging healthy peer relation-

ships for young patients with thalassemia is important

not only for improving compliance (because the patient

then may not attempt to evade treatment to feel morenormal) but also for developing their future ability to

engage in healthy relationships as adults and for easing

much of the psychological trauma of thalassemia. Moti-

vating self-acceptance and confidence in relationships

may build a solid foundation for the patients. It is alsoworth noting that patients with thalassemia may be

more likely to engage in drug abuse, smoking, and alco-

hol 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 topatients 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. Be-

cause this can severely affect the physical and emotionalrelationships 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 thepatient if natural conception is not possible.

Psychiatric Disorders

In the biopsychosocial model describing the pathogene-

sis of psychiatric conditions, the development of psychi-atric 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 seenclearly that thalassemia could affect the latter 2 factors.

This means that the chances of patients with thalassemia

developing serious psychiatric conditions or mood disor-

ders, beyond the anxiety, guilt, shame, or depression

that may accompany any normal stressful situation, are

increased. Therefore, careful monitoring and any neces-sary referrals to a psychiatrist by the specialized adult

psychologist are recommended.

CONCLUSIONS

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 ben-eficial. 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 cen-ters 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,

TABLE 1 Thalassemia Pediatric to Adult Health Care Transition

Domain Challenge Recommendations

Clinical

Setting Hardware Modification of equipment

Staff Lack of adult specialists and adult psychologists in pediatric

thalassemia centers

Employment of missing staff members

Lack of thalassemia specialists in adult health care centers Employment of missing staff members

Authorities Lack of insight regarding health care transit ion problems Provision of funding and policies

Research Lack of studies about the extent to which adolescents are receiving

health care transition services

More research about psychosocial aspects and enhancement

of treatment modalities to improve compliance

Psychosocial

Education Increased awareness regarding the nature of the disease Psychosocial management

Independence Physical Financial and directive support

Psychological Psychosocial management

Work Absenteeism because of occupancy with management or lack

of energy

Psychosocial management

Relationships Limited because of lack of energy, occupancy, shame, nonacceptance,

or denial

Psychosocial management

Marriage Financial, sexual, and emotional burdens Psychosocial management

Psychiatric disorders Increased vulnerability Psychosocial management

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well-coordinated transition should be the primary tar-

get.6 In fact, many attempts are made each day to de-

velop 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 psy-

chological management is to consider scheduling regular

appointments (every 3 or 6 months) with an adult psy-

chologist 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 psychologists main role is to help

patients and parents discuss and express their feelings

about the illness, to help patients accept the illness andtake care of themselves, to facilitate a normal lifestyle,

and to support full control of the various issues men-

tioned 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 specialisteach time.

Fourth, more research should focus on the needs of

patients with thalassemia who are transferring from pe-

diatric to adult care. Determining the extent to which

adolescents are receiving health care transition servicesand understanding the main factors influencing the ad-

equacy 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 ofservice modification, recruiting the appropriate person-

nel, and providing adequate medical insurance. Qualita-tive and quantitative studies should be the main focus of

health care societies in searching for ideal health care for

thalassemia8,9 (Table 1).

REFERENCES

1. Laurice M. Caring for adult thalassemics in a pediatric world.

Presented at the Cooleys Anemia Eighth Symposium, Lake

Buena Vista, FL; March 1719, 2005

2. Newacheck PW, Taylor WR. Childhood chronic illness: preva-

lence, severity, and impact. Am J Public Health. 1992;82(3):

364371

3. Blum RW. Transition to adult health care: setting the stage. J

Adolesc Health. 1995;17(1):35

4. Scal P, Ireland M. Addressing transition to adult health care foradolescents with special health care needs. Pediatrics. 2005;

115(6):16071612

5. Zahed L, Mourad FH, Alameddine R, Aoun S, Koussa S, Taher

A. Effect of oral iron chelation therapy with deferiprone (L1) on

the psychosocial status of thalassemia patients. Haematologia.

2002;31(4):333339

6. American Psychiatric Association. Diagnostic and Statistical Man-

ual of Mental Disorders. 4th ed. Washington, DC: American Psy-

chiatric Association; 2000

7. American Academy of Pediatrics, American Academy of Family

Physicians, American College of Physicians, American Society of

Internal Medicine. A consensus statement on health care tran-

sitions for young adults with special health care needs. Pediatrics.

2002;110(6):13041306

8. Galanello R. A thalassemic child becomes adult. Rev Clin Exp

Hematol. 2003;7(1):421

9. Reiss J, Gibson R. Health care transition: destination unknown.

Pediatrics. 2002;110(6):13071314

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DOI: 10.1542/peds.2007-1944

2008;121;e1426PediatricsKhaled Musallam, Maria D. Cappellini and Ali Taher

Transitioning From Childhood to AdulthoodChallenges Associated With Prolonged Survival of Patients With Thalassemia:

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Challenges Associated With Prolonged Survival of Patients With Thalassemia