HEMATOONCOLOGY DIVISION

EVIDENCE BASED CASE REPORT

QUALITY OF LIFE IN CHILDREN

WITH THALASSEMIA MAJOR

BONNIE ARSENO
INTRODUCTION
 Thalassemia Genetic disorder

Autosomal recessive

An estimated 1,5% of worlds

population are carriersProblem
of thalassemia
all over the world

Trachtenberg dkk.
Relationship among chelator adherence, change in chelators, and quality of life in Thalassemia.
Qual Life Res.2014(23):227788.
Caocci G.
Health related quality of life in Middle Eastern children with beta-thalassemia.
BMC Blood Disorders 2012: 12:6
 Impaired
Ineffective
Thalassemia synthesis of the
erythropoiesis
globin chains

Decreased
Anemia production of
red blood cells

BLOOD
TRANSFUSION

Galanello R. Beta-thalassemia. Orphanet Journal of Rare Diseases 2010:5:11

 Repeated Formation of increased
blood ineffective absorbtion of
transfusions erythropoiesis iron

Elevated levels
Multiple organ of iron in the
inolvement serum

Galanello R. Beta-thalassemia. Orphanet Journal of Rare Diseases 2010:5:11

Abetz L BJF, Jones P, Rofail D.
The impact of iron overload and its treatment on quality of life: results from a literature review.
Health and Quality of Life Outcomes. 2006(4:73).
Mishra A K TA.
Iron Overload in Beta Thalassaemia Major and Intermedia Patients.
MAEDICA a Journal of Clinical Medicine 2013(8(4)):328-32.
Bejaoui M GN.
Beta Thalassemia Major in a Developing Country: Epidemiological, Clinical and Evolutionary Aspects.
Mediterr J Hematol Infect Dis. 2013
 Thalassemia major
Life threatening
Disrupt social and educational activities
Children rarely go to school
Personality of children with thalassemia can be
disturbed and they become dependent on
others
Psychosocial burden affects many aspects of the
patient's life, such as education, leisure time,
physical activity, skills, abilities, and family
adjustment
Caocci G.
Health related quality of life in Middle Eastern children with beta-thalassemia.
BMC Blood Disorders 2012: 12:6
Shaligram D GSC, Chaturvedi S.K.
Psychological problems and quality of life in children with thalassemia.
I ndian journal of pediatrics 2007
Torcharus K PT.
Health-related quality of life in thai thalassemic children treated with iron chelation.
South East Asian JTRop med publc Health. 2011
Ayoub dkk.
Quality of life among children with beta-thalassemia major treated in Western Saudi Arabia.
Saudi Med J 2013;Vol. 34 ((12): ):1281-6
 This EBCR will discuss :

Prognosis of thalassemia major disease in the

form of quality of life based on iron chelation
therapy adherence.
 CASE
ILLUSTRATIONS
Case 1 Case 2 Case 3 Case 4

Male, 10 years old Male, 9 years old Female, 9 years old Female, 12 years old
Known to suffer Known to suffer Known to suffer Known to suffer
thalassemia since the thalassemia since the thalassemia since the thalassemia since the
age of 3 months age of 4 months age of 4 months age of 9 months
Blood trasfusions Blood trasfusions Blood trasfusions Blood trasfusions
every 1-2 week, every 1- 2 week, every 3-4 week, every 4 week,
received iron chelator, received iron chelator, received iron chelator, received iron chelator,
but dont comply daily but dont comply daily and comply daily and comply daily
 Ferritin Level
Case 1 Case 2 Case 3 Case 4

3267 ng/mL, 3411 ng/mL, 1297 ng/mL, 3756 ng/mL,

6666 ng/mL, 3706 ng/mL, 2482 ng/mL, 2241 ng/mL,

4875 ng/mL, 4845 ng/mL, 3112 ng/mL, 2653 ng/mL,.

4551 ng/Ll, 3775 ng/mL, 2263 ng/mL, 2070 ng/mL,

4320 ng/mL 6567 ng/mL. 1522 ng/mL. 1577 ng/mL

CLINICAL
QUESTION
Clinical question:

How is the effect of

adherece to iron chelation
on quality of life in children
with thalassemia?
 The problem is elaborated in the form of PICO
components as follows:

P: Patient/Population Children with thalassemia major

I : Intervention/Indicator Provision regular iron chelation

C: Comparison/Control Adherence : good or not

O: Outcome Quality of life

How the effect of regular iron chelation on

quality of life with thalassemia?
JOURNAL
SEARCH
 METHODS
Literature search procedures to address the
problem above is by browsing the online
literature search using :

Pubmed
Clinical Queries
Cochrane Library and or
EMBASH.
 METHODS
The methods also include and consider
alternative terminology, synonyms, or spelling.
Search using the "AND" to limit the search,
and "OR" to broaden the search.
Keywords that are used are :
Thalassemia major AND blood transfusion
AND children AND chelation therapy outcome.
 Type Pubmed Clinical Queries, Articles
28 articles (all
in english
Cochrane
language)

All articles 19 irrelevant No9filter

relevant 9
articles articles

Journals Cohort
3 cohort
4 cross 3
sectional
journals
(publication type)
journals

Journals Cross sectional 4

Critical
(publication type)
appraisal
 Relationship Among Chelator Adherence,
Change in Chelators and Quality of Life in
Thalassemia
Trachtenberg F L, Gerstenberger E, Yan Xu, Mednick L, Sobota A, Ware H,
Quality of life research. October 2014, Volume 23, Issue 8, pp 2277-2288

To examine stability of health-related quality of life (HRQOL) in thalassemia and

adherence to chelation therapy over time, especially after changes in chelator choice.

Chelation adherence was defined as self-reported percent of doses administered in

the last 4 weeks

Physical HRQOL improved among those with lower iron burden (better health status)
at baseline who made a single change in chelator, but declined among participants
with multiple changes and/or high iron burden (worse health status). Mental health
improved among participants with lower iron burden, but iron overload was
negatively associated with social functioning
 CRITICAL APPRAISAL
ASPECT OF PROGNOSIS
SCREENING
Background of the research ?
To determine the quality of life in children with beta
thalassemia

Is the study design appropriate?

Yes

Are PICO research in accordance with the formulation of

clinical questions?
Yes
 Are the prognosis aspect of this study
is valid?
1. Is the sample representative, well defined and taken at the
same time with the course of their illness? Yes

2.Did the observations is sufficiently long and complete? Yes

3. Is the patient observations completed? Yes
4. Is the desired outcome predetermined?
Yes
Are there any criteria for outcomes that have been
Yes
obtained?
5. Are the outcomes assessed in a "blind"? (without knowing
the clinical characteristics and prognostic factors of No
patients?)
 Is the evidence of the aspects of
prognosis is important ?

1.What is the risk of outcome over time?

Decreased quality of life

2. How precise estimation of outcomes?

(P value) <0,05
 APPLICATION OF
CLINICAL PROBLEMS
 Management
Iron chelation of
complications

Regular blood
Splenectomy
transfusions
Supportive
management of
thalassemia major

Ikram N HK, Younas M, Amanat S.

Ferritin Levels in Patients of Beta Thalassaemia Major. International Journal of Pathology. 2004; :71-4.
Trachtenberg dkk.
Beliefs about chelation among thalassemia patients. Health and Quality of Life Outcomes. 2012:10:148
Porter J d.
Health-Related Quality of Life, Treatment Satisfaction, Adherence and Persistence in -Thalassemia and
Myelodysplastic Syndrome Patients with Iron Overload Receiving Deferasirox:Results from the EPIC Clinical Trial.
Hindawi Publishing Corporation. Anemia 2012
 Thalassemia major is usually diagnosed in the
first 2 years of life and require regular blood
transfusions.

However, regular blood transfusions can lead to a

iron overload

Complications that can occur is a disorder of the

endocrine hormone, dilated cardiomyopathy,
fibrosis liver and cirrhosis

Galanello R. Beta-thalassemia. Orphanet Journal of Rare Diseases 2010:5:11

Abetz L BJF, Jones P, Rofail D.
The impact of iron overload and its treatment on quality of life: results from a literature review.
Health and Quality of Life Outcomes. 2006(4:73).
Mishra A K TA. Iron Overload in Beta Thalassaemia Major and Intermedia Patients.
MAEDICA a Journal of Clinical Medicine 2013(8(4)):328-32.
Bejaoui M GN.
Beta Thalassemia Major in a Developing Country: Epidemiological, Clinical and Evolutionary Aspects.
Mediterr J Hematol Infect Dis. 2013
 Prognosis of children
with beta-thalassemia is Advancement of
getting better in the last medical treatment
20 years.

Compliance or
adherence of blood
transfusions and iron
chelation will improve
the quality of life.

Trachtenberg dkk.
Relationship among chelator adherence, change in chelators, and quality of life in Thalassemia.
Qual Life Res.2014(23):227788.
Caocci G.
Health related quality of life in Middle Eastern children with beta-thalassemia.
BMC Blood Disorders 2012: 12:6
Galanello R. Beta-thalassemia. Orphanet Journal of Rare Diseases 2010:5:11
 Generally, Health
Various types of
Related Quality of Studies investigated
questionnaires
Life (HRQOL) is the HRQOL in
difficult to do
conceptualized as a thalassemia
comparison
multidimensional

Caocci G.
Health related quality of life in Middle Eastern children with beta-thalassemia.
BMC Blood Disorders 2012: 12:6
 Varni et al
Physical functioning,
emotional and social,
and school functioning as
described by the World
Health Organization
2003
(WHO).

Multidimensional Measure the core

PedsQL 4.0 domains of
questionnaires HRQOL

Torcharus K PT.
Health-related quality of life in thai thalassemic children treated with iron chelation.
South East Asian JTRop med publc Health. 2011;Vol 42 (No. 4 ).
Ayoub dkk.
Quality of life among children with beta-thalassemia major treated in Western Saudi Arabia.
Saudi Med J 2013;Vol. 34 ((12): ):1281-6
 Child and parent reports of PedsQL 4.0
measurement model is sensitive to cognitive
development and contains a form that is
suitable for children and their parents.

Several recent studies have reported using

PedsQL 4.0 to assess HRQOL in patients with
thalassemia from developing countries

Torcharus K PT.
Health-related quality of life in thai thalassemic children treated with iron chelation.
South East Asian JTRop med publc Health. 2011;Vol 42 (No. 4 ).
Ayoub dkk.
Quality of life among children with beta-thalassemia major treated in Western Saudi Arabia.
Saudi Med J 2013;Vol. 34 ((12): ):1281-6
 Physical function
(8 questions)

Emotional
function (5
Generic core questions)
23 questions
scale
Social function
PedsQL
(5 questions)
Specific diseases
School function
(5 questions).

Torcharus K PT.
Health-related quality of life in thai thalassemic children treated with iron chelation.
South East Asian JTRop med publc Health. 2011;Vol 42 (No. 4 ).
Ayoub dkk.
Quality of life among children with beta-thalassemia major treated in Western Saudi Arabia.
Saudi Med J 2013;Vol. 34 ((12): ):1281-6
 Forms
development 23 items
staging

PedsQL
2-18 years
4 minutes
old

Community
and school

Torcharus K PT.
Health-related quality of life in thai thalassemic children treated with iron chelation.
South East Asian JTRop med publc Health. 2011;Vol 42 (No. 4 ).
Ayoub dkk.
Quality of life among children with beta-thalassemia major treated in Western Saudi Arabia.
Saudi Med J 2013;Vol. 34 ((12): ):1281-6
 PedsQL
Assessment instruments include physical
functioning, emotional, social and school.
Total scale score of 0.88 to 0.90 for the self-
report and parent proxy report.
These instruments distinguish between
healthy children and children with acute or
chronic disease conditions.
This instrument also distinguish the severity of
chronic disease

Torcharus K PT.
Health-related quality of life in thai thalassemic children treated with iron chelation.
South East Asian JTRop med publc Health. 2011;Vol 42 (No. 4 ).
Ayoub dkk.
Quality of life among children with beta-thalassemia major treated in Western Saudi Arabia.
Saudi Med J 2013;Vol. 34 ((12): ):1281-6
 PedsQL results
Case 1 Case 2 Case 3 Case 4

Physical 200 650 775 800

Emotion 175 400 500 500

Social 125 450 325 400

School 125 375 375 400

PedsQL (mean) 156,25 493,75 493,75 525

In this case, each of the parents and patients
filled out a questionnaire PedsQL 4.0.

irregularly take
iron chelation Regularly take
iron chelation

Better quality of
life
OUTCOME
ANALYSIS
4 cases of thalassemia major
Regular blood transfusions
2 cases Regular iron chelation (good adherence)
Good quality of life

Regular blood transfusions

2 cases


Irregular iron chelation (poor adherence)
Blood ferritin levels
Declining quality of life
SUMMARY
Quality of life children with thalassemia major
with good adherence to iron chelation is
better than children with poor adherence.
 Education about the benefits of iron
chelation
Monitoring iron chelating drug for
children with thalassemia

To improve the quality of life children

with thalassemia
Thank you
Objectives: To assess the quality of life among children and adolescents with
thalassemia major.
Methods: This cross-sectional study used the Pediatric Quality of Life
Inventory (PedsQL). Children and adolescents with beta-
thalassemia major who attended the Day Care Unit at King
Abdulaziz University Hospital, Jeddah, Saudi Arabia from October
2012 to February 2013 were surveyed. The questions highlighted 4
health status scales, namely physical functioning (PF), emotional
functioning (EF), school performance (SC), and social functioning
(SF). Scores were calculated for each patient and data were
analyzed using the Statistical Package for Social Sciences
Results:
We recruited 46 children (60.9% males). The median age of the sample
was 12 years (range, 2-18years). Most patients (84.8%) had 3 weekly blood
transfusions. The meanSD physical functioning (PF), score was 57.225.9;
the EF score was 74.120.3, SF score was 78.524, and SC score was
54.324.2. The PF score was significantly lower in patients with a family
history of thalassemia (p=0.003), and in those whose families had low
incomes (p=0.049). Conversely, the SF score was significantly higher in
school-educated patients (p=0.01).

Conclusion:
The quality of life of thalassemic children is affected by multiple factors,
such as family income and a family history of thalassemia. Education
appeared to increase patient functionality. Supportive measures could
improve the quality of life in thalassemic patients.

Saudi Med J 2013; Vol. 34 (12): 1281-1286