Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox

NCT ID: NCT03358498

Last Updated: 2017-11-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

75 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-12-01

Study Completion Date

2020-03-01

Brief Summary

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Regular blood transfusions are essential for the management of haematological conditions such as β-thalassemia major. As a result, however, patients with these conditions are susceptible to the development of transfusion-dependent iron overload (hemosiderosis or secondary iron overload

Detailed Description

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In the absence of a naturally occurring physiological mechanism for the removal of excess iron in the body, life-long treatment and adherence to iron chelation therapy (ICT) are necessary to prevent the morbidity and mortality that may result if excess iron is allowed to .

Deferasirox (DFO),is the oldest available form of ICT used by patients with transfusion-dependent disorders. Improvements in ICT administration convenience and tolerability are expected to improve patient's satisfaction with ICT and Health Related Quality of Life (HRQOL), thus promoting adherence to ICT regimens and potentially reducing iron overload-related morbidity/mortality and associated healthcare costs

Conditions

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β-thalassemia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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β-thalassemia group

SICT It is a questionnaire to assess patient satisfaction with ICT regimens. It comprises 19 items assessing four domains: perceived effectiveness of ICT (PE), burden of ICT (BD), acceptance of ICT (AC), and side effects of ICT (SE). Patients rate all items on scale from 1 "very dissatisfied" to 5 "very satisfied".

Lab methods :

1. full history and thorough clinical evaluation.
2. . Complete blood count. .3- Serum ferritin .

4-Renal function tests. 5-liver function tests.

The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) It

Intervention Type OTHER

SF-36v2 is questionnaire comprising 36items measuring eight dimensions of general HRQOL: physical functioning 10 items, physical health problems 4 items, bodily pain 2 items, general health perceptions 5 items, vitality 4 items, social functioning (2 items), role limitations due to emotional problems (3 items), and general mental health 5 items.

.

Interventions

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The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) It

SF-36v2 is questionnaire comprising 36items measuring eight dimensions of general HRQOL: physical functioning 10 items, physical health problems 4 items, bodily pain 2 items, general health perceptions 5 items, vitality 4 items, social functioning (2 items), role limitations due to emotional problems (3 items), and general mental health 5 items.

.

Intervention Type OTHER

Other Intervention Names

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Satisfaction with ICT Questionnaire (SICT) Complete blood count. - Serum ferritin . -Renal function tests. -liver function tests.

Eligibility Criteria

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Inclusion Criteria

1 - Age more than 16 years 2-transfusion related iron over load (serum ferritin more than 1000 ng/ml) 3-patients on oral iron chelation (deferasirox ) for one year or more

Exclusion

1. primary haemochromatosis
2. thalassemia minor patients
3. preseance of systemic disease that prevent patient from treatment ,
Minimum Eligible Age

16 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Assiut University

OTHER

Sponsor Role lead

Responsible Party

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Rania

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Assiut University Hospital

Asyut, , Egypt

Site Status

Countries

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Egypt

Central Contacts

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Osama A Ibrahiem, Prof

Role: CONTACT

Phone: 00201006372498

Email: [email protected]

Rania M Hafez, doctor

Role: CONTACT

Phone: 00201000019198

Email: [email protected]

References

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Delea TE, Edelsberg J, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, Coates TD. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007 Oct;47(10):1919-29. doi: 10.1111/j.1537-2995.2007.01416.x.

Reference Type BACKGROUND
PMID: 17880620 (View on PubMed)

Bollig C, Schell LK, Rucker G, Allert R, Motschall E, Niemeyer CM, Bassler D, Meerpohl JJ. Deferasirox for managing iron overload in people with thalassaemia. Cochrane Database Syst Rev. 2017 Aug 15;8(8):CD007476. doi: 10.1002/14651858.CD007476.pub3.

Reference Type BACKGROUND
PMID: 28809446 (View on PubMed)

Payne KA, Desrosiers MP, Caro JJ, Baladi JF, Lordan N, Proskorovsky I, Ishak K, Rofail D. Clinical and economic burden of infused iron chelation therapy in the United States. Transfusion. 2007 Oct;47(10):1820-9. doi: 10.1111/j.1537-2995.2007.01398.x.

Reference Type BACKGROUND
PMID: 17880607 (View on PubMed)

Payne KA, Rofail D, Baladi JF, Viala M, Abetz L, Desrosiers MP, Lordan N, Ishak K, Proskorovsky I. Iron chelation therapy: clinical effectiveness, economic burden and quality of life in patients with iron overload. Adv Ther. 2008 Aug;25(8):725-42. doi: 10.1007/s12325-008-0085-z.

Reference Type BACKGROUND
PMID: 18704280 (View on PubMed)

Fisher SA, Brunskill SJ, Doree C, Gooding S, Chowdhury O, Roberts DJ. Desferrioxamine mesylate for managing transfusional iron overload in people with transfusion-dependent thalassaemia. Cochrane Database Syst Rev. 2013 Aug 21;2013(8):CD004450. doi: 10.1002/14651858.CD004450.pub3.

Reference Type BACKGROUND
PMID: 23963793 (View on PubMed)

Other Identifiers

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QOLATSIBTPRD

Identifier Type: -

Identifier Source: org_study_id