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

NCT03358498 | Unknown

• 1 other identifier: QOLATSIBTPRD
• Study Type: observational
• Target: 75
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

β-thalassemia

Outcome Measures

Primary Outcomes (1)

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

  \- The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) It is a self-administered questionnaire comprising 36-items measuring eight dimensions of general HRQOL: physical functioning (10 items), role limitation due to 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). In addition to scores for individual dimensions, two summary scores assessing physical and mental dimensions of health and well-being can also be calculated: Physical Component Summary (PCS) score and the Mental Component Summary (MCS) score, respectively

  Baseline

Study Arms (1)

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.

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

Interventions

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

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

β-thalassemia group

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Seventy five thalassemic patients will be included in the study from haematology unit in internal medicine department in Assiut University Hospital.

You may qualify if:

• 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

You may not qualify if:

• primary haemochromatosis
• thalassemia minor patients
• preseance of systemic disease that prevent patient from treatment ,

Sponsors & Collaborators

• Assiut University: lead

Study Sites (1)

Assiut University Hospital

Asyut, Assiut University71515, Egypt

Location

Related Publications (5)

• 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.

  PMID: 17880620 BACKGROUND

• 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.

  PMID: 28809446 BACKGROUND

• 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.

  PMID: 17880607 BACKGROUND

• 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.

  PMID: 18704280 BACKGROUND

• 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.

  PMID: 23963793 BACKGROUND

Central Study Contacts

Osama A Ibrahiem, Prof

CONTACT

00201006372498; oibrahiem@yahoo.com

Rania M Hafez, doctor

CONTACT

00201000019198; raniahafez@ymail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 27, 2017
• First Posted: November 30, 2017
• Study Start: December 1, 2017
• Primary Completion: December 1, 2018
• Study Completion: March 1, 2020
• Last Updated: November 30, 2017

Record last verified: 2017-11

Locations

EG (1)