Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients

Mohsen S. Elalfy, Amira M. Adly, Yasser Wali, Samir Tony, Ahmad Samir, Yasmine I. Elhenawy

Research output: Contribution to journalArticle

36 Citations (Scopus)

Abstract

Objective: Minimal data are available on the combined two oral iron chelators in β-thalassemia major (β-TM). Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and deferasirox (DFX) were studied. Methods: A prospective randomized trial (NCT01511848) was conducted on 96 young β-TM patients with severe iron overload. Patients were randomized to receive either DFP with DFO (arm 1) or DFP and DFX (arm 2). Efficacy endpoints were the difference between two groups in the change of serum ferritin (SF), liver iron concentration (LIC), cardiac MRI, and quality of life (QoL). Results: In both arms, SF and LIC at 12 months were significantly lower, and geometric mean cardiac T2* was higher compared to baseline. On regression analysis of change in each studied variable against time, significant difference between slopes of the two groups regarding cardiac T2* (P = 0.001 with more improvement in DFP/DFX patients) was found with no significant difference in the slopes of SF and LIC (P = 0.218 and 0.340). Conclusion: Both iron chelation combination regimens were equally effective in reducing iron overload and improving QoL.DFP/DFX combination proved superior in improving cardiac T2*, treatment compliance, and patients satisfaction with no greater adverse events.

Original languageEnglish
Pages (from-to)411-420
Number of pages10
JournalEuropean Journal of Haematology
Volume95
Issue number5
DOIs
Publication statusPublished - Nov 1 2015

Fingerprint

Deferoxamine
beta-Thalassemia
Chelating Agents
Iron
Safety
Ferritins
Iron Overload
Liver
Serum
Quality of Life
Patient Satisfaction
Compliance
Regression Analysis
deferasirox
deferiprone

Keywords

  • Cardiac MRI
  • Combined oral iron chelators
  • Liver iron
  • Quality of life
  • Thalassemia major

ASJC Scopus subject areas

  • Hematology

Cite this

Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients. / Elalfy, Mohsen S.; Adly, Amira M.; Wali, Yasser; Tony, Samir; Samir, Ahmad; Elhenawy, Yasmine I.

In: European Journal of Haematology, Vol. 95, No. 5, 01.11.2015, p. 411-420.

Research output: Contribution to journalArticle

@article{1b672f0a0cb74e37b440e2176164667f,
title = "Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients",
abstract = "Objective: Minimal data are available on the combined two oral iron chelators in β-thalassemia major (β-TM). Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and deferasirox (DFX) were studied. Methods: A prospective randomized trial (NCT01511848) was conducted on 96 young β-TM patients with severe iron overload. Patients were randomized to receive either DFP with DFO (arm 1) or DFP and DFX (arm 2). Efficacy endpoints were the difference between two groups in the change of serum ferritin (SF), liver iron concentration (LIC), cardiac MRI, and quality of life (QoL). Results: In both arms, SF and LIC at 12 months were significantly lower, and geometric mean cardiac T2* was higher compared to baseline. On regression analysis of change in each studied variable against time, significant difference between slopes of the two groups regarding cardiac T2* (P = 0.001 with more improvement in DFP/DFX patients) was found with no significant difference in the slopes of SF and LIC (P = 0.218 and 0.340). Conclusion: Both iron chelation combination regimens were equally effective in reducing iron overload and improving QoL.DFP/DFX combination proved superior in improving cardiac T2*, treatment compliance, and patients satisfaction with no greater adverse events.",
keywords = "Cardiac MRI, Combined oral iron chelators, Liver iron, Quality of life, Thalassemia major",
author = "Elalfy, {Mohsen S.} and Adly, {Amira M.} and Yasser Wali and Samir Tony and Ahmad Samir and Elhenawy, {Yasmine I.}",
year = "2015",
month = "11",
day = "1",
doi = "10.1111/ejh.12507",
language = "English",
volume = "95",
pages = "411--420",
journal = "European Journal of Haematology",
issn = "0902-4441",
publisher = "Wiley-Blackwell",
number = "5",

}

TY - JOUR

T1 - Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients

AU - Elalfy, Mohsen S.

AU - Adly, Amira M.

AU - Wali, Yasser

AU - Tony, Samir

AU - Samir, Ahmad

AU - Elhenawy, Yasmine I.

PY - 2015/11/1

Y1 - 2015/11/1

N2 - Objective: Minimal data are available on the combined two oral iron chelators in β-thalassemia major (β-TM). Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and deferasirox (DFX) were studied. Methods: A prospective randomized trial (NCT01511848) was conducted on 96 young β-TM patients with severe iron overload. Patients were randomized to receive either DFP with DFO (arm 1) or DFP and DFX (arm 2). Efficacy endpoints were the difference between two groups in the change of serum ferritin (SF), liver iron concentration (LIC), cardiac MRI, and quality of life (QoL). Results: In both arms, SF and LIC at 12 months were significantly lower, and geometric mean cardiac T2* was higher compared to baseline. On regression analysis of change in each studied variable against time, significant difference between slopes of the two groups regarding cardiac T2* (P = 0.001 with more improvement in DFP/DFX patients) was found with no significant difference in the slopes of SF and LIC (P = 0.218 and 0.340). Conclusion: Both iron chelation combination regimens were equally effective in reducing iron overload and improving QoL.DFP/DFX combination proved superior in improving cardiac T2*, treatment compliance, and patients satisfaction with no greater adverse events.

AB - Objective: Minimal data are available on the combined two oral iron chelators in β-thalassemia major (β-TM). Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and deferasirox (DFX) were studied. Methods: A prospective randomized trial (NCT01511848) was conducted on 96 young β-TM patients with severe iron overload. Patients were randomized to receive either DFP with DFO (arm 1) or DFP and DFX (arm 2). Efficacy endpoints were the difference between two groups in the change of serum ferritin (SF), liver iron concentration (LIC), cardiac MRI, and quality of life (QoL). Results: In both arms, SF and LIC at 12 months were significantly lower, and geometric mean cardiac T2* was higher compared to baseline. On regression analysis of change in each studied variable against time, significant difference between slopes of the two groups regarding cardiac T2* (P = 0.001 with more improvement in DFP/DFX patients) was found with no significant difference in the slopes of SF and LIC (P = 0.218 and 0.340). Conclusion: Both iron chelation combination regimens were equally effective in reducing iron overload and improving QoL.DFP/DFX combination proved superior in improving cardiac T2*, treatment compliance, and patients satisfaction with no greater adverse events.

KW - Cardiac MRI

KW - Combined oral iron chelators

KW - Liver iron

KW - Quality of life

KW - Thalassemia major

UR - http://www.scopus.com/inward/record.url?scp=84940782881&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84940782881&partnerID=8YFLogxK

U2 - 10.1111/ejh.12507

DO - 10.1111/ejh.12507

M3 - Article

VL - 95

SP - 411

EP - 420

JO - European Journal of Haematology

JF - European Journal of Haematology

SN - 0902-4441

IS - 5

ER -