Beta
356025

Outcome of Beta Thalassemia Major in Paediatric Patients Allografted from Fully Matched Related Donors; in Egypt

Article

Last updated: 04 Jan 2025

Subjects

-

Tags

Cancer Stem cells targeted therapy

Abstract

Background Thalassemia is the most common genetic disease worldwide. Allogeneic hemopoietic stem cell transplantation is a method available to cure transfusion-dependent thalassemia major patients. Aim To report incidence of post-transplant complications and survival outcome with factors affecting it in beta thalassemia major patients. Patients & methods We concluded this retrospective study of pediatric patients (≤18 years old) who underwent their first allo-HSCT during 5 years (2015-2019), all patients received allografts from fully matched related donors. A total 115 BTM patients' data were collected and patient classified into 3 classes according to Pesaro classification. Results post-transplant complications reported in 80% of all patients with most common one was infections (63.5%) which seen more in class III (64.7%). Also, acute graft versus host disease (AGVHD), chronic GVHD, cardiovascular toxicity, endocrinopathies and graft failure were more in class III patients (35.3%, 11.8%, 11.8%, 17.6% and 11.8% respectively). While pulmonary complications, neurotoxicity and hemorrhagic cystitis reported more in class II patients (40%, 21.8% and 21.8% respectively). Three-year overall survival (OS) and thalassemia free survival (TFS) of all patients were 80% and 77.4%, with the worst outcome reported in class III patients (58.8% for both OS and TFS). Patients' age group 5-10 years and MTX included GVHD prophylaxis were associated with better survival outcomes than other factors. Conclusion Addition of Fludarabine to the conditioning regimen in class III BTM patients didn't improve the occurrence of GVHD, graft failure or survival outcomes in comparison to class I and II patients.

DOI

10.21608/jcbr.2024.279173.1346

Keywords

Allogeneic hemopoietic stem cell transplantation, Pesaro classification, thalassemia free survival

Authors

First Name

Azza

Last Name

Shibl

MiddleName

-

Affiliation

Pediatric Oncology department, South Egypt Cancer Institute, Assiut University, Assiut, Egypt

Email

azzashibl@aun.edu.eg

City

Assiut

Orcid

0000000343206597

First Name

Heba

Last Name

Sayed

MiddleName

-

Affiliation

Pediatric Oncology department, South Egypt Cancer Institute, Assiut University

Email

hebadina2007@aun.edu.eg

City

Assiut

Orcid

-

First Name

Mohamed

Last Name

Zakaria

MiddleName

-

Affiliation

Paediatric oncology department, South Egypt Cancer Institute, Assiut, Egypt.

Email

mohammed.zakarya.3140@gmail.com

City

Asyut

Orcid

0009000605159960

First Name

Alaa

Last Name

Elhaddad

MiddleName

-

Affiliation

Pediatric oncology department, National Cancer Institute, Cairo University, Cairo, Egypt.

Email

alaaelhaddad@hotmail.com

City

Cairo

Orcid

-

Volume

8

Article Issue

2

Related Issue

47868

Issue Date

2024-06-01

Receive Date

2024-03-24

Publish Date

2024-06-01

Page Start

35

Page End

45

Print ISSN

3009-6391

Online ISSN

3009-7312

Link

https://jcbr.journals.ekb.eg/article_356025.html

Detail API

https://jcbr.journals.ekb.eg/service?article_code=356025

Order

356,025

Type

Original Article

Type Code

885

Publication Type

Journal

Publication Title

Egyptian Journal of Cancer and Biomedical Research

Publication Link

https://jcbr.journals.ekb.eg/

MainTitle

Outcome of Beta Thalassemia Major in Paediatric Patients Allografted from Fully Matched Related Donors; in Egypt

Details

Type

Article

Created At

30 Dec 2024