ALLOGENEIC BONE
MARROW TRANSPLANTATION FOR CHILDREN WITH b-THALASSAEMIA MAJOR:
EGYPTIAN EXPERIENCE
AL-Tonbary Y1, Mahmoud H2, Salama O1,
Abo EL-Kheir M1, Sarhan M1
1 Mansoura University Children’s Hospital, Mansoura, Egypt
2 National Cancer Institute, Cairo, Egypt
Objective: Bone marrow transplantation (BMT) has an
established role in the treatment of immunological, hematological,
oncological, and genetic diseases.
The aim of this work is to evaluate the efficacy, cost benefit, and
morbidity of allogeneic BMT in children with b-thalassaemia major.
Methods: BMT has been performed in 20 transfusion-dependant thalassaemic
children (10 males and 10 females) with age range 1.2-13 years (median
=3.25) and body weight ranged from 10 to 50 kg (median =12.25). Based on the presence or absence of
hepatomegaly, hepatic fibrosis, and/or poor chelation therapy, patients
were classified into risk group I (n. =10), risk group II (n. =9), and risk
group III (n. =1). Conditioning regimen includes busulfan (3.5mg/kg/day for
4 days), cyclophosphamide (50mg/kg/day for 4 days), and antithymocyte
globulin (10mg/kg/day for 10 days).
Results: The overall survival was 100%, while
disease-free survival was 85% as graft failure had occurred in 15% of
cases. Acute and chronic
graft-versus-host diseases (GVHD) were encountered in 30% and 10% of cases
respectively. The median time
for WBCs engraftment was 16 days and for platelet engraftment was 21.5
days. The median dose of CD34+
cells was 11.9 x 106/kg of recipient’s body weight.
Conclusion: Allogeneic BMT has cured most of
children with b-thalassaemia major. The
use of peripheral blood stem cells yields a good CD 34+-cell dose and is
associated with rapid engraftment and tolerable complications.