Abstract book 6th RMS 16.indd

402
BMT for Haemoglobinopathies
Ayad Ahmed Hussein MD (Jordan)
Thalassemia major and sickle cell disease
are the two most widely disseminated
hereditary hemoglobinopathies in the
world. The outlook for affected individuals
has improved in recent years due to
advances in medical management and
attention paid to prevention and treatment
of complications. However, hematopoietic
stem cell transplantation (HSCT) is currently
the only curative option. The use of HSCT
has been increasing, and the outcomes
today are much improved in the last three
decades, with more than 90% of patients
surviving transplantation and more than
80% of them being disease-free.
Hemoglobinopathies are common in the
Mediterranean region, Middle East and
Africa. In many of these countries; the
delivery of the regular treatment and
adequate supportive care such as safe
blood transfusion and aggressive chelation
therapy is often problematic which leading
to poor survival with medical therapy and
can negatively influence the outcome
following HSCT. Also; because of various
socio-economic factors; patients are
often referred late for transplant in their
disease process. To improve the outcome
following HSCT; we have adopted at
KHCC a novel risk adopted approach
by using a reduced intensity preparative
regimen for high risk patients and fully
ablative conditioning for low risks patients.
403
Recovery of Neutrophil Function
by Hematopoietic Stem Cell
Transplantation
Adel M. Al-Wahadneh MD (Jordan)
Mohammed Mutaereen , Adel Alwahadneh,
Mohammad Abu-shoukear, Zeyad Habahbeh,
Read Zyoud, Hiffa Bin-Dahman
Background: Inherited neutrophil
Disorders are a heterogeneous group
of diseases that are characterized by
increased susceptibility to serious and lifethreatening
infections and non-infectious
complications. Hematopoietic stem cell
transplantation (HSCT) is a curative option
for these disorders.
Patients and Method: We retrospectively
reviewed the charts of 15 consecutive
patients who underwent HSCT for
inherited neutrophilic disorders in king
Hussein Medical Center/Queen Rania
Children Hospital (KHMC/QRCH) in Jordan
between 2004 and 2012. Data were
obtained from KHMC/QRCH data base.
The total number of patients was 15. The
total number of transplant was 17. Nine
patients (60%) were male and six patients
(40%) were female. Diagnoses were
chronic granulomatous disease (CGD)
(n=6, 40%), Chediak-Higashi Syndrome
(n=2, 13%), Griscelli Syndrome type II
(GS II) (n=1, 7%), Leukocyte Adhesion
Deficiency type I (LADI) (n=5, 33%), Severe
Congenital Neutropenia (SCN) (n=1,
7%). Pre-transplant comorbidities include
invasive fungal disease in CGD patients,
fasciitis in LAD I patients and accelerated
phase in patient with CHS and GS II.
Results: the total number of transplant was
17, two patients needed second transplant
for primary and secondary graft failure
respectively. The median age for transplant
was 3years (0.3-12 years). Two-third
of patients received transplant from
matched sibling donor and the remaining
from Matched family donor (at least 8/8
Loci). HSCT sources were bone marrow
(n=13, 76%), including RBC-depleted,
and peripheral blood stem cell PBSC
(n=4, 24%). Cyclophosphamide-based
myeloablative conditioning regimen was
used in four transplants. In 12 transplants
Fludarabine-busulfan-based regimen
were used with or without Rabbit ATG
and only one transplant was carried out
without conditioning. Cyclosporine was
used for the prophylaxis against GVHD in
majority of transplants (n=15, 88%) while
tacrolimus was used in only 2 transplants.
Second agents include MMF (n=13) and
MTX (n=4). The total number of CD34+
cells infused on day 0 ranged from 2.5 to
10 X 106/kg (median, 5.5X106/kg). The
total events of acute GVHD was 6 (35%)
with stage I-II in majority of patients. All
patients were engrafted except two who
had primary and secondary graft failure
respectively and underwent successful
2nd transplant from the same donor. The
median time of myeloid engraftment was
D +13 (11-15) and of platelet engraftment
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