12th Congress of the European Hematology ... - Haematologica

12 th Congress of the European Hematology Association
SF, ESD and LVEF. The stability of SF and ESD in asymptomatic patients
may be attributed to inadequate dosage of DFO. Long term administration
of both chelators had satisfactory effect on ferritin levels and cardiac
T2 and T2*.
Table 1.
Table 2.
0802
BONE MICROARCHITECTURE IN THALASSAEMIA
R. Grosse, 1 I. Frieling, 2 E.B. Fung, 3 R. Fischer, 1 H.P. Kruse, 2 G.E. Janka1 1 University Hospital Hamburg/UKE, HAMBURG; 2 Osteoporosis Center Hamburg-Neuer
Wall, HAMBURG; 3 Children's Hospital & Research Center, OAK-
LAND, USA
Background. Due to improved blood transfusion and chelation therapy,
survival has been increased in thalassaemia patients with the consequence
of complications like osteoporosis not seen during childhood
and adolescence. The diagnosis of osteoporosis or osteopenia is assessed
by endocrinological parameters and bone mineral density (BMD) measurements.
Aims. The obvious shortcomings of conventional BMD methods
like dual energy x-ray absorptiometry (DXA), can be overcome by
simultaneously assessing the microarchitecture of the bone using highresolution
peripheral quantitative computed tomography (HR-pQCT),
which may improve the estimation of the fracture risk in patients with
thalassaemia. Patients and Methods. In 17 regularly transfused patients
(age: 13-43 y, 9/17 female) with β-thalassaemia major (n=10), -intermedia
(n=6), and CDA-II (n=1), the BMD of lumbar spine (LS) and total hip
was measured by DXA (Hologic QDR1000, Waltham, USA). Age related
z-scores were calculated from BMD. In addition, we assessed the
volumetric BMD and the trabecular architecture of the non-dominant
distal radius and tibia by HR-pQCT (XtremeCT ® , SCANCO Medical
AG, Bassersdorf, Schweiz). Liver iron concentration and endocrinological
parameters were also determined. Nonparametric statistical analysis
was used. Results. In 15/17 patients low BMD values (LS z-score
range: -1.1 to -3.1) were measured by DXA in significant correlation
with total volumetric density (range: 91-388 mg/cm 2 ; Rs = 0.70, p=0.002)
measured by HR-pQCT at the distal radius. Seven patients with LS zscores
570 µm). In 6/17
patients (>28 y), all with latent hypogonadism, the trabecular inhomogeneity
parameter TbSp SD at the distal radius deviated by more than
100% from the upper normal value (Boutroy et al., 2005) and their spongiosa
was porous or nearly dissolved. Patients with hypogonadism (n=9)
were significantly different from normals with respect to radial TbSp SD
((p=0.02), but not to LS z-score ((p=0.4). Patients with fractures (n = 5)
had lower total densities (p=0.02) and trabecular TbSp SD ((p=0.02) at
the tibia and started blood transfusions (Tx-age) at a higher age
(p=0.023). However, z-scores did not reflect the fracture risk in this
patient group ((p=0.11). Only the trabecular thickness of the tibia seems
to be correlated with the Tx-age (Rs=0.62, (p=0.007), which was higher
in the patients with thalassaemia intermedia and CDA-II (> 5 y). Liver
iron was mainly correlated with tibial TbSp SD (Rs=0.54, (p=0.025).
Summary. In diagnosing osteopenia or osteoporosis in patients with
thalassaemia z- or T-scores seem to underestimate the fracture risk
because a normal cortical thickness and density may conceal a porous
300 | haematologica/the hematology journal | 2007; 92(s1)
trabecular structure. Endocrinological failures, especially hypogonadism,
were responsible for the pathological microarchitecture of distal radius
and tibia, while bone marrow expansion as in thalassaemia intermedia
and liver iron concentration seem to play a minor role. These first results
from bone microarchitecture measurements in thalassaemia have to be
confirmed by larger patient numbers of different gender and age.
0803
COMPARISON OF EFFECTS OF DIFFERENT, LONG-TERM IRON CHELATION REGIMENS ON
MYOCARDIAL AND HEPATIC IRON CONCENTRATIONS ASSESSED WITH T2* MRI IN
PATIENTS WITH β-THALASSAEMIA MAJOR
V. Perifanis, 1 A. Christoforidis, 1 E. Vlachaki, 1 I. Tsatra, 1 G. Spanos, 2
M. Athanassiou-Metaxa1 1 2 Ippokration Hospital, THESSALONIKI; Diagnostic Laboratory Eurodiagnosis,
THESSALONIKI, Greece
Background. Iron overload in patients with β-thalassaemia, develops
insidiously and leads to multi-organ failure and premature death.
Exceeded iron is initially accumulated in the reticuloendothelial system;
however, iron-induced cardiomyopathy is the commonest cause of
death among thalassaemic patients. Magnetic Resonance Imaging (MRI)
has long been considered as a useful, noninvasive tool for estimating tissue
iron overload. Additionally, as new chelation agents are being developed,
MRI could represent a useful marker for comparing the efficacy
of different chelation regimens in removing tissue iron overload. Aims.
The aim of this study was to compare the efficacy of different iron chelation
regimens in controlling myocardial and hepatic iron with the use
of T2* MRI technique and to correlate these results to clinical and biochemical
parameters. Methods. From a pool of 167 patients with β-thalassaemia
major based on our Unit in this study we selected all patients
aged ≥17 years that maintained unaltered iron chelation treatment and
dosage for longer than 4 years. Sixty-four patients (28M and 36F) were
finally enrolled in the study. Their mean age at the time of MR scanning
was 26.49±5.8 years. Regarding their chelation therapy, patients were
divided into three groups: the first group, was receiving deferiprone
(DFP) at a dose of 75 mg/kg/day orally, the second group was receiving
deferoxamine (DFO) at a dose of 30-50 mg/kg/day at least 5 times a
week by a subcutaneous infusion overnight and the third group was
chelated with combination of DFO (30-50 mg/kg/day, 3-4 days a week)
and DFP (75 mg/kg/day, 7 days a week). Myocardial and Hepatic T2*
measurements were acquired on a 1.5 Tesla Unit, based on the protocol
developed by Prof. Pennell et al. Additionally, ventricular volumes and
ejection fractions were measured by standard cardiovascular MR techniques.
Means of serum ferritin concentrations and daily iron accumulation
derived from total amount of transfused red blood cells were calculated
for one year prior to MR scanning.
Table 1.
Results. Demographic, laboratorial and MR characteristics of the three
study groups are compare in Table 1. DFP group and combined group
had significantly less myocardial iron than DFO group (mean T2*±S.D.:
35.77±18.3 and 38.05±15.3 vs 23.77±13, p=0.02 and p=0.001, respectively).
DFO group and combined group had significantly less hepatic iron
than DFP group (mean T2*±S.D.: 8.16±8.4 and 11.3±10.9 vs 3.29±2.5,