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

12 th Congress of the European Hematology Association
1355
SURVIVAL AND COMPLICATIONS OF TRANSFUSION DEPENDENT β-THALASSEMIA
MAJOR IN LEBANON; A DECADES EXPERIENCE
M. Naja, 1 H. Ismaeel, 1 N. Zeineh, 1 W. Gharzuddin, 1
M. Charafeddine 1 , A. Inati, 2 S. Koussa, 3 K. Charafeddine, 1 A. Taher 1
1 American University of Beirut Med Cen, BEIRUT; 2 Nini Hospital, TRIPOLI;
3 Chronic Care Center, HAZMIEH, Lebanon
Background. β-thalassemia major (TM) is a debilitating disease with a
considerable prevalence in Lebanon. While the prognosis of patients
affected by β-thalassemia major has improved after the introduction of
regular blood transfusion regimens, complications and mortality related
to iron overload started to emerge. With the introduction of desferrioxamine
in the 1970s, the prospects of the disease improved. The
chronic nature of both the illness and its treatment modalities have lead
to the creation of the Chronic Care Center (CCC) in the year 1994, an
institution where patient care and information is centralized. Aims. Since
its inauguration, the CCC has significantly contributed to decreasing
the burden of β-thalassemia, as well as spreading awareness about this
disease. The present article deals primarily with our experience to this
day, specifically pertaining to the mortality of TM and development of
complications among the patients. Methods. All TM patients (218) included
in this study are regularly followed up at CCC with 30 patients who
were lost to follow-up being censored. Patients have been divided into
three groups according to the following birth cohorts: patients born in
1970-1983, 1984-1993, and patients born in 1994 and beyond. Data collected,
through chart review, included: year of birth, age at diagnosis,
gender, consanguinity status, date and cause of death, as well as date of
appearance of any of the following complications: hypothyroidism
requiring replacement, hypogonadism, infection with the hepatitis C
virus, HIV infection, or thrombotic events. Results. A total of 15 deaths
was recorded, nine (60%) of which were due to cardiac causes (Table
1). Heart failure affects 6.5% of patients (n=14). Hypogonadism was
shown to affect 27.9% of patients (n=60) and hypothyroidism was present
in 20.9% (n=45). Fourteen percent (n=30) of the patients have
Hepatitis C virus (HCV) infection. Though not statistically significant
(p=0.555), complication free survival period was noted to be slightly
better among patients with mean serum ferritin levels below 2500
ng/mL as compared to patients with higher levels. Patients born in 1994
and beyond were found to have a significantly more extended complication-free
survival period. Discussions. The centralization of care and the
more consistent follow-up at the CCC led to an increase in the complication-free
survival period. Patients are being diagnosed at an earlier age
and chelation therapy is being initiated considerably earlier (7.20±5.59
years of age for patients born from 1970 to 1984, compared to 1.07±1.47
years of age for patients born after 1994). Complications due to iron
overload still persist however, and the non-compliance to desferrioxamine
regimens is thought to be the main reason for this. Conclusions.
The advent and introduction of new oral iron chelators as well as better
iron overload quantitation methods will most likely lead to new findings
and decrease in the sequale of the disease, thus a follow up study
will be required to examine their impact and the new survival trends.
Table 1. Causes of Death.
488 | haematologica/the hematology journal | 2007; 92(s1)
1356
MONITORING ERYTHROCYTES CREATINE, DENSITY AND DEFORMABILITY IN DETECTING
PRECLINICAL ACTIVITY OF HEMOLYSIS IN THERAPY OF AUTOIMMUNE HEMOLYTIC ANEMIA
O. Nikulina, V. Tsvetaeva, G. Dmitrieva, S. Shurhina, A. Levina,
A. Levina, D. Khoroshko
Hematological Research Center, MOSCOW, Russian Federation
Autoimmune hemolytic anemias (AIHA) are acquired diseases, characterized
by selective destruction of erythrocytes by autoantibodies.
The reticulocyte count, creatine level and deformability of erythrocytes
are considered to be quantitave indicators of hemolytic process, which
can show preclinical hemolytic activity, but complex investigation of
hemolysis by different indices hasn , t been done yet. In 1967 Griffith
suggested that creatine level of erythrocyte reflects the mean age of erythrocyte
population. Aim. To detect minimal hemolytical activity with
the main and additional diagnostical tests of hemolytic process (hemoglobin
-Hb, reticulocyte - Rt, bilirubin, creatine, density, deformability
of erythrocyte which can help to estimate the efficacy of treatment and
prevent the development of severe hemolytic crises. Materials and methods.
47 patient with autoimmune hemolytic anemia (AIHA) were investigated:
18 male, 29 female, 22-68 y.o. Results. During hemolytic crises
the hemoglobin level dropped (58,2±15,5 g/l), reticulocyte count
increased up to 10,6±2,8%, bilirubin level also increased to 47,4±24,7 (N-
0- 20 mkm/l); mean level of creatine in erythrocytes was 5 time above
normal value (28,7±13,0 mg/dL, N - 5,7±1,5 mg/dL), density and
deformability of erythrocyte were also elevated (d l 16,8±5,4% N -
0,3±0,3%; d t 20,6±3,4%, N - 0,6±0,3%). 26 patient with chronic AIHA
received prednisolone 1-2 mg/kg. After 1 month therapy Hb, Rt, bilirubin
normalized, but creatine and deformability of erythrocyte still normal
ranges after 2 month remission. Patients with partial remission were
characterized by normal level of hemoglobin, high content of reticulocytes;
but at the same time creatine, density and deformability of erythrocyte
exceeded normal values and didn , t reach normal parametres.
Patients with acute form AIHA (n=15) were treated with high dose
methylprednisolon for 3 days, 4-5 cycles. Hb, Rt, bilirubin normalized
after 4 cycles of pulse therapy. After Hb, Rt, Bi had been normalized, creatine
of erythrocytes was decreasing slowly (11,4 mg/dL) and normalized
within 3-4 month. In remission (n=5) all values were normal. Three
patients with resistant form AIHA were treated with Rituximab (375
mg/m2 ). Three month later all parameters of hemolytic activity (main
and additional) normalized . Their remission duration is 5, 9, 19 month.
During remission, monitoring all parameters have been kept normal. In
relapse erythrocyte creatine levels were found to be rised at first as well
as erythrocyte deformability. Monitoring parameters of one patient after
the first course of Rituximab (RTX ) had shown slowly increasing creatine
level and deformability of erythrocyte, with stable level of hemoglobin
and reticulocytes. At primary period of hemolyse (without clinical
manifestation) RTX was applied again. It can prevent the development
of severe hemolytic crises. Complete remission having been
observed for 3 years. We observed high correlation between reticulocyte
count, creatine and deformability erythrocytes (r=0,86, r=0,78 p