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THALASSEMIA MAJOR AND INTERMEDIA IN PATIENTS OLDER THAN THIRTY-FIVE YEARS -FROM A FATAL TO A CHRONIC DISEASE
Author(s):
Oren Pasvolsky
,
Oren Pasvolsky
Affiliations:
beilinson hospital,Petach Tikva,Israel
liat shargian alon
,
liat shargian alon
Affiliations:
beilinson hospital,Petach Tikva,Israel
pia raanani
pia raanani
Affiliations:
beilinson hospital,Petach Tikva,Israel
(Abstract release date: 05/18/17) EHA Library. Pasvolsky O. 05/18/17; 182906; PB2193
Dr. Oren Pasvolsky
Dr. Oren Pasvolsky
Contributions
PDF Abstract
Abstract

Abstract: PB2193

Type: Publication Only

Background
During the past four decades beta thalassemia major (TM) and beta thalassemia intermedia (TI) have transformed from a universally fatal disease at a young age, into a chronic disease, with a constantly increasing life expectancy. This is attributed, amongst others, to the use of improved chelation therapy. Since prolongation of life expectancy has occurred only in recent years, there is little data regarding the older population with TM and TI.

Aims
We aimed to characterize disease and patients’ characteristics in patients above 35 years of age in an adult thalassemia center in Israel.

Methods
We conducted a retrospective analysis of 14 adult patients over the age of 35 years with TM (N=10) and TI (N=4) treated in a single center, specializing in the care of adult thalassemia patients. We used descriptive statistics to describe characteristics of disease and patients and the Mann-Whiteny test to compare between patients with TI and patients with TM.

Results
Between 2006 and 2016, 14 adult patients older than 35 years with TM (n=10) & TI (n=4) were followed and treated in our center. Median patients' age was 37 (range, 35-51) years, with 66% males and 50% of Arab ethnicity. Most of the patients had at least high school education (85%), and 78% were employed.

Thirteen patients (all TM patients and 3 out of the 4 TI patients) were treated regularly with blood transfusions. All patients received chelation treatment. Median hemoglobin (Hb) levels and mean corpuscular volume (MCV) levels were lower in patients with TI compared to TM (8.1 vs. 10 g/dl, p=.002 and 72.4 vs. 84 fl, p=.004, respectively). Median LDH levels and indirect bilirubin levels were higher in patients with TI compared to TM (603 vs. 330 u/L, p=.004 and 2.02 vs. 1.1 mg/dl, p=.06, respectively) indicating increased hemolysis.
All patients underwent splenectomy and had secondary thrombocytosis. All but two patients were treated with at least two different chelation modalities, either as single agent, including subcutaneous (SC) or intravenous (IV) deferioxamine (DFO), deferiprone (DFP), or deferasirox (DFX), or as various combination therapy options. The median number of chelation treatment lines was 3. All patients treated with chelation suffered from at least one adverse event, necessitating temporary discontinuation and usually substitution of treatment. The median number of adverse events was 1.5 per patient. Nine patients (64.2%) had good compliance with current chelation therapy.
Four patients with acute heart failure secondary to cardiac iron overload, and all four improved with intensified chelation treatment. Four TM patients (40%) were hypothyroid, half of them requiring thyroid hormone replacement therapy. All TM patients had hypogonadism. All females had amenorrhea and were treated with hormone replacement therapy, and none of them tried to conceive. Six of the seven male TM patients were treated with monthly testosterone injections, and three of them fathered children. All TM patients had osteoporosis, and three TI patients (75%) had metabolic bone disease.
Figure 1 shows the relative rates of symptomatic cardiac iron overload and endocrine dysfunction in the cohort.
Three patients (21.4%) had significant liver overload according to liver T2* MRI, necessitating chelation treatment intensification.
None of the patients in our cohort underwent allogeneic hematopoietic stem cell transplantation and none developed secondary malignancy during follow-up.

Conclusion
Advances in the treatment of thalassemia patients have enabled the majority of these patients prolonged survival into adulthood. However, this has brought a new set of challenges for both patients and healthcare. This study delineates the challenges faced while treating adult patients with TI and TM in the new era.

Session topic: 26. Thalassemias

Keyword(s): Treatment, Thalassemia, adult

Abstract: PB2193

Type: Publication Only

Background
During the past four decades beta thalassemia major (TM) and beta thalassemia intermedia (TI) have transformed from a universally fatal disease at a young age, into a chronic disease, with a constantly increasing life expectancy. This is attributed, amongst others, to the use of improved chelation therapy. Since prolongation of life expectancy has occurred only in recent years, there is little data regarding the older population with TM and TI.

Aims
We aimed to characterize disease and patients’ characteristics in patients above 35 years of age in an adult thalassemia center in Israel.

Methods
We conducted a retrospective analysis of 14 adult patients over the age of 35 years with TM (N=10) and TI (N=4) treated in a single center, specializing in the care of adult thalassemia patients. We used descriptive statistics to describe characteristics of disease and patients and the Mann-Whiteny test to compare between patients with TI and patients with TM.

Results
Between 2006 and 2016, 14 adult patients older than 35 years with TM (n=10) & TI (n=4) were followed and treated in our center. Median patients' age was 37 (range, 35-51) years, with 66% males and 50% of Arab ethnicity. Most of the patients had at least high school education (85%), and 78% were employed.

Thirteen patients (all TM patients and 3 out of the 4 TI patients) were treated regularly with blood transfusions. All patients received chelation treatment. Median hemoglobin (Hb) levels and mean corpuscular volume (MCV) levels were lower in patients with TI compared to TM (8.1 vs. 10 g/dl, p=.002 and 72.4 vs. 84 fl, p=.004, respectively). Median LDH levels and indirect bilirubin levels were higher in patients with TI compared to TM (603 vs. 330 u/L, p=.004 and 2.02 vs. 1.1 mg/dl, p=.06, respectively) indicating increased hemolysis.
All patients underwent splenectomy and had secondary thrombocytosis. All but two patients were treated with at least two different chelation modalities, either as single agent, including subcutaneous (SC) or intravenous (IV) deferioxamine (DFO), deferiprone (DFP), or deferasirox (DFX), or as various combination therapy options. The median number of chelation treatment lines was 3. All patients treated with chelation suffered from at least one adverse event, necessitating temporary discontinuation and usually substitution of treatment. The median number of adverse events was 1.5 per patient. Nine patients (64.2%) had good compliance with current chelation therapy.
Four patients with acute heart failure secondary to cardiac iron overload, and all four improved with intensified chelation treatment. Four TM patients (40%) were hypothyroid, half of them requiring thyroid hormone replacement therapy. All TM patients had hypogonadism. All females had amenorrhea and were treated with hormone replacement therapy, and none of them tried to conceive. Six of the seven male TM patients were treated with monthly testosterone injections, and three of them fathered children. All TM patients had osteoporosis, and three TI patients (75%) had metabolic bone disease.
Figure 1 shows the relative rates of symptomatic cardiac iron overload and endocrine dysfunction in the cohort.
Three patients (21.4%) had significant liver overload according to liver T2* MRI, necessitating chelation treatment intensification.
None of the patients in our cohort underwent allogeneic hematopoietic stem cell transplantation and none developed secondary malignancy during follow-up.

Conclusion
Advances in the treatment of thalassemia patients have enabled the majority of these patients prolonged survival into adulthood. However, this has brought a new set of challenges for both patients and healthcare. This study delineates the challenges faced while treating adult patients with TI and TM in the new era.

Session topic: 26. Thalassemias

Keyword(s): Treatment, Thalassemia, adult

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