ASSESSMENT OF A COHORT OF BETA THALASSEMIA PATIENTS: NEW CHALLENGES
(Abstract release date: 05/19/16)
EHA Library. Benmegherbi F. 06/09/16; 134963; PB2063
Fatma Zohra Benmegherbi
Contributions
Contributions
Abstract
Abstract: PB2063
Type: Publication Only
Background
The combination of transfusion and chelation therapy has dramatically extended the life expectancy of Beta-thalassemic patients who can now survive into their fourth and fifth decades of life. The goal of long-term transfusional support is to maintain the patient's hemoglobin level at 9-10 g/dl. However, complications are still frequent and affect the patients' quality of life, particularly in countries with limited resources.
Aims
The main objective of this study is to determine the prevalence of prominent complications in a cohort of Algerian thalassemia patients.
Methods
This study was conducted to assess 81 polytransfused Beta-thalassemic patients, mean age 16 years (6 – 31), sex ratio: 37M/44G: We assess blood transfusion regime, pre-transfusion hemoglobin, iron overload, by serum ferritin level, hepatic iron concentration. Bone mineral density (BMD) measurements at lumbar and femoral regions have been done using dual x-ray absorptiometry. We assess Growth and pubertal maturation (Taner stage), and dosing gonadic hormone dosage, calcium, vitamin D, Parathyroid, and thyroid hormone.
Results
The pretransfusion hemoglobin level was maintained between 6 and 9 g/dl in 90% of patients. Additionally, 5% had levels less than 6 g/dL, 5% from 9 to 11 g/dL. Fifty-five percent of patients had undergone splenectomy at a median age of 9 years (range, 1-31 years). Ferritin levels ranged: 147 to 13500 ng/mL (median, 4992 ng/mL). Among 44 patients assessed, 77% had values of 15 mg/g dry weight or higher, 50% LCI>43 mg/gdw, 20% had moderate LCI 15 to 7 mg/gdw, only 1 patient < 7mg/gdw. Hepatic biopsy, performed in 26 patients (assessed for bone morrow transplantation), all had hepatic fibrosis moderate to severe (Metavir score). BMD performed in 49 patients, prevalence of lumbar osteoporosis and osteopenia were 38% and 62%. Dosage of vitamin D performed in 34 patients:5 < 10ng/ml, 17 [10-20ng/ml], 8 [20-30ng ml], 4 normal > 30ng/ml. 12 patients receive Biphosphonate treatment. Short stature was seen in 65% of our patients. Hypogonadism was diagnosed in 22,9% of 74 patients who had reached pubertal age: 50% of hypogonadic females and males were receiving hormonal substitution. No patient had Hypoparathyroidism and primary hypothyroidism. 3 cases of primary amenorrhea, and 2 diabetes aged 17 and 31 years. Despite high ferritine serum no patients had heart disease requiring medication. The goal of long-term hypertransfusional support is to maintain the patient's hemoglobin level at 9-10 g/d. This threefold is rarely reached in our patients, for mixture of reasons (compliance, limited blood pack). Iron chelation has been formerly limited, reason of the high prevalence of hemochromatosis in the majority of our patients. Intensification of iron therapy is now assessed to obtain reversal tissues lesions. Well treated 90% of thalassemics patients reached normal puberty; in contrast, in our group of patients, only 20% achieved normal pubertal status after 16 years. Poor pubertal growth and impaired sexual maturation, and endocrine abnormalities in children, adolescents and young adults have been observed because of conventional treatment deficiency, substitutive hormonal therapy is indicated. BMD is a good index of bone status in patients with Thalassemia and should be done in these patients annual.
Conclusion
High prevalence of complications among our thalassemics signifies the importance of more detailed studies along with therapeutic interventions. The survival of patients with thalassemia major improving, but the prevalence of severe complications is still high.
Session topic: E-poster
Keyword(s): Bone mineral density, Hemoglobinopathy, Iron chelation
Type: Publication Only
Background
The combination of transfusion and chelation therapy has dramatically extended the life expectancy of Beta-thalassemic patients who can now survive into their fourth and fifth decades of life. The goal of long-term transfusional support is to maintain the patient's hemoglobin level at 9-10 g/dl. However, complications are still frequent and affect the patients' quality of life, particularly in countries with limited resources.
Aims
The main objective of this study is to determine the prevalence of prominent complications in a cohort of Algerian thalassemia patients.
Methods
This study was conducted to assess 81 polytransfused Beta-thalassemic patients, mean age 16 years (6 – 31), sex ratio: 37M/44G: We assess blood transfusion regime, pre-transfusion hemoglobin, iron overload, by serum ferritin level, hepatic iron concentration. Bone mineral density (BMD) measurements at lumbar and femoral regions have been done using dual x-ray absorptiometry. We assess Growth and pubertal maturation (Taner stage), and dosing gonadic hormone dosage, calcium, vitamin D, Parathyroid, and thyroid hormone.
Results
The pretransfusion hemoglobin level was maintained between 6 and 9 g/dl in 90% of patients. Additionally, 5% had levels less than 6 g/dL, 5% from 9 to 11 g/dL. Fifty-five percent of patients had undergone splenectomy at a median age of 9 years (range, 1-31 years). Ferritin levels ranged: 147 to 13500 ng/mL (median, 4992 ng/mL). Among 44 patients assessed, 77% had values of 15 mg/g dry weight or higher, 50% LCI>43 mg/gdw, 20% had moderate LCI 15 to 7 mg/gdw, only 1 patient < 7mg/gdw. Hepatic biopsy, performed in 26 patients (assessed for bone morrow transplantation), all had hepatic fibrosis moderate to severe (Metavir score). BMD performed in 49 patients, prevalence of lumbar osteoporosis and osteopenia were 38% and 62%. Dosage of vitamin D performed in 34 patients:5 < 10ng/ml, 17 [10-20ng/ml], 8 [20-30ng ml], 4 normal > 30ng/ml. 12 patients receive Biphosphonate treatment. Short stature was seen in 65% of our patients. Hypogonadism was diagnosed in 22,9% of 74 patients who had reached pubertal age: 50% of hypogonadic females and males were receiving hormonal substitution. No patient had Hypoparathyroidism and primary hypothyroidism. 3 cases of primary amenorrhea, and 2 diabetes aged 17 and 31 years. Despite high ferritine serum no patients had heart disease requiring medication. The goal of long-term hypertransfusional support is to maintain the patient's hemoglobin level at 9-10 g/d. This threefold is rarely reached in our patients, for mixture of reasons (compliance, limited blood pack). Iron chelation has been formerly limited, reason of the high prevalence of hemochromatosis in the majority of our patients. Intensification of iron therapy is now assessed to obtain reversal tissues lesions. Well treated 90% of thalassemics patients reached normal puberty; in contrast, in our group of patients, only 20% achieved normal pubertal status after 16 years. Poor pubertal growth and impaired sexual maturation, and endocrine abnormalities in children, adolescents and young adults have been observed because of conventional treatment deficiency, substitutive hormonal therapy is indicated. BMD is a good index of bone status in patients with Thalassemia and should be done in these patients annual.
Conclusion
High prevalence of complications among our thalassemics signifies the importance of more detailed studies along with therapeutic interventions. The survival of patients with thalassemia major improving, but the prevalence of severe complications is still high.
Session topic: E-poster
Keyword(s): Bone mineral density, Hemoglobinopathy, Iron chelation
Abstract: PB2063
Type: Publication Only
Background
The combination of transfusion and chelation therapy has dramatically extended the life expectancy of Beta-thalassemic patients who can now survive into their fourth and fifth decades of life. The goal of long-term transfusional support is to maintain the patient's hemoglobin level at 9-10 g/dl. However, complications are still frequent and affect the patients' quality of life, particularly in countries with limited resources.
Aims
The main objective of this study is to determine the prevalence of prominent complications in a cohort of Algerian thalassemia patients.
Methods
This study was conducted to assess 81 polytransfused Beta-thalassemic patients, mean age 16 years (6 – 31), sex ratio: 37M/44G: We assess blood transfusion regime, pre-transfusion hemoglobin, iron overload, by serum ferritin level, hepatic iron concentration. Bone mineral density (BMD) measurements at lumbar and femoral regions have been done using dual x-ray absorptiometry. We assess Growth and pubertal maturation (Taner stage), and dosing gonadic hormone dosage, calcium, vitamin D, Parathyroid, and thyroid hormone.
Results
The pretransfusion hemoglobin level was maintained between 6 and 9 g/dl in 90% of patients. Additionally, 5% had levels less than 6 g/dL, 5% from 9 to 11 g/dL. Fifty-five percent of patients had undergone splenectomy at a median age of 9 years (range, 1-31 years). Ferritin levels ranged: 147 to 13500 ng/mL (median, 4992 ng/mL). Among 44 patients assessed, 77% had values of 15 mg/g dry weight or higher, 50% LCI>43 mg/gdw, 20% had moderate LCI 15 to 7 mg/gdw, only 1 patient < 7mg/gdw. Hepatic biopsy, performed in 26 patients (assessed for bone morrow transplantation), all had hepatic fibrosis moderate to severe (Metavir score). BMD performed in 49 patients, prevalence of lumbar osteoporosis and osteopenia were 38% and 62%. Dosage of vitamin D performed in 34 patients:5 < 10ng/ml, 17 [10-20ng/ml], 8 [20-30ng ml], 4 normal > 30ng/ml. 12 patients receive Biphosphonate treatment. Short stature was seen in 65% of our patients. Hypogonadism was diagnosed in 22,9% of 74 patients who had reached pubertal age: 50% of hypogonadic females and males were receiving hormonal substitution. No patient had Hypoparathyroidism and primary hypothyroidism. 3 cases of primary amenorrhea, and 2 diabetes aged 17 and 31 years. Despite high ferritine serum no patients had heart disease requiring medication. The goal of long-term hypertransfusional support is to maintain the patient's hemoglobin level at 9-10 g/d. This threefold is rarely reached in our patients, for mixture of reasons (compliance, limited blood pack). Iron chelation has been formerly limited, reason of the high prevalence of hemochromatosis in the majority of our patients. Intensification of iron therapy is now assessed to obtain reversal tissues lesions. Well treated 90% of thalassemics patients reached normal puberty; in contrast, in our group of patients, only 20% achieved normal pubertal status after 16 years. Poor pubertal growth and impaired sexual maturation, and endocrine abnormalities in children, adolescents and young adults have been observed because of conventional treatment deficiency, substitutive hormonal therapy is indicated. BMD is a good index of bone status in patients with Thalassemia and should be done in these patients annual.
Conclusion
High prevalence of complications among our thalassemics signifies the importance of more detailed studies along with therapeutic interventions. The survival of patients with thalassemia major improving, but the prevalence of severe complications is still high.
Session topic: E-poster
Keyword(s): Bone mineral density, Hemoglobinopathy, Iron chelation
Type: Publication Only
Background
The combination of transfusion and chelation therapy has dramatically extended the life expectancy of Beta-thalassemic patients who can now survive into their fourth and fifth decades of life. The goal of long-term transfusional support is to maintain the patient's hemoglobin level at 9-10 g/dl. However, complications are still frequent and affect the patients' quality of life, particularly in countries with limited resources.
Aims
The main objective of this study is to determine the prevalence of prominent complications in a cohort of Algerian thalassemia patients.
Methods
This study was conducted to assess 81 polytransfused Beta-thalassemic patients, mean age 16 years (6 – 31), sex ratio: 37M/44G: We assess blood transfusion regime, pre-transfusion hemoglobin, iron overload, by serum ferritin level, hepatic iron concentration. Bone mineral density (BMD) measurements at lumbar and femoral regions have been done using dual x-ray absorptiometry. We assess Growth and pubertal maturation (Taner stage), and dosing gonadic hormone dosage, calcium, vitamin D, Parathyroid, and thyroid hormone.
Results
The pretransfusion hemoglobin level was maintained between 6 and 9 g/dl in 90% of patients. Additionally, 5% had levels less than 6 g/dL, 5% from 9 to 11 g/dL. Fifty-five percent of patients had undergone splenectomy at a median age of 9 years (range, 1-31 years). Ferritin levels ranged: 147 to 13500 ng/mL (median, 4992 ng/mL). Among 44 patients assessed, 77% had values of 15 mg/g dry weight or higher, 50% LCI>43 mg/gdw, 20% had moderate LCI 15 to 7 mg/gdw, only 1 patient < 7mg/gdw. Hepatic biopsy, performed in 26 patients (assessed for bone morrow transplantation), all had hepatic fibrosis moderate to severe (Metavir score). BMD performed in 49 patients, prevalence of lumbar osteoporosis and osteopenia were 38% and 62%. Dosage of vitamin D performed in 34 patients:5 < 10ng/ml, 17 [10-20ng/ml], 8 [20-30ng ml], 4 normal > 30ng/ml. 12 patients receive Biphosphonate treatment. Short stature was seen in 65% of our patients. Hypogonadism was diagnosed in 22,9% of 74 patients who had reached pubertal age: 50% of hypogonadic females and males were receiving hormonal substitution. No patient had Hypoparathyroidism and primary hypothyroidism. 3 cases of primary amenorrhea, and 2 diabetes aged 17 and 31 years. Despite high ferritine serum no patients had heart disease requiring medication. The goal of long-term hypertransfusional support is to maintain the patient's hemoglobin level at 9-10 g/d. This threefold is rarely reached in our patients, for mixture of reasons (compliance, limited blood pack). Iron chelation has been formerly limited, reason of the high prevalence of hemochromatosis in the majority of our patients. Intensification of iron therapy is now assessed to obtain reversal tissues lesions. Well treated 90% of thalassemics patients reached normal puberty; in contrast, in our group of patients, only 20% achieved normal pubertal status after 16 years. Poor pubertal growth and impaired sexual maturation, and endocrine abnormalities in children, adolescents and young adults have been observed because of conventional treatment deficiency, substitutive hormonal therapy is indicated. BMD is a good index of bone status in patients with Thalassemia and should be done in these patients annual.
Conclusion
High prevalence of complications among our thalassemics signifies the importance of more detailed studies along with therapeutic interventions. The survival of patients with thalassemia major improving, but the prevalence of severe complications is still high.
Session topic: E-poster
Keyword(s): Bone mineral density, Hemoglobinopathy, Iron chelation
{{ help_message }}
{{filter}}