scholarly journals Clinical-Pathological Features of Non-Transfusion Dependent Thalassemia in Adults — a Single Center Study in Hong Kong

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4900-4900
Author(s):  
Ka Lok Luke Chan ◽  
Vivien W M Mak ◽  
Kate F S Leung ◽  
Joyce H Y Kwong ◽  
Nelson C N Chan ◽  
...  
Keyword(s):  
Hong Kong ◽  
Liver Fibrosis ◽  
Iron Overload ◽  
Serum Ferritin ◽  
Transfusion Requirement ◽  
Liver Stiffness ◽  
Leg Ulcer ◽  
Liver Iron ◽  
Liver Iron Overload ◽  
Hbh Disease

Abstract Introduction: Non-transfusion dependent thalassemia (NTDT) includes HbH disease, β-thalassemia intermedia (β-TI) and HbE/β-thalassemia. The transfusion requirements of patients with NTDT are variable and they are all at risk of developing iron overload and other complications. The prevalent genetic changes in thalassemia are different across geographical territories, accounting largely for the diverse clinical outcomes. The complication profile of NTDT in Southern China is less well studied than other areas. Data on age-related complications is sparse. The present study aims to describe the clinical-pathological features of adult NTDT patients in Hong Kong, and to evaluate the risk factors associated with complications. Method: A single-center observational study was performed during Jan 2017 to Jun 2018. Data collection included review of medical records for demographics; globin genotypes; hepatitis B and C status; transfusion requirement; splenectomy; iron chelation therapy; complications including gallstone disease, hypothyroidism or hypogonadism, diabetes mellitus (DM), extramedullary hematopoiesis (EMH), leg ulcer, venous thrombosis and cerebral ischemia. During the study period, steady state hemoglobin (Hb) and serum ferritin levels in the recent year were obtained; organ iron deposition assessed using liver and cardiac T2* at 1.5T MRI; liver stiffness measured by Fibroscan; and presence of pulmonary arterial hypertension (PAH) evaluated by echocardiography. The disease profile and prevalence of complications were described with descriptive statistics. Factors impacting clinical parameters and development of complication were studied with univariate regressions. Age and sex adjusted β-coefficients or odd ratios were then determined with multivariable regression analysis. Results: A total of 96 Chinese patients were recruited (mean age 50±15 years; 31% patients >60 years; females 66%). There were 63 (65%) patients having deletional HbH disease, 20 (21%) with non-deletional HbH disease (ND-HbHD), and 13 (14%) with β-TI. The mean Hb was 8.9±1.1 g/dL. Transfusion requirement was never in 39 (41%), occasional in 50 (52%), and regular in 7 (7%) patients. Ten (10%) patients were splenectomized. Iron chelation was given to 21 (21%) patients and the median duration of therapy was 3 (range 1-21) years. Respectively 4 (4%) and 2 (2%) patients were hepatitis B and C carriers. The median serum ferritin was 473 [IQR 217-1029] ng/mL. The median liver iron concentration (LIC) estimated by MRI T2* was 3.6 [IQR 1.7-7.1] mg Fe/g with 26% of patients having moderate to severe liver iron overload (≥7.1 Fe mg/g). In the study population, the prevalence of liver fibrosis (liver stiffness ≥7.1kPa) was 26%, gallstones 50%, hypo-thyroidism/-gonadism 8%, DM 16%, EMH 5%, leg ulcer 1%, thrombotic events 2%, cardiac iron overload 1%. No patients had PAH (Table 1). In multivariable regression, advanced age (>60 years) was associated with lower Hb (p=0.03), higher risk of liver fibrosis (p=0.04) and DM (p<0.01). β-TI was associated with higher transfusion requirement (p<0.01), higher serum ferritin (p<0.01) and higher risk of hypo-thyroidism/-gonadism(p=0.01). Both ND-HbHD and β-TI were associated with higher LIC (p<0.01). Patients requiring regular transfusions had higher serum ferritin (p<0.01) and LIC (p<0.01). Occasionally transfused (p=0.01) and female patients (p=0.01) had higher risk of developing gallstones. Splenectomy was associated with higher serum ferritin level (p=0.03). Patients with moderate to severe liver iron overload had increased risk of liver fibrosis (p=0.01) (Table 2). Conclusions: The present study including 1/3 of elderly patients illustrates the heterogeneous clinical features of NTDT in Hong Kong in terms of transfusion requirement and body iron store. HbH disease is the most frequent type of NTDT. Liver iron overload, liver fibrosis, gallstones and DM were the most common complications. Advancing age, β-TI, ND-HbHD were associated with higher risk of developing complications. A low prevalence of EMH, cardiac iron overload, PAH, thrombosis and leg ulcer was observed. Larger prospective study is needed to confirm the prevalence of complications in NTDT in Chinese, which may be different from other ethnic groups, and at the same time to provide more insight to the pathophysiology of NTDT and facilitate establishment of future local management strategies. Disclosures No relevant conflicts of interest to declare.

Liver Transient Elastography (FibroScan) Correlates with Liver Iron Concentration and Reflects Liver Fibrosis In Patients with Sickle Cell Disease

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 1646-1646 ◽  
Author(s):  
Ersi Voskaridou ◽  
Maria Schina ◽  
Eleni Plata ◽  
Dimitrios Christoulas ◽  
Maria Tsalkani ◽  
...  
Keyword(s):  
Liver Fibrosis ◽  
Iron Overload ◽  
Hepatic Fibrosis ◽  
Sickle Cell ◽  
Serum Ferritin ◽  
Transient Elastography ◽  
Iron Concentration ◽  
Liver Stiffness ◽  
Cell Disease ◽  
Liver Iron

Abstract Abstract 1646 Liver transient elastography (FibroScan) is an interesting new technology that allows estimation of hepatic fibrosis through measurement of liver stiffness. The technique is based on changes in tissue elasticity induced by hepatic fibrosis and is considered as a noninvasive, reproducible and reliable method to assess hepatic fibrosis as well as to diagnose liver cirrhosis. Hepatic iron overload is a severe complication of chronic transfusion therapy in patients with hemoglobinopathies and plays an important role in the development of hepatic fibrosis and cirrhosis. Iron overload is present in several cases of sickle cell disease (SCD) including sickle cell anemia (HbS/HbS) and double heterozygous sickle-cell/beta-thalassemia (HbS/beta-thal). The aim of the study was to evaluate liver fibrosis by measuring the liver rigidity (Liver Stiffness Measurement, LSM, kPascals) using transient elastography (FibroScan, Echosens, Paris, France) in patients with SCD and explore possible correlations with clinical and laboratory characteristics of the patients, including iron overload. We studied 110 consecutive patients with SCD who are followed-up in the Thalassemia Center of Laikon General Hospital in Athens, Greece. Forty-four patients were males and 66 females; their median age was 44 years (range: 21–73 years). Twenty-two patients had HbS/HbS and 88 patients had HbS/beta-thal. On the day of Fibroscan, all patients had a thorough hematology and biochemical evaluation, including hemoglobin, reticulocyte counts, serum ferritin, liver biochemistry, bilirubin, lactate dehydrogenase (LDH) and serology for viral hepatitis. Liver iron concentration was evaluated by magnetic resonance imaging (MRI) T2* in all patients. The median LSM of all patients was 6.1 kPascals (range: 3.4–48.8 kPascals) with no differences between HbS/HbS (6.1 kPascals, 3.5–17.3 kPascals) and HbS/beta-thal (6.1 kPascals, 3.4–48.8 kPascals) patients (p=0.835). LSM values strongly correlated with liver MRI T2* values (r=0.337, p<0.001), serum ferritin (r=0.328, p=0.001), number of transfusions (r=0.332, p=0.001), bilirubin (r=0.299, p=0.003), LDH (r=0.287, p=0.004), Hb (r=-0.275, p=0.006) and reticulocyte counts (r=0.244, p=0.015). LSM values showed also strong positive correlations with biochemical indicators of liver function: gamma-glutamyl transpeptidase (r=0.522, p<0.0001), glutamic oxaloacetic transaminase (r=0.484, p<0.0001), glutamic pyruvic transaminase (r=0.422, p<0.0001), alkaline phosphatase (r=0.334, p=0.001), gamma-globulin (r=0.296, p=0.005) and weak correlation with PT-International Normalized Ratio (r=0.184, p=0.094). The above correlations were similar in patients with HbS/HbS and in patients with HbS/beta-thal. However, in HbS/HbS patients the correlation between LSM and liver T2* values was very strong (r=0.770, p=0.001). Patients who were regularly transfused had higher values of LSM (median: 6.7 kPascals, range: 2.3–48.8 kPascals) compared with patients who were sporadically transfused or were not transfused (4.4 kPascals, 3.6–17.5 kPascals, p=0.003). Patients who were under iron chelation therapy had lower values of LSM (6.3 kPascals, 3.4–15 kPascals) compared with those who did not receive iron chelators (13.9 kPascals, 8.5–17.3 kPascals, p=0.013). We found no correlations between the presence of HBV or HCV positivity and the levels of LSM. In conclusion, FibroScan may constitute a reliable and easy to apply noninvasive method to assess liver fibrosis in patients with SCD; the strong correlations between LSM values with MRI T2* values and serum ferritin supports this observation. Furthermore, FibroScan seems also to reflect the presence of chronic hepatic injury in these patients. If our results are confirmed by other studies, FibroScan may be regularly used in the management of SCD patients in whom liver is the main target organ of the disease. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Iron Overload Status in Patients with Non-Transfusion-Dependent Thalassemia in China

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 1287-1287
Author(s):  
Rong rong Liu ◽  
Yu mei Huang ◽  
Peng Peng ◽  
Xiao yun Wei ◽  
Yu Lei ◽  
...  
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Genetic Disorder ◽  
Chinese Patients ◽  
Beta Thalassemia ◽  
Iron Level ◽  
Liver Iron ◽  
Liver Iron Overload ◽  
Medical University ◽  
Hbh Disease

Abstract Background: Non-transfusion-dependent thalassemia (NTDT) is a genetic disorder most commonly including beta-thalassemia intermedia (Beta-TI), HbE/Beta thalassemia (HbE/Beta thalassemia), and hemoglobin H disease (HbH disease). NTDT patients can be at risk of iron overload due to increased intestinal iron absorption triggered by chronic anemia, ineffective erythropoiesis and, possibly, decreased serum hepcidin. Despite NTDT is popular in southern China, there is little data evaluating iron overload in Chinese patients. This study aimed at investigating the occurrence, prevalence and severity of iron overload in Chinese population with NTDT. Methods: We evaluated the serum ferritin (SF), liver iron concentration (LIC) and cardiac T2* in 158 NTDT patients (83 with HbH disease, 45 with Beta-TI and 30 with HbE/Beta thalassemia) in China. The median age was 22 years old. The main characteristics of these patients along with the main results of the study were summarized in Table 1. Blood samples were obtained for the assessment of hemoglobin (Hb) and serum ferritin (SF) levels. LIC was assessed by using validated R2 magnetic resonance imaging [MRI] (FerriScan®). Cardiac iron level was measured by MRI T2*. Patients were scanned with MRI 1.5 T (Siemens Avanto, Germany). The study was performed at the First Affiliated Hospital of Guangxi Medical University. LIC < 3mg Fe/g dw and cardiac T2* > 20ms was considered normal. Abnormal LIC can be divided into mild: 3-7mg Fe/g dw, moderate: 7-15mg Fe/g dw, severe: >15mg Fe/g dw. All patients or parents/guardians provided their written informed consent to participate in this study. The study was approved by the Medical Ethics Committee of the First Affiliated Hospital of Guangxi Medical University. Results: The median SF level of 158 NTDT patients was 1,037(27.0-19,704) ng/ ml. LIC was detected in 155 patients (98.1%) and the median LIC value was 8.9(0.6-43) mg Fe/g dw. There were 15 patients (60%) (8 with HbH disease, 5 with Beta-TI and 2 with HbE/Beta thalassemia)<10 years old found liver iron overload. The youngest patient with liver iron overload was 5 years old with 5.6mg Fe/g dw in LIC. Cardiac T2*was assessed in 111 patients (70.2%) and the median cardiac T2* was 32.8(7.5-75.1)ms. The 7 patients (4.4%) (4 with HbH disease and 3 with Beta-TI) had cardiac T2*=<20ms. There was a significant correlation between LIC and SF (r=0.809, p<0.001). No correlation between LIC and Hb, cardiac T2* values can be verified. There was a significant correlation between LIC and age (r=0.497, p<0.001)(Fig 1). The levels of LIC in patients > 30-year old group are significantly higher than those in other groups (Fig 2).The patients with Beta-TI and HbE/Beta thalassemia showed a statistically significant lower Hb and higher values of SF and LIC than those of HbH disease patients. Conclusions: Chinese NTDT patients have a high prevalence of iron overload. The iron overload in patients with Beta-TI and HbE/Beta thalassemia are more serious than those in HbH disease patients. The age of patient is a risk factor of iron overload in NTDT patient. Patients > 30 years old have a high burden of iron overload. Our data shows that the first assessment of MRI LIC should be performed as early as 5 years old. Disclosures No relevant conflicts of interest to declare.

Non Invasive Assessment of Hepatic Fibrosis by Measurement of Liver Stiffness in Post Transfusional Iron Overload: Preliminary Results in 15 Patients.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3740-3740
Author(s):  
T. Mirault ◽  
D. Lucidarme ◽  
B. Turlin ◽  
Y. Deugnier ◽  
P. Brissot ◽  
...  
Keyword(s):  
At Risk ◽  
Liver Fibrosis ◽  
Iron Overload ◽  
Predictive Value ◽  
Liver Stiffness ◽  
Liver Iron ◽  
Preliminary Results ◽  
Non Invasive ◽  
Patients At Risk ◽  
Severe Fibrosis

Abstract Background: The prognosis of liver iron overload is highly dependent on liver iron content (LIC) and the extent of liver fibrosis. Liver biopsy is the invasive reference method for fibrosis evaluation but with several limitations. Non-invasive biological methods (FibroTest, APRI) are not applicable in blood diseases. Transient elastography (TE) is a new non-invasive and rapid bedside method used to measure liver stiffness.This technique has been extensively studied with success in evaluation of liver fibrosis in HCV infection (Ziol, Hepatology 2005; Castera, Gastroenterology 2005). Until now, no data are available on evaluation of liver fibrosis by TE in highly transfused patients with iron overload. Methods: We assessed liver stiffness (kPa) by elastography measurement (Fibroscan; Echosens, Paris, France). Ishak’s and Metavir’s scales were used to stage fibrosis and hepatitis activity; LIC was measured by atomic absorption spectometry in a central laboratory (Rennes, France) and by MRI procedure according to Gandon (Lancet 2004). Cardiac MRI T2* (indirect estimation of iron heart content) was also measured by MRI according to Anderson (Eur Heart J 2001). Serum ferritin level and HCV serology were assayed in all cases. Correlation statistical analysis used Spearman’s Rho and Pearson tests and mean comparisons were done by a non-parametric Mann-Whitney test. Results: 15 liver samples (weight >1mg except 1) from monthly transfused patients (13 major beta Thalassemia, 1 sickle cell disease, 1 myelodysplastic syndrome) were analyzed. Median age was 37 years (7–75). Histological results were: 3 cirrhosis (METAVIR F=4 (F4), or Ishak’s Staging =6 (IS6)) among 5 patients with severe fibrosis (F3,F4) or (IS4-6) and 10 patients with no or mild fibrosis (F0-2) or (IS0-3). Mean ferritin was 2579 ng/ml CI 95% [1372–3786]. 2 patients were HCV positive. Correlation between LIC and ferritin was 0.82 (p<0,05); between LIC and MRI LIC: 0.88 (p<0,05); LIC and MRI Heart T2*: 0.72(p<0,05); METAVIR F score and Ishak’s grading score: 0.86 (p<0,05). A correlation was also observed between METAVIR F and elastography: 0.60(p<0,05); and mean values of elastography were significally different in patients with severe fibrosis: 9.1kPa CI95% [4.5–13.7] vs 5.9kPa CI95% [4.6–7.2] in those without sever fibrosis (p<0,05) (fig). A value of elastography above 6.25kPa (Se=80%; Sp=70%; AUROC=0.820) identified patients at risk for severe fibrosis (F3,F4 or IS 4–6) (Negative Predictive Value = 88%; Positive Predictive Value = 57%). Conclusion: A significant correlation between Metavir Fibrosis scale and elastography values was found. This new non-invasive method would be helpful to evaluate liver fibrosis and to determine patients who could avoid invasive procedures in particular in regularly transfused low risk myelodysplastic patients at risk of bleeding due to abnormal platelet function. These preliminary results will have to be confirmed in a larger population. Figure Figure

Deferasirox Safety Profile in Patients with Transfusion-Dependent Anaemias:Results From the Interim Analysis of a Hellenic Study,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3183-3183
Author(s):  
Vassilis Ladis ◽  
Marouso Drossou ◽  
Dimitria Vini ◽  
Ersi Voskaridou ◽  
Miranda Athanasiou-Metaxa ◽  
...  
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Safety Profile ◽  
Interim Analysis ◽  
Pediatric Patients ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Liver Iron ◽  
Liver Iron Overload

Abstract Abstract 3183 Background: The introduction of iron chelation treatment has led to a significant improvement in morbidity and overall survival in patients with transfusion-dependent anemias. Deferasirox is a once-daily, oral iron chelator approved for the treatment of transfusional iron overload in both adult and pediatric patients. The efficacy and safety of deferasirox in a variety of transfusion-dependent anemias has been established in numerous Phase II/III clinical trials. Since most patients with transfusion-dependent anemias require lifelong iron chelation therapy, there is a need to assess the long-term safety of deferasirox in both adult and pediatric patients. Aim: To assess the safety profile of deferasirox in patients with transfusional iron overload in a real-world clinical setting. To further investigate the safety profile of deferasirox in patients with congenital erythrocyte disorders and transfusional iron overload, with ferritin levels <4000 ng/ml and without severe cardiac siderosis. Methods: Between July 2009 and September 2010, 85 patients with transfusion-induced iron overload treated with deferasirox as per the approved product labeling were enrolled in the study. These data represent the 24-week planned interim analysis of a 12-month observational study on deferasirox safety profile in the treatment of pediatric and adult patients with transfusion-dependent anemias who were newly-treated with deferasirox. Safety was evaluated through the monitoring and recording of all adverse events and serious adverse events, as well as routine laboratory testing, including hematology, blood chemistry and hepatic function assessments. Results: The population had a median age of 37.6 years (range: 5.3–61.4) and a female to male ratio of 1.3. Beta-thalassemia (67.1%) was the most common transfusion-dependent anemia, followed by thalassemia intermedia requiring periodic transfusions (20.0%) and sickle cell anemia (12.9%). Mean baseline ferritin levels were 1502.1±870.5 (pediatric group: 1480.2±522.8 and adult group: 1503.6±891.4), while 53 out of the 85 patients (62.4%) had serum ferritin level above 1000 ng/ml. Mean baseline liver T2* value was 10.4±9.7 ms; 44.4% of patients demonstrated minimal liver iron deposition (MRI T2* > 6.3 ms), 51.4% had mild to moderate liver iron overload (T2* ≤ 6.3 ms), and 4.2% had severe liver iron overload (T2*<1.4 ms). 54 (63.5%) of patients analysed had been pre-treated with iron chelators and 31 (36.5%) were chelation-naïve. The initial average daily dose of deferasirox was 25.9±4.8 mg/kg, and 70.6% of patients had no dose modification during the 24-week follow-up period. A statistical significant decrease in median serum ferritin levels was observed by Week 24 (mean absolute change from baseline:-214.5 ng/mL; p=0.009) [Figure 1]. No statistically significant changes were observed in creatitine levels, creatinine clearance and transaminases by Week 24 [Figure 1]. 37 ADRs were reported by 17 patients (20%) over the 24-week period. Among the most frequently observed ADRs (>5%) were epigastralgia reported by 7.1% of patients (6/85) and loose stools/diarrhoea by 5.9% of patients (5/85). The majority of ADRs reported (nevents=25; 67.6%) were graded as mild in severity, while 21.6% (nevents=8) were graded as moderate and 10.8% (nevents=4) as severe. Most ADRs (nevents=31; 83.8%) resulted in full recovery by Week 24. The overall incidence of SADRs was as low as 1.2% (in particular one patient experienced severe epigastralgia and upper extremity pain which resulted in her withdrawal from the study after four months of treatment). The all-cause discontinuation rate was 9.4% (8/85), while only two patients (2.4%) discontinued the study therapy due to ADR; 1 patient due to increased transaminase levels and 1 patient due to the aforementioned SADR. Conclusions: These data highlight the safety profile of deferasirox in both adult and pediatric patients; the regular monitoring of serum ferritin levels as well as other iron-overload parameters and transfusion requirements play a major role in determining and optimizing the outcome of iron chelation therapy. Disclosures: Ladis: Novartis Hellas S.A.C.I.: Investigator participating in a trial sponsored by Novartis. Drossou:Novartis Pharmaceuticals: Investigator participating in a trial sponsored by Novartis. Vini:Novartis Pharmaceuticals: Investigator participating in a trial sponsored by Novartis. Athanasiou-Metaxa:Novartis Hellas S.A.C.I.: Research Funding. Oikonomou:Novartis Hellas S.A.C.I.: Investigator participating in a trial sponsored by Novartis. Vlachaki:Novartis Hellas S.A.C.I.: Investigator participating in a trial sponsored by Novartis. Tigka:Novartis Hellas S.A.C.I.: Employment. Tzavelas:Novartis Hellas S.A.C.I.: Employment. Liakopoulou:Novartis Hellas S.A.C.I.: Investigator participating in a trial sponsored by Novartis. Adamopoulos:Novartis Hellas S.A.C.I.: Investigator participating in a trial sponsored by Novartis. Kattamis:Novartis Hellas S.A.C.I.: Honoraria, Membership on an entity's Board of Directors or advisory committees.

The Effect of Combination Iron Chelating Agents on Reducing the Severity Grading of Heart and Liver Iron Overload in β-Thalassemia Patients

2019 ◽  
Author(s):  
Majid Ghanavat ◽  
Alireza Fazeli Varzaneh ◽  
Nahid Reisi
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Ferritin Level ◽  
Iron Deposition ◽  
Liver Iron ◽  
Liver Iron Overload ◽  
Severity Grading ◽  
Group A ◽  
Group B

Background: Deferasirox (DFX), Deferoxamine (DFO), and Deferiprone (DFP) are iron chelators that can be used in thalassemic patients with iron overload. Materials and Methods: This clinical trial was performed on 108 thalassemic patients who were randomly divided into group A (n=54) and B (n=54). Group A received combination of DFX and DFP, and group B received DFO and DFP for six months. Serum ferritin level was measured at the beginning of the study, 3, and 6 months after the treatment; The heart and liver iron deposition rates were also measured at the beginning of the study, and 6 months after the treatment  in both groups and compared using Magnetic Resonance Imaging T2 plus (MRI T2*). Results: The mean age of patients in group A and B was 17.29±4.3 and 17.89±5.61 years old, respectively. Serum ferritin level significantly reduced after the treatment (Serum ferritin level at baseline, 3, and 6 months after the treatment in Group A: 2476.25±1289.32, 2089.62±1051.64 and 1290.22±724.78 ng/ml, respectively; in Group B: 2044.63±989.82, 1341.30±887.62 and 1229.41±701.22 ng/ml, respectively) (p<0.01, for both groups). MRI T2* heart and liver was also improved at the end of the study in both groups (p<0.01, for both groups). However, the combination of DFO/DFP significantly decreased severity grades of liver iron deposition in comparison to DFX/DFP regimen after six months (p<0.01). Conclusion: The results of the present study indicated that both combination therapies of DFO/DFP and DFX/DFP could improve heart and liver MRI T2*. However, DFO/DFP combination therapy was more effective in reducing the severity grades of liver iron deposition.     

scholarly journals Serum ferritin in the diagnosis of cardiac and liver iron overload in thalassaemia patients real-world practice: a multicentre study

2017 ◽  
Vol 182 (2) ◽  
pp. 301-305 ◽  
Author(s):  
Rungroj Krittayaphong ◽  
Vip Viprakasit ◽  
Pairash Saiviroonporn ◽  
Wipaporn Wangworatrakul ◽  
John C. Wood
Keyword(s):  
Multicentre Study ◽  
Iron Overload ◽  
Serum Ferritin ◽  
Real World ◽  
Liver Iron ◽  
Liver Iron Overload

Transient Elastography May Predict Liver Iron Overload in Adult Thalassaemia Patients

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 5407-5407 ◽  
Author(s):  
Vasilios Perifanis ◽  
Efthimia Vlachaki ◽  
Emmanouil Sinakos ◽  
Ioanna Tsatra ◽  
Maria Raptopoulou-Gigi ◽  
...  
Keyword(s):  
Liver Fibrosis ◽  
Iron Overload ◽  
Hepatic Fibrosis ◽  
Transient Elastography ◽  
Iron Concentration ◽  
Thalassaemia Major ◽  
Liver Iron Concentration ◽  
Liver Iron ◽  
Liver Iron Overload ◽  
T2 Mri

Abstract Although it is life saving, transfusion therapy has resulted in the majority of thalassemia patients being at risk for hemosiderosis-induced organ damage. Liver Iron Concentration (LIC) assessed by liver biopsy is considered the most accurate and sensitive method for determining body iron burden in patients with iron overload. The development of liver fibrosis is more closely related to liver iron concentration. Transient elastography (FibroScan, FS), which measures liver stiffness (LS), is a novel, noninvasive method to assess liver fibrosis. Whether FS is useful in the detection of preexisting liver iron overload in patients presenting with thalassaemia major without chronic viral hepatitis, is unclear. On the other hand, Magnetic Resonance Imaging (MRI) is a relatively inexpensive, widely available but more time consuming method that has long been considered as a useful tool for the non-invasive estimation of tissue iron content in multiple transfused patients with thalassemia. Aim: To study the prevalence and severity of liver fibrosis of transfusion dependent thalassaemia major patients, and correlate the MRI.LIC with the measurements of FS. Methods-Patients: The applicability for FS (Echosens, Paris, France) was defined as at least 10 valid measures and a success rate (number of valid measures/total number of LS Measures, LSM) ≥60% and a ratio of interquartile range/stiffness ≤0,2. Most subjects with FS scores below 5.1 kilopascals (kPa) are considered to have minimal fibrosis (grade F0 or F1, METAVIR score) according to the literature. The cut off FS values for diagnosing different stages of hepatic fibrosis were defined as &gt; 7.9kPa for F≥2, &gt; 10.3kPa for F≥3 and &gt; 11.9kPa for F=4. A total of 43 thalassaemic patients 23 males/20 females, median age 26,8±4,9 years, regularly transfused (pre-transfusion haemoglobin 9,7g/dl) were included in the study. All patients were hepatitis C virus (HCV) negative and chelated with different drugs (13 on deferasirox, 12 on deferiprone, 5 on desferrioxamine and 13 on combined therapy). Median ferritin levels were 1552±1576ng/ml. Liver tests (AST, ALT, γGT and Alkaline Phosphatase) were done simultaneously to all patients. Twenty-two of the 43 patients underwent liver iron determination (LIC) simultaneously by two methods: T2* Magnetic Imaging (T2*MRI) assessment and by calculation of MR-Hepatic Iron Concentration (MR.HIC) values (based on an algorithm developed by Gandon et al (Lancet 2004), using liver to muscle ratios in five axial gradient-echo sequences). T-test was used in statistical analysis to compare means. Results: Applicability of LSM was 100%. Overall median LSM was 8,25±6,05kPa (range 4–40,3kPa). Nineteen (44,1%) patients had FS&lt;6,1kPa (notably 8/19 patients below 5,1kPa), 13 (30,2%) had &lt;7,9kPa, 4 (9,3%) had &lt;10,3kPa, 2 (4,7%) had &lt;11,9kPa and 5 (11,7%) above 11,9kPa. Total FS correlated with Ferritin (r=0,39, p=0,008). Using the cutt-off value of 6,1 kPa for FS measurements, patients were divided in two groups with different ferritin levels: A (&lt;6,1kPa) 1039±758ng/ml vs B (&gt;6,1kPa) 1833±1742ng/ml, p&lt;0,03. FS values of the three different major therapy groups did not differ significantly. FS (22pts) correlated negatively with T2*MRI results (r=−0,39, p=0,07) and positively with MR.HIC results (r=0,49, p=0,02). There was no correlation with liver function tests. Conclusions: Severe haemosiderosis and hepatic fibrosis are common in patients with thalassaemia major despite the use of chelation therapy and the absence of HCV. Elastography has several characteristics that make it a desirable method for assessing hepatic fibrosis. In addition to being noninvasive and painless, it is also quick, inexpensive, and produces consistent results. It can also be useful as an alternative to check for liver iron overload, as abnormal results predict heavy liver iron overload. Further longitudinal and prospective studies are necessary to confirm these preliminary data.

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