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  • 1
    Online Resource
    Online Resource
    A Cost-Utility Analysis of Deferiprone Compared to Desferrioxamine and Deferasirox for the Treatment of Chronic Myocardial Iron Overload in Thalassemia Patients
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 2077-2077
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 2077-2077
    Abstract: Background: In thalassemia major (TM) three iron chelators are available to treat chronic iron overload due to blood transfusions: subcutaneous desferrioxamine (DFO), oral deferiprone (DFP) and oral deferasirox (DFX). Aims: This study evaluated the relative cost effectiveness of the three chelators in monotherapy. Methods: The cost-effectiveness model used is an Italian adaptation within the MIOT (Myocardial Iron Overload in Thalassemia) project of the previously built and published UK model (Bentley A et al. Pharmacoeconomics 2013;31:807-22). Based on literature and MIOT data, it was assumed that all the monotherapies had a comparable effect on and liver iron. Data about adverse events (AE) and cardiac morbidity were taken from the MIOT Network. Four different efficacy-based scenarios were explored in respect to cardiac morbidity and mortality using Markov-type models. Cost data were updated to reflect the Italian market: the tariffs applied in Veneto Region in the year 2014 were considered for the drugs, the administration of desferrioxamine and the monitoring. In Italy Veneto Region was proved to be one of the most upright region in the health costs management. Incremental costs and quality-adjusted life-years (QALYs) were calculated for each treatment, with cost effectiveness expressed as incremental cost per QALY. Results: Within the MIOT project, none of the AE considered in the UK model (neutropenia, agranulocytosis, Franconi syndrome, hepatitis) were detected for the 193 TM patients who had been received the same chelator for at least 18 months and were considered as reference group. The table shows costs and QALYs in the different modelled scenarios. For the first three scenarios a 5-year period was considered per patient while for the last scenario a 1-year time horizon was used and the discount rate of 3% was not applied. DFP was the dominant strategy in all scenarios, providing greater QALY at a lower cost. There was a higher QALY gain with DFX compared with DFO, but at a greater cost. Conclusions: The results of this analysis indicate that, from an Italian perspective, DFP is the most cost-effective treatment available for managing chronic iron overload in β-thalassaemia patients. Use of DFP in these patients could therefore result in substantial cost savings. Table 1. Drug cost Administration costs Monitoring costs AE costs Total costs QALYs Scenario 1: iron chelators impact cardiac morbidity and mortality. Time horizon: 5 years. DFP €14,815 (€15,704) €0 (€0) €1,277 (€1,354) 0 €16,092 (€17,058) 3.962 (4.200) DFX €158,122 (€167,508) €0 (€0) €1,495 (€1,583) 0 €159,618 (€169,091) 3.876 (4.106) DFO €55,009 (€58,274) €12,034 (€12,748) €788 (€835) 0 €67,831 (€71,857) 3.285 (3.479) Scenario 2: iron chelators impact only cardiac mortality. Time horizon: 5 years. DFP €14,815 (€15,704) €0 (€0) €1,277 (€1,354) 0 €16,092 (€17,058) 3.962 (4.200) DFX €158,122 (€167,508) €0 (€0) €1,495 (€1,583) 0 €159,618 (€169,091) 3.888 (4.119) DFO €55,009 (€58,274) €12,034 (€12,748) €788 (€835) 0 €67,831 (€71,857) 3.294 (3.490) Scenario 3: iron chelators impact only cardiac morbidity. Time horizon: 5 years. DFP €14,815 (€15,704) €0 (€0) €1,277 (€1,354) 0 €16,092 (€17,058) 3.962 (4.200) DFX €161,155 (€170,820) €0 (€0) €1,524 (€1,615) 0 €162,679 (€172,435) 3.951 (4.187) DFO €56,064 (€59,427) €12,264 (€13,000) €803 (€851) 0 €69,132 (€73,278) 3.348 (3.548) Scenario 4: iron chelators are all equivalent. Time horizon: 1 year. No discount. DFP €3,141 €0 €271 0 €3,412 0.840 DFX €34,164 €0 €334 0 €34,498 0.840 DFO €11,885 €2,600 €170 0 €14,656 0.712 Discounted valuea (Undiscounted value) Disclosures Pepe: ApoPharma Inc: Speakers Bureau; Novartis: Speakers Bureau; Chiesi: Speakers Bureau.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.2077.2077
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
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  • 2
    Online Resource
    Online Resource
    Children with Thalassemia Major Display Abnormal Renal Tubular Function
    American Society of Hematology ; 2016
    In:  Blood Vol. 128, No. 22 ( 2016-12-02), p. 3628-3628
    Details
    In: Blood, American Society of Hematology, Vol. 128, No. 22 ( 2016-12-02), p. 3628-3628
    Abstract: Introduction. Chronic hypoxia, transfusional iron overload and chelation therapy are all contributing factors to the deterioration of renal function in thalassemia major (TM ) patients. Among chelators, deferasirox (DFX) is considered the most toxic drug for kidneys: increment of more than 30% of serum creatinine levels is used for dosage modulation or interruption. Recently tubular abnormalities have also been described. We have observed renal toxicity in nine children affected by TM and treated with DFX or deferiprone (DFP) and described the differences in glomerular or tubular function between the two oral chelators. Material and methods. Nine children, 4 males and 5 females, aged 3-17 years (median 7 y), were retrospectively evaluated. They were regularly transfused every 2-3 weeks, receiving a median of 260 g erythrocytes concentrates/kg/year. The median serum ferritin level at baseline before starting oral chelation was 1763 ug/L (range 1127-4198). Four patients (pts) received only one type of chelator (1 DFP and 3 DFX). Five pts received both DFX or DFP in different periods. Overall 8 DFX treatments (dosage 15-30 mg/kg, for 18-112 months, mean 40) and 6 DFP treatments (dosage 75-130 mg/kg for 14-54 months, mean 24) were administered. Three pts had previously received deferoxamine; 3/8 DFX pts and 1/6 DFP pts were naïf to chelation before first renal evaluation. The following renal indices were considered: − glomerular function: serum creatinine and cystatine C levels, estimated glomerular filtration rate (eGFR), according to modified Schwartz formula; urine albumin to creatinine ratio (ACR), urine protein excretion. − tubular function: glycosuria, urinary N-acetyl-beta-D-glucosamidase activity/u-creatinine (u-NAG/u-cr), (index of acute tubular damage); urine daily Beta2 microglobulin (B2MG) and Alfa1 microglobulin (A1MG) excretion (both indices of chronic tubular damage). Analyses were performed at different intervals, according to clinical conditions and patients' age, with different frequency among children. Creatinine impairment was defined as an increase over 30% of basal level. All indices were graded according to local laboratory range. Toxicity was considered significant when the tests were abnormal in at least 25% of evaluations. Results. Initial renal function was normal in all patients (median eGFR 209 ml/min/1.73 m2, range 157-295) and eGFR remained stable over time in all cases. A total of 5 pts with DFX and 3 pts with DFP showed an increase of creatinine over 30% of basal levels. Increase of ACR 〉 30 mg/g cr was seen in 6/8 pts (75%) in DFX and in 1/6 pts (17%) in DFP; 2 pts (1/8 in DFX and 1/6 in DFP) had macroalbuminuria (ACR 〉 300 mg/g creatinine). No association was found between cumulative DFP dose and eGFR over time (Pearson coefficient, r = 0.06), while a moderate association was found with DFX (r = 0.5; p = 0.06). Symptomatic acute tubular toxicity was observed in 2 pts, with tubular acidosis, glycosuria and abnormal acute tubular indices (u-NAG/u-cr). A1MG and B2MG were also altered, suggesting a chronic injury substrate. Both pts were in DFX group (receiving a dose of 20 and 40 mg/kg/day, respectively) and had experienced a fast reduction in ferritin levels over a short period of time. No signs of concomitant infection were detected. u-NAG/u-cr was always abnormal in all children in both groups; mean values were 2.8 U/mmol for DFX and 2.4 U/mmol for DFP. B2MG was altered in 6/8 DFX pts and in 2/6 DFP pts (75% vs 33%). Median values were 3.0 mg/24 h for DFX and 0.5 mg/24 h for DFP, respectively. A1MG was altered in 4/8 DFX pts and 3/6 DFP pts, with median values of 164 mg/24 h for DFX and 55 mg/24 h for DFP, respectively. Conclusions. Patients withTM can experience abnormalities in renal function, especially as regards tubular damage. Acute tubular damage indices were altered in all chelated pts, whereas tests for chronic damage were abnormal more often in the DFX group than in DFP-treated pts. Albeit the significance of our findings needs further confirmation in a longer follow up, these results suggest that extensive renal evaluation is needed, in particular in children whose life expectancy is open and long-term prognosis for multiple organ function is an important issue. Disclosures No relevant conflicts of interest to declare.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V128.22.3628.3628
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2016
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  • 3
    Online Resource
    Online Resource
    Cost-Utility Analysis of Three Iron Chelators Used in Monotherapy for the Treatment of Chronic Iron Overload in β-Thalassaemia Major Patients: An Italian Perspective
    Springer Science and Business Media LLC ; 2017
    In:  Clinical Drug Investigation Vol. 37, No. 5 ( 2017-5), p. 453-464
    Details
    In: Clinical Drug Investigation, Springer Science and Business Media LLC, Vol. 37, No. 5 ( 2017-5), p. 453-464
    Type of Medium: Online Resource
    ISSN: 1173-2563 , 1179-1918
    URL: Article
    DOI: 10.1007/s40261-017-0496-1
    Language: English
    Publisher: Springer Science and Business Media LLC
    Publication Date: 2017
    detail.hit.zdb_id: 2043793-6
    SSG: 15,3
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  • 4
    Online Resource
    Online Resource
    Three Dimensional Speckle Tracking Echocardiography (3D-STE) for Early Detection of Subtle Myocardial Deformation Dysfunction in Thalassemic Patients
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 2158-2158
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 2158-2158
    Abstract: Background: Among patients with beta-thalassemia early detection of transfusion-induced myocardial iron loading and its intervention with aggressive chelation therapy may delay or reverse heart failure. Three dimensional speckle tracking echocardiography (3D-STE) is a novel tool that may early detect myocardial affection in these patients. Methods: Thirty-two thalassemic patients with a mean age of 18.1± 7.03 years and 30 aged matched healthy control subjects have been included in the study. Patients have been recruited from pediatric hematology clinics in both Cairo University, Egypt (n=18) and Padova University, Italy (n=14). 3D-STE was performed to all patients and control subjects in addition to the myocardial relaxometry T2* by cardiac MRI. Results: The left ventricular ejection fraction (LVEF) derived from 3D echocardiography among the studied thalassemia patients was within normal range 62.5± 5.6%. Compared to the normal subjects, thalassemia patients had a statistical significant reduction of the left ventricular global longitudinal strain (-16.81± 0.93% vs -18.76 ± 1.12 %, p=0.001), the left ventricular global circumferential strain (-10.56 ± 0.61% vs -11.83 ± 0.71 %, p=0.001 ) and the left ventricular global area strain (-20.13 ±1.18% vs -22.48 ±,1.29 %, p=0.001). No statistical significant correlation was found between the severity of myocardial iron overload measured by T2* and the measured left ventricular global strain. Conclusion: In asymptomatic thalassemia patients with preserved left ventricular global systolic function 3D-STE derived strain can detect early subtle myocardial dysfunction. The observed subtle myocardial deformation dysfunction is not related to the extent of myocardial iron deposition. Disclosures No relevant conflicts of interest to declare.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.2158.2158
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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