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Laura Mangiarini 1

The multiregional Italian Thalassemia Registry: patient’s population changes and related iron chelation approach. Laura Mangiarini 1. R. Padula 1 , D. Bonifazi 1 , G. Del Vecchio 2 , P. Baiardi 3. 1 Consorzio per Valutazioni Biologiche e Farmacologiche, Pavia ( Italy )

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Laura Mangiarini 1

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  1. The multiregional Italian Thalassemia Registry: patient’s population changes and related iron chelation approach Laura Mangiarini1 R. Padula1, D. Bonifazi1, G. Del Vecchio2, P. Baiardi3 1Consorzio per Valutazioni Biologiche e Farmacologiche, Pavia (Italy) 2U.O. Pediatria "Federico Vecchio"A. O. U. Consorziale Policlinico di Bari (Italy) 3Fondazione Salvatore Maugeri, Pavia (Italy)

  2. -Thalassemia– Mediterranean Anemia in Italy FACTS In Italy 4,000-6000affected Transfusion => iron overload => iron chelation therapy Iron overload monitoring=> ferritin, hepatic and cardiac MRI

  3. The Italian Registry of • Thalassemia patients • The “Inter-regional Network for Thalassemia” was promoted by the Italian MoH in 2010. • A registry of thalassemia patients was set up to provide a flexible platform for the assessment of patients’ characteristics and disease management, utilization of chelating agents, treatment outcome, AEs rate, methodologies for iron deposition evaluation and cost of therapies. • Data were used to perform a prevalence study aimed at: • evaluating the demographic characteristics of the study population 11 and 5 years respectively after the introduction of the two oral chelators DFP and DFX • describing the current management of iron overload in a large cohort of different age subsets of patients.

  4. alltogether=> 1899 patients (adults and paediatrics)in 31 centri: 1100 • The Italian Registry of • Thalassemia patients in 2011 • NUMBERS • 16 regions • 60 clinical centres • 1-150 patientseach • 36 centres: paediatric and adultpatients • 22 centres: onlyadults • 2 centres: onlypaediatrics

  5. Demographic data of patients participating to the study Averageage of patientsis 30 years The progressive increase of the global meanageisdetermined by the sistematic use of ironchelatingagents

  6. Patients distribution stratified by age and gender 13,8% are paediatrics (263 patients) 8,0% of the Italianthalassemicpopulationisyoungerthan 12 years

  7. Iron chelation in Italy no therapy 1,20% DFX 32,70% DFO 24,10% DFP 20,30% 53% of patientsis under treatment with an oralchelator 45,80% of patientsis under treatment with DFO (monotherapy or combined)

  8. Iron chelation approach in paediatrics vs adults DFX 29,40% DFX 58,60% DFP 21,90% DFO 24,30% DFP 11,40% DFO 26,20% Combined 24,40% Combined 3,80%

  9. Distribution of iron chelation therapy DFO Combined DFP DFX Over 50% of children < 5 yearsis with DFX, DFO isused by 37% The subset 12-17 yearsis the highestuser of oralchelators(72.8%)

  10. Distribution of iron chelation therapy DFO Combined DFP DFX Adultpatients are equallydistributedamongthe therapeuticoptions DFO remains the first therapeuticchoice for patients >45

  11. Reasoning leading to the selection of iron chelation therapy • A structured interview was conducted with a subgroup (15) of the participating clinical centres aimed at exploring the medical reasons for: • changing from the parenteral to an oral iron chelation therapy • changing from the monotherapy to a combination therapy • The interviewwasbased on a multiple choicequestionnaire and to eachanswer a score 0 => 3 wasattributed: • 0 = never • 1 = true for lessthan 30% of mypatients • 2 = true for 30 -60% of mypatients • 3 = true for > 60% of mypatients

  12. Reasoning leading to the selection of iron chelation therapy

  13. Reasoning leading to the selection of iron chelation therapy • The introduction of the oralchelatorshasprogressivelychanges the prescriptionhabits in children and youngpatients: • DFOisprogressivelyexcluded from the chelation treatment in youngpatients • DFX is the preferredtherapeuticapproachbecausewellaccepted • DFPisacknowledged for depletingcardiaciron

  14. Conclusions • To date, in patients > 45 yearsthe electivetherapyremains DFO or DFP (asmonotherapy or combinationtherapy): • thereislittleinclination in changingtherapywhenefficacious • => the therapeuticeffectispredominant on the route of administration • The availability of alternative therapeuticoptionshassensiblyincreased the survival of the thalassemicpatient

  15. Conclusions • Thereis no perfectironchelating agent • but • threechelators are available with differentpharmacologicalprofile: • => itiscritical to makethemavailablealso for paediatricpatients in order to increase the possibility to provide the mostsuitable treatment for eachpatient in terms of safety, efficacy and compliance • => itisimportant to search for new chelators

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