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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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slide1

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)

slide2

-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

slide3

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.
slide4

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
slide5

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

slide6

Patients distribution stratified

by age and gender

13,8% are paediatrics (263 patients)

8,0% of the Italianthalassemicpopulationisyoungerthan 12 years

slide7

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)

slide8

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%

slide9

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%)

slide10

Distribution of

iron chelation therapy

DFO

Combined

DFP

DFX

Adultpatients are equallydistributedamongthe therapeuticoptions

DFO remains the first therapeuticchoice for patients >45

slide11

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
slide13

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
slide14

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
slide15

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