HemoRec in Poland. Summary of bleeding episodes of haemophilia patients with inhibitor recorded in the years 2008 and 2009 04/2010



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Transkrypt:

HemoRec in Poland Summary of bleeding episodes of haemophilia patients with inhibitor recorded in the years 2008 and 2009 04/2010 Institute of Biostatistics and Analyses. Masaryk University. Brno

Participating centres in Poland Participating centres: 3 1. I Klinika Chorób Dzieci (OOD). Szczecin 2. Katedra i Klinika HNKTS. Wroclaw 1 13 14 12 3. Klinika Hematologii AM w Gdansku 4. Klinika Hematologii Dzieciecej AM. Wroclaw 5. Klinika Hematoonkologii I Transplantacji Szpiku. Lublin 6 10 8 9 6. Oddzial Chorób Wewnetrznych I Hematologii SJS. Poznan 7. Klinika Hematologii CMUJ. Krakow 2 4 11 15 5 8. IHiT Warsaw 9. Paediatric Clinic Warsaw 10. Paediatric Clinic Poznan 7 11. Wojewódzki Szpital im. M. Kopernika. Lodz 12. Wojewódzki Specjalistyczny Szpital Zespolony. Olsztyn 13. Szpital Wojskowy. Szczecin 14. Klinika Pediatrii. Hematologii i Onkologii. Zabrze 15. Świętokrzyskie Centrum Onkologii. Kielce

Number of records Number of patients Basic patient information Bleeding episodes Medical centre 0 100 200 300 I Klinika Chorób Dzieci (OOD), Szczecin 34 Katedra i Klinika HNKTS, Wroclaw 39 Klinika Hematologii AM w Gdansku 75 Klinika Hematologii Dzieciecej AM, Wroclaw 56 Klinika Hematoonkologii I Transplantacji Szpiku, Lublin 122 Oddzial Chorób Wewnetrznych I Hematologii SJS, Poznan 93 Klinika Hematologii CMUJ, Krakow 130 IHiT Warsaw 27 Paediatric Clinic Warsaw 221 Paediatric Clinic Poznan 39 Wojewódzki Szpital im. M. Kopernika, Lodz 40 Wojewódzki Specjalistyczny Szpital Zespolony, Olsztyn 27 Szpital Wojskowy, Szczecin 29 Klinika Pediatrii. Hematologii i Onkologii, Zabrze 81 Świętokrzyskie Centrum Onkologii, Kielce 89 0 100 200 300 34 29 62 53 82 81 1 198 39 31 27 29 81 65 0 500 1000 1500 2000 901 111 61 1610 579 160 6 11 27 886 318

Diagnosis Overall number of patients registered in Polish centres: N = 1102 (as of 17/08/2009) Diagnosis N % Haemophilia A 707 67.9 Haemophilia B 124 11.9 Haemophilia A with inhibitor 105 10.1 Von Willebrand disease 51 4.9 Coagulation factor deficiency 43 4.1 Haemophilia B with inhibitor 5 0.5 Haemophilia A & Haemophilia B 3 0.3 Haemophilia A & Von Willebrand disease, type other 1 0.1 Haemophilia A with inhibitor & Haemophilia B with inhibitor 1 0.1 Haemophilia A with inhibitor & Von Willebrand disease, type unknown 1 0.1 Haemophilia B & Von Willebrand disease, type 1 1 0.1 Total 1042* 100.0 Overall number of patients with inhibitor: N = 112 * Diagnosis is not filled for 60 patients.

Number of patients according to centres Overall number of patients registered in Polish centres: N = 1102 Overall number of patients with inhibitor : N = 112 Medical centre Inhibitor All patients patients N % N % I Klinika Chorób Dzieci (OOD). Szczecin 34 3.1 11 9.8 Katedra i Klinika HNKTS. Wroclaw 39 3.5 0 0.0 Klinika Hematologii AM w Gdansku 75 6.8 9 8.0 Klinika Hematologii Dzieciecej AM. Wroclaw 56 5.1 2 1.8 Klinika Hematoonkologii I Transplantacji Szpiku. Lublin 122 11.1 10 8.9 Oddzial Chorób Wewnetrznych I Hematologii SJS. Poznan 93 8.4 8 7.1 Klinika Hematologii CMUJ. Krakow 130 11.8 13 11.6 IHiT Warsaw 27 2.5 22 19.6 Paediatric Clinic Warsaw 221 20.1 18 16.1 Paediatric Clinic Poznan 39 3.5 1 0.9 Wojewódzki Szpital im. M. Kopernika. Lodz 40 3.6 5 4.5 Wojewódzki Specjalistyczny Szpital Zespolony. Olsztyn 27 2.5 0 0.0 Szpital Wojskowy. Szczecin 29 2.6 0 0.0 Klinika Pediatrii. Hematologii i Onkologii. Zabrze 81 7.4 6 5.4 Świętokrzyskie Centrum Onkologii. Kielce 89 8.1 7 6.3 Total 1102 100.0 112 100.0 Distribution of inhibitor patients 0% 5% 10% 15% 20% 9.8% 0.0% 8.0% 1.8% 8.9% 7.1% 11.6% 19.6% 16.1% 0.9% 4.5% 0.0% 0.0% 5.4% 6.3%

Proportion of inhibitor patients Overall number of registered patients with inhibitor : N = 112 Medical centre All patients Inhibitor patients N N % I Klinika Chorób Dzieci (OOD). Szczecin 34 11 32.4% Katedra i Klinika HNKTS. Wroclaw 39 0 0.0% Klinika Hematologii AM w Gdansku 75 9 12.0% Klinika Hematologii Dzieciecej AM. Wroclaw 56 2 3.6% Klinika Hematoonkologii I Transplantacji Szpiku. Lublin 122 10 8.2% Oddzial Chorób Wewnetrznych I Hematologii SJS. Poznan 93 8 8.6% Klinika Hematologii CMUJ. Krakow 130 13 10.0% IHiT Warsaw 27 22 81.5% Paediatric Clinic Warsaw 221 18 8.1% Paediatric Clinic Poznan 39 1 2.6% Wojewódzki Szpital im. M. Kopernika. Lodz 40 5 12.5% Wojewódzki Specjalistyczny Szpital Zespolony. Olsztyn 27 0 0.0% Szpital Wojskowy. Szczecin 29 0 0.0% Klinika Pediatrii. Hematologii i Onkologii. Zabrze 81 6 7.4% Świętokrzyskie Centrum Onkologii. Kielce 89 7 7.9% Total 1102 112 10.2% Proportion of inhibitor patients 0% 25% 50% 75% 100% Patients with inhibitor Patients without inhibitor or with unfilled diagnosis

History of patients registration Overall number of patients registered in Polish centres: N = 1102 Overall number of patients with inhibitor : N = 112 % of patients 25% 20% 15% 10% 5% Date of registration in the HemoRec registry Patients without inhibitor or with unfilled diagnosis Patients with inhibitor 0% 2 Q 2006 3 Q 2006 4 Q 2006 1 Q 2007 2 Q 2007 3 Q 2007 4 Q 2007 1 Q 2008 2 Q 2008 3 Q 2008 4 Q 2008 1 Q 2009 2 Q 2009 3 Q 2009 Inhibitor 2 Q 2006 3 Q 2006 4 Q 2006 1 Q 2007 2 Q 2007 3 Q 2007 4 Q 2007 1 Q 2008 2 Q 2008 3 Q 2008 4 Q 2008 1 Q 2009 2 Q 2009 3 Q 2009 Total yes 0 15 31 21 13 13 9 1 0 0 5 2 2 0 112 no or N/A 1 22 201 163 203 186 88 7 2 10 67 29 10 1 990 Total 1 37 232 184 216 199 97 8 2 10 72 31 12 1 1102

Inhibitor patients - sex, age distribution Overall number of patients with inhibitor : N = 112 Sex Current age 36.6% children 98.2% 1.8% % of patients 20 15 10 9.8 12.5 14.3 11.6 15.2 17.0 8.0 8.9 Male Female 5 2.7 N % Male 110 98.2 Female 2 1.8 Total 112 100.0 0 0-6 7-12 13-18 19-25 26-35 36-45 Age (years) 46-55 56-65 > 65 N 112 Mean 29.6 Median (Min - Max) 28.8 (1.2 75.7)

Bleeding episodes of inhibitor patients Overall number of recorded bleeding episodes of patients with inhibitor is 270 (for 28 different patients). Number of episodes per patient Location of bleeding N % 10 Joints 175 65.8 Number of patients 8 6 4 2 4 5 8 8 3 Joints & Muscle** 10 3.8 Muscle 32 12.0 Muscle & Subcutaneous** 3 1.1 Head 31 11.7 Head & Torso** 1 0.4 Genital 6 2.3 Subcutaneous 4 1.5 Torso 2 0.8 0 1 2-4 5-9 10-19 20 and more Skin 1 0.4 Skin & Subcutaneous** 1 0.4 Total 266* 100.0 Number of bleeding episodes * Location is not filled for 4 bleeding episodes. ** 15 bleeding episodes (5.6%) were into more than one location

History of bleeding of inhibitor patients Overall number of recorded bleeding episodes of patients with inhibitor is 270 (for 28 different patients). Number of episodes 40 30 20 10 0 3 Q 2005 Number of bleeding episodes Number of registered inhibitor patients Number of inhibitor patients with bleeding Average number of episodes per patient 2 2 2 2 3 4 Q 2005 3 Q 2005 1 Q 2006 4 Q 2005 2 Q 2006 1 Q 2006 Number of recorded bleeding episodes 3 Q 2006 2 Q 2006 4 Q 2006 3 Q 2006 1 Q 2007 20 4 Q 2006 2 Q 2007 35 1 Q 2007 3 Q 2007 38 2 Q 2007 4 Q 2007 Quarter and year 28 27 28 3 Q 2007 1 Q 2008 4 Q 2007 2 Q 2008 1 Q 2008 3 Q 2008 26 2 Q 2008 4 Q 2008 16 3 Q 2008 1 Q 2009 21 4 Q 2008 2 Q 2009 18 1 Q 2009 3 Q 2009 2 2 Q 2009 3 Q 2009 Total 2 2 0 2 2 3 20 35 38 28 27 28 26 16 21 18 2 270 0 0 0 0 15 46 67 80 93 102 103 103 103 108 110 112 112 112 1 1 0 1 2 2 8 14 11 13 19 7 13 8 9 7 1-2.0 2.0-2.0 1.0 1.5 2.5 2.5 3.5 2.2 1.4 4.0 2.0 2.0 2.3 2.6 2.0 -

Bleeding patterns of 28 inhibitor patients 2005 2006 2007 2008 2009 Bleeding episode Re-bleeding episode * 01/2005 01/2006 01/2007 01/2008 01/2009 * Bleeding into the same location within 48 hours after the end of the previous bleeding. Bleedings Re-bleedings Patient N N % 1 5 0-2 5 0-3 17 2 11.8 4 12 0-5 9 0-6 31 2 6.5 7 11 0-8 18 1 5.6 9 1 0-10 1 0-11 1 0-12 13 0-13 19 0-14 6 1 16.7 15 11 0-16 6 0-17 2 0-18 5 1 20.0 19 3 0-20 9 0-21 20 11 55.0 22 2 0-23 1 0-24 15 0-25 2 0-26 2 0-27 6 0-28 37 1 2.7 Total 270 20 7.0

Bleeding and medication - inhibitor patients 2005 2006 2007 2008 2009 2005-2009 Number of inhibitor patients 0 46 102 108 112 112 Bleeding episodes Number of bleeding episodes 4 7 121 97 41 270 % of re-bleeding episodes 0.0% 0.0% 5.0% 10.3% 7.3% 7.0% Number of patients with recorded bleeding episode Average number of bleeding episodes per patient Dosage Number of bleeding episodes with medication 1 2 20 17 13 28 4.0 3.5 6.1 5.7 3.2 9.6 4 6 119 97 40 266 Number of medications 4 10 165 101 62 342 Average number of medications per bleeding episode Number of bleeding episodes with more than one medication % of bleeding episodes with more than one medication 1.0 1.7 1.4 1.0 1.6 1.3 0 4 22 7 3 36 0.0% 66.7% 18.5% 7.2% 7.5% 13.5%

Bleeding and medication - non-inhibitor patients 2005 and earlier 2006 2007 2008 2009 2005-2009 Number of non-inhibitor patients 0 124 864 9850 990 990 Bleeding episodes Number of bleeding episodes 2 178 2218 1681 320 4400* Number of patients with recorded bleeding episode Average number of bleeding episodes per patient Dosage Number of bleeding episodes with medication 2 27 237 192 59 286 1.0 6.6 9.4 8.8 5.4 15.4 2 176 2211 1680 320 4389 Number of medications 5 193 2469 1869 358 4894 Average number of medications per bleeding episode Number of bleeding episodes with more than one medication % of bleeding episodes with more than one medication 2.5 1.1 1.1 1.1 1.1 1.1 0 16 168 114 32 330 0.0% 9.1% 7.6% 6.8% 10.0% 7.5% * There is missing date for 1 bleeding episode.

Duration of bleeding - inhibitor patients Overall number of recorded bleeding episodes of patients with inhibitor is 270 (for 28 different patients). Time from start of bleeding to the first infusion Time from the first infusion to the end of bleeding Proportion of episodes 40% 30% 20% 10% 0% 33.3% 34.4% 16.3% 10.0% 5.9% 1 2-3 4-6 7-12 > 12 Proportion of episodes 40% 30% 20% 10% 0% 19.6% 33.0% 29.3% 8.9% 9.3% 1 2-3 4-6 7-12 > 12 Time (hours) Time (hours) N = 270 N = 270 Mean: 4.4 hours Median: 2 hours Min - Max: 1-24 hours Mean: 5.2 hours Median: 3 hours Min - Max: 1-24 hours

Duration of bleeding non-inhibitor patients Overall number of recorded bleeding episodes of patients without inhibitor is 4400 (for 286 different patients). Time from start of bleeding to the first infusion Time from the first infusion to the end of bleeding Proportion of episodes 70% 60% 50% 40% 30% 20% 10% 0% 18.8% 61.3% 12.6% 6.6% 0.6% 1 2-3 4-6 7-12 > 12 Proportion of episodes 70% 60% 50% 40% 30% 20% 10% 0% 10.1% 40.0% 38.2% 4.8% 6.8% 1 2-3 4-6 7-12 > 12 Time (hours) Time (hours) N = 4297* N = 4266** Mean: 2.8 hours Median: 2 hours Min - Max: 1-24 hours Mean: 4.9 hours Median: 3 hours Min - Max: 1-24 hours * Information missing for 103 bleeding episodes. ** Information missing for 134 bleeding episodes.

Evaluation of episode - inhibitor patients Overall number of recorded bleeding episodes of patients with inhibitor is 270 (for 28 different patients). Evaluation of entire episode Proportion of episodes 100% 80% 60% 40% 20% 0% 77.0% Effective 21.5% Partially effective 1.5% Ineffective Evaluation

Evaluation of episode - non-inhibitor patients Overall number of recorded bleeding episodes of patients without inhibitor is 4400 (for 286 different patients). Evaluation of entire episode Proportion of episodes 100% 80% 60% 40% 20% 0% 95.1% Effective 3.9% 0.4% 0.6% Partially effective Evaluation Ineffective N/A

Medication of bleeding episodes - inhibitor patients Overall number of applied medications is 342. Medication was filled for 266 of 270 bleeding episodes. Dose Medication N % Mean Median Min Max NovoSeven (mg) 216 63.2 4.9 4.8 1.2 30.0 Other human factor VIII (IU) 82 24.0 1588 2000 500 3000 Feiba (IU) 23 6.7 1900 1000 500 4500 Immunate (IU) 15 4.4 1200 1000 250 4000 Purified factor IX, other (IU) 2 0.6 1000 1000 1000 1000 Fanhdi (IU) 1 0.3 1000 1000 1000 1000 Octanate (IU) 1 0.3 500 500 500 500 Octanine (mg) 1 0.3 4.8 4.8 4.8 4.8 Other recombinant factor IX (IU) 1 0.3 2000 2000 2000 2000 Total 342* 100.0 0 20 40 60 80 100 % 6.7 4.4 0.6 0.3 0.3 0.3 0.3 % of episodes 24.0 63.2 * more than one medication was used for 36 bleeding episodes

Medication of bleeding episodes non-inhibitor patients Overall number of applied medications is 4894. Medication was filled for 4389 of 4399 bleeding episodes. Dose Medication N % Mean Median Min Max % of episodes 0 20 40 60 80 100 % Immunate (IU) 2014 41.2 2555 1500 150 17500 Other human factor VIII 1760 36.0 - - - - Octanate (IU) 404 8.3 1519 1000 250 8000 Immunine (IU) 224 4.6 1676 1500 3 6000 Fanhdi (IU) 184 3.8 1330 1000 250 6000 Purified factor IX, other (IU) 142 2.9 1324 1200 500 2400 Prothrombin complex, other (IU) 76 1.6 979 1200 500 1200 Octanine (IU) 42 0.9 1548 1500 500 6000 Hemofil M (IU) 12 0.2 576 500 306 1000 Other recombinant factor IX (U) 9 0.2 1425 1000 325 4000 Recombinant, other (IU) 7 0.1 943 1200 600 1200 Coagulation FVIII (Red cross) (IU) 6 0.1 1333 1000 1000 2000 Hyate C (IU) 6 0.1 750 750 500 1000 Activated prothrombin complex, other (U) 2 0.0 1100 1100 1000 1200 NovoSeven (mg) 2 0.0 3.6 3.6 2.4 4.8 Koate (IU) 1 0.0 1000 1000 1000 1000 Kogenate (IU) 1 0.0 1000 1000 1000 1000 Plasma derived factor I (IU) 1 0.0 1200 1200 1200 1200 Recombinant factor IX, other (IU) 1 0.0 2000 2000 2000 2000 Total 4894* 100.0 8.3 4.6 3.8 2.9 1.6 0.9 0.2 0.2 0.1 0.1 0.1 0.0 0.0 0.0 0.0 0.0 0.0 41.2 36.0 * more than one medication was used for 330 bleeding episodes