ČNHP Současný stav. ÚspěchyČNHP (ve spolupráci s ČHS) Podpora odborných společností Po svémvznikučnhp požádalo podporutyto odborné společnosti

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1 ČNHP Současný stav Blatný J, Smejkal P. Koordinátoři ČNHP Organizační struktura Vznikala postupně zezdola po dobu 4 let Je demokraticky formovaná a odpovídá současným požadavkům center Rozhoduje většina(hlasováním), volební období Je formálně zakotvená(deklarace) Jednotlivá centra nominují zástupce Zvláště děti, zvláště dospělí Zástupci center tvoří KR KR volívr Ta vybere ze svého středu koordinátory Podpora odborných společností Po svémvznikučnhp požádalo podporutyto odborné společnosti ČHS ČSTH ČPS Ty souhlasily, což deklarováno podpisy předsedů pod deklarací ČNHP ČNHP však není součástí/strukturou žádné odborné společnosti, ale je mezioborovým gremiem zaineteresovaných odborníků ÚspěchyČNHP (ve spolupráci s ČHS) Vytvoření formální sítě center a jejich akreditace za účasti odborné veřejnosti a VZP První audit, Re-audit vždyá roky V roce4 iaudit MZda ZP Úspěšné jednání s NRC/MZd vedoucí k vynětí úhradyléčbyhemofiliez DRG a z paušálů Úzká spoluráce v rámci projektů Evropské komise EUHASS, EUHANET a stím související PR české hemofilické péče

2 Úspěchy ČNHP Spolupráce s WFH, EHC a ISTH Od roku opětdodávámedata pro WFH po přetržce od roku 996 Jubilejní kongres EHC v Praze Spolupráce na QoL dotaznících pro EU stran léčby hemofilie Edukační aktivity v rámci OPVK Reprezentativnídata o léčběhemofiliea obdobných onemocnění v zemi Tvorba standardů péče, publikace Plány do budoucna Pravidelné audity center obou úrovní ve spolupráci s odbornými společnostmi, MZd, ZP Pokračování certifikace EU center v rámci EUHANET Udržení výkonové úhrady antihemofilik Rozvíjení systémů standardů a doporučení v rámci péče o vrozené krv. choroby Edukace/sběr dat a feed-back /zlepšování PR Spolupráce s odbornými společnostmi Trvale udržitelný rozvoj(nejen v rámci OPVK) Financování registru ČNHP The current status of care for persons with haemophilia and von Willebrand s disease registered within CNHP registry Jan Blatný, Petra Ovesná on behalfof Centres contributing to common database of the CNHP (Czech National Haemophilia Programme) April 4 Registr ČNHP vznikl díky projektu OPVK, za přispěníevropskéhosociálníhofondu, MŠMT ČR, Evropské Unie V současnédoběje ČNHP financovánz grantu a sponsorských darů skrze MU Brno. Sponsoři ani poskytovatelé darů nezasahují do databáze ČNHP, ani jinak neovliňují sběr a zpracování dat

3 Sample size, valid records N=9 Persons registered in HemIS/CNHP in whole history until 3..3 Validation* N=7 Records excluded from further analyses: -4deceased persons - 6 persons lost from evidence -64persons switched to other centre, which does not participate in CNHP - 4 unfilled minimal registration record - 5 duplicate records - women-carriersofhaemophilia - 4 persons with changed diagnosis valid persons N=65 N=48 Part A Persons with haemophilia (PWH) Haemophilia Hereditary deficiency of other clotting Von Willebrand s disease Sample size Participating centres in CNHP N=7 Valid persons Valid persons Paediatric centres N % Adult centres N % Haemophilia N=65 valid persons Hereditary deficiency of other clotting N=48 Von Willebrand s disease FN Motol Dpt. of PediatricHaematology and Oncology 78. FN Brno DN Dpt. of Pediatric Haematology FNHK Dpt. of Pediatric Medicine FN Ostrava Dpt. of Pediatric Medicine UnL Pediatric Dpt. Haematology CB Pediatric Dpt. 3. FN Plzen Pediatric Dpt..7 FN Olomouc Dpt. of Pediatric Medicine 9.4 FN Brno OKH 3.5 FN Ostrava Blood centre 67.4 FN Olomouc Haemato-Oncology Dpt FN Plzen UKBH KN Liberec OKH CB OKH FN a LF HK IV. IHK 6 4. * N = 33 N = 4 * Persons under 9 years old in 3

4 Age Type and severity of haemophilia I Age at diagnosis (years) N 5* Current age (years) N 644 Type of haemophilia Haemophilia A (N=556) Severity of haemophilia(n=643*) Mild (N=96) Mean 8.3 Median (min-max) ( 8) Mean 3.6 Median(min -max) 5 ( 9) Haemophilia B (N=88) Moderate (N=) Severe (N=46) 4, 5, 35, 3, 5,, 5,, 5,, 3,3 3,,9 8, 4,8 3,,6 3,, 5,, 5, 8, 8, 4,,,7 9,3,7 5, 3,7% 86,3% 38,3% 5,7% 46,%,, >65 age (years) * Missing information on year of diagnosis in 4 persons. age (years) * Severity of haemophilia not known in person. Type and severity of haemophilia II Current age according to type and severity of haemophilia Typ hemofilie a věk při diagnóze N = 4 Haemophilia A (N=555 ) ) Mild (N=67) Moderate (N=74) Severe (N=4) Haemophilia B (N=88) Mild (N=9) Moderate (N=7) Severe (N=3) Current age (years) median th 9 th percentile Current age (years) ,6% 48,% 36,4% 33,% Haem A Haem B Mild Moderate Severe Inhibitor 3,3% 3,7% Haemophilia A Haemophilia B Currentage (years) Mild* Moderate* Severe* Inhibitor N Mean ( 9) 5 ( 68) Median(min max) 5 ( 9) 9 ( 7) 7.5 ( 74) ( 7) ) Severity not known in person with haemophilia A. Current age = age reached in year 3 * including persons with inhibitor + in 3

5 Hepatitis experienced Hepatitis experienced N=33 Experienced hepatitis Experienced hepatitis Yes (N=49) Yes (N=) No (N=455) Not known (N=4) 6,% 3,% N=49 5% % 5% % 9,9%,5% No (N=3) 99,6% N= One child has hepatitis C. 7,7% 5% 4,8% % Hep A (N=9) Hep B (N=6) Hep C (N=4) type of hepatitis,4% Data from last annual report of each person. Type of hepatitis not specified in persons. One person may have recorded more types of hepatitis. Data from last annual report of each person. Hepatitis experienced N=4 HIV Experienced hepatitis HIV 54,3% Yes (N=48) No (N=3) Not known (N=4) 9,7% 36,% N=48 35% 3% 5% % 5% % 5% % 7,8% Hep A (N=9) 6,% Hep B (N=6) 33,% Hep C (N=3) type of hepatitis 74,4% Positive (N=3) Negative (N=479) Not known / not available (N=6) 5,% N=3,5% HIV-positive persons are adults. Data from last annual report of each person. Type of hepatitis not specified in adults. One person may have recorded more types of hepatitis. Data from last annual report of each person.

6 Data fromyear3 sample size Dětští pacienti: aktualizace údajů za rok Valid persons Persons with annual report in 3 Persons examined in 3 Persons treated in 3 N % N % N % N % 644 % % % % of them with inhibitor 33 % % 3 9.4% % of them with inhibitor % % % 3 56.% of them with inhibitor Persons with haemophilia with inhibitors inhibitor was recorded in persons in year 3-7 children and 5 adults - haemophiliaa and heamophiliab -severe, moderate and mildhaemophilia -8 HR and LR (other two patients had in 3 low level of inhibitor due to by-pass therapy only) -3 patients were treated with rfviia, patient with apcc, other patients both with rfviiaand apcc - ITT was started in 3 children - patients were without any recorded treatment N=634 Frequency of bleeding requiring treatment in 3 N=634 Frequency of bleeding requiring treatment in 3 N=9 6% 5% 4% 3% % % % > 9% 8% 7% 6% 5% 4% 3% % % % > 5% 45% 4% 35% 3% 5% % 5% % 5% % > 8% 7% 6% 5% 4% 3% % % % > Haemophilia A Haemophilia B Frequency of bleeding Mild* Moderate* Severe* Inhibitor N total N valid Mean.4.7 / ( 4) ( 53) Median(min max) ( 7) ( 3) 5.5 / 4 ( 4) 6 ( 3) Frequency of bleeding is missing in 7 persons. mean and median of frequency of bleeding in persons with severe haemophilia without inhibitor on permanent prophylaxis Haemophilia A Haemophilia B Frequency of bleeding Mild* Moderate* Severe* Inhibitor N total N valid Mean / ( 59) ( 7) Median(min max) ( 7) ( ) 6 / 6 ( 59) ( 3) Frequency of bleeding is missing in 9 children. mean and median of frequency of bleeding in children with severe haemophilia without inhibitor on permanent prophylaxis

7 6% 5% 4% 3% % % % Frequency of bleeding requiring treatment in > Haemophilia A Haemophilia B Frequency of bleeding Mild* Moderate* Severe* Inhibitor N total N valid Mean.4.5. / ( 4).5 ( 53) Median(min max) ( 7) ( 3) 5 / 3 ( 4) ( 5) Frequency of bleeding is missing in 88 adults. 9% 8% 7% 6% 5% 4% 3% % % % N= > mean and median of frequency of bleeding in adults with severe haemophilia without inhibitor on permanent prophylaxis Location of (any) bleeds in 3 3 (56.8%) children experienced bleeding requiring treatment at least once in year; 856 bleeds were recorded in total, 48 bleeds required hospitalization. 99 (43.%) children recorded no bleed during year 3. In total 33 persons recorded bleeds of any location*. * all recorded bleeds, regardless of requirement of treatment Joints (N=9) Muscles (N=43) Subcutaneous (N=6) Urogenital tract (N=3) GIT (N=) Tooth extraction (N=7) Surgery (N=) Other (N=5),3%,% 7,4% 8,7% 8,8%,7% 6,% N=9 39,3% % % % 3% 4% 5% Location of (any) bleeds in 3 N=45 Treatment N= (49.5%) adults experienced bleeding requiring treatment at least once in year; 975 bleeds were recorded in total, 4 bleeds required hospitalization. 3 (5.5%) adults have recorded no bleed during year 3. Information on frequency of bleeding is missing in 3 examined adults. In total 38 persons recorded bleeds of any location*. Joints (N=) 7,6% Muscles (N=44) Subcutaneous (N=) Urogenital tract (N=7) GIT (N=) Tooth extraction (N=5) Surgery (N=3) Other (N=3),9% 3,%,7%,5% 3,7% 3,% 7,5% Without bleeding (N=3) 5,5% % % 4% 6% 8% Immunate Fanhdi Immunine Octanate Octanine Haemoctin Not known Feiba Advate Kogenate Recombinate Other FIX Other FVIII NovoSeven Plasma-derived N = 77 Recombinant N = (6.4%) persons received factor concentrates in 3 (39 of them received more than one type/make of concentrate). Plasma-derived were administered more frequently in 77 (43.7% of all PWHs) persons, whereas recombinant in 3 (9.4% of all PWHs) persons. Twenty persons were treated with both plasma-derived and recombinant factor. * all recorded bleeds, regardless of requirement of treatment 5 5 number of persons

8 Treatment N=9 Treatment N=45 Immunate Fanhdi Immunine Octanate Octanine Haemoctin Not known Feiba Advate Kogenate Recombinate Other FIX Other FVIII NovoSeven Plasma-derived N = 7 Recombinant N = (6.7%) children received factor concentrates in 3 ( of them received more than one type/make of concentrate). Plasma-derived were administered in 7 (3.4% of all) children, recombinant in 77 (33.6% of all) children. Twelve children were treated with both plasma-derived and recombinant factor. Immunate Fanhdi Immunine Octanate Octanine Haemoctin Not known Feiba Advate Kogenate Recombinate Other FIX Other FVIII NovoSeven Plasma-derived N = 5 Recombinant N = (6.%) adults received factor concentrates in 3 (7 of them received more than one type/make of concentrate). Plasma-derived were administered more frequently in 5 (5.6% of all) adults, whereas recombinant in46 (.4% of all)adults. Eight adults were treated with both plasma-derived and recombinant factor number of persons 5 5 number of persons Type of treatment N=634 Bleeding requiring treatment according to prophylaxis N=9 % 8% 6% 4% % % 7,3% Home treatment (N=37) 38,8% Prophylaxis (N=7) type of treatment 4,7% 5,7% 79,7% Type of prophylaxis (N=7) Permanent (N=37) Frequency of bleeding per year median th 9 th percentile no yes no yes no yes no yes Frequency of bleeding Mild* Moderate* Severe* Inhibitor N Mean permanent prophylaxis Temporary weeks(n=8) Median(min max) ( 7) ( ) ( 6) 8 ( 59) 6 ( 45) 7 ( 3).5 (7 9) Temporary < weeks(n=7) childrenon permanent prophylaxis (%) 7 (5.6%) 6 (84.9%) 4 (57.%) % ofdrugs(fviii andfix) consumedby childrenon permanent prophylaxix % 6.6 % 75.6 %. %

9 Joint and otherbleedsaccordingto prophylaxis Frequency of bleeding Mild* Moderate* Severe* Inhibitor N total N valid JOINT BLEEDS Mean Median(range) ( 4) ( ) ( ) 6 ( 3) ( 9) 9 ( 8) 6 (4 9) Total bleeding rate OTHER BLEEDS Mean Median(range) ( 5) ( ) ( 6) ( 36) 3 ( 6) 8 ( ) 5.5 (3 ) Total bleeding rate Frequency of bleeding per year no yes no yes Joint bleeds Other bleeds 3 median th 9 th percentile permanent prophylaxis N=9 Bleeding requiring treatment according to prophylaxis Frequency of bleeding per year median th 9 th percentile, 3, no yes no yes no yes no yes Frequency of bleeding Mild* Moderate* Severe* Inhibitor N Mean Median(min max) ( 6) ( 3) ( ) ( 4) 3 ( 35) ( 5) adultson permanent prophylaxis 7 (6.7%) 57 (38.3%) % ofdrugs(fviii andfix) consumedby adultson permanent prophylaxix % 56. % - N=45 permanent prophylaxis Joint and otherbleedsaccordingto prophylaxis Frequency of bleeding Mild* Moderate* Severe* Inhibitor N total N valid JOINT BLEEDS Mean Median(range) ( 5) ( 7) ( ) ( 4) ( 3) Total bleeding rate OTHER BLEEDS Mean Median(range) ( 3) ( 3) ( 4) ( 8) ( ) Total bleeding rate Frequency of bleeding per year no yes no yes Joint bleeds Other bleeds median th 9 th percentile permanent prophylaxis N=45 Drug Consumption of drugs Total annual consumption Všichni pacienti treated persons treated person examined persons examined person IU Immunate IU IU Fanhdi IU IU IU Octanate 8 75 IU IU IU Haemoctin 38 5 IU IU 84.9 IU FVIII Advate IU IU IU 474 Kogenate IU IU 83. IU Recombinate 858 IU IU IU Bax IU IU 3. IU Other recombinant 4 5 IU IU 59.5 IU FVIII celkem IU IU IU Immunine IU IU 8.3 IU FIX Octanine 388 IU IU IU 77 Other recombinant IU IU 8 3. IU FIX celkem IU IU IU apcc Feiba 35 IU IU rfviia NovoSeven 4 mg 5 8. mg Plasma-derived - TOTAL* IU IU IU Recombinant - TOTAL* IU IU IU TOTAL CONSUMTION * IU IU IU plasma-derived = Immunate, Fanhdi, Octanate, Immunine, Octanine, Other plasma-derived recombinant = Advate, Kogenate, Recombinate, BAX 36, Other recombinant *TOTAL CONSUMPTION = all mentioned drugs excluding Feiba and NovoSeven

10 FVIII Drug Consumption of drugs Total annual consumption Všichni pacienti treated persons treated child examined children examined child IU Immunate IU IU Fanhdi 58 5 IU IU IU Octanate IU IU 9 5. IU Haemoctin 65 IU 65. IU 9.6 IU Advate IU IU IU Kogenate 75 IU IU 6. IU BAX IU IU IU Studie gena IU IU 39.7 IU FVIII celkem IU IU IU Immunine IU IU IU Octanine IU IU IU FIX 33 Other recombinant 4 IU IU IU FIX celkem IU IU IU apcc Feiba 3 IU 5. IU rfviia NovoSeven 695 mg mg Plasma-derived - TOTAL* 5 49 IU IU IU Recombinant - TOTAL* IU IU IU TOTAL CONSUMTION * 5 96 IU IU IU plasma-derived = Immunate, Fanhdi, Octanate, Immunine, Octanine, Other plasma-derived recombinant = Advate, Kogenate, Recombinate, BAX 36, Other recombinant *TOTAL CONSUMPTION = all mentioned drugs excluding Feiba and NovoSeven FVIII Drug Consumption of drugs Total annual consumption Všichni pacienti treated persons treated person examined adults examined adult IU Immunate IU IU Fanhdi 4 7 IU IU IU Octanate 388 IU IU IU Haemoctin 6 5 IU 8 5. IU 744. IU Advate 88 IU 8. IU IU Kogenate 3 IU IU 65.9 IU Recombinate 858 IU IU IU BAX IU 5 5. IU 6.5 IU FVIII celkem IU IU IU Immunine 57 9 IU IU IU Octanine 33 IU IU IU FIX 44 Other recombinant IU IU IU FIX celkem IU IU IU apcc Feiba IU. IU rfviia NovoSeven 445 mg 445. mg Plasma-derived - TOTAL* 4 95 IU IU IU Recombinant - TOTAL* IU IU IU TOTAL CONSUMTION * IU IU IU plasma-derived = Immunate, Fanhdi, Octanate, Immunine, Octanine, Other plasma-derived recombinant = Advate, Kogenate, Recombinate, BAX 36, Other recombinant *TOTAL CONSUMPTION = all mentioned drugs excluding Feiba and NovoSeven Děkuji za pozornost Poděkovánílékařům, sestráma datamanagerům všech center ČNHP Poděkování IBA Poděkování sponsorům projektu Více na