A repo t r t f ro m th e Nati tional Haemophili hili a Database

Transcription

1 A report from the National Haemophilia Database

2 Coagulation Defect Male Female Total New Registrations Number of patients newly registered at UK Haemophilia Centres between April 2009 & March 2010 showing their coagulation defect and gender Haemophilia A Haemophilia B Females with VIII deficiency Females with IX deficiency von Willebrand disease F.V deficiency F.VII deficiency FIX Leyden 1 1 F.X deficiency F.XI Deficiency F.XIII Deficiency Fibrinogen Deficiency Prothrombin Deficiency 1 1 Dysfibrinogenaemia Combined V+VIII Deficiency 1 1 Othercombined diagnoses Acquired Haemophilia A Acquired von Willebrands Acquired Deficiency (other) 1 1 Severe PlateletDisorders Glanzmanns Thrombasthenia Severe Platelet Disorders Bernard Soulier 2 2 Other platelet defects Miscellaneous Unclassified Pseudo vw 1 1 Probable von Willebrand's disease Total

3 New Registrations ti of Haemophilia A & B between April 2009 & March 2010, by age at the end of March 2010 and disease severity Coagulation Age Number of Patients Factor VIII/IX level iu/dl Defect (years) 1 >1 and <5 5 Not Known Total Haemophilia A 0 : : : : : : : : : Total Haemophilia B 0 : : : : : : Total

4 Coagulation Defect Number of Patients In Register Treated Percent Treated Haemophilia A 5,346 2, % Haemophilia B 1, % Females with VIII deficiency 1, % Females with IX deficiency % von Willebrand disease 8, % F.V deficiency % FVIId F.VII deficiency i % FIX Leyden % FIX Leyden Carrier % F.X deficiency % F.XI Deficiency 1, % F.XIII Deficiency % Fibrinogen Deficiency % Prothrombin Deficiency % Dysfibrinogenaemia % Combined dii+vii+ix+x IX X Deficiency i % 00% Combined V+VIII Deficiency % In Register The Other combined diagnoses % total Number of Acquired Haemophilia A % Acquired Haemophilia B % patients t with all Acquired von Willebrands % types of bleeding Acquired F.XIII Deficiency % disorder currently in Acquired Deficiency (other) % Severe Platelet Disorders Glanzmann's Thrombasthenia % Severe Platelet l tdisorders Bernard dsoulier % the register and the Other platelet defects 1, % number treated Haemophilia A with Liver Transplant % Haemophilia B with Liver Transplant % during April 2009 & Pseudo vw % March 2010 Probable von Willebrand's disease % Miscellaneous % Unclassified % Total 22,189 5, %

5 In Register The total number of haemophilia A, B and von Willebrand's disease patientscurrently inthe register, by severity, age group and gender Males Females Coagulation Defect 1 <18 years =>18 years <18 years =>18 years >1 & Sub >1 & Sub >1 & Sub >1 & Sub 5 N/K 1 5 N/K 1 5 N/K 1 5 N/K <5 Total <5 Total <5 Total <5 Total Overall Total Haemophilia A Haemophilia B Female with FVIII deficiency Female with FIX deficiency von Willebrand disease

6 Factor VIII Usage (inc. inhibitor) divided by number of Severe Haemophilia A patients by commissioning region

7 Total Number of Severe Haemophilia A & B Patients by commissioning region

8 FVIII Units usage per capita of population

9 Factor IX Usage (inc. inhibitor) divided by number of Severe Haemophilia B patients by commissioning region

10 Factor VIII Usage by commissioning region for Severe Haemophilia A patients (inc. inhibitor) SCG / Region Population * Number of Severe Haemophillia A Patients Total FVIII Units Used Mean Usage Haemophilia A FVIII Units Per Capita Pan Thames Haemophilia Consortium 15,318, ,142, , East Midlands Specialised Commissioning Group 4,433, ,277, , East of England Specialised Commissioning Group 2,335, ,130, , North East Specialised Commissioning Group 2,575, ,679, , North West Specialised Commissioning Group 6,875, ,279, , South West Specialised Commissioning Group 5,209, ,330, , South Central Specialised Commissioning Group 4,062, ,436, , West Midlands Specialised Commissioning Group 5,411, ,728, , Yorkshire and Humber Specialised Commissioning Group 5,213, ,565, , Wales 2,993, ,669, , East Scotland 2,953, ,513, , West Scotland 2,215, ,639, , Northern Ireland 1,775, ,882, ,

11 Factor IX Usage by commissioning region for Severe Haemophilia B patients (inc. inhibitor) SCG / Region Population * Number of Patients Severe Total FIX Units Mean Usage FIX Units Per Haemophillia B Used Haemophilia B Capita Pan Thames Haemophilia Consortium 15,318, ,604, , East Midlands Specialised Commissioning Group 4,433, ,112, , East of England Specialised Commissioning Group 2,335, ,101, , North East Specialised Commissioning Group 2,575, ,493, , North West Specialised Commissioning Group 6,875, ,311, , South West Specialised Commissioning Group 5,209, ,855, , South Central Specialised Commissioning Group 4,062, ,806, , West Midlands Specialised Commissioning Group 5,411, ,516, , Yorkshire and Humber Specialised Commissioning Group 5,213, ,897, , Wales 2,993, ,923, , East Scotland 2,953, ,420, , West Scotland 2,215, ,409, , Northern Ireland 1,775, ,041, ,

12 FVIII usage in severe haemophilia A patients with no current inhibitor by centre, aged less than 18 years Manchester centres with 5-9 patients meeting criteria centres with <=4 patients meeting criteria ,000 Median Units (thousands) per Patient t Centre Patients Total Units Median Units Birmingham Belfast 21 2,355, ,000 Bristol Birmingham 42 3,350,750 64,875 Liverpool Sheffield Bristol 12 1,537,750 75,500 Belfast Cambridge 19 2,595, , Cardiff Cardiff 12 1,402, ,000 Leeds Hammersmith Glasgow 26 4,967, ,375 Nottingham Great Ormond Street 60 13,421, ,000 Oxford Hammersmith 14 2,519,000, 119,000 Newcastle upon Tyne Leeds 28 4,074, ,750 Liverpool 19 1,807,000 78,250 Cambridge Manchester 20 1,314,250 63,750 Royal London Newcastle upon Tyne 25 3,891, ,000 Glasgow Great Ormond Street Nottingham 11 1,336, ,918 St Thomas' Oxford 41 5,601, ,000 Royal Free Royal Free 26 7,486, ,500 Royal London 13 1,993, ,250 excludes outside values Sheffield 29 2,713,000 84,950 St Thomas' 30 7,646, ,000 centres with <=4 patients meeting criteria 41 6,156, ,078 centres with 5 9 patients meeting criteria 86 13,010, ,500

13 FVIII usage in severe haemophilia A patients with no current inhibitor by centre, aged 18 years or more Bristol Nottingham Birmingham Liverpool North Hampshire Glasgow Manchester Canterbury centres with 5-9 patients meeting criteria centres with <=4 patients meeting criteria Cardiff Southampton Hammersmith Oxford Newcastle upon Tyne Edinburgh Royal Free Leeds Cambridge Leicester St George's Royal London St Thomas' Belfast Centre Patients Total Units Median Units Bristol 18 1,634,500 78,250 Nottingham 27 4,571, ,000 Birmingham 68 10,774, ,250 Liverpool 29 5,558, ,000 g North Hampshire 32 5,409, ,500 Glasgow 42 7,169, ,112 Manchester 69 11,853, ,500 Canterbury 18 3,613, ,000 centres with 5 9 patients meeting criteria ,172, ,250 centres with <=4 patients meeting criteria 27 6,902, ,000 Sheffield Cardiff 26 5,179, ,250 Hammersmith 15 3,195, ,500 Southampton 14 3,227, ,500 Sheffield 38 8,349, ,000 Oxford 84 19,177, ,000 Leicester Newcastle upontyne ,000 Edinburgh 19 4,970, ,211 Royal Free 86 19,927, ,750 Leeds 25 6,475, ,000 Cambridge 19 5,337, , ,000 Median Units (thousands) per Patient excludes outside values Leicester 15 3,476,000, 285,000 St George's 22 6,492, ,500 Royal London 35 11,488, ,000 St Thomas' 98 32,836, ,000 Belfast 29 9,610, ,000

14 Mean weight of Severe Haemophilia A males compared to the general population Mean Weight (HpA Males) Lower 95% CI (HpA Males) Upper 95% CI (HpA Males) Mean Weight (Gen Pop) Lower 95% CI (Gen Pop) Upper 95% CI (Gen Pop) yrs yrs yrs yrs yrs yrs 75+ yrs

15 Median FVIII units used per kilogram body weight per year in Severe Haemophilia A patients, 2009/10 10,000 9,000 8,000 ts per kilogram Unit 7,000 6,000 5,000 4,000 3,000 2,000 1, yrs yrs yrs yrs yrs yrs yrs 75+ yrs excludes outside values

16 Severe Haemophilia A patients treated with FVIII products with no current inhibitor: median IU issued Median Units (thousan nds) per Patie ent 1, excludes outside values / /10

17 Severe Haemophilia A patients with no current inhibitor between April 2009 & March 2010: median usage by inhibitor history 1,000 thousands) pe er Patient Median Units (t No inhibitor history Inhibitor history No inhibitor history Inhibitor history Aged <18 years excludes outside values Aged >=18 years

18 Severe Haemophilia A & B patients with no current inhibitor using only one productbetweenapril2009 & March 2010:medianusage 1, per Patient Median Units (thousands) Kogenate-Helixate Advate Refacto Benefix Plasma-derived Factor IX Kogenate-Helixate Advate Refacto Benefix Plasma-derived Factor IX Aged <18 years Aged >=18 years excludes outside values

19 Factor VIII units used by UK Haemophilia Centres Coagulation Defect Patients Treated Plasma FVIII Recombinant FVIII Total FVIII Haemophilia A 2,669 40,041, ,375, ,417,195 Females with VIII deficiency , ,011 von Willebrand disease ,390,135 46,082 17,436,217 Combined V+VIII Deficiency 6 29,598 29,598 Other combined diagnoses 15 1,759,500, 1,017,250, 2,776,750, Acquired Haemophilia A 7 214, ,744 Acquired von Willebrands ,500 5, ,500 Miscellaneous 3 38,500 7,500 46,000 Total 3,329 59,805, ,152, ,958,015

20 Factor VIII units by UK Haemophilia Centres: by calendar year between 1989 and 2007 and by financial year from April 2008 onwards 500,000, ,000,000 Recombinant Plasma ,000, ,000, ,000, Units 250,000, ,000, ,000, ,000, ,000,000 0 Time Period

21 Manufacturer Product Total Units Desmopressin Octim Sub cut (15mcg/ml) 5,645 Desmopressin DDAVP (4mcg/ml) 1,265 Desmopressin Octim nasal spray (150mcg/2.5ml vial) 64,090 Haemocompletten P (g) 28 Baxter Advate 179,032,922 FEIBA 19,873,181 Bayer Kogenate 114,536,110 BPL FVIII 8Y 803,820 Optivate 1,340,690 CSL Behring Beriplex 4,000 Haemate P 311,500 Helixate Nexgen 78,804, Grifols Alphanate 59,000 Products usedfor Haemophilia A (including inhibitors) Fanhdi 26,419,790 Novo Nordisk NovoSeven (mg) 23,232.4 Octapharma Octanate 11,107,000 Octaplas 2 Octaplex 1,000 Pfizer ReFacto AF 6,618,765 Refacto 11,381,804

22 Market Share of factor VIII concentrates known to have been used by UK Haemophilia Centres Grifols Plasma 6.77% Ot Octapharma Plasma 2.46% Pfizer Recombinant 4.01% Baxter Recombinant 39.68% CSL Behring Recombinant 17.63% CSL Behring Plasma 3.49% BPL Plasma 0.52% Bayer Recombinant 25.44%

23 Inhibitors by disease severity Number of Patients ever known to have an inhibitor by disease severity Diagnosis 1 iu/dl >1 and <5 iu/dl 5 iu/dl In Reg Inhib. Pts % In Reg Inhib. Pts % In Reg Inhib. Pts % Haemophilia A % % % Haemophilia B % % % von Willebrand disease % % % Patients with a current inhibitor treated between April 2009 and March 2010 by disease severity Diagnosis 1 iu/dl >1 and <5 iu/dl 5 iu/dl In Reg Inhib. % In Reg Inhib. % In Reg Inhib. Pts Pts Pts % Haemophilia A % % % Haemophilia B % % % von Willebrand disease % % %

24 FIX usage in severe Haemophilia B patients with no current inhibitor by centre, aged less than 18years centres with 2 patients meeting criteria Kingston upon Hull Centre Patients Total Units Median Units Cambridge Oxford Manchester centres with 1 patient meeting criteria Hammersmith Belfast Canterbury Great Ormond Street Royal London Newcastle upon Tyne Belfast 4 842,000 92,500 Cambridge 3 67,500 18,000 Canterbury 6 815,000 95,000 Glasgow 5 619, ,750 Great Ormond Street 18 2,430, ,500 Hammersmith 4 358,010 80,505 Kingston upon Hull 3 49,140 11,838 St Thomas' Manchester ,000 55,500 Glasgow Newcastle upon Tyne 5 989, ,000 Nottingham 3 511, ,420 Oxford Nottingham ,500 29,000 Royal Free Royal Free 5 1,461, ,390 Royal London 3 1,018, , ,000 St Thomas' 3 377, ,000 Median Units (thousands) per Patient centres with 1 patient meeting criteria 14 1,192, ,500 excludes outside values centres with 2 patients meeting criteria 16 1,065,250 48,250

25 FIX usage in severe Haemophilia B patients with no current inhibitor by centre, aged 18 years or more Centre Patients Total Units Median Units Lewisham Canterbury Kingston upon Hull Norwich Hammersmith centres with 1 patient meeting criteria Newcastle upon Tyne Birminghami 9 1,779, , Cambridge 17 3,476, ,000 Canterbury 6 894,000 84,000 Cardiff 9 1,555, ,000 Dundee 6 1,058, ,790 Leeds Edinburgh 3 532, ,024 Glasgow 16 2,664, ,500 Hammersmith 3 368, ,000 Kingston upon Hull 4 635,729 91,903 Cardiff Leeds 5 770, ,500 Leicester Glasgow Manchester Cambridge Dundee Birmingham North Hampshire Oxford Royal London Edinburgh centres with 2 patients meeting criteria Leicester 4 550, ,980 Lewisham 5 1,335,426 55,808 Manchester 16 2,983, ,000 Newcastle upon Tyne 11 2,075, ,000 North Hampshire 6 1,161, ,000 Royal Free Norwich 3 340, ,000 St Thomas' Oxford 21 5,359, ,000 Sheffield Royal Free 25 6,369, ,224 Royal London 7 2,748, , ,000 Median Units (thousands) per Patient Sheffield 9 2,376,575, 310,000 St Thomas' 15 3,534, ,000 excludes outside values centres with 1 patient meeting criteria 14 2,045, ,250 centres with 2 patients meeting criteria 14 4,215, ,000

26 Factor IX units used by UK Haemophilia Centres Coagulation Defect Patients Treated Plasma FIX Recombinant FIX Total FIX Units Haemophilia B 611 9,137,700 70,438,519 79,576,219 Females with IX deficiency , ,738 FIX Leyden Carrier 1 14,000 14,000 Combined diagnoses 1 106, , Total 650 9,137,700 71,037,257 80,174,957

27 Total factor IX units by UK Haemophilia Centres: by calendar year between 1989 and 2007 and by financial year from April 2008 onwards 90,000,000 80,000,000 Recombinant Plasma ,000, ,000, Units 50,000,000 40,000,000, 30,000,000 20,000, ,000,000 0 Time Period

28 Products used for Haemophilia B (including inhibitors) Manufacturer Product Total Units Baxter FEIBA 1,819,000 BPL Replenine 3,326,370 CSL Behring Mononine 451,000 Grifols Alphanine 4,924,330 Novo Nordisk NovoSeven (mg) 5,018 Octapharma Nanotiv 436,000 Pfizer BeneFix 70,654,127

29 Market Share of factor IX concentrates known to have been used by UK Haemophilia Centres BPL Plasma 4.15% CSL Behring Plasma 0.56% Grifols Plasma 6.14% Octapharma Plasma 0.54% Pfizer Recombinant 88.60%

30 Products used in the treatment of von Willebrand s patients Manufacturer Product Total Units DDAVP 45 Desmopressin DDAVP (4mcg/ml) 2,002 Desmopressin Octim nasal spray (150mcg/2.5ml vial) 74,672 Desmopressin Octim Sub cut (15mcg/ml) 9,608 Baxter Advate 83,974 Bayer Kogenate 28,500 BPL BPL FVIII 137,655 CSL Behring Haemate P 14,627,600 Haemocompletten P (g) 4 Grifols Alphanate 2,578,880 Fanhdi 46,000 Wilfactin 540,350 Novo Nordisk NovoSeven (mg) 450 Octapharma Wilate 279, Pfizer Refacto AF 1,500

31 Von Willebrand's Sub Types vw Sub types currently in register Number of patients A 4 1B A 171 2B 70 2D 1 2M 85 Normandy (2N) Vincenza 2 Other 30 Type unreported 6066 Total 8879

32 Acquired defects: Materials used by number of patients Product Acquired Acquired von Acquired FX Haemophilia A Willebrands Deficiency Total Advate Desmopressin Octim Sub cut (15mcg/ml) 1 1 Feiba Haemate P Haemocompletten P (g) 2 2 Helixate Nexgen 2 2 Kogenate 1 1 NovoSeven (mg) Octaplex 1 1 Refacto 1 1

33 Adverse events Adverse Event Number of Events New Inhibitor 14 Non A, Non B or Hepatitis C Transmission 0 Other Events * 2 Transfusion Reaction 5 Total 21

34 Coagulation Defect Alive at 31/03/2010 Died by 31/03/2010 Total Haemophilia A 1,882 1,571 3,453 Haemophilia B Females with VIII deficiency Females with IX deficiency von Willebrand disease F.VII deficiency 5 5 F.X deficiency F.XI Deficiency Prothrombin Deficiency 1 1 Combined V+VIII Deficiency 2 2 Other combined diagnoses Acquired Haemophilia A Acquired dhaemophilia B Acquired von Willebrands Estimate of number of Platelet defects 1 1 patients exposed to Haemophilia A with Liver Transplant hepatitis C, based on Haemophilia B with Liver Transplant historical clotting factor Factor X deficiency with Liver Transplant 1 1 concentrate exposure Miscellaneous 1 1 Unclassified 9 9 Total 2,775 1,900 4,675

35 Annualised UK Deaths from Liver Disease Year of death Hepatocellular Carcinoma Liver Failure Total Total

36 Annualised UK Deaths from Liver Disease Hepatocellular Carcinoma Liver Failure Total Number of deaths Year of Death

37 Coagulation Defect Patients at public health risk Patients known to have received implicated batch(es) Haemophilia A Haemophilia B Females with VIII deficiency 59 2 Females with IX deficiency 55 3 von Willebrand disease F.VII deficiency 35 - F.X deficiency 29 8 F.XI Deficiency F.XII (Hageman) defect 2 - F.XIII Deficiency 22 - Fibrinogen Deficiency 2 - vcjd Combined II+VII+IX+X IX X Deficiency 1 - Combined V+VIII Deficiency 7 - Antithrombin III 11 - Acquired Haemophilia A 32 5 Acquired Haemophilia B 1 1 Acquired von Willebrands 6 1 Acquired F.XIII Deficiency 1 - Severe Platelet Disorders - Other 6 - Haemophilia A with Liver Transplant 10 2 Haemophilia B with Liver Transplant 3 1 Misc. 1 - Unclassified 14 1 Temporary coagulation defect, now normal 3 - Combined diagnoses 8 1 Total

38 Report on the enhanced surveillance of patients at risk of CJD Description of age groups at exposure by exposure group At risk group Age group at exposure¹ >80 Total No DOB Recipients of implicated products Recipients of nonimplicated products , ,071 1 Date of exposure by exposure group Total patients by year group of exposure¹ At risk group Missing 2010 year group Total Recipients of implicated products Recipients of nonimplicated products 2, ,072

39 Report on the enhanced surveillance of patients at risk of CJD (Cont d) Description of genotype and sex by exposure groups Gender Exposure group Male Female Not known Recipients of implicated products Recipients of nonimplicated products , Description of genotype and sex by exposure groups Exposure group Total at risk Total deaths Person Years at risk¹ Total Median Range Total post mortems done Recipients of implicated products Recipients i of nonimplicated products , , ,

40 Report on the enhanced surveillance of patients at risk of CJD (Cont d) Age group g distribution of patients who are currently alive by exposure group At risk group Current Age group of living at risk patients (maximum alive) Total No DOB Recipients of implicated products Recipients of non-implicated products ,728 1 Number ofyears lived following exposure for patients currently alive Number of years since exposure Current age group of living patients (maximum alive) Total alive

41 Cause of Death Severity Severe Moderate Mild AIDS Carcinoma Encephalopathy Epilepsy Haemorrhage Hepatocellular Carcinoma Infection (Bacterial) Ischaemic Heart Disease Liver Failure 2-1 Causes of death in Haemophilia A, B, Females with FVIII/FIX deficiency & von Willebrand s disease patients Lymphoproliferative p Malignancy Renal Failure 2-1 Stroke (thrombotic) Stroke (Unknown) Suicide Unknown 1-10 Total

42 Causes of death Rarer Defects Coagulation Defect Cause of Death Number of deaths Acquired Haemophilia A Carcinoma 1 COAD 1 Haemorrhage (Misc) 1 Ischaemic Heart Disease 3 Renal Failure 1 Stroke (Unknown) 1 Unknown 9 Acquired von Willebrands Carcinoma 1 Dysfibrinogenaemia Unknown 1 F.VII deficiency COAD 1 F.X deficiency Ischaemic Heart Disease 1 F.XI Deficiency Carcinoma 3 Infection (Bacterial) 2 Unknown 1 Fibrinogen Deficiency Infection (Bacterial) 1 Unknown 1 Glanzmanns Thrombasthenia Accident 1 Haemophilia A with Liver Transplant Liver Failure 1 Lymphoproliferative Malignancy 1 Defects Combined diagnoses Cerebral haemorrhage 1 Ischaemic Heart Disease 2 Platelet defects (misc) Infection (Bacterial) 1 Ischaemic Heart Disease 1 Liver Failure 1 Peripheral vascular disease 1 Total 39

43 Total number of patients with Haemophilia A, Haemophilia B or von Willebrand's Disease treated by UK Haemophilia Centres Number of Centres : 9 10: 19 20: 29 30: 39 40: 59 60: : : : :299 Number of treated patients

44 Total number of severely affected patients with Haemophilia A, Haemophilia B or von Willebrand's Disease treated by UK Haemophilia Centres Number of Centres : 9 10: 19 20: 39 40: 59 60: : :180 Number of treated patients