Treatment of Patients with Haemophilia in China: Sub-analysis of the HERO study

Transcription

1 Treatment of Patients with Haemophilia in China: Sub-analysis of the HERO study Baolai HUA Peking Union Medical College Hospital On behalf of Chinese HERO National Advisory Board 12-OCT-2013 Slide 1

2 Japanese Society of Hematology COI disclosure Presenter: Baolai HUA I have no relevant financial relationship to disclose. Slide 2

3 Background and Aims Method Results Conclusions Slide 3

4 Background Haemophilia A and B are serious congenital bleeding disorders whose prognosis and outcomes are improved by comprehensive care and access to treatment 1. Prophylaxis regimen has significant advantages over on-demand treatment for people with haemophilia (PWH) by reducing all types of bleeding with comparable safety 2,3. Home therapy and self-infusion emancipates PWH from frequent clinic visits and improves the quality of life for both PWH and his family 1. The deficiency in replacement factor products and budget in developing countries leads to greater mortality and morbidity Srivastava A, et al. Guidelines for the management of hemophilia. 2 nd edition. 2. Colvin BT, et al. Haemophilia. 2008, 14: Antunes SV, et al. Haemophilia. 2013, 4. Young G. Hematology Am Soc Hematol Educ Program. 2012, 2012: Slide 4

5 Background The Haemophilia Experiences, Results and Opportunities (HERO) study Is a multinational, multidisciplinary research. Focused on psychosocial issues in people with haemophilia and parents of children with haemophilia International advisory board Preliminary literature review Qualitative survey: Interivew based 150 people 7 countries More extensive literature review 1 Quantitative survey: questionnaire-based 1236 people 10 countries 1. Cassis FR, et al. Haemophilia. 2012; 18(3):e Slide 5

6 Aim The primary objective of the HERO study was to quantify the impact of key psychosocial factors affecting PWH and parents of children with haemophilia. The aim of this presentation is to describe the self-reported treatment characteristics of Chinese adults with haemophilia ( 18 years) and compare the results with those of the 9 other countries in this study. Slide 6

7 Background and Aims Method Results Conclusions Slide 7

8 Method As a part of the quantitative survey in the HERO study, a questionnaire survey targeting patients aged 18 years with haemophilia (PWH) was conducted in 10 countries. Key inclusion criteria: Males aged 18 years with haemophilia A or B (with or without inhibitors) Patients currently receiving treatment for haemophilia with one of the following: replacement factor VIII or IX a bypassing agent (recombinant factor VIIa or apcc) Patients not currently receiving treatment but with 1 spontaneous bleed in past 12 months Data on treatment reported by Chinese PWH was extracted and compared with the 9 other countries in this study. Slide 8

9 Background and Aims Method Results Conclusions Slide 9

10 Results Distribution A total of 110 Chinese PWH and 565 non-chinese PWH completed the survey. Most of patients, both Chinese and non-chinese, were with haemophilia A (HA). Total HA HB HWI China, n (%) 110 (100.0) 90 (81.8) 16 (14.5) 4 (3.6) Non-China, n (%) 565 (100.0) 408 (72.2) 70 (12.4) 87 (15.4) Total, n (%) 675 (100.0) 498 (73.8) 86 (12.7) 91 (13.5) HA: Haemophilia A without inhibitors; HB: Haemophilia B without inhibitors; HWI: Haemophilia with inhibitors. Slide 10

11 Results Treatment agents Replacement factor infusion was the dominant treatment for PWH without inhibitors. More Chinese PWH than non-chinese PWH received other remedial treatment. HA Responders FVIII** Cryoprecipitate** FFP** DDAVP* China, n (%) 90 (100.0) 83 (92.2) 58 (64.4) 56 (62.2) 11 (12.2) Non-China, n (%) 408 (100.0) 404 (99.0) 45 (11.0) 23 (5.6) 22 (5.4) HB Responders FIX** FFP** China, n (%) 16 (100.0) 13 (81.3) 10 (62.5) Non-China, n (%) 70 (100.0) 69 (98.6) 12 (17.1) HA: Haemophilia A without inhibitors; HB: Haemophilia B without inhibitors; FVIII: replacement factor VIII; FIX: replacement factor IX; FFP: fresh frozen plasma; DDAVP: desmopressin. *P < 0.05 for China vs. non-china; **P < 0.01 for China vs. non-china. Slide 11

12 Results Treatment strategy Compared with non-chinese PWH: Higher percentage of Chinese PWH adopted on-demand treatment. Lower percentage of Chinese PWH were treated for prophylaxis. OD* PPX* China n = 97 OD + STP Non-China n = Percentage of responders (%) OD: on-demand; PPX: regular prophylaxis; OD + STP: on-demand plus short term prophylaxis. *P < 0.01 for China vs. Non-China. Slide 12

13 Results Treatment location More than a half of Chinese PWH reported their haemophilia treatment infused mostly/always at home. Always at home* Mostly at home but sometimes at the clinic/hospital * Mostly at the clinic/hospital but sometimes at home* First dose at home and if needed further doses at the clinic/hospital Always at the clinic/hospital China n = 97 Non-China n = Percentage of responders (%) *P < 0.01 for China vs. Non-China. Slide 13

14 Results Compliance to prescription Compared with non-chinese PWH: Less Chinese PWH were using replacement therapy exactly as instructed. More Chinese PWH admitted using replacement therapy a lot less than instructed. A lot more than instructed A little more than instructed Exactly as instructed* A little less than instructed A lot less than instructed* Sometimes more, sometimes less China n = 97 Non-China n = *P < 0.01 for China vs. Non-China. Percentage of responders (%) Slide 14

15 Results Barriers Compared with non-chinese PWH, more Chinese PWH reported difficulty in obtaining replacement factor products in the past 5 years. 97.3% of Chinese vs. 29.6% of non-chinese, P <0.01 Over 60% of Chinese PWH considered it was difficult to visit the treatment centre. Very difficult: 23.6% of Chinese vs. 5.7% of non-chinese, P < 0.01 Quite difficult: 37.3% of Chinese vs. 20.7% of non-chinese, P < 0.01 Slide 15

16 Results Difficulties More Chinese PWH than non-chinese PWH considered personal affordability, insurance issues and lack of supply as problems in obtaining replacement factor products. Could not personally afford it** Difficulty with health insurance** Lack of supply at the hospital* Lack of supply throughout the country** Unpredictability of supply Physician reluctant to prescribe it China n = 107 Non-China n = Percentage of responders (%) *P < 0.05 for China vs. Non-China, **P < 0.01 for China vs. Non-China. Slide 16

17 Results Outcomes More Chinese PWH with HA or HB reported spontaneous joint bleeding during the past year, compared with non-chinese PWH. HA: 95.6% of Chinese vs. 71.3% of non-chinese, P <0.01 HB: 100.0% of Chinese vs. 60.0% of non-chinese, P < 0.01 The mean number of bleeds requiring treatment in the last year reported by Chinese PWH was nearly double that reported by non-chinese PWH times for Chinese vs times for non-chinese, P <0.01 Haemophilia-related arthritis was reported in higher percentage of Chinese PWH than non-chinese PWH. 76.4% of Chinese vs. 42.8% of non-chinese, P <0.01 Slide 17

18 Results Perceived disease control Chinese PWH reported lower mean score of disease control than non-chinese PWH. On a 10-point scale (10 = extremely well controlled) Mean score: 5.55 for Chinese vs for non-chinese, P < 0.01 China n = 110 Non-China n = 565 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% 1-Not at all controlled* 2* 3* 4* 5* 6 7 8* 9* 10-Extremely well controlled* *P < 0.01 for China vs. Non-China. Percentage of responders Slide 18

19 Background and Aims Method Results Conclusions Slide 19

20 Conclusions Chinese adults with haemophilia: Mostly adopted on-demand treatment strategy. Were using far less replacement factor products than instructed. Were facing reimbursement issues and personal affordability troubles generally. Experienced more disease difficulties and greater difficulty in obtaining treatment resources compared with the non-chinese population. Slide 20

21 Acknowledgement All investigators with contribution to the HERO study All patients and parents participating the survey Novo Nordisk company Slide 21