Secondary prophylaxis treatment versus on-demand treatment for patients with severe haemophilia A: comparisons of cost and outcomes in Taiwan

Haemophilia ◽  
2010 ◽  
Vol 17 (1) ◽  
pp. 45-54 ◽  
Author(s):  
W.-S. LIOU ◽  
T.-C. TU ◽  
S.-N. CHENG ◽  
T.-Y. CHOU ◽  
C.-F. LEE ◽  
...  
Keyword(s):  
Secondary Prophylaxis ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand

Effects of primary and secondary prophylaxis on the clinical expression of joint damage in children with severe haemophilia A

2008 ◽  
Vol 99 (01) ◽  
pp. 71-76 ◽  
Author(s):  
Karin Kurnik ◽  
Frauke Friedrichs ◽  
Susan Halimeh ◽  
Anne Krümpel ◽  
Christoph Bidlingmaier ◽  
...  
Keyword(s):  
Bleeding Episode ◽  
Joint Damage ◽  
Primary Prophylaxis ◽  
Secondary Prophylaxis ◽  
Outcome Variable ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand ◽  
Haemophilic Arthropathy ◽  
Bleeding Episodes

SummaryPatients with severe haemophilia A (HA) can either be treated by regular FVIII infusions twice or three times per week (prophylaxis), or only in case of bleeding episodes (on-demand). Whereas prophylaxis reduces the number of bleeding episodes and may therefore prevent the development of haemophilic arthropathy, there is still a lot of controversy surrounding recommendations on age and dose at start of prophylactic regimens. The present database study was performed to investigate the role of primary versus secondary prophylaxis in HA children. The outcome variable was imaging-proven haemophilic joint damage. Forty-two children were initially treated with primary prophylaxis following the first bleeding episode, and were frequency-matched (year of birth, catchment area) to 67 pa- tients receiving “on-demand” therapy with an early switch to “secondary prophylaxis”. In multivariate analysis adjusted for the HA mutation type and the presence or absence of thrombophilia, the Pettersson score investigated at a median age of 12.5 years in joints with at least one documented bleeding episode was not significantly different between the two patient groups (p=0.944),and no statistically significant differences were found in patients with target joints (p=0.3), nor in children in whom synovitis had occurred (p=0.77). No conclusion can be drawn from the data presented herein whether primary prophylaxis or an early start of secondary prophylaxis is superior with respect to joint outcome in children with severe HA.

Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study

2015 ◽  
Vol 114 (07) ◽  
pp. 35-45 ◽  
Author(s):  
Giulio Feola ◽  
Angelo Claudio Molinari ◽  
Cristina Santoro ◽  
Gianna Franca Rivolta ◽  
Dorina Bianca Cultrera ◽  
...  
Keyword(s):  
Controlled Trial ◽  
Secondary Prophylaxis ◽  
Annual Number ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Open Label ◽  
On Demand ◽  
Adolescents And Adults ◽  
Bleeding Episodes

SummaryRigorous evidence is lacking on long-term outcomes of factor VIII (FVIII) prophylaxis initiated in adolescent or adult patients with severe haemophilia A. The prospective, open-label Prophylaxis versus On-demand Therapy Through Economic Report (POTTER) study (Clinical-Trials.gov NCT01159587) compared long-term late secondary prophylaxis (recombinant FVIII-FS 20–30 IU/kg thrice weekly) with on-demand treatment in patients aged 12 to 55 years with severe haemophilia A. The annual number of joint bleeding episodes (primary endpoint), total bleeding episodes, orthopaedic and radiologic (Pettersson) scores, health-related quality of life (HRQoL), pharmacoeconomic impact, and safety were evaluated over a > 5-year period (2004–2010). Fifty-eight patients were enrolled at 11 centres in Italy; 53 (27 prophylaxis, 26 on demand) were evaluated and stratified into 2 age subgroups (12–25 and 26–55 years). Patients receiving prophylaxis experienced a significantly lower number of joint bleeding episodes vs the on–demand group (annualised bleeding rate, 1.97 vs 16.80 and 2.46 vs 16.71 in younger and older patients, respectively; p=0.0043). Results were similar for total bleeding episodes. Prophylaxis was associated with significantly fewer target joints (p< 0.001), better orthopaedic (p=0.0019) and Pettersson (p=0.0177) scores, better HRQoL, and fewer days of everyday activities lost (p< 0.0001) but required significantly higher FVIII product consumption. The POTTER study is the first prospective, controlled trial documenting long-term benefits of late secondary prophylaxis in adolescents and adults with severe haemophilia A. The benefits of reduced bleeding frequency, improved joint status, and HRQoL may offset the higher FVIII consumption and costs.

Secondary prophylaxis in an adult patient with severe haemophilia A

2013 ◽  
Vol 33 (03) ◽  
pp. 241-244 ◽  
Author(s):  
C. Schubert ◽  
S. Schlenkrich
Keyword(s):  
Factor Viii ◽  
Prospective Studies ◽  
Primary Prophylaxis ◽  
Secondary Prophylaxis ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Bleeding Rate ◽  
On Demand ◽  
Optimal Dosing ◽  
Joint Protection

SummaryPrimary prophylaxis has been proven as the gold standard for preserving joint function in severe haemophilia. Secondary prophylaxis in children has also been shown to have a more beneficial effect on joint protection as on-demand treatment. The outcome of delayed secondary prophylaxis started in adulthood is rarely studied. The case of a man (age: 47 years) with severe haemophilia A demonstrates the successful switch from on-demand therapy to prophylaxis with sub-sequent reduction of annual bleeding rate and number of days of work lost. The small increase of factor VIII consumption (16%) and the decrease of bleeding rate are compared with data from two recently published prospective studies.An optimal dosing schedule needs to be discussed and larger and longer prospective studies targeting the benefits of secondary prophylaxis with factor VIII started in adulthood are necessary.

scholarly journals Cost-Utility of Prophylaxis VS. On-Demand Treatment In Severe Haemophilia A Patients

2016 ◽  
Vol 19 (7) ◽  
pp. A591
Author(s):  
Z Gharibnaseri ◽  
M Davari ◽  
A Cheraghali ◽  
P Eshghi ◽  
R Ravanbod ◽  
...  
Keyword(s):  
Cost Utility ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand

Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates

2016 ◽  
Vol 115 (05) ◽  
pp. 931-938 ◽  
Author(s):  
Annelies Nijdam ◽  
Wouter Foppen ◽  
Piet de Kleijn ◽  
Evelien P. Mauser-Bunschoten ◽  
Goris Roosendaal ◽  
...  
Keyword(s):  
Functional Limitations ◽  
Objective Assessment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand ◽  
Recommended Treatment ◽  
Objective Monitoring ◽  
Joint Health ◽  
Observational Cohort

SummaryProphylaxis is the recommended treatment for children with severe haemophilia A, but whether prophylaxis should be continued in adulthood is still under debate. Previous studies with limited follow-up have suggested that some patients may be able to stop prophylaxis in adulthood, while maintaining good joint health. This single-centre observational cohort study examined patients with severe haemophilia A born 1970–1988 without inhibitor development, and assessed the long-term consequences of discontinuing prophylaxis. Patient-initiated changes in prophylaxis, including all switches to on-demand treatment lasting a minimum of two consecutive weeks, were recorded from the time self-infusion began until the last evaluation. Sixty-six patients were evaluated at a median age of 32.4 years: 26 of patients had stopped prophylaxis for a median of 10 years, 15 had interrupted prophylaxis and 59 had continued prophylaxis. Annual joint bleeding rate (AJBR), Haemophilia Joint Health Score (HJHS-2.1; 0–124 points), radiological Pettersson score (0–78 points) and Haemophilia Activities List score (HAL; 100–0 points) were compared between patients who stopped and patients who continued prophylaxis. Although self-reported bleeding rates and functional limitations were similar in both groups (AJBR: 1.5 vs 1.2 and HAL: 84 vs 84 for those who stopped and continued prophylaxis, respectively), objective assessment of joint status showed increased arthropathy after 10 years of on-demand treatment in patients who stopped prophylaxis compared with those who continued (HJHS: 23 vs. 14 and Pettersson: 16 vs 5, respectively; P< 0.01). These results support continuation of long-term prophylaxis in adults and demonstrate the need for objective monitoring of joint status.Trial registration: Dutch Trial Registry number 3098; UTN U1111–1121–7069.

scholarly journals Haemophilia A: Pharmacoeconomic Review of Prophylaxis Treatment versus On-Demand

2015 ◽  
Vol 2015 ◽  
pp. 1-9 ◽  
Author(s):  
Brigid Unim ◽  
Maria Assunta Veneziano ◽  
Antonio Boccia ◽  
Walter Ricciardi ◽  
Giuseppe La Torre
Keyword(s):  
English Language ◽  
Cost Effective ◽  
Primary Prophylaxis ◽  
Secondary Prophylaxis ◽  
Economic Evaluations ◽  
Haemophilia A ◽  
On Demand ◽  
Use Of Resources ◽  
Medium Quality ◽  
Methodological Aspects

Objectives. Haemophilia A is a congenital disorder of coagulation that mainly affects males and causes a considerable use of resources, especially when hemophilic patients are treated with prophylaxis. The aim of the present review was to discuss and appraise the methodological aspects and results of published economic evaluations of haemophilia A treatments in the last decade.Methods. The literature search, performed by consulting four engines, covered studies published between 2002 and 2014. Full economic evaluations published in English language were identified and included in the review. A quality assessment of the studies was also carried out based on Drummond’s checklist.Results. After careful evaluations of the identified records, 5 studies were reviewed. Primary and secondary prophylaxis resulted cost-effective compared to on-demand therapy: the ICER of primary prophylaxis ranged from €40.236 to €59.315/QALY gained, while the ICER of secondary prophylaxis was €40.229/QALY gained. Furthermore, 60% were high quality and 40% were medium quality studies.Conclusions. The review underlines the cost-effectiveness of prophylaxis versus on-demand treatment and the different methodological approaches applied. Further economic evaluations are required with models that reflect the clinical reality and consumption of resources in each country.

Case-Control Pilot Study of the Immune Modulating Effect of FEIBA® on Patients with Haemophilia a and Inhibitors

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4688-4688
Author(s):  
Ihosvany Fernandez Bello ◽  
María Teresa Álvarez Román ◽  
Victor Jiménez Yuste ◽  
Monica Martín Salces ◽  
Gemma Iruin ◽  
...  
Keyword(s):  
Research Funding ◽  
Fas Ligand ◽  
Transforming Growth Factor ◽  
Transforming Growth Factor Β ◽  
Time Frame ◽  
Immunomodulatory Activity ◽  
Type I ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand

Abstract Background: Immune tolerance therapy (ITT) is the only available therapy for eliminating the inhibitor in patients with haemophilia and high-responding inhibitors. However, it is a very expensive treatment with an efficacy of up to 60%-75%. Factors influencing the outcome of ITT are not completely understood. Some studies have suggested that plasma derived factor VIII (FVIII) concentrates might offer certain advantages in the elimination of the inhibitor when compared with recombinant derivatives. The presence of von Willebrand factor (Auerswald et al,Haematologica 2003;88:EREP05; Kreuz et al,Haematologica 2001;86:16-20) and some molecules with immunomodulatory effect like anti-idiotype antibodies, transforming growth factor-β, HLA type I molecules and soluble FAS ligand in plasma derived concentrates may be involved in such effect (Berntorp et al,Haemophilia 2001;7:109-13; Hodge et al,Br J Haematol 2001;115:376-81; Hodge et al, Haemophilia 2006;12:133-9; Ghio et al, Thromb Haemost 2003;89:365-73). The activated prothrombin complex concentrate (FEIBA®) is a plasma derived product used for bleeding control in patients with haemophilia A and high-responding inhibitors. We hypothesize that FEIBA® may have an immunomodulatory activity due to its plasmatic origin. This effect might be observed in patients with intense treatment with FEIBA® (e.g. prophylaxis treatment) by the reduction in levels of antibodies against FVIII compared with patients on demand treatment with this product. Objective: The aim of this study was to compare levels of inhibitors in patients with severe haemophilia A and inhibitors on demand or under prophylactic treatment with FEIBA®. Methods and results: This was a prospective, observational and multicenter study. A total of 12 adult patients with severe haemophilia A and high-responding inhibitors (6 patients on demand and 6 patients under prophylaxis treatment with FEIBA®) were included. All patients had normal levels of serum creatinine, serum alanine aminotransferase and CD4+ lymphocyte counts at inclusion. Levels of inhibitors were determined by Bethesda assay. Levels of inhibitors in patients on prophylaxis with FEIBA® were lower than those observed in patients on demand treatment but differences were not statistical significant. In most of the patients on prophylaxis, levels of inhibitors went down along the prophylaxis regimen. Table. Medical history of patients. Patient Nº Age Type of treatment Usual dose (IU/kg) Frecuency Titer (BU) Time frame with start of prophylaxis (months)** 480101 53 Prophylaxis 60 Three times a week 64* 4.4 80 4.1 96 1.3 250 -1.3 480102 58 Prophylaxis 55 Three times a week 5* 3.3 12 2.1 7 -3.8 12 -6.1 150101 UNK Prophylaxis 60 Three times a week 4* 3.0 5 -6.0 6 -8.0 280101 28 Prophylaxis 56 Every other day 109* 7.3 77 4.1 101 -8.3 101 -9.7 280102 57 On Demand 56 x 8 doses N/A 174* N/A 37 N/A 83 N/A 21 N/A 25 N/A 184 N/A 280103 57 On Demand 55 x 6 doses N/A 8000* N/A 64 N/A 5 N/A 21 N/A 280104 58 On Demand 68 x 6 doses N/A 152* N/A 280105 23 On Demand 68 x 3 doses 40 x 6 doses N/A 30* N/A 080101 28 On Demand 50 x 5 doses N/A 5.2* N/A 7.0 7.2 4.0 7.8 460101 37 Prophylaxis 66 every 48h Negative 21,0 460102 57 On demand 70 2 doses/ bleeding usually 66.5* More than 20 years 300001 50 Prophylaxis 85 Three times a week 8* 3 *Titer at inclusion. **Positive values represent number of months after starting prophylaxis and negative values are referred to number of months before starting prophylaxis. For example, the patient number 480101 had 250 BU at 1.3 months before starting prophylaxis (in the table= -1.3 months) and 64 UB at month 4.4 after initiation of prophylaxis (in the table= 4.4 months). N/A: not applicable. Conclusion: Prophylaxis with FEIBA® might decrease levels of inhibitors in patients with severe haemophilia A and high-responding inhibitors. However, a broader study is needed to confirm this result. Disclosures Iruin: Baxalta: Research Funding. Bonanad:Baxalta: Research Funding. López Fernández:Baxalta: Research Funding. Altisent:Baxalta: Research Funding. García Candel:Baxalta: Research Funding. Rivas Pollmar:Baxalta: Research Funding. Canales:Baxalta: Research Funding.

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