Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study

Haemophilia ◽  
2013 ◽  
Vol 19 (5) ◽  
pp. 727-735 ◽  
Author(s):  
M. J. Manco-Johnson ◽  
J. Sanders ◽  
N. Ewing ◽  
N. Rodriguez ◽  
M. Tarantino ◽  
...  
Keyword(s):  
Prophylactic Treatment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand

Haemophilia registry of the Medical Committee of the Swiss Hemophilia Society

2012 ◽  
Vol 32 (S 01) ◽  
pp. S20-S24 ◽  
Author(s):  
N. von der Weid
Keyword(s):  
Quality Control ◽  
Young Adults ◽  
Prophylactic Treatment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Coagulation Disorders ◽  
On Demand ◽  
Viii And Ix ◽  
Year 2000 ◽  
Low Rate

SummaryThe Haemophilia Registry of the Swiss Haemo philia Society was created in the year 2000. The latest records from October 31st 2011 are presented here. Included are all patients with haemophilia A or B and other inherited coagulation disorders (including VWD patients with R-Co activity below 10%) known and followed by the 11 paediatric and 12 adult haemophilia treatment or reference centers. Currently there are 950 patients registered, the majority of which (585) having haemophilia A. Disease severity is graded according to ISTH criteria and its distribution between mild, moderate and severe haemophilia is similar to data from other European and American registries. The majority (about two thirds) of Swiss patients with haemophilia A or B are treated on-demand, with only about 20% of patients being on prophylaxis. The figure is different in paediatrics and young adults (1st and 2nd decades), where 80 to 90% of patients with haemophilia A are under regular prophylaxis. Interestingly enough, use of factor concentrates, although readily available, is rather low in Switzerland, especially when taking the country’s GDP into account: The total amount of factor VIII and IX was 4.94 U pro capita, comparable to other European countries with distinctly lower incomes (Poland, Slovakia, Hungary). This finding is mainly due to the afore mentioned low rate of prophylactic treatment of haemophilia in our country. Our registry remains an important instrument of quality control of haemophilia therapy in Switzerland.

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

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

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.

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.

scholarly journals Once‐weekly prophylactic treatment vs. on‐demand treatment with nonacog alfa in patients with moderately severe to severe haemophilia B

Haemophilia ◽  
2016 ◽  
Vol 22 (3) ◽  
pp. 381-388 ◽  
Author(s):  
K. Kavakli ◽  
L. Smith ◽  
K. Kuliczkowski ◽  
J. Korth‐Bradley ◽  
C. W. You ◽  
...  
Keyword(s):  
Prophylactic Treatment ◽  
Severe Haemophilia ◽  
Haemophilia B ◽  
On Demand

Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B

1992 ◽  
Vol 232 (1) ◽  
pp. 25-32 ◽  
Author(s):  
I. M. NILSSON ◽  
E. BERNTORP ◽  
T. LÖFQVIST ◽  
H. PETTERSSON
Keyword(s):  
Prophylactic Treatment ◽  
Haemophilia A ◽  
Severe Haemophilia

Human-Cl Rhfviii Effectively and Safely Prevents Bleeding Episodes in Previously Treated Adult Patients with Severe Haemophilia A

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 1132-1132
Author(s):  
Sigurd Knaub ◽  
Toshko Lissitchkov ◽  
Kingsley Hampton ◽  
Mario Von Depka ◽  
Savita Rangarajan ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Research Funding ◽  
Prophylactic Treatment ◽  
Hemophilia A ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Open Label ◽  
Bleeding Rate ◽  
Previously Treated

Abstract Abstract 1132 The main purpose of this prospective, multi-center, open-label phase 3 study was to assess the efficacy of prophylactic treatment with Human-cl rhFVIII, the first human cell-line derived recombinant FVIII, in previously treated patients (PTPs) with severe haemophilia A. Patients were to receive 30–40 international units (IU) FVIII of Human-cl rhFVIII per kg every other day for 6 months. Efficacy of preventing and treating bleeds were judged using objective criteria taking the monthly bleeding rate and the number of infusions needed to manage a break-through bleed into account. In-vivo recovery (IVR) was determined at the beginning of the study and after 3 and 6 months. FVIII:C was measured by validated chromogenic (CHR) and one-stage (OS) assays in a central laboratory, which also assigned drug potencies. Inhibitor activity was determined using the Nijmegen modification of the Bethesda assay before the first administration and at defined intervals thereafter. Thirty-two patients between 18 and 75 years of age were enrolled from 11 centres in Europe and treated prophylactically for 6.0±0.9 months (mean ± SD) with a mean prophylactic dose of 32.8 IU/kg. Sixteen patients never bled, 11 patients bled once and 5 more than once. The mean total and spontaneous monthly bleeding rate was 0.188±0.307 and 0.095±0.211, respectively. Efficacy of the prophylactic treatment was “excellent” in all patients for spontaneous BEs and “excellent” or “good” in all patients but one for all types of bleeds. All treatments of bleeds were rated as “excellent” (71.4%) or “good” (28.6%). The IVR at baseline was 2.6±0.5 % per IU/kg for the CHR and 2.2±0.5 % per IU/kg for the OS assay and remained stable during the study. A total of 2921 infusions were given in the study. Human-cl rhFVIII was well tolerated and no patient experienced a related serious adverse event. No FVIII inhibitors were detected. Conclusion: The data indicate that Human-cl rh FVIII is safe and efficacious in preventing and treating bleeds in PTPs with severe haemophlia A. Disclosures: Knaub: Octapharma AG: Employment. Lissitchkov:Octapharma AG: PI Other. Tuddenham:College London: Consultancy, Employment, Gene therapy for hemophilia A, Gene therapy for hemophilia A Patents & Royalties, Research Funding. Collins:Octapharma AG: Consultancy. Oldenburg:d and e: Baxter, Bayer, Biotest, CSL-Behring, Grifols, Inspiration, NovoNordisk, Octapharma, Pfizer e: Biogen IDec, Swedish Orphan Biovitrum: Honoraria, Research Funding. Bichler:Octapharma AG: Employment.

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