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.

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.

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.

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

A cost-effectiveness analysis of primary prophylaxis (PP) versus secondary prophylaxis (SP) with biosimilar myeloid growth factors (MGFs) for preventing chemotherapy-induced febrile neutropenia (FN) in non-Hodgkin lymphoma (NHL) patients at intermediate risk.

2019 ◽  
Vol 37 (27_suppl) ◽  
pp. 107-107
Author(s):  
Gary H. Lyman ◽  
Dylan Mezzio ◽  
Edward C. Li ◽  
Kim Campbell ◽  
Sanjeev Balu
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Cost Effective ◽  
Primary Prophylaxis ◽  
The United States ◽  
Secondary Prophylaxis ◽  
Sensitivity Analyses ◽  
Intermediate Risk ◽  
Related Factors ◽  
Hospitalization Costs

107 Background: Historically, PP with a MGF was recommended in patients with a ≥40% risk for developing chemotherapy-induced FN, based on clinical and regimen-related factors. Previous economic studies provided evidence to lower the threshold to 20%, the current high-risk threshold listed by practice guidelines. Biosimilar MGFs, such as filgrastim-sndz or LA-EP2006 (a proposed pegfilgrastim biosimilar), offer an opportunity to evaluate whether it is cost-effective to further lower the threshold for intermediate-risk regimens (i.e., 10-20% FN risk). Methods: A Markov model was constructed to evaluate the total costs and clinical outcomes of biosimilar or reference MGFs when used as PP vs. SP in patients 55 years old with NHL receiving 6 cycles of R-CHOP chemotherapy (rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone) with no additional FN risk factors. Model inputs, including MGF efficacy and acquisition costs, were estimated from publically available data and literature, as were FN hospitalization costs and clinical utilities. Incremental cost-effectiveness ratios (ICERs) were calculated for cost per FN event avoided, life-year saved (LYS), and quality-adjusted life-year (QALY) gained from a payer perspective within the United States. Deterministic and probabilistic sensitivity analyses were conducted. Results: Use of filgrastim-sndz as PP vs. SP provided an additional 0.130 QALYs (0.144 LYS) at an incremental cost of $5,999. The ICERs were $50,676, $41,761, and $46,207 for cost per FN event avoided, cost per LYS, and cost per QALY gained, respectively. Using LA-EP2006 as PP vs. SP provided an additional 0.166 QALYs (0.184 LYS) at an incremental cost of $17,648. The ICERs were $123,840, $95,963, and $106,265 for cost per FN event avoided, cost per LYS, and cost per QALY gained, respectively. Conclusions: Within NHL patients receiving R-CHOP at an intermediate risk for FN, PP with filgrastim-sndz and LA-EP2006 are cost-effective compared to their respective use as SP based on a cost-effectiveness threshold of $150,000/QALY.

Secondary Prophylaxis in a Cohort of Adolescent and Adult Italian Hemophiliacs.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 1148-1148
Author(s):  
Annarita Tagliaferri ◽  
Gianna Franca Rivolta ◽  
Antonio Coppola ◽  
Cristina Santoro ◽  
Gina Rossetti ◽  
...  
Keyword(s):  
Therapeutic Approach ◽  
Well Being ◽  
Primary Prophylaxis ◽  
Secondary Prophylaxis ◽  
Clinical Impact ◽  
Clotting Factor ◽  
Severe Hemophilia ◽  
Limited Data ◽  
On Demand

Abstract Although primary prophylaxis is a well-established and widely recommended practice for the management of patients with severe hemophilia, there are limited data in the literature documenting the benefit of secondary prophylaxis initiated in adolescence or adulthood. In order to address this issue we have conducted a retrospective study among the 10 Italian Hemophilia Centers on adolescent and adult patients with severe hemophilia switched from on-demand treatment to a long term secondary prophylaxis regimen. On the whole, we collected information on 73 consecutive patients with severe hemophilia (66 hemophilia A and 7 hemophilia B). Their median age started prophylaxis was 12 (range 10–17) for 23 adolescents and 30 (17.4–72) for the 50 adults and continued for a median of years 5.8 (2–14.7) in adolescents and 4 years (1.5–12) in adults. The clinical impact of secondary prophylaxis with respect to the last year on-demand regimen before switching is showed in the following table for each group of patients. Adolescents (n=23) Adults (n=50) On-Demand Prophylaxis On-Demand Prophylaxis Values are reported as mean (standard deviation) Bleedings 32.4(24.8) 2.8 (3.2) 42.7 (24.5) 5.5 (5.2) Joint bleedings 25.6 (22.1) 1.9 (2.3) 36.4 (24.5) 4.2 (4.1) Orthopaedic score 8.4 (7.5) 3.7 (2.4) 21.6 (12.2) 17.7 (12.2) Pettersson score 5.1 (3.4) 5.0 (3.3) 14.7 (13.8) 15.2 (14.3) Work/school days lost 35.1 (34.5) 1.0 (1.5) 33.8 (20.9) 2 (0–17) Concentrate consumption U/Kg/y 2449.8 (1761.9) 3508.3(803.1) 3222.6 (2241.8) 4058.5 (952.3) The decreased joint bleeding, better orthopaedic score, less work/school days lost, delayed progression of arthropathy and improved well-being observed with secondary prophylaxis show the benefit of this therapeutic approach and appear to counterbalance its higher clotting factor consumption. In conclusion large, prospective, long-term randomized studies are needed to definitevely assess the clinical impact of this strategy in adolescent and adult hemophiliacs and to identify those groups of patients who would benefit from this therapeutic approach.

Secondary Prophylaxis Versus On-Demand Treatment in Adult Hemophilia Patients – Increased Factor Use without Increased Benefit?

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2289-2289
Author(s):  
Vipra Sharma ◽  
Ellen White ◽  
Olanrewaju Ladipo ◽  
Alice J. Cohen
Keyword(s):  
Conflicts Of Interest ◽  
Joint Damage ◽  
Well Being ◽  
Primary Prophylaxis ◽  
Secondary Prophylaxis ◽  
Clotting Factor ◽  
Psychiatric History ◽  
Advantages And Disadvantages ◽  
On Demand ◽  
Significant Difference

Abstract Abstract 2289 Introduction: Primary prophylaxis treatment for hemophilia (hem) patients (pts) during childhood has become the standard of care, however there is little consensus on the guidelines for adult management. Currently depending on the physician preference and experience, an adult hem pt can either be treated in response to bleeding (on-demand(OD)), or with regular infusion of clotting factor to prevent bleeding and further joint damage (secondary prophylaxis(P)). This study was undertaken to assess the differences in annual bleeds rates and utilization of clotting factor concentrates between P and OD treatment strategies in adults. Methods: A retrospective study of the 46 patients with moderate or severe hem at the Newark Beth Israel Comprehensive Hemophilia Center was undertaken. Data on treatment and outcomes were evaluated for pts treated with P and those treated with OD. A chart review was done to evaluate the extent of joint arthropathy, incidence of bleeds per calendar year, type of treatment P or OD, and quantity of factor utilized annually. Data was also collected on the utilization of analgesics medications, employment history, and psychiatric history. Results: The data was evaluable for 5 Hemophilia B pts, 4 severe, and 1 moderate; and 41 Hemophilia A pts, 25 severe, and 16 moderate. Forty-six percent (21/46) of the hem pts were receiving P as adults. Fifteen percent (7/46) hem pts had received P as a child or adolescent, and continued into adulthood. The pts ages ranged from 18 to 66 years of age. The mean age of pts receiving P was 32 years, while it was 34 years in the OD group. There was no difference in the average number of target joints between the two groups, with a mean of 2 target joints per patient. Fifty-two percent of hem on P (11/21) had 0–1 bleed per month, while only 40% of OD pts (12/25) had 0–1 bleed per month. In the P group 48% (10/21) had more than 1 bleed per month, while 60% in the OD group had more than 1 bleed per month. Overall there was no statically significant difference between the incidence of annual bleeds between the two groups, p=0.51. Forty-six percent of patients (21/46) were taking analgesic managements, and of these 48% (10/21) were on P, p=.806. Thirty-three percent (15/46) of the pts were unemployed or not enrolled as a student, and more than half (8/15) of those pts were on P treatment. Twenty-four percent (11/46) of hem pts were found to have a psychiatric history with either depression, or anxiety, of which 82% were on P. In 43 of the 46 hem pts the amount of factor utilized over a 5-year period (2006–2010) was available for analysis. Patients receiving P utilized more factor than pts on OD, 385,124 units versus 115,367 units, p =.0013. Conclusion: The initiation of primary prophylaxis in childhood has decreased much of the morbidity associated with hemophilia, however the benefit of secondary prophylaxis has not been clearly established. The treatment of hemophilia is costly, and there are both medical and economical advantages and disadvantages. Prophylaxis in this older population of moderate and severe hem pts did not have an impact on the reduction of annual bleeds, or the number of target joints, though pts on P utilized more units of clotting factor concentrate. Additionally, our pts did not have improvement in quality of life factors such as pain alleviation, increased employment, or mental well-being. Lack of benefit may be obscured as these pts who had not received primary prophylaxis, already had significant joint damage. Studies on continuation of P or OD in pts who have received primary prophylaxis will be necessary to determine long term prophylaxis in these pts. Disclosures: No relevant conflicts of interest to declare.

Effects of “Primary Prophylaxis” and “On-Demand” Therapy on the Clinical Expression of Severe Hemophilia A in Children - Results of a Multicenter Study.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 3146-3146
Author(s):  
Rosemarie Schobess ◽  
Karin Kurnik ◽  
Wolfhart Kreuz ◽  
Frauke Friedrichs ◽  
Anne Krumpel ◽  
...  
Keyword(s):  
Hemophilia A ◽  
Bleeding Episode ◽  
Joint Damage ◽  
Primary Prophylaxis ◽  
Secondary Prophylaxis ◽  
Outcome Variable ◽  
Severe Hemophilia ◽  
On Demand ◽  
Imaging Results ◽  
Bleeding Episodes

Abstract Background: Patients with severe hemophilia A (HA) can either be treated by regular FVIII infusions twice or thrice 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 hemophilic arthropathy, recommendations regarding age and dose at start of prophylactic regimens are still a matter of debate. The present cohort study was performed to investigate the role of “primary prophylaxis” versus “on-demand” therapy in HA children. The outcome variable was imaging-proven hemophilic joint damage. Methods: 42 children were initially treated with primary prophylaxis following the first bleeding episode, and were frequency-matched (year of birth, catchment area) to 67 patients receiving “on-demand” therapy with an early switch to “secondary prophylaxis”. Results: 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 no 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), or in children in which synovitis had occurred (p=0.77). Imaging results obtained showed a substantial agreement (87.14%) beyond that expected by chance alone (42.4%) between local and central readers in the patients tested (kappa=0.77; Z= 17.27; p < 0.001). Conclusion: In cases with severe HA where primary prophylaxis is impossible, the procedure to switch from “on-demand” to early secondary prophylaxis can be achieved in the majority of young children affected. In addition, the needs of parents around the time of diagnosis of severe HA could be better addressed.

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