The Evolution Between 2001 and 2010 In the Uk Costs of Resolving a Mild to Moderate Bleeding Episode with Pd-aPCC Versus rFVIIa In Haemophilia A Patients with inhibitors.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 1518-1518
Author(s):  
Paresh Sewpaul ◽  
Donna Ashley ◽  
Paul Riley
Keyword(s):  
Bleeding Episode ◽  
Haemophilia A ◽  
Line Treatment ◽  
First Line ◽  
Treatment Regimens ◽  
Bypassing Agents ◽  
Third Line ◽  
The Uk ◽  
Third Line Treatment ◽  
First Line Treatment

Abstract Abstract 1518 Introduction: Haemophilia A is a rare inherited bleeding disorder characterised by spontaneous or trauma-related bleeding, most commonly into the joints, leading to pain, swelling and limited movement. Some 30–50% of patients with haemophilia A develop inhibitors to their standard treatment, comprised of FVIII concentrates. There are two bypassing agents used for the management of bleeding episodes in patients with haemophilia A with inhibitors in the UK: recombinant factor VII (rFVIIa, NovoSeven®) and plasma-derived activated prothrombin complex concentrate (pd-aPCC, Feiba®). Objective: A systematic review of the cost-effectiveness of pd-aPCC versus rFVIIa was published in 2003. Since the analysis, costs of bypassing agents in the UK have changed significantly (pd-aPCC +30% from ≤0.57/U to ≤0.74/U; rFVIIa -11% from ≤0.51/mcg to ≤0.46/mcg). The present study examines the evolution in costs of resolving a mild to moderate bleeding episode with treatment initiated outside hospital. Methods: A decision analytic model estimated the expected costs and outcomes of resolving a mild-moderate bleeding episode in patients with haemophilia A with inhibitors. The model compared three treatment regimens in patients with a mean weight 70kg. Each regimen comprised first-, second- and third-line treatment: (1) pd-aPCC-pd-aPCC-rFVIIa; (2) pd-aPCC-rFVIIa-rFVIIa; and (3) rFVIIa-rFVIIa-rFVIIa. Efficacy and dosing of pd-aPCC and rFVIIa were estimated from the published literature, with rFVIIa assumed to resolve 92% of mild-moderate bleeds when used as first and second line treatment and pd-aPCC resolving 79% and 88% of mild-moderate bleeds when used first and second line respectively. Patients whose bleeding episodes were not resolved with first-line treatment were admitted to hospital for second- and third-line treatment. Costs included in the economic model were bypassing agent costs and hospitalisation costs. Results: The different treatment regimens result in different total costs for the resolution of a single mild-moderate bleeding episode with up to three lines of treatment: (1) GBP 13,542 (EUR 15,180); (2) GBP 12,985 (EUR 14,556); and (3) GBP 8,569 (EUR 9,605). Since 2001, the costs of regimens 1 and 2 have increased by 26% and 18%, but regimen 3 has decreased by 11% in the UK. Conclusion: Divergence in the price of pd-aPCC and rFVIIa since 2001 has increased the cost-effectiveness of rFVIIa. The results reinforce the fact that effective first-line treatment with rFVIIa results in lower overall treatment costs due to a reduced need for further treatment and associated hospitalisation costs. Adopting rFVIIa as first-line treatment is cost-saving and more effective compared to reserving rFVIIa as a second- or third-line treatment option. Disclosures: Sewpaul: Novo Nordisk : Employment. Ashley:Novo Nordisk : Employment. Riley:Novo Nordisk Ltd: Employment.

scholarly journals Disease Management and Resource Use for the Management of Melanoma stage IIIc or IV Positive for BRAF V600 Mutations in Greece

2021 ◽  
Vol 2 (1) ◽  
Author(s):  
Carayanni V ◽  
Gogas H ◽  
Bafaloukos D ◽  
Boukovinas I ◽  
Latsou D ◽  
...  
Keyword(s):  
Resource Use ◽  
Line Treatment ◽  
Second Line ◽  
First Line ◽  
Cost Of Treatment ◽  
Management Scenario ◽  
Third Line ◽  
The Cost ◽  
Third Line Treatment ◽  
First Line Treatment

Objective: Melanoma is one of the most aggressive cancers and is responsible for the majority of skin cancer deaths, with the presence of metastases prognostic for poor survival. At a time when most cancer incidences are falling, the annual incidence of melanoma has risen as rapidly as 4-6% in many European countries, with a substantial economic burden in advanced stages. The objective of this study is the investigation of treatment pathways and healthcare resource use related to advanced BRAF-mutated melanoma in Greece. Methods: This study is based on the information collected by an expert panel comprising of 3 oncologists of major public and private melanoma clinics around Greece. A 3-round survey was undertaken, according to a modified Delphi method. The treatment phases studied were: pre-progression; disease progression and terminal care. Oncology drug costs, medical visits, laboratory tests, imaging examinations, hospitalization and concomitant medications were the resources considered in the context of the Greek National Services Organization (EOPYY). Results: Τhe most common management scenario (80% of cases) in Greece for patients of stage IV BRAF V600 mutated melanoma was: targeted therapies as first line treatment at 95%, followed by immunotherapies at 100% as second line as well as third line treatment at 65% of cases. The weighted annual cost of treatment was 89.215,78 €, (90%CI:62,451.05; 115,980.51) for first line treatment at list price and around 41.584,50 (90%CI:29,109.15; 54,059.85) based on the negotiated price. At second line, the cost of treatment has been estimated between 15,704.272 (90%CI:10,992.990; 20,415.553) and 19,800.92€, (90%CI: 16,489; 30,622) for the two most common management scenarios for immunotherapies. For third line treatment the cost was 37,778.93 (90%CI 26,445.25; 49,112.61€) for the mostly used management scenario (50% ipilimumab). Conclusions: Μetastatic BRAF mutant melanoma requires prolonged and costly treatment with new therapies shown to substantially increase life expectancy. Identifying the appropriate treatment options in order to optimize health outcomes should be an important priority in healthcare system.

The Cost Effectiveness of a New Formulation of Recombinant Factor VIIa in the Home Treatment of Mild to Moderate Bleeding Episodes in Haemophilia a Patients with Inhibitors in the United Kingdom.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 1296-1296
Author(s):  
Vicki Sunro
Keyword(s):  
Room Temperature ◽  
Bleeding Episode ◽  
Factor Viia ◽  
Cost Effective ◽  
Home Treatment ◽  
Haemophilia A ◽  
Line Treatment ◽  
Bleeding Episodes ◽  
Third Line ◽  
Third Line Treatment

Abstract The introduction of room temperature stable recombinant Factor VIIa (rFVIIa) (eptacog alfa, activated, [NovoSeven®]) in the United Kingdom (UK) provides a step forward in care for haemophilia A patients with inhibitors through enabling greater patient independence and freedom. It allows patients to treat bleeds earlier which has been shown to be associated with a greater rate of success and fewer injections being required1. Delayed treatment resulting in prolonged bleeding may result in longer hospitalization, and in some patients, a subsequent need for surgical procedures which results in an increase in direct medical costs. The objective of this analysis was to assess whether the benefits of room temperature stable rFVIIa would offset the small price premium associated with the new formulation compared to the original formulation as well as remain cost-effective compared to the other bypassing agent, activated prothrombin complex concentrate (aPCC). The study used a decision analytic approach, similar to a published economic modeling study2, to model the expected costs and outcomes of room temperature stable rFVIIa, original rFVIIa and aPCC in the home treatment of a mild to moderate bleeding episode in haemophilia A patients with inhibitors. The mean weight of patients was assumed to be 70kg. Each treatment regimen comprised first-, second- and third- line treatment: original rFVIIa - original rFVIIa - original rFVIIa ; room temperature stable rFVIIa - room temperature stable rFVIIa – room temperature stable rFVIIa; and aPCC – aPCC – room temperature stable rFVIIa. The efficacy and dosing of aPCC and original rFVIIa were obtained from the published literature. The efficacy of room temperature stable rFVIIa was assumed to be slightly higher than that for original rFVIIa and the dose of room temperature stable rFVIIa was assumed to be slightly less than that for original rFVIIa. Patients whose mild to moderate bleeding episodes were not resolved with first-line home treatment were admitted to hospital for second- and third-line treatment. All bleeding episodes resolved with third-line treatment. The outcome used in the analysis was resolved mild to moderate bleeding episode. Only the costs of the haemostatic agents were included in the base-case analysis. The different treatment regimens for the treatment of a mild to moderate bleeding episode of haemophilia A patients with inhibitors in the UK resulted in different costs attributable to resolving a mild to moderate bleeding episode: £9,644 (EUR 12,151); £9,458 (EUR 11,917); and £14,097 (EUR 17,762). Sensitivity analysis showed the results to be robust to changes in key parameters. The results show that both original rFVIIa and room temperature stable rFVIIa are cost-effective compared to aPCC, in the home treatment of a mild to moderate bleeding episode, in haemophilia A patients with inhibitors. In addition, the analysis highlights the fact that price alone does not determine overall treatment costs, and that even small improvements in efficacy and dosing associated with earlier treatment may reduce overall treatment costs.

scholarly journals Two Cases of Long-Term Control of Metastatic Colorectal Cancer via FTD/TPI plus Bevacizumab in Elderly Patients

2018 ◽  
Vol 11 (3) ◽  
pp. 800-805 ◽  
Author(s):  
Masatsune Shibutani ◽  
Kiyoshi Maeda ◽  
Hisashi Nagahara ◽  
Tatsunari Fukuoka ◽  
Yasuhito Iseki ◽  
...  
Keyword(s):  
Adverse Events ◽  
Elderly Patients ◽  
Intensive Chemotherapy ◽  
Line Treatment ◽  
First Line ◽  
Severe Adverse Events ◽  
Third Line ◽  
Third Line Treatment ◽  
First Line Treatment

With advances in new cytotoxic drugs and molecular-targeted drugs, the prognosis of patients with metastatic colorectal cancer (mCRC) has improved. However, physicians often hesitate to administer intensive standard regimens to elderly patients with mCRC. Recently, first-line regimens that are effective in and well-tolerated by patients who are not eligible for intensive chemotherapy have been established. However, the therapeutic strategies to adopt after the failure of first-line treatment for patients who are not eligible for intensive chemotherapy remain unclear. We herein report two cases of long-term control of mCRC via FTD/TPI+bevacizumab (Bmab) as second- or third-line treatment in elderly patients without severe adverse events. In case 1, first-line treatment with Tegafur-Uracil, which is a prodrug of 5-FU, caused disease progression in a short period after the initiation of chemotherapy. In case 2, intensive first-line treatment caused severe adverse events, and treatment was discontinued. However, in both cases, disease control was obtained for a long time without severe adverse events by subsequent treatment with FTD/TPI+Bmab. The success in these present cases indicates that FTD/TPI+Bmab as a second- or third-line treatment is a therapeutic option for elderly patients with mCRC who are not eligible for intensive chemotherapy, even after failure of treatment with 5-FU.

A low dose rituximab regimen for first‐line treatment of acquired haemophilia A

2021 ◽  
Author(s):  
Stewart Hunt ◽  
Jeremy Robertson ◽  
Jason Conn ◽  
John Casey ◽  
Jane Royle ◽  
...  
Keyword(s):  
Low Dose ◽  
Haemophilia A ◽  
Line Treatment ◽  
First Line ◽  
Acquired Haemophilia ◽  
First Line Treatment

Efficacy of Coenzyme Q10 in the Treatment of Cyclic Vomiting Syndrome in Children

2021 ◽  
Vol 6 (1) ◽  
Author(s):  
Brezin F ◽  
◽  
Wiedemann A ◽  
Bansept C ◽  
Albuisson E ◽  
...  
Keyword(s):  
Coenzyme Q10 ◽  
Gastrointestinal Disorder ◽  
Cyclic Vomiting Syndrome ◽  
Line Treatment ◽  
First Line ◽  
Excellent Safety Profile ◽  
Third Line ◽  
One Year ◽  
First Line Treatment ◽  
Cyclic Vomiting

Cyclic Vomiting Syndrome (CVS) is a chronic functional gastrointestinal disorder related to migraine, characterized by episodic nausea and vomiting. The treatment of CVS remains based on tricyclic antidepressants, triptans and antiepileptics. As mitochondriopathy has been involved in the pathophysiology of CVS, Coenzyme Q10 (CoQ10), a mitochondrial cofactor, has been used as the third line treatment in CVS. Considering the excellent safety profile of CoQ10, we decided to use it as the first line treatment in CVS. We retrospectively studied the evolution of 23 CVS patients who were treated for one year by CoQ10 alone. We recorded the characteristics of patients and their CVS history and compared data obtained the year before and the year following the prescription of CoQ10 treatment. We found a significant decrease in the number of vomiting episodes between the year before and the year after the start of CoQ10 (median [IQR]: 18.0 [15.75] vs. 3.00 [5.0]; p <0.001). This decrease persisted with time (2 and 3 years of treatment). The treatment was very efficient in 17/23 patients and did not decrease the number of vomiting episodes in 3 patients. Only one mild side effect related to the drug has been reported. Conclusions: CoQ10 is an efficient and safe treatment of CVS and should be used as the first line treatment in this episodic syndrome related to migraine.

A nationwide population-based study comparing survival in unresectable advanced or synchronous metastatic esophageal and gastric adenocarcinoma.

2020 ◽  
Vol 38 (4_suppl) ◽  
pp. 308-308
Author(s):  
Marieke Pape ◽  
Pauline A.J. Vissers ◽  
David Bertwistle ◽  
Laura McDonald ◽  
Hanneke W.M. Van Laarhoven ◽  
...  
Keyword(s):  
Systemic Treatment ◽  
Rank Test ◽  
Tumor Characteristics ◽  
Line Treatment ◽  
First Line ◽  
Treatment Regimens ◽  
Netherlands Cancer Registry ◽  
Number Of Patients ◽  
Significant Difference ◽  
First Line Treatment

308 Background: Similarities between esophageal and gastric adenocarcinomas have been identified in terms of genomic characteristics. There is however no consensus on the combined or stratified inclusion of esophageal adenocarcinoma (EAC) within gastric cancer (GC) clinical trials. The aim of our study was to compare patient and tumor characteristics, first-line treatment regimens and overall survival (OS) of patients with EAC and GC. Methods: We selected patients with unresectable advanced and/or synchronous metastatic EAC (n = 1554) or GC (including junction tumors; n = 2095) diagnosed in the period 2015-2017 from the nationwide Netherlands Cancer Registry. Patients with a positive HER2 test result and/or receiving trastuzumab as a first-line treatment were excluded. Data on OS were analyzed using Kaplan-Meier curves with Log-Rank test. Results: The EAC patient population had significantly more male patients (83% vs 66%), lower median age (68 vs 71 years) and higher median BMI (25.4 vs 24.5). Significant differences in location of metastases were identified, with higher percentages in non-regional lymph nodes (48% vs 28%), liver (50% vs 35%), lung (21% vs 9%) and bone (19% vs 7%) and lower in peritoneum (5% vs 42%), in EAC versus GC patients respectively. EAC patients more often received any type (supportive or active systemic) of treatment (76% vs 60%). Median OS was longer in EAC than GC patients (EAC: 4.8 vs GC: 4.1 months; p < 0.01). The percentages of patients receiving first-line systemic treatment were equal in both groups (43%). The number of patients receiving CapOx or FOLFOX was not significantly different (43% vs 47%). Carboplatin+paclitaxel was more frequently given in EAC versus GC (34% vs 3%), while EOX or ECC was given less frequently (12% vs 30%). No significant difference was observed in median OS between EAC and GC patients receiving first-line active systemic treatment (8.0 vs 7.6 months; p = 0.28). Conclusions: Patient characteristics, tumor characteristics, treatment regimens and OS differ between EAC and GC patients. Despite these differences, in patients receiving first-line active systemic treatment no significant differences in OS were found.

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