The value of early treatment in patients with haemophilia and inhibitors

Abstract

Development of inhibitors to infused factor concentrates represents a major clinical and economic challenge in the treatment of haemophilic patients. It has been shown that a delay in initiation of treatment leads to requirement of a larger number of injections to stop the bleeding but this has never been formally linked to costs associated with the bleeding. The objectives of this study were to assess the relationship between time to initiation of NovoSeven and total costs, number of doses administered and time to bleeding resolution in mild to moderate bleeding episodes. Data on time to treatment initiation, time to bleeding resolution and on all resource use related to the bleeding were extracted from medical records in Turkey for 129 bleeding episodes. Regression analysis was used to assess the impact of time to treatment on outcomes. Longer time to treatment initiation increased both total costs associated with the bleeding, the number of doses needed and the time to bleeding resolution. Treatment in hospital was associated with significantly longer time to treatment, higher costs and longer time to bleeding resolution as compared with home treatment or outpatient treatment. When controlling for other bleeding characteristics, the cost of bleedings treated in hospital was more than 150% higher. This study shows that treatment with NovoSeven should be initiated as soon as possible after the onset of bleeding in order to minimize costs and optimize outcomes. Home treatment reduces time to treatment initiation and also reduces costs related to the bleeding.

PERSEPT 1: a phase 3 trial of activated eptacog beta for on‐demand treatment of haemophilia inhibitor‐related bleeding

“…An axiom of haemophilic therapy is that the time spent bleeding impacts short‐ and long‐term outcomes. Convincing studies have demonstrated that delay of treatment initiation during an active bleeding episode impacts the achievement of haemostasis . In addition, multiple lines of evidence suggest that total haeme (iron) accumulation within a haemophilic joint affects the temporal progression of haemophilic arthropathy and may be difficult to detect .…”

“…Assumptions included a true proportion of success of 0.70, a correlation among bleeding episodes for a given subject of 0.1, and 8 mild/moderate bleeding episodes per treatment regimen per subject. The true success proportion was defined as 15% greater than a 12‐hour objective performance criterion (OPC) of 0.55, derived from the success rates used to support eptacog alfa registration or reported in the inhibitor literature between 1998 and 2013 …”

Recombinant Activated Factor VII: Mechanisms of Action and Current Indications

“…A recent study of 129 bleeding episodes showed that treatment with rFVIIa should be initiated as soon as possible after the onset of bleeding to reduce the total costs of a bleed (p ¼ 0.005) and optimize outcomes. 58 Pharmacoeconomic analyses conducted from a health-care payer perspective (using literature published between 1980 and November 2007) predicted that the use of rFVIIa is cost saving or cost neutral versus pdaPCC for controlling mild to moderate bleeds in inhibitor patients. 59 Furthermore, maintenance of hemostasis with rFVIIa in inhibitor patients undergoing orthopedic surgery is generally cost saving over the medium to long term compared with not having surgery.…”

Hemophilia Care in India: A Review and Experience from a Tertiary Care Centre in Uttar Pradesh

“…They found that rFVIIa if initiated as soon as possible after the onset of bleeding could minimize costs and optimize outcomes [36]. Elective orthopedic surgical procedures have also been made possible for PWH with inhibitors [37].…”