Approaches towards successful home treatment in patients with inhibitors

Abstract

Significant advances have been achieved in prevention of haemophilic disability through prophylactic administration of concentrates and early administration of coagulation factors to control new bleeding episodes, but there is only limited experience with home treatment in haemophilia patients with inhibitors. A home treatment programme using recombinant activated factor VII (rFVIIa; NovoSeven®, Novo Nordisk, Bagsværd, Denmark) in early intervention against minor bleeds in patients with high responding inhibitors was initiated in Denmark in June 1994. Following careful education and instruction, 2–3 doses each giving 90–100 μg/kg bodyweight of rFVIIa were stored in each patient's home. At the onset of a new bleeding episode patients were instructed to inject 1 dose of rFVIIa, and to call the Haemophilia Centre to discuss further management of the episode. If the drug was not completely effective after 1–2 h, a second dose was injected after 3 h. Patients were further instructed to contact us the following day for final efficacy reporting. In total, 7 patients have been enrolled into the study, and to date 114 bleeding episodes have been managed at home with a mean of 2.1 doses per bleed. On 4 occasions, recurrence of bleeding was noted within 24 h. Hospital admission was required in 9 cases, because of a serious injury, insufficient compliance or, in 2 cases, because bleeding required prolonged treatment. Management of these bleeding episodes required a mean of 18 doses. We propose and discuss key criteria for selection of patients for a home treatment programme. The results of this study demonstrate that early intervention in the home setting with rFVIIa is safe and effective in the management of minor bleeding episodes in haemophilia patients with inhibitors.

Home treatment with bypassing products in inhibitor patients: a 7.5‐year experience

“…However, the time period investigated in most studies are comparably short [22–26] and our study is the first to show that the safety using bypassing products at home is very good also over an extended time period such as 7.5 years.…”

“…To date, studies and reports on bypassing products in home treatment have primarily been focusing on the efficacy in a shorter time perspective and have not been evaluating the feasibility, efficacy and safety of the treatment for a long time period [20–26].…”

A review of evidence about behavioural and psychological aspects of chronic joint pain among people with haemophilia

“…Self‐management initiatives for haemophilia have focused mainly on self‐administration of clotting factor concentrates , which is vitally important because clotting factor treatment is the key to effective haemophilia management. Physical activity is also important, for physiotherapy was associated with reduced pain and improved functional ability , and exercise programmes designed specifically for people with bleeding disorders led to improvements in joint motion, strength and walking ability .…”

Efficacy of recombinant activated factor VII vs. activated prothrombin complex concentrate for patients suffering from haemophilia complicated with inhibitors: a Bayesian meta‐regression

“…Reason for exclusion Zwahlen and Beck [33] Efficacy not reported Sjamsoedin et al [31] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Macik et al [34] Not enough information on dosage, frequency or timing Lindley et al [35] Efficacy not reported Schmidt et al [36] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Lusher [37] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients McPherson et al [38] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Rice and Savidge [39] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients DiMichelle [40] No clinical study Hay et al [41] Not enough information on dosage, frequency or timing Arkin et al [42] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Ingerslev et al [43] Efficacy not reported Ingerslev et al [44] Not enough information on dosage, frequency or timing Lusher [45] No clinical study Lusher et al [46] No clinical study Mauser-Bunschoten et al [47] No bolus infusion Petrini and Klementz [48] No clinical study Shapiro et al [49] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Scharrer [50] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Brackmann et al [51] Efficacy not reported Liebman et al [52] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Lusher [53] No clinical study Negrier and Lienhart [54] No clinical study Berrettini et al [55] Efficacy not reported Friederich et al [56] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Hedner [57] No clinical study Santagostino et al [58] No bolus infusion Shirahata et al [27] Not enough information on dosage, frequency or timing Smith et al [59] No (separate) evaluation of non-surgical joint bleeds in inhibitor patients Levi…”