Successful self‐infusion of activated prothrombin complex concentrate for prophylaxis in a child with a factor VIII inhibitor

Ohga, Shouichi; Nomura, Akihiko; Takada, Hidetoshi; Suga, Naohiro; Hara, Toshiro

doi:10.1002/ajh.20751

Cited by 11 publications

(9 citation statements)

References 13 publications

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“…Joint bleeds were generally associated with small traumas, particularly during sports and physical activities. Most previous reports on FEIBA prophylaxis have also indicated a dramatic reduction in bleeds [18, 20–23, 25, 26]. By contrast, in the study of seven patients by Hilgartner et al.…”

Section: Discussionmentioning

confidence: 92%

“…An increasing number of case or case series reports on FEIBA prophylaxis have appeared in recent years [15][16][17][18][19][20][21][22][23][24][25][26]. In a questionnaire study involving 72 haemophilia treatment centres in Europe and the United States, prophylactic treatment with FEIBA resulted in improved or stabilized clinical orthopaedic status in 11 of 13 patients (85%) [27].…”

Section: Introductionmentioning

confidence: 99%

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Early long‐term FEIBA prophylaxis in haemophilia A patients with inhibitor after failing immune tolerance induction: A prospective clinical case series

Ettingshausen¹,

Kreuz²

2009

Haemophilia

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Persistent high-titre inhibitors after immune tolerance induction (ITI) increase the risks of haemorrhage and arthropathy, resulting in high morbidity and mortality. Long-term prophylaxis with bypassing agents may avert these risks. This study was performed to assess the effectiveness and safety of early prophylaxis with FEIBA in preventing bleeding and joint damage after failed ITI. Seven paediatric patients proceeded immediately after failed ITI to long-term FEIBA prophylaxis at 60-100 IU kg(-1) dosages and various dosing intervals depending upon bleeding tendency. Bleeding episodes and joint status were assessed. Thrombin generation assays (TGA) were also used to gauge treatment response. FEIBA prophylaxis was commenced at a median age of 6.0 years (range, 1.5-11.8 years) and continued for a median duration of 6.9 years (range, 0.8-17.1 years). The mean annual incidence of joint bleeding was 1.5 episodes per year with a 95% CI of 0.7-3.0 episodes per year. Muscle bleeding incidence was 0.9 episodes per year (CI, 0.6-1.2 episodes per year). No patient experienced major joint damage during prophylaxis. Median Pettersson and orthopaedic joint scores at the last follow-up evaluation were 4 (range, 0-12) and 2 (range, 0-4) respectively. Endogenous thrombin potential (ETP) measured by TGA exceeded 80% of normal after FEIBA infusion in the majority of the patients. Between regular prophylactic infusions, mean trough ETP equalled 2.6 fold of the inhibitor plasma control mean. FEIBA prophylaxis was well-tolerated without serious thrombotic or other complications. The only adverse event involved venous access. Therefore early long-term FEIBA prophylaxis is valuable in controlling bleeding and preserving joint integrity in young patients failing ITI.

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Section: Discussionmentioning

confidence: 92%

Section: Introductionmentioning

confidence: 99%

Early long‐term FEIBA prophylaxis in haemophilia A patients with inhibitor after failing immune tolerance induction: A prospective clinical case series

Ettingshausen¹,

Kreuz²

2009

Haemophilia

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“…The patient had a high‐titre FVIII inhibitor, and the bleedings were successfully controlled with self‐infusion of FEIBA. The detailed course was reported previously [2]. The diagnosis of haemophilia A was based on a neonatal hepatic bleeding and <1% of FVIII activity.…”

Section: Major Bleedings and Anamnestic Responses Triggered By High‐mentioning

confidence: 99%

Complete immunotolerance induction after FEIBA prophylaxis in a haemophilia A patient with high‐titre inhibitor

et al. 2012

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The standard goal in the current management of haemophilia children is the early establishment of prophylaxis using recombinant factor VIII or IX (FVIII/IX) agents. The development of inhibitors neutralizing the clotting factor activity is the most challenging complication associated with the treatment. Immune tolerance induction (ITI) is usually applicable for haemophilia patients having low, but not extremely high-titre of FVIII or FIX inhibitor [1]. Bypass therapy using recombinant activated FVII (rFVIIa) (NovosevenÒ) or activated prothrombin complex concentrate (aPCC) (anti-inhibitor coagulant complex, FEIBA VHÒ) is the only measure to control bleeding in patients with high-titre inhibitors. Recent retrospective studies indicated the safety and efficacy of aPCC or rFVIIa in decreasing the frequency of bleeding episodes in patients with high-titre inhibitors. However, anamnestic response is one of the major obstacles for the introduction of ITI, because it often occurs even after aPCC infusion in patients with haemophilia A or B and each high responding inhibitor. rFVIIa agent does not precipitate anamnesis, although the shorter biological half-life confronts a dilemma for the effective prophylaxis. Thus, the ultimate goal, Ôthe eradication of high responding inhibitor in hemophilia childrenÕ is beyond our scope of practice. We herein report a paediatric case of haemophilia A with high-titre inhibitor who succeeded in ITI until age 20 years following 4 yearsÕ FEIBA prophylaxis.A 19-year-old Japanese boy with haemophilia A was hospitalized because of the left knee joint pain. The patient had a high-titre FVIII inhibitor, and the bleedings were successfully controlled with selfinfusion of FEIBA. The detailed course was reported previously [2]. The diagnosis of haemophilia A was based on a neonatal hepatic bleeding and <1% of FVIII activity. For 7 yearsÕ on-demand therapy with plasma-derived FVIII concentrates (Hemofil-MÒ/Crosseight-MÒ), intracranial bleedings adjacent to a subarachnoid cyst repeated at age 1 and 2 year(s). Thereafter, he showed 127 BU/mL of FVIII inhibitor (Table 1). Despite the on-demand therapy with Proplex-STÒ, he frequently bled into the target joints. At age 10 years, 1 BU/mL of the inhibitor level elevated to 989 BU/mL after high-dose Crosseight-M therapy for the third intracranial haemorrhage. During the consequent regular infusions of Proplex (50 U/kg/day, tri-weekly), joint and mucosal bleedings recurred. An accidental quadriceps bleeding and shock at 14 years of age prompted FEIBA prophylaxis (50 U/kg/day, tri-weekly), that led to a successful school life followed by a professional life of care-worker in hospital. He was on dietary therapy for type 2 diabetes mellitus since age 15 years. No adverse events occurred during the self-FEIBA infusion without the use of central venous access devices.At age 19 years, he visited our emergency room because of the left knee injury resulting from an accidental fall from stairs on collision with passengers at the station. On admission, he complaine...

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“…Effects on bleeding frequency were largely maintained throughout a 3-month post-prophylaxis evaluation period. In many reports from case series, pd-aPCC and rFVIIa appeared to be effective and well tolerated as prophylactic therapy, with a reduced incidence of bleeding episodes, fewer workdays missed, improved quality of life, and less need for on-demand therapy, all variously reported 36–42. However, concerns have been raised about the risks (particularly of thromboembolism) with repeated and frequent use of pd-aPCC and the potential for anamnestic increases in antibody titers 43.…”

Section: Bypassing Therapy For Patients With Inhibitorsmentioning

confidence: 99%

Current difficulties and recent advances in bypass therapy for the management of hemophilia with inhibitors: a new and practical formulation of recombinant factor VIIa

Butros¹,

Boayue²,

Mathew³

2011

DDDT

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Bypassing agents are the mainstay of treatment for patients with hemophilia with high-titer inhibitors. Whereas the availability of these agents has greatly advanced the management of bleeding episodes in this population, timely administration of bypassing agents continues to be hampered by a number of practical limitations, including the need for refrigerated storage of the agent and its reconstitution at room temperature prior to administration, among others. In this review, the importance of early treatment of bleeds and factors that influence this more timely therapeutic approach are highlighted, together with the advantages offered by the use of a new formulation of recombinant activated factor VII that permits improved storage and portability, potentially optimizing timely bypassing agent administration.

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Successful self‐infusion of activated prothrombin complex concentrate for prophylaxis in a child with a factor VIII inhibitor

Cited by 11 publications

References 13 publications

Early long‐term FEIBA prophylaxis in haemophilia A patients with inhibitor after failing immune tolerance induction: A prospective clinical case series

Early long‐term FEIBA prophylaxis in haemophilia A patients with inhibitor after failing immune tolerance induction: A prospective clinical case series

Complete immunotolerance induction after FEIBA prophylaxis in a haemophilia A patient with high‐titre inhibitor

Current difficulties and recent advances in bypass therapy for the management of hemophilia with inhibitors: a new and practical formulation of recombinant factor VIIa

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