Unlabeled uses of factor VIIa (recombinant) in pediatric patients

Hong, Irene S.; Stachnik, Joan M

doi:10.2146/ajhp090667

Cited by 13 publications

(7 citation statements)

References 85 publications

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“…rFVIIa is known to be effective in treating bleeding episodes in Glanzmann thrombasthenia. Based on the case reports and case series response to treatment varies between 65 and 95% [11][12][13]. It is more effective when used before an invasive procedure rather than used after the bleeding starts [5].…”

Section: Discussionmentioning

confidence: 99%

Weekly low-dose recombinant factor VIIa prophylaxis in Glanzmann thrombasthenia

Andıç

Oğuz

Gündüz

et al. 2021

Blood Coagulation &Amp; Fibrinolysis

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Glanzmann thrombasthenia is an inherited disease causing bleeding episodes due to platelet dysfunction. The standard treatment for moderate-severe bleeding is platelet transfusion. Recombinant factor VIIa (rFVIIa) is successfully used in bleeding episodes and invasive procedures. Here, we present a patient with Glanzmann thrombasthenia, whose bleeding episodes could only be controlled by rFVIIa. The patient is a 28 years old male, who has had frequent bleeding episodes unresponsive to local hemostatic agents and tranexamic acid and had an anaphylactoid reaction to platelet transfusion. We started the patient on a low-dose (20 μg/kg) rFVIIa once a week. The patient has no spontaneous bleeding since then. This is the first case report of a Glanzmann thrombasthenia patient on routine prophylaxis with low-dose rFVIIa.

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Section: Discussionmentioning

confidence: 99%

Weekly low-dose recombinant factor VIIa prophylaxis in Glanzmann thrombasthenia

Andıç

Oğuz

Gündüz

et al. 2021

Blood Coagulation &Amp; Fibrinolysis

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“…Reported decreased in the requirement of blood product transfusion after the use of factor VIIa (recombinant) versus standard therapies or placebo was commonly observed but was not statistically signifi cant in many cases. 47 Given the lack of well-designed controlled studies and cost-effectiveness of its use, the current evidence is inconclusive regarding the safety and effi cacy of factor VIIa (recombinant) for unlabeled indications in paediatric patients with severe trauma and during MTP.…”

Section: Use Of Recombinant Factor Viia In Severe Bleeding In Major T...mentioning

confidence: 99%

Physiologically-guided Balanced Resuscitation: An Evidence-based Approach for Acute Fluid Management in Paediatric Major Trauma

Kua

Ong

2014

Ann Acad Med Singap

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Trauma is a major cause of death, and haemorrhage represents an important target for improving outcomes after severe injury. Volume replacement with crystalloids in resuscitation might become harmful in large amounts because of coagulopathy. A fine balance must be achieved between haemodynamic and haemostatic resuscitation. Permissive hypotension refers to permitting some degree of hypotension in such adult patients in an attempt to attain this fine balance. For patients who require a significant volume of blood product resuscitation, the term ‘massive transfusion protocol’ (MTP) is used. There is very little data on transfusion protocols for paediatric trauma patients, and children respond to hypovolemic shock in a different physiological manner compared to adults. Hence, concepts such as permissive hypotension may not be appropriate when treating children involved in major trauma. We recently embarked on a plan to streamline the management of blood transfusion in massive bleeding during paediatric trauma, to reduce the logistical problems associated with the transport of blood products from the blood bank to the patient. From this, we evolved a MTP for paediatric major trauma. Nonetheless, further studies will be needed to see if there is indeed improved outcome after MTP in paediatric major trauma as current evidence is extrapolated from adult studies. Key words: Compensated versus decompensated shock, Damage control resuscitation, Massive transfusion protocol, Permissive tachycardia

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“…More than 60 case reports describing the use of rFVIIa in children have been published to date. 35 Since case reports showing a beneficial effect of a new therapy are more likely to be published than studies that do not show a benefit, the initial successes of rFVIIa in achieving hemostasis in children with a variety of conditions may be attributed to publication bias, and a higher level of evidence should be sought.…”

Section: Off-label Use Of Rfviiamentioning

confidence: 99%

Overview of the use of recombinant factor VIIa in children

Puetz¹

2014

PHMT

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Recombinant factor VIIa (rFVIIa) was initially developed as a bypassing agent to control bleeding in people with hemophilia who have inhibitors. Due to its potential as a universal hemostatic agent, rFVIIa has also been used for a variety of other clinical indications. Investigations into the use of rFVIIa in children have demonstrated clinical efficacy and excellent safety when used for children with hemophilia and inhibitors. Investigations into the use of rFVIIa in children for alternative indications have been hampered by the lack of high-quality evidence. Most studies have shown that rFVIIa can be effective in reducing bleeding and blood product usage when given off-label in children. However, thrombotic events have been reported. In addition, no mortality benefit has been shown for rFVIIa. Despite this, over the last decade, the use of rFVIIa for indications beyond hemophilia has increased dramatically.

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Unlabeled uses of factor VIIa (recombinant) in pediatric patients

Abstract: Given the lack of well-designed controlled studies, current evidence is inconclusive regarding the safety and efficacy of factor VIIa (recombinant) for unlabeled indications in pediatric patients.

Cited by 13 publications

References 85 publications

Weekly low-dose recombinant factor VIIa prophylaxis in Glanzmann thrombasthenia

Weekly low-dose recombinant factor VIIa prophylaxis in Glanzmann thrombasthenia

Physiologically-guided Balanced Resuscitation: An Evidence-based Approach for Acute Fluid Management in Paediatric Major Trauma

Overview of the use of recombinant factor VIIa in children

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