Turoctocog alfa (trade name NovoEight) is a recombinant antihemophilic factor VIII used for the treatment of and prophylaxis of bleeding patients with haemophilia A. It is marketed by Novo Nordisk. It was approved in the United States, the European Union, and Japan in 2013. Medical uses
Turoctocog alfa is indicated for the treatment and prophylaxis of bleeding in adults and children with haemophilia A (congenital factor VIII deficiency).Turoctocog alfa pegol is indicated for the treatment and prophylaxis of bleeding in adults and children twelve years and above with haemophilia A (congenital factor VIII deficiency).In the safety and efficacy trial for prevention and treatment of bleeds, in hemophilia patients the success rate for treatment of bleeds was 84.5% (excluding bleeds for which there was no outcome reported) and out of a total of nine surgeries in nine patients performed during the trial, haemostasis was successful in all the surgeries and no treatment failures were reported. It is also used for perioperative management and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. Turoctocog alfa is not indicated for the treatment of von Willebrand disease. Benefits and risks
In a study conducted with 150 patients aged twelve and above, adolescents after using turoctocog alfa as a treatment, had an average of 5.55 bleedings per year while the adults had an average of 6.68 bleeding per year. According to data, turoctocog alfa was considered an excellent treatment for 403 out of 499 bleeding episodes.
Smoking does however affect the growth of a fetus, and low birth weight is shown to increase the likelihood of all the disorders encompassed by TDS. Maternal obesity, resulting in gestational diabetes, has also been shown to be a risk factor for impaired testes development and TDS symptoms in sons. Pathogenesis
The TDS hypothesis proposes that testicular dysgenesis, which has various primary causes, can lead to abnormalities in Sertoli and/or Leydig cell function. This leads to both impaired germ cell development and hormonal changes during male sexual differentiation. For instance, insufficient production of testosterone can result in incomplete masculinisation, whilst reduced expression of insulin-like factor 3 can lead to incomplete testes descent. The downstream disorders of such abnormalities can include both genital malformations (e.g. hypospadias and cryptorchidism) and delayed reproductive disorders (e.g. testicular cancer and poor semen quality) which comprise TDS. Diagnosis
Hypospadias
Hypospadias presents as an abnormal location for the end of the urethra which is typically found on the distal end of the penis. It is generally diagnosed at birth from visual confirmation of the hallmark features. As well as an unusual location of the urethra, the prepuce (foreskin) is typically incomplete as well. The abnormal ‘hooded’ prepuce is what often draws attention to the condition but can occur separately to hypospadias. Cryptorchidism
In Cryptorchidism a diagnosis is made from a physical examination which is performed when the baby is lacking one or both testes in the dependant portion of the scrotal sac.