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Factor IX therapy seems safe, effective in young kids

Antihemophilic factor

SCOTTSDALE, ARIZONA—The recombinant factor IX product trenonacog alfa appears safe and effective for previously treated patients with hemophilia B who are younger than 12 years of age, according to researchers.

The team conducted a pooled analysis of 2 studies, which included a total of 12 patients.

The median annualized bleeding rate (ABR) was low among patients who received trenonacog alfa as prophylaxis.

Among all patients, 72% of bleeding episodes were resolved with a single infusion of trenonacog alfa.

None of the patients developed factor IX inhibitors, and treatment-related adverse events consisted of fever and hyperhidrosis.

These results were presented in a poster at the Hemostasis and Thrombosis Research Society 2017 Scientific Symposium.

The research was conducted by employees of Aptevo Therapeutics, the company marketing trenonacog alfa as IXINITY®. The poster is available on the company’s website.

The researchers conducted a pooled analysis of 2 prospective, non-randomized studies of 12 children with hemophilia B under the age of 12.

The patients’ median age was 9.5 (range, 2-11). All patients were male. Half were Asian, 3 were white, 2 were Pacific Islanders, and 1 belonged to an “other” racial/ethnic group.

Eleven patients received trenonacog alfa as prophylaxis, and 1 patient received the treatment on demand.

Among the patients on prophylaxis, the median number of exposure days was 254 (range, 111-404), and the median dose per infusion was 75.3 IU/kg (range, 25.3-111.0).

For the patients on prophylaxis, the median number of bleeding episodes was 1.0 (range, 0-11), and the median ABR was 0.3 (range, 0-4.0).  Two patients had no bleeding episodes.

The patient who received trenonacog alfa on demand had 23 bleeding episodes and an ABR of 11.1.

There were a total of 61 bleeding episodes in this study. Most (72%, n=44) resolved after 1 infusion of trenonacog alfa, and 10% (n=6) resolved without any infusions.

Eight percent of the bleeding episodes (n=5) required 2 infusions of trenonacog alfa, and 10% (n=6) required 3, 4, or 5 infusions.

Adverse events thought to be related to trenonacog alfa were hyperhidrosis and fever in 1 patient, and hyperhidrosis in another patient.

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Antihemophilic factor

SCOTTSDALE, ARIZONA—The recombinant factor IX product trenonacog alfa appears safe and effective for previously treated patients with hemophilia B who are younger than 12 years of age, according to researchers.

The team conducted a pooled analysis of 2 studies, which included a total of 12 patients.

The median annualized bleeding rate (ABR) was low among patients who received trenonacog alfa as prophylaxis.

Among all patients, 72% of bleeding episodes were resolved with a single infusion of trenonacog alfa.

None of the patients developed factor IX inhibitors, and treatment-related adverse events consisted of fever and hyperhidrosis.

These results were presented in a poster at the Hemostasis and Thrombosis Research Society 2017 Scientific Symposium.

The research was conducted by employees of Aptevo Therapeutics, the company marketing trenonacog alfa as IXINITY®. The poster is available on the company’s website.

The researchers conducted a pooled analysis of 2 prospective, non-randomized studies of 12 children with hemophilia B under the age of 12.

The patients’ median age was 9.5 (range, 2-11). All patients were male. Half were Asian, 3 were white, 2 were Pacific Islanders, and 1 belonged to an “other” racial/ethnic group.

Eleven patients received trenonacog alfa as prophylaxis, and 1 patient received the treatment on demand.

Among the patients on prophylaxis, the median number of exposure days was 254 (range, 111-404), and the median dose per infusion was 75.3 IU/kg (range, 25.3-111.0).

For the patients on prophylaxis, the median number of bleeding episodes was 1.0 (range, 0-11), and the median ABR was 0.3 (range, 0-4.0).  Two patients had no bleeding episodes.

The patient who received trenonacog alfa on demand had 23 bleeding episodes and an ABR of 11.1.

There were a total of 61 bleeding episodes in this study. Most (72%, n=44) resolved after 1 infusion of trenonacog alfa, and 10% (n=6) resolved without any infusions.

Eight percent of the bleeding episodes (n=5) required 2 infusions of trenonacog alfa, and 10% (n=6) required 3, 4, or 5 infusions.

Adverse events thought to be related to trenonacog alfa were hyperhidrosis and fever in 1 patient, and hyperhidrosis in another patient.

Antihemophilic factor

SCOTTSDALE, ARIZONA—The recombinant factor IX product trenonacog alfa appears safe and effective for previously treated patients with hemophilia B who are younger than 12 years of age, according to researchers.

The team conducted a pooled analysis of 2 studies, which included a total of 12 patients.

The median annualized bleeding rate (ABR) was low among patients who received trenonacog alfa as prophylaxis.

Among all patients, 72% of bleeding episodes were resolved with a single infusion of trenonacog alfa.

None of the patients developed factor IX inhibitors, and treatment-related adverse events consisted of fever and hyperhidrosis.

These results were presented in a poster at the Hemostasis and Thrombosis Research Society 2017 Scientific Symposium.

The research was conducted by employees of Aptevo Therapeutics, the company marketing trenonacog alfa as IXINITY®. The poster is available on the company’s website.

The researchers conducted a pooled analysis of 2 prospective, non-randomized studies of 12 children with hemophilia B under the age of 12.

The patients’ median age was 9.5 (range, 2-11). All patients were male. Half were Asian, 3 were white, 2 were Pacific Islanders, and 1 belonged to an “other” racial/ethnic group.

Eleven patients received trenonacog alfa as prophylaxis, and 1 patient received the treatment on demand.

Among the patients on prophylaxis, the median number of exposure days was 254 (range, 111-404), and the median dose per infusion was 75.3 IU/kg (range, 25.3-111.0).

For the patients on prophylaxis, the median number of bleeding episodes was 1.0 (range, 0-11), and the median ABR was 0.3 (range, 0-4.0).  Two patients had no bleeding episodes.

The patient who received trenonacog alfa on demand had 23 bleeding episodes and an ABR of 11.1.

There were a total of 61 bleeding episodes in this study. Most (72%, n=44) resolved after 1 infusion of trenonacog alfa, and 10% (n=6) resolved without any infusions.

Eight percent of the bleeding episodes (n=5) required 2 infusions of trenonacog alfa, and 10% (n=6) required 3, 4, or 5 infusions.

Adverse events thought to be related to trenonacog alfa were hyperhidrosis and fever in 1 patient, and hyperhidrosis in another patient.

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