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Bevacizumab no better than placebo for epistaxis in HHT

A hand spraying a mist

of nasal spray

Bevacizumab nasal spray is no more effective than a placebo for reducing epistaxis caused by hereditary hemorrhagic telangiectasia (HHT), according to a study published in JAMA.

Researchers compared 3 different doses of bevacizumab to a sodium chloride placebo and found no significant difference between the treatment groups in the frequency or duration of epistaxis, the need for transfusion, or hemoglobin/ferritin levels.

Sophie Dupuis-Girod, MD, PhD, of the Hopital Femme-Mere-Enfants in Bron, France, and her colleagues conducted this trial, enrolling 80 patients with HHT and a history of epistaxis.

The patients were randomized to receive placebo (0.9% sodium chloride, n=21) or 1 of 3 doses of bevacizumab nasal spray.

Patients in the bevacizumab groups received 25 mg (n=20), 50 mg (n=20), or 75 mg (n=19) in 3 doses, 14 days apart, for a total treatment duration of 4 weeks, resulting in a total dose of 75 mg, 150 mg, or 225 mg.

Epistaxis duration

The researchers measured the mean monthly epistaxis duration at 3 months and found no significant difference between the placebo group and the bevacizumab groups (P=0.57). Likewise, there were no significant differences between the bevacizumab groups.

However, most of the groups experienced improvements in epistaxis duration after treatment.

The mean monthly epistaxis duration (in minutes) before treatment (from day −90 to day −1) and after treatment (from day 29 to day 118), respectively, was:

  • 262.8 and 200.4 in the placebo group
  • 285.5 and 259.2 in the 25 mg group
  • 229.0 and 244.0 in the 50 mg group
  • 272.9 and 215.0 in the 75 mg group.

Epistaxis frequency

There was no significant difference between the bevacizumab groups and the placebo group when it came to the number of epistaxis episodes (P=0.55). However, patients in all of the groups saw a reduction in episodes after treatment.

The mean number of epistaxis episodes before treatment (from day −90 to day −1) and after (from day 29 to day 118), respectively, were:

  • 31.37 and 24.27 in the placebo group
  • 30.99 and 23.13 in the 25 mg bevacizumab group
  • 25.06 and 20.01 in the 50 mg bevacizumab group
  • 27.84 and 24.35 in the 75 mg bevacizumab group.

Other outcomes

There was no significant difference between the bevacizumab groups and the placebo group regarding the number of red blood cell transfusions at 3 months (P=0.35) and 6 months (P=0.39), mean hemoglobin levels (P=0.66), and mean ferritin levels (P=0.86).

There were no adverse events (AEs) thought to be treatment-related. There were 161 AEs overall and no significant differences between the groups—47 AEs in the 25 mg bevacizumab group, 33 in the 50 mg bevacizumab group, 38 in the 75 mg bevacizumab group, and 43 in the placebo group.

After an interim analysis, an independent data monitoring committee recommended terminating this study early due to treatment futility. So the study was stopped before entering phase 3.

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A hand spraying a mist

of nasal spray

Bevacizumab nasal spray is no more effective than a placebo for reducing epistaxis caused by hereditary hemorrhagic telangiectasia (HHT), according to a study published in JAMA.

Researchers compared 3 different doses of bevacizumab to a sodium chloride placebo and found no significant difference between the treatment groups in the frequency or duration of epistaxis, the need for transfusion, or hemoglobin/ferritin levels.

Sophie Dupuis-Girod, MD, PhD, of the Hopital Femme-Mere-Enfants in Bron, France, and her colleagues conducted this trial, enrolling 80 patients with HHT and a history of epistaxis.

The patients were randomized to receive placebo (0.9% sodium chloride, n=21) or 1 of 3 doses of bevacizumab nasal spray.

Patients in the bevacizumab groups received 25 mg (n=20), 50 mg (n=20), or 75 mg (n=19) in 3 doses, 14 days apart, for a total treatment duration of 4 weeks, resulting in a total dose of 75 mg, 150 mg, or 225 mg.

Epistaxis duration

The researchers measured the mean monthly epistaxis duration at 3 months and found no significant difference between the placebo group and the bevacizumab groups (P=0.57). Likewise, there were no significant differences between the bevacizumab groups.

However, most of the groups experienced improvements in epistaxis duration after treatment.

The mean monthly epistaxis duration (in minutes) before treatment (from day −90 to day −1) and after treatment (from day 29 to day 118), respectively, was:

  • 262.8 and 200.4 in the placebo group
  • 285.5 and 259.2 in the 25 mg group
  • 229.0 and 244.0 in the 50 mg group
  • 272.9 and 215.0 in the 75 mg group.

Epistaxis frequency

There was no significant difference between the bevacizumab groups and the placebo group when it came to the number of epistaxis episodes (P=0.55). However, patients in all of the groups saw a reduction in episodes after treatment.

The mean number of epistaxis episodes before treatment (from day −90 to day −1) and after (from day 29 to day 118), respectively, were:

  • 31.37 and 24.27 in the placebo group
  • 30.99 and 23.13 in the 25 mg bevacizumab group
  • 25.06 and 20.01 in the 50 mg bevacizumab group
  • 27.84 and 24.35 in the 75 mg bevacizumab group.

Other outcomes

There was no significant difference between the bevacizumab groups and the placebo group regarding the number of red blood cell transfusions at 3 months (P=0.35) and 6 months (P=0.39), mean hemoglobin levels (P=0.66), and mean ferritin levels (P=0.86).

There were no adverse events (AEs) thought to be treatment-related. There were 161 AEs overall and no significant differences between the groups—47 AEs in the 25 mg bevacizumab group, 33 in the 50 mg bevacizumab group, 38 in the 75 mg bevacizumab group, and 43 in the placebo group.

After an interim analysis, an independent data monitoring committee recommended terminating this study early due to treatment futility. So the study was stopped before entering phase 3.

A hand spraying a mist

of nasal spray

Bevacizumab nasal spray is no more effective than a placebo for reducing epistaxis caused by hereditary hemorrhagic telangiectasia (HHT), according to a study published in JAMA.

Researchers compared 3 different doses of bevacizumab to a sodium chloride placebo and found no significant difference between the treatment groups in the frequency or duration of epistaxis, the need for transfusion, or hemoglobin/ferritin levels.

Sophie Dupuis-Girod, MD, PhD, of the Hopital Femme-Mere-Enfants in Bron, France, and her colleagues conducted this trial, enrolling 80 patients with HHT and a history of epistaxis.

The patients were randomized to receive placebo (0.9% sodium chloride, n=21) or 1 of 3 doses of bevacizumab nasal spray.

Patients in the bevacizumab groups received 25 mg (n=20), 50 mg (n=20), or 75 mg (n=19) in 3 doses, 14 days apart, for a total treatment duration of 4 weeks, resulting in a total dose of 75 mg, 150 mg, or 225 mg.

Epistaxis duration

The researchers measured the mean monthly epistaxis duration at 3 months and found no significant difference between the placebo group and the bevacizumab groups (P=0.57). Likewise, there were no significant differences between the bevacizumab groups.

However, most of the groups experienced improvements in epistaxis duration after treatment.

The mean monthly epistaxis duration (in minutes) before treatment (from day −90 to day −1) and after treatment (from day 29 to day 118), respectively, was:

  • 262.8 and 200.4 in the placebo group
  • 285.5 and 259.2 in the 25 mg group
  • 229.0 and 244.0 in the 50 mg group
  • 272.9 and 215.0 in the 75 mg group.

Epistaxis frequency

There was no significant difference between the bevacizumab groups and the placebo group when it came to the number of epistaxis episodes (P=0.55). However, patients in all of the groups saw a reduction in episodes after treatment.

The mean number of epistaxis episodes before treatment (from day −90 to day −1) and after (from day 29 to day 118), respectively, were:

  • 31.37 and 24.27 in the placebo group
  • 30.99 and 23.13 in the 25 mg bevacizumab group
  • 25.06 and 20.01 in the 50 mg bevacizumab group
  • 27.84 and 24.35 in the 75 mg bevacizumab group.

Other outcomes

There was no significant difference between the bevacizumab groups and the placebo group regarding the number of red blood cell transfusions at 3 months (P=0.35) and 6 months (P=0.39), mean hemoglobin levels (P=0.66), and mean ferritin levels (P=0.86).

There were no adverse events (AEs) thought to be treatment-related. There were 161 AEs overall and no significant differences between the groups—47 AEs in the 25 mg bevacizumab group, 33 in the 50 mg bevacizumab group, 38 in the 75 mg bevacizumab group, and 43 in the placebo group.

After an interim analysis, an independent data monitoring committee recommended terminating this study early due to treatment futility. So the study was stopped before entering phase 3.

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