NPM1m AML
Revealing a potential way to remission
The AUGMENT-101 clinical trial was designed to measure the safety and effectiveness of Revuforj in people with acute myeloid leukemia (AML) with an NPM1 mutation whose disease had come back or had not improved after previous treatment(s)
Adults and children (1 year and older) who had AML with an NPM1 mutation
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64 adults and 1 child met the criteria to be evaluated for effectiveness
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The youngest person was 11 years old and the oldest was 84 years old
A specific dose of Revuforj was given based on the person’s body weight
All doses were taken by mouth 2x a day
People continued taking Revuforj until they experienced side effects or worsening of their leukemia
Remission is the main goal of AML treatment
~1 in 4 people achieved CR + CRh
In the clinical trial, about 23% of people (15 out of 65) achieved complete remission (CR) or complete remission with a partial hematological recovery (CRh).
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CR=signs of acute leukemia are gone, and blood cell counts are back to normal
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CRh=signs of acute leukemia are gone but some blood cell counts did not fully return to normal
2.8
MONTHS
Of the 15 people who achieved CR + CRh:
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Half responded within 2.8 months of treatment (median time to CR + CRh was 2.8 months; range=1.8 to 9.6 months)
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Half stayed in remission for more than 4 and a half months
Revuforj helped some people achieve complete remission
Actor portrayals.
Actor portrayals.
Rate of transplant
11
%
(7 out of 65)
of the people in the trial went on to receive a
stem cell transplant
transplant
following treatment with Revuforj
Rate of transfusion independence
17
%
(8 out of 46)
of people who needed transfusions at the start of the trial became transfusion-free
68
%
(13 out of 19)
of people who were transfusion-free at the start of the trial remained transfusion-free
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Revuforj will not work for everyone. Individual results may vary.
KMT2A AML & ALL
Complete remission may be possible with Revuforj
The AUGMENT-101 clinical trial was designed to measure the safety and effectiveness of Revuforj in people with acute leukemia with a KMT2A translocation whose disease had come back or had not improved after previous treatment(s)
Adults and children (1 year and older) who had acute leukemia with a KMT2A translocation
-
The youngest person was ~1 year old and the oldest was 79 years old
A specific dose of Revuforj was given based on the person’s body weight
All doses were taken by mouth 2x a day
People continued taking Revuforj until they experienced side effects or worsening of their leukemia
Remission is the main goal of AML and ALL treatment
~1 in 5 people achieved CR + CRh
In the clinical trial, 21% of people (22 out of 104) achieved complete remission (CR) or complete remission with a partial hematological recovery (CRh).
-
CR=signs of acute leukemia are gone, and blood cell counts are back to normal
-
CRh=signs of acute leukemia are gone but some blood cell counts did not fully return to normal
1
MONTH
Of the 22 people who achieved CR + CRh:
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Some responded in as early as 1 month
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Half responded within the first 2 months of treatment (median time to CR + CRh was 1.9 months; range=0.9 to 5.6 months)
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Half stayed in remission for more than 6.4 months
Revuforj helped some people achieve complete remission
Actor portrayal.
Actor portrayal.
Rate of transplant
23
%
(24 out of 104)
of the people in the trial went on to receive a
stem cell transplant
transplant
following treatment with Revuforj
-
Revuforj will not work for everyone. Individual results may vary.
Side effects
What are the possible side effects?
Revuforj may cause serious side effects, including differentiation syndrome
Differentiation syndrome is a serious but common condition that affects your blood cells, which may be life-threatening or lead to death if not treated. Differentiation syndrome has happened as early as 3 days and up to 41 days after starting Revuforj. Tell any healthcare provider caring for you that you are taking a medicine that can cause differentiation syndrome.
Call your healthcare provider or go to the nearest hospital emergency room right away if you develop any of the following symptoms of differentiation syndrome during treatment with Revuforj:
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fever
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cough
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shortness of breath
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severe headache
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confusion
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dizziness or lightheadedness
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fast weight gain
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swelling of arms, legs, neck, groin, or underarm area
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decreased urination
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rash
Revuforj may also cause changes in electrical activity of your heart, called “QT prolongation”
QT prolongation is a serious but common side effect that can cause irregular heartbeats that can be life-threatening, such as a heart attack, and can lead to death. Your healthcare provider will check the electrical activity of your heart with a test called an electrocardiogram (ECG) and will also do blood tests to check your potassium and magnesium levels before and during treatment with Revuforj.
Tell your healthcare provider right away if you feel faint, lightheaded, dizzy, or if you feel your heart beating irregularly or fast during treatment with Revuforj.
ECG monitoring and blood tests are required for all FDA-approved menin inhibitors.
Revuforj can harm your unborn baby
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Revuforj.
Females who are able to become pregnant:
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your healthcare provider will perform a pregnancy test within 7 days before you start treatment with Revuforj
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use effective birth control (contraception) during treatment with Revuforj and for 4 months after the last dose of Revuforj
Males who have female partners who are able to become pregnant:
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use effective birth control during treatment with Revuforj and for 4 months after the last dose of Revuforj
It is not known if Revuforj passes into your breast milk. Do not breastfeed during your treatment with Revuforj or for 1 week after your last dose of Revuforj.
Revuforj may cause fertility problems in females and males, which may affect your ability to have children. Talk to your healthcare provider if this is a concern for you.
Talk to your healthcare provider about birth control methods you can use during this time
The most common side effects of Revuforj include:
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infections, including bacterial and viral
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changes in liver function tests
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bleeding (hemorrhage)
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nausea and vomiting
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muscle pain
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low white blood cell counts with fever
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diarrhea
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tiredness
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swelling in the arms and legs
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decreased appetite
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constipation