Watch a theoretical patient case and results from the pivotal trial
View two experts discussing a theoretical patient with R/R AML who received IDHIFA® and outcomes from the IDHIFA® pivotal trial.
Dr Hetty Carraway and Dr Daniel A Pollyea have been paid by Bristol Myers Squibb to participate in this video.
Dr Hetty Carraway is an Associate Professor of Medicine at the Cleveland Clinic.
Dr Daniel A Pollyea is an Associate Professor of Medicine and Clinical Director of Leukemia Services, Division of Hematology at the University of Colorado School of Medicine.
IDHIFA® Clinical Trial Design
FDA approval of IDHIFA® was based on results from
THE FIRST PIVOTAL TRIAL EXCLUSIVELY IN R/R AML WITH AN IDH2 MUTATION
IDHIFA® was studied in an open-label, single-arm, multicenter, clinical trial of patients who had R/R AML with an IDH2 mutation who were assigned a starting dose of 100 mg daily until disease progression or unacceptable toxicity. Dose reductions were allowed to manage adverse events. The efficacy analysis included 199 patients; the safety analysis included 214 patients.
Efficacy was established on the basis of:
Rate of complete response (CR): defined as <5% of blasts in the bone marrow, no evidence of disease, and full recovery of peripheral blood counts (platelets >100,000/µL and absolute neutrophil counts [ANC] >1,000/µL)
Rate of complete response with partial hematologic recovery (CRh): defined as CR requirement for blast count, no evidence of disease, but only partial recovery of peripheral blood counts (platelets >50,000/µL and ANC >500/µL)
Duration of response (DOR): defined as time since first response of CR or CRh to relapse or death, whichever is earlier
Rate of conversion from transfusion dependence to transfusion independence*
The median follow-up was 6.6 months (range, 0.4 to 27.7).
For patients in the efficacy analysis, IDH2 mutations were either prospectively identified or retrospectively confirmed by the Abbott RealTime™ IDH2 assay.
Of the patients enrolled and treated, 93% (199/214) had IDH2 mutations detected by the Abbott RealTime™ IDH2 assay and had R/R status confirmed by FDA. Therefore, the 199 patients were included in the efficacy analysis1
*Patients were defined as transfusion independent if they received no RBC or platelet transfusions within any 56-day post-baseline period.
IDHIFA®: the first and only therapy indicated for R/R AML with an IDH2 mutation Targeted therapy delivered in an oral tablet
IDHIFA® (enasidenib) is indicated for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.
Patients treated with IDHIFA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, acute respiratory distress, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, lymphadenopathy, bone pain, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.
Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 14% of patients treated with IDHIFA experienced differentiation syndrome, which may be life-threatening or fatal if not treated. Differentiation syndrome has been observed with and without concomitant hyperleukocytosis, in as early as 1 day and up to 5 months after IDHIFA initiation. Symptoms in patients treated with IDHIFA included acute respiratory distress represented by dyspnea and/or hypoxia and need for supplemental oxygen; pulmonary infiltrates and pleural effusion; renal impairment; fever; lymphadenopathy; bone pain; peripheral edema with rapid weight gain; and pericardial effusion. Hepatic, renal, and multi-organ dysfunction have also been observed. If differentiation syndrome is suspected, initiate systemic corticosteroids and hemodynamic monitoring until improvement. Taper corticosteroids only after resolution of symptoms. Differentiation syndrome symptoms may recur with premature discontinuation of corticosteroids. If severe pulmonary symptoms requiring intubation or ventilator support and/or renal dysfunction persist for more than 48 hours after initiation of corticosteroids, interrupt IDHIFA until signs and symptoms are no longer severe. Hospitalization for close observation and monitoring of patients with pulmonary and/or renal manifestation is recommended.
Embryo-Fetal Toxicity: Based on animal embryo-fetal toxicity studies, IDHIFA can cause embryo-fetal harm when administered to a pregnant woman. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with IDHIFA and for at least 2 months after the last dose. Advise pregnant women of the potential risk to the fetus.
The most common adverse reactions (≥20%) included total bilirubin increased (81%), calcium decreased (74%), nausea (50%), diarrhea (43%), potassium decreased (41%), vomiting (34%), decreased appetite (34%), and phosphorus decreased (27%)
The most frequently reported ≥Grade 3 adverse reactions (≥5%) included total bilirubin increased (15%), potassium decreased (15%), phosphorus decreased (8%), calcium decreased (8%), diarrhea (8%), differentiation syndrome (7%), non-infectious leukocytosis (6%), tumor lysis syndrome (6%), and nausea (5%)
Serious adverse reactions were reported in 77.1% of patients. The most frequent serious adverse reactions (≥2%) were leukocytosis (10%), diarrhea (6%), nausea (5%), vomiting (3%), decreased appetite (3%), tumor lysis syndrome (5%), and differentiation syndrome (8%). Differentiation syndrome events characterized as serious included pyrexia, renal failure acute, hypoxia, respiratory failure, and multi-organ failure
Coadministration of IDHIFA increases the exposure of OATP1B1, OATP1B3, BCRP, and P‑glycoprotein (P‑gp) substrates, which may increase the incidence and severity of adverse reactions of these substrates. If coadministered, decrease the dosage of the substrate as recommended in the respective prescribing information and as clinically indicated.
Because of the potential for adverse reactions in the breastfed child, advise women not to breastfeed during treatment with IDHIFA and for at least 2 months after the last dose.
Data on file, Celgene Corporation. Summit, New Jersey.
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