Table 3.

Adverse events of special interest with molecularly targeted therapies and venetoclax

Adverse eventClinical characteristicsTargeted agentIncidenceDays to onset median (range)Management
Differentiation syndrome (DS) associated with targeted inhibitors
 
Dyspnea, pulmonary infiltrates, weight gain, fever, renal failure, hypotension, leukocytosis Ivosidenib 11%-19% 20 (1-78) Dexamethasone 10 mg twice daily for 72 hours or until improvement in symptoms. Rule out other etiologies for symptoms. Hydroxyurea for co-occurring leukocytosis. For severe cases, temporarily interrupt targeted therapy.
 
Olutasidenib 14% 17.5 (1-561) 
Enasidenib 14%-19% 19 (1-86) 
Gilteritinib ∼ 3% 2-75 
Quizartinib Not reported Not reported 
Revumenib 16% 18 (5-41) 
IVO-AZA 14% 19.5 (3-33) 
Tumor lysis syndrome Hyperkalemia, hyperphosphatemia, hyperuricemia, hypocalcemia, renal failure, cardiac arrhythmias, seizures Venetoclax 3% Days 1-3 (venetoclax ramp) Employ venetoclax ramp-up dosing on days 1-3 of initiation of therapy. Administer prophylactic uric acid reducing agents and intravenous fluids when initiating therapy. Monitor chemistry panel every 4–6 hours when initiating therapy. 
QTc prolongation
 
Prolongation of the QT interval beyond 450  msec. Grade 3 QT prolongation defined as ≥500  msec or >60  msec change from baseline. Gilteritinib 9% Interrupt treatment for QTcF >500 msec and reduce gilteritinib dosage. 
Quizartinib 2%-14% Reduce quizartinib dose for QTcF >481-500. Interrupt treatment for QTcF >500  msec and reduce quizartinib dosage. 
Revumenib 53% (grade 3: 13%) Revumenib interrupted for grade ≥3 QT prolongation and resumed at lower dose-level. 
Ivosidenib 8%-18% Interrupt treatment for QTcF >480-500  msec. Restart ivosidenib once QTcF <480 and monitor EKG weekly for 2 weeks. 
Hepatic abnormalities
 
Elevated liver function tests Olutasidenib 12% (ALT) 9% (AST) For grade ≥3 AEs, interrupt treatment until ≤ grade 1 and resume at 1 dose-level lower. Can increase to full dose if no recurrence within 30-days after initial event. 
Hyperbilirubinemia Enasidenib 12%-31% Reduce enasidenib to 50  mg/d if bilirubin ≥3  ×  upper limit of normal. Can resume at 100  mg/d if bilirubin resolves to <2  ×  upper limit of normal. 
Norsworthy et al. CCR 2020;63 Fathi et al. JAMA Onc. 2018;64 de Botton et al. Blood Adv. 2023;65 Fathi et al. AJH 2021 (Differentiation syndrome);66 DiNardo et al. NEJM 2020 (Tumor lysis syndrome),9 Cortez et al. Lancet Haematology 2019,68 Issa et al. Nature 2023;26 Stein et al. Blood 2017;69 Roboz et al. Blood 2020;67 U.S. Package inserts for ivosidenib, olutasidenib, enasidenib, gilteritinib, quizartinib. 
Adverse eventClinical characteristicsTargeted agentIncidenceDays to onset median (range)Management
Differentiation syndrome (DS) associated with targeted inhibitors
 
Dyspnea, pulmonary infiltrates, weight gain, fever, renal failure, hypotension, leukocytosis Ivosidenib 11%-19% 20 (1-78) Dexamethasone 10 mg twice daily for 72 hours or until improvement in symptoms. Rule out other etiologies for symptoms. Hydroxyurea for co-occurring leukocytosis. For severe cases, temporarily interrupt targeted therapy.
 
Olutasidenib 14% 17.5 (1-561) 
Enasidenib 14%-19% 19 (1-86) 
Gilteritinib ∼ 3% 2-75 
Quizartinib Not reported Not reported 
Revumenib 16% 18 (5-41) 
IVO-AZA 14% 19.5 (3-33) 
Tumor lysis syndrome Hyperkalemia, hyperphosphatemia, hyperuricemia, hypocalcemia, renal failure, cardiac arrhythmias, seizures Venetoclax 3% Days 1-3 (venetoclax ramp) Employ venetoclax ramp-up dosing on days 1-3 of initiation of therapy. Administer prophylactic uric acid reducing agents and intravenous fluids when initiating therapy. Monitor chemistry panel every 4–6 hours when initiating therapy. 
QTc prolongation
 
Prolongation of the QT interval beyond 450  msec. Grade 3 QT prolongation defined as ≥500  msec or >60  msec change from baseline. Gilteritinib 9% Interrupt treatment for QTcF >500 msec and reduce gilteritinib dosage. 
Quizartinib 2%-14% Reduce quizartinib dose for QTcF >481-500. Interrupt treatment for QTcF >500  msec and reduce quizartinib dosage. 
Revumenib 53% (grade 3: 13%) Revumenib interrupted for grade ≥3 QT prolongation and resumed at lower dose-level. 
Ivosidenib 8%-18% Interrupt treatment for QTcF >480-500  msec. Restart ivosidenib once QTcF <480 and monitor EKG weekly for 2 weeks. 
Hepatic abnormalities
 
Elevated liver function tests Olutasidenib 12% (ALT) 9% (AST) For grade ≥3 AEs, interrupt treatment until ≤ grade 1 and resume at 1 dose-level lower. Can increase to full dose if no recurrence within 30-days after initial event. 
Hyperbilirubinemia Enasidenib 12%-31% Reduce enasidenib to 50  mg/d if bilirubin ≥3  ×  upper limit of normal. Can resume at 100  mg/d if bilirubin resolves to <2  ×  upper limit of normal. 
Norsworthy et al. CCR 2020;63 Fathi et al. JAMA Onc. 2018;64 de Botton et al. Blood Adv. 2023;65 Fathi et al. AJH 2021 (Differentiation syndrome);66 DiNardo et al. NEJM 2020 (Tumor lysis syndrome),9 Cortez et al. Lancet Haematology 2019,68 Issa et al. Nature 2023;26 Stein et al. Blood 2017;69 Roboz et al. Blood 2020;67 U.S. Package inserts for ivosidenib, olutasidenib, enasidenib, gilteritinib, quizartinib. 

ALT, alanine aminotransferase; AST, aspartate aminotransferase; EKG, electrocardiogram.

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