Abstract: P215
Type: Poster Presentation
Presentation during EHA22: On Friday, June 23, 2017 from 17:15 - 18:45
Location: Poster area (Hall 7)
Background
Aims
To characterize the prevalence, characteristics, and course of IDH-DS in pts with relapsed or refractory (R/R) AML receiving enasidenib 100 mg daily in a phase 1 dose-escalation and expansion study (NCT01915498). This dose is currently under study in a multicenter, randomized, phase 3 trial comparing enasidenib with conventional care regimens in R/R AML pts (NCT02577406).
Methods
An independent Differentiation Syndrome Review Committee (DSRC) was formed to review potential cases of IDH-DS. The DSRC identified and agreed upon a series of signs and symptoms possibly characteristic of IDH-DS, including fever, lung infiltrates, pleural or pericardial effusions, rapid weight gain, edema, and azotemia. In all, 27 cases (8 of investigator-reported IDH-DS and 19 with characteristics suggestive of IDH-DS) were identified and retrospectively reviewed by the DSRC to determine their consistency with IDH-DS.
Results
The DSRC determined 13 cases (11.9% of 109 R/R AML pts in the enasidenib 100 mg/day dosing cohort) to be consistent with IDH-DS. Median time to onset was 30 days (range 7-116). Manifestations of IDH-DS in >2 pts were dyspnea (n=10), pyrexia (9), lung infiltrates (8), pleural effusion (5), and kidney injury (3). IDH-DS was effectively managed with systemic corticosteroids in 12/13 cases. Leukocytosis accompanied 4/13 cases, for which hydroxyurea was employed for cytoreduction. Enasidenib was interrupted for 9 pts (for a median of 7 days), but dose reductions or enasidenib discontinuation were not required for pts with IDH-DS. Six of the 13 pts had clinical responses (2 complete remissions [CR], 2 CRs with incomplete hematologic recovery, 1 partial remission, and 1 morphologic leukemia-free state), 6 pts had stable disease, and 1 pt had progressive disease.
Conclusion
Session topic: 4. Acute myeloid leukemia - Clinical
Keyword(s): Clinical Trial, Acute Myeloid Leukemia, Safety, Mutation
Abstract: P215
Type: Poster Presentation
Presentation during EHA22: On Friday, June 23, 2017 from 17:15 - 18:45
Location: Poster area (Hall 7)
Background
Aims
To characterize the prevalence, characteristics, and course of IDH-DS in pts with relapsed or refractory (R/R) AML receiving enasidenib 100 mg daily in a phase 1 dose-escalation and expansion study (NCT01915498). This dose is currently under study in a multicenter, randomized, phase 3 trial comparing enasidenib with conventional care regimens in R/R AML pts (NCT02577406).
Methods
An independent Differentiation Syndrome Review Committee (DSRC) was formed to review potential cases of IDH-DS. The DSRC identified and agreed upon a series of signs and symptoms possibly characteristic of IDH-DS, including fever, lung infiltrates, pleural or pericardial effusions, rapid weight gain, edema, and azotemia. In all, 27 cases (8 of investigator-reported IDH-DS and 19 with characteristics suggestive of IDH-DS) were identified and retrospectively reviewed by the DSRC to determine their consistency with IDH-DS.
Results
The DSRC determined 13 cases (11.9% of 109 R/R AML pts in the enasidenib 100 mg/day dosing cohort) to be consistent with IDH-DS. Median time to onset was 30 days (range 7-116). Manifestations of IDH-DS in >2 pts were dyspnea (n=10), pyrexia (9), lung infiltrates (8), pleural effusion (5), and kidney injury (3). IDH-DS was effectively managed with systemic corticosteroids in 12/13 cases. Leukocytosis accompanied 4/13 cases, for which hydroxyurea was employed for cytoreduction. Enasidenib was interrupted for 9 pts (for a median of 7 days), but dose reductions or enasidenib discontinuation were not required for pts with IDH-DS. Six of the 13 pts had clinical responses (2 complete remissions [CR], 2 CRs with incomplete hematologic recovery, 1 partial remission, and 1 morphologic leukemia-free state), 6 pts had stable disease, and 1 pt had progressive disease.
Conclusion
Session topic: 4. Acute myeloid leukemia - Clinical
Keyword(s): Clinical Trial, Acute Myeloid Leukemia, Safety, Mutation