Acute myeloid leukemia, FLT3-positive
Section editor | |
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Martin W. Schoen, MD, MPH Saint Louis University St. Louis, MO mwschoen |
Note: these are regimens tested in biomarker-specific populations for patients with FLT3 internal tandem duplicated (FLT3-ITD) or tyrosine kinase domain mutated (FLT3-TKD) AML, please see the main AML page for other regimens.
22 regimens on this page
27 variants on this page
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Upfront induction therapy, standard patients
7+3d (intermediate-dose)
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7+3d: 7 days of cytarabine + 3 days of daunorubicin
Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
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Stone et al. 2017 (RATIFY) | 2008-2011 | Phase III (C) | 7+3d & Midostaurin | Inferior OS |
Chemotherapy
- Cytarabine (Ara-C) 200 mg/m2/day IV continuous infusion over 7 days, started on day 1 (total dose: 1400 mg/m2)
- Daunorubicin (Cerubidine) 60 mg/m2 IV over 5 minutes once per day on days 1 to 3
Supportive medications
- "According to commonly accepted guidelines with no prophylactic IV antibiotics"
- Granulocyte colony-stimulating factor recommended only for patients older than 50 years old whose leukemic blasts were negative for CD114 expression
7-day course
References
- RATIFY: Stone RM, Mandrekar SJ, Sanford BL, Laumann K, Geyer S, Bloomfield CD, Thiede C, Prior TW, Döhner K, Marcucci G, Lo-Coco F, Klisovic RB, Wei A, Sierra J, Sanz MA, Brandwein JM, de Witte T, Niederwieser D, Appelbaum FR, Medeiros BC, Tallman MS, Krauter J, Schlenk RF, Ganser A, Serve H, Ehninger G, Amadori S, Larson RA, Döhner H. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med. 2017 Aug 3;377(5):454-464. Epub 2017 Jun 23. link to original article link to PMC article contains verified protocol PubMed NCT00651261
- Q-SOC: NCT04676243
- QuANTUM-First: NCT02668653
7+3d & Midostaurin
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7+3d & Midostaurin: 7 days of cytarabine, 3 days of daunorubicin, Midostaurin
Regimen
FDA-recommended dose |
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
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Stone et al. 2017 (RATIFY) | 2008-2011 | Phase III (E-RT-esc) | 7+3d (intermediate-dose) | Superior OS |
Chemotherapy
- Cytarabine (Ara-C) 200 mg/m2/day IV continuous infusion on days 1 to 7 (total dose: 1400 mg/m2)
- Daunorubicin (Cerubidine) 60 mg/m2 IV once per day on days 1 to 3
Targeted therapy
- Midostaurin (Rydapt) 50 mg PO twice per day on days 8 to 21
Supportive medications
- Hydroxyurea (Hydrea) (no dosage specified) was allowed to be used for up to 5 days before the start of therapy while waiting for results of FLT3 mutation testing
21-day course; retreatment with a second course was allowed if day 21 bone marrow biopsy showed residual AML.
Subsequent treatment
- Patients who achieved complete remission (CR): HiDAC & Midostaurin consolidation. Stem cell transplantation was allowed.
References
- RATIFY: Stone RM, Mandrekar SJ, Sanford BL, Laumann K, Geyer S, Bloomfield CD, Thiede C, Prior TW, Döhner K, Marcucci G, Lo-Coco F, Klisovic RB, Wei A, Sierra J, Sanz MA, Brandwein JM, de Witte T, Niederwieser D, Appelbaum FR, Medeiros BC, Tallman MS, Krauter J, Schlenk RF, Ganser A, Serve H, Ehninger G, Amadori S, Larson RA, Döhner H. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med. 2017 Aug 3;377(5):454-464. Epub 2017 Jun 23. link to original article link to PMC article contains verified protocol PubMed NCT00651261
First-line induction therapy, older patients or "unfit" patients
7+3d & Sorafenib
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Regimen
Study | Evidence |
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Uy et al. 2017 (CALGB 11001) | Phase II |
Chemotherapy
- Cytarabine (Ara-C) 100 mg/m2/day IV continuous infusion on days 1 to 7 (total dose: 700 mg/m2)
- Daunorubicin (Cerubidine) 60 mg/m2 IV once per day on days 1 to 3
Targeted therapy
- Sorafenib (Nexavar) 400 mg PO twice per day on days 1 to 7
7-day course
Subsequent treatment
- Patients not achieving a hypoplastic marrow on day 14: 5+2 & sorafenib re-induction
- Patients achieving a CR or CRi: IDAC & sorafenib consolidation
References
- CALGB 11001: Uy GL, Mandrekar SJ, Laumann K, Marcucci G, Zhao W, Levis MJ, Klepin HD, Baer MR, Powell BL, Westervelt P, DeAngelo DJ, Stock W, Sanford B, Blum WG, Bloomfield CD, Stone RM, Larson RA. A phase 2 study incorporating sorafenib into the chemotherapy for older adults with FLT3-mutated acute myeloid leukemia: CALGB 11001. Blood Adv. 2017 Jan 24;1(5):331-340. link to original article contains verified protocol link to PMC article PubMed NCT01253070
Consolidation after upfront therapy
HiDAC & Midostaurin
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HiDAC & Midostaurin: High Dose Ara-C (Cytarabine) & Midostaurin
Regimen
FDA-recommended dose |
Study | Evidence |
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Stone et al. 2017 (RATIFY) | Non-randomized portion of RCT |
Preceding treatment
- 7+3d & midostaurin induction, with CR
Chemotherapy
- Cytarabine (Ara-C) 3000 mg/m2 every 12 hours IV over 3 hours every 12 hours on days 1, 3, 5 (total dose per cycle: 18,000 mg/m2)
Targeted therapy
- Midostaurin (Rydapt) 50 mg PO twice per day on days 8 to 21
28-day cycle for 4 cycles
Subsequent treatment
- Stem cell transplantation "was allowed" for eligible patients; others proceeded to midostaurin maintenance
References
- RATIFY: Stone RM, Mandrekar SJ, Sanford BL, Laumann K, Geyer S, Bloomfield CD, Thiede C, Prior TW, Döhner K, Marcucci G, Lo-Coco F, Klisovic RB, Wei A, Sierra J, Sanz MA, Brandwein JM, de Witte T, Niederwieser D, Appelbaum FR, Medeiros BC, Tallman MS, Krauter J, Schlenk RF, Ganser A, Serve H, Ehninger G, Amadori S, Larson RA, Döhner H. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med. 2017 Aug 3;377(5):454-464. Epub 2017 Jun 23. link to original article link to PMC article contains verified protocol PubMed NCT00651261
IDAC & Sorafenib
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IDAC & Sorafenib: Intermediate Dose Ara-C (Cytarabine) & Sorafenib
Regimen
Study | Evidence |
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Uy et al. 2017 (CALGB 11001) | Phase II |
Preceding treatment
Chemotherapy
- Cytarabine (Ara-C) 2000 mg/m2 IV over 3 hours once per day on days 1 to 5
Targeted therapy
- Sorafenib (Nexavar) 400 mg PO twice per day on days 1 to 28
4- to 6-week cycle for 2 cycles
Subsequent treatment
References
- CALGB 11001: Uy GL, Mandrekar SJ, Laumann K, Marcucci G, Zhao W, Levis MJ, Klepin HD, Baer MR, Powell BL, Westervelt P, DeAngelo DJ, Stock W, Sanford B, Blum WG, Bloomfield CD, Stone RM, Larson RA. A phase 2 study incorporating sorafenib into the chemotherapy for older adults with FLT3-mutated acute myeloid leukemia: CALGB 11001. Blood Adv. 2017 Jan 24;1(5):331-340. link to original article contains verified protocol link to PMC article PubMed NCT01253070
Maintenance after upfront therapy, including allogeneic HSCT
Midostaurin monotherapy
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Regimen
Study | Evidence | Efficacy |
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Stone et al. 2017 (RATIFY) | Non-randomized portion of RCT | CR rate: 59% after induction |
Preceding treatment
Targeted therapy
- Midostaurin (Rydapt) 50 mg PO twice per day
28-day cycle for up to 13 cycles (1 year)
References
- RATIFY: Stone RM, Mandrekar SJ, Sanford BL, Laumann K, Geyer S, Bloomfield CD, Thiede C, Prior TW, Döhner K, Marcucci G, Lo-Coco F, Klisovic RB, Wei A, Sierra J, Sanz MA, Brandwein JM, de Witte T, Niederwieser D, Appelbaum FR, Medeiros BC, Tallman MS, Krauter J, Schlenk RF, Ganser A, Serve H, Ehninger G, Amadori S, Larson RA, Döhner H. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med. 2017 Aug 3;377(5):454-464. Epub 2017 Jun 23. link to original article link to PMC article contains verified protocol PubMed NCT00651261
- ARO-021: NCT03258931
- HOVON 156 AML: NCT04027309
Sorafenib monotherapy
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Regimen variant #1, 6 months
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
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Xuan et al. 2020 (Sorafenib-Flt3 AML-2015) | 2015-2018 | Phase III (E-esc) | Observation | Superior 1-year cumulative incidence of relapse |
Preceding treatment
- Allogeneic stem cell transplant
Targeted therapy
- Sorafenib (Nexavar) 400 mg PO twice per day on days +30 to +180
6-month course
Regimen variant #2, 12 mos
Study | Evidence |
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Uy et al. 2017 (CALGB 11001) | Phase II |
Preceding treatment
Targeted therapy
- Sorafenib (Nexavar) 400 mg PO twice per day
28-day cycle for up to 12 cycles
Regimen variant #3, 2 years
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
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Burchert et al. 2020 (SORMAIN) | 2010-2016 | Phase III (E-esc) | Placebo | Seems to have superior RFS |
Preceding treatment
- Allogeneic stem cell transplant
Targeted therapy
- Sorafenib (Nexavar) as follows:
- Cycle 1: 200 mg PO twice per day on days 1 to 14, then increasing to 400 mg PO twice per day if tolerated
- Cycles 2 to 26: 400 mg PO twice per day
28-day cycle for up to 26 cycles (2 years)
References
- CALGB 11001: Uy GL, Mandrekar SJ, Laumann K, Marcucci G, Zhao W, Levis MJ, Klepin HD, Baer MR, Powell BL, Westervelt P, DeAngelo DJ, Stock W, Sanford B, Blum WG, Bloomfield CD, Stone RM, Larson RA. A phase 2 study incorporating sorafenib into the chemotherapy for older adults with FLT3-mutated acute myeloid leukemia: CALGB 11001. Blood Adv. 2017 Jan 24;1(5):331-340. link to original article contains verified protocol link to PMC article PubMed NCT01253070
- SORMAIN: Burchert A, Bug G, Fritz LV, Finke J, Stelljes M, Röllig C, Wollmer E, Wäsch R, Bornhäuser M, Berg T, Lang F, Ehninger G, Serve H, Zeiser R, Wagner EM, Kröger N, Wolschke C, Schleuning M, Götze KS, Schmid C, Crysandt M, Eßeling E, Wolf D, Wang Y, Böhm A, Thiede C, Haferlach T, Michel C, Bethge W, Wündisch T, Brandts C, Harnisch S, Wittenberg M, Hoeffkes HG, Rospleszcz S, Burchardt A, Neubauer A, Brugger M, Strauch K, Schade-Brittinger C, Metzelder SK. Sorafenib Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia With FLT3-Internal Tandem Duplication Mutation (SORMAIN). J Clin Oncol. 2020 Sep 10;38(26):2993-3002. Epub 2020 Jul 16. link to original article contains verified protocol PubMed Link to clinical trial registration DRKS00000591
- Sorafenib-Flt3 AML-2015: Xuan L, Wang Y, Huang F, Fan Z, Xu Y, Sun J, Xu N, Deng L, Li X, Liang X, Luo X, Shi P, Liu H, Wang Z, Jiang L, Yu C, Zhou X, Lin R, Chen Y, Tu S, Huang X, Liu Q. Sorafenib maintenance in patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised phase 3 trial. Lancet Oncol. 2020 Sep;21(9):1201-1212. Epub 2020 Aug 10. link to original article PubMed NCT02474290
Relapsed or refractory, salvage therapy
Midostaurin monotherapy
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Regimen variant #1
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Fischer et al. 2010 (CPKC412A2104) | 2002-NR | Randomized Phase IIB (E-de-esc) | Midostaurin; 100 mg twice per day | Not reported |
Targeted therapy
- Midostaurin (Rydapt) 50 mg PO twice per day
Continued indefinitely
Regimen variant #2
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Fischer et al. 2010 (CPKC412A2104) | 2002-NR | Randomized Phase IIB (E-esc) | Midostaurin; 50 mg twice per day | Not reported |
Targeted therapy
- Midostaurin (Rydapt) 100 mg PO twice per day
Continued indefinitely
Regimen variant #3
Study | Evidence |
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Stone et al. 2004 | Phase II |
Patients were required to have a FLT3 ITD or FLT3 p.D835Y mutation.
Targeted therapy
- Midostaurin (Rydapt) 75 mg PO three times per day
28-day cycles
References
- Stone RM, DeAngelo DJ, Klimek V, Galinsky I, Estey E, Nimer SD, Grandin W, Lebwohl D, Wang Y, Cohen P, Fox EA, Neuberg D, Clark J, Gilliland DG, Griffin JD. Patients with acute myeloid leukemia and an activating mutation in FLT3 respond to a small-molecule FLT3 tyrosine kinase inhibitor, PKC412. Blood. 2005 Jan 1;105(1):54-60. Epub 2004 Sep 2. link to original article contains verified protocol PubMed
- CPKC412A2104: Fischer T, Stone RM, Deangelo DJ, Galinsky I, Estey E, Lanza C, Fox E, Ehninger G, Feldman EJ, Schiller GJ, Klimek VM, Nimer SD, Gilliland DG, Dutreix C, Huntsman-Labed A, Virkus J, Giles FJ. Phase IIB trial of oral midostaurin (PKC412), the FMS-like tyrosine kinase 3 receptor (FLT3) and multi-targeted kinase inhibitor, in patients with acute myeloid leukemia and high-risk myelodysplastic syndrome with either wild-type or mutated FLT3. J Clin Oncol. 2010 Oct 1;28(28):4339-45. Epub 2010 Aug 23. link to original article link to PMC article contains verified protocol PubMed NCT00045942
Relapsed or refractory, further lines of therapy
Azacitidine & Sorafenib
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Regimen
Study | Evidence |
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Ravandi et al. 2013 (MDACC 2010-0511) | Phase II |
Chemotherapy
- Azacitidine (Vidaza) 75 mg/m2 IV or SC once per day on days 1 to 7
Targeted therapy
- Sorafenib (Nexavar) 400 mg PO twice per day
Supportive medications
- "All patients received antimicrobials, supportive care, and transfusions of blood products according to the institutional guidelines."
4- to 8-week cycles
References
- MDACC 2010-0511: Ravandi F, Alattar ML, Grunwald MR, Rudek MA, Rajkhowa T, Richie MA, Pierce S, Daver N, Garcia-Manero G, Faderl S, Nazha A, Konopleva M, Borthakur G, Burger J, Kadia T, Dellasala S, Andreeff M, Cortes J, Kantarjian H, Levis M. Phase 2 study of azacytidine plus sorafenib in patients with acute myeloid leukemia and FLT-3 internal tandem duplication mutation. Blood. 2013 Jun 6;121(23):4655-62. Epub 2013 Apr 23. link to original article contains verified protocol link to PMC article PubMed NCT01254890
FLAG-Ida
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FLAG-Ida: FLudarabine, Ara-C (Cytarabine), G-CSF (Filgrastim), Idarubicin
Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Cortes et al. 2019 (QuANTUM-R) | 2014-2017 | Phase III (C) | Quizartinib | Seems to have inferior OS |
Chemotherapy
- Fludarabine (Fludara) 30 mg/m2 IV over 30 minutes once per day on days 2 to 6
- Cytarabine (Ara-C) 2000 mg/m2 IV once per day on days 2 to 6
- Idarubicin (Idamycin) 10 mg/m2 IV once per day on days 2 to 4
Growth factor therapy
- Filgrastim (Neupogen) by one of the following:
- 5 mcg/kg SC once per day on days 1 to 5
- 300 mcg/m2 SC once per day on days 1 to 5
28-day cycle for up to 2 cycles
References
- QuANTUM-R: Cortes JE, Khaled S, Martinelli G, Perl AE, Ganguly S, Russell N, Krämer A, Dombret H, Hogge D, Jonas BA, Leung AY, Mehta P, Montesinos P, Radsak M, Sica S, Arunachalam M, Holmes M, Kobayashi K, Namuyinga R, Ge N, Yver A, Zhang Y, Levis MJ. Quizartinib versus salvage chemotherapy in relapsed or refractory FLT3-ITD acute myeloid leukaemia (QuANTUM-R): a multicentre, randomised, controlled, open-label, phase 3 trial. Lancet Oncol. 2019 Jul;20(7):984-997. Epub 2019 Jun 4. Erratum in: Lancet Oncol. 2019 Jul;20(7):e346. link to original article PubMed NCT02039726
- ARO-013: NCT03250338
Gilteritinib monotherapy
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Regimen
FDA-recommended dose |
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
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Perl et al. 2017 (2215-CL-0101) | 2013-2015 | Phase I/II | ||
Perl et al. 2019 (ADMIRAL) | 2015-2018 | Phase III (E-RT-switch-ooc) | Salvage Chemotherapy | Superior OS Median OS: 9.3 mo vs 5.6 mo (HR 0.64, 95% CI 0.49-0.83) |
Targeted therapy
- Gilteritinib (Xospata) 120 mg PO once per day
28-day cycles
References
- 2215-CL-0101: Perl AE, Altman JK, Cortes J, Smith C, Litzow M, Baer MR, Claxton D, Erba HP, Gill S, Goldberg S, Jurcic JG, Larson RA, Liu C, Ritchie E, Schiller G, Spira AI, Strickland SA, Tibes R, Ustun C, Wang ES, Stuart R, Röllig C, Neubauer A, Martinelli G, Bahceci E, Levis M. Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1-2 study. Lancet Oncol. 2017 Aug;18(8):1061-1075. Epub 2017 Jun 20. link to original article PubMed NCT02014558
- ADMIRAL: Perl AE, Martinelli G, Cortes JE, Neubauer A, Berman E, Paolini S, Montesinos P, Baer MR, Larson RA, Ustun C, Fabbiano F, Erba HP, Di Stasi A, Stuart R, Olin R, Kasner M, Ciceri F, Chou W-C, Podoltsev N, Recher C, Yokoyama H, Hosono N, Yoon S-S, Lee J-H, Pardee T, Fathi AT, Liu C, Hasabou N, Liu X, Bahceci E, Levis MJ. Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML. N Engl J Med. 2019;381:1728-40. link to original article contains verified protocol PubMed NCT02421939
Low-dose Cytarabine monotherapy (LoDAC)
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LoDAC: Low Dose Ara-C (cytarabine)
LDAC: Low Dose Ara-C (cytarabine)
Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Cortes et al. 2019 (QuANTUM-R) | 2014-2017 | Phase III (C) | Quizartinib | Seems to have inferior OS |
Chemotherapy
- Cytarabine (Ara-C) 20 mg SC twice per day on days 1 to 10
28-day cycles
References
- QuANTUM-R: Cortes JE, Khaled S, Martinelli G, Perl AE, Ganguly S, Russell N, Krämer A, Dombret H, Hogge D, Jonas BA, Leung AY, Mehta P, Montesinos P, Radsak M, Sica S, Arunachalam M, Holmes M, Kobayashi K, Namuyinga R, Ge N, Yver A, Zhang Y, Levis MJ. Quizartinib versus salvage chemotherapy in relapsed or refractory FLT3-ITD acute myeloid leukaemia (QuANTUM-R): a multicentre, randomised, controlled, open-label, phase 3 trial. Lancet Oncol. 2019 Jul;20(7):984-997. Epub 2019 Jun 4. Erratum in: Lancet Oncol. 2019 Jul;20(7):e346. link to original article contains protocol PubMed NCT02039726
MEC
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MEC: Mitoxantrone, Etoposide, Cytarabine
Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Cortes et al. 2019 (QuANTUM-R) | 2014-2017 | Phase III (C) | Quizartinib | Seems to have inferior OS |
Chemotherapy
- Mitoxantrone (Novantrone) 8 mg/m2 IV push once per day on days 1 to 5, given third
- Etoposide (Vepesid) 100 mg/m2 IV over 2 hours once per day on days 1 to 5, given first
- Cytarabine (Ara-C) 1000 mg/m2 IV once per day on days 1 to 5, given second
28-day cycle for up to 2 cycles
References
- QuANTUM-R: Cortes JE, Khaled S, Martinelli G, Perl AE, Ganguly S, Russell N, Krämer A, Dombret H, Hogge D, Jonas BA, Leung AY, Mehta P, Montesinos P, Radsak M, Sica S, Arunachalam M, Holmes M, Kobayashi K, Namuyinga R, Ge N, Yver A, Zhang Y, Levis MJ. Quizartinib versus salvage chemotherapy in relapsed or refractory FLT3-ITD acute myeloid leukaemia (QuANTUM-R): a multicentre, randomised, controlled, open-label, phase 3 trial. Lancet Oncol. 2019 Jul;20(7):984-997. Epub 2019 Jun 4. Erratum in: Lancet Oncol. 2019 Jul;20(7):e346. link to original article contains protocol PubMed NCT02039726
Quizartinib monotherapy
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Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Cortes et al. 2019 (QuANTUM-R) | 2014-2017 | Phase III (E-switch-ooc) | Investigator's choice of: 1. LoDAC 2. MEC 3. FLAG-Ida |
Seems to have superior OS |
Note: Quizartinib is not yet approved in any domain.
Targeted therapy
- Quizartinib 60 mg PO once per day
Continued indefinitely
References
- QuANTUM-R: Cortes JE, Khaled S, Martinelli G, Perl AE, Ganguly S, Russell N, Krämer A, Dombret H, Hogge D, Jonas BA, Leung AY, Mehta P, Montesinos P, Radsak M, Sica S, Arunachalam M, Holmes M, Kobayashi K, Namuyinga R, Ge N, Yver A, Zhang Y, Levis MJ. Quizartinib versus salvage chemotherapy in relapsed or refractory FLT3-ITD acute myeloid leukaemia (QuANTUM-R): a multicentre, randomised, controlled, open-label, phase 3 trial. Lancet Oncol. 2019 Jul;20(7):984-997. link to original article PubMed NCT02039726
Prognosis
Prognosis in cytogenetically normal AML
- Seminal paper comparing the mutational status of NPM1, FLT3, CEBPA, MLL, and NRAS with clinical outcome: Schlenk RF, Döhner K, Krauter J, Fröhling S, Corbacioglu A, Bullinger L, Habdank M, Späth D, Morgan M, Benner A, Schlegelberger B, Heil G, Ganser A, Döhner H; German-Austrian Acute Myeloid Leukemia Study Group. Mutations and treatment outcome in cytogenetically normal acute myeloid leukemia. N Engl J Med. 2008 May 1;358(18):1909-18. link to original article PubMed
Investigational agents
These are drugs under study with at least some promising results for this disease.