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Jeffrey E. Rubnitz, MD, PhD
Jeffrey E. Rubnitz, MD, PhD


MD – University of California at San Diego (1988)
PhD – University of California at San Diego (1987)

Research Interests

 I joined the Leukemia/Lymphoma Division in 1995 and have focused my efforts on improving the outcome of children with acute myeloid leukemia (AML). I was the principal investigator of three multi-institutional clinical trials (AML02, AML08, and AML16) for pediatric patients with newly diagnosed AML and I am currently the principal investigator of our AML23 trial. The recently activated AML23 trial is the first trial to use venetoclax in pediatric patients with newly diagnosed AML. In addition, I developed and led SELHEM, the first trial of selinexor in pediatric patients, and VENAML, the first pediatric trial of venetoclax. I am currently leading the SELCLAX trial, which is testing the novel combination of venetoclax plus selinexor, and RAVAML, which will be the first pediatric trial of revumenib + azacitidine + venetoclax.

Selected Publications

Karol SE, Coustan-Smith E, Pounds SB, Wang L, Inaba H, Ribeiro RC, Pui CH, Klco JM, Rubnitz JE. Clinical Impact of Minimal Residual Disease in Blood and Bone Marrow of Children with Acute Myeloid Leukemia. Blood Adv 7:3651-3657, 2023

Zarnegar-Lumley S, Caldwell K, Rubnitz JE. Relapsed Acute Myeloid Leukemia in Children and Adolescents: Current Treatment Options and Future Strategies. Leukemia 36:1951-1960, 2022.

Rubnitz JE, Kaspers GJL. How I treat pediatric acute myeloid leukemia. Blood 138:1009-1018, 2021.

Jeha S, Choi J, Roberts KG, Pei D, Coustan-Smith E, Inaba H, Rubnitz JE, Ribeiro RC, Gruber T, Raimondi SC, Karol SE, Qu C, Brady SW, Gu Z, Yang JJ, Cheng C, Downing JR, Evans WE, Relling MV, Campana D, Mullighan CG, Pui CH. Clinical significance of novel subtypes of acute lymphoblastic leukemia in the context of minimal residual disease-directed therapy. Blood Cancer Discovery 2:326-337, 2021.

Caldwell KJ, Budhraja A, Opferman JT, Pui CH, Klco JM, Rubnitz JE. Activity of venetoclax against relapsed acute undifferentiated leukemia. Cancer 127:2608-2611, 2021.

Karol SE, Alexander TB, Budhraja A, Pounds SB, Canavera K, Wang L, Wolf J, Klco JM, Mead PE, Das Gupta S, Kim SY, Salem AH, Palenski T, Lacayo NJ, Pui CH, Opferman JT, Rubnitz JE. Venetoclax in combination with cytarabine with or without idarubicin in children with relapsed or refractory acute myeloid leukaemia: a phase 1, dose-escalation study. Lancet Oncol 21:551–60, 2020.

Rubnitz JE, Lacayo NJ, Inaba H, Heym K, Ribeiro RC, Taub J, McNeer J, Degar B, Schiff D, Yeoh AE, Coustan-Smith E, Wang L, Triplett B, Raimondi SC, Klco J, Choi J, Pounds S, Pui C-H. Clofarabine can replace anthracyclines and etoposide in remission induction therapy for childhood acute myeloid leukemia: the AML08 multicenter, randomized phase 3 trial. J Clin Oncol 37:2072-2081, 2019.

Nguyen R, Wu H, Pounds S, Inaba H, Ribeiro RC, Cullins D, Rooney B, Bell T, Lacayo NJ, Heym K, Degar B, Schiff D, Janssen WE, Triplett B, Pui CH, Leung W, Rubnitz JE.  A phase II clinical trial of adoptive transfer of haploidentical natural killer cells for consolidation therapy of pediatric acute myeloid leukemia. J Immunother Cancer 7:81, 2019.

Alexander TB, Lacayo NJ, Choi J, Ribeiro RC, Pui C-H, Rubnitz JE. Phase I Study of Selinexor, a Selective Inhibitor of Nuclear Export, in Combination with Fludarabine and Cytarabine in Pediatric Relapsed or Refractory Acute Leukemia. J Clin Oncol 34:4094-4101, 2016.

Rubnitz JE, Inaba H, Leung W, Pounds S, Cao X, Campana D, Ribeiro RC, Pui CH. Definition of cure in childhood acute myeloid leukemia. Cancer 120:2490-2496, 2014.

Inaba H, Gaur AH, Cao X, Flynn PM, Pounds SB, Avutu V, Marszal LN, Howard SC, Pui CH, Ribeiro RC, Hayden RT, Rubnitz JE. Feasibility, efficacy, and adverse effects of outpatient antibacterial prophylaxis in children with acute myeloid leukemia. Cancer 120:1985–1992, 2014.

Rubnitz JE, Inaba H, Dahl G, Ribeiro RC, Bowman WP, Taub J, Pounds S, Razzouk BI, Lacayo NJ, Cao X, Meshinchi S, Degar B, Airewele G, Raimondi SC, Onciu M, Coustan-Smith E, Downing JR, Leung W, Pui C-H, Campana D. Minimal Residual Disease–Directed Therapy for Childhood Acute Myeloid Leukemia: Results of the AML02 Multicenter Trial. The Lancet Oncology 11:543-552, 2010.

Last update: September 2023