Elliot Stieglitz, MD

Juvenile myelomonocytic leukemia (JMML) is a blood cancer that affects young children and is difficult to diagnose. Currently available therapies cure only half of patients, with some children experiencing an aggressive disease course while some children get better with very little treatment. We have now shown that the presence of secondary mutations at diagnosis predicts a poor outcome. We have also shown that DNA methylation profiling can identify patients most likely to experience favorable outcomes.

We recently completed a clinical trial through the Children's Oncology Group for patients with relapsed and refractory JMML. This trial tested the safety and efficacy of the oral MEK inhibitor, trametinib and met its primary objective. This trial was based on extensive pre-clinical testing and a strong genomic rationale. This trial marks an important step towards more effectively treating patients with Ras driven leukemia using targeted agents.

Our current trial (NCT05849662) risk-stratifies patients with newly diagnosed JMML to receive different intensities of therapy based on the number of mutations and DNA methylation signatures at diagnosis. It also combines trametinib with standard-of-care therapy for newly diagnosed patients.
Education
2014 - Pediatric Hematology Oncology Fellowship, University of California, San Francisco
2011 - Pediatric Residency, New York Presbyterian - Weill Cornell
MD, 2008 - School of Medicine, Stony Brook
Honors and Awards
  • R Accelerated Award, Alex's Lemonade Stand Foundation, 2024-2028
  • Translational Research Grant, Leukemia Lymphomoa Society, 2023-2027
  • Research Grant, Cookies for Kids' Cancer, 2023-2024
  • R37, NIH NCI, 2022-2027
  • Translational Research Award, V Foundation, 2019-2021
  • Research Award, Pediatric Cancer Research Foundation, 2019-2020
  • Individual Research Award, American Cancer Society, 2016-2017
  • Translational Research Training in Hematology, ASH/EHA, 2016
  • Young Investigator Award, Alex's Lemonade Stand Foundation, 2015-2017
  • Fellow in Clinical Research, St. Baldrick's Foundation Fellowship, 2014-2016
Websites
  1. Timothy T. Ferng, Samantha M. Pintar, Vanessa E. Kennedy, Shaheen Kabir, Theodore C. Tarver, Veronica Steri, Paul Phojanakong, Juwita Hübner, Carolina E. Morales, Jose M. Rivera, Aaron C. Logan, Benjamin Braun, Elliot Stieglitz, Luke A. Gilbert, Catherine C. Smith. CDK8 Inhibition Represses Monocyte-like Gene Expression in Acute Myeloid Leukemia Cells and Antagonizes In Vivo Resistance to FLT3 Inhibition. Blood 2024. PMID:


  2. Jeremy Grenier, Sarah K. Tasian, Elliot Stieglitz, Zied Abdullaev, Gerald Wertheim, Marilyn M. Li, Charles A Phillips. Effective Azacytidine Monotherapy in an Infant with Mosaic NRAS-Driven JMML. Blood 2024. PMID:


  3. Hiroyuki Takamatsu, Emilio Ramos, Bonell Patino-Escobar, Corynn Kasap, Adila Izgutdina, Juwita Werner, Akihito Inagaki, Takeshi Yoroidaka, Fernando Salangsang, Paul Phojanakong, Juan Antonio Camara Serrano, Veronica Steri, Elliot Stieglitz, Masaki Nagato, Satoshi Tatsuno, Arun P Wiita. HLA-Pan-DP CAR-T Demonstrates Cytotoxicity Against Multiple Hematological Malignancies. Blood 2024. PMID:


  4. Matthew Decker, Benjamin J Huang, Tim Ware, Christopher Boone, Michelle Tang, Aishwarya Ballapuram, Julia Ybarra, Katrine Taran, Marcos Amendáriz, Camille Leung, Max Harris, Elliot Stieglitz, Micah Niphakis, Benjamin Cravatt, Kevin Shannon. In Vivo and In Vitro Chemical Synthetic Lethality of ABHD17 and MEK Inhibition in NRAS -Mutant Acute Myeloid Leukemia. Blood 2024. PMID:


  5. Bogdan Popescu, Madison Piao, Sydney Abelson, Carolina E. Morales, Khadija Yousuf, Jose M. Rivera, Cheryl A C Peretz, Elliot Stieglitz, Catherine C. Smith. Preclinical Activity of RAS(ON) Multi-Selective Inhibitor RMC-7977 and Therapeutic Combinations in AML with Signaling Mutations. Blood 2024. PMID:


  6. Johns C, Erickson C, Jacobs A, Moon J, Baggott C, Dagher R, Nadel H, Balagtas J, Aftandilian C, Ramakrishna S, Lacayo N, Davis KL, Stieglitz E, Schultz L. Case report: sub-clinical extramedullary B-ALL in the setting of relapse following targeted therapy. Frontiers in immunology 2024. PMID: 39386214


  7. Stieglitz E, Lee AG, Angus SP, Davis C, Barkauskas DA, Hall D, Kogan SC, Meyer J, Rhodes SD, Tasian SK, Xuei X, Shannon K, Loh ML, Fox E, Weigel BJ. Efficacy of the Allosteric MEK Inhibitor Trametinib in Relapsed and Refractory Juvenile Myelomonocytic Leukemia: a Report from the Children's Oncology Group. Cancer discovery 2024. PMID: 38867349


  8. Zheng H, Zhao P, Tan Z, Yu WM, Werner J, Stieglitz E, Porter C, Chandrakasan S, Wechsler D, Mendez-Ferrer S, Qu CK. Prototypical innate immune mechanism hijacked by leukemia-initiating mutant stem cells for selective advantage and immune evasion in Ptpn11-associated juvenile myelomonocytic leukemia. Research square 2024. PMID: 39149498


  9. Wintering A, Hecht A, Meyer J, Wong EB, Hübner J, Abelson S, Feldman K, Kennedy VE, Peretz CAC, French DL, Maguire JA, Jobaliya C, Vasquez MR, Desai S, Dulman R, Nemecek E, Haines H, Hammad M, El Haddad A, Kogan SC, Abdullaev Z, Chehab FF, Tasian SK, Smith CC, Loh ML, Stieglitz E. LNK/SH2B3 as a novel driver in juvenile myelomonocytic leukemia. Haematologica 2023. PMID: 38152053


  10. Kasap C, Izgutdina A, Patiño-Escobar B, Kang A, Chilakapati N, Akagi N, Johnson H, Rashid T, Werner J, Barpanda A, Geng H, Lin YT, Rampersaud S, Gil-Alós D, Sobh A, Dupéré-Richer D, Wicaksono G, Kawehi Kelii KM, Dalal R, Ramos E, Vijayanarayanan A, Salangsang F, Phojanakong P, Serrano JAC, Zakraoui O, Tariq I, Steri V, Shanmugam M, Boise LH, Kortemme T, Stieglitz E, Licht JD, Karlon WJ, Barwick BG, Wiita AP. Targeting high-risk multiple myeloma genotypes with optimized anti-CD70 CAR-T cells. bioRxiv : the preprint server for biology 2024. PMID: 38463958




  11. William C. Temple, Adila Izgutdina, Matthew A. Nix, Benjamin J. Huang, Paul Phojanakong, Juan Antonio Camara Serrano, Fernando Salangsang, Veronica Steri, Simayijiang Xirenayi, Elliot Stieglitz, Arun P. Wiita. Preclinical Development and Characterization of Humanized Anti-CD72 Nanobody Chimeric Antigen Receptor T Cells for B Cell Malignancies. Transplantation and cellular therapy 2024. PMID:


  12. Verma A, Chi YY, Malvar J, Lamble A, Chaudhury S, Agarwal A, Li HT, Liang G, Leong R, Brown PA, Kaplan J, Schafer ES, Slone T, Pauly M, Chang BH, Stieglitz E, Wayne AS, Hijiya N, Bhojwani D. Nivolumab Plus 5-Azacitidine in Pediatric Relapsed/Refractory Acute Myeloid Leukemia (AML): Phase I/II Trial Results from the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) Consortium. Cancers 2024. PMID: 38339248



  13. Elliot Stieglitz, Alex Lee, Steven P. Angus, Christopher Davis, Donald Barkauskas, David Hall, Scott C. Kogan, Julia Meyer, Steven D. Rhodes, Xiaoling Xuei, Kevin Shannon, Mignon L. Loh, Elizabeth Fox, Brenda J. Weigel. Efficacy of the Allosteric MEK Inhibitor Trametinib in Relapsed and Refractory Juvenile Myelomonocytic Leukemia: A Report from the Children's Oncology Group. Blood 2023. PMID:


  14. Timothy T. Ferng, Samantha M. Pintar, Vanessa E. Kennedy, Theodore C. Tarver, Shaheen Kabir, Veronica Steri, Juan Antonio Camara Serrano, Fernando Salangsang, Paul Phojanakong, Juwita Hübner, Carolina E. Morales, Jose M. Rivera, Aaron C. Logan, Benjamin Braun, Elliot Stieglitz, Luke A. Gilbert, Catherine C. Smith. Mediator Kinase/CDK8 Inhibition As a Strategy to Improve FLT3 Inhibitor Activity in Acute Myeloid Leukemia. Blood 2023. PMID:



  15. Elliot Stieglitz, Yueh-Yun Chi, Bill H. Chang, Sarah K. Tasian, Marielle Yohe, Christopher C Dvorak, Erica Southworth, Janel R. Long-Boyle, Jessica Van Ziffle, Zied Abdullaev, Roy Leong, Alan S. Wayne, Deepa Bhojwani, Mignon L. Loh. Risk Stratified Treatment for Patients with Newly Diagnosed Juvenile Myelomonocytic Leukemia: A Phase 1/2 Non-Randomized Study of Trametinib and Azacitidine with or without Chemotherapy. Blood 2023. PMID:


  16. Cheryl A C Peretz, Elaine Tran, Bogdan Popescu, Vanessa E. Kennedy, Tanya Kumar, Lisa McGary, Natalie Tantisira, Eric Wong, Jose M. Rivera, Simayijiang Xirenayi, Elliot Stieglitz, Catherine C. Smith, Juwita Hübner. Threshold Attenuation of Perk Signaling Is Sufficient to Overcome RAS-Mediated FLT3 Inhibitor Resistance. Blood 2023. PMID:


  17. Temple WC, Nix MA, Naik A, Izgutdina A, Huang BJ, Wicaksono G, Phojanakong P, Serrano JAC, Young EP, Ramos E, Salangsang F, Steri V, Xirenayi S, Hermiston M, Logan AC, Stieglitz E, Wiita AP. Framework humanization optimizes potency of anti-CD72 nanobody CAR-T cells for B-cell malignancies. Journal for immunotherapy of cancer 2023. PMID: 38007238


  18. Popescu B, Stahlhut C, Tarver TC, Wishner S, Lee BJ, Peretz CAC, Luck C, Phojanakong P, Camara Serrano JA, Hongo H, Rivera JM, Xirenayi S, Chukinas JA, Steri V, Tasian SK, Stieglitz E, Smith CC. Allosteric SHP2 inhibition increases apoptotic dependency on BCL2 and synergizes with venetoclax in FLT3- and KIT-mutant AML. Cell reports. Medicine 2023. PMID: 37992684


  19. Pierpont EI, Bennett AM, Schoyer L, Stronach B, Anschutz A, Borrie SC, Briggs B, Burkitt-Wright E, Castel P, Cirstea IC, Draaisma F, Ellis M, Fear VS, Frone MN, Flex E, Gelb BD, Green T, Gripp KW, Khoshkhoo S, Kieran MW, Kleemann K, Klein-Tasman BP, Kontaridis MI, Kruszka P, Leoni C, Liu CZ, Merchant N, Magoulas PL, Moertel C, Prada CE, Rauen KA, Roelofs R, Rossignol R, Sevilla C, Sevilla G, Sheedy R, Stieglitz E, Sun D, Tiemens D, White F, Wingbermühle E, Wolf C, Zenker M, Andelfinger G. The 8th International RASopathies Symposium: Expanding research and care practice through global collaboration and advocacy. American journal of medical genetics. Part A 2023. PMID: 37969032


  20. Shumock SS, Temple WC, Marinoff A, Aaronson K, Southworth E, Xirenayi S, Lee AG, Leung SG, Sweet-Cordero EA, Hermiston M, Higham C, Stieglitz E. Pre-and post-HSCT use of TKI therapy for fusion-driven B-ALL: A case series of five pediatric, adolescent and young adult patients. Cancer reports (Hoboken, N.J.) 2023. PMID: 37933765


  21. Mandal K, Wicaksono G, Yu C, Adams JJ, Hoopmann MR, Temple WC, Izgutdina A, Escobar BP, Gorelik M, Ihling CH, Nix MA, Naik A, Xie WH, Hübner J, Rollins LA, Reid SM, Ramos E, Kasap C, Steri V, Serrano JAC, Salangsang F, Phojanakong P, McMillan M, Gavallos V, Leavitt AD, Logan AC, Rooney CM, Eyquem J, Sinz A, Huang BJ, Stieglitz E, Smith CC, Moritz RL, Sidhu SS, Huang L, Wiita AP. Structural surfaceomics reveals an AML-specific conformation of integrin ß2 as a CAR T cellular therapy target. Nature cancer 2023. PMID: 37904046


  22. Stieglitz E, Gu CJ, Richardson M, Kita R, Santaguida MT, Ali KA, Strachan DC, Dhar A, Yam G, Anderson W, Anderson E, Hübner J, Tasian SK, Loh ML, Lacher MD. Tretinoin Enhances the Effects of Chemotherapy in Juvenile Myelomonocytic Leukemia Using an Ex Vivo Drug Sensitivity Assay. JCO precision oncology 2023. PMID: 37944074


  23. Jeffrey Rubnitz, Ghayas C. Issa, Eytan M. Stein, Neerav Shukla, Wendy Stock, Andrius Žucenka, Sajad Khazal, Nandita Khera, Elliot Stieglitz, Jeremy Rubinstein, Galit Rosen, Rachel Ghiraldi, Huy Van Nguyen, Nicole Mcneer, C. Michel Zwaan. P561: REVUMENIB IN PATIENTS WITH ACUTE LEUKEMIAS: COMPASSIONATE USE PROGRAM EXPERIENCE. HemaSphere 2023. PMID:


  24. Juwita Huebner, Sherin Xirenayi, Jose Rivera, Bonell Patiño Escobar, Alex G Lee, Kamal Mandal, Ernesto Diaz-Flores, Arun Wiita, Elliot Stieglitz. P985: CLL-1 IS AN ADOPTIVE IMMUNOTHERAPY TARGET IN JUVENILE MYELOMONOCYTIC LEUKEMIA. HemaSphere 2023. PMID:


  25. Elliot Stieglitz, Christine Gu, Anukriti Dhar, Juwita Huebner, Kamran Ali, Markus Lacher. P998: TRETINOIN AND CALCITRIOL ENHANCE TREATMENT REGIMENS FOR JUVENILE MYELOMONOCYTIC LEUKEMIA IN EX VIVO DRUG SENSITIVITY ASSAYS. HemaSphere 2023. PMID:


  26. Marinoff AE, Aaronson K, Agrawal AK, Braun BS, Golden C, Huang BJ, Michlitsch J, Southworth E, Thrall A, Vo KT, Stieglitz E. Venetoclax in combination with chemotherapy as treatment for pediatric advanced hematologic malignancies. Pediatric blood & cancer 2023. PMID: 37036306


  27. Pasupuleti SK, Chao K, Ramdas B, Kanumuri R, Palam LR, Liu S, Wan J, Annesley C, Loh M, Stieglitz E, Burke MJ, Kapur R. Potential clinical use of Azacitidine and MEK inhibitor combination therapy in PTPN11-mutated Juvenile myelomonocytic leukemia. Molecular therapy : the journal of the American Society of Gene Therapy 2023. PMID: 36739480



  28. Astrid Wintering, Anna Hecht, Julia Meyer, Eric Brandon Wong, Deborah L French, Jean Ann Maguire, Chintan Jobaliya, Marta Rojas Vasquez, Sunil J. Desai, Robin Yates Dulman, Eneida R. Nemecek, Farid F Chehab, Mignon L. Loh, Elliot Stieglitz. LNK/SH2B3 As a Novel Driver in Juvenile Myelomonocytic Leukemia. Blood 2022. PMID:


  29. de Blank PMK, Gross AM, Akshintala S, Blakeley JO, Bollag G, Cannon A, Dombi E, Fangusaro J, Gelb BD, Hargrave D, Kim A, Klesse LJ, Loh M, Martin S, Moertel C, Packer R, Payne JM, Rauen KA, Rios JJ, Robison N, Schorry EK, Shannon K, Stevenson DA, Stieglitz E, Ullrich NJ, Walsh KS, Weiss BD, Wolters PL, Yohay K, Yohe ME, Widemann BC, Fisher MJ. MEK Inhibitors for Neurofibromatosis Type 1 Manifestations: Clinical Evidence and Consensus. Neuro-oncology 2022. PMID: 35788692


  30. Niemeyer CM, Rudelius M, Shimamura A, Flotho C, Hasle H, Stieglitz E, Strahm B, Godley LA, Weinberg OK, Orazi A, Calvo KR. Classification of rare pediatric myeloid neoplasia-Quo vadis? Leukemia 2022. PMID: 36271151


  31. Pearson AD, Allen C, Fangusaro J, Hutter C, Witt O, Weiner S, Reaman G, Russo M, Bandopadhayay P, Ahsan S, Barone A, Barry E, de Rojas T, Fisher M, Fox E, Bender JG, Gore L, Hargrave D, Hawkins D, Kreider B, Langseth AJ, Lesa G, Ligas F, Marotti M, Marshall LV, Nasri K, Norga K, Nysom K, Pappo A, Rossato G, Scobie N, Smith M, Stieglitz E, Weigel B, Weinstein A, Viana R, Karres D, Vassal G. Paediatric Strategy Forum for medicinal product development in mitogen-activated protein kinase pathway inhibitors: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. European journal of cancer (Oxford, England : 1990) 2022. PMID: 36335782


  32. Imaizumi T, Meyer J, Wakamatsu M, Kitazawa H, Murakami N, Okuno Y, Yoshida T, Sajiki D, Hama A, Kojima S, Takahashi Y, Loh M, Stieglitz E, Muramatsu H. Clinical parameter-based prediction of DNA methylation classification generates a prediction model of prognosis in patients with juvenile myelomonocytic leukemia. Scientific Reports 2022. PMID: 36042365


  33. Smith SM, Lee A, Tong S, Leung S, Hongo H, Rivera J, Sweet-Cordero A, Michlitsch J, Stieglitz E. Detection of a GLIS3 fusion in an infant with AML refractory to chemotherapy. Cold Spring Harbor molecular case studies 2022. PMID: 35927023


  34. Lu KL, Menke JR, Ng D, Ruiz-Cordero R, Marinoff A, Stieglitz E, Gollapudi S, Singh K, Ohgami RS, Vohra P. Cytomorphologic features of pediatric-type follicular lymphoma on fine needle aspiration biopsy: case series and a review of the literature. Journal of the American Society of Cytopathology 2022. PMID: 35843844


  35. Ramdas B, Yuen LD, Palam LR, Patel R, Pasupuleti SK, Jideonwo V, Zhang J, Maguire C, Wong E, Kanumuri R, Zhang C, Sandusky G, Chan RJ, Zhang C, Stieglitz E, Haneline L, Kapur R. Inhibition of BTK and PI3Kδ impairs the development of human JMML stem and progenitor cells. 2022. PMID: 35443935


  36. Miltiadous O, Petrova-Drus K, Kaicker S, Mathew S, Kluk M, Geyer JT, Rodriguez-Sanchez MI, Bouvier N, Inghirami GG, Stieglitz E, Nafa K, Benayed R, Richardson M, Anderson W, Benhamida J, You D, Londono D, Kung AL, Prockop S, Roshal M, Zhang Y, Shukla NN. Successful treatment and integrated genomic analysis of an infant with FIP1L1-RARA fusion-associated myeloid neoplasm. Blood advances 2021. PMID: 34551074


  37. Yan Y, Dong L, Chen C, Bunting KD, Li Q, Stieglitz E, Loh ML, Qu CK. JMML tumor cells disrupt normal hematopoietic stem cells by imposing inflammatory stress through overproduction of IL-1β. Blood advances 2021. PMID: 34555844


  38. Hecht A, Meyer JA, Behnert A, Wong E, Chehab F, Olshen A, Hechmer A, Aftandilian C, Bhat R, Choi SW, Chonat S, Farrar JE, Fluchel M, Frangoul H, Han JH, Kolb EA, Kuo DJ, MacMillan ML, Maese L, Maloney KW, Narendran A, Oshrine B, Schultz KR, Sulis ML, Van Mater D, Tasian SK, Hofmann WK, Loh ML, Stieglitz E. Molecular and phenotypic diversity of CBL-mutated juvenile myelomonocytic leukemia. Haematologica 2020. PMID: 33375775


  39. Kitazawa H, Okuno Y, Muramatsu H, Aoki K, Murakami N, Wakamatsu M, Suzuki K, Narita K, Kataoka S, Ichikawa D, Hamada M, Taniguchi R, Kawashima N, Nishikawa E, Narita A, Nishio N, Hama A, Loh ML, Stieglitz E, Kojima S, Takahashi Y. A simple and robust methylation test for risk stratification of patients with juvenile myelomonocytic leukemia. Blood advances 2021. PMID: 34580726


  40. Wintering A, Smith S, Fuh B, Rangaswami A, Dahl G, Chien M, Gruber TA, Dang J, Li LS, Lenzen A, Savelli S, Dvorak CC, Agrawal AK, Stieglitz E. Therapy-related myeloid neoplasms resembling juvenile myelomonocytic leukemia: a case series and review of the literature. 2021. PMID: 34939322



  41. Bogdan Popescu, Carlos Stahlhut, Theodore C. Tarver, Timothy T. Ferng, Cheryl Peretz, Sydney Wishner, Paul Phojanakong, Veronica Steri, Eric B. Wong, Jose M. Rivera, Elliot Stieglitz, Catherine C. Smith. Allosteric SHP2 Inhibitor RMC4550 Synergizes with Venetoclax in FLT3 and KIT Mutant Acute Myeloid Leukemia. 2021. PMID:


  42. Elliot Stieglitz, Mignon L. Loh, Julia Meyer, Chujing Zhang, Donald A Barkauskas, David Hall, Elizabeth Fox, Brenda J. Weigel. MEK Inhibition Demonstrates Activity in Relapsed, Refractory Patients with Juvenile Myelomonocytic Leukemia: Results from COG Study ADVL1521. 2021. PMID:


  43. Santhosh Kumar Pasupuleti, Baskar Ramdas, Kai Yang, Chujing Zhang, Elliot Stieglitz, Reuben Kapur. Targeting M2-Tumor Associated Macrophages By Arginase-1 and PD-L1 in Regulating Juvenile Myelomonocytic Leukemia (JMML) Development and Relapse. 2021. PMID:


  44. Morales CE, Stieglitz E, Kogan SC, Loh ML, Braun BS. Nf1 and Sh2b3 mutations cooperate in vivo in a mouse model of juvenile myelomonocytic leukemia. Blood advances 2021. PMID: 34464969


  45. Behnert A, Lee AG, Young EP, Breese MR, Leung SG, Behroozfard I, Maruffi M, Sweet-Cordero EA, Dvorak CC, Chu J, Stieglitz E. NUP98-NSD1 Driven MDS/MPN in Childhood Masquerading as JMML. Journal of pediatric hematology/oncology 2020. PMID: 32815876


  46. Behnert A, Meyer J, Parsa JY, Hechmer A, Loh ML, Olshen A, de Smith AJ, Stieglitz E. Exploring the genetic and epigenetic origins of juvenile myelomonocytic leukemia using newborn screening samples. Leukemia 2021. PMID: 34183765


  47. Nix MA, Mandal K, Geng H, Paranjape N, Lin YT, Rivera JM, Marcoulis M, White KL, Whitman JD, Bapat SP, Parker KR, Ramirez J, Deucher A, Phojanokong P, Steri V, Fattahi F, Hann BC, Satpathy AT, Manglik A, Stieglitz E, Wiita AP. Surface Proteomics Reveals CD72 as a Target for In Vitro-Evolved Nanobody-Based CAR-T Cells in KMT2A/MLL1-Rearranged B-ALL. Cancer discovery 2021. PMID: 33727310


  48. Kennedy AL, Myers KC, Bowman J, Gibson CJ, Camarda ND, Furutani E, Muscato GM, Klein RH, Ballotti K, Liu S, Harris CE, Galvin A, Malsch M, Dale D, Gansner JM, Nakano TA, Bertuch A, Vlachos A, Lipton JM, Castillo P, Connelly J, Churpek J, Edwards JR, Hijiya N, Ho RH, Hofmann I, Huang JN, Keel S, Lamble A, Lau BW, Norkin M, Stieglitz E, Stock W, Walkovich K, Boettcher S, Brendel C, Fleming MD, Davies SM, Weller EA, Bahl C, Carter SL, Shimamura A, Lindsley RC. Distinct genetic pathways define pre-malignant versus compensatory clonal hematopoiesis in Shwachman-Diamond syndrome. Nature communications 2021. PMID: 33637765


  49. Pikman Y, Tasian SK, Sulis ML, Stevenson K, Blonquist TM, Apsel Winger B, Cooper TM, Pauly M, Maloney KW, Burke MJ, Brown PA, Gossai N, McNeer JL, Shukla NN, Cole PD, Kahn JM, Chen J, Barth MJ, Magee JA, Gennarini L, Adhav AA, Clinton CM, Ocasio-Martinez N, Gotti G, Li Y, Lin S, Imamovic A, Tognon CE, Patel T, Faust HL, Contreras CF, Cremer A, Cortopassi WA, Garrido Ruiz D, Jacobson MP, Dharia NV, Su A, Robichaud AL, Saur Conway A, Tarlock K, Stieglitz E, Place AE, Puissant A, Hunger SP, Kim AS, Lindeman NI, Gore L, Janeway KA, Silverman LB, Tyner JW, Harris MH, Loh ML, Stegmaier K. Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium. Cancer discovery 2021. PMID: 33563661



  50. Alyssa L. Kennedy, Kasiani C. Myers, James Bowman, Christopher J. Gibson, Gwen M. Muscato, Robert Klein, Kaitlyn Ballotti, Nicholas Camarda, Elissa M. Furutani, Chad E. Harris, Shanshan Liu, Ashley Galvin, Maggie Malsch, David C. Dale, John M Gansner, Taizo A. Nakano, Alison A. Bertuch, Adrianna Vlachos, Jeff H. Lipton, Paul Castillo, James Connelly, John Edwards, Nobuko Hijiya, Richard Ho, Inga Hofmann, James N Huang, Sioban B. Keel, Adam J. Lamble, Bonnie W Lau, Kelly J. Walkovich, Maxim Norkin, Wendy Stock, Steffen Boettcher, Christian Brendel, Elliot Stieglitz, Mark D. Fleming, Stella M. Davies, Edie A. Weller, Chris Bahl, Scott L. Carter, Akiko Shimamura, R. Coleman Lindsley. Distinct Genetic Pathways Define Leukemia Predisposition Versus Adaptive Clonal Hematopoiesis in Shwachman-Diamond Syndrome. Blood 2020. PMID:


  51. Astrid Behnert, Julia Meyer, Jahan-Yar Parsa, Aaron Hechmer, Mignon L. Loh, Adam B. Olshen, Adam J. de Smith, Elliot Stieglitz. Genetic Alterations Precede DNA Methylation Changes in Juvenile Myelomonocytic Leukemia. Blood 2020. PMID:


  52. Levinson A, Lee AG, Martell HJ, Breese MR, Zaloudek C, Van Ziffle J, Laguna B, Leung SG, Chen MD, Chen LM, Pfeil J, Ladwig NR, Shah AT, Behroozfard I, Rao AA, Salama SR, Sweet-Cordero EA, Stieglitz E. Complete Response to PD-1 Inhibition in an Adolescent With Relapsed Clear Cell Adenocarcinoma of the Cervix Predicted by Neoepitope Burden and APOBEC Signature. JCO precision oncology 2020. PMID: 33283136


  53. Schönung M, Meyer J, Nöllke P, Olshen AB, Hartmann M, Murakami N, Wakamatsu M, Okuno Y, Plass C, Loh ML, Niemeyer CM, Muramatsu H, Flotho C, Stieglitz E, Lipka DB. International Consensus Definition of DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia. Clinical cancer research : an official journal of the American Association for Cancer Research 2020. PMID: 33139265



  54. Fabrizio VA, Kernan NA, Boulad F, Cancio M, Allen J, Higman M, Margossian SP, Mauguen A, Prockop S, Scaradavou A, Shah N, Spitzer B, Stieglitz E, Yeager N, O'Reilly RJ, Brentjens RJ, Jan Boelens J, Curran KJ. Low toxicity and favorable overall survival in relapsed/refractory B-ALL following CAR T cells and CD34-selected T-cell depleted allogeneic hematopoietic cell transplant. Bone marrow transplantation 2020. PMID: 32390002


  55. Dueck ME, Lin R, Zayac A, Gallagher S, Chao AK, Jiang L, Datwani SS, Hung P, Stieglitz E. Precision cancer monitoring using a novel, fully integrated, microfluidic array partitioning digital PCR platform. Scientific Reports 2019. PMID: 31862911


  56. Friend BD, Bailey-Olson M, Melton A, Shimano KA, Kharbanda S, Higham C, Winestone LE, Huang J, Stieglitz E, Dvorak CC. The impact of total body irradiation-based regimens on outcomes in children and young adults with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation. Pediatric blood & cancer 2019. PMID: 31724815


  57. Julia Meyer, Maximillian Schönung, Christian Flotho, Adam B. Olshen, Mark Hartmann, Christoph Plass, Yusuke Okuno, Yoshiyuki Takahashi, Norihiro Murakami, Manabu Wakamatsu, Mignon L. Loh, Charlotte M. Niemeyer, Hideki Muramatsu, Daniel B. Lipka, Elliot Stieglitz. DNA Methylation As a Biomarker of Outcome in JMML: An International Effort Towards Clinical Implementation. Blood 2019. PMID:


  58. Matthew Nix, Yu-Hsiu T. Lin, Huimin Geng, Makeba Marcoulis, Paul Phojanakong, Donghui Wang, Veronica Steri, Jeffrey Whitman, Sagar Bapat, Elliot Stieglitz, Anne Deucher, Kristie L White, Byron C Hann, Arun P. Wiita. In Vitro-Selected Nanobody-Based Cellular Therapy Targeting CD72 for Treatment of Refractory B-Cell Malignancies. Blood 2019. PMID:


  59. Chao AK, Meyer JA, Lee AG, Hecht A, Tarver T, Van Ziffle J, Koegel AK, Golden C, Braun BS, Sweet-Cordero EA, Smith CC, Dvorak CC, Loh ML, Stieglitz E. Fusion driven JMML: a novel CCDC88C-FLT3 fusion responsive to sorafenib identified by RNA sequencing. Leukemia 2019. PMID: 31511612


  60. Hecht A, Meyer J, Chehab FF, White KL, Magruder K, Dvorak CC, Loh ML, Stieglitz E. Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia. Pediatric blood & cancer 2019. PMID: 31347788


  61. de Smith AJ, Lavoie G, Walsh KM, Aujla S, Evans E, Hansen HM, Smirnov I, Kang AY, Zenker M, Ceremsak JJ, Stieglitz E, Muskens IS, Roberts W, McKean-Cowdin R, Metayer C, Roux PP, Wiemels JL. Predisposing germline mutations in high hyperdiploid acute lymphoblastic leukemia in children. Genes, chromosomes & cancer 2019. PMID: 31102422


  62. Capri Y, Flex E, Krumbach OHF, Carpentieri G, Cecchetti S, Lißewski C, Rezaei Adariani S, Schanze D, Brinkmann J, Piard J, Pantaleoni F, Lepri FR, Goh ES, Chong K, Stieglitz E, Meyer J, Kuechler A, Bramswig NC, Sacharow S, Strullu M, Vial Y, Vignal C, Kensah G, Cuturilo G, Kazemein Jasemi NS, Dvorsky R, Monaghan KG, Vincent LM, Cavé H, Verloes A, Ahmadian MR, Tartaglia M, Zenker M. Activating Mutations of RRAS2 Are a Rare Cause of Noonan Syndrome. American journal of human genetics 2019. PMID: 31130282


  63. Iacobucci I, Wen J, Meggendorfer M, Choi JK, Shi L, Pounds SB, Carmichael CL, Masih KE, Morris SM, Lindsley RC, Janke LJ, Alexander TB, Song G, Qu C, Li Y, Payne-Turner D, Tomizawa D, Kiyokawa N, Valentine M, Valentine V, Basso G, Locatelli F, Enemark EJ, Kham SKY, Yeoh AEJ, Ma X, Zhou X, Sioson E, Rusch M, Ries RE, Stieglitz E, Hunger SP, Wei AH, To LB, Lewis ID, D'Andrea RJ, Kile BT, Brown AL, Scott HS, Hahn CN, Marlton P, Pei D, Cheng C, Loh ML, Ebert BL, Meshinchi S, Haferlach T, Mullighan CG. Genomic subtyping and therapeutic targeting of acute erythroleukemia. Nature genetics 2019. PMID: 30926971


  64. Maximilian Schoenung, Elliot Stieglitz, Hideki Muramatsu, Norihiro Murakami, Mark Hartmann, Manuel Wiesenfarth, Yusuke Okuno, Adam B. Olshen, Christoph Plass, Mignon L. Loh, Charlotte Niemeyer, Christian Flotho, Daniel B. Lipka. DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia: An International Collaborative Analysis and Development of a Common Diagnostic Platform. Blood 2018. PMID:


  65. Adam J de Smith, Geneviève Lavoie, Kyle M Walsh, Sumeet Aujla, Erica Evans, Helen M Hansen, Ivan Smirnov, Alice Y Kang, Martin Zenker, John J Ceremsak, Elliot Stieglitz, Roberta McKean-Cowdin, Catherine Metayer, Philippe P Roux, Joseph L Wiemels. Germline GAB2 Mutations in Childhood Acute Lymphoblastic Leukemia. Blood 2018. PMID:


  66. Andrew E. Place, Traci M. Blonquist, Elliot Stieglitz, Todd M Cooper, Lia Gore, Richard Aplenc, Mignon L. Loh, Melinda Pauly, Rachel E. Rau, Michael J. Burke, Julia Etchin, A. Thomas Look, Maria Luisa Sulis, Lewis B. Silverman. Phase I Study of the Selinexor in Relapsed/Refractory Childhood Acute Leukemia. Blood 2018. PMID:


  67. Kong G, You X, Wen Z, Chang YI, Qian S, Ranheim EA, Letson C, Zhang X, Zhou Y, Liu Y, Rajagopalan A, Zhang J, Stieglitz E, Loh M, Hofmann I, Yang D, Zhong X, Padron E, Zhou L, Pear WS, Zhang J. Downregulating Notch counteracts KrasG12D-induced ERK activation and oxidative phosphorylation in myeloproliferative neoplasm. Leukemia 2018. PMID: 30206308



  68. Dvorak CC, Satwani P, Stieglitz E, Cairo MS, Dang H, Pei Q, Gao Y, Wall D, Mazor T, Olshen AB, Parker JS, Kahwash S, Hirsch B, Raimondi S, Patel N, Skeens M, Cooper T, Mehta PA, Grupp SA, Loh ML. Disease burden and conditioning regimens in ASCT1221, a randomized phase II trial in children with juvenile myelomonocytic leukemia: A Children's Oncology Group study. Pediatric blood & cancer 2018. PMID: 29528181


  69. Stieglitz E, Mazor T, Olshen AB, Geng H, Gelston LC, Akutagawa J, Lipka DB, Plass C, Flotho C, Chehab FF, Braun BS, Costello JF, Loh ML. Genome-wide DNA methylation is predictive of outcome in juvenile myelomonocytic leukemia. Nature communications 2017. PMID: 29259179


  70. Elisa Dorantes-Acosta, Hui Huang, Sara P. Garcia, Elliot Stieglitz, Mignon Loh, Guo-Cheng Yuan, Alan B. Cantor. Abstract 26: RUNX1 as a transcriptional target of activated Shp2 (PTPN11) in juvenile myelomonocytic leukemia. Leukemia 2017. PMID:


  71. Alan Cantor, Elisa Dorantes-Costa, Hui Huang, Sara Garcia, Elliot Stieglitz, Mignon Loh, Guo-Cheng Yuan. Dysregulation of the transcription factor runx1 in juvenile myelomonocytic leukemia. Experimental hematology 2017. PMID:


  72. Yoshimi A, Balasis ME, Vedder A, Feldman K, Ma Y, Zhang H, Lee SC, Letson C, Niyongere S, Lu SX, Ball M, Taylor J, Zhang Q, Zhao Y, Youssef S, Chung YR, Zhang XJ, Durham BH, Yang W, List AF, Loh ML, Klimek V, Berger MF, Stieglitz E, Padron E, Abdel-Wahab O. Robust patient-derived xenografts of MDS/MPN overlap syndromes capture the unique characteristics of CMML and JMML. Blood 2017. PMID: 28576879



  73. Whale AS, Devonshire AS, Karlin-Neumann G, Regan J, Javier L, Cowen S, Fernandez-Gonzalez A, Jones GM, Redshaw N, Beck J, Berger AW, Combaret V, Dahl Kjersgaard N, Davis L, Fina F, Forshew T, Fredslund Andersen R, Galbiati S, González Hernández Á, Haynes CA, Janku F, Lacave R, Lee J, Mistry V, Pender A, Pradines A, Proudhon C, Saal LH, Stieglitz E, Ulrich B, Foy CA, Parkes H, Tzonev S, Huggett JF. International Interlaboratory Digital PCR Study Demonstrating High Reproducibility for the Measurement of a Rare Sequence Variant. 2017. PMID: 27935690


  74. Elisa Dorantes Acosta, Hui Huang, Sara P. Garcia, Elliot Stieglitz, Mignon L. Loh, G C Yuan, Alan B. Cantor. RUNX1 Is a Candidate Transcriptional Effector in Juvenile Myelomonocytic Leukemia. 2016. PMID:


  75. Ilaria Iacobucci, Ji Wen, Manja Meggendorfer, Catherine Carmichael, John K Choi, Katherine Masih, Yongjin Li, Debbie Payne, Daisuke Tomizawa, Nobutaka Kiyokawa, Laura J. Janke, Thomas B. Alexander, Stanley Pounds, Lei Shi, Marcus Valentine, Giuseppe Basso, Franco Locatelli, Shirley Kham Kow Yin, Allen Yeoh Eng Juh, Ries E Rhonda, Elliot Stieglitz, Andrew Wei, Ian D Lewis, Richard D'Andrea, Benjamin T. Kile, Jinghui Zhang, Mignon L. Loh, Soheil Meshinchi, Torsten Haferlach, Charles Mullighan. The Genomic Landscape of Childhood and Adult Acute Erythroid Leukemia. 2016. PMID:


  76. Ceremsak JJ, Yu A, Esquivel E, Lissewski C, Zenker M, Loh ML, Stieglitz E. Germline RRAS2 mutations are not associated with Noonan syndrome. Journal of medical genetics 2016. PMID: 27055474


  77. Stieglitz E, Loh ML. Pediatric MDS: GATA screen the germline. Blood 2016. PMID: 26989184


  78. Stieglitz E, Taylor-Weiner AN, Chang TY, Gelston LC, Wang YD, Mazor T, Esquivel E, Yu A, Seepo S, Olsen SR, Rosenberg M, Archambeault SL, Abusin G, Beckman K, Brown PA, Briones M, Carcamo B, Cooper T, Dahl GV, Emanuel PD, Fluchel MN, Goyal RK, Hayashi RJ, Hitzler J, Hugge C, Liu YL, Messinger YH, Mahoney DH, Monteleone P, Nemecek ER, Roehrs PA, Schore RJ, Stine KC, Takemoto CM, Toretsky JA, Costello JF, Olshen AB, Stewart C, Li Y, Ma J, Gerbing RB, Alonzo TA, Getz G, Gruber TA, Golub TR, Stegmaier K, Loh ML. Corrigendum: The genomic landscape of juvenile myelomonocytic leukemia. Nature genetics 2016. PMID: 26711114


  79. Stieglitz E, Ward AF, Gerbing RB, Alonzo TA, Arceci RJ, Liu YL, Emanuel PD, Widemann BC, Cheng JW, Jayaprakash N, Balis FM, Castleberry RP, Bunin NJ, Loh ML, Cooper TM. Phase II/III trial of a pre-transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: a report from the Children's Oncology Group. Pediatric blood & cancer 2014. PMID: 25704135


  80. Elliot Stieglitz, Camille B Troup, Laura C Gelston, Eric D Chow, Kristie B Yu, Jon Akutagawa, Amaro N Taylor-Weiner, Y. Lucy Liu, Peter D Emanuel, Benjamin S Braun, Robert B. Gerbing, Todd A. Alonzo, Mignon L. Loh. Subclonal Mutations in SETBP1 Predict Relapse in Juvenile Myelomonocytic Leukemia. 2014. PMID:


  81. Stieglitz E, Troup CB, Gelston LC, Haliburton J, Chow ED, Yu KB, Akutagawa J, Taylor-Weiner AN, Liu YL, Wang YD, Beckman K, Emanuel PD, Braun BS, Abate A, Gerbing RB, Alonzo TA, Loh ML. Subclonal mutations in SETBP1 confer a poor prognosis in juvenile myelomonocytic leukemia. Blood 2014. PMID: 25395418


  82. Stieglitz E, Liu YL, Emanuel PD, Castleberry RP, Cooper TM, Shannon KM, Loh ML. Mutations in GATA2 are rare in juvenile myelomonocytic leukemia. Blood 2014. PMID: 24578498


  83. Stieglitz E, Hsiang MS, Simko JP, Hirose S, Goldsby RE. Pulmonary coccidiomycosis masquerading as refractory metastatic Ewing sarcoma. Journal of pediatric hematology/oncology 2014. PMID: 23588326


  84. Elliot Stieglitz, Y. Lucy Liu, Peter D. Emanuel, Robert P. Castleberry, Todd Michael Cooper, Kevin Shannon, Mignon L. Loh. Mutations In GATA2 Are Rare In Juvenile Myelomonocytic Leukemia. 2013. PMID: