Elliot Stieglitz, MD

Title(s)Associate Professor, Pediatrics
SchoolSchool of Medicine
Address1450 Third Street, #243
San Francisco CA 94158
ORCID ORCID Icon0000-0001-7032-4623 Additional info
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    Collapse Biography 
    Collapse Education and Training
    Stony Brook MD2008School of Medicine
    New York Presbyterian - Weill Cornell2011Pediatric Residency
    University of California, San Francisco2014Pediatric Hematology Oncology Fellowship
    Collapse Awards and Honors
    NIH NCI2022  - 2027R37
    Alex's Lemonade Stand Foundation 2024  - 2028R Accelerated Award
    Leukemia Lymphomoa Society2023  - 2027Translational Research Grant
    Cookies for Kids' Cancer2023  - 2024Research Grant
    V Foundation2019  - 2021Translational Research Award
    Pediatric Cancer Research Foundation2019  - 2020Research Award
    ASH/EHA2016  - 2016Translational Research Training in Hematology
    Alex's Lemonade Stand Foundation2015  - 2017Young Investigator Award
    American Cancer Society2016  - 2017Individual Research Award
    St. Baldrick's Foundation Fellowship2014  - 2016Fellow in Clinical Research

    Collapse Overview 
    Collapse Overview
    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 conducted 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 next trial will risk-stratify patients with newly diagnosed JMML to receive different therapies tailored to the risk of relapse predicted by both the number of mutations and DNA methylation signatures at diagnosis.

    Collapse Research 
    Collapse Research Activities and Funding
    Implementation of an interventional, risk-adapted clinical trial for children with newly diagnosed juvenile myelomonocytic leukemia.
    NIH R37CA266550Aug 1, 2022 - Jul 31, 2027
    Role: Principal Investigator
    Targeting SETBP1 in Myeloproliferative Disorders
    NIH K08HL135434Jan 1, 2017 - Dec 31, 2021
    Role: Principal Investigator

    Collapse ORNG Applications 
    Collapse Clinical Trials
    Collapse Featured Publications

    Collapse Bibliographic 
    Collapse Publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Targeting high-risk multiple myeloma genotypes with optimized anti-CD70 CAR-T cells. bioRxiv. 2024 Feb 28. 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. PMID: 38463958; PMCID: PMC10925123.
      View in: PubMed   Mentions:
    2. Risk-stratified Treatment for Pediatric Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia. The Hematologist. 2024 Feb 21; 21(2). Stieglitz SE, Pommert PL. .
      View in: Publisher Site   Mentions:
    3. Desensitization using pegaspargase in the era of commercially available Erwinia: A single-institution report on efficacy, cost, and resource utilization. Pediatr Blood Cancer. 2024 Apr; 71(4):e30891. Feldman K, Aaronson K, Gu T, Ige K, Southworth E, Sanchez L, Stieglitz E. PMID: 38311802.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    4. Preclinical Development and Characterization of Humanized Anti-CD72 Nanobody Chimeric Antigen Receptor T Cells for B Cell Malignancies. Transplantation and Cellular Therapy. 2024 Feb 1; 30(2):s174-s175. Temple TW, Izgutdina IA, Nix NM, Huang HB, Phojanakong PP, Serrano SJ, Salangsang SF, Steri SV, Xirenayi XS, Stieglitz SE, Wiita WA. .
      View in: Publisher Site   Mentions:
    5. 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 (Basel). 2024 Jan 24; 16(3). 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. PMID: 38339248; PMCID: PMC10854518.
      View in: PubMed   Mentions:
    6. LNK/SH2B3 as a novel driver in juvenile myelomonocytic leukemia. Haematologica. 2023 Dec 28. 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. PMID: 38152053.
      View in: PubMed   Mentions:    Fields:    
    7. Too many white cells-TAM, JMML, or something else? Hematology Am Soc Hematol Educ Program. 2023 Dec 08; 2023(1):37-42. Satty A, Stieglitz E, Kucine N. PMID: 38066851; PMCID: PMC10727065.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    8. Efficacy of the Allosteric MEK Inhibitor Trametinib in Relapsed and Refractory Juvenile Myelomonocytic Leukemia: A Report from the Children's Oncology Group. Blood. 2023 Nov 28; 142(Supplement 1):74. Stieglitz SE, Lee LA, Angus AS, Davis DC, Barkauskas BD, Hall HD, Kogan KS, Meyer MJ, Rhodes RS, Xuei XX, Shannon SK, Loh LM, Fox FE, Weigel WB. .
      View in: Publisher Site   Mentions:
    9. Mediator Kinase/CDK8 Inhibition As a Strategy to Improve FLT3 Inhibitor Activity in Acute Myeloid Leukemia. Blood. 2023 Nov 28; 142(Supplement 1):4173. Ferng FT, Pintar PS, Kennedy KV, Tarver TT, Kabir KS, Steri SV, Serrano SJ, Salangsang SF, Phojanakong PP, Hübner HJ, Morales MC, Rivera RJ, Logan LA, Braun BB, Stieglitz SE, Gilbert GL, Smith SC. .
      View in: Publisher Site   Mentions:
    10. RAS MULTI(ON) Inhibitor RMC-7977 Targets Oncogenic RAS Mutations and Overcomes RAS/MAPK-Mediated Resistance to FLT3 Inhibitors in AML Models. Blood. 2023 Nov 28; 142(Supplement 1):2793. Popescu PB, Stieglitz SE, Smith SC. .
      View in: Publisher Site   Mentions:
    11. Threshold Attenuation of Perk Signaling Is Sufficient to Overcome RAS-Mediated FLT3 Inhibitor Resistance. Blood. 2023 Nov 28; 142(Supplement 1):4171. Peretz PC, Tran TE, Popescu PB, Kennedy KV, Kumar KT, McGary ML, Tantisira TN, Wong WE, Rivera RJ, Xirenayi XS, Stieglitz SE, Smith SC, Hübner HJ. .
      View in: Publisher Site   Mentions:
    12. 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 Nov 28; 142(Supplement 1):3210. Stieglitz SE, Chi CY, Chang CB, Tasian TS, Yohe YM, Dvorak DC, Southworth SE, Long-Boyle LJ, Van Ziffle VJ, Abdullaev AZ, Leong LR, Wayne WA, Bhojwani BD, Loh LM. .
      View in: Publisher Site   Mentions:
    13. Framework humanization optimizes potency of anti-CD72 nanobody CAR-T cells for B-cell malignancies. J Immunother Cancer. 2023 11 24; 11(11). 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. PMID: 38007238; PMCID: PMC10680002.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimalsCells
    14. Allosteric SHP2 inhibition increases apoptotic dependency on BCL2 and synergizes with venetoclax in FLT3- and KIT-mutant AML. Cell Rep Med. 2023 11 21; 4(11):101290. 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. PMID: 37992684; PMCID: PMC10694768.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    15. The 8th International RASopathies Symposium: Expanding research and care practice through global collaboration and advocacy. Am J Med Genet A. 2024 Apr; 194(4):e63477. 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. PMID: 37969032; PMCID: PMC10939912.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    16. 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 Rep (Hoboken). 2023 12; 6(12):e1901. 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. PMID: 37933765; PMCID: PMC10728537.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    17. Structural surfaceomics reveals an AML-specific conformation of integrin β2 as a CAR T cellular therapy target. Nat Cancer. 2023 Nov; 4(11):1592-1609. 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. PMID: 37904046; PMCID: PMC10663162.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    18. Tretinoin Enhances the Effects of Chemotherapy in Juvenile Myelomonocytic Leukemia Using an Ex Vivo Drug Sensitivity Assay. JCO Precis Oncol. 2023 Sep; 7:e2300302. 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. PMID: 37944074; PMCID: PMC10645413.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
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    20. P985: CLL-1 IS AN ADOPTIVE IMMUNOTHERAPY TARGET IN JUVENILE MYELOMONOCYTIC LEUKEMIA. HemaSphere. 2023 Aug 1; 7(Suppl):e40333db. Huebner HJ, Xirenayi XS, Rivera RJ, Escobar EB, Lee LA, Mandal MK, Diaz-Flores DE, Wiita WA, Stieglitz SE. .
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    21. Venetoclax in combination with chemotherapy as treatment for pediatric advanced hematologic malignancies. Pediatr Blood Cancer. 2023 06; 70(6):e30335. Marinoff AE, Aaronson K, Agrawal AK, Braun BS, Golden C, Huang BJ, Michlitsch J, Southworth E, Thrall A, Vo KT, Stieglitz E. PMID: 37036306; PMCID: PMC10133180.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    22. Potential clinical use of azacitidine and MEK inhibitor combination therapy in PTPN11-mutated juvenile myelomonocytic leukemia. Mol Ther. 2023 04 05; 31(4):986-1001. Pasupuleti SK, Chao K, Ramdas B, Kanumuri R, Palam LR, Liu S, Wan J, Annesley C, Loh ML, Stieglitz E, Burke MJ, Kapur R. PMID: 36739480; PMCID: PMC10124140.
      View in: PubMed   Mentions: 1     Fields:    
    23. Targeting Arg-1 and PD-L1 in M2-Tumor Associated Macrophages Impairs Juvenile Myelomonocytic Leukemia (JMML) Cell Proliferation and Migration. Proceedings of IMPRS. 2023 Jan 26; 5(1). Young YK, Pasupuleti PS, Stieglitz SE, Kapur KR. .
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    24. LNK/SH2B3 As a Novel Driver in Juvenile Myelomonocytic Leukemia. Blood. 2022 Nov 15; 140(Supplement 1):6863-6864. Wintering WA, Hecht HA, Meyer MJ, Wong WE, French FD, Maguire MJ, Jobaliya JC, Rojas Vasquez RM, Desai DS, Dulman DR, Nemecek NE, Chehab CF, Loh LM, Stieglitz SE. .
      View in: Publisher Site   Mentions:
    25. MEK inhibitors for neurofibromatosis type 1 manifestations: Clinical evidence and consensus. Neuro Oncol. 2022 11 02; 24(11):1845-1856. 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. PMID: 35788692; PMCID: PMC9629420.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    26. Classification of rare pediatric myeloid neoplasia-Quo vadis? Leukemia. 2022 12; 36(12):2947-2948. Niemeyer CM, Rudelius M, Shimamura A, Flotho C, Hasle H, Stieglitz E, Strahm B, Godley LA, Weinberg OK, Orazi A, Calvo KR. PMID: 36271151.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    27. 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. Eur J Cancer. 2022 Dec; 177:120-142. 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. PMID: 36335782.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    28. Clinical parameter-based prediction of DNA methylation classification generates a prediction model of prognosis in patients with juvenile myelomonocytic leukemia. Sci Rep. 2022 08 30; 12(1):14753. 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. PMID: 36042365; PMCID: PMC9427938.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    29. Detection of a GLIS3 fusion in an infant with AML refractory to chemotherapy. Cold Spring Harb Mol Case Stud. 2022 Aug 04. Smith SM, Lee A, Tong S, Leung S, Hongo H, Rivera J, Sweet-Cordero A, Michlitsch J, Stieglitz E. PMID: 35927023; PMCID: PMC9528968.
      View in: PubMed   Mentions: 2     Fields:    
    30. Cytomorphologic features of pediatric-type follicular lymphoma on fine needle aspiration biopsy: case series and a review of the literature. J Am Soc Cytopathol. 2022 Sep-Oct; 11(5):281-294. Lu KL, Menke JR, Ng D, Ruiz-Cordero R, Marinoff A, Stieglitz E, Gollapudi S, Singh K, Ohgami RS, Vohra P. PMID: 35843844.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    31. Inhibition of BTK and PI3Kδ impairs the development of human JMML stem and progenitor cells. Mol Ther. 2022 07 06; 30(7):2505-2521. 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. PMID: 35443935; PMCID: PMC9263321.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimalsCells
    32. Successful treatment and integrated genomic analysis of an infant with FIP1L1-RARA fusion-associated myeloid neoplasm. Blood Adv. 2022 02 22; 6(4):1137-1142. Miltiadous O, Petrova-Drus K, Kaicker S, Mathew S, Kluk M, Geyer JT, Rodriguez-Sanchez I, Bouvier N, Inghirami G, Stieglitz E, Khedoudja N, Benayed R, Richardson M, Anderson W, Benhamida J, You D, Londono D, Kung AL, Prockop SE, Roshal M, Zhang Y, Shukla N. PMID: 34551074; PMCID: PMC8864666.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    33. JMML tumor cells disrupt normal hematopoietic stem cells by imposing inflammatory stress through overproduction of IL-1β. Blood Adv. 2022 01 11; 6(1):200-206. Yan Y, Dong L, Chen C, Bunting KD, Li Q, Stieglitz E, Loh ML, Qu CK. PMID: 34555844; PMCID: PMC8753218.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansAnimalsCells
    34. Molecular and phenotypic diversity of CBL-mutated juvenile myelomonocytic leukemia. Haematologica. 2022 01 01; 107(1):178-186. 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. PMID: 33375775; PMCID: PMC8719097.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    35. Simple and robust methylation test for risk stratification of patients with juvenile myelomonocytic leukemia. Blood Adv. 2021 12 28; 5(24):5507-5518. 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. PMID: 34580726; PMCID: PMC8714717.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCells
    36. Therapy-related myeloid neoplasms resembling juvenile myelomonocytic leukemia: a case series and review of the literature. Pediatr Blood Cancer. 2022 05; 69(5):e29499. 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. PMID: 34939322; PMCID: PMC8957526.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    37. Juvenile myelomonocytic leukemia in the molecular era: a clinician's guide to diagnosis, risk stratification, and treatment. Blood Adv. 2021 11 23; 5(22):4783-4793. Wintering A, Dvorak CC, Stieglitz E, Loh ML. PMID: 34525182; PMCID: PMC8759142.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    38. MEK Inhibition Demonstrates Activity in Relapsed, Refractory Patients with Juvenile Myelomonocytic Leukemia: Results from COG Study ADVL1521. Blood. 2021 Nov 5; 138(Supplement 1):3679-3679. Stieglitz SE, Loh LM, Meyer MJ, Zhang ZC, Barkauskas BD, Hall HD, Fox FE, Weigel WB. .
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    39. Targeting M2-Tumor Associated Macrophages By Arginase-1 and PD-L1 in Regulating Juvenile Myelomonocytic Leukemia (JMML) Development and Relapse. Blood. 2021 Nov 5; 138(Supplement 1):1471-1471. Pasupuleti PS, Ramdas RB, Yang YK, Zhang ZC, Stieglitz SE, Kapur KR. .
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    40. Allosteric SHP2 Inhibitor RMC4550 Synergizes with Venetoclax in FLT3 and KIT Mutant Acute Myeloid Leukemia. Blood. 2021 Nov 5; 138(Supplement 1):2231-2231. Popescu PB, Stahlhut SC, Tarver TT, Ferng FT, Peretz PC, Wishner WS, Phojanakong PP, Steri SV, Wong WE, Rivera RJ, Stieglitz SE, Smith SC. .
      View in: Publisher Site   Mentions:
    41. Nf1 and Sh2b3 mutations cooperate in vivo in a mouse model of juvenile myelomonocytic leukemia. Blood Adv. 2021 09 28; 5(18):3587-3591. Morales CE, Stieglitz E, Kogan SC, Loh ML, Braun BS. PMID: 34464969; PMCID: PMC8945579.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansAnimalsCells
    42. NUP98-NSD1 Driven MDS/MPN in Childhood Masquerading as JMML. J Pediatr Hematol Oncol. 2021 08 01; 43(6):e808-e811. Behnert A, Lee AG, Young EP, Breese MR, Leung SG, Behroozfard I, Maruffi M, Sweet-Cordero EA, Dvorak CC, Chu J, Stieglitz E. PMID: 32815876; PMCID: PMC7889745.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    43. Exploring the genetic and epigenetic origins of juvenile myelomonocytic leukemia using newborn screening samples. Leukemia. 2022 01; 36(1):279-282. Behnert A, Meyer J, Parsa JY, Hechmer A, Loh ML, Olshen A, de Smith AJ, Stieglitz E. PMID: 34183765; PMCID: PMC8720242.
      View in: PubMed   Mentions: 4     Fields:    
    44. Surface Proteomics Reveals CD72 as a Target for In Vitro-Evolved Nanobody-Based CAR-T Cells in KMT2A/MLL1-Rearranged B-ALL. Cancer Discov. 2021 08; 11(8):2032-2049. 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. PMID: 33727310; PMCID: PMC8338785.
      View in: PubMed   Mentions: 21     Fields:    Translation:Humans
    45. Distinct genetic pathways define pre-malignant versus compensatory clonal hematopoiesis in Shwachman-Diamond syndrome. Nat Commun. 2021 02 26; 12(1):1334. 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. PMID: 33637765; PMCID: PMC7910481.
      View in: PubMed   Mentions: 61     Fields:    Translation:HumansCells
    46. Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium. Cancer Discov. 2021 06; 11(6):1424-1439. 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. PMID: 33563661; PMCID: PMC8178162.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCTClinical Trials
    47. Targeting the Ras pathway in pediatric hematologic malignancies. Curr Opin Pediatr. 2021 02 01; 33(1):49-58. Pikman Y, Stieglitz E. PMID: 33394740; PMCID: PMC7880552.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    48. Genetic Alterations Precede DNA Methylation Changes in Juvenile Myelomonocytic Leukemia. Blood. 2020 Nov 5; 136(Supplement 1):19-20. Behnert BA, Meyer MJ, Parsa PJ, Hechmer HA, Loh LM, Olshen OA, de Smith dA, Stieglitz SE. .
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    49. Distinct Genetic Pathways Define Leukemia Predisposition Versus Adaptive Clonal Hematopoiesis in Shwachman-Diamond Syndrome. Blood. 2020 Nov 5; 136(Supplement 1):35-36. Kennedy KA, Myers MK, Bowman BJ, Gibson GC, Muscato MG, Klein KR, Ballotti BK, Camarda CN, Furutani FE, Harris HC, Liu LS, Galvin GA, Malsch MM, Dale DD, Gansner GJ, Nakano NT, Bertuch BA, Vlachos VA, Lipton LJ, Castillo CP, Connelly CJ, Edwards EJ, Hijiya HN, Ho HR, Hofmann HI, Huang HJ, Keel KS, Lamble LA, Lau LB, Walkovich WK, Norkin NM, Stock SW, Boettcher BS, Brendel BC, Stieglitz SE, Fleming FM, Davies DS, Weller WE, Bahl BC, Carter CS, Shimamura SA, Lindsley LR. .
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    50. International Consensus Definition of DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia. Clin Cancer Res. 2021 01 01; 27(1):158-168. 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. PMID: 33139265; PMCID: PMC7785676.
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    51. 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 Precis Oncol. 2020; 4. 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. PMID: 33283136; PMCID: PMC7713560.
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    52. Clinical utilization of blinatumomab and inotuzumab immunotherapy in children with relapsed or refractory B-acute lymphoblastic leukemia. Pediatr Blood Cancer. 2021 01; 68(1):e28718. Contreras CF, Higham CS, Behnert A, Kim K, Stieglitz E, Tasian SK. PMID: 33098744; PMCID: PMC7688575.
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    53. 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 Transplant. 2020 11; 55(11):2160-2169. 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. PMID: 32390002; PMCID: PMC7606268.
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    54. Precision cancer monitoring using a novel, fully integrated, microfluidic array partitioning digital PCR platform. Sci Rep. 2019 12 20; 9(1):19606. Dueck ME, Lin R, Zayac A, Gallagher S, Chao AK, Jiang L, Datwani SS, Hung P, Stieglitz E. PMID: 31862911; PMCID: PMC6925289.
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    55. 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. Pediatr Blood Cancer. 2020 02; 67(2):e28079. Friend BD, Bailey-Olson M, Melton A, Shimano KA, Kharbanda S, Higham C, Winestone LE, Huang J, Stieglitz E, Dvorak CC. PMID: 31724815.
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    56. In Vitro-Selected Nanobody-Based Cellular Therapy Targeting CD72 for Treatment of Refractory B-Cell Malignancies. Blood. 2019 Nov 13; 134(Supplement_1):1337-1337. Nix NM, Lin LY, Geng GH, Marcoulis MM, Phojanakong PP, Wang WD, Steri SV, Whitman WJ, Bapat BS, Stieglitz SE, Deucher DA, White WK, Hann HB, Wiita WA. .
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    57. DNA Methylation As a Biomarker of Outcome in JMML: An International Effort Towards Clinical Implementation. Blood. 2019 Nov 13; 134(Supplement_1):1693-1693. Meyer MJ, Schönung SM, Flotho FC, Olshen OA, Hartmann HM, Plass PC, Okuno OY, Takahashi TY, Murakami MN, Wakamatsu WM, Loh LM, Niemeyer NC, Muramatsu MH, Lipka LD, Stieglitz SE. .
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    58. Fusion driven JMML: a novel CCDC88C-FLT3 fusion responsive to sorafenib identified by RNA sequencing. Leukemia. 2020 02; 34(2):662-666. 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. PMID: 31511612; PMCID: PMC6995757.
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    59. Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia. Pediatr Blood Cancer. 2019 11; 66(11):e27948. Hecht A, Meyer J, Chehab FF, White KL, Magruder K, Dvorak CC, Loh ML, Stieglitz E. PMID: 31347788; PMCID: PMC6754267.
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    60. Predisposing germline mutations in high hyperdiploid acute lymphoblastic leukemia in children. Genes Chromosomes Cancer. 2019 10; 58(10):723-730. 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, Wiemels JL. PMID: 31102422; PMCID: PMC6684857.
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    61. Activating Mutations of RRAS2 Are a Rare Cause of Noonan Syndrome. Am J Hum Genet. 2019 06 06; 104(6):1223-1232. 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. PMID: 31130282; PMCID: PMC6562003.
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    62. Genomic subtyping and therapeutic targeting of acute erythroleukemia. Nat Genet. 2019 04; 51(4):694-704. 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. PMID: 30926971; PMCID: PMC6828160.
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    63. Phase I Study of the Selinexor in Relapsed/Refractory Childhood Acute Leukemia. Blood. 2018 Nov 29; 132(Supplement 1):1405-1405. Place PA, Blonquist BT, Stieglitz SE, Cooper CT, Gore GL, Aplenc AR, Loh LM, Pauly PM, Rau RR, Burke BM, Etchin EJ, Look LA, Sulis SM, Silverman SL. .
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    64. Germline GAB2 Mutations in Childhood Acute Lymphoblastic Leukemia. Blood. 2018 Nov 29; 132(Supplement 1):388-388. de Smith dA, Lavoie LG, Walsh WK, Aujla AS, Evans EE, Hansen HH, Smirnov SI, Kang KA, Zenker ZM, Ceremsak CJ, Stieglitz SE, McKean-Cowdin MR, Metayer MC, Roux RP, Wiemels WJ. .
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    65. DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia: An International Collaborative Analysis and Development of a Common Diagnostic Platform. Blood. 2018 Nov 29; 132(Supplement 1):3093-3093. Schoenung SM, Stieglitz SE, Muramatsu MH, Murakami MN, Hartmann HM, Wiesenfarth WM, Okuno OY, Olshen OA, Plass PC, Loh LM, Niemeyer NC, Flotho FC, Lipka LD. .
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    66. Downregulating Notch counteracts KrasG12D-induced ERK activation and oxidative phosphorylation in myeloproliferative neoplasm. Leukemia. 2019 03; 33(3):671-685. 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. PMID: 30206308; PMCID: PMC6405304.
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    67. ABVD Without Radiation for Newly Diagnosed Pediatric and Young Adult Patients With Hodgkin Lymphoma: A Single Center Retrospective Analysis of 28 Consecutive Patients. J Pediatr Hematol Oncol. 2018 05; 40(4):290-294. Stieglitz E, Dinh T, Phelps AS, Pampaloni MH, Olshen AB, Robbins E. PMID: 29432308.
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    68. Disease burden and conditioning regimens in ASCT1221, a randomized phase II trial in children with juvenile myelomonocytic leukemia: A Children's Oncology Group study. Pediatr Blood Cancer. 2018 07; 65(7):e27034. 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. PMID: 29528181; PMCID: PMC5980696.
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    69. Genome-wide DNA methylation is predictive of outcome in juvenile myelomonocytic leukemia. Nat Commun. 2017 12 19; 8(1):2127. 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. PMID: 29259179; PMCID: PMC5736624.
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    70. Abstract 26: RUNX1 as a transcriptional target of activated Shp2 (PTPN11) in juvenile myelomonocytic leukemia. Leukemia. 2017 Dec 15; 26-26. Dorantes-Acosta DE, Huang HH, Garcia GS, Stieglitz SE, Loh LM, Yuan YG, Cantor CA. .
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    71. Dysregulation of the transcription factor runx1 in juvenile myelomonocytic leukemia. Experimental Hematology. 2017 Sep 1; 53:s51. Cantor CA, Dorantes-Costa DE, Huang HH, Garcia GS, Stieglitz SE, Loh LM, Yuan YG. .
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    72. Robust patient-derived xenografts of MDS/MPN overlap syndromes capture the unique characteristics of CMML and JMML. Blood. 2017 07 27; 130(4):397-407. 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. PMID: 28576879; PMCID: PMC5533204.
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    73. A Risk Stratified Treatment Algorithm for Patients with Juvenile Myelomonocytic Leukemia. . 2017 Mar 1; 14(2). Koegel KA, Stieglitz SE. .
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    74. International Interlaboratory Digital PCR Study Demonstrating High Reproducibility for the Measurement of a Rare Sequence Variant. Anal Chem. 2017 02 07; 89(3):1724-1733. 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. PMID: 27935690.
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    75. RUNX1 Is a Candidate Transcriptional Effector in Juvenile Myelomonocytic Leukemia. Blood. 2016 Dec 2; 128(22):2699-2699. Dorantes Acosta DE, Huang HH, Garcia GS, Stieglitz SE, Loh LM, Yuan YG, Cantor CA. .
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    76. The Genomic Landscape of Childhood and Adult Acute Erythroid Leukemia. Blood. 2016 Dec 2; 128(22):39-39. Iacobucci II, Wen WJ, Meggendorfer MM, Carmichael CC, Choi CJ, Masih MK, Li LY, Payne PD, Tomizawa TD, Kiyokawa KN, Janke JL, Alexander AT, Pounds PS, Shi SL, Valentine VM, Basso BG, Locatelli LF, Kham Kow Yin KS, Yeoh Eng Juh YA, Rhonda RR, Stieglitz SE, Wei WA, Lewis LI, D'Andrea DR, Kile KB, Zhang ZJ, Loh LM, Meshinchi MS, Haferlach HT, Mullighan MC. .
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    77. Germline RRAS2 mutations are not associated with Noonan syndrome. J Med Genet. 2016 Nov; 53(11):728. Ceremsak JJ, Yu A, Esquivel E, Lissewski C, Zenker M, Loh ML, Stieglitz E. PMID: 27055474.
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    78. Pediatric MDS: GATA screen the germline. Blood. 2016 Mar 17; 127(11):1377-8. Stieglitz E, Loh ML. PMID: 26989184.
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    79. Corrigendum: The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet. 2016 Jan; 48(1):101. 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. PMID: 26711114.
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    80. Phase II/III trial of a pre-transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2015 Apr; 62(4):629-36. 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. PMID: 25704135; PMCID: PMC4339233.
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    81. Subclonal Mutations in SETBP1 Predict Relapse in Juvenile Myelomonocytic Leukemia. Blood. 2014 Dec 6; 124(21):410-410. Stieglitz SE, Troup TC, Gelston GL, Chow CE, Yu YK, Akutagawa AJ, Taylor-Weiner TA, Liu LY, Emanuel EP, Braun BB, Gerbing GR, Alonzo AT, Loh LM. .
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    82. Subclonal mutations in SETBP1 confer a poor prognosis in juvenile myelomonocytic leukemia. Blood. 2015 Jan 15; 125(3):516-24. 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. PMID: 25395418; PMCID: PMC4296011.
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    83. Mutations in GATA2 are rare in juvenile myelomonocytic leukemia. Blood. 2014 Feb 27; 123(9):1426-7. Stieglitz E, Liu YL, Emanuel PD, Castleberry RP, Cooper TM, Shannon KM, Loh ML. PMID: 24578498; PMCID: PMC3938154.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansCells
    84. Pulmonary coccidiomycosis masquerading as refractory metastatic Ewing sarcoma. J Pediatr Hematol Oncol. 2014 Jan; 36(1):e57-60. Stieglitz E, Hsiang MS, Simko JP, Hirose S, Goldsby RE. PMID: 23588326.
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    85. Mutations In GATA2 Are Rare In Juvenile Myelomonocytic Leukemia. Blood. 2013 Nov 15; 122(21):1526-1526. Stieglitz SE, Liu LY, Emanuel EP, Castleberry CR, Cooper CT, Shannon SK, Loh LM. .
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    86. Genetic predispositions to childhood leukemia. Ther Adv Hematol. 2013 Aug; 4(4):270-90. Stieglitz E, Loh ML. PMID: 23926459; PMCID: PMC3734905.
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