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Items: 1 to 50 of 121

1.

Low mutation burden and frequent loss of CDKN2A/B and SMARCA2, but not PRC2, define pre-malignant neurofibromatosis type 1-associated atypical neurofibromas.

Pemov A, Hansen NF, Sindiri S, Patidar R, Higham CS, Dombi E, Miettinen MM, Fetsch P, Brems H, Chandrasekharappa S, Jones K, Zhu B, Wei JS; NISC Comparative Sequencing Program; NCI DCEG Cancer Genomics Research Laboratory, Mullikin JC, Wallace MR, Khan J, Legius E, Widemann BC, Stewart DR.

Neuro Oncol. 2019 Feb 5. doi: 10.1093/neuonc/noz028. [Epub ahead of print]

PMID:
30722027
2.

Proceedings of the fifth international RASopathies symposium: When development and cancer intersect.

Rauen KA, Schoyer L, Schill L, Stronach B, Albeck J, Andresen BS, Cavé H, Ellis M, Fruchtman SM, Gelb BD, Gibson CC, Gripp K, Hefner E, Huang WYC, Itkin M, Kerr B, Linardic CM, McMahon M, Oberlander B, Perlstein E, Ratner N, Rogers L, Schenck A, Shankar S, Shvartsman S, Stevenson DA, Stites EC, Stork PJS, Sun C, Therrien M, Ullian EM, Widemann BC, Yeh E, Zampino G, Zenker M, Timmer W, McCormick F.

Am J Med Genet A. 2018 Dec;176(12):2924-2929. doi: 10.1002/ajmg.a.40632. Epub 2018 Oct 10.

PMID:
30302932
3.

Genetically engineered minipigs model the major clinical features of human neurofibromatosis type 1.

Isakson SH, Rizzardi AE, Coutts AW, Carlson DF, Kirstein MN, Fisher J, Vitte J, Williams KB, Pluhar GE, Dahiya S, Widemann BC, Dombi E, Rizvi T, Ratner N, Messiaen L, Stemmer-Rachamimov AO, Fahrenkrug SC, Gutmann DH, Giovannini M, Moertel CL, Largaespada DA, Watson AL.

Commun Biol. 2018 Oct 2;1:158. doi: 10.1038/s42003-018-0163-y. eCollection 2018.

4.

Safety and efficacy of low-dose sirolimus in the PIK3CA-related overgrowth spectrum.

Parker VER, Keppler-Noreuil KM, Faivre L, Luu M, Oden NL, De Silva L, Sapp JC, Andrews K, Bardou M, Chen KY, Darling TN, Gautier E, Goldspiel BR, Hadj-Rabia S, Harris J, Kounidas G, Kumar P, Lindhurst MJ, Loffroy R, Martin L, Phan A, Rother KI, Widemann BC, Wolters PL, Coubes C, Pinson L, Willems M, Vincent-Delorme C; PROMISE Working Group, Vabres P, Semple RK, Biesecker LG.

Genet Med. 2018 Oct 1. doi: 10.1038/s41436-018-0297-9. [Epub ahead of print]

PMID:
30270358
5.

Clinical trial design for cutaneous neurofibromas.

Cannon A, Jarnagin K, Korf B, Widemann BC, Casey D, Ko HS, Blakeley JO, Verma SK, Pichard DC.

Neurology. 2018 Jul 10;91(2 Supplement 1):S31-S37. doi: 10.1212/WNL.0000000000005790.

PMID:
29987133
6.

Creating a comprehensive research strategy for cutaneous neurofibromas.

Blakeley JO, Wolkenstein P, Widemann BC, Lee J, Le LQ, Jackson R, Stathis M, Verma SK.

Neurology. 2018 Jul 10;91(2 Supplement 1):S1-S4. doi: 10.1212/WNL.0000000000005789.

PMID:
29987129
7.

Improved CNS exposure to tocilizumab after cerebrospinal fluid compared to intravenous administration in rhesus macaques.

Nellan A, McCully CML, Cruz Garcia R, Jayaprakash N, Widemann BC, Lee DW, Warren KE.

Blood. 2018 Aug 9;132(6):662-666. doi: 10.1182/blood-2018-05-846428. Epub 2018 Jun 28. No abstract available.

PMID:
29954750
8.

Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1.

Gross AM, Singh G, Akshintala S, Baldwin A, Dombi E, Ukwuani S, Goodwin A, Liewehr DJ, Steinberg SM, Widemann BC.

Neuro Oncol. 2018 Nov 12;20(12):1643-1651. doi: 10.1093/neuonc/noy067.

PMID:
29718344
9.

A phase 1 study of cabozantinib in children and adolescents with recurrent or refractory solid tumors, including CNS tumors: Trial ADVL1211, a report from the Children's Oncology Group.

Chuk MK, Widemann BC, Minard CG, Liu X, Kim A, Bernhardt MB, Kudgus RA, Reid JM, Voss SD, Blaney S, Fox E, Weigel BJ.

Pediatr Blood Cancer. 2018 Aug;65(8):e27077. doi: 10.1002/pbc.27077. Epub 2018 Apr 25.

PMID:
29693796
10.

2016 Children's Tumor Foundation conference on neurofibromatosis type 1, neurofibromatosis type 2, and schwannomatosis.

Fisher MJ, Belzberg AJ, de Blank P, De Raedt T, Elefteriou F, Ferner RE, Giovannini M, Harris GJ, Kalamarides M, Karajannis MA, Kim A, Lázaro C, Le LQ, Li W, Listernick R, Martin S, Morrison H, Pasmant E, Ratner N, Schorry E, Ullrich NJ, Viskochil D, Weiss B, Widemann BC, Zhu Y, Bakker A, Serra E.

Am J Med Genet A. 2018 May;176(5):1258-1269. doi: 10.1002/ajmg.a.38675.

PMID:
29681099
11.

Improvement in Patient-reported Hearing After Treatment With Bevacizumab in People With Neurofibromatosis Type 2.

Huang V, Bergner AL, Halpin C, Merker VL, Sheridan MR, Widemann BC, Blakeley JO, Plotkin SR.

Otol Neurotol. 2018 Jun;39(5):632-638. doi: 10.1097/MAO.0000000000001781.

PMID:
29649040
12.

The characteristics of 76 atypical neurofibromas as precursors to neurofibromatosis 1 associated malignant peripheral nerve sheath tumors.

Higham CS, Dombi E, Rogiers A, Bhaumik S, Pans S, Connor SEJ, Miettinen M, Sciot R, Tirabosco R, Brems H, Baldwin A, Legius E, Widemann BC, Ferner RE.

Neuro Oncol. 2018 May 18;20(6):818-825. doi: 10.1093/neuonc/noy013.

PMID:
29409029
13.
14.

Outcomes of Children and Adolescents with Advanced Hereditary Medullary Thyroid Carcinoma Treated with Vandetanib.

Kraft IL, Akshintala S, Zhu Y, Lei H, Derse-Anthony C, Dombi E, Steinberg SM, Lodish M, Waguespack SG, Kapustina O, Fox E, Balis FM, Merino MJ, Meltzer PS, Glod JW, Shern JF, Widemann BC.

Clin Cancer Res. 2018 Feb 15;24(4):753-765. doi: 10.1158/1078-0432.CCR-17-2101. Epub 2017 Nov 29.

15.

SARC006: Phase II Trial of Chemotherapy in Sporadic and Neurofibromatosis Type 1 Associated Chemotherapy-Naive Malignant Peripheral Nerve Sheath Tumors.

Higham CS, Steinberg SM, Dombi E, Perry A, Helman LJ, Schuetze SM, Ludwig JA, Staddon A, Milhem MM, Rushing D, Jones RL, Livingston M, Goldman S, Moertel C, Wagner L, Janhofer D, Annunziata CM, Reinke D, Long L, Viskochil D, Baker L, Widemann BC.

Sarcoma. 2017;2017:8685638. doi: 10.1155/2017/8685638. Epub 2017 Sep 12.

16.

Neurofibromatosis Type 1-Associated MPNST State of the Science: Outlining a Research Agenda for the Future.

Reilly KM, Kim A, Blakely J, Ferner RE, Gutmann DH, Legius E, Miettinen MM, Randall RL, Ratner N, Jumbé NL, Bakker A, Viskochil D, Widemann BC, Stewart DR.

J Natl Cancer Inst. 2017 Aug 1;109(8). doi: 10.1093/jnci/djx124. Review.

17.

Volumetric MRI Analysis of Plexiform Neurofibromas in Neurofibromatosis Type 1: Comparison of Two Methods.

Cai W, Steinberg SM, Bredella MA, Basinsky G, Somarouthu B, Plotkin SR, Solomon J, Widemann BC, Harris GJ, Dombi E.

Acad Radiol. 2018 Feb;25(2):144-152. doi: 10.1016/j.acra.2017.09.004. Epub 2017 Oct 31.

18.

Consensus Guideline for Use of Glucarpidase in Patients with High-Dose Methotrexate Induced Acute Kidney Injury and Delayed Methotrexate Clearance.

Ramsey LB, Balis FM, O'Brien MM, Schmiegelow K, Pauley JL, Bleyer A, Widemann BC, Askenazi D, Bergeron S, Shirali A, Schwartz S, Vinks AA, Heldrup J.

Oncologist. 2018 Jan;23(1):52-61. doi: 10.1634/theoncologist.2017-0243. Epub 2017 Oct 27.

19.

Editorial: US Cancer Statistics of Survival: Achievements, Challenges, and Future Directions.

Gadalla SM, Widemann BC.

J Natl Cancer Inst. 2017 Sep 1;109(9). doi: 10.1093/jnci/djx070. No abstract available.

20.

A phase I/II trial and pharmacokinetic study of mithramycin in children and adults with refractory Ewing sarcoma and EWS-FLI1 fusion transcript.

Grohar PJ, Glod J, Peer CJ, Sissung TM, Arnaldez FI, Long L, Figg WD, Whitcomb P, Helman LJ, Widemann BC.

Cancer Chemother Pharmacol. 2017 Sep;80(3):645-652. doi: 10.1007/s00280-017-3382-x. Epub 2017 Jul 22.

PMID:
28735378
21.

Identifying Symptoms of Distress in Youth Living with Neurofibromatosis Type 1 (NF1).

Wiener L, Battles H, Bedoya SZ, Baldwin A, Widemann BC, Pao M.

J Genet Couns. 2018 Feb;27(1):115-123. doi: 10.1007/s10897-017-0128-1. Epub 2017 Jul 23.

22.

Malignant Peripheral Nerve Sheath Tumors State of the Science: Leveraging Clinical and Biological Insights into Effective Therapies.

Kim A, Stewart DR, Reilly KM, Viskochil D, Miettinen MM, Widemann BC.

Sarcoma. 2017;2017:7429697. doi: 10.1155/2017/7429697. Epub 2017 May 16. Review.

23.

Inhibition of B Cell Receptor Signaling by Ibrutinib in Primary CNS Lymphoma.

Lionakis MS, Dunleavy K, Roschewski M, Widemann BC, Butman JA, Schmitz R, Yang Y, Cole DE, Melani C, Higham CS, Desai JV, Ceribelli M, Chen L, Thomas CJ, Little RF, Gea-Banacloche J, Bhaumik S, Stetler-Stevenson M, Pittaluga S, Jaffe ES, Heiss J, Lucas N, Steinberg SM, Staudt LM, Wilson WH.

Cancer Cell. 2017 Jun 12;31(6):833-843.e5. doi: 10.1016/j.ccell.2017.04.012. Epub 2017 May 25.

24.

Selumetinib in Plexiform Neurofibromas.

Gross A, Bishop R, Widemann BC.

N Engl J Med. 2017 Mar 23;376(12):1195. doi: 10.1056/NEJMc1701029. No abstract available.

PMID:
28328340
25.

The primacy of NF1 loss as the driver of tumorigenesis in neurofibromatosis type 1-associated plexiform neurofibromas.

Pemov A, Li H, Patidar R, Hansen NF, Sindiri S, Hartley SW, Wei JS, Elkahloun A, Chandrasekharappa SC; NISC Comparative Sequencing Program, Boland JF, Bass S; NCI DCEG Cancer Genomics Research Laboratory, Mullikin JC, Khan J, Widemann BC, Wallace MR, Stewart DR.

Oncogene. 2017 Jun 1;36(22):3168-3177. doi: 10.1038/onc.2016.464. Epub 2017 Jan 9.

PMID:
28068329
26.

Activity of Selumetinib in Neurofibromatosis Type 1-Related Plexiform Neurofibromas.

Dombi E, Baldwin A, Marcus LJ, Fisher MJ, Weiss B, Kim A, Whitcomb P, Martin S, Aschbacher-Smith LE, Rizvi TA, Wu J, Ershler R, Wolters P, Therrien J, Glod J, Belasco JB, Schorry E, Brofferio A, Starosta AJ, Gillespie A, Doyle AL, Ratner N, Widemann BC.

N Engl J Med. 2016 Dec 29;375(26):2550-2560. doi: 10.1056/NEJMoa1605943.

27.

Orbital/Periorbital Plexiform Neurofibromas in Children with Neurofibromatosis Type 1: Multidisciplinary Recommendations for Care.

Avery RA, Katowitz JA, Fisher MJ, Heidary G, Dombi E, Packer RJ, Widemann BC; OPPN Working Group.

Ophthalmology. 2017 Jan;124(1):123-132. doi: 10.1016/j.ophtha.2016.09.020. Epub 2016 Nov 3. Review.

28.

Sequence-Specific Pharmacokinetic and Pharmacodynamic Phase I/Ib Study of Olaparib Tablets and Carboplatin in Women's Cancer.

Lee JM, Peer CJ, Yu M, Amable L, Gordon N, Annunziata CM, Houston N, Goey AK, Sissung TM, Parker B, Minasian L, Chiou VL, Murphy RF, Widemann BC, Figg WD, Kohn EC.

Clin Cancer Res. 2017 Mar 15;23(6):1397-1406. doi: 10.1158/1078-0432.CCR-16-1546. Epub 2016 Sep 23.

29.

Current status and recommendations for biomarkers and biobanking in neurofibromatosis.

Hanemann CO, Blakeley JO, Nunes FP, Robertson K, Stemmer-Rachamimov A, Mautner V, Kurtz A, Ferguson M, Widemann BC, Evans DG, Ferner R, Carroll SL, Korf B, Wolkenstein P, Knight P, Plotkin SR; REiNS International Collaboration.

Neurology. 2016 Aug 16;87(7 Suppl 1):S40-8. doi: 10.1212/WNL.0000000000002932.

30.

Sleep and pulmonary outcomes for clinical trials of airway plexiform neurofibromas in NF1.

Plotkin SR, Davis SD, Robertson KA, Akshintala S, Allen J, Fisher MJ, Blakeley JO, Widemann BC, Ferner RE, Marcus CL; REiNS International Collaboration.

Neurology. 2016 Aug 16;87(7 Suppl 1):S13-20. doi: 10.1212/WNL.0000000000002933.

31.

Phase II trial of pegylated interferon alfa-2b in young patients with neurofibromatosis type 1 and unresectable plexiform neurofibromas.

Jakacki RI, Dombi E, Steinberg SM, Goldman S, Kieran MW, Ullrich NJ, Pollack IF, Goodwin A, Manley PE, Fangusaro J, Allen R, Widemann BC.

Neuro Oncol. 2017 Feb 1;19(2):289-297. doi: 10.1093/neuonc/now158.

32.

Outcome of Patients With Recurrent Osteosarcoma Enrolled in Seven Phase II Trials Through Children's Cancer Group, Pediatric Oncology Group, and Children's Oncology Group: Learning From the Past to Move Forward.

Lagmay JP, Krailo MD, Dang H, Kim A, Hawkins DS, Beaty O 3rd, Widemann BC, Zwerdling T, Bomgaars L, Langevin AM, Grier HE, Weigel B, Blaney SM, Gorlick R, Janeway KA.

J Clin Oncol. 2016 Sep 1;34(25):3031-8. doi: 10.1200/JCO.2015.65.5381. Epub 2016 Jul 11.

33.

The Fourth International Symposium on Genetic Disorders of the Ras/MAPK pathway.

Stevenson DA, Schill L, Schoyer L, Andresen BS, Bakker A, Bayrak-Toydemir P, Burkitt-Wright E, Chatfield K, Elefteriou F, Elgersma Y, Fisher MJ, Franz D, Gelb BD, Goriely A, Gripp KW, Hardan AY, Keppler-Noreuil KM, Kerr B, Korf B, Leoni C, McCormick F, Plotkin SR, Rauen KA, Reilly K, Roberts A, Sandler A, Siegel D, Walsh K, Widemann BC.

Am J Med Genet A. 2016 Aug;170(8):1959-66. doi: 10.1002/ajmg.a.37723. Epub 2016 May 7.

34.

MultiDimensional ClinOmics for Precision Therapy of Children and Adolescent Young Adults with Relapsed and Refractory Cancer: A Report from the Center for Cancer Research.

Chang W, Brohl AS, Patidar R, Sindiri S, Shern JF, Wei JS, Song YK, Yohe ME, Gryder B, Zhang S, Calzone KA, Shivaprasad N, Wen X, Badgett TC, Miettinen M, Hartman KR, League-Pascual JC, Trahair TN, Widemann BC, Merchant MS, Kaplan RN, Lin JC, Khan J.

Clin Cancer Res. 2016 Aug 1;22(15):3810-20. doi: 10.1158/1078-0432.CCR-15-2717. Epub 2016 Mar 18.

35.

Efficacy and Biomarker Study of Bevacizumab for Hearing Loss Resulting From Neurofibromatosis Type 2-Associated Vestibular Schwannomas.

Blakeley JO, Ye X, Duda DG, Halpin CF, Bergner AL, Muzikansky A, Merker VL, Gerstner ER, Fayad LM, Ahlawat S, Jacobs MA, Jain RK, Zalewski C, Dombi E, Widemann BC, Plotkin SR.

J Clin Oncol. 2016 May 10;34(14):1669-75. doi: 10.1200/JCO.2015.64.3817. Epub 2016 Mar 14.

36.

Vincristine Sulfate Liposomes Injection (VSLI, Marqibo®): Results From a Phase I Study in Children, Adolescents, and Young Adults With Refractory Solid Tumors or Leukemias.

Shah NN, Merchant MS, Cole DE, Jayaprakash N, Bernstein D, Delbrook C, Richards K, Widemann BC, Wayne AS.

Pediatr Blood Cancer. 2016 Jun;63(6):997-1005. doi: 10.1002/pbc.25937. Epub 2016 Feb 17.

PMID:
26891067
37.

Population pharmacokinetics of sirolimus in pediatric patients with neurofibromatosis type 1-Erratum.

Scott JR, Courter JD, Saldaña SN, Widemann BC, Fisher M, Weiss B, Perentesis J, Vinks AA.

Ther Drug Monit. 2016 Jan 21. [Epub ahead of print] No abstract available.

38.

Plasma and cerebrospinal fluid pharmacokinetics of vincristine and vincristine sulfate liposomes injection (VSLI, marqibo®) after intravenous administration in Non-human primates.

Shah NN, Cole DE, Lester-McCully CM, Wayne AS, Warren KE, Widemann BC.

Invest New Drugs. 2016 Feb;34(1):61-5. doi: 10.1007/s10637-015-0311-x. Epub 2015 Dec 10.

39.

Validity, specificity, feasibility and acceptability of a brief pediatric distress thermometer in outpatient clinics.

Wiener L, Battles H, Zadeh S, Widemann BC, Pao M.

Psychooncology. 2017 Apr;26(4):461-468. doi: 10.1002/pon.4038. Epub 2015 Nov 30.

40.

Reply to: Glucarpidase for the Treatment of Methotrexate-Induced Renal Dysfunction and Delayed Methotrexate Excretion.

Widemann BC.

Pediatr Blood Cancer. 2016 Feb;63(2):366. doi: 10.1002/pbc.25798. Epub 2015 Oct 21. No abstract available.

PMID:
26488622
41.

Pharmacokinetic and pharmacodynamic study of tariquidar (XR9576), a P-glycoprotein inhibitor, in combination with doxorubicin, vinorelbine, or docetaxel in children and adolescents with refractory solid tumors.

Fox E, Widemann BC, Pastakia D, Chen CC, Yang SX, Cole D, Balis FM.

Cancer Chemother Pharmacol. 2015 Dec;76(6):1273-83. doi: 10.1007/s00280-015-2845-1. Epub 2015 Oct 20.

PMID:
26486517
42.

Cephalometry in adults and children with neurofibromatosis type 1: Implications for the pathogenesis of sphenoid wing dysplasia and the "NF1 facies".

Cung W, Freedman LA, Khan NE, Romberg E, Gardner PJ, Bassim CW, Baldwin AM, Widemann BC, Stewart DR.

Eur J Med Genet. 2015 Nov;58(11):584-90. doi: 10.1016/j.ejmg.2015.09.001. Epub 2015 Sep 8.

43.

Phase 2 trial of sorafenib in children and young adults with refractory solid tumors: A report from the Children's Oncology Group.

Kim A, Widemann BC, Krailo M, Jayaprakash N, Fox E, Weigel B, Blaney SM.

Pediatr Blood Cancer. 2015 Sep;62(9):1562-6. doi: 10.1002/pbc.25548. Epub 2015 Apr 27.

44.

Pain interference in youth with neurofibromatosis type 1 and plexiform neurofibromas and relation to disease severity, social-emotional functioning, and quality of life.

Wolters PL, Burns KM, Martin S, Baldwin A, Dombi E, Toledo-Tamula MA, Dudley WN, Gillespie A, Widemann BC.

Am J Med Genet A. 2015 Sep;167A(9):2103-13. doi: 10.1002/ajmg.a.37123. Epub 2015 May 14.

PMID:
25976979
45.

Practical considerations for the administration of glucarpidase in high-dose methotrexate (HDMTX) induced renal dysfunction.

Widemann BC.

Pediatr Blood Cancer. 2015 Sep;62(9):1512-3. doi: 10.1002/pbc.25577. Epub 2015 May 4. No abstract available.

PMID:
25940351
46.

Plasma and cerebrospinal fluid pharmacokinetics of the Akt inhibitor, perifosine, in a non-human primate model.

Cole DE, Lester-McCully CM, Widemann BC, Warren KE.

Cancer Chemother Pharmacol. 2015 May;75(5):923-8. doi: 10.1007/s00280-015-2711-1. Epub 2015 Mar 5.

47.

Phase II/III trial of a pre-transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: a report from the Children's Oncology Group.

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.

Pediatr Blood Cancer. 2015 Apr;62(4):629-36. doi: 10.1002/pbc.25342. Epub 2014 Dec 8.

48.

Phase 1 trial and pharmacokinetic study of the oral platinum analog satraplatin in children and young adults with refractory solid tumors including brain tumors.

Akshintala S, Marcus L, Warren KE, Murphy RF, Sissung TM, Srivastava A, Goodspeed WJ, Goodwin A, Brewer CC, Zalewski C, King KA, Kim A, Figg WD, Widemann BC.

Pediatr Blood Cancer. 2015 Apr;62(4):603-10. doi: 10.1002/pbc.25344. Epub 2015 Jan 3.

49.

Radiation therapy in management of sporadic and neurofibromatosis type 1-associated malignant peripheral nerve sheath tumors.

Kahn J, Gillespie A, Tsokos M, Ondos J, Dombi E, Camphausen K, Widemann BC, Kaushal A.

Front Oncol. 2014 Nov 17;4:324. doi: 10.3389/fonc.2014.00324. eCollection 2014.

50.

Genetic modifiers of neurofibromatosis type 1-associated café-au-lait macule count identified using multi-platform analysis.

Pemov A, Sung H, Hyland PL, Sloan JL, Ruppert SL, Baldwin AM, Boland JF, Bass SE, Lee HJ, Jones KM, Zhang X; NISC Comparative Sequencing Program, Mullikin JC, Widemann BC, Wilson AF, Stewart DR.

PLoS Genet. 2014 Oct 16;10(10):e1004575. doi: 10.1371/journal.pgen.1004575. eCollection 2014 Oct.

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