Katherine K. Matthay, MD

Katherine K. Matthay, MD

Professor, Department of Pediatrics; Leader, Pediatric Malignancies Program, UCSF Helen Diller Family Comprehensive Cancer Center
Mildred V. Strouss Endowed Chair in Translational Research in Pediatric Oncology, UCSF

Phone: (415) 476-3831 (appts)
Box 0106, UCSF
San Francisco, CA 94143-0106

UCSF Profiles

Cancer Center Membership

Program Member » Pediatric Malignancies

Research Summary

I am professor of pediatrics at the UCSF School of Medicine. I am the leader of the Pediatric Malignancies Program of the Helen Diller Family Comprehensive Cancer Center at UCSF and am one of the leading translational investigators in neuroblastoma therapy internationally. I have extensive experience over the last 25 years in clinical trials and translational research in neuroblastoma and pediatric oncology. I was the chair of the Neuroblastoma Strategy Group in the Children’s Cancer Group for ten years and continue on the Neuroblastoma Steering committee. In this role, I initiated several landmark randomized trials in high-risk neuroblastoma, improving outcome with myeloablative therapy and with two biologic targeted therapies. I am the Principal Investigator for the UCSF Alex Lemonade Center of Excellence for Developmental Therapeutics. I have established and lead the NCI-funded NANT clinical trials consortium for targeted therapy of neuroblastoma, now in its third 5-year cycle. I have been a leader in the development of response evaluation internationally and targeted radiotherapy with the radiopharmaceutical, metaiodobenzylguanidine (131I-mIBG). I have led multiple phase I, II, and III clinical trials for advanced neuroblastoma, as well as collaborative work in pre-clinical studies of combination targeted therapy, mechanisms of MIBG uptake, and MIBG imaging for dosimetry and prognosis. I have also collaborated closely on preclinical work in MYCN and PI3 kinase pathways.


Bryn Mawr College, Bryn Mawr, PA, B.A., 1969, Chemistry
University of Pennsylvania, Philadelphia, PA, M.D., 1973, Medicine
University of Colorado, Denver, CO, Resident, 1976, Pediatrics
University of California, San Francisco, CA, Fellow, 1979, Pediatric Hematology/Oncology

Professional Experience

  • 1980-87
    Assistant Professor in Residence, Pediatrics, Univ. of Calif., San Francisco
  • 1987-93
    Associate Professor in Residence, Pediatrics, Univ. of Calif., San Francisco
  • 1991-2001
    Chair, Neuroblastoma Strategy Group, Children's Cancer Group
  • 1992-present
    Chief, Pediatric Clinical Oncology, Univ. of California, San Francisco
  • 1993-present
    Professor, Pediatrics, Univ. of California, San Francisco
  • 2000-2001; 2009-10
    Visiting Professor, Institut Curie, Paris
  • 2003-2006
    Fellowship Director, Pediatric Hematology/Oncology, Univ. of California, San Francisco
  • 2003-2013
    Division Chief, Pediatric Hematology/Oncology, Univ. of California, San Francisco
  • 2005-present
    Leader, Pediatric Malignancies Program in the HDF Comprehensive Cancer Center

Honors & Awards

  • 1992
    American Cancer Society Scholar Award
  • 2000-01
    Bourse Henri de Rothschild for Neuroblastoma Research Award
  • 2006
    Mildred V. Strouss Endowed Chair in Translational Research
  • 2006-12
    SIOP North American continental President
  • 2007
    Hadassah Doctor of Distinction Award
  • 2012-16
    President, Advances in Neuroblastoma Research Association

Selected Publications

  1. Huibregtse KE, Vo KT, DuBois SG, Fetzko S, Neuhaus J, Batra V, Maris JM, Weiss B, Marachelian A, Yanik GA, Matthay KK. Incidence and risk factors for secondary malignancy in patients with neuroblastoma after treatment with (131)I-metaiodobenzylguanidine. Eur J Cancer. 2016 Aug 26; 66:144-152.
    View on PubMed
  2. Edmondson DA, Karski EE, Kohlgruber A, Koneru H, Matthay KK, Allen S, Hartmann CL, Peterson LE, DuBois SG, Coleman MA. Transcript Analysis for Internal Biodosimetry Using Peripheral Blood from Neuroblastoma Patients Treated with (131)I-mIBG, a Targeted Radionuclide. Radiat Res. 2016 Sep; 186(3):235-44.
    View on PubMed
  3. Morgenstern DA, London WB, Stephens D, Volchenboum SL, Simon T, Nakagawara A, Shimada H, Schleiermacher G, Matthay KK, Cohn SL, Pearson AD, Irwin MS. Prognostic significance of pattern and burden of metastatic disease in patients with stage 4 neuroblastoma: A study from the International Neuroblastoma Risk Group database. Eur J Cancer. 2016 Sep; 65:1-10.
    View on PubMed
  4. Lee JS, Matthay K, DuBois SG, Roy-Burman A. Reply: Is Extended Sedation Necessary for Young Children Receiving High-Dose (131) I-MIBG Therapy? Pediatr Blood Cancer. 2016 Oct; 63(10):1868.
    View on PubMed
  5. Vo KT, Matthay KK, DuBois SG. Targeted antiangiogenic agents in combination with cytotoxic chemotherapy in preclinical and clinical studies in sarcoma. Clin Sarcoma Res. 2016; 6:9.
    View on PubMed
  6. Villablanca JG, Volchenboum SL, Cho H, Kang MH, Cohn SL, Anderson CP, Marachelian A, Groshen S, Tsao-Wei D, Matthay KK, Maris JM, Hasenauer CE, Czarnecki S, Lai H, Goodarzian F, Shimada H, Reynolds CP. A Phase I New Approaches to Neuroblastoma Therapy Study of Buthionine Sulfoximine and Melphalan With Autologous Stem Cells for Recurrent/Refractory High-Risk Neuroblastoma. Pediatr Blood Cancer. 2016 Aug; 63(8):1349-56.
    View on PubMed
  7. DuBois SG, Marachelian A, Fox E, Kudgus RA, Reid JM, Groshen S, Malvar J, Bagatell R, Wagner L, Maris JM, Hawkins R, Courtier J, Lai H, Goodarzian F, Shimada H, Czarnecki S, Tsao-Wei D, Matthay KK, Mosse YP. Phase I Study of the Aurora A Kinase Inhibitor Alisertib in Combination With Irinotecan and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma: A NANT (New Approaches to Neuroblastoma Therapy) Trial. J Clin Oncol. 2016 Apr 20; 34(12):1368-75.
    View on PubMed
  8. Vo KT, Edwards JV, Epling CL, Sinclair E, Hawkins DS, Grier HE, Janeway KA, Barnette P, McIlvaine E, Krailo MD, Barkauskas DA, Matthay KK, Womer RB, Gorlick RG, Lessnick SL, Mackall CL, DuBois SG. Impact of Two Measures of Micrometastatic Disease on Clinical Outcomes in Patients with Newly Diagnosed Ewing Sarcoma: A Report from the Children's Oncology Group. Clin Cancer Res. 2016 Jul 15; 22(14):3643-50.
    View on PubMed
  9. Balkin EM, Thompson D, Colson KE, Lam CG, Matthay KK. Physician Perspectives on Palliative Care for Children With Neuroblastoma: An International Context. Pediatr Blood Cancer. 2016 May; 63(5):872-9.
    View on PubMed
  10. Thompson D, Vo KT, London WB, Fischer M, Ambros PF, Nakagawara A, Brodeur GM, Matthay KK, DuBois SG. Identification of patient subgroups with markedly disparate rates of MYCN amplification in neuroblastoma: A report from the International Neuroblastoma Risk Group project. Cancer. 2016 Mar 15; 122(6):935-45.
    View on PubMed
  11. Lee JS, Wu R, Wong T, DuBois SG, Matthay K, Gustafson C, Hawkins R, Roy-Burman A. Extended Sedation With Continuous Midazolam or Dexmedetomidine Infusion for Young Children Receiving (131) I-MIBG Radiopharmaceutical Therapy for Advanced Neuroblastoma. Pediatr Blood Cancer. 2016 Mar; 63(3):471-8.
    View on PubMed
  12. Cohler C, Jumanne S, Kaijage J, DuBois SG, Scanlan P, Matthay KK. Evaluation and Outcome of Central Nervous System Involvement in Pediatric Acute Lymphoblastic Leukemia in Dar es Salaam, Tanzania. Pediatr Blood Cancer. 2016 Mar; 63(3):458-64.
    View on PubMed
  13. Trieu M, DuBois SG, Pon E, Nardo L, Hawkins RA, Marachelian A, Twist CJ, Park JR, Matthay KK. Impact of Whole-Body Radiation Dose on Response and Toxicity in Patients With Neuroblastoma After Therapy With (131) I-Metaiodobenzylguanidine (MIBG). Pediatr Blood Cancer. 2016 Mar; 63(3):436-42.
    View on PubMed
  14. Temple W, Mendelsohn L, Kim GE, Nekritz E, Gustafson WC, Lin L, Giacomini K, Naranjo A, Van Ryn C, Yanik GA, Kreissman SG, Hogarty M, Matthay KK, DuBois SG. Vesicular monoamine transporter protein expression correlates with clinical features, tumor biology, and MIBG avidity in neuroblastoma: a report from the Children's Oncology Group. Eur J Nucl Med Mol Imaging. 2016 Mar; 43(3):474-81.
    View on PubMed
  15. Pinto NR, Applebaum MA, Volchenboum SL, Matthay KK, London WB, Ambros PF, Nakagawara A, Berthold F, Schleiermacher G, Park JR, Valteau-Couanet D, Pearson AD, Cohn SL. Advances in Risk Classification and Treatment Strategies for Neuroblastoma. J Clin Oncol. 2015 Sep 20; 33(27):3008-17.
    View on PubMed
  16. Zhou MJ, Doral MY, DuBois SG, Villablanca JG, Yanik GA, Matthay KK. Different outcomes for relapsed versus refractory neuroblastoma after therapy with (131)I-metaiodobenzylguanidine ((131)I-MIBG). Eur J Cancer. 2015 Nov; 51(16):2465-72.
    View on PubMed
  17. Anderson CP, Matthay KK, Perentesis JP, Neglia JP, Bailey HH, Villablanca JG, Groshen S, Hasenauer B, Maris JM, Seeger RC, Reynolds CP. Pilot study of intravenous melphalan combined with continuous infusion L-S,R-buthionine sulfoximine for children with recurrent neuroblastoma. Pediatr Blood Cancer. 2015 Oct; 62(10):1739-46.
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  18. Huang SY, Bolch WE, Lee C, Van Brocklin HF, Pampaloni MH, Hawkins RA, Sznewajs A, DuBois SG, Matthay KK, Seo Y. Patient-specific dosimetry using pretherapy [¹²4I]m-iodobenzylguanidine ([¹²4I]mIBG) dynamic PET/CT imaging before [¹³¹I]mIBG targeted radionuclide therapy for neuroblastoma. Mol Imaging Biol. 2015 Apr; 17(2):284-94.
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  19. Parikh NS, Howard SC, Chantada G, Israels T, Khattab M, Alcasabas P, Lam CG, Faulkner L, Park JR, London WB, Matthay KK. SIOP-PODC adapted risk stratification and treatment guidelines: Recommendations for neuroblastoma in low- and middle-income settings. Pediatr Blood Cancer. 2015 Aug; 62(8):1305-16.
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  20. DuBois SG, Groshen S, Park JR, Haas-Kogan DA, Yang X, Geier E, Chen E, Giacomini K, Weiss B, Cohn SL, Granger MM, Yanik GA, Hawkins R, Courtier J, Jackson H, Goodarzian F, Shimada H, Czarnecki S, Tsao-Wei D, Villablanca JG, Marachelian A, Matthay KK. Phase I Study of Vorinostat as a Radiation Sensitizer with 131I-Metaiodobenzylguanidine (131I-MIBG) for Patients with Relapsed or Refractory Neuroblastoma. Clin Cancer Res. 2015 Jun 15; 21(12):2715-21.
    View on PubMed

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