Pediatric Hematology-Oncology - UT Southwestern

Stephen X. Skapek, M.D.
Professor
Director, Pediatric Hematology-Oncology
Pediatric Hematology-Oncology
2011 Academic Review
This past academic year brought substantial changes in leadership in the Division
of Pediatric Hematology-Oncology, as well as continuing renovations to the patient
care facilities in the Center for Cancer and Blood Disorders (CCBD). The long-term
goal is for Children’s Medical Center’s (CMC) 6 th floor to be entirely devoted to the
CCBD. After renovating the Stem Cell Transplant unit over recent years, Phase I of
the current renovations are being completed now. This phase, just the first of a fourphase
project, represents approximately 14,000 square feet of new outpatient clinic
space in the B Tower of CMC. It is scheduled to open for our hematology patients
in February 2012. Phase II, scheduled to begin in April 2012, involves renovation of
another approximately 14,000 square feet of space for additional inpatient beds and an
18-bay infusion area which will serve as a day hospital for CCBD patients.
Dr. Stephen Skapek has begun to lead the Division through a process of critical
appraisal of academic programs in the CCBD. This effort will be dually focused on
the training programs and the research efforts. The former is primarily represented
by our ACGME-accredited, three-year hematology/oncology fellowship program.
We continue to accept four fellows per year into this program. Research efforts are
composed of clinical, translational and laboratory work addressing fundamental
problems in hematology, oncology and stem cell transplantation. By a combination
of critical re-direction of efforts, targeted recruitment of physician-scientists and clinical scholars, and rejuvenated efforts
to leverage phenomenal strengths already on the UT Southwestern campus, the Division and the CCBD look forward to
tremendous growth in what are already very strong academic programs.
Faculty
At the close of 2011, the Division included 24 faculty members ranging in rank from Instructor to Professor. With the
growth of the program and aforementioned changes in leadership, each faculty member is now aligned with specific clinical
areas. This realignment places 12 faculty members in the oncology program, five in the hematology program, four within
the stem cell transplant group, and three “general” hematologists/oncologists with full-time clinical responsibilities on
the Legacy campus of CMC. Organization along these clinical service lines allows individual faculty members to develop
greater, disease-specific expertise, and it also fosters opportunities for academic programs to emerge from the clinical
programs.
In addition to Dr. Skapek, new faculty members include the following:
Jeffrey Magee, M.D., Ph.D.
• Dr. Magee is a physician-scientist who joined the faculty as an Instructor in the Division of Hematology/
Oncology after completing Pediatric Hematology/Oncology training at the University of Michigan.
As a member of the oncology program, his primary responsibilities are to carry out laboratory-based
research into the biology of leukemia cells and how this biology is coupled to normal developmental
changes that occur with age. This work is carried out under the direct guidance of Dr. Sean Morrison,
Director of the Children’s Research Institute.
Alecia Nero, M.D.
• Dr. Nero is a clinical scholar who joined our faculty as an Assistant Professor with joint appointments
in the Departments of Medicine and Pediatrics. She completed Medicine/Pediatrics residency and
Medical Hematology training at Case Western Reserve University prior to joining our faculty. As
a member of the hematology program, her dual academic and clinical goals are to develop a robust
program to facilitate the transition of care for adolescents and young adults with sickle cell disease
into adult programs on our campus. To accomplish this important work, she is forging collaborative
mentor relationships with leaders in hematology and the Department of Clinical Sciences.
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Tanya Watt, M.D.
• Dr. Watt joined the Division as an Assistant Professor of Pediatrics. She completed her Pediatric
Hematology/Oncology training at the Children’s Hospital of Atlanta, during which time she also
received a Masters of Science in Clinical Research as she opened Phase I clinical trials for children
with cancer. She has joint responsibilities with the oncology and stem cell programs, which mesh
perfectly with her primary goals of further strengthening our clinical and translational research
efforts in neuroblastoma.
Yanbin Zheng, Ph.D.
• Dr. Yanbin Zheng, a highly-skilled biologist with overlapping interests in cancer and developmental
biology, moved from th University of Chicago Biological Sciences Division to join the Division of
Hematology/Oncology as an Assistant Professor. He is working closely with Dr. Stephen Skapek,
leading molecular and cellular biology studies investigating certain genes that help to block cancer
formation in children.
The remaining division faculty members are:
James Amatruda, M.D., Ph.D.
Andrew Koh, M.D.
Assistant Professor, Pediatrics, Molecular Biology and Internal Assistant Professor, Pediatrics, Microbiology and Simmons
Medicine
Cancer Center
Kristine Appel, M.D.
Patrick Leavey, M.D.
Assistant Professor
Associate Professor
Victor Aquino, M.D.
Timothy McCavit, M.D.
Associate Professor
Assistant Professor
Daniel C. Bowers, M.D.
Zora R. Rogers, M.D.
Associate Professor
Professor
George R. Buchanan, M.D.
Tiffany Simms-Waldrip, M.D.
Professor
Assistant Professor
MaryEllen Cavalier, M.D.
Tamra Slone, M.D.
Assistant Professor
Assistant Professor
Alexander Gozman, M.D.
Martha Stegner, M.D.
Assistant Professor
Assistant Professor
Paul Harker-Murray, M.D., Ph.D.
Jon Wickiser, M.D.
Assistant Professor
Assistant Professor
Janna Journeycake, M.D.
Naomi J. Winick, M.D.
Associate Professor
Professor
Laura Klesse, M.D., Ph.D.
Assistant Professor
Major accomplishments of existing faculty members include the following:
• James Amatruda published a manuscript in Proceedings of the National Academy of Sciences describing a new
zebrafish model of malignant germ cell tumor driven by deregulation of the Type 1B Bone Morphogenetic Protein
(BMP) receptor.
• George Buchanan continues to Co-Chair the NHLBI’s panel to create evidence-based guidelines for management
of children and adults with sickle cell disease. He also serves as Director of the American Society of Pediatric
Hematology-Oncology board review course held every two years in Dallas.
• Andrew Koh led our Stem Cell Transplant program through a successful re-accreditation with the Foundation for
Accreditation of Cellular Therapy organization – a key benchmark for our program.
• Patrick Leavey was appointed as Co-Chairman of the Children’s Oncology Group Ewing Sarcoma AEWS 1031 Study
Committee, an international Phase II clinical trial for children with localized Ewing Sarcoma.
• Zora Rogers co-authored a manuscript in the Lancet describing her work on the BABY HUG study, and she was
elected as Chair of the BABY HUG Followup Study, funded by the NHLBI.
• Tiffany Simms-Waldrip was named an American Society of Blood and Marrow Transplantation Clinical Research
Training Scholar.
• Tanya Watt was named a Dedman Family Scholar in Clinical Care at UT Southwestern.
• Naomi Winick led our Division to become a part of the Therapeutic Advances in Leukemia Consortium (TACL),
a national organization that will bring early-phase therapeutic agents to the pediatric leukemia program at UT
Southwestern.
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Research Activities
Carrying out research activities continues to represent a key part of our mission. Dr. Skapek joined the faculty with the
intention to recruit up to five additional physician-scientists who will complement laboratory-based scientists already in
the division. Indeed, essentially all of the faculty members in the Division are currently engaged in clinical, laboratory and
translational research addressing major gaps in our understanding of cancer and blood disorders in children.
Clinical Research
Clinical research programs include our participation in:
• NIH-sponsored cooperative group trials such as BABY HUG and many trials operated through the Children’s Oncology
Group and the aforementioned TACL Phase I/II study group;
• industry-sponsored therapeutics trials;
• other multi-site collaborative studies; and
• investigator-initiated trials at our own institutions.
Topics range from experimental therapeutics for cancer, stem cell transplant and sickle cell disease and thalassemia to
supportive care and health outcomes research addressing topics such as depression and fatigue in cancer patients and
metabolic syndrome in childhood cancer survivors. Nearly all of the current research programs are supported by funds
from a wide range of extramural sources. Of particular note, over one-third of the faculty members in the Division hold
leadership positions within national clinical trial organizations like the Children’s Oncology Group.
Laboratory-based Research
Laboratory-based research programs in the Division also cover a wide-range of topics. Examples include studying:
• pathogen- and host-dependent determinants of invasive fungal and bacterial disease in immune compromised
patients;
• molecular pathogenesis of malignant germ cell tumors using high-resolution genomics tools;
• the role of BMP signaling in germ cell tumor using a novel non-mammalian model in zebrafish;
• molecular pathogenesis of Ewing sarcoma family tumors and other soft tissue sarcomas in zebrafish and mouse
models;
• investigating molecular factors that drive initial phase of skeletal myogenesis, a step towards differentiation-inducing
therapeutics for rhabdomyosarcoma;
• the role that the ARF tumor suppressor gene plays to guide vascular remodeling in the developing eye; and
• how developmental changes in hematopoietic stem cells are recapitulated in leukemia arising from infants versus
older children.
As with the clinical research, nearly all of the laboratory-based efforts are supported by extramural funds, such as from the
Cancer Prevention and Research Institute of Texas (CPRIT), National Cancer Institute (NCI), National Eye Institute (NEI),
National Heart, Lung, and Blood Institute (NHLBI) and numerous other charitable foundations such as St. Baldrick’s
Foundation, Wipe Out Kids Cancer, Hyundai Hope on Wheels and the Children’s Cancer Fund.
Translational Research
Several new translational research initiatives highlight the unparalleled potential brought together by the pooled clinical
research resources from Children’s Medical Center, the scientific strengths in the Division, and the broader UT Southwestern
Medical Center Campus. Examples include new projects investigating:
• the potential for microRNA species to serve as novel biomarkers of anthracycline-induced cardiac toxicity;
• serum proteins that can serve as early markers of metabolic syndrome in young children following treatment for
brain tumor;
• structural genetic changes – such as DNA copy number gains or losses – in relapsed medulloblastoma and various
types of malignant germ cell tumors; and
• the presence of potentially-actionable mutations in non-rhabdomyosarcoma soft tissue sarcoma specimens.
In addition to being supported by extramural sources, these new translational programs are gratifying because they bring
together investigators with expertise in pathology, cardiology and cardiac developmental biology, metabolism and genomics
and systems biology from across the UT Southwestern campus and nationally.
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New ResearchGrants
Over 40 research grants were submitted from members of the Division this past year. Numerous were awarded from
the NHLBI to support BABY HUG and TwiTCH clinical trials (Zora Rogers); Department of Defense to support
neurofibromatosis clinical trial (Laura Klesse); St. Baldrick’s Foundation to support molecular genetics studies of soft tissue
sarcoma (Stephen Skapek); Hyundai Hope on Wheels to support molecular biology studies of germ cell tumor (James
Amatruda) and survivorship studies (Raven Cooksey); and Wipe Out Kids Cancer to support molecular genetics studies of
medulloblastoma (Laura Klesse) and clinical/translational studies of metabolic syndrome in survivors of childhood brain
tumor (Raven Cooksey/Daniel Bowers).
Clinical Activities
The staff at UT Southwestern and Children’s who support Hematology-Oncology clinical care and research number more
than 150, including administrative assistants, clinical and research nurses, mid-level providers, pharmacists, social workers,
psychologists, child-life experts and clinical research associates. In addition to UT Southwestern faculty members, CCBD
leadership includes Kaye Schmidt, Senior Director; Barbara Obert, Operations Director; and Kathy Carlisle, Nursing
Director.
Clinical services are largely divided along three service lines of Hematology, Oncology, and Stem Cell Transplant, which are
led by Drs. Zora Rogers, Jonathan Wickiser and Andrew Koh, respectively. Dr. Patrick Leavey acts as Associate Director of
Clinical Affairs, providing oversight of clinical operations in each of the Divisions and on the Legacy campus. Consistent
with the overall program goals, individual physician faculty members are now aligned with one of the service lines to
assist their development of greater expertise in a particular
area. Disease-specific clinical programs are developing
Outpatient Visits - Dallas
within the larger services. These include a leukemia program,
neuroblastoma program, sarcoma program, neuro-oncology
program, neurofibromatosis and cancer susceptibility program,
and the “After Cancer Experience” (ACE) survivorship program.
As these groups coalesce, physician leaders are developing
specific agendas to enhance clinical care, develop new research
opportunities, and extend the reach of our program. In all
cases, members of specific programs meet weekly or bi-weekly
in multidisciplinary meetings focused primarily on reviewing
clinical care, while also providing academic opportunities.
Clinical care is provided in both inpatient and outpatient
facilities on the Dallas and the Legacy campuses. The outpatient
care on the Dallas campus has been provided in the Bright
Building, adjacent to the Hospital. The first phase opening the
new clinic space on the 6 th floor of the hospital will leave the Hematology and Oncology clinics separated for the next year
as renovations on the 6 th floor are completed. Inpatient care on the Dallas campus is primarily centered on the 24 beds on
the 6th floor of the D Tower; however, as the average daily census is close to 30, inpatients care is routinely extended to other
floors in the hospital. Twenty-five to forty inpatients with cancer and hematologic disorders are usually under direct care
in a dedicated 24-bed unit in Tower D and on other floors throughout the hospital.
Inpatient and outpatient activities on the Legacy campus are primarily provided by three full-time clinical faculty (Kristine
Appel, MaryEllen Cavalier and Alexander Gozman); several additional
Dallas-based faculty hold bi-weekly or monthly clinics at Legacy.
Outpatient Visits - Legacy
Sixty to seventy-five patients are seen daily in outpatient clinic on the Dallas
campus, which is staffed by three or four full-time faculty members, several
fellows and mid-level providers who are now oriented along hematology,
oncology and stem cell transplant service lines. Many of the children
receive bone marrow aspiration, lumbar puncture, and intravenous drug
treatments or blood product transfusions as part of their regular outpatient
visit.
The inpatient care on the Dallas campus is provided by four separate
inpatient services (Oncology A, Oncology B, Hematology and Stem Cell
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Transplant). Each service is staffed by an attending physician, who typically leads a team containing a first-year hematology/
oncology fellow, pediatric resident housestaff, medical students, pharmacists, nutrition experts, social workers, and nurse
case managers. To meet the changing levels of support provided by pediatric resident trainees, the Oncology B team will be
fully supported by mid-level providers working with an attending physician.
Clinical Program Growth and Regional Outreach
As is clear from the figures below, total clinical volume and workload continue to increase. This continued growth raises
new challenges as well as opportunities. Among them are our plans to strategically consider (a) increased utilization of
the Legacy campus for patients with certain types of problems; and (b) opportunities to deliver more care in the outpatient
setting by taking advantage of increased clinic space and a new day-hospital facility planned in the next phases of renovations.
Moreover, as the program grows, we are considering mechanisms to assure, and improve where needed, communications
with referring providers.
Hematology
Stem Cell Transplants
Total New Patients
Oncology
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Education and Training
Our Division continues to support an ACGME-accredited Pediatric Hematology/
Oncology Fellowship training program, with up to four fellows selected for each
year of a three-year program. We continue to strive to train exceptional clinicians
who will be future leaders in Pediatric Hematology/Oncology. Graduates this
past year have taken faculty positions at prestigious institutions such as St. Jude
Children’s Research Hospital and Cincinnati Children’s Hospital Medical Center.
Four of the eight current upper-level clinical fellows are funded by extramural
grants from:
• St Baldrick’s Foundation (Rachel Thienprayoon),
• Hyundai Hope on Wheels (Raven Cooksey),
• Children’s Cancer Fund (Scott Furlan), and
• a T32 grant from the Cancer Center (Kenneth Chen).
Another laboratory-based postdoctoral fellow (Stefanie Leacock) is funded
by a grant from the American Cancer Society for her work in the Amatruda
laboratory.
Three fellows graduated in 2011:
• Carrye Cost went to Cincinnatti Children’s Hospital as an Instructor in the Department of Pediatrics at the University
of Cincinnatti, Ohio.
• Amy Fowler accepted a position at Austin Regional Clinic, Austin, Texas.
• Nicholas Fustino accepted a position at Coventry Health Care of Iowa in Des Moines.
All clinical faculty members in the Division of Hematology-Oncology embrace the commitment to teaching medical
students, residents, fellows, primary care and referring physicians, and other healthcare professionals. Teaching is done
through one-on-one interactions, small groups, seminars and formal lectures in a variety of settings. In addition to clinical
teaching, faculty members are also affiliated with the Genetics and Development and the Cancer Biology programs, and
provide formal lectures to graduate students in the Biological Sciences.
Clinical teaching conferences within the Division are held from several times each week to monthly. Regular conferences
include Pediatric Hematology-Oncology Grand Rounds, Oncology Tumor Board, Neuro-oncology Tumor Board,
Hematology Case Conference, and Journal Club. Further, laboratory science education is provided by monthly conferences
led by physician-scientists and aimed at providing guidance to clinical fellows and junior faculty members. Lastly, for more
than a decade, the Division has offered an annual Continuing Medical Education course each October at UT Southwestern
aimed at primary care physicians. Between 50 and 70 attendees participate regularly and provide uniformly positive reviews.
Presentations
Dr. Carrye Cost
Oral presentation at the 2 nd Annual
Pediatric Senior Fellow Research Day
James Amatruda, M.D., Ph.D.
• “The Role of BMP Signaling in Germ Cell Tumorigenesis,” International Meeting on Childhood Germ Cell Tumors,
UT Southwestern, March 2011
• “Genomic Approaches to Target Identification in Childhood Germ Cell Tumors,” Children’s Oncology Group Spring
Meeting, Los Angeles, CA, March 2011
• “Modeling Childhood Cancers in the Zebrafish,” Grand Rounds, Division of Pediatric Hematology-Oncology,
UT MD Anderson Cancer Center, Houston, TX, April 2011
• “BMP Signaling in Germline Development and Germ Cell Tumorigenesis,” Zebrafish Disease Models 4: Cancer and
Immunity Models, Edinburgh, UK, July 2011 (invited speaker)
• “Genetic and Translational Models of Childhood Cancer,” University of Utah, Huntsman Cancer Institute, Center for
Children’s Cancer Retreat, Salt Lake City, UT, October 2011 (keynote speaker)
• “Genetic and Translational Models of Childhood Cancer,” UT MD Anderson Carcinogenesis Research Center,
Smithville, TX, November 2011
• “Developmental Signaling Pathways in Germ Cell Tumors,” 2 nd International Meeting on Germ Cell Tumors and AYA
Oncology, Cambridge, UK, December 2011 (invited speaker)
Victor Aquino, M.D.
• “Hematopoietic Stem Cell Transplantation for Congenital Immunodeficiencies,” Pediatric Allergy and Immunology
Conference, UT Southwestern, January 2011
• “Engaging Physicians in the Work of the Collaborative,” Pediatric Hematology/Oncology Central Line-associated
Blood Stream Infection (CLABSI) Workshop, Chicago, IL, March 2011
• “Histiocytic Disorders,” Pediatric Hematology/Oncology Nursing Teaching Days, Children’s Medical Center, October
and November 2011
Daniel Bowers, M.D.
• “Survivors of Childhood Brain Tumors: Entering the Adult World: Challenges and Avenues for Intervention,”
Innovations in Cancer Prevention and Research Conference, The Cancer Prevention and Research Institute of Texas
(CPRIT), Austin, TX, November 2011
George Buchanan, M.D.
• “Being Keen on the Spleen,” Visiting Professor, Rady Children’s Hospital and University of California, San Diego, CA,
January 2011
• “Nutritional Anemia,” American Society of Pediatric Hematology/Oncologh (ASPHO) Review Course, Dallas, TX,
February 2011
• “Iron Deficiency During Childhood,” Visiting Professor, Children’s Hospital of New Jersey/Robert Wood Johnson
School of Medicine, Newark, NJ, April 2011
• “Sickle Cell Disease Research Update,” Texas Department of State Health Services Newborn Screening Program
Annual Consultants Meeting, Austin, TX, April 2011
• “Immune Thrombocytopenia During Childhood,” Symposium Honoring the Accomplishments of Dr. Victor
Blanchette, Hospital for Sick Children, Toronto, Ontario, Canada, May 2011
• “Diagnosis and Management of Anemia in Infants and Children,” University of South Dakota School of Medicine,
Sioux Falls, SD, September 2011 (visiting professor)
• “Progress and Management of Patients with Sickle Cell Disease,” Tarrant County Health Department Annual
Symposium, Fort Worth, TX, September 2011
• “Anemia in Childhood: Iron Deficiency and Beyond,” American Academy of Pediatrics National Conference and
Exposition, Boston, MA, October 2011
• “Hereditary Hemochromatosis: The Other Iron Problem,” Annual
Hematology-Oncology CME Course, Children’s Medical Center, November
2011
MaryEllen Cavalier, M.D.
• “Iron Deficiency and Other Nutritional Anemias,” CME Event, Trinity
Mother Frances Medical Center, Tyler, TX, August 2011
Paul Harker-Murray, M.D., Ph.D.
• “Lymphoma,” American Society of Pediatric Hematology and Oncology,
Review Course, February 2011
• “Lymphadenopathy and Lymphoma,” Hematology/Oncology CME Course,
UT Southwestern, October 2011
Janna Journeycake, M.D., M.S.C.S.
• “Update on Inhibitors,” Texas Hemophilia Regional Conference, San
Antonio, TX, June 2011
• “Immunosuppression for Inhibitors,” National Hemophilia Foundation
Inhibitor Summit, San Francisco, CA, July 2011
Andrew Koh, M.D.
Dr. Amy Fowler
Poster presentation at the 2 nd Annual
Pediatric Senior Fellow Research Day
• “Pseudomonas Aeruginosa and Candida Albicans: Gastrointestinal Colonization and Dissemination,” Meidcal Scientist
Training Program (MSTP), UT Southwestern, January 2011
• “Pseudomonas aeruginosa and Candida albicans: Gastrointestinal Colonization and Dissemination,” UT Arlington,
Department of Biology, Colloquium Series, Arlington, TX, February 2011
• “Hematopoietic Stem Cell Transplant Program at Children’s and UT Southwestern,” Children’s Medical Center Annual
Leadership Retreat, Dallas, TX, March 2011
Patrick Leavey, M.D.
• “New Approaches to Relapsed Ewing Sarcoma,” Medical College of Wisconsin and Children’s Hospital of Wisconsin,
February 2011
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• “Report of Fellow’s Survey Regarding Scholarship Choices,” Presented by
invitation to the Board of Directors for the American Society of Pediatric
Hematology/Oncology, Baltimore, MD, May 2011
• “Alternative Pathways in Fellowship Training and Education – Educator
Pathways,” American Society of Hematology Annual Meeting, San Diego,
CA, December 2011
Stephen Skapek, M.D.
• “The Arf Tumor Suppressor Gene: At the Intersection of Cancer and
Developmental Biology,” Hematology/Oncology Grand Rounds, Texas
Children’s Hospital, Houston, TX, November 2011
Awards and Honors
James Amatruda, M.D., Ph.D.
• Outstanding Teacher Award, 2010-2011 MS1 Class
Daniel Bowers, M.D.
• Best Pediatric Specialists in Dallas, D Magazine
George Buchanan, M.D.
• Best Pediatric Specialists in Dallas, D Magazine
• Top US Doctors, U.S. News
• Selected by Nominating Committee of the American Society of Hematology to serve as one of two candidates for
election as Vice President-President Elect-President of the American Society of Hematology from 2012 through 2014
(offer declined)
Paul Harker-Murray, M.D., Ph.D.
• UT Southwestern Advocacy Mentor Award for the Project “A Partnership between Parkland Memorial Hospital and
the Texas Cord Blood Bank for Collection and Storage of Umbilical Cord Blood that Reflects the Rich Ethnic Diversity
of Texans
Janna Journeycake, M.D., M.S.C.S.
• Best Pediatric Specialists in Dallas, D Magazine
Patrick Leavey, M.D.
• Best Pediatric Specialists in Dallas, D Magazine
Zora Rogers, M.D.
• Best Pediatric Specialists in Dallas, D Magazine
Tiffany Simms-Waldrip, M.D.
• American Society of Blood and Marrow Transplantation Clinical Research Training Scholar
Zora Rogers, M.D.
• Election to the Board of Trustees American Society of Pediatric Hematology/Oncology (ASPHO)
• Election to the National Executive Committee American Academy of Pediatrics, Section on Pediatric Hematology/
Oncology (SOHO)
• Member, Editorial Board, The Journal of Pediatrics
Tanya Watt, M.D., MS.c.
• Dedman Family Scholar in Clinical Care
Naomi Winick, M.D.
• Best Pediatric Specialists in Dallas, D Magazine
• Texas Super Doctors, Texas Monthly
• Top US Doctors, U.S. News
Fellows:
Raven Cooksey, M.D.
• “Call for Cash” Recipient, Children’s Cancer Fund
• Hyundai Hope on Wheels Research Scholar, Hyundai Motors America
Scott Furlan, M.D.
• 2011 Research Fellow Award, Children’s Cancer Fund
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Dr. Nicholas Fustino
Poster presentation at the 2 nd Annual
Pediatric Senior Fellow Research Day
Publications
Articles
1. Al-Tahan A, Sarkis O, Harajly M, Baghdadi OK, Zibara K, Boulos F, Dighe D, Kregel S, Bazarbachi A, El-Sabban M,
Skapek SX, Saab R. Retinoic acid fails to induce cell cycle arrest with myogenic differentiation in rhabdomyosarcoma.
Pediatr Blood Cancer;58:877-84.
2. Bowers DC, Kucejova B, Margraf L, Gargan L, Brugarolas J. mTORC1 activation in childhood ependymoma and
response to sirolimus. J Neurooncol;103:797-801.
3. Boztug K, Rosenberg PS, Dorda M, Banka S, Moulton T, Curtin J, Rezaei N, Corns J, Innin JW, Avci Z, Tran HC,
Pellier I, Pierani P, Fruge R, Rarvaneh N, Mamishi S, Mody R, Darbyshire P, Motwani J, Murray J, Buchanan GR, et al.
Extended spectrum of human glucose-6-phosphatase catalytic subunit 3 deficiency: novel genotypes and phenotypic
variability in severe congenital neutropenia. J Pediatr;160:679-83 e2.
4. Bussel JB, Buchanan GR, Nugent DJ, et al. A randomized, double-blind study of romiplostim to determine its safety
and efficacy in children with immune thrombocytopenia. Blood;118:28-36.
5. Chen KS, Neunert CE, Crary SE, Buchanan GR. Hemolytic non-uremic syndrome. Pediatr Blood Cancer.
6. Cox JA, DeMasi J, McCollom S, Jackson G, Scothorn D, Aquino VM. The diagnostic utility of routine chest radiography
in the evaluation of the initial fever in patients undergoing hematopoietic stem cell. Pediatr Blood Cancer;57:666-8.
7. Crary SE, Hall K, Buchanan GR. Intravenous iron sucrose for children with iron deficiency failing to respond to oral
iron therapy. Pediatr Blood Cancer;56:615-9.
8. Crary SE, Ramaciotti C, Buchanan GR. Prevalence of pulmonary hypertension in hereditary spherocytosis. Am J
Hematol;86:E73-6.
9. Dale JC, Cochran CJ, Roy L, Jernigan E, Buchanan GR. Health-related quality of life in children and adolescents with
sickle cell disease. J Pediatr Health Care;25:208-15.
10. Dampier C, LeBeau P, Rhee S, Lieff S, Kesler K, Ballas S, Rogers Z, Wang W, for the Comprehensive Sickle Cell Centers
(CSCC) Clinical Trial Consortium (CTC) Site Investigators. Health-related quality of life in adults with sickle cell
disease (SCD): a report from the comprehensive sickle cell centers clinical trial consortium. Am J Hematol;86:203-5.
11. Fustino N, Rakheja D, Ateek CS, Neumann JC, Amatruda JF. Bone morphogenetic protein signalling activity
distinguishes histological subsets of paediatric germ cell tumours. Int J Androl;34:e218-33.
12. Grace RF, Bennett CM, Ritchey AK, Jeng M, Thornburg CD, Lambert MP, Neier M, Recht M, Kumar M, Blanchette
V, Klaassen RJ, Buchanan GR, et al. Response to steroids predicts response to rituximab in pediatric chronic immune
thrombocytopenia. Pediatr Blood Cancer;58:221-5.
13. Hajeri VA, Amatruda JF. Studying synthetic lethal interactions in the zebrafish system: insight into disease genes and
mechanisms. Dis Model Mech;5:33-7.
14. Jacob LS, Wu X, Dodge ME, Fan CW, Kulak O, Chen B, Tang W, Wang B, Amatruda JF, Lum L. Genome-wide RNAi
screen reveals disease-associated genes that are common to Hedgehog and Wnt signaling. Sci Signal;4:ra4.
15. Kolins JA, Zbylut C, McCollom S, Aquino VM. Hematopoietic stem cell transplantation in children. Crit Care Nurs
Clin North Am;23:349-76.
16. Koral K, Roy D, Timmons CF, Gargan L, Bowers DC. Low-grade bone lesions in survivors of childhood
medulloblastoma/primitive neuroectodermal tumor. Acad Radiol;19:35-9.
17. Kurtzberg J, Asselin B, Bernstein M, Buchanan GR, Pollock BH, Camitta BM. Polyethylene Glycol-conjugated
L-asparaginase versus native L-asparaginase in combination with standard agents for children with acute
lymphoblastic leukemia in second bone marrow relapse: a Children’s Oncology Group Study (POG 8866). J Pediatr
Hematol Oncol;33:610-6.
18. Leacock SW, Basse AN, Chandler GL, Kirk AM, Rakheja D, Amatruda JF. A zebrafish transgenic model of Ewing’s
sarcoma reveals conserved mediators of EWS-FLI1 tumorigenesis. Dis Model Mech;5:95-106.
19. Lebensburger JD, Miller ST, Howard TH, Casella JF, Brown RC, Lu M, Iyer RV, Sarnaik S, Rogers ZR, Wang WC.
Influence of severity of anemia on clinical findings in infants with sickle cell anemia: Analyses from the BABY HUG
study. Pediatr Blood Cancer.
20. Lehmann HP, Dambita N, Buchanan GR, Casella JF. Decision modeling of disagreements: pediatric hematologists’
management of idiopathic thrombocytopenic purpura. Med Decis Making;31:805-15.
21. Lopez-Medina E, Neubauer MM, Pier GB, Koh AY. RNA isolation of Pseudomonas aeruginosa colonizing the murine
gastrointestinal tract. J Vis Exp.
22. Margraf LR, Gargan L, Butt Y, Raghunathan N, Bowers DC. Proliferative and metabolic markers in incompletely
excised pediatric pilocytic astrocytomas--an assessment of 3 new variables in predicting clinical outcome. Neuro
Oncol;13:767-74.
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23. McCarville MB, Luo Z, Huang X, Rees RC, Rogers ZR, et al. Abdominal ultrasound with scintigraphic and
clinical correlates in infants with sickle cell anemia: baseline data from the BABY HUG trial. AJR Am J
Roentgenol;196:1399-404.
24. McCarville MB, Rogers ZR, Sarnaik S, et al. Effects of chronic transfusions on abdominal sonographic abnormalities
in children with sickle cell anemia. J Pediatr;160:281-5 e1.
25. McCavit TL, Quinn CT, Techasaensiri C, Rogers ZR. Increase in invasive Streptococcus pneumoniae infections in
children with sickle cell disease since pneumococcal conjugate vaccine licensure. J Pediatr;158:505-7.
26. Miller ST, Rey K, He J, Flanagan J, Fish BJ, Rogers ZR, et al. Massive accidental overdose of hydroxyurea in a young
child with sickle cell anemia. Pediatr Blood Cancer.
27. Neumann JC, Chandler GL, Damoulis VA, Fustino NJ, Lillard K, Looijenga L, Margraf L, Rakheja D, Amatruda JF.
Mutation in the type IB bone morphogenetic protein receptor Alk6b impairs germ-cell differentiation and causes
germ-cell tumors in zebrafish. Proc Natl Acad Sci U S A;108:13153-8.
28. Neumann JC, Lillard K, Damoulis V, Amatruda JF. Zebrafish models of germ cell tumor. Methods Cell Biol;105:3-24.
29. Paulson V, Chandler G, Rakheja D, Galindo RL, Wilson K, Amatruda JF, Cameron S. High-resolution array CGH
identifies common mechanisms that drive embryonal rhabdomyosarcoma pathogenesis. Genes Chromosomes
Cancer;50:397-408.
30. Quinn CT, Stuart MJ, Kesler K, Ataga KI, Wang WC, Styles L, Smith-Whitley K, Wun T, Raj A, Hsu LL, Krishnan S,
Kuypers FA, Setty Y, Rhee S, Key NS, Buchanan GR, and on Behalf of the Investigators of the Comprehensive Sickle
Cell Centers. Tapered oral dexamethasone for the acute chest syndrome of sickle cell disease. Br J Haematol;155:263-7.
31. Rodeberg DA, Garcia-Henriquez N, Lyden ER, Davicioni E,, Parham DM, Skapek SX, et al. Prognostic significance
and tumor biology of regional lymph node disease in patients with rhabdomyosarcoma: a report from the Children’s
Oncology Group. J Clin Oncol;29:1304-11.
32. Rogers ZR, Wang WC, Luo Z, et al. Biomarkers of splenic function in infants with sickle cell anemia: baseline data
from the BABY HUG Trial. Blood;117:2614-7.
33. Saab R, Spunt SL, Skapek SX. Myogenesis and rhabdomyosarcoma the Jekyll and Hyde of skeletal muscle. Curr Top
Dev Biol;94:197-234.
34. Sarode R, Matevosyan K, Rogers ZR, Burner JD, Rutherford C. Advantages of isovolemic hemodilution-red cell
exchange therapy to prevent recurrent stroke in sickle cell anemia patients. J Clin Apher;26:200-7.
35. Spunt SL, Vargas SO, Coffin CM, Skapek SX, et al. The clinical, research, and social value of autopsy after any cancer death: A
perspective from the Children’s Oncology Group Soft Tissue Sarcoma Committee. Cancer 2011 Oct 17
36. Verduzco D, Amatruda JF. Analysis of cell proliferation, senescence, and cell death in zebrafish embryos. Methods
Cell Biol;101:19-38.
37. Wang W, Brugnara C, Snyder C, Rogers Z, et al. The effects of hydroxycarbamide and magnesium on haemoglobin SC
disease: results of the multi-centre CHAMPS trial. Br J Haematol;152:771-6.
38. Wang WC, Ware RE, Miller ST, Iyer RV, Casella JF, Minniti CP, Rana S, Thornburg CD, Rogers ZR, et al.
Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY
HUG). Lancet;377:1663-72.
39. Ware RE, Schultz WH, Yovetich N, Mortier NA, Alvarez O, Hilliard L, Iyer RV, Miller ST, Rogers ZR, et al. Stroke With
Transfusions Changing to Hydroxyurea (SWiTCH): a phase III randomized clinical trial for treatment of children with
sickle cell anemia, stroke, and iron overload. Pediatr Blood Cancer;57:1011-7.
40. Watt TC, Cooper T. Sorafenib as treatment for relapsed or refractory pediatric acute myelogenous leukemia. Pediatr
Blood Cancer.
41. Zaremba CM, Oliver D, Cavalier M, Fuda F, Karandikar NJ, Chen W. Distinct immunophenotype of early T-cell
progenitors in T lymphoblastic leukemia/lymphoma may predict FMS-like tyrosine kinase 3 mutations. Ann Diagn
Pathol;16:16-20.
Book Chapters
1. Koh AY, Pizzo PA. Infectious Complications in Pediatric Cancer Patients. In: Pizzo PA, Poplack DG, eds. Principles
and Practice of Pediatric Oncology. 6th ed. Philadelphia: Lippincott Williams & Wilkins; 2011.
2. Rogers ZR. Chapter 504: Anemia Due to Diminished Red Blood Cell Production. In: Rudolph, Lister, Gershon, First,
Rudolph, eds. Rudolph’s Pediatrics. 22nd ed. New York: McGraw-Hill; 2011.
Grants
NIH and Other Government Grants & Contracts
James F. Amatruda, M.D., Ph.D.
Generation and Rapid Mapping of Low-penetrance Disease Alleles in Zebrafish
NIH, 5 R01 CA135731-04
09/01/08 - 07/31/13
Genetic Approach to Target EWS-FLI1 Oncoprotrein in Ewing’s Sarcoma
Cancer Research and Prevention Institute of Texas (CPRIT), RP110395-02
12/01/10 - 11/30/12
Identification of Novel Targets for Therapy of Pediatric Germ Cell Tumors
Cancer Research and Prevention Institute of Texas (CPRIT), RP110394-02
12/01/10 - 11/30/13
George R. Buchanan, M.D.
Health Care and Other Facilities
HRSA, 1-C76-HF10995-01-00
09/01/08 – 08/31/11
Pediatric Hematology and Oncology Research Training
NIH, 5T32CA00964019
07/01/08 - 06/30/11
UT Southwestern Comprehensive Sickle Cell Centers
NIH, 5 U54 HL070588-08
06/14/08 – 02/29/12
Janna Journeycake, M.D., M.S.
Identification and Treatment of Clinically Silent Catheter-related Deep Vein Thrombosis in Children with Cancer
NIH, 5 K23 HL084097-05
05/01/06 - 04/30/12
Jeff Magee, MD, PhD
Antecedents and Sequelae of Childhood Cancer
NIH K12 Child Career Development Award
Zora R. Rogers, M.D.
Pediatric Hydroxyurea Phase III Clinical Trial (Baby Hug)
NIH, N01- HB-07159-12
09/01/00 - 06/30/12
BABY HUG – Follow up Study II
NIH/NHLBI and NICHD, HHSN268201200019C
01/12/2012 - 12/31/17
Stephen X. Skapek, M.D.
ARF Controls Vascular Regression DuringEye Development
NIH/National Eye Institute(NEI), 7 R01 EY014368-11
12/01/08 – 7/31/13
Tgfβ2Controls p19Arf During Eye Development
NIH/National Eye Institute(NEI), 7 R01 EY019942-03
12/01/09 - 11/30/12
Translation of Predictive Cancer Biomarkers into Clinical Practice
NIH/NCI, 1 RC2 CA148216
09/29/2009 – 08/31/2011
10 11

NIH and Other Government Subcontracts
Victor Aquino, M.D.
Pediatric Blood and Marrow Transplant Consortium
National Childhood Cancer Fund, U01 HL069254;#1021
09/01/06 - 12/31/11
Daniel Bowers, M.D.
Childhood Cancer Survivor Study
NIH/NCI: U24 CA055727-15
11/30/09 - present
Zora Rogers, M.D.
TWiTCH: TCD with Transfusion Changing to Hydroxyurea
NHLBI/Baylor College of Medicine, R01 HL095647
8/21/10 – present
SWiTCH: Stroke with Transfusion in Transition to Hydroxyurea)
NHLBI/Baylor College of Medicine, R01 HL 078787
08/01/10-07/31/11
Thalassemia Longitudinal Cohort – Planning Grant for comparative effectiveness research application
NHLBI/Harvard
09/01/11 – 08/31/13
Pharmacokinetics and Relative Bioavailability of a Liquid Formulation of Hydroxyurea in Pediatric Patients with Sickle
Cell Anemia
NICHD/ Duke Pediatric Trial Network
4/1/2011-9/30/2012
Promoting Education and Outreach of Persons w/Hereditary Blood Disorders: Identifying Patients w/ Diamond
Blackfan Anemia
DHHS Centers for Disease Control, U58 DD 000564
Naomi J. Winick, M.D.
Children’s Oncology Group/Agreement #97452-1021
National Childhood Cancer Foundation, U01CA9745207
08/01/08 - 07/31/11
Children’s Oncology Group/Agreement #1233
National Childhood Cancer Foundation, 5U10CA9854303
03/01/05 - Open
Multi-center consortium conducting therapeutic research in childhood cancer.
NCI/Children’s Oncology Group Chair’s Grant
03/01/03 – 02/28/13
MTX Study
NCI /Children’s Oncology Group Chair’s Grant Supplement
03/01/06 – 02/28/13
Non-governmental Organization Grants and Contracts
James F. Amatruda, M.D., Ph.D.
Fellowship for Stephanie Leacock
American Cancer Society, PF-08
09/01/09 – 08/31/11
Chemical Genetic Approaches to the Biology and Treatment of Childhood Cancers
Robert A. Welch Foundation, 1679
06/01/08 - 05/31/11
Hyundai Hope on Wheels Award
Hyundai Foundation
10/01/11-09/30/13
Daniel Bowers, M.D.
Phase 2 Study of Valproic Acid & XRT Followed by Post-XRT Maintenance Therapy
Baylor College of Medicine/TOPNOC Addendum #1
06/01/10 - 05/31/13
Phase II Gleevec/Imatinib Mesylate (Sti-571, NSC 716051)
Neurofibromatosis (NF1) Subjects with Plexiform Neurofibromas
Indiana University, CSTI571BUS248
12/20/06 – Present
A Pharmacokinetic and Phase II Study of Oral Cyclophosphamide and Oral Topotecan for Children with Recurrent
Solid Tumors
Children’s Clinical Research Advisory Committee (CCRAC), 1862168
10/18/02 – Present
Metabolic Syndrome in Childhood Brain Tumor Survivors
Wipe Out Kids Cancer
01/01/12 – present
George R. Buchanan, M.D.
North American Chronic ITP Registry
Children’s Hospital Boston
12/03/06 – Open
The Children’s Cancer Fund District Chair/Pediatric Oncology-Hematology
CCF/Anonymous, GL#623180
03/19/92 – Open
Andrew Koh, M.D.
The Role of Commensal Microbial Flora in Acute Intestinal Graft Versus Host Disease
American Cancer Society Institutional Research Grant, ACS-IRG-02-196
01/01/11 - 12/31/11.
Stephen X. Skapek, M.D.
Histologic Criteria and Surrogate Markers Alveolar Rhabdomyosarcoma
St. Baldrick’s Foundation
07/01/10 - 06/30/11
Molecular Targeting in Non-rhabdomyosarcoma Soft Tissue Sarcoma
St. Baldrick’s Foundation
07/01/11 – 06/30/14
Sponsored Clinical Trials
Victor M. Aquino, M.D.
Pediatric Blood and Marrow Consortium
Children’s Mercy Hospital, 1U01HL6925401
04/01/02 – Present
Intravenous Temsirolimus (Cc10779) in Pediatrics Subjects
Wyeth Pharmaceuticals, 3066K1139US
10/23/07 – Present
Kevin Weir Ewing Sarcoma Fund
Cytokine Gene Polymorphisms in Pediatric Patients Receiving Tacro
Catholic Foundation/G. Platt/D. Weir
North Shore University Hospital
11/20/07 - Open
10/01/02 – Present
12 13

Myeloablative Therapy and Allogenic Hematopoietic Stem Cell Transplantation
Amgen Inc., 20010133
08/01/06 – Present
Toward a Less Toxic Yet Highly Effective Conditioning Regimen in Allogeneic Hematopoietic Stem Cell Transplantation
for Children and Adolescents with Severe Sickle Cell Disease: a Pilot Study (The SCD Conditioning Trial)
Children’s Healthcare of Atlanta/AFLAC Cancer Center and Blood Disorders Service.
A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorder Pediatric
Blood Marrow Transplant Consortium, RDCRN PIDTC 6901.
A Retrospective and Cross-Sectional Analysis of Patients Treated for SCID (1968-2010) Pediatric Blood Marrow
Transplant Consortium, DAIT NIAID PIDTC 6902.
A Randomized, Placebo-controlled, Multi-site Phase 2 Study Evaluating the Safety and Efficacy of Preemptive
Treatment with CMX001 for the Prevention of Adenovirus Disease Following Hematopoietic Stem Cell Transplantation
(The ADV HALT Trial) CTI/Chimerics Inc., CMX001-202.
Daniel Bowers, M.D.
Transfusional Iron Overload in Children Treated for Cancer
Novartis Pharmaceuticals, CICL670AUS41T
08/31/10 – 09/01/12
A Randomized Phase 2 Study of Single-agent Erlotinib vs. Oral Etoposide in Patients w/Recurrent or Refractory
Pediatric Ependymoma (PETEY)
OSI Pharmaceuticals Inc. OSI-774-205
05/01/10 – 04/30/12
George R. Buchanan, M.D.
Transfusion Medicine/Hemostasis (TMH) Clinical Trials Network
New England Research Institute
09/01/07 – Present
A Prospective Multicenter, Open-label, Phase 3B Study of Human Plasma-Derived Factor XIII Concentrate in Subjects
w/Congenital Factor XIII Deficiency
CSL Behring Inc. B171023-3001
01/01/10 – 02/28/11
Andrew Koh, M.D.
The Use of Anti-Candida Monoclonal Antibody Therapy for Candida albicans Gastrointestinal Colonization and
Dissemination
Regeneron Pharmaceuticals, Inc.
10/28/11-10/28/13
Patrick Leavey, M.D.
A Phase III Randomized Trial of Adding Vincristine-topotecan-cyclophosphamide to Standard
Chemotherapy in Initial Treatment of Non-metastatic Ewing Sarcoma
NCI/Children’s Oncology Group, U10 CA98543-09
2011 - 2012
Phase I/II Study of Cp-751,871 in Patients With Relapsed and/or Refractory Ewing’s Sarcoma Family of Tumors
Pfizer Inc./Research, A4021020
03/17/08 - Present
Zora Rogers, M.D.
Pediatric Hydroxyurea Phase III Clinical Trial – Clinical Center (BABY HUG)
National Heart, Lung and Blood Institute
NO1-HB-07159
Pediatrics Subjects with Chronic Immune (Idiopathic) Thrombocytope
Amgen, Inc., 20060195
09/26/07 – 08/31/12
An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of Romiplostin in Thrombocytopenic
Pediatric Subjects with Immune (Idiopathic) Thrombocytopenic Purpura (ITP
Amgen, Inc., 20090340
A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Patients with Sickle Cell Disease
HemaQuest Pharmaceuticals, HQP 1001-SCD-006
Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients
(MBCTPs) when Exposed to Plasma-Derived vonWillebrand Factor-Containing Factor VIII (VWF/FVIII)
Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre,
Prospective, Controlled, Randomized, Open Label, Clinical Trial (SIPPET)
Fondazion Angelo Bianchi Bonomi (Milan, Italy), ABB-09-001
Paul Harker-Murray, M.D. Ph.D.
An Open Label Study of Bendamustine Hydrochloride for the Treatment of Pediatric Patients w/Relapsed or Refractory
Acute Leukemia
Cephalon Inc. C18083/2046
08/01/10 – 07/30/12
Janna Journeycake, M.D., M.S.C.S.
Evaluation of Prophylaxis Treatment and Characterization of B-domain
Wyeth Pharmaceuticals
02/17/09 – Present
14 15