Department of Pediatrics Annual Report – 2020

Hematology, Oncology, and Stem Cell Transplantation

The region’s only center to offer early-phase trials for children with cancer

The Division of Hematology, Oncology, and Stem Cell Transplantation’s more than 30 faculty members and 75 staff provide compassionate state-of-the-art care, perform cutting-edge, extramurally supported research, and train the next generation of leaders in our field.


Clinical Services

We are home to the largest and most comprehensive pediatric oncology program in the tristate area, and one of the largest centers in the nation for stem cell and bone marrow transplantation. Our clinical programs include:

  • Pediatric Cancer Foundation Developmental Therapeutics Program: The only National Cancer Institute-sponsored Children’s Oncology Group Phase 1 Consortium site in the New York-New Jersey-Connecticut area, and one of the founding members of the Therapeutic Approaches to Childhood Leukemia (TACL) consortium
  • Pediatric Liver Cancer Program: With four board-certified pediatric gastrointestinal transplant specialists, we have performed more than twice as many pediatric liver transplants as any other program in the region (see the Scientific Registry of Transplant Recipients)
  • Precision in Pediatric Sequencing (PIPseq) Program: Our PIPseq program is one of a few in the U.S. and the only one in New York to prospectively sequence cancers, create personalized avatars, and use the results to make clinical decisions
  • Sickle Cell Disease
  • Stem Cell Transplant Program
  • Center for Comprehensive Wellness (CCW): The CCW assimilates integrative treatments into each patient’s care and conducts research on the patterns of use and overall effectiveness of complementary and alternative medicine and nutrition throughout the world
  • Center for Survivor Wellness: We provide continuity of care to address the ongoing impact of cancer treatment on the family, relationships, body image, school, and employment

Research

With NIH funding, faculty members in the division conduct basic research on the causes of leukemia and brain tumors and have identified new genetic changes resulting in cancer formation. We are also investigating the mechanisms that enable leukemia cells to evade the immune system in the bone marrow.

Members of our translational research division have advanced several new therapeutic approaches for pediatric leukemia, brain tumors, sarcomas, and neuroblastoma. We also have active studies aimed at improving the treatment of childhood blood disorders, including bleeding disorders, iron deficiency anemia, and sickle cell disease.

We maintain a large number of clinical trials that provide bone marrow transplantation for children with cancer and blood disorders. We are studying approaches that may help improve control of symptoms in children with sickle cell disease as well as definitive methods to cure the disease. Our clinical research program brings the latest therapies to every child with cancer we treat, and our precision medicine program allows us to tailor therapies based on state-of-the-art technologies.


Education

We provide clinical training and research opportunities to medical students and residents. Our three-year Oncology, Hematology, and Stem Cell Transplant Fellowship Program has both a clinical and research focus.


New Appointments


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Honors and Awards

Stergios Zacharoulis, MD

  • Herbert & Florence Irving Professorship in Pediatric Neuro-Oncology

Nobuko Hijiya, MD

  • Herbert & Florence Irving Professorship in Pediatric Developmental Therapeutics

Shan Zha, MD, PhD

  • James A. Wolff Associate Professorship of Pediatrics

Monica Bhatia, MD

  • Chair, Sickle Cell Transplant Advocacy and Research Alliance

Gary Brittenham, MD

  • Visiting Professor, Laboratory of Human Nutrition, Swiss Federal Institute of Technology (ETH Zürich), 2018–2019

Nancy Green, MD

  • Research award for being among the top NIH-funded faculty, Dept. of Pediatrics, 2018, 2019

Lenat Joffe, MD

  • Fellow of the NIH National Institute of Child Health and Human Development (NICHD)

Justine Kahn, MD, MS

  • NCORP Pediatric Oncology Representative at Herbert Irving Comprehensive Cancer Center, 2019
  • Columbia University Irving Medical Center Minority/Underserved National Cancer Institute (NCI) Community Oncology Research Program (NCORP)
  • Young Investigator Award Best Research Abstract, International Symposium on Hodgkin Lymphoma (ISHL-11), Cologne, Germany, 2018

Margaret Lee, MD

  • Distinguished Service Award, 26th Annual Lifetime Achievement Gala Sickle Cell Thalassemia Patient Network, 2019

Cindy Neunert, MD

  • Appointed Member of the American Society of Hematology Committee on Quality
  • Chair of the American Society of Hematology Guideline committee for Immune Thrombocytopenia

Jovana Pavisic, MD

  • CUIMC: Oberfield Prize in Translational Science, 2019

Dara Steinberg, PhD

  • Elected Co-Chair, Research, Intervention & Outcomes, Consultation Liaison Special Interest Group, Society of Pediatric Psychology (APA Division 54)

Katherine Ender, MD

  • Nominated to the VP&S Academy of Clinical Excellence

Major Grants

  • Probiotics for prevention of acute graft-vs-host disease in children with cancer. NIH/National Cancer Institute; R01CA201788-05. Co-Principal Investigator: Monica Bhatia, MD.
  • Daily vitamin D for sickle-cell respiratory complications. US Food & Drug Administration; R01FD006372-01-04. Co-Principal Investigator: Gary Brittenham (M. Lee, MD, co-PI).
  • QSM to guide iron chelating therapy in transfusional iron overload. NIH/ National Institute of Diabetes and Digestive and Kidney Diseases; R01DK116126-02. Co-Principal Investigator: Gary Brittenham (co-PIs: Y. Wang, S. Sheth).
  • Prebiotic GOS and lactoferrin for beneficial gut microbiota with iron supplements. NIH/National Institute of Diabetes and Digestive and Kidney Diseases; R01DK115449-03. Principal Investigator: Gary Brittenham.
  • Neurocognitive effects of iron deficiency in blood donors. NIH/National Heart, Lung, and Blood Institute; R01HL139489-03. Co-investigator: Gary Brittenham (co-PI: E. Hod).
  • Red blood cells from iron-deficient donors: Recovery and storage quality. NIH/National Heart, Lung, and Blood Institute; R01HL133049-05. Co-investigator: Gary Brittenham (co-PIs: S. Spitalnik, E. Hod).
  • Burden and risk of neurological and cognitive impairment in pediatric sickle cell anemia in Uganda (BRAIN SAFE II). NIH/Eunice Kennedy Shriver National Institute of Child Health & Human Development; R21HD089791-02S1. Co-Principal Investigators: Richard Idro, Nancy Green.
  • Hydroxyurea adherence for “Personal Best” in sickle cell treatment: “HABIT.” NIH/National Institute of Nursing Research; R01NR017206-04. Multiple Principal Investigators: Nancy Green, Arlene Smaldone.
  • Adolescent/young adult lymphoma correlative studies grant. Lymphoma Research Foundation. Investigator: Justine Kahn.
  • Daily vitamin D for sickle-cell respiratory complications. U.S. FDA Orphan Products Development Grant Program; R01FD006372-01. Co-Principal Investigators: Gary Brittenham and Margaret Lee.
  • DISPLACE (Dissemination and Implementation of Stroke Prevention Looking at the Care Environment); National multicenter clinical trial. NIH/National Heart, Lung, and Blood Institute (NHLBI) NIH Subaward (UAB); R01HL133896-04. Co-Investigator: Margaret Lee.
  • Assessing coverage and reimbursement for whole-exome sequencing in pediatric oncology practice (pilot grant). National Human Genome Research Institute (NHGRI). 2RM1HG007257-07 (Appelbaum, PI). Columbia Center for Research on Ethical, Legal & Social Implications of Psychiatric, Neurologic & Behavioral Genetics. Co-Principal Investigator: Jennifer Oberg.
  • The COMIDITA Study: Urban Latino toddlers, diet intake and developmental outcomes. NIH/National Institute on Minority Health and Health Disparities; R21MD013622. Principal Investigator: Manuela Orjuela.
  • Targeting master regulator dependencies in pediatric osteosarcoma. St. Baldrick’s Foundation Fellow Award. Recipient: Jovana Pavisic.
  • National Library of Medicine T15 Award for Training in Biomedical Informatics at Columbia University. Recipient: Jovana Pavisic.
  • Targeting master regulator dependencies in high risk osteosarcoma. Hyundai Hope on Wheels Quantum Collaboration. Principal Investigator: Darrell Yamashiro, Co-PI: Julia Glade Bender.
  • Columbia University Developmental Therapeutics Program: Striving for Excellence. Alex’s Lemonade Stand. Principal Investigator: Darrell Yamashiro.

Selected Publications

Boboila S, Lopez G, Yu J, Banerjee D, Kadenhe-Chiweshe A, Connolly EP, Kandel JJ, Rajbhandari P, Silva JM, Califano A, Yamashiro DJ. Transcription factor activating protein 4 is synthetically lethal and a master regulator of MYCN-amplified neuroblastoma. Oncogene. 2018; 37, 5451-5465.

Hijiya N, Suttorp M. How I treat chronic myeloid leukemia in children and adolescents, Blood. 2019; 133: 2374-2384.

Gartrell-Corrado RD, Chen AX, Rizk EM, Marks DK, Bogardus MH, Hart TD, Silverman AM, Bayan CY, Finkel GG, Barker LW, Komatsubara KM, Carvajal RD, Horst BA, Chang R, Monod A, Rabadan R, Saenger YM. Linking transcriptomic and imaging data defines features of a favorable tumor immune microenvironment and identifies a combination biomarker for primary melanoma. Cancer Res. 2020.

Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kuhne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society Of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019; 3, 3829-3866.

Green NS, Munube D, Bangirana P, Buluma LR, Kebirungi B, Opoka R, Mupere E, Kasirye P, Kiguli S, Birabwa A, Kawooya MS, Lubowa SK, Sekibira R, Kayongo E, Hume H, Elkind M, Peng W, Li G, Rosano C, LaRussa P, Minja FJ, Boehme A, Idro R. Burden of neurological and neurocognitive impairment in pediatric sickle cell anemia in Uganda (BRAIN SAFE): A cross-sectional study. BMC Pediatr. 2019; 19, 381.

Bourgeois W, Ricci A, Jin Z, Hall M, George D, Bhatia M, Garvin J, Satwani P. Health care utilization and cost among pediatric patients receiving unrelated donor allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 2019; 54, 691-699.

D'Angelo F, Ceccarelli M, Tala, Garofano L, Zhang J, Frattini V, Caruso FP, Lewis G, Alfaro KD, Bauchet L, Berzero G, Cachia D, Cangiano M, Capelle L, de Groot J, DiMeco F, Ducray F, Farah W, Finocchiaro G, Goutagny S, Kamiya-Matsuoka C, Lavarino C, Loiseau H, Lorgis V, Marras CE, McCutcheon I, Nam DH, Ronchi S, Saletti V, Seizeur R, Slopis J, Sunol M, Vandenbos F, Varlet P, Vidaud D, Watts C, Tabar V, Reuss DE, Kim SK, Meyronet D, Mokhtari K, Salvador H, Bhat KP, Eoli M, Sanson M, Lasorella A, Iavarone A. The molecular landscape of glioma in patients with neurofibromatosis 1. Nat Med. 2019; 25, 176-187.


Highlights


Florence and Herbert Irving Clinical Research Career Award: Finding New Uses for Existing Therapies

Novel therapies are often considered the most innovative tools to improve care for cancers with poor prognoses. But a team of Columbia researchers is using a different tactic: they are exploring ways to repurpose drugs approved by the FDA for other conditions and existing experimental therapies in these difficult-to-treat cancers.

Department Newsroom


Clinical Trial Begins for Promising Sickle Cell Gene Editing Treatment

Promising new research is now bringing hope for patients with SCD. Monica Bhatia, MD, director of Columbia’s Pediatric Stem Cell Transplant Program, is participating in an international clinical trial evaluating the use of gene editing as a curative treatment for patients with sickle cell disease and the related condition, thalassemia.

Department Newsroom


A New Paradigm for Brain Tumors

The brain is shielded from most toxins and pathogens by a highly complex network of blood vessels—the blood-brain barrier—that restricts the size and chemical composition of molecules that can cross vessel walls into brain tissue. This barrier is also one of the major challenges neuro-oncologists face in treating brain tumors: most chemotherapeutic agents are not able to pass through the blood-brain barrier to reach brain and spinal cord tumors, or do so only in very low and ineffective amounts.

Department Newsroom


Adolfo Ferrando Awarded the William Dameshek Prize for Outstanding Hematologic Research

Adolfo Ferrando, MD, PhD, a member of the Institute for Cancer Genetics (ICG) and the Herbert Irving Comprehensive Cancer Center (HICCC), has been awarded the William Dameshek Prize for his contributions in advancing acute lymphoblastic leukemia (ALL) and T-cell acute lymphoblastic leukemia (T-ALL) research.

HICCC News